Elliott Vichinsky, MD

Professor In Residence
Pediatrics

SUMMARY OF INTERESTS:
Protecting and advancing the health and well-being of children through clinical care, teaching, and research; hematology; hemoglobin, hemoglobin disorders; designing and leading clinical research studies; understanding the pathophysiology of hemoglobinopathies; comprehensive clinical care; evaluating therapeutic interventions to improve disease outcomes; expanding patient access to quality healthcare; improving patient quality of life; health equity; establishing, improving, and expanding networks of care for hemoglobinopathy patients; designing and leading collaborative multi-center trials; forming and leading regional, national, and international collaborative research networks; implementation studies; data collection; registry and surveillance systems for hemoglobinopathies; expanding enrollment in clinical trials; accelerating the pace of clinical research; writing, editing, and reviewing grant applications, abstracts, peer-reviewed manuscripts, chapters, and books; publishing research findings; presenting research findings to diverse audiences; developing, updating, publishing, and teaching standards of care guidelines for optimal management of hemoglobinopathy patients; training and expanding the healthcare workforce knowledgeable in the standard of care treatments; developing and teaching academic curricula; junior and mid-career faculty career development; recruiting and mentoring under-represented faculty in medicine and science; diversity in medicine and research; mentoring others to become leading independent clinician scientists equipped to advance the field; sickle cell disease; sickle cell anemia; sickle cell trait; pain management; acute chest syndrome; avascular necrosis; bone disease; organ failure; priapism; pulmonary hypertension; hemolysis; alloimmunization; iron dysregulation; brain iron; chelation therapy, neurodegeneration; NBIA; PKAN; thalassemia syndromes; alpha thalassemia major; fetal hemoglobin; in utero transfusion, intrauterine stem cell transplantation; Bart's fetalis syndrome; stroke; transcranial doppler; TCD screening; prenatal and newborn screening; genetic counseling; BMT; gene and stem cell therapy; red blood cell biology; laboratory medicine; pheresis medicine; transfusion medicine; global medicine; political advocacy to improve healthcare policy; collaborating with community advisory boards and involving them as part of the research team to ensure our research activities are guided by the needs and concerns of people affected.

I am an endowed physician and professor in residence at the University of California San Francisco Benioff Children's Hospital in Oakland, California. For nearly 40 years I have been on forefront of research into better treatments and potential cures for hemoglobinopathies. My directorship of the largest and most comprehensive program for individuals with sickle cell disease and their families in the western United States began in 1978. Renamed the UCSF Sickle Cell Center of Excellence and rebuilt stronger, my center continues its mission to improve clinical outcomes and quality of life for individuals living with sickle cell and their families across the lifespan.

I direct the Hemoglobinopathy Reference Laboratory for the State of California at UCSF Benioff Children's Hospital Oakland, which has been a national resource to aid in the diagnosis of clinically relevant hemoglobinopathies since 1973 (the laboratory has been a vital component of the state's newborn screening follow-up program, evaluating clinically relevant hemoglobin variants, providing confirmatory testing on all newborns in California with presumptive sickle cell disease, beta thalassemia or HbH disease, and assisting NBS programs across the country.

Throughout my career I have developed successful teams that bring basic and clinical investigators together in partnership with the biomedical and pharmaceutical industry to develop and evaluate new treatments for hemoglobinopathies. I was lead investigator of the safety and efficacy of several important therapies for sickle cell disease, such as arginine, mitapivat, and other promising drug trials. I was the senior/principal author of the drug trials of voxelotor and glutamine and played a pivotal role securing their FDA-approval.

In addition to leading the development of novel therapeutics, I serve as PI or Co-PI on several key evidence-based clinical research networks, such as the American Society of Hematology’s Research Collaborative (as the Western States Sickle Cell Disease Clinical Trials Network); the NHLBI’s SC-Links study (Linking the Sickle Cell Community, Providers and Systems to Improve Sickle Cell Disease Care (SCDIC); the NHLBI’s REDS-IV-P study (Recipient Epidemiology and Donor Evaluation Study-IV-Pediatric, Domestic Hubs); the Network for California Sickle Cell Care and HRSA’s Pacific Sickle Cell Regional Collaboration, which aims to reduce morbidity and mortality by improving the quality of coordinated and comprehensive services to individuals with sickle cell disease and their families. These networks link together researchers, clinicians, and communities affected by sickle cell disease to guide evidence-based treatment and improve patient care.

I am commitment to fostering the next generation of translational and clinical scientists and guide the academic development of my colleagues through participation in programs like the American Society of Pediatric Hematology Oncology’s Mid-Career Mentoring Program, the Doris Duke Charitable Foundation’s Clinical Research Mentorship Program, and the NIH’s Mentored Research Scientist Career Development Program (K series). I have supported young students from disadvantaged backgrounds explore potential career opportunities in healthcare and science through my involvement in the UCSF Summer Student Program.

I am a passionate advocate to improve healthcare policies for people living with sickle cell disease. Over the years I have provided scientific insight and medical expertise to legislators and health care policy makers at the regional, state, and federal levels of government, including my work with California Congresswoman Barbara Lee, State Superintendent of Public Instruction Tony Thurmond, California Assemblymember Mike Gibson, the California State Health Subcommittee, and multiple mayoral leaders of the City of Oakland, California to improve and increase access to comprehensive care for Californians living with sickle cell disease.

Publications: 

Physician perspectives about the diagnosis and management of acute chest syndrome.

Transfusion

Bhasin N, LeBlanc DM, Yates S, Eichbaum Q, Pham A, Sharma D, Zhang L, Vichinsky EP, Sarode R

COVID-19 mRNA Vaccination Responses in Individuals With Sickle Cell Disease: ASH RC Sickle Cell Research Network Study.

Blood advances

Anderson AR, Strouse JJ, Manwani D, Brandow AM, Vichinsky EP, Campbell A, Leavey PJ, Nero A, Ibrahim IF, Field JJ, Baer A, Soto-Calderon H, Vincent L, Zhao Y, Santos JJS, Hensley SE, Mortier N, Lanzkron S, Neuberg DS, Abrams CS

Disease burden, management strategies, and unmet needs in α-thalassemia due to hemoglobin H disease.

American journal of hematology

Lal A, Viprakasit V, Vichinsky E, Lai Y, Lu MY, Kattamis A

Neurocognitive outcome in children with sickle cell disease after myeloimmunoablative conditioning and haploidentical hematopoietic stem cell transplantation: a non-randomized clinical trial.

Frontiers in neurology

Braniecki S, Vichinsky E, Walters MC, Shenoy S, Shi Q, Moore TB, Talano JA, Parsons SK, Flower A, Panarella A, Fabricatore S, Morris E, Mahanti H, Milner J, McKinstry RC, Duncan CN, van de Ven C, Cairo MS

Sickle Cell Disease Treatment with Arginine Therapy (STArT): study protocol for a phase 3 randomized controlled trial.

Trials

Rees CA, Brousseau DC, Cohen DM, Villella A, Dampier C, Brown K, Campbell A, Chumpitazi CE, Airewele G, Chang T, Denton C, Ellison A, Thompson A, Ahmad F, Bakshi N, Coleman KD, Leibovich S, Leake D, Hatabah D, Wilkinson H, Robinson M, Casper TC, Vichinsky E, Morris CR, SCD Arginine Study Group and PECARN

Fertility and Pregnancy in Women with Transfusion-Dependent Thalassemia.

Hematology/oncology clinics of North America

Sayani FA, Singer ST, Carlberg KT, Vichinsky EP

The Clinical Phenotypes of Alpha Thalassemia.

Hematology/oncology clinics of North America

Lal A, Vichinsky E

Assessment of total and unbound cell-free heme in plasma of patients with sickle cell disease.

Experimental biology and medicine (Maywood, N.J.)

Vissa M, Larkin SK, Vichinsky EP, Kuypers FA, Soupene E

COVID-19 Infection and Outcomes in Newborn Screening Cohorts of Sickle Cell Trait and Sickle Cell Disease in Michigan and Georgia.

Journal of pediatric hematology/oncology

Paulukonis ST, Snyder A, Smeltzer MP, Sutaria AN, Hurden I, Latta K, Chennuri S, Vichinsky E, Reeves SL

The impact of in utero transfusions on perinatal outcomes in patients with alpha thalassemia major: the UCSF registry.

Blood advances

Schwab ME, Lianoglou BR, Gano D, Gonzalez Velez J, Allen IE, Arvon R, Baschat A, Bianchi DW, Bitanga M, Bourguignon A, Brown RN, Chen B, Chien M, Davis-Nelson S, de Laat MWM, Ekwattanakit S, Gollin Y, Hirata G, Jelin A, Jolley J, Meyer P, Miller J, Norton ME, Ogasawara KK, Panchalee T, Schindewolf E, Shaw SW, Stumbaugh T, Thompson AA, Towner D, Tsai PS, Viprakasit V, Volanakis E, Zhang L, Vichinsky E, MacKenzie TC

Development of a Thalassemia International Prognostic Scoring System (TIPSS).

Blood cells, molecules & diseases

Vitrano A, Musallam KM, Meloni A, Karimi M, Daar S, Ricchi P, Costantini S, Vlachaki E, Di Marco V, El-Beshlawy A, Hajipour M, Ansari SH, Filosa A, Ceci A, Singer ST, Naserullah ZA, Pepe A, Cademartiri F, Pollina SA, Scondotto S, Dardanoni G, Bonifazi F, Sankaran VG, Vichinsky E, Taher AT, Maggio A, International Working Group on Thalassemia (IWG-THAL)

Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion dependent a-thalassaemia or �-thalassaemia: an open-label, multicentre, phase 2 study.

Lancet (London, England)

Kuo KHM, Layton DM, Lal A, Al-Samkari H, Bhatia J, Kosinski PA, Tong B, Lynch M, Uhlig K, Vichinsky EP

The effects of glutamine supplementation on markers of apoptosis and autophagy in sickle cell disease peripheral blood mononuclear cells.

Complementary therapies in medicine

Walter PB, Hohman LS, Rokeby A, Lum JJ, Hagar R, Lavrisha L, Saulys A, Kuypers FA, Vichinsky E, Morris CR

Implications for the metabolic fate of oral glutamine supplementation within plasma and erythrocytes of patients with sickle cell disease: A pharmacokinetics study.

Complementary therapies in medicine

Morris CR, Kuypers FA, Hagar R, Larkin S, Lavrisha L, Saulys A, Vichinsky EP, Suh JH

Consensus statement for the perinatal management of patients with a thalassemia major.

Blood advances

MacKenzie TC, Amid A, Angastiniotis M, Butler C, Gilbert S, Gonzalez J, Keller RL, Kharbanda S, Kirby-Allen M, Koenig BA, Kyono W, Lal A, Lianoglou BR, Norton ME, Ogasawara KK, Panchalee T, Rosner M, Schwab M, Thompson A, Waye JS, Vichinsky E

Risk of mortality from anemia and iron overload in nontransfusion-dependent �-thalassemia.

American journal of hematology

Musallam KM, Vitrano A, Meloni A, Pollina SA, Karimi M, El-Beshlawy A, Hajipour M, Di Marco V, Ansari SH, Filosa A, Ricchi P, Ceci A, Daar S, Vlachaki E, Singer ST, Naserullah ZA, Pepe A, Scondotto S, Dardanoni G, Bonifazi F, Sankaran VG, Vichinsky E, Taher AT, Maggio A, International Working Group on Thalassemia (IWG-THAL)

An update on the US adult thalassaemia population: a report from the CDC thalassaemia treatment centres.

British journal of haematology

Chapin J, Cohen AR, Neufeld EJ, Vichinsky E, Giardina PJ, Boudreaux J, Le BC, Kenney K, Trimble S, Thompson AA

Anterior Pituitary Volume in Patients with Transfusion Dependent Anemias: Volumetric Approaches and Relation to Pituitary MRI-R2.

Clinical neuroradiology

Berliner C, Wang ZJ, Singer ST, Grosse R, McDonough RV, Padua E, Yuan Q, Weyhmiller M, James E, Vichinsky E, Adam G, Yamamura J, Bannas P, Fischer R, Schoennagel BP

Primary HBB gene mutation severity and long-term outcomes in a global cohort of �-thalassaemia.

British journal of haematology

Musallam KM, Vitrano A, Meloni A, Addario Pollina S, Di Marco V, Hussain Ansari S, Filosa A, Ricchi P, Ceci A, Daar S, Vlachaki E, Singer ST, Naserullah ZA, Pepe A, Scondotto S, Dardanoni G, Karimi M, El-Beshlawy A, Hajipour M, Bonifazi F, Vichinsky E, Taher AT, Sankaran VG, Maggio A, International Working Group on Thalassemia (IWG-THAL)

Survival and causes of death in 2,033 patients with non-transfusion-dependent �-thalassemia

Haematologica

Musallam KM, Vitrano A, Meloni A, Pollina SA, Karimi M, El-Beshlawy A, Hajipour M, Di Marco V, Ansari SH, Filosa A, Ricchi P, Ceci A, Daar S, Vlachaki E, Singer ST, Naserullah ZA, Pepe A, Scondotto S, Dardanoni G, Bonifazi F, Sankaran VG, Vichinsky E, Taher AT, Maggio A

The transfusion management of beta thalassemia in the United States.

Transfusion

Lal A, Wong T, Keel S, Pagano M, Chung J, Kamdar A, Rao L, Ikeda A, Puthenveetil G, Shah S, Yu J, Vichinsky E

Time to rethink haemoglobin threshold guidelines in sickle cell disease.

British journal of haematology

Ballas SK, Kuypers FA, Gordeuk VR, Hankins JS, Thompson AA, Vichinsky E

Pituitary iron and factors predictive of fertility status in transfusion dependent thalassemia.

Haematologica

Singer ST, Fischer R, Allen I, Lal A, Vichinsky E, Yuan Q, Wang ZJ

Stable to improved cardiac and pulmonary function in children with high-risk sickle cell disease following haploidentical stem cell transplantation.

Bone marrow transplantation

Friedman D, Dozor AJ, Milner J, D'Souza M, Talano JA, Moore TB, Shenoy S, Shi Q, Walters MC, Vichinsky E, Parsons SK, Braniecki S, Moorthy CR, Ayello J, Flower A, Morris E, Mahanti H, Fabricatore S, Klejmont L, van de Ven C, Baxter-Lowe LA, Cairo MS

Voxelotor in adolescents and adults with sickle cell disease (HOPE): long-term follow-up results of an international, randomised, double-blind, placebo-controlled, phase 3 trial.

The Lancet. Haematology

Howard J, Ataga KI, Brown RC, Achebe M, Nduba V, El-Beshlawy A, Hassab H, Agodoa I, Tonda M, Gray S, Lehrer-Graiwer J, Vichinsky E

Voxelotor for the treatment of sickle cell disease.

Expert review of hematology

Vissa M, Vichinsky E

Iron Deficiency: Implications Before Anemia.

Pediatrics in review

Tong S, Vichinsky E

A complication risk score to evaluate clinical severity of thalassaemia syndromes.

British journal of haematology

Vitrano A, Meloni A, Addario Pollina W, Karimi M, El-Beshlawy A, Hajipour M, Di Marco V, Hussain Ansari S, Filosa A, Ricchi P, Ceci A, Daar S, Titi Singer S, Naserullah ZA, Pepe A, Scondotto S, Dardanoni G, Bonifazi F, Vichinsky E, Maggio A

Effect of Inhaled Cannabis for Pain in Adults With Sickle Cell Disease: A Randomized Clinical Trial.

JAMA network open

Abrams DI, Couey P, Dixit N, Sagi V, Hagar W, Vichinsky E, Kelly ME, Connett JE, Gupta K

American Society of Hematology 2020 guidelines for sickle cell disease: prevention, diagnosis, and treatment of cerebrovascular disease in children and adults.

Blood advances

DeBaun MR, Jordan LC, King AA, Schatz J, Vichinsky E, Fox CK, McKinstry RC, Telfer P, Kraut MA, Daraz L, Kirkham FJ, Murad MH

Influence of sickle cell disease on susceptibility to HIV infection.

PloS one

Kelly S, Jacobs ES, Stone M, Keating SM, Lee TH, Chafets D, Heitman J, Dimapasoc M, Operskalski E, Hagar W, Vichinsky E, Busch MP, Norris PJ, Custer B, Recipient Epidemiology and Donor Evaluation Study (REDS-III)

Identifying Clinical and Research Priorities in Sickle Cell Lung Disease. An Official American Thoracic Society Workshop Report.

Annals of the American Thoracic Society

Ruhl AP, Sadreameli SC, Allen JL, Bennett DP, Campbell AD, Coates TD, Diallo DA, Field JJ, Fiorino EK, Gladwin MT, Glassberg JA, Gordeuk VR, Graham LM, Greenough A, Howard J, Kato GJ, Knight-Madden J, Kopp BT, Koumbourlis AC, Lanzkron SM, Liem RI, Machado RF, Mehari A, Morris CR, Ogunlesi FO, Rosen CL, Smith-Whitley K, Tauber D, Terry N, Thein SL, Vichinsky E, Weir NA, Cohen RT, Klings ES

Safety and efficacy of deferiprone for pantothenate kinase-associated neurodegeneration: a randomised, double-blind, controlled trial and an open-label extension study.

The Lancet. Neurology

Klopstock T, Tricta F, Neumayr L, Karin I, Zorzi G, Fradette C, Kmiec T, B?chner B, Steele HE, Horvath R, Chinnery PF, Basu A, K?pper C, Neuhofer C, K?lm?n B, Du?ek P, Yapici Z, Wilson I, Zhao F, Zibordi F, Nardocci N, Aguilar C, Hayflick SJ, Spino M, Blamire AM, Hogarth P, Vichinsky E

A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease.

The New England journal of medicine

Vichinsky E, Hoppe CC, Ataga KI, Ware RE, Nduba V, El-Beshlawy A, Hassab H, Achebe MM, Alkindi S, Brown RC, Diuguid DL, Telfer P, Tsitsikas DA, Elghandour A, Gordeuk VR, Kanter J, Abboud MR, Lehrer-Graiwer J, Tonda M, Intondi A, Tong B, Howard J, HOPE Trial Investigators

Vincristine-induced anemia in hereditary spherocytosis.

Experimental biology and medicine (Maywood, N.J.)

Michlitsch J, Larkin S, Vichinsky E, Kuypers FA

Evaluation of Mandible Fractures in Patients With Sickle Cell Anemia-A Nationwide Study.

Journal of oral and maxillofacial surgery : official journal of the American Association of Oral and Maxillofacial Surgeons

Akbari M, Patel R, Carrao V, Hagar W, Vichinsky E, Chuang SK

Transfusion practices and complications in thalassemia.

Transfusion

Lal A, Wong TE, Andrews J, Balasa VV, Chung JH, Forester CM, Ikeda AK, Keel SB, Pagano MB, Puthenveetil G, Shah SJ, Yu JC, Vichinsky EP

A Phase 3 Trial of l-Glutamine in Sickle Cell Disease.

The New England journal of medicine

Niihara Y, Miller ST, Kanter J, Lanzkron S, Smith WR, Hsu LL, Gordeuk VR, Viswanathan K, Sarnaik S, Osunkwo I, Guillaume E, Sadanandan S, Sieger L, Lasky JL, Panosyan EH, Blake OA, New TN, Bellevue R, Tran LT, Razon RL, Stark CW, Neumayr LD, Vichinsky EP, Investigators of the Phase 3 Trial of l-Glutamine in Sickle Cell Disease

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia.

The New England journal of medicine

Thompson AA, Walters MC, Kwiatkowski J, Rasko JEJ, Ribeil JA, Hongeng S, Magrin E, Schiller GJ, Payen E, Semeraro M, Moshous D, Lefrere F, Puy H, Bourget P, Magnani A, Caccavelli L, Diana JS, Suarez F, Monpoux F, Brousse V, Poirot C, Brouzes C, Meritet JF, Pondarré C, Beuzard Y, Chrétien S, Lefebvre T, Teachey DT, Anurathapan U, Ho PJ, von Kalle C, Kletzel M, Vichinsky E, Soni S, Veres G, Negre O, Ross RW, Davidson D, Petrusich A, Sandler L, Asmal M, Hermine O, De Montalembert M, Hacein-Bey-Abina S, Blanche S, Leboulch P, Cavazzana M

Epidemiologic and clinical characteristics of nontransfusion-dependent thalassemia in the United States.

Pediatric blood & cancer

Vichinsky E, Cohen A, Thompson AA, Giardina PJ, Lal A, Paley C, Cheng WY, McCormick N, Sasane M, Qiu Y, Kwiatkowski JL

Fertility and Pregnancy in Women with Transfusion-Dependent Thalassemia.

Hematology/oncology clinics of North America

Carlberg KT, Singer ST, Vichinsky EP

Sickle cell disease.

Nature reviews. Disease primers

Kato GJ, Piel FB, Reid CD, Gaston MH, Ohene-Frempong K, Krishnamurti L, Smith WR, Panepinto JA, Weatherall DJ, Costa FF, Vichinsky EP

Dietary nonheme iron is equally bioavailable from ferritin or ferrous sulfate in thalassemia intermedia.

Pediatric hematology and oncology

Khurana M, Fung EB, Vichinsky EP, Theil EC

Chronic organ failure in adult sickle cell disease.

Hematology. American Society of Hematology. Education Program

Vichinsky E

Encephaloduroarteriosynangiosis (EDAS) in young patients with cerebrovascular complications of sickle cell disease: Single-institution experience.

Pediatric hematology and oncology

Winstead M, Sun PP, Martin K, Earl J, Neumayr L, Hoppe C, Vichinsky E

Lifespan care in SCD: Whom to transition, the patients or the health care system?

American journal of hematology

Minniti CP, Vichinsky E

Simvastatin reduces vaso-occlusive pain in sickle cell anaemia: a pilot efficacy trial.

British journal of haematology

Hoppe C, Jacob E, Styles L, Kuypers F, Larkin S, Vichinsky E

Long-term safety and efficacy of deferasirox in young pediatric patients with transfusional hemosiderosis: Results from a 5-year observational study (ENTRUST).

Pediatric blood & cancer

Vichinsky E, El-Beshlawy A, Al Zoebie A, Kamdem A, Koussa S, Chotsampancharoen T, Bruederle A, Gilotti G, Han J, Elalfy M

Emergency department utilization by Californians with sickle cell disease, 2005-2014.

Pediatric blood & cancer

Paulukonis ST, Feuchtbaum LB, Coates TD, Neumayr LD, Treadwell MJ, Vichinsky EP, Hulihan MM

Favorable outcomes after in utero transfusion in fetuses with alpha thalassemia major: a case series and review of the literature.

Prenatal diagnosis

Kreger EM, Singer ST, Witt RG, Sweeters N, Lianoglou B, Lal A, Mackenzie TC, Vichinsky E

Fertility in transfusion-dependent thalassemia men: effects of iron burden on the reproductive axis.

American journal of hematology

Singer ST, Killilea D, Suh JH, Wang ZJ, Yuan Q, Ivani K, Evans P, Vichinsky E, Fischer R, Smith JF

Dysregulated arginine metabolism and cardiopulmonary dysfunction in patients with thalassaemia.

British journal of haematology

Morris CR, Kim HY, Klings ES, Wood J, Porter JB, Trachtenberg F, Sweeters N, Olivieri NF, Kwiatkowski JL, Virzi L, Hassell K, Taher A, Neufeld EJ, Thompson AA, Larkin S, Suh JH, Vichinsky EP, Kuypers FA, Thalassemia Clinical Research Network

In utero hematopoietic cell transplantation for hemoglobinopathies.

Frontiers in pharmacology

Derderian SC, Jeanty C, Walters MC, Vichinsky E, MacKenzie TC

Mechanisms of plasma non-transferrin bound iron generation: insights from comparing transfused diamond blackfan anaemia with sickle cell and thalassaemia patients.

British journal of haematology

Porter JB, Walter PB, Neumayr LD, Evans P, Bansal S, Garbowski M, Weyhmiller MG, Harmatz PR, Wood JC, Miller JL, Byrnes C, Weiss G, Seifert M, Grosse R, Grabowski D, Schmidt A, Fischer R, Nielsen P, Niemeyer C, Vichinsky E

Emerging Therapies Targeting the Pathophysiology of Sickle Cell Disease

Hematology/oncology clinics of North America

Vichinsky, Elliott P., ed.

Relationship among chelator adherence, change in chelators, and quality of life in thalassemia.

Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation

Trachtenberg FL, Gerstenberger E, Xu Y, Mednick L, Sobota A, Ware H, Thompson AA, Neufeld EJ, Yamashita R, Thalassemia Clinical Research Network

Elevated tricuspid regurgitant jet velocity in subgroups of thalassemia patients: insight into pathophysiology and the effect of splenectomy.

Annals of hematology

Singer ST, Kuypers F, Fineman J, Gildengorin G, Larkin S, Sweeters N, Rosenfeld H, Kurio G, Higa A, Jeng M, Huang J, Vichinsky EP

Neuroimaging abnormalities in adults with sickle cell anemia: associations with cognition.

Neurology

Mackin RS, Insel P, Truran D, Vichinsky EP, Neumayr LD, Armstrong FD, Gold JI, Kesler K, Brewer J, Weiner MW, Neuropsychological Dysfunction and Neuroimaging Adult Sickle Cell Anemia Study Group

Validation and reliability of a disease-specific quality of life measure (the TranQol) in adults and children with thalassaemia major.

British journal of haematology

Klaassen RJ, Barrowman N, Merelles-Pulcini M, Vichinsky EP, Sweeters N, Kirby-Allen M, Neufeld EJ, Kwiatkowski JL, Wu J, Vickars L, Blanchette VS, Forgie M, Yamashita R, Wong-Rieger D, Young NL

Pain over time and its effects on life in thalassemia.

American journal of hematology

Oliveros O, Trachtenberg F, Haines D, Gerstenberger E, Martin M, Carson S, Green S, Calamaras D, Hess P, Yamashita R, Vichinsky E, Thalassemia Clinical Research Network

Renal medullary carcinoma in an adolescent with sickle cell anemia.

Pediatric blood & cancer

Marsh A, Golden C, Hoppe C, Quirolo K, Vichinsky E

Efficacy and safety of deferasirox compared with deferoxamine in sickle cell disease: two-year results including pharmacokinetics and concomitant hydroxyurea.

American journal of hematology

Vichinsky E, Torres M, Minniti CP, Barrette S, Habr D, Zhang Y, Files B, study CICL670A2201 investigators

Zinc supplementation improves bone density in patients with thalassemia: a double-blind, randomized, placebo-controlled trial.

The American journal of clinical nutrition

Fung EB, Kwiatkowski JL, Huang JN, Gildengorin G, King JC, Vichinsky EP

Treatment of classic pantothenate kinase-associated neurodegeneration with deferiprone and intrathecal baclofen.

American journal of physical medicine & rehabilitation

Pratini NR, Sweeters N, Vichinsky E, Neufeld JA

Transfusion complications in thalassemia patients: a report from the Centers for Disease Control and Prevention (CME).

Transfusion

Vichinsky E, Neumayr L, Trimble S, Giardina PJ, Cohen AR, Coates T, Boudreaux J, Neufeld EJ, Kenney K, Grant A, Thompson AA, CDC Thalassemia Investigators

Pregnancy outcomes in women with thalassemia in North America and the United Kingdom.

American journal of hematology

Thompson AA, Kim HY, Singer ST, Vichinsky E, Eile J, Yamashita R, Giardina PJ, Olivieri N, Parmar N, Trachtenberg F, Neufeld EJ, Kwiatkowski JL, Thalassemia Clinical Research Network

Non-transfusion-dependent thalassemias.

Haematologica

Musallam KM, Rivella S, Vichinsky E, Rachmilewitz EA

Treatment of heart failure in adults with thalassemia major: response in patients randomised to deferoxamine with or without deferiprone.

Journal of cardiovascular magnetic resonance : official journal of the Society for Cardiovascular Magnetic Resonance

Porter JB, Wood J, Olivieri N, Vichinsky EP, Taher A, Neufeld E, Giardina P, Thompson A, Moore B, Evans P, Kim HY, Macklin EA, Trachtenberg F

A randomized, placebo-controlled trial of arginine therapy for the treatment of children with sickle cell disease hospitalized with vaso-occlusive pain episodes.

Haematologica

Morris CR, Kuypers FA, Lavrisha L, Ansari M, Sweeters N, Stewart M, Gildengorin G, Neumayr L, Vichinsky EP

Clinical manifestations of a-thalassemia.

Cold Spring Harbor perspectives in medicine

Vichinsky EP

The palatability and tolerability of deferasirox taken with different beverages or foods.

Pediatric blood & cancer

Goldberg SL, Giardina PJ, Chirnomas D, Esposito J, Paley C, Vichinsky E

Sildenafil therapy in thalassemia patients with Doppler-defined risk of pulmonary hypertension.

Haematologica

Morris CR, Kim HY, Wood J, Porter JB, Klings ES, Trachtenberg FL, Sweeters N, Olivieri NF, Kwiatkowski JL, Virzi L, Singer ST, Taher A, Neufeld EJ, Thompson AA, Sachdev V, Larkin S, Suh JH, Kuypers FA, Vichinsky EP, Thalassemia Clinical Research Network

Human T cell lymphotropic virus type 1 infection among U.S. thalassemia patients.

AIDS research and human retroviruses

Switzer WM, Shankar A, Trimble SR, Thompson AA, Giardina PJ, Cohen AR, Coates TD, Vichinsky E, Neufeld EJ, Boudreaux JM, Heneine W

Pain in thalassaemia: the effects of age on pain frequency and severity.

British journal of haematology

Haines D, Martin M, Carson S, Oliveros O, Green S, Coates T, Eile J, Schilling L, Dinu B, Mendoza T, Gerstenberger E, Trachtenberg F, Vichinsky E, Thalassemia Clinical Research Network

Increased leucocyte apoptosis in transfused �-thalassaemia patients.

British journal of haematology

Walter PB, Porter J, Evans P, Kwiatkowski JL, Neufeld EJ, Coates T, Giardina PJ, Grady RW, Vichinsky E, Olivieri N, Trachtenberg F, Alberti D, Fung E, Ames B, Higa A, Harmatz P, Thalassemia Clinical Research Network

Combined chelation therapy with deferasirox and deferoxamine in thalassemia.

Blood cells, molecules & diseases

Lal A, Porter J, Sweeters N, Ng V, Evans P, Neumayr L, Kurio G, Harmatz P, Vichinsky E

A phase 1/2 trial of HQK-1001, an oral fetal globin inducer, in sickle cell disease.

American journal of hematology

Kutlar A, Ataga K, Reid M, Vichinsky EP, Neumayr L, Blair-Britt L, Labotka R, Glass J, Keefer JR, Wargin WA, Berenson R, Perrine SP

The effect of whole body vibration therapy on bone density in patients with thalassemia: a pilot study.

American journal of hematology

Fung EB, Gariepy CA, Sawyer AJ, Higa A, Vichinsky EP

Inadequate dietary intake in patients with thalassemia.

Journal of the Academy of Nutrition and Dietetics

Fung EB, Xu Y, Trachtenberg F, Odame I, Kwiatkowski JL, Neufeld EJ, Thompson AA, Boudreaux J, Quinn CT, Vichinsky EP, Thalassemia Clinical Research Network

A potent oral P-selectin blocking agent improves microcirculatory blood flow and a marker of endothelial cell injury in patients with sickle cell disease.

American journal of hematology

Kutlar A, Ataga KI, McMahon L, Howard J, Galacteros F, Hagar W, Vichinsky E, Cheung AT, Matsui N, Embury SH

Silent cerebral infarcts: a review on a prevalent and progressive cause of neurologic injury in sickle cell anemia.

Blood

DeBaun MR, Armstrong FD, McKinstry RC, Ware RE, Vichinsky E, Kirkham FJ

British Journal of Haematology

Safety of deferasirox in sickle cell disease patients with co-existing liver impairment – response to Sinakos et al

Vichinsky E.

Chelation use and iron burden in North American and British thalassemia patients: a report from the Thalassemia Longitudinal Cohort.

Blood

Kwiatkowski JL, Kim HY, Thompson AA, Quinn CT, Mueller BU, Odame I, Giardina PJ, Vichinsky EP, Boudreaux JM, Cohen AR, Porter JB, Coates T, Olivieri NF, Neufeld EJ, Thalassemia Clinical Research Network

Emerging 'A' therapies in hemoglobinopathies: agonists, antagonists, antioxidants, and arginine.

Hematology. American Society of Hematology. Education Program

Vichinsky E

Hb E/beta-thalassaemia: a common & clinically diverse disorder.

The Indian journal of medical research

Olivieri NF, Pakbaz Z, Vichinsky E

Risk factors and mortality associated with an elevated tricuspid regurgitant jet velocity measured by Doppler-echocardiography in thalassemia: a Thalassemia Clinical Research Network report.

Blood

Morris CR, Kim HY, Trachtenberg F, Wood J, Quinn CT, Sweeters N, Kwiatkowski JL, Thompson AA, Giardina PJ, Boudreaux J, Olivieri NF, Porter JB, Neufeld EJ, Vichinsky EP, Thalassemia Clinical Research Network

Reproductive capacity in iron overloaded women with thalassemia major.

Blood

Singer ST, Vichinsky EP, Gildengorin G, van Disseldorp J, Rosen M, Cedars MI

A pilot study of subcutaneous decitabine in �-thalassemia intermedia.

Blood

Olivieri NF, Saunthararajah Y, Thayalasuthan V, Kwiatkowski J, Ware RE, Kuypers FA, Kim HY, Trachtenberg FL, Vichinsky EP, Thalassemia Clinical Research Network

Long-term safety and efficacy of deferasirox (Exjade) for up to 5 years in transfusional iron-overloaded patients with sickle cell disease.

British journal of haematology

Vichinsky E, Bernaudin F, Forni GL, Gardner R, Hassell K, Heeney MM, Inusa B, Kutlar A, Lane P, Mathias L, Porter J, Tebbi C, Wilson F, Griffel L, Deng W, Giannone V, Coates T

Iron chelation adherence to deferoxamine and deferasirox in thalassemia.

American journal of hematology

Trachtenberg F, Vichinsky E, Haines D, Pakbaz Z, Mednick L, Sobota A, Kwiatkowski J, Thompson AA, Porter J, Coates T, Giardina PJ, Olivieri N, Yamashita R, Neufeld EJ, Thalassemia Clinical Research Network

Darbepoetin alfa for the treatment of anaemia in alpha- or beta- thalassaemia intermedia syndromes.

British journal of haematology

Singer ST, Vichinsky EP, Sweeters N, Rachmilewitz E

A pilot study of the short-term use of simvastatin in sickle cell disease: effects on markers of vascular dysfunction.

British journal of haematology

Hoppe C, Kuypers F, Larkin S, Hagar W, Vichinsky E, Styles L

Characterization of low bone mass in young patients with thalassemia by DXA, pQCT and markers of bone turnover.

Bone

Fung EB, Vichinsky EP, Kwiatkowski JL, Huang J, Bachrach LK, Sawyer AJ, Zemel BS

Heterogeneity of hemoglobin H disease in childhood.

The New England journal of medicine

Lal A, Goldrich ML, Haines DA, Azimi M, Singer ST, Vichinsky EP

Red cell alloimmunization in a diverse population of transfused patients with thalassaemia.

British journal of haematology

Thompson AA, Cunningham MJ, Singer ST, Neufeld EJ, Vichinsky E, Yamashita R, Giardina P, Kim HY, Trachtenberg F, Kwiatkowski JL, Thalassemia Clinical Research Network Investigators

The effects of hydroxycarbamide and magnesium on haemoglobin SC disease: results of the multi-centre CHAMPS trial

British journal of haematology

Wang, W., Brugnara, C., Snyder, C., Wynn, L., Rogers, Z., Kalinyak, K., Brown, C., Qureshi, A., Bigelow, C., Neumayr, L., Smith-Whitley, K., Chui, D. H. K., Delahunty, M., Woolson, R., Steinberg, M., Telen, M. and Kesler, K

Novel influenza A (H1N1) viral infection in pediatric patients with sickle-cell disease.

Pediatric blood & cancer

Jacobs JE, Quirolo K, Vichinsky E

A phase 1 dose-escalation study: safety, tolerability, and pharmacokinetics of FBS0701, a novel oral iron chelator for the treatment of transfusional iron overload.

Haematologica

Rienhoff HY, Viprakasit V, Tay L, Harmatz P, Vichinsky E, Chirnomas D, Kwiatkowski JL, Tapper A, Kramer W, Porter JB, Neufeld EJ

HbE/�-thalassemia: basis of marked clinical diversity.

Hematology/oncology clinics of North America

Olivieri NF, Pakbaz Z, Vichinsky E

Chart Card: feasibility of a tool for improving emergency department care in sickle cell disease.

Journal of the National Medical Association

Neumayr L, Pringle S, Giles S, Quirolo KC, Paulukonis S, Vichinsky EP, Treadwell MJ

Education and employment status of children and adults with thalassemia in North America.

Pediatric blood & cancer

Pakbaz Z, Treadwell M, Kim HY, Trachtenberg F, Parmar N, Kwiatkowski JL, Cunningham MJ, Martin M, Sweeters N, Neufeld EJ, Giardina PJ, Olivieri N, Yamashita RC, Vichinsky E

Pulmonary hypertension and NO in sickle cell.

Blood

Gladwin MT, Barst RJ, Castro OL, Gordeuk VR, Hillery CA, Kato GJ, Kim-Shapiro DB, Machado R, Morris CR, Steinberg MH, Vichinsky EP

Fertility potential in thalassemia major women: current findings and future diagnostic tools.

Annals of the New York Academy of Sciences

Singer ST, Sweeters N, Vega O, Higa A, Vichinsky E, Cedars M

Preface to Cooley's Anemia: Ninth Symposium.

Annals of the New York Academy of Sciences

Vichinsky EP, Neufeld EJ

Pulmonary hypertension in thalassemia.

Annals of the New York Academy of Sciences

Morris CR, Vichinsky EP

Approaches to transfusion therapy and iron overload in patients with sickle cell disease: results of an international survey.

Pediatric hematology and oncology

Vichinsky EP, Ohene-Frempong K, Transfusion Committee

Transfusion and chelation practices in sickle cell disease: a regional perspective.

Pediatric hematology and oncology

Vichinsky EP, Ohene-Frempong K, Thein SL, Lobo CL, Inati A, Thompson AA, Smith-Whitley K, Kwiatkowski JL, Swerdlow PS, Porter JB, Marks PW

Relationship between chronic transfusion therapy and body composition in subjects with thalassemia.

The Journal of pediatrics

Fung EB, Xu Y, Kwiatkowski JL, Vogiatzi MG, Neufeld E, Olivieri N, Vichinsky EP, Giardina PJ, Thalassemia Clinical Research Network

Neuropsychological dysfunction and neuroimaging abnormalities in neurologically intact adults with sickle cell anemia.

JAMA

Vichinsky EP, Neumayr LD, Gold JI, Weiner MW, Rule RR, Truran D, Kasten J, Eggleston B, Kesler K, McMahon L, Orringer EP, Harrington T, Kalinyak K, De Castro LM, Kutlar A, Rutherford CJ, Johnson C, Bessman JD, Jordan LB, Armstrong FD, Neuropsychological Dysfunction and Neuroimaging Adult Sickle Cell Anemia Study Group

Elevated exhaled carbon monoxide concentration in hemoglobinopathies and its relation to red blood cell transfusion therapy.

Pediatric hematology and oncology

James EB, Vreman HJ, Wong RJ, Stevenson DK, Vichinsky E, Schumacher L, Hall JY, Simon J, Golden DW, Harmatz P

Pulmonary hypertension in hemolytic anemias.

F1000 medicine reports

Wahl S, Vichinsky E

Hemoglobin Hakkari: an autosomal dominant form of beta thalassemia with inclusion bodies arising from de novo mutation in exon 2 of beta globin gene.

Pediatric blood & cancer

Kanathezhath B, Hazard FK, Guo H, Kidd J, Azimi M, Kuypers FA, Vichinsky EP, Lal A

Assessment of cardiac iron by MRI susceptometry and R2* in patients with thalassemia.

Magnetic resonance imaging

Wang ZJ, Fischer R, Chu Z, Mahoney DH, Mueller BU, Muthupillai R, James EB, Krishnamurthy R, Chung T, Padua E, Vichinsky E, Harmatz P

Low dose, oral epsilon aminocaproic acid for renal papillary necrosis and massive hemorrhage in hemoglobin SC disease.

Pediatric blood & cancer

Gabrovsky A, Aderinto A, Aderinto A, Spevak M, Vichinsky E, Resar LM

Iron metabolism and iron chelation in sickle cell disease.

Acta haematologica

Walter PB, Harmatz P, Vichinsky E

Hemoglobin H-constant spring in North America: an alpha thalassemia with frequent complications.

American journal of hematology

Singer ST, Kim HY, Olivieri NF, Kwiatkowski JL, Coates TD, Carson S, Neufeld E, Cunningham MJ, Giardina PJ, Mueller BU, Quinn CT, Fung E, Vichinsky E, Thalassemia Clinical Research Network

Severe sickle cell disease--pathophysiology and therapy.

Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation

Buchanan G, Vichinsky E, Krishnamurti L, Shenoy S

Efficacy and safety of deferasirox doses of >30 mg/kg per d in patients with transfusion-dependent anaemia and iron overload.

British journal of haematology

Taher A, Cappellini MD, Vichinsky E, Galanello R, Piga A, Lawniczek T, Clark J, Habr D, Porter JB

Serum ferritin level changes in children with sickle cell disease on chronic blood transfusion are nonlinear and are associated with iron load and liver injury.

Blood

Adamkiewicz TV, Abboud MR, Paley C, Olivieri N, Kirby-Allen M, Vichinsky E, Casella JF, Alvarez OA, Barredo JC, Lee MT, Iyer RV, Kutlar A, McKie KM, McKie V, Odo N, Gee B, Kwiatkowski JL, Woods GM, Coates T, Wang W, Adams RJ

Differences in the prevalence of growth, endocrine and vitamin D abnormalities among the various thalassaemia syndromes in North America.

British journal of haematology

Vogiatzi MG, Macklin EA, Trachtenberg FL, Fung EB, Cheung AM, Vichinsky E, Olivieri N, Kirby M, Kwiatkowski JL, Cunningham M, Holm IA, Fleisher M, Grady RW, Peterson CM, Giardina PJ, Thalassemia Clinical Research Network

HLA type and risk of alloimmunization in sickle cell disease.

American journal of hematology

Hoppe C, Klitz W, Vichinsky E, Styles L

Newborn screening for hemoglobinopathies in California.

Pediatric blood & cancer

Michlitsch J, Azimi M, Hoppe C, Walters MC, Lubin B, Lorey F, Vichinsky E

Bone disease in thalassemia: a frequent and still unresolved problem.

Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research

Vogiatzi MG, Macklin EA, Fung EB, Cheung AM, Vichinsky E, Olivieri N, Kirby M, Kwiatkowski JL, Cunningham M, Holm IA, Lane J, Schneider R, Fleisher M, Grady RW, Peterson CC, Giardina PJ, Thalassemia Clinical Research Network

Alpha thalassemia major--new mutations, intrauterine management, and outcomes.

Hematology. American Society of Hematology. Education Program

Vichinsky EP

Pulmonary complications of sickle cell disease.

The New England journal of medicine

Gladwin MT, Vichinsky E

Hemoglobinopathies.

Current molecular medicine

Vichinsky E, Rodgers GP, Rachmilewitz E

Hydroxycarbamide-induced changes in E/beta thalassemia red blood cells.

American journal of hematology

Singer ST, Vichinsky EP, Larkin S, Olivieri N, Sweeters N, Kuypers FA, E/beta Thalassemia Study Group

Psychosocial and behavioral outcomes in children with sickle cell disease and their healthy siblings.

Journal of behavioral medicine

Gold JI, Mahrer NE, Treadwell M, Weissman L, Vichinsky E

Safety and efficacy of pegylated interferon alpha-2a and ribavirin for the treatment of hepatitis C in patients with thalassemia.

Haematologica

Harmatz P, Jonas MM, Kwiatkowski JL, Wright EC, Fischer R, Vichinsky E, Giardina PJ, Neufeld EJ, Porter J, Olivieri N, Thalassemia Clinical Research Network

Detection and assessment of stroke in patients with sickle cell disease: neuropsychological functioning and magnetic resonance imaging.

Pediatric hematology and oncology

Gold JI, Johnson CB, Treadwell MJ, Hans N, Vichinsky E

Disparity in the management of iron overload between patients with sickle cell disease and thalassemia who received transfusions.

Transfusion

Fung EB, Harmatz PR, Milet M, Balasa V, Ballas SK, Casella JF, Hilliard L, Kutlar A, McClain KL, Olivieri NF, Porter JB, Vichinsky EP, Multi-Center Iron Overload Research Group

Inflammation and oxidant-stress in beta-thalassemia patients treated with iron chelators deferasirox (ICL670) or deferoxamine: an ancillary study of the Novartis CICL670A0107 trial.

Haematologica

Walter PB, Macklin EA, Porter J, Evans P, Kwiatkowski JL, Neufeld EJ, Coates T, Giardina PJ, Vichinsky E, Olivieri N, Alberti D, Holland J, Harmatz P, Thalassemia Clinical Research Network

Clinical application of deferasirox: practical patient management.

American journal of hematology

Vichinsky E

Patient-reported outcomes of deferasirox (Exjade, ICL670) versus deferoxamine in sickle cell disease patients with transfusional hemosiderosis. Substudy of a randomized open-label phase II trial.

Acta haematologica

Vichinsky E, Pakbaz Z, Onyekwere O, Porter J, Swerdlow P, Coates T, Lane P, Files B, Mueller BU, Coïc L, Forni GL, Fischer R, Marks P, Rofail D, Abetz L, Baladi JF

Fracture prevalence and relationship to endocrinopathy in iron overloaded patients with sickle cell disease and thalassemia.

Bone

Fung EB, Harmatz PR, Milet M, Coates TD, Thompson AA, Ranalli M, Mignaca R, Scher C, Giardina P, Robertson S, Neumayr L, Vichinsky EP, Multi-Center Iron Overload Study Group

Advances in clinical research in sickle cell disease.

British journal of haematology

Hagar W, Vichinsky E

Transfusional iron burden and liver toxicity after bone marrow transplantation for acute myelogenous leukemia and hemoglobinopathies.

Pediatric blood & cancer

Jastaniah W, Harmatz P, Pakbaz Z, Fischer R, Vichinsky E, Walters MC

Efficacy and safety of the Gardos channel blocker, senicapoc (ICA-17043), in patients with sickle cell anemia.

Blood

Ataga KI, Smith WR, De Castro LM, Swerdlow P, Saunthararajah Y, Castro O, Vichinsky E, Kutlar A, Orringer EP, Rigdon GC, Stocker JW, ICA-17043-05 Investigators

Relative response of patients with myelodysplastic syndromes and other transfusion-dependent anaemias to deferasirox (ICL670): a 1-yr prospective study.

European journal of haematology

Porter J, Galanello R, Saglio G, Neufeld EJ, Vichinsky E, Cappellini MD, Olivieri N, Piga A, Cunningham MJ, Soulières D, Gattermann N, Tchernia G, Maertens J, Giardina P, Kwiatkowski J, Quarta G, Jeng M, Forni GL, Stadler M, Cario H, Debusscher L, Della Porta M, Cazzola M, Greenberg P, Alimena G, Rabault B, Gathmann I, Ford JM, Alberti D, Rose C

Clinical differences between children and adults with pulmonary hypertension and sickle cell disease.

British journal of haematology

Hagar RW, Michlitsch JG, Gardner J, Vichinsky EP, Morris CR

Erythrocyte glutamine depletion, altered redox environment, and pulmonary hypertension in sickle cell disease.

Blood

Morris CR, Suh JH, Hagar W, Larkin S, Bland DA, Steinberg MH, Vichinsky EP, Shigenaga M, Ames B, Kuypers FA, Klings ES

Phase Ib clinical trial of starch-conjugated deferoxamine (40SD02): a novel long-acting iron chelator.

British journal of haematology

Harmatz P, Grady RW, Dragsten P, Vichinsky E, Giardina P, Madden J, Jeng M, Miller B, Hanson G, Hedlund B

Iron homeostasis during transfusional iron overload in beta-thalassemia and sickle cell disease: changes in iron regulatory protein, hepcidin, and ferritin expression.

Pediatric hematology and oncology

Jenkins ZA, Hagar W, Bowlus CL, Johansson HE, Harmatz P, Vichinsky EP, Theil EC

Prospective evaluation of patient-reported outcomes during treatment with deferasirox or deferoxamine for iron overload in patients with beta-thalassemia.

Clinical therapeutics

Cappellini MD, Bejaoui M, Agaoglu L, Porter J, Coates T, Jeng M, Lai ME, Mangiagli A, Strauss G, Girot R, Watman N, Ferster A, Loggetto S, Abish S, Cario H, Zoumbos N, Vichinsky E, Opitz H, Ressayre-Djaffer C, Abetz L, Rofail D, Baladi JF

Morbidity and mortality in chronically transfused subjects with thalassemia and sickle cell disease: A report from the multi-center study of iron overload.

American journal of hematology

Fung EB, Harmatz P, Milet M, Ballas SK, De Castro L, Hagar W, Owen W, Olivieri N, Smith-Whitley K, Darbari D, Wang W, Vichinsky E, Multi-Center Study of Iron Overload Research Group

A randomised comparison of deferasirox versus deferoxamine for the treatment of transfusional iron overload in sickle cell disease.

British journal of haematology

Vichinsky E, Onyekwere O, Porter J, Swerdlow P, Eckman J, Lane P, Files B, Hassell K, Kelly P, Wilson F, Bernaudin F, Forni GL, Okpala I, Ressayre-Djaffer C, Alberti D, Holland J, Marks P, Fung E, Fischer R, Mueller BU, Coates T, Deferasirox in Sickle Cell Investigators

Caregiving time in sickle cell disease: psychological effects in maternal caregivers.

Pediatric blood & cancer

Moskowitz JT, Butensky E, Harmatz P, Vichinsky E, Heyman MB, Acree M, Wrubel J, Wilson L, Folkman S

Hemoglobin e syndromes.

Hematology. American Society of Hematology. Education Program

Vichinsky E

Bone mineral density in children with sickle cell anemia.

Pediatric blood & cancer

Lal A, Fung EB, Pakbaz Z, Hackney-Stephens E, Vichinsky EP

Physical therapy alone compared with core decompression and physical therapy for femoral head osteonecrosis in sickle cell disease. Results of a multicenter study at a mean of three years after treatment.

The Journal of bone and joint surgery. American volume

Neumayr LD, Aguilar C, Earles AN, Jergesen HE, Haberkern CM, Kammen BF, Nancarrow PA, Padua E, Milet M, Stulberg BN, Williams RA, Orringer EP, Graber N, Robertson SM, Vichinsky EP, National Osteonecrosis Trial in Sickle Cell Anemia Study Group

Dose-escalation study of ICA-17043 in patients with sickle cell disease.

Pharmacotherapy

Ataga KI, Orringer EP, Styles L, Vichinsky EP, Swerdlow P, Davis GA, Desimone PA, Stocker JW

Increased prevalence of iron-overload associated endocrinopathy in thalassaemia versus sickle-cell disease.

British journal of haematology

Fung EB, Harmatz PR, Lee PD, Milet M, Bellevue R, Jeng MR, Kalinyak KA, Hudes M, Bhatia S, Vichinsky EP, Multi-Centre Study of Iron Overload Research Group

Oxidative stress and inflammation in iron-overloaded patients with beta-thalassaemia or sickle cell disease.

British journal of haematology

Walter PB, Fung EB, Killilea DW, Jiang Q, Hudes M, Madden J, Porter J, Evans P, Vichinsky E, Harmatz P

Prevalence of HFE mutations in California newborns.

Pediatric hematology and oncology

Hoppe C, Watson RM, Long CM, Lorey F, Robles L, Klitz W, Styles L, Vichinsky E

Pulmonary hypertension in thalassemia: association with platelet activation and hypercoagulable state.

American journal of hematology

Singer ST, Kuypers FA, Styles L, Vichinsky EP, Foote D, Rosenfeld H

Prevalence of fractures among the Thalassemia syndromes in North America.

Bone

Vogiatzi MG, Macklin EA, Fung EB, Vichinsky E, Olivieri N, Kwiatkowski J, Cohen A, Neufeld E, Giardina PJ

Changes in the epidemiology of thalassemia in North America: a new minority disease.

Pediatrics

Vichinsky EP, MacKlin EA, Waye JS, Lorey F, Olivieri NF

Fetal haemoglobin augmentation in E/beta(0) thalassaemia: clinical and haematological outcome.

British journal of haematology

Singer ST, Kuypers FA, Olivieri NF, Weatherall DJ, Mignacca R, Coates TD, Davies S, Sweeters N, Vichinsky EP, E/beta Thalassaemia Study Group

Bone and joint disease in sickle cell disease.

Hematology/oncology clinics of North America

Aguilar C, Vichinsky E, Neumayr L

Comparison of organ dysfunction in transfused patients with SCD or beta thalassemia.

American journal of hematology

Vichinsky E, Butensky E, Fung E, Hudes M, Theil E, Ferrell L, Williams R, Louie L, Lee PD, Harmatz P

Dysregulated arginine metabolism, hemolysis-associated pulmonary hypertension, and mortality in sickle cell disease.

JAMA

Morris CR, Kato GJ, Poljakovic M, Wang X, Blackwelder WC, Sachdev V, Hazen SL, Vichinsky EP, Morris SM, Gladwin MT

Clinical evaluation of avascular necrosis in patients with sickle cell disease: Children's Hospital Oakland Hip Evaluation Scale--a modification of the Harris Hip Score.

Archives of physical medicine and rehabilitation

Aguilar CM, Neumayr LD, Eggleston BE, Earles AN, Robertson SM, Jergesen HE, Stulberg BN, Vichinsky EP

Barriers to adherence of deferoxamine usage in sickle cell disease.

Pediatric blood & cancer

Treadwell MJ, Law AW, Sung J, Hackney-Stephens E, Quirolo K, Murray E, Glendenning GA, Vichinsky E

A simple model to assess and improve adherence to iron chelation therapy with deferoxamine in patients with thalassemia.

Annals of the New York Academy of Sciences

Pakbaz Z, Fischer R, Treadwell M, Yamashita R, Fung EB, Calvelli L, Quirolo K, Foote D, Harmatz P, Vichinsky EP

Changing patterns of thalassemia worldwide.

Annals of the New York Academy of Sciences

Vichinsky EP

Hemoglobin E-beta-thalassemia: Progress report from the International Study Group.

Annals of the New York Academy of Sciences

Premawardhena A, De Silver S, Arambepola M, Olivieri NF, Vichinsky EP, Merson L, Muraco G, Allen A, Fisher C, Peto T, Weatherall DJ

Hemolysis-associated pulmonary hypertension in thalassemia.

Annals of the New York Academy of Sciences

Morris CR, Kuypers FA, Kato GJ, Lavrisha L, Larkin S, Singer T, Vichinsky EP

Measuring chromosome breaks in patients with thalassemia.

Annals of the New York Academy of Sciences

Offer T, Bhagat A, Lal A, Atamna W, Singer ST, Vichinsky EP, Kuypers FA, Ames BN

Outcomes of preimplantation genetic diagnosis therapy in treatment of beta-thalassemia: A retrospective analysis.

Annals of the New York Academy of Sciences

Qureshi N, Foote D, Walters MC, Singer ST, Quirolo K, Vichinsky EP

Quality of life in patients with thalassemia intermedia compared to thalassemia major.

Annals of the New York Academy of Sciences

Pakbaz Z, Treadwell M, Yamashita R, Quirolo K, Foote D, Quill L, Singer T, Vichinsky EP

Single and combination drug therapy for fetal hemoglobin augmentation in hemoglobin E-beta 0-thalassemia: Considerations for treatment.

Annals of the New York Academy of Sciences

Singer ST, Kuypers FA, Olivieri NF, Weatherall DJ, Mignacca R, Coates TD, Davies S, Sweeters N, Vichinsky EP

Treatment of hepatitis C virus infection in thalassemia.

Annals of the New York Academy of Sciences

Butensky E, Pakbaz Z, Foote D, Walters MC, Vichinsky EP, Harmatz P

Utility of Holter electrocardiogram in iron-overloaded hemoglobinopathies.

Annals of the New York Academy of Sciences

Qureshi N, Avasarala K, Foote D, Vichinsky EP

Variability in hepatic iron concentration in percutaneous needle biopsy specimens from patients with transfusional hemosiderosis.

American journal of clinical pathology

Butensky E, Fischer R, Hudes M, Schumacher L, Williams R, Moyer TP, Vichinsky E, Harmatz P

Hematologic problems in immigrants from Southeast Asia.

Hematology/oncology clinics of North America

Jeng MR, Vichinsky E

The new SQUID biosusceptometer at Oakland: first year of experience.

Neurology & clinical neurophysiology : NCN

Fung EB, Fischer R, Pakbaz Z, Fagaly RL, Vichinsky E, Starr TN, Ewing T, Paulson DN, Hassenzahl WV, Harmatz P

Can peak systolic velocities be used for prediction of stroke in sickle cell anemia?

Pediatric radiology

Jones A, Granger S, Brambilla D, Gallagher D, Vichinsky E, Woods G, Berman B, Roach S, Nichols F, Adams RJ

Thalassemia in Sri Lanka: a progress report.

Human molecular genetics

Premawardhena A, De Silva S, Arambepola M, Olivieri N, Merson L, Muraco J, Allen A, Fisher C, Peto T, Vichinsky E, Weatherall D

The role of fetal hemoglobin-enhancing agents in thalassemia.

Seminars in hematology

Lal A, Vichinsky E

Pulmonary hypertension in sickle cell disease.

The New England journal of medicine

Vichinsky EP

Stroke and conversion to high risk in children screened with transcranial Doppler ultrasound during the STOP study.

Blood

Adams RJ, Brambilla DJ, Granger S, Gallagher D, Vichinsky E, Abboud MR, Pegelow CH, Woods G, Rohde EM, Nichols FT, Jones A, Luden JP, Bowman L, Hagner S, Morales KH, Roach ES, STOP Study

Thalassemia.

Hematology. American Society of Hematology. Education Program

Cohen AR, Galanello R, Pennell DJ, Cunningham MJ, Vichinsky E

Managing sickle cell disease.

BMJ (Clinical research ed.)

Claster S, Vichinsky EP

Gene interactions and stroke risk in children with sickle cell anemia.

Blood

Hoppe C, Klitz W, Cheng S, Apple R, Steiner L, Robles L, Girard T, Vichinsky E, Styles L, CSSCD Investigators

Invasive pneumococcal infections in children with sickle cell disease in the era of penicillin prophylaxis, antibiotic resistance, and 23-valent pneumococcal polysaccharide vaccination.

The Journal of pediatrics

Adamkiewicz TV, Sarnaik S, Buchanan GR, Iyer RV, Miller ST, Pegelow CH, Rogers ZR, Vichinsky E, Elliott J, Facklam RR, O'Brien KL, Schwartz B, Van Beneden CA, Cannon MJ, Eckman JR, Keyserling H, Sullivan K, Wong WY, Wang WC

Hydroxyurea and arginine therapy: impact on nitric oxide production in sickle cell disease.

Journal of pediatric hematology/oncology

Morris CR, Vichinsky EP, van Warmerdam J, Machado L, Kepka-Lenhart D, Morris SM, Kuypers FA

Mycoplasma disease and acute chest syndrome in sickle cell disease.

Pediatrics

Neumayr L, Lennette E, Kelly D, Earles A, Embury S, Groncy P, Grossi M, Grover R, McMahon L, Swerdlow P, Waldron P, Vichinsky E

Deferiprone and hepatic fibrosis.

Blood

Brittenham GM, Nathan DG, Olivieri NF, Porter JB, Pippard M, Vichinsky EP, Weatherall DJ

Effect of hydroxyurea on mortality and morbidity in adult sickle cell anemia: risks and benefits up to 9 years of treatment.

JAMA

Steinberg MH, Barton F, Castro O, Pegelow CH, Ballas SK, Kutlar A, Orringer E, Bellevue R, Olivieri N, Eckman J, Varma M, Ramirez G, Adler B, Smith W, Carlos T, Ataga K, DeCastro L, Bigelow C, Saunthararajah Y, Telfer M, Vichinsky E, Claster S, Shurin S, Bridges K, Waclawiw M, Bonds D, Terrin M

Liver ferritin subunit ratios in neonatal hemochromatosis.

Pediatric hematology and oncology

Hagar W, Vichinsky EP, Theil EC

Arginine therapy: a new treatment for pulmonary hypertension in sickle cell disease?

American journal of respiratory and critical care medicine

Morris CR, Morris SM, Hagar W, Van Warmerdam J, Claster S, Kepka-Lenhart D, Machado L, Kuypers FA, Vichinsky EP

Chlamydia pneumoniae and acute chest syndrome in patients with sickle cell disease.

Journal of pediatric hematology/oncology

Dean D, Neumayr L, Kelly DM, Ballas SK, Kleman K, Robertson S, Iyer RV, Ware RE, Koshy M, Rackoff WR, Pegelow CH, Waldron P, Benjamin L, Vichinsky E, Acute Chest Syndrome Study Group

Distinct HLA associations by stroke subtype in children with sickle cell anemia.

Blood

Hoppe C, Klitz W, Noble J, Vigil L, Vichinsky E, Styles L

Pancytopenia induced by hypothermia.

Journal of pediatric hematology/oncology

Lo L, Singer ST, Vichinsky E

Diseases of iron metabolism.

Pediatric clinics of North America

Hagar W, Theil EC, Vichinsky EP

New therapies in sickle cell disease.

Lancet (London, England)

Vichinsky E

Bioelectrical impedance analysis of the body composition of children and adolescents with sickle cell disease.

The Journal of pediatrics

VanderJagt DJ, Harmatz P, Scott-Emuakpor AB, Vichinsky E, Glew RH

Longitudinal changes in ferritin during chronic transfusion: a report from the Stroke Prevention Trial in Sickle Cell Anemia (STOP).

Journal of pediatric hematology/oncology

Files B, Brambilla D, Kutlar A, Miller S, Vichinsky E, Wang W, Granger S, Adams RJ

Longitudinal changes in brain magnetic resonance imaging findings in children with sickle cell disease.

Blood

Pegelow CH, Macklin EA, Moser FG, Wang WC, Bello JA, Miller ST, Vichinsky EP, DeBaun MR, Guarini L, Zimmerman RA, Younkin DP, Gallagher DM, Kinney TR

Assessment of sickle cell pain in children and young adults using the adolescent pediatric pain tool.

Journal of pain and symptom management

Franck LS, Treadwell M, Jacob E, Vichinsky E

Central venous catheter complications in sickle cell disease.

American journal of hematology

Jeng MR, Feusner J, Skibola C, Vichinsky E

Effect of hydroxyurea on growth in children with sickle cell anemia: results of the HUG-KIDS Study.

The Journal of pediatrics

Wang WC, Helms RW, Lynn HS, Redding-Lallinger R, Gee BE, Ohene-Frempong K, Smith-Whitley K, Waclawiw MA, Vichinsky EP, Styles LA, Ware RE, Kinney TR

Using quality improvement strategies to enhance pediatric pain assessment.

International journal for quality in health care : journal of the International Society for Quality in Health Care

Treadwell MJ, Franck LS, Vichinsky E

Novel therapeutic approaches in sickle cell disease.

Hematology. American Society of Hematology. Education Program

Walters MC, Nienhuis AW, Vichinsky E

Silent infarcts in children with sickle cell anemia and abnormal cerebral artery velocity.

Archives of neurology

Pegelow CH, Wang W, Granger S, Hsu LL, Vichinsky E, Moser FG, Bello J, Zimmerman RA, Adams RJ, Brambilla D, STOP Trial

A novel multilocus genotyping assay to identify genetic predictors of stroke in sickle cell anaemia.

British journal of haematology

Hoppe C, Cheng S, Grow M, Silbergleit A, Klitz W, Trachtenberg E, Erlich H, Vichinsky E, Styles L

Neuropsychologic performance in school-aged children with sickle cell disease: a report from the Cooperative Study of Sickle Cell Disease.

The Journal of pediatrics

Wang W, Enos L, Gallagher D, Thompson R, Guarini L, Vichinsky E, Wright E, Zimmerman R, Armstrong FD, Cooperative Study of Sickle Cell Disease

Prospective RBC phenotype matching in a stroke-prevention trial in sickle cell anemia: a multicenter transfusion trial.

Transfusion

Vichinsky EP, Luban NL, Wright E, Olivieri N, Driscoll C, Pegelow CH, Adams RJ, Stroke Prevention Trail in Sickle Cell Anemia

Silent infarction as a risk factor for overt stroke in children with sickle cell anemia: a report from the Cooperative Study of Sickle Cell Disease.

The Journal of pediatrics

Miller ST, Macklin EA, Pegelow CH, Kinney TR, Sleeper LA, Bello JA, DeWitt LD, Gallagher DM, Guarini L, Moser FG, Ohene-Frempong K, Sanchez N, Vichinsky EP, Wang WC, Wethers DL, Younkin DP, Zimmerman RA, DeBaun MR, Cooperative Study of Sickle Cell Disease

Transfusion therapy: a coming-of-age treatment for patients with sickle cell disease.

Journal of pediatric hematology/oncology

Reed W, Vichinsky EP

Nesidioblastosis in sickle cell disease.

Pediatric pathology & molecular medicine

Culberson DE, Manci EA, Shah AK, Haynes J, Ballas SK, Pegelow C, Vichinsky E

Stable mixed hematopoietic chimerism after bone marrow transplantation for sickle cell anemia.

Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation

Walters MC, Patience M, Leisenring W, Rogers ZR, Aquino VM, Buchanan GR, Roberts IA, Yeager AM, Hsu L, Adamkiewicz T, Kurtzberg J, Vichinsky E, Storer B, Storb R, Sullivan KM, Multicenter Investigation of Bone Marrow Transplantation for Sickle Cell Disease

Stem cell transplantation for sickle cell disease: can we reduce the toxicity?

Pediatric pathology & molecular medicine

Fixler J, Vichinsky E, Walters MC

Universal newborn screening for Hb H disease in California.

Genetic testing

Lorey F, Cunningham G, Vichinsky EP, Lubin BH, Witkowska HE, Matsunaga A, Azimi M, Sherwin J, Eastman J, Farina F, Waye JS, Chui DH

Arginine therapy: a novel strategy to induce nitric oxide production in sickle cell disease.

British journal of haematology

Morris CR, Kuypers FA, Larkin S, Sweeters N, Simon J, Vichinsky EP, Styles LA

Changing outcome of homozygous alpha-thalassemia: cautious optimism.

Journal of pediatric hematology/oncology

Singer ST, Styles L, Bojanowski J, Quirolo K, Foote D, Vichinsky EP

Patterns of arginine and nitric oxide in patients with sickle cell disease with vaso-occlusive crisis and acute chest syndrome.

Journal of pediatric hematology/oncology

Morris CR, Kuypers FA, Larkin S, Vichinsky EP, Styles LA

Multicenter comparison of magnetic resonance imaging and transcranial Doppler ultrasonography in the evaluation of the central nervous system in children with sickle cell disease.

Journal of pediatric hematology/oncology

Wang WC, Gallagher DM, Pegelow CH, Wright EC, Vichinsky EP, Abboud MR, Moser FG, Adams RJ

Severity of iron overload in patients with sickle cell disease receiving chronic red blood cell transfusion therapy.

Blood

Harmatz P, Butensky E, Quirolo K, Williams R, Ferrell L, Moyer T, Golden D, Neumayr L, Vichinsky E

Use of hydroxyurea in children ages 2 to 5 years with sickle cell disease.

Journal of pediatric hematology/oncology

Hoppe C, Vichinsky E, Quirolo K, van Warmerdam J, Allen K, Styles L

Causes and outcomes of the acute chest syndrome in sickle cell disease. National Acute Chest Syndrome Study Group.

The New England journal of medicine

Vichinsky EP, Neumayr LD, Earles AN, Williams R, Lennette ET, Dean D, Nickerson B, Orringer E, McKie V, Bellevue R, Daeschner C, Manci EA

Evidence for HLA-related susceptibility for stroke in children with sickle cell disease.

Blood

Styles LA, Hoppe C, Klitz W, Vichinsky E, Lubin B, Trachtenberg E

Impact of bone marrow transplantation for symptomatic sickle cell disease: an interim report. Multicenter investigation of bone marrow transplantation for sickle cell disease.

Blood

Walters MC, Storb R, Patience M, Leisenring W, Taylor T, Sanders JE, Buchanan GE, Rogers ZR, Dinndorf P, Davies SC, Roberts IA, Dickerhoff R, Yeager AM, Hsu L, Kurtzberg J, Ohene-Frempong K, Bunin N, Bernaudin F, Wong WY, Scott JP, Margolis D, Vichinsky E, Wall DA, Wayne AS, Pegelow C, Redding-Lallinger R, Wiley J, Klemperer M, Mentzer WC, Smith FO, Sullivan KM

Sickle-cell disease not identified by newborn screening because of prior transfusion.

The Journal of pediatrics

Reed W, Lane PA, Lorey F, Bojanowski J, Glass M, Louie RR, Lubin BH, Vichinsky EP

Major changes in sickle cell disease.

Advances in pediatrics

Hagar RW, Vichinsky EP

Hydroxyurea and sodium phenylbutyrate therapy in thalassemia intermedia.

American journal of hematology

Hoppe C, Vichinsky E, Lewis B, Foote D, Styles L

The perioperative complication rate of orthopedic surgery in sickle cell disease: report of the National Sickle Cell Surgery Study Group.

American journal of hematology

Vichinsky EP, Neumayr LD, Haberkern C, Earles AN, Eckman J, Koshy M, Black DM

Transfusion practice for patients with sickle cell disease.

Current opinion in hematology

Reed WF, Vichinsky EP

Bone disease in beta-thalassaemia.

Lancet (London, England)

Singer ST, Vichinsky EP

Safety of hydroxyurea in children with sickle cell anemia: results of the HUG-KIDS study, a phase I/II trial. Pediatric Hydroxyurea Group.

Blood

Kinney TR, Helms RW, O'Branski EE, Ohene-Frempong K, Wang W, Daeschner C, Vichinsky E, Redding-Lallinger R, Gee B, Platt OS, Ware RE

Effects of red blood cell transfusion on resting energy expenditure in adolescents with sickle cell anemia.

Journal of pediatric gastroenterology and nutrition

Harmatz P, Heyman MB, Cunningham J, Lee PD, Styles L, Quirolo K, Kopp-Hoolihan L, Ghiron J, Hintz RL, Vichinsky E

Silent cerebral infarcts in sickle cell anemia: a risk factor analysis. The Cooperative Study of Sickle Cell Disease.

Pediatrics

Kinney TR, Sleeper LA, Wang WC, Zimmerman RA, Pegelow CH, Ohene-Frempong K, Wethers DL, Bello JA, Vichinsky EP, Moser FG, Gallagher DM, DeBaun MR, Platt OS, Miller ST

Tonsillectomy, adenoidectomy, and myringotomy in sickle cell disease: perioperative morbidity. Preoperative Transfusion in Sickle Cell Disease Study Group.

Journal of pediatric hematology/oncology

Waldron P, Pegelow C, Neumayr L, Haberkern C, Earles A, Wesman R, Vichinsky E

Deferoxamine treatment during pregnancy: is it harmful?

American journal of hematology

Singer ST, Vichinsky EP

Prevention of a first stroke by transfusions in children with sickle cell anemia and abnormal results on transcranial Doppler ultrasonography.

The New England journal of medicine

Adams RJ, McKie VC, Hsu L, Files B, Vichinsky E, Pegelow C, Abboud M, Gallagher D, Kutlar A, Nichols FT, Bonds DR, Brambilla D

Approaches to working with adult thalassemia patients in pediatric settings.

Annals of the New York Academy of Sciences

Weissman L, Treadwell M, Foote D, Heer N, Vichinsky EP

Bone marrow transplant in thalassemia. A role for radiation?

Annals of the New York Academy of Sciences

Lee YS, Kristovich KM, Ducore JM, Vichinsky E, Crouse VL, Glader BE, Amylon MD

Outreach strategies for Asian Pacific Island (API) communities.

Annals of the New York Academy of Sciences

Choy J, Yamashita RC, Foote D, Heer N, Vichinsky EP

Preliminary report: hydroxyurea produces significant clinical response in thalassemia intermedia.

Annals of the New York Academy of Sciences

Styles L, Lewis B, Foote D, Cuda L, Vichinsky EP

The hemoglobin E syndromes.

Annals of the New York Academy of Sciences

Rees DC, Styles L, Vichinsky EP, Clegg JB, Weatherall DJ

The morbidity of bone disease in thalassemia.

Annals of the New York Academy of Sciences

Vichinsky EP

The social impact of migration on disease. Cooley's anemia, thalassemia, and new Asian immigrants.

Annals of the New York Academy of Sciences

Heer N, Choy J, Vichinsky EP

Acute chest syndrome and sickle cell disease.

Current opinion in hematology

Golden C, Styles L, Vichinsky E

Stroke prevention trial in sickle cell anemia.

Controlled clinical trials

Adams RJ, McKie VC, Brambilla D, Carl E, Gallagher D, Nichols FT, Roach S, Abboud M, Berman B, Driscoll C, Files B, Hsu L, Hurlet A, Miller S, Olivieri N, Pegelow C, Scher C, Vichinsky E, Wang W, Woods G, Kutlar A, Wright E, Hagner S, Tighe F, Waclawiw MA

Surgery in patients with hemoglobin SC disease. Preoperative Transfusion in Sickle Cell Disease Study Group.

American journal of hematology

Neumayr L, Koshy M, Haberkern C, Earles AN, Bellevue R, Hassell K, Miller S, Black D, Vichinsky E

The natural history of sickle cell disease.

Current opinion in pediatrics

Hoppe C, Styles L, Vichinsky E

New considerations in the treatment of sickle cell disease.

Annual review of medicine

Reed W, Vichinsky EP

Hydroxyurea in children: present and future.

Seminars in hematology

Vichinsky EP

Influence of penicillin prophylaxis on antimicrobial resistance in nasopharyngeal S. pneumoniae among children with sickle cell anemia. The Ancillary Nasopharyngeal Culture Study of Prophylactic Penicillin Study II.

Journal of pediatric hematology/oncology

Woods GM, Jorgensen JH, Waclawiw MA, Reid C, Wang W, Pegelow CH, Rogers ZR, Iyer RV, Holbrook CT, Kinney TR, Vichinsky E, DeBaun MR, Grossman NJ, Thomas MD, Falletta JM

Umbilical cord blood stem cells: application for the treatment of patients with hemoglobinopathies.

The Journal of pediatrics

Kelly P, Kurtzberg J, Vichinsky E, Lubin B

Natural history of blood pressure in sickle cell disease: risks for stroke and death associated with relative hypertension in sickle cell anemia.

The American journal of medicine

Pegelow CH, Colangelo L, Steinberg M, Wright EC, Smith J, Phillips G, Vichinsky E

New therapies and approaches to transfusion in sickle cell disease in children.

Current opinion in pediatrics

Styles LA, Vichinsky EP

Pulmonary complications.

Hematology/oncology clinics of North America

Vichinsky E, Styles L

Serotype-specific immunoglobulin G antibody responses to pneumococcal polysaccharide vaccine in children with sickle cell anemia: effects of continued penicillin prophylaxis.

The Journal of pediatrics

Bjornson AB, Falletta JM, Verter JI, Buchanan GR, Miller ST, Pegelow CH, Iyer RV, Johnstone HS, DeBaun MR, Wethers DL, Wang WC, Woods GM, Holbrook CT, Becton DL, Kinney TR, Reaman GH, Kalinyak K, Grossman NJ, Vichinsky E, Reid CD

Core decompression in avascular necrosis of the hip in sickle-cell disease.

American journal of hematology

Styles LA, Vichinsky EP

Barriers to bone marrow transplantation for sickle cell anemia.

Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation

Walters MC, Patience M, Leisenring W, Eckman JR, Buchanan GR, Rogers ZR, Olivieri NE, Vichinsky E, Davies SC, Mentzer WC, Powars D, Scott JP, Bernaudin F, Ohene-Frempong K, Darbyshire PJ, Wayne A, Roberts IA, Dinndorf P, Brandalise S, Sanders JE, Matthews DC, Appelbaum FR, Storb R, Sullivan KM

The spectrum of brain MR abnormalities in sickle-cell disease: a report from the Cooperative Study of Sickle Cell Disease.

AJNR. American journal of neuroradiology

Moser FG, Miller ST, Bello JA, Pegelow CH, Zimmerman RA, Wang WC, Ohene-Frempong K, Schwartz A, Vichinsky EP, Gallagher D, Kinney TR

Phospholipase A2 levels in acute chest syndrome of sickle cell disease.

Blood

Styles LA, Schalkwijk CG, Aarsman AJ, Vichinsky EP, Lubin BH, Kuypers FA

Newborn screening for sickle cell disease: 4 years of experience from California's newborn screening program.

Journal of pediatric hematology/oncology

Shafer FE, Lorey F, Cunningham GC, Klumpp C, Vichinsky E, Lubin B

Ototoxicity in hemoglobinopathy patients chelated with desferrioxamine.

Journal of pediatric hematology/oncology

Styles LA, Vichinsky EP

Surgery and anesthesia in sickle cell disease. Cooperative Study of Sickle Cell Diseases.

Blood

Koshy M, Weiner SJ, Miller ST, Sleeper LA, Vichinsky E, Brown AK, Khakoo Y, Kinney TR

Discontinuing penicillin prophylaxis in children with sickle cell anemia. Prophylactic Penicillin Study II.

The Journal of pediatrics

Falletta JM, Woods GM, Verter JI, Buchanan GR, Pegelow CH, Iyer RV, Miller ST, Holbrook CT, Kinney TR, Vichinsky E

A comparison of conservative and aggressive transfusion regimens in the perioperative management of sickle cell disease. The Preoperative Transfusion in Sickle Cell Disease Study Group.

The New England journal of medicine

Vichinsky EP, Haberkern CM, Neumayr L, Earles AN, Black D, Koshy M, Pegelow C, Abboud M, Ohene-Frempong K, Iyer RV

Clinical events in the first decade in a cohort of infants with sickle cell disease. Cooperative Study of Sickle Cell Disease.

Blood

Gill FM, Sleeper LA, Weiner SJ, Brown AK, Bellevue R, Grover R, Pegelow CH, Vichinsky E

Risk of recurrent stroke in patients with sickle cell disease treated with erythrocyte transfusions.

The Journal of pediatrics

Pegelow CH, Adams RJ, McKie V, Abboud M, Berman B, Miller ST, Olivieri N, Vichinsky E, Wang W, Brambilla D

Liver transplantation in a child with sickle cell anemia.

Transplantation

Lang T, Berquist WE, So SK, Cox KL, Rich EJ, Vichinsky E, Concepcion W, Esquivel CO

Chronic pulmonary disorders in sickle cell disease: findings at thin-section CT.

Radiology

Aquino SL, Gamsu G, Fahy JV, Claster S, Embury SH, Mentzer WC, Vichinsky EP

Pulmonary fat embolism: a distinct cause of severe acute chest syndrome in sickle cell anemia.

Blood

Vichinsky E, Williams R, Das M, Earles AN, Lewis N, Adler A, McQuitty J

New advances in the pathophysiology and management of sickle cell disease.

Current opinion in hematology

Shafer FE, Vichinsky E

Availability of related donors for bone marrow transplantation in sickle cell anemia.

The American journal of pediatric hematology/oncology

Mentzer WC, Heller S, Pearle PR, Hackney E, Vichinsky E

Butyrate derivatives. New agents for stimulating fetal globin production in the beta-globin disorders.

The American journal of pediatric hematology/oncology

Perrine SP, Olivieri NF, Faller DV, Vichinsky EP, Dover GJ, Ginder GD

Universal screening for hemoglobinopathies using high-performance liquid chromatography: clinical results of 2.2 million screens.

European journal of human genetics : EJHG

Lorey F, Cunningham G, Shafer F, Lubin B, Vichinsky E

Detection of Hb E/beta-thalassemia versus homozygous EE using high-performance liquid chromatography results from newborns.

Biochemical medicine and metabolic biology

Lorey FW, Cunningham GC, Vichinsky E, Lubin B, Shafer F, Eastman J

A short-term trial of butyrate to stimulate fetal-globin-gene expression in the beta-globin disorders.

The New England journal of medicine

Perrine SP, Ginder GD, Faller DV, Dover GH, Ikuta T, Witkowska HE, Cai SP, Vichinsky EP, Olivieri NF

F reticulocyte response in sickle cell anemia treated with recombinant human erythropoietin: a double-blind study.

Blood

Nagel RL, Vichinsky E, Shah M, Johnson R, Spadacino E, Fabry ME, Mangahas L, Abel R, Stamatoyannopoulos G

Sickle cell disease in a patient with sickle cell trait and compound heterozygosity for hemoglobin S and hemoglobin Quebec-Chori.

The New England journal of medicine

Witkowska HE, Lubin BH, Beuzard Y, Baruchel S, Esseltine DW, Vichinsky EP, Kleman KM, Bardakdjian-Michau J, Pinkoski L, Cahn S

Pain in sickle cell disease. Rates and risk factors.

The New England journal of medicine

Platt OS, Thorington BD, Brambilla DJ, Milner PF, Rosse WF, Vichinsky E, Kinney TR

Alloimmunization in sickle cell anemia and transfusion of racially unmatched blood.

The New England journal of medicine

Vichinsky EP, Earles A, Johnson RA, Hoag MS, Williams A, Lubin B

Vitamin C deficiency in patients with sickle cell anemia.

The American journal of pediatric hematology/oncology

Chiu D, Vichinsky E, Ho SL, Liu T, Lubin BH

Sodium butyrate enhances fetal globin gene expression in erythroid progenitors of patients with Hb SS and beta thalassemia.

Blood

Perrine SP, Miller BA, Faller DV, Cohen RA, Vichinsky EP, Hurst D, Lubin BH, Papayannopoulou T

Experience with newborn screening using isoelectric focusing.

Pediatrics

Kleman KM, Vichinsky E, Lubin BH

Elevated fasting breath hydrogen and abnormal hydrogen breath tests in children with sickle cell disease: a preliminary report.

The American journal of clinical nutrition

Heyman MB, Lande W, Vichinsky E, Mentzer W

Butyrate analogues modulate globin gene expression in human and ovine erythroid cells.

Progress in clinical and biological research

Perrine SP, Miller BA, Papayannopoulou T, Faller DV, Rudolph AM, Vichinsky E, Hurst D, Kan YW

Hematopoietic hormones: from cloning to clinic.

The American journal of pediatric hematology/oncology

Perrine SP, Vichinsky E, Faller DV

Newborn screening for sickle cell disease: effect on mortality.

Pediatrics

Vichinsky E, Hurst D, Earles A, Kleman K, Lubin B

Contraceptive use in the chronically ill adolescent female.

Journal of adolescent health care : official publication of the Society for Adolescent Medicine

Vichinsky E, McDonald C

Suggested guidelines for the treatment of children with sickle cell anemia.

Hematology/oncology clinics of North America

Vichinsky E, Lubin BH

Prophylaxis with oral penicillin in children with sickle cell anemia. A randomized trial.

The New England journal of medicine

Gaston MH, Verter JI, Woods G, Pegelow C, Kelleher J, Presbury G, Zarkowsky H, Vichinsky E, Iyer R, Lobel JS

Growth retardation in sickle-cell disease treated by nutritional support.

Lancet (London, England)

Heyman MB, Vichinsky E, Katz R, Gaffield B, Hurst D, Castillo R, Chiu D, Kleman K, Ammann AJ, Thaler MM

Long-term follow-up and booster immunization with polyvalent pneumococcal polysaccharide in patients with sickle cell anemia.

The Journal of pediatrics

Weintrub PS, Schiffman G, Addiego JE, Matthay KK, Vichinsky E, Johnson R, Lubin B, Mentzer WC, Ammann AJ

Inadequate erythroid response to hypoxia in cystic fibrosis.

The Journal of pediatrics

Vichinsky EP, Pennathur-Das R, Nickerson B, Minor M, Kleman K, Higashino S, Lubin B

Evidence for a heterogeneous response to erythropoietin in the CFUE pool of human bone marrow.

Experimental hematology

Pennathur-Das R, Alpen E, Vichinsky E, Lubin B

Iron deficiency in health and disease.

Advances in pediatrics

Reeves JD, Vichinsky E, Addiego J, Lubin BH

Concurrent sickle-cell anemia and alpha-thalassemia: effect on severity of anemia.

The New England journal of medicine

Embury SH, Dozy AM, Miller J, Davis JR, Kleman KM, Preisler H, Vichinsky E, Lande WN, Lubin BH, Kan YW, Mentzer WC

Multidisciplinary approach to pain management in sickle cell disease.

The American journal of pediatric hematology/oncology

Vichinsky EP, Johnson R, Lubin BH

Peroxidation, vitamin E, and sickle-cell anemia.

Annals of the New York Academy of Sciences

Chiu D, Vichinsky E, Yee M, Kleman K, Lubin B

The diagnosis of iron deficiency anemia in sickle cell disease.

Blood

Vichinsky E, Kleman K, Embury S, Lubin B

Current treatment of sickle cell disease.

Current problems in pediatrics

Davis JR, Vichinsky EP, Lubin BH

Sickle cell anemia and related hemoglobinopathies.

Pediatric clinics of North America

Vichinsky EP, Lubin BH

Unstable hemoglobins, hemoglobins with altered oxygen affinity, and m-hemoglobins.

Pediatric clinics of North America

Vichinsky EP, Lubin BH

Anemia in the newborn period.

Pediatric annals

Lubin B, Vichinsky E

Anemia associated with rheumatoid arthritis.

The Journal of pediatrics

Vichinsky EP

Immune response after splenectomy.

Lancet (London, England)

Sullivan JL, Ochs HD, Schiffman G, Hammerschlag MR, Miser J, Vichinsky E, Wedgwood RJ

Preparation and properties of a cell-free system from rat skin that catalyzes sterol biosynthesis.

Journal of lipid research

Gaylor JL, Delwiche CV, Brady DR, Green AJ

Guidelines for the Management of Non Tranfusion Dependent Thalassemia (NTDT). Thalassemia International Federation, 2013

Guidelines for the Management of Non Tranfusion Dependent Thalassemia (NTDT). Thalassemia International Federation, 2013.

Taher A, Vichinsky E, Musallam K, Cappellini MD, Viprakasit V.

Role: