selected publications
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Safety and pharmacokinetics of the oral iron chelator SP-420 in β-thalassemia.
American journal of hematology.
2017
Academic Article
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Times cited: 7 -
Evidence for multidimensional resilience in adult patients with transfusion-dependent thalassemias: Is it more common than we think?.
Transfusion medicine (Oxford, England).
2016
Academic Article
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Times cited: 2 -
Crucial role for the VWF A1 domain in binding to type IV collagen.
Blood.
2015
Academic Article
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Times cited: 79 -
Mutations in TRNT1 cause congenital sideroblastic anemia with immunodeficiency, fevers, and developmental delay (SIFD).
Blood.
2014
Academic Article
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Times cited: 146 -
Contextually appropriate emotional word use predicts adaptive health behavior: Emotion context sensitivity and treatment adherence.
Journal of health psychology.
2014
Academic Article
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Times cited: 15 -
Red blood cell transfusions for thalassemia: results of a survey assessing current practice and proposal of evidence-based guidelines.
Transfusion.
2014
Review
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Times cited: 23 -
Negative affect differentiation and adherence during treatment for thalassemia.
International journal of behavioral medicine.
2014
Academic Article
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Times cited: 10 -
Safe mobilization of CD34+ cells in adults with β-thalassemia and validation of effective globin gene transfer for clinical investigation.
Blood.
2014
Academic Article
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Times cited: 58 -
Transfusion complications in thalassemia patients: a report from the Centers for Disease Control and Prevention (CME).
Transfusion.
2013
Academic Article
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Times cited: 106 -
Pregnancy outcomes in women with thalassemia in North America and the United Kingdom.
American journal of hematology.
2013
Academic Article
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Times cited: 22 -
Longitudinal monitoring of cardiac siderosis using cardiovascular magnetic resonance T2* in patients with thalassemia major on various chelation regimens: a 6-year study.
American journal of hematology.
2013
Academic Article
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Times cited: 12 -
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.
2013
Academic Article
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Times cited: 44 -
The palatability and tolerability of deferasirox taken with different beverages or foods.
Pediatric blood & cancer.
2013
Academic Article
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Times cited: 18 -
Unreliable oral glucose tolerance test and haemoglobin A1C in beta thalassaemia major--a case for continuous glucose monitoring?.
British journal of haematology.
2013
Letter
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Times cited: 18 -
A novel syndrome of congenital sideroblastic anemia, B-cell immunodeficiency, periodic fevers, and developmental delay (SIFD).
Blood.
2013
Academic Article
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Times cited: 93 -
Macrophages support pathological erythropoiesis in polycythemia vera and β-thalassemia.
Nature medicine.
2013
Academic Article
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Times cited: 188 -
Human T cell lymphotropic virus type 1 infection among U.S. thalassemia patients.
AIDS research and human retroviruses.
2013
Academic Article
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Times cited: 4 -
Candidate sequence variants and fetal hemoglobin in children with sickle cell disease treated with hydroxyurea.
PloS one.
2013
Academic Article
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Times cited: 26 -
Increased leucocyte apoptosis in transfused β-thalassaemia patients.
British journal of haematology.
2012
Academic Article
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Times cited: 7 -
Parental and other factors associated with hydroxyurea use for pediatric sickle cell disease.
Pediatric blood & cancer.
2012
Academic Article
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Times cited: 58 -
Toward optimizing the use of deferasirox: potential benefits of combined use with deferoxamine.
Haematologica.
2012
Academic Article
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Times cited: 34 -
Therapeutic hemoglobin levels after gene transfer in β-thalassemia mice and in hematopoietic cells of β-thalassemia and sickle cells disease patients.
PloS one.
2012
Academic Article
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Times cited: 81 -
Managing incidentally diagnosed isolated factor VII deficiency perioperatively: a brief expert consensus report.
Expert review of hematology.
2012
Academic Article
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Times cited: 6 -
Chelation use and iron burden in North American and British thalassemia patients: a report from the Thalassemia Longitudinal Cohort.
Blood.
2012
Academic Article
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Times cited: 74 -
How I treat thalassemia.
Blood.
2011
Review
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Times cited: 222 -
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.
2011
Academic Article
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Times cited: 53 -
Iron chelation adherence to deferoxamine and deferasirox in thalassemia.
American journal of hematology.
2011
Academic Article
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Times cited: 65 -
Relationship between labile plasma iron, liver iron concentration and cardiac response in a deferasirox monotherapy trial.
Haematologica.
2011
Academic Article
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Times cited: 35 -
Renal dysfunction in patients with thalassaemia.
British journal of haematology.
2011
Academic Article
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Times cited: 94 -
Red cell alloimmunization in a diverse population of transfused patients with thalassaemia.
British journal of haematology.
2011
Academic Article
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Times cited: 96 -
Genomic safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells.
Nature biotechnology.
2010
Academic Article
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Times cited: 264 -
Hepcidin as a therapeutic tool to limit iron overload and improve anemia in β-thalassemic mice.
The Journal of clinical investigation.
2010
Academic Article
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Times cited: 192 -
Education and employment status of children and adults with thalassemia in North America.
Pediatric blood & cancer.
2010
Academic Article
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Times cited: 13 -
Bone loss caused by iron overload in a murine model: importance of oxidative stress.
Blood.
2010
Academic Article
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Times cited: 273 -
Relationship between chronic transfusion therapy and body composition in subjects with thalassemia.
The Journal of pediatrics.
2010
Academic Article
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Times cited: 37 -
Changes in bone microarchitecture and biomechanical properties in the th3 thalassemia mouse are associated with decreased bone turnover and occur during the period of bone accrual.
Calcified tissue international.
2010
Academic Article
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Times cited: 26 -
The effect of deferasirox on cardiac iron in thalassemia major: impact of total body iron stores.
Blood.
2010
Academic Article
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Times cited: 108 -
The significance of a positive DAT in thalassemia patients.
Immunohematology.
2010
Academic Article
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Times cited: 3 -
Differences in the prevalence of growth, endocrine and vitamin D abnormalities among the various thalassaemia syndromes in North America.
British journal of haematology.
2009
Academic Article
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Times cited: 135 -
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.
2009
Academic Article
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Times cited: 197 -
Safety and efficacy of pegylated interferon alpha-2a and ribavirin for the treatment of hepatitis C in patients with thalassemia.
Haematologica.
2008
Academic Article
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Times cited: 42 -
Disparity in the management of iron overload between patients with sickle cell disease and thalassemia who received transfusions.
Transfusion.
2008
Academic Article
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Times cited: 29 -
Decreased differentiation of erythroid cells exacerbates ineffective erythropoiesis in beta-thalassemia.
Blood.
2008
Academic Article
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Times cited: 142 -
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.
2008
Academic Article
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Times cited: 62 -
Fracture prevalence and relationship to endocrinopathy in iron overloaded patients with sickle cell disease and thalassemia.
Bone.
2008
Academic Article
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Times cited: 63 -
Relative response of patients with myelodysplastic syndromes and other transfusion-dependent anaemias to deferasirox (ICL670): a 1-yr prospective study.
European journal of haematology.
2007
Academic Article
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Times cited: 218 -
Phase Ib clinical trial of starch-conjugated deferoxamine (40SD02): a novel long-acting iron chelator.
British journal of haematology.
2007
Academic Article
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Times cited: 33 -
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.
2007
Academic Article
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Times cited: 103 -
Ineffective erythropoiesis in beta-thalassemia is characterized by increased iron absorption mediated by down-regulation of hepcidin and up-regulation of ferroportin.
Blood.
2007
Academic Article
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Times cited: 270 -
Therapeutic options for patients with severe beta-thalassemia: the need for globin gene therapy.
Human gene therapy.
2007
Review
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Times cited: 40 -
A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia.
Blood.
2005
Academic Article
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Times cited: 554 -
Prevalence of fractures among the Thalassemia syndromes in North America.
Bone.
2005
Academic Article
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Times cited: 87 -
Low bone mass in prepubertal children with thalassemia major: insights into the pathogenesis of low bone mass in thalassemia.
Journal of pediatric endocrinology & metabolism : JPEM.
2004
Academic Article
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Times cited: 40 -
Effectiveness and safety of ICL670 in iron-loaded patients with thalassaemia: a randomised, double-blind, placebo-controlled, dose-escalation trial.
Lancet (London, England).
2003
Academic Article
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Times cited: 288 -
A novel beta-thalassemia intermedia phenotype containing Nt494+129T-->C and NT494+132C-->A mutations in cis and a Nt168C-->T (beta(o) 39 point) mutation in trans.
American journal of hematology.
2001
Letter
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Times cited: 2 -
Effect of transfusional iron overload on immune response.
The Journal of infectious diseases.
2000
Academic Article
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Times cited: 60 -
Bone marrow transplantation for homozygous beta-thalassemia. The Memorial Sloan-Kettering Cancer Center experience.
Annals of the New York Academy of Sciences.
1998
Academic Article
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Times cited: 34 -
Survival and morbidity in transfusion-dependent thalassemic patients on subcutaneous desferrioxamine chelation. Nearly two decades of experience.
Annals of the New York Academy of Sciences.
1998
Academic Article
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Times cited: 21 -
Microchimerism and tolerance following intrauterine transplantation and transfusion for alpha-thalassemia-1.
Fetal diagnosis and therapy.
1998
Academic Article
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Times cited: 54 -
Serum ferritin iron, a new test, measures human body iron stores unconfounded by inflammation.
Stem cells (Dayton, Ohio).
1997
Academic Article
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Times cited: 63 -
The changing profile of homozygous beta-thalassemia: demography, ethnicity, and age distribution of current North American patients and changes in two decades.
Pediatrics.
1996
Academic Article
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Times cited: 33 -
Chelation therapy in beta-thalassemia: the benefits and limitations of desferrioxamine.
Seminars in hematology.
1995
Review
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Times cited: 73 -
Oral sodium phenylbutyrate therapy in homozygous beta thalassemia: a clinical trial.
Blood.
1995
Academic Article
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Times cited: 232 -
Pulmonary function abnormalities in thalassemia major and the role of iron overload.
American journal of respiratory and critical care medicine.
1994
Academic Article
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Times cited: 55 -
Behavioral contracting to improve adherence in patients with thalassemia.
Journal of pediatric nursing.
1993
Academic Article
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Times cited: 14 -
The biology of stem cell factor, a new hematopoietic growth factor involved in stem cell regulation.
International journal of clinical & laboratory research.
1993
Review
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Times cited: 11 -
Long-term generation of colony-forming cells in liquid culture of CD34+ cord blood cells in the presence of recombinant human stem cell factor.
Blood.
1992
Academic Article
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Times cited: 133 -
Update on thalassemia.
Pediatrics in review.
1992
Review
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Times cited: 11 -
Sickle and thalassemic erythroid progenitor cells are different from normal.
Hemoglobin.
1992
Academic Article
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Times cited: 8 -
Effects of recombinant human stem cell factor (SCF) on the growth of human progenitor cells in vitro.
Journal of cellular physiology.
1991
Academic Article
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Times cited: 62 -
Prolonged survival in patients with beta-thalassemia major treated with deferoxamine.
The Journal of pediatrics.
1991
Academic Article
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Times cited: 102 -
Deferoxamine-induced bone dysplasia in patients with thalassemia major.
AJR. American journal of roentgenology.
1991
Academic Article
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Times cited: 85 -
Experimental approach to the study of immune function in children with possible human immunodeficiency virus infection.
Journal of clinical laboratory analysis.
1990
Academic Article
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Times cited: 1 -
Beta-thalassemia due to two novel nucleotide substitutions in consensus acceptor splice sequences of the beta-globin gene.
Blood.
1989
Academic Article
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Times cited: 60 -
Modulation of the defective natural killer activity seen in thalassaemia major with desferrioxamine and alpha-interferon.
Clinical and experimental immunology.
1987
Academic Article
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Times cited: 11 -
HTLV-III neutralizing antibody development in transfusion-dependent seropositive patients with beta-thalassemia.
Journal of immunology (Baltimore, Md. : 1950).
1987
Academic Article
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Times cited: 27 -
Transfusion-related immunologic abnormalities in beta-thalassemia major.
Birth defects original article series.
1987
Academic Article
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Times cited: 7 -
Decreased natural killer activity in thalassemia major: a possible consequence of iron overload.
Journal of immunology (Baltimore, Md. : 1950).
1986
Academic Article
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Times cited: 46 -
Immunological abnormalities in thalassaemia major. I. A transfusion-related increase in circulating cytoplasmic immunoglobulin-positive cells.
Clinical and experimental immunology.
1985
Academic Article
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Times cited: 12 -
Disproportionate lymphoid cell subsets in thalassaemia major: the relative contributions of transfusion and splenectomy.
British journal of haematology.
1985
Academic Article
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Times cited: 59 -
A psychosocial needs assessment of patients with homozygous beta-thalassemia.
Annals of the New York Academy of Sciences.
1985
Academic Article
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Times cited: 15 -
Beta thalassemia and heart disease: three decades of gradual progress.
Transactions of the American Clinical and Climatological Association.
1985
Academic Article
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Times cited: 4 -
The effect of subcutaneous deferoxamine on the cardiac profile of thalassemia major: a five-year study.
Annals of the New York Academy of Sciences.
1985
Academic Article
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Times cited: 17 - beta-Thalassemia in Chinese: use of in vivo RNA analysis and oligonucleotide hybridization in systematic characterization of molecular defects. Proceedings of the National Academy of Sciences of the United States of America. 1984 Academic Article GET IT
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ATA box transcription mutation in beta-thalassemia.
Nucleic acids research.
1983
Academic Article
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Times cited: 75 - Study of A- and B-cell function in beta-thalassemia major. Acta diabetologica latina. 1983 Academic Article GET IT
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Linkage of beta-thalassaemia mutations and beta-globin gene polymorphisms with DNA polymorphisms in human beta-globin gene cluster.
Nature.
1982
Academic Article
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Times cited: 680 -
Nonrandom association of polymorphic restriction sites in the beta-globin gene cluster.
Proceedings of the National Academy of Sciences of the United States of America.
1982
Academic Article
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Times cited: 269 -
Chelation therapy in beta-thalassemia major. III. The role of splenectomy in achieving iron balance.
The Journal of pediatrics.
1981
Academic Article
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Times cited: 39 -
Thrombocytosis and increased circulating platelet aggregates in newborn infants of polydrug users.
The Journal of pediatrics.
1979
Academic Article
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Times cited: 21 -
Liver dysfunction in patients with hemophilia A, B, and von Willebrand's disease.
Transfusion.
1977
Academic Article
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Times cited: 17 -
Chronic lymphocytic leukaemia. Clinical studies based on 189 cases followed for a long time.
Scandinavian journal of haematology. Supplementum.
1973
Academic Article
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Times cited: 81