Information

Related Research Units

Dana Farber Boston Childrens Cancer and Blood Disorders Center Research
Hematology and Oncology Research

Research Overview

Dr. Heeney conducts clinical research focused on sickle cell disorders and their treatment. He has been the Children's Hospital Boston Principal Investigator for several clinical trial consortia including the former Boston Sickle Cell Disease Research Network (SCDCRN), and Boston 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. 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.

Goals of Dr. Heeney's work include: to improve the understanding of the pathophysiology and treatment of sickle cell anemia through multicenter clinical trials and to elucidate the genetic basis of iron homeostasis and its role in human disease.

Research Background

Dr. Heeney received his MD at the University of Calgary, Alberta, completed his residency at Montreal Children's Hospital, McGill University, and a pediatric hemotology/oncology fellowship at Duke University.

 

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

Publications

  1. 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 Abstract
  2. 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 Abstract
  3. Increasing COVID-19 Vaccination Rates for Children With Sickle Cell Disease. Pediatrics. 2023 Oct 01; 152(4). View Abstract
  4. Ketamine use for management of vaso-occlusive pain in pediatric sickle cell disease. Pediatr Blood Cancer. 2023 05; 70(5):e30254. View Abstract
  5. Patient Controlled Analgesia for Vaso-Occlusive Episodes in Children: A Retrospective Study. J Pain Symptom Manage. 2023 05; 65(5):e409-e415. View Abstract
  6. Sickle Cell Disease and Gene Therapy - Patient and Physician Perspectives. N Engl J Med. 2022 09 29; 387(13):e28. View Abstract
  7. 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 Abstract
  8. Consensus definition of essential, optimal, and suggested components of a pediatric sickle cell disease center. Pediatr Blood Cancer. 2023 01; 70(1):e29961. View Abstract
  9. 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 Abstract
  10. Standardizing Opioid Prescribing in a Pediatric Hospital: A Quality Improvement Effort. Hosp Pediatr. 2022 02 01; 12(2):164-173. View Abstract
  11. Belzutifan, a Potent HIF2a Inhibitor, in the Pacak-Zhuang Syndrome. N Engl J Med. 2021 11 25; 385(22):2059-2065. View Abstract
  12. 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 Abstract
  13. 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 Abstract
  14. 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 Abstract
  15. Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease. N Engl J Med. 2021 01 21; 384(3):205-215. View Abstract
  16. 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 Abstract
  17. Mutations in the iron-sulfur cluster biogenesis protein HSCB cause congenital sideroblastic anemia. J Clin Invest. 2020 10 01; 130(10):5245-5256. View Abstract
  18. Biochemical and therapeutic effects of Omega-3 fatty acids in sickle cell disease. Complement Ther Med. 2020 Aug; 52:102482. View Abstract
  19. An emerging role for endothelial barrier support therapy for congenital disorders of glycosylation. J Inherit Metab Dis. 2020 07; 43(4):880-890. View Abstract
  20. 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 Abstract
  21. 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 Abstract
  22. Clinical and laboratory outcomes following total or partial splenectomy in patients with hereditary spherocytosis. Pediatr Hematol Oncol. 2019 Sep; 36(6):382-389. View Abstract
  23. Patient- and Nurse-Controlled Analgesia: 22-Year Experience in a Pediatric Hospital. Hosp Pediatr. 2019 02; 9(2):129-133. View Abstract
  24. 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 Abstract
  25. Bronchodilator Use for Acute Chest Syndrome Among Large Pediatric Hospitals in North America. Clin Pediatr (Phila). 2018 12; 57(14):1630-1637. View Abstract
  26. 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 Abstract
  27. 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 Abstract
  28. 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 Abstract
  29. Normalizing hepcidin predicts TMPRSS6 mutation status in patients with chronic iron deficiency. Blood. 2018 07 26; 132(4):448-452. View Abstract
  30. A Scientific Renaissance: Novel Drugs in Sickle Cell Disease. Pediatr Clin North Am. 2018 06; 65(3):445-464. View Abstract
  31. 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 Abstract
  32. 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 Abstract
  33. 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 Abstract
  34. 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 Abstract
  35. Sickle Cell Disease. N Engl J Med. 2017 07 20; 377(3):304. View Abstract
  36. Neonatal anemia: Revisiting the enigmatic pyknocyte. Am J Hematol. 2017 Jul; 92(7):717-721. View Abstract
  37. 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 Abstract
  38. Ringed sideroblasts in ß-thalassemia. Pediatr Blood Cancer. 2017 05; 64(5). View Abstract
  39. Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype. J Clin Invest. 2016 10 03; 126(10):3868-3878. View Abstract
  40. 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 Abstract
  41. Prasugrel for Sickle Cell Vaso-Occlusive Events. N Engl J Med. 2016 07 14; 375(2):185-6. View Abstract
  42. Newborn Screening for Sickle Cell Disease in Liberia: A Pilot Study. Pediatr Blood Cancer. 2016 Apr; 63(4):671-6. View Abstract
  43. A Multinational Trial of Prasugrel for Sickle Cell Vaso-Occlusive Events. N Engl J Med. 2016 Feb 18; 374(7):625-35. View Abstract
  44. 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 Abstract
  45. Hematologic outcomes after total splenectomy and partial splenectomy for congenital hemolytic anemia. J Pediatr Surg. 2016 Jan; 51(1):122-7. View Abstract
  46. Congenital sideroblastic anemia due to mutations in the mitochondrial HSP70 homologue HSPA9. Blood. 2015 Dec 17; 126(25):2734-8. View Abstract
  47. 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 Abstract
  48. 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 Abstract
  49. 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 Abstract
  50. 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 Abstract
  51. 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 Abstract
  52. 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 Abstract
  53. Mutations in TRNT1 cause congenital sideroblastic anemia with immunodeficiency, fevers, and developmental delay (SIFD). Blood. 2014 Oct 30; 124(18):2867-71. View Abstract
  54. Iron disorders. Hematol Oncol Clin North Am. 2014 Aug; 28(4):ix-x. View Abstract
  55. 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 Abstract
  56. Iron-refractory iron deficiency anemia (IRIDA). Hematol Oncol Clin North Am. 2014 Aug; 28(4):637-52, v. View Abstract
  57. 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 Abstract
  58. Systemic corticosteroids in acute chest syndrome: friend or foe? Paediatr Respir Rev. 2014 Mar; 15(1):24-7. View Abstract
  59. 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 Abstract
  60. Bacteremia risk and outpatient management of febrile patients with sickle cell disease. Pediatrics. 2013 Jun; 131(6):1035-41. View Abstract
  61. 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 Abstract
  62. Platelet activation and inhibition in sickle cell disease (pains) study. Platelets. 2014; 25(1):27-35. View Abstract
  63. Weight status of children with sickle cell disease. Pediatrics. 2013 Apr; 131(4):e1168-73. View Abstract
  64. 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 Abstract
  65. Stroke With Transfusions Changing to Hydroxyurea (SWiTCH). Blood. 2012 Apr 26; 119(17):3925-32. View Abstract
  66. Effects of chronic transfusions on abdominal sonographic abnormalities in children with sickle cell anemia. J Pediatr. 2012 Feb; 160(2):281-285.e1. View Abstract
  67. 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 Abstract
  68. 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 Abstract
  69. 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 Abstract
  70. 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 Abstract
  71. Rudolph’s Pediatrics. Iron Deficiency Anemia: Definition, Pathophysiology and Classification. 2011. View Abstract
  72. Rudolph’s Pediatrics (Rudolph AM, Rudolph C, First L, Lister G, Gershon AA. Editors). Anemia: Definition, Pathophysiology and Classification. 2011. View Abstract
  73. Common variants at 10 genomic loci influence hemoglobin A1(C) levels via glycemic and nonglycemic pathways. Diabetes. 2010 Dec; 59(12):3229-39. View Abstract
  74. Encephalopathy from lead poisoning masquerading as a flu-like syndrome in an autistic child. Pediatr Emerg Care. 2010 May; 26(5):370-3. View Abstract
  75. 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 Abstract
  76. Hydroxyurea for children with sickle cell disease. Hematol Oncol Clin North Am. 2010 Feb; 24(1):199-214. View Abstract
  77. 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 Abstract
  78. Definitions of the phenotypic manifestations of sickle cell disease. Am J Hematol. 2010 Jan; 85(1):6-13. View Abstract
  79. 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 Abstract
  80. Pial synangiosis in patients with moyamoya syndrome and sickle cell anemia: perioperative management and surgical outcome. Neurosurg Focus. 2009 Apr; 26(4):E10. View Abstract
  81. Clinical practice guideline improves the treatment of sickle cell disease vasoocclusive pain. Pediatr Blood Cancer. 2009 Mar; 52(3):369-72. View Abstract
  82. 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. View Abstract
  83. Cefepime-induced neutropenia in a teenager. Pediatr Blood Cancer. 2008 Nov; 51(5):715-6. View Abstract
  84. Mutations in TMPRSS6 cause iron-refractory iron deficiency anemia (IRIDA). Nat Genet. 2008 May; 40(5):569-71. View Abstract
  85. Hydroxyurea for children with sickle cell disease. Pediatr Clin North Am. 2008 Apr; 55(2):483-501, x. View Abstract
  86. Chronic transfusion practice for children with sickle cell anaemia and stroke. Br J Haematol. 2009 May; 145(4):524-8. View Abstract
  87. Nathan and Oski’s Hematology of Infancy and Childhood (Nathan DG, Orkin SH, Ginsburg D, Look AT. Editors). Sickle Cell Disease. 2008; 949-1014. View Abstract
  88. 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 Abstract
  89. Sickle cell disease caused by Hb S/Québec-CHORI: treatment with hydroxyurea and response. Pediatr Blood Cancer. 2007 Aug; 49(2):207-10. View Abstract
  90. Hemoglobin SE disease: a concise review. Am J Hematol. 2007 Jul; 82(7):643-9. View Abstract
  91. Acquired immune cytopenias post-cardiac transplantation respond to rituximab. Pediatr Blood Cancer. 2007 Mar; 48(3):339-44. View Abstract
  92. Management of Sickle Cell Disease,. Comprehensive Pediatric Hospital Medicine (Zaoutis LD, Chiang VW). 2007; 731-737. View Abstract
  93. Hematologic Disorders. Manual of Pediatric Therapeutics (Greenes DS, Graeff JW.). 2007; 466-499. View Abstract
  94. 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 Abstract
  95. Risk of cholelithiasis in sickle cell anemia. Haematologica. 2005 Feb; 90(2):147. View Abstract
  96. Iron homeostasis and inherited iron overload disorders: an overview. Hematol Oncol Clin North Am. 2004 Dec; 18(6):1379-403, ix. View Abstract
  97. Chemical and functional analysis of hydroxyurea oral solutions. J Pediatr Hematol Oncol. 2004 Mar; 26(3):179-84. View Abstract
  98. Childhood autoimmune cytopenia secondary to unsuspected common variable immunodeficiency. J Pediatr. 2003 Nov; 143(5):662-5. View Abstract
  99. UGT1A promoter polymorphisms influence bilirubin response to hydroxyurea therapy in sickle cell anemia. J Lab Clin Med. 2003 Apr; 141(4):279-82. View Abstract
  100. 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 Abstract
  101. Diagnostic Approach to Sickle Hemoglobinopathies. Practical Algorithms in Pediatric Hematology-Oncology (Sills R). 2003. View Abstract
  102. Newborn Screening for Hemoglobinopathies. Practical Algorithms in Pediatric Hematology Oncology (Sills R.). 2003. View Abstract
  103. A multi-step model for the pathogenesis and evolution of PNH. Paroxysmal Nocturnal Hemoglobinuria (PNH) and Related Disorders. 2003; 41-54. View Abstract
  104. Interpretation of fetal hemoglobin only on newborn screening for hemoglobinopathy. J Pediatr Hematol Oncol. 2002 Aug-Sep; 24(6):499-502. View Abstract
  105. 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 Abstract
  106. 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 Abstract

Contact Matthew M. Heeney

Print Profile