Matthew M. Heeney | Medical Services
Specialties
Programs & Services
- Cancer and Blood Disorders Center
- Rare Anemias and Iron Disorders Program
- Sickle Cell Disease Program
Languages
- English
Matthew M. Heeney | Education
Undergraduate School
Trinity College, University of Toronto
1992, Toronto, Canada
Medical School
University of Calgary
1995, Calgary, Canada
Residency
Montreal Children's Hospital, McGill University
1999, Montreal, Canada
Fellowship
Pediatric Hematology-Oncology
Duke University Medical Center
2002, Durham, NC
Matthew M. Heeney | Certifications
- American Board of Pediatrics (Hematology-Oncology)
Matthew M. Heeney | Professional History
Matthew Heeney, MD, is board certified in pediatrics and pediatric hematology/oncology. He is the Associate Chief for Hematology in the Division of Hematology/Oncology and the Director of the Sickle Cell Program in Dana-Farber/Boston Children’s Cancer and Blood Disorders Center. Dr. Heeney’s research interests are focused on sickle cell disease and congenital disorders of iron metabolism. The goals of Dr. Heeney's research include improving the understanding of the pathophysiology and treatment of sickle cell anemia through clinical investigation and elucidating the genetic basis of iron homeostasis and its role in human disease.
Dr. Heeney has been the Boston Children's Hospital Site Investigator for several clinical trial consortia including the former Sickle Cell Disease Research Network (SCDCRN) and Comprehensive Sickle Cell Center (CSCC). He is also the Site Investigator for the multicenter SWiTCH and TWiTCH trials, which are exploring the use of Hydroxyurea as an alternative to chronic transfusion for stroke prevention in sickle cell disease. He is also involved in early phase trials of prasugrel and regadenoson in sickle cell disease. Dr. Heeney also conducts translational research in inherited disorders of iron homeostasis in humans. In particular, he is interested in investigating the genetic basis inherited disorders of iron deficiency, sideroblastic anemia, and iron overload.
Dr. Heeney is an Associate Professor of Pediatrics at Harvard Medical School (HMS) and is committed to medical education as the Co-Director of the Hematology course for second year HMS students. He is a frequent lecturer on hematology topics in HMS-sponsored continuing medical education and BCH training programs.
Dr. Heeney received his MD at the University of Calgary, Alberta; completed his pediatrics residency at Montreal Children's Hospital, McGill University; and completed a pediatric hematology/oncology fellowship at Duke University.
Matthew M. Heeney | Publications
Screen of the ReFRAME Compound Library for Therapeutic Agents to Prevent Red Blood Cell Sickling Using an Improved High Throughput Sickling Assay. ACS Omega. 2025 Apr 29; 10(16):16497-16505. View Screen of the ReFRAME Compound Library for Therapeutic Agents to Prevent Red Blood Cell Sickling Using an Improved High Throughput Sickling Assay. Abstract
X-linked sideroblastic anemia in females. Blood. 2025 Apr 03; 145(14):1583-1587. View X-linked sideroblastic anemia in females. Abstract
Correlation of Genetic Mutation With Outcomes in Children With Hereditary Spherocytosis Undergoing Partial Splenectomy: A Multicentre Study. J Pediatr Surg. 2025 Apr; 60(4):162229. View Correlation of Genetic Mutation With Outcomes in Children With Hereditary Spherocytosis Undergoing Partial Splenectomy: A Multicentre Study. Abstract
Sustained increase in annual transcranial Doppler screening rates in children with sickle cell disease: A quality improvement project. Pediatr Blood Cancer. 2024 Aug; 71(8):e31088. View Sustained increase in annual transcranial Doppler screening rates in children with sickle cell disease: A quality improvement project. Abstract
The Risk of Kidney Injury in Patients With Sickle Cell Disease Treated With Ketorolac for Acute Pain. J Pediatr Hematol Oncol. 2024 07 01; 46(5):e290-e295. View The Risk of Kidney Injury in Patients With Sickle Cell Disease Treated With Ketorolac for Acute Pain. Abstract
Increasing COVID-19 Vaccination Rates for Children With Sickle Cell Disease. Pediatrics. 2023 Oct 01; 152(4). View Increasing COVID-19 Vaccination Rates for Children With Sickle Cell Disease. Abstract
Ketamine use for management of vaso-occlusive pain in pediatric sickle cell disease. Pediatr Blood Cancer. 2023 05; 70(5):e30254. View Ketamine use for management of vaso-occlusive pain in pediatric sickle cell disease. Abstract
Patient Controlled Analgesia for Vaso-Occlusive Episodes in Children: A Retrospective Study. J Pain Symptom Manage. 2023 05; 65(5):e409-e415. View Patient Controlled Analgesia for Vaso-Occlusive Episodes in Children: A Retrospective Study. Abstract
Sickle Cell Disease and Gene Therapy - Patient and Physician Perspectives. N Engl J Med. 2022 09 29; 387(13):e28. View Sickle Cell Disease and Gene Therapy - Patient and Physician Perspectives. Abstract
Ticagrelor vs placebo for the reduction of vaso-occlusive crises in pediatric sickle cell disease: the HESTIA3 study. Blood. 2022 09 29; 140(13):1470-1481. View Ticagrelor vs placebo for the reduction of vaso-occlusive crises in pediatric sickle cell disease: the HESTIA3 study. Abstract
Consensus definition of essential, optimal, and suggested components of a pediatric sickle cell disease center. Pediatr Blood Cancer. 2023 01; 70(1):e29961. View Consensus definition of essential, optimal, and suggested components of a pediatric sickle cell disease center. Abstract
High-Throughput Assay to Screen Small Molecules for Their Ability to Prevent Sickling of Red Blood Cells. ACS Omega. 2022 Apr 26; 7(16):14009-14016. View High-Throughput Assay to Screen Small Molecules for Their Ability to Prevent Sickling of Red Blood Cells. Abstract
Standardizing Opioid Prescribing in a Pediatric Hospital: A Quality Improvement Effort. Hosp Pediatr. 2022 02 01; 12(2):164-173. View Standardizing Opioid Prescribing in a Pediatric Hospital: A Quality Improvement Effort. Abstract
Belzutifan, a Potent HIF2a Inhibitor, in the Pacak-Zhuang Syndrome. N Engl J Med. 2021 11 25; 385(22):2059-2065. View Belzutifan, a Potent HIF2a Inhibitor, in the Pacak-Zhuang Syndrome. Abstract
Prevalence and Predictors of Iron Deficiency in Adolescent and Young Adult Outpatients: Implications for Screening. Clin Pediatr (Phila). 2022 01; 61(1):66-75. View Prevalence and Predictors of Iron Deficiency in Adolescent and Young Adult Outpatients: Implications for Screening. Abstract
SLC25A38 congenital sideroblastic anemia: Phenotypes and genotypes of 31 individuals from 24 families, including 11 novel mutations, and a review of the literature. Hum Mutat. 2021 11; 42(11):1367-1383. View SLC25A38 congenital sideroblastic anemia: Phenotypes and genotypes of 31 individuals from 24 families, including 11 novel mutations, and a review of the literature. Abstract
A systematic review of ketamine for the management of vaso-occlusive pain in sickle cell disease. Pediatr Blood Cancer. 2021 07; 68(7):e28989. View A systematic review of ketamine for the management of vaso-occlusive pain in sickle cell disease. Abstract
Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease. N Engl J Med. 2021 01 21; 384(3):205-215. View Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease. Abstract
Hydroxyurea Optimization through Precision Study (HOPS): study protocol for a randomized, multicenter trial in children with sickle cell anemia. Trials. 2020 Nov 27; 21(1):983. View Hydroxyurea Optimization through Precision Study (HOPS): study protocol for a randomized, multicenter trial in children with sickle cell anemia. Abstract
Mutations in the iron-sulfur cluster biogenesis protein HSCB cause congenital sideroblastic anemia. J Clin Invest. 2020 10 01; 130(10):5245-5256. View Mutations in the iron-sulfur cluster biogenesis protein HSCB cause congenital sideroblastic anemia. Abstract
Biochemical and therapeutic effects of Omega-3 fatty acids in sickle cell disease. Complement Ther Med. 2020 Aug; 52:102482. View Biochemical and therapeutic effects of Omega-3 fatty acids in sickle cell disease. Abstract
An emerging role for endothelial barrier support therapy for congenital disorders of glycosylation. J Inherit Metab Dis. 2020 07; 43(4):880-890. View An emerging role for endothelial barrier support therapy for congenital disorders of glycosylation. Abstract
Geographic Differences in Phenotype and Treatment of Children with Sickle Cell Anemia from the Multinational DOVE Study. J Clin Med. 2019 Nov 17; 8(11). View Geographic Differences in Phenotype and Treatment of Children with Sickle Cell Anemia from the Multinational DOVE Study. Abstract
Ticagrelor versus placebo for the reduction of vaso-occlusive crises in pediatric sickle cell disease: Rationale and design of a randomized, double-blind, parallel-group, multicenter phase 3 study (HESTIA3). Contemp Clin Trials. 2019 10; 85:105835. View Ticagrelor versus placebo for the reduction of vaso-occlusive crises in pediatric sickle cell disease: Rationale and design of a randomized, double-blind, parallel-group, multicenter phase 3 study (HESTIA3). Abstract
Clinical and laboratory outcomes following total or partial splenectomy in patients with hereditary spherocytosis. Pediatr Hematol Oncol. 2019 Sep; 36(6):382-389. View Clinical and laboratory outcomes following total or partial splenectomy in patients with hereditary spherocytosis. Abstract
Patient- and Nurse-Controlled Analgesia: 22-Year Experience in a Pediatric Hospital. Hosp Pediatr. 2019 02; 9(2):129-133. View Patient- and Nurse-Controlled Analgesia: 22-Year Experience in a Pediatric Hospital. Abstract
Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients. Blood Adv. 2018 10 09; 2(19):2505-2512. View Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients. Abstract
Bronchodilator Use for Acute Chest Syndrome Among Large Pediatric Hospitals in North America. Clin Pediatr (Phila). 2018 12; 57(14):1630-1637. View Bronchodilator Use for Acute Chest Syndrome Among Large Pediatric Hospitals in North America. Abstract
Double-blind, randomized, multicenter phase 2 study of SC411 in children with sickle cell disease (SCOT trial). Blood Adv. 2018 08 14; 2(15):1969-1979. View Double-blind, randomized, multicenter phase 2 study of SC411 in children with sickle cell disease (SCOT trial). Abstract
The phenotypic spectrum of germline YARS2 variants: from isolated sideroblastic anemia to mitochondrial myopathy, lactic acidosis and sideroblastic anemia 2. Haematologica. 2018 12; 103(12):2008-2015. View The phenotypic spectrum of germline YARS2 variants: from isolated sideroblastic anemia to mitochondrial myopathy, lactic acidosis and sideroblastic anemia 2. Abstract
Recurrent heteroplasmy for the MT-ATP6 p.Ser148Asn (m.8969G>A) mutation in patients with syndromic congenital sideroblastic anemia of variable clinical severity. Haematologica. 2018 12; 103(12):e561-e563. View Recurrent heteroplasmy for the MT-ATP6 p.Ser148Asn (m.8969G>A) mutation in patients with syndromic congenital sideroblastic anemia of variable clinical severity. Abstract
Normalizing hepcidin predicts TMPRSS6 mutation status in patients with chronic iron deficiency. Blood. 2018 07 26; 132(4):448-452. View Normalizing hepcidin predicts TMPRSS6 mutation status in patients with chronic iron deficiency. Abstract
A Scientific Renaissance: Novel Drugs in Sickle Cell Disease. Pediatr Clin North Am. 2018 06; 65(3):445-464. View A Scientific Renaissance: Novel Drugs in Sickle Cell Disease. Abstract
The effect of iron chelation therapy on overall survival in sickle cell disease and ß-thalassemia: A systematic review. Am J Hematol. 2018 07; 93(7):943-952. View The effect of iron chelation therapy on overall survival in sickle cell disease and ß-thalassemia: A systematic review. Abstract
Alu element insertion in PKLR gene as a novel cause of pyruvate kinase deficiency in Middle Eastern patients. Hum Mutat. 2018 03; 39(3):389-393. View Alu element insertion in PKLR gene as a novel cause of pyruvate kinase deficiency in Middle Eastern patients. Abstract
Randomized phase 2 trial of regadenoson for treatment of acute vaso-occlusive crises in sickle cell disease. Blood Adv. 2017 Sep 12; 1(20):1645-1649. View Randomized phase 2 trial of regadenoson for treatment of acute vaso-occlusive crises in sickle cell disease. Abstract
Successful utilization of an electronic pain diary in a multinational phase 3 interventional study of pediatric sickle cell anemia. Clin Trials. 2017 Dec; 14(6):563-571. View Successful utilization of an electronic pain diary in a multinational phase 3 interventional study of pediatric sickle cell anemia. Abstract
Sickle Cell Disease. N Engl J Med. 2017 07 20; 377(3):304. View Sickle Cell Disease. Abstract
Neonatal anemia: Revisiting the enigmatic pyknocyte. Am J Hematol. 2017 Jul; 92(7):717-721. View Neonatal anemia: Revisiting the enigmatic pyknocyte. Abstract
Real-time dose adjustment using point-of-care platelet reactivity testing in a double-blind study of prasugrel in children with sickle cell anaemia. Thromb Haemost. 2017 02 28; 117(3):580-588. View Real-time dose adjustment using point-of-care platelet reactivity testing in a double-blind study of prasugrel in children with sickle cell anaemia. Abstract
Ringed sideroblasts in ß-thalassemia. Pediatr Blood Cancer. 2017 05; 64(5). View Ringed sideroblasts in ß-thalassemia. Abstract
Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype. J Clin Invest. 2016 10 03; 126(10):3868-3878. View Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype. Abstract
A recurring mutation in the respiratory complex 1 protein NDUFB11 is responsible for a novel form of X-linked sideroblastic anemia. Blood. 2016 10 13; 128(15):1913-1917. View A recurring mutation in the respiratory complex 1 protein NDUFB11 is responsible for a novel form of X-linked sideroblastic anemia. Abstract
Prasugrel for Sickle Cell Vaso-Occlusive Events. N Engl J Med. 2016 07 14; 375(2):185-6. View Prasugrel for Sickle Cell Vaso-Occlusive Events. Abstract
Newborn Screening for Sickle Cell Disease in Liberia: A Pilot Study. Pediatr Blood Cancer. 2016 Apr; 63(4):671-6. View Newborn Screening for Sickle Cell Disease in Liberia: A Pilot Study. Abstract
A Multinational Trial of Prasugrel for Sickle Cell Vaso-Occlusive Events. N Engl J Med. 2016 Feb 18; 374(7):625-35. View A Multinational Trial of Prasugrel for Sickle Cell Vaso-Occlusive Events. Abstract
Hydroxycarbamide versus chronic transfusion for maintenance of transcranial doppler flow velocities in children with sickle cell anaemia-TCD With Transfusions Changing to Hydroxyurea (TWiTCH): a multicentre, open-label, phase 3, non-inferiority trial. Lancet. 2016 Feb 13; 387(10019):661-670. View Hydroxycarbamide versus chronic transfusion for maintenance of transcranial doppler flow velocities in children with sickle cell anaemia-TCD With Transfusions Changing to Hydroxyurea (TWiTCH): a multicentre, open-label, phase 3, non-inferiority trial. Abstract
Hematologic outcomes after total splenectomy and partial splenectomy for congenital hemolytic anemia. J Pediatr Surg. 2016 Jan; 51(1):122-7. View Hematologic outcomes after total splenectomy and partial splenectomy for congenital hemolytic anemia. Abstract
Congenital sideroblastic anemia due to mutations in the mitochondrial HSP70 homologue HSPA9. Blood. 2015 Dec 17; 126(25):2734-8. View Congenital sideroblastic anemia due to mutations in the mitochondrial HSP70 homologue HSPA9. Abstract
Design of the DOVE (Determining Effects of Platelet Inhibition on Vaso-Occlusive Events) trial: A global Phase 3 double-blind, randomized, placebo-controlled, multicenter study of the efficacy and safety of prasugrel in pediatric patients with sickle cell anemia utilizing a dose titration strategy. Pediatr Blood Cancer. 2016 Feb; 63(2):299-305. View Design of the DOVE (Determining Effects of Platelet Inhibition on Vaso-Occlusive Events) trial: A global Phase 3 double-blind, randomized, placebo-controlled, multicenter study of the efficacy and safety of prasugrel in pediatric patients with sickle cell anemia utilizing a dose titration strategy. Abstract
Liver iron concentration measurements by MRI in chronically transfused children with sickle cell anemia: baseline results from the TWiTCH trial. Am J Hematol. 2015 Sep; 90(9):806-10. View Liver iron concentration measurements by MRI in chronically transfused children with sickle cell anemia: baseline results from the TWiTCH trial. Abstract
Therapeutic phlebotomy is safe in children with sickle cell anaemia and can be effective treatment for transfusional iron overload. Br J Haematol. 2015 Apr; 169(2):262-6. View Therapeutic phlebotomy is safe in children with sickle cell anaemia and can be effective treatment for transfusional iron overload. Abstract
Prasugrel in children with sickle cell disease: pharmacokinetic and pharmacodynamic data from an open-label, adaptive-design, dose-ranging study. J Pediatr Hematol Oncol. 2015 Jan; 37(1):1-9. View Prasugrel in children with sickle cell disease: pharmacokinetic and pharmacodynamic data from an open-label, adaptive-design, dose-ranging study. Abstract
Clinical outcomes of splenectomy in children: report of the splenectomy in congenital hemolytic anemia registry. Am J Hematol. 2015 Mar; 90(3):187-92. View Clinical outcomes of splenectomy in children: report of the splenectomy in congenital hemolytic anemia registry. Abstract
Iron clad: iron homeostasis and the diagnosis of hereditary iron overload. Hematology Am Soc Hematol Educ Program. 2014 Dec 05; 2014(1):202-9. View Iron clad: iron homeostasis and the diagnosis of hereditary iron overload. Abstract
Mutations in TRNT1 cause congenital sideroblastic anemia with immunodeficiency, fevers, and developmental delay (SIFD). Blood. 2014 Oct 30; 124(18):2867-71. View Mutations in TRNT1 cause congenital sideroblastic anemia with immunodeficiency, fevers, and developmental delay (SIFD). Abstract
Iron disorders. Hematol Oncol Clin North Am. 2014 Aug; 28(4):ix-x. View Iron disorders. Abstract
Magnetic resonance imaging/angiography and transcranial Doppler velocities in sickle cell anemia: results from the SWiTCH trial. Blood. 2014 Aug 07; 124(6):891-8. View Magnetic resonance imaging/angiography and transcranial Doppler velocities in sickle cell anemia: results from the SWiTCH trial. Abstract
Iron-refractory iron deficiency anemia (IRIDA). Hematol Oncol Clin North Am. 2014 Aug; 28(4):637-52, v. View Iron-refractory iron deficiency anemia (IRIDA). Abstract
X-linked sideroblastic anemia due to ALAS2 intron 1 enhancer element GATA-binding site mutations. Am J Hematol. 2014 Mar; 89(3):315-9. View X-linked sideroblastic anemia due to ALAS2 intron 1 enhancer element GATA-binding site mutations. Abstract
Systemic corticosteroids in acute chest syndrome: friend or foe? Paediatr Respir Rev. 2014 Mar; 15(1):24-7. View Systemic corticosteroids in acute chest syndrome: friend or foe? Abstract
Pain and other non-neurological adverse events in children with sickle cell anemia and previous stroke who received hydroxyurea and phlebotomy or chronic transfusions and chelation: results from the SWiTCH clinical trial. Am J Hematol. 2013 Nov; 88(11):932-8. View Pain and other non-neurological adverse events in children with sickle cell anemia and previous stroke who received hydroxyurea and phlebotomy or chronic transfusions and chelation: results from the SWiTCH clinical trial. Abstract
Bacteremia risk and outpatient management of febrile patients with sickle cell disease. Pediatrics. 2013 Jun; 131(6):1035-41. View Bacteremia risk and outpatient management of febrile patients with sickle cell disease. Abstract
A novel syndrome of congenital sideroblastic anemia, B-cell immunodeficiency, periodic fevers, and developmental delay (SIFD). Blood. 2013 Jul 04; 122(1):112-23. View A novel syndrome of congenital sideroblastic anemia, B-cell immunodeficiency, periodic fevers, and developmental delay (SIFD). Abstract
Platelet activation and inhibition in sickle cell disease (pains) study. Platelets. 2014; 25(1):27-35. View Platelet activation and inhibition in sickle cell disease (pains) study. Abstract
Weight status of children with sickle cell disease. Pediatrics. 2013 Apr; 131(4):e1168-73. View Weight status of children with sickle cell disease. Abstract
Hydroxyurea for the treatment of sickle cell disease: efficacy, barriers, toxicity, and management in children. Pediatr Blood Cancer. 2012 Aug; 59(2):365-71. View Hydroxyurea for the treatment of sickle cell disease: efficacy, barriers, toxicity, and management in children. Abstract
Stroke With Transfusions Changing to Hydroxyurea (SWiTCH). Blood. 2012 Apr 26; 119(17):3925-32. View Stroke With Transfusions Changing to Hydroxyurea (SWiTCH). Abstract
Effects of chronic transfusions on abdominal sonographic abnormalities in children with sickle cell anemia. J Pediatr. 2012 Feb; 160(2):281-285.e1. View Effects of chronic transfusions on abdominal sonographic abnormalities in children with sickle cell anemia. Abstract
Loss-of-function and gain-of-function phenotypes of stomatocytosis mutant RhAG F65S. Am J Physiol Cell Physiol. 2011 Dec; 301(6):C1325-43. View Loss-of-function and gain-of-function phenotypes of stomatocytosis mutant RhAG F65S. Abstract
Thirty-day readmission rates following hospitalization for pediatric sickle cell crisis at freestanding children's hospitals: risk factors and hospital variation. Pediatr Blood Cancer. 2012 Jan; 58(1):61-5. View Thirty-day readmission rates following hospitalization for pediatric sickle cell crisis at freestanding children's hospitals: risk factors and hospital variation. Abstract
Transition from pediatric to adult care for sickle cell disease: results of a survey of pediatric providers. Am J Hematol. 2011 Jun; 86(6):512-5. View Transition from pediatric to adult care for sickle cell disease: results of a survey of pediatric providers. Abstract
Long-term safety and efficacy of deferasirox (Exjade) for up to 5 years in transfusional iron-overloaded patients with sickle cell disease. Br J Haematol. 2011 Aug; 154(3):387-97. View Long-term safety and efficacy of deferasirox (Exjade) for up to 5 years in transfusional iron-overloaded patients with sickle cell disease. Abstract
Rudolph’s Pediatrics. Iron Deficiency Anemia: Definition, Pathophysiology and Classification. 2011.
Rudolph’s Pediatrics (Rudolph AM, Rudolph C, First L, Lister G, Gershon AA. Editors). Anemia: Definition, Pathophysiology and Classification. 2011.
Common variants at 10 genomic loci influence hemoglobin A1(C) levels via glycemic and nonglycemic pathways. Diabetes. 2010 Dec; 59(12):3229-39. View Common variants at 10 genomic loci influence hemoglobin A1(C) levels via glycemic and nonglycemic pathways. Abstract
Encephalopathy from lead poisoning masquerading as a flu-like syndrome in an autistic child. Pediatr Emerg Care. 2010 May; 26(5):370-3. View Encephalopathy from lead poisoning masquerading as a flu-like syndrome in an autistic child. Abstract
High-throughput matrix-assisted laser desorption ionization-time-of-flight mass spectrometry method for quantification of hepcidin in human urine. Anal Chem. 2010 Feb 15; 82(4):1551-5. View High-throughput matrix-assisted laser desorption ionization-time-of-flight mass spectrometry method for quantification of hepcidin in human urine. Abstract
Hydroxyurea for children with sickle cell disease. Hematol Oncol Clin North Am. 2010 Feb; 24(1):199-214. View Hydroxyurea for children with sickle cell disease. Abstract
Resolution of cerebral artery stenosis in a child with sickle cell anemia treated with hydroxyurea. Am J Hematol. 2010 Feb; 85(2):135-7. View Resolution of cerebral artery stenosis in a child with sickle cell anemia treated with hydroxyurea. Abstract
Definitions of the phenotypic manifestations of sickle cell disease. Am J Hematol. 2010 Jan; 85(1):6-13. View Definitions of the phenotypic manifestations of sickle cell disease. Abstract
Corticosteroids for acute chest syndrome in children with sickle cell disease: variation in use and association with length of stay and readmission. Am J Hematol. 2010 Jan; 85(1):24-8. View Corticosteroids for acute chest syndrome in children with sickle cell disease: variation in use and association with length of stay and readmission. Abstract
Pial synangiosis in patients with moyamoya syndrome and sickle cell anemia: perioperative management and surgical outcome. Neurosurg Focus. 2009 Apr; 26(4):E10. View Pial synangiosis in patients with moyamoya syndrome and sickle cell anemia: perioperative management and surgical outcome. Abstract
Clinical practice guideline improves the treatment of sickle cell disease vasoocclusive pain. Pediatr Blood Cancer. 2009 Mar; 52(3):369-72. View Clinical practice guideline improves the treatment of sickle cell disease vasoocclusive pain. Abstract
Disorders of Hemoglobin: Genetics, Pathophysiology, and Clinical Management (Steinberg MH, Forget BG, Higgs DR, Weatherall DJ. Editors). Clinical and Pathophysiological Aspects of Sickle Cell Anemia. 2009; 437-496.
Cefepime-induced neutropenia in a teenager. Pediatr Blood Cancer. 2008 Nov; 51(5):715-6. View Cefepime-induced neutropenia in a teenager. Abstract
Mutations in TMPRSS6 cause iron-refractory iron deficiency anemia (IRIDA). Nat Genet. 2008 May; 40(5):569-71. View Mutations in TMPRSS6 cause iron-refractory iron deficiency anemia (IRIDA). Abstract
Hydroxyurea for children with sickle cell disease. Pediatr Clin North Am. 2008 Apr; 55(2):483-501, x. View Hydroxyurea for children with sickle cell disease. Abstract
Chronic transfusion practice for children with sickle cell anaemia and stroke. Br J Haematol. 2009 May; 145(4):524-8. View Chronic transfusion practice for children with sickle cell anaemia and stroke. Abstract
Nathan and Oski’s Hematology of Infancy and Childhood (Nathan DG, Orkin SH, Ginsburg D, Look AT. Editors). Sickle Cell Disease. 2008; 949-1014.
Preventing stroke among children with sickle cell anemia: an analysis of strategies that involve transcranial Doppler testing and chronic transfusion. Pediatrics. 2007 Oct; 120(4):e1107-16. View Preventing stroke among children with sickle cell anemia: an analysis of strategies that involve transcranial Doppler testing and chronic transfusion. Abstract
Sickle cell disease caused by Hb S/Québec-CHORI: treatment with hydroxyurea and response. Pediatr Blood Cancer. 2007 Aug; 49(2):207-10. View Sickle cell disease caused by Hb S/Québec-CHORI: treatment with hydroxyurea and response. Abstract
Hemoglobin SE disease: a concise review. Am J Hematol. 2007 Jul; 82(7):643-9. View Hemoglobin SE disease: a concise review. Abstract
Acquired immune cytopenias post-cardiac transplantation respond to rituximab. Pediatr Blood Cancer. 2007 Mar; 48(3):339-44. View Acquired immune cytopenias post-cardiac transplantation respond to rituximab. Abstract
Management of Sickle Cell Disease,. Comprehensive Pediatric Hospital Medicine (Zaoutis LD, Chiang VW). 2007; 731-737.
Hematologic Disorders. Manual of Pediatric Therapeutics (Greenes DS, Graeff JW.). 2007; 466-499.
Hemoglobinopathies mimicking Hb S/beta-thalassemia: Hb S/S with alpha-thalassemia and Hb S/Volga. Am J Hematol. 2006 May; 81(5):361-5. View Hemoglobinopathies mimicking Hb S/beta-thalassemia: Hb S/S with alpha-thalassemia and Hb S/Volga. Abstract
Risk of cholelithiasis in sickle cell anemia. Haematologica. 2005 Feb; 90(2):147. View Risk of cholelithiasis in sickle cell anemia. Abstract
Iron homeostasis and inherited iron overload disorders: an overview. Hematol Oncol Clin North Am. 2004 Dec; 18(6):1379-403, ix. View Iron homeostasis and inherited iron overload disorders: an overview. Abstract
Chemical and functional analysis of hydroxyurea oral solutions. J Pediatr Hematol Oncol. 2004 Mar; 26(3):179-84. View Chemical and functional analysis of hydroxyurea oral solutions. Abstract
Childhood autoimmune cytopenia secondary to unsuspected common variable immunodeficiency. J Pediatr. 2003 Nov; 143(5):662-5. View Childhood autoimmune cytopenia secondary to unsuspected common variable immunodeficiency. Abstract
UGT1A promoter polymorphisms influence bilirubin response to hydroxyurea therapy in sickle cell anemia. J Lab Clin Med. 2003 Apr; 141(4):279-82. View UGT1A promoter polymorphisms influence bilirubin response to hydroxyurea therapy in sickle cell anemia. Abstract
Increased expression of anti-apoptosis genes in peripheral blood cells from patients with paroxysmal nocturnal hemoglobinuria. Mol Genet Metab. 2003 Apr; 78(4):291-4. View Increased expression of anti-apoptosis genes in peripheral blood cells from patients with paroxysmal nocturnal hemoglobinuria. Abstract
Diagnostic Approach to Sickle Hemoglobinopathies. Practical Algorithms in Pediatric Hematology-Oncology (Sills R). 2003.
Newborn Screening for Hemoglobinopathies. Practical Algorithms in Pediatric Hematology Oncology (Sills R.). 2003.
A multi-step model for the pathogenesis and evolution of PNH. Paroxysmal Nocturnal Hemoglobinuria (PNH) and Related Disorders. 2003; 41-54.
Interpretation of fetal hemoglobin only on newborn screening for hemoglobinopathy. J Pediatr Hematol Oncol. 2002 Aug-Sep; 24(6):499-502. View Interpretation of fetal hemoglobin only on newborn screening for hemoglobinopathy. Abstract
Distinct stage-specific cis-active transcriptional mechanisms control expression of T cell coreceptor CD8 alpha at double- and single-positive stages of thymic development. J Immunol. 1998 Sep 01; 161(5):2254-66. View Distinct stage-specific cis-active transcriptional mechanisms control expression of T cell coreceptor CD8 alpha at double- and single-positive stages of thymic development. Abstract
A 150-base pair 5' region of the MHC class I HLA-B7 gene is sufficient to direct tissue-specific expression and locus control region activity: the alpha site determines efficient expression and in vivo occupancy at multiple cis-active sites throughout this region. J Immunol. 1997 Nov 15; 159(10):4913-29. View A 150-base pair 5' region of the MHC class I HLA-B7 gene is sufficient to direct tissue-specific expression and locus control region activity: the alpha site determines efficient expression and in vivo occupancy at multiple cis-active sites throughout this region. Abstract