Chapter 31. Evidence-Based Treatment of Thalassemia Major

  1. Mark A. Crowther MD, MSc, FRCPC Professor Division Director3,4,
  2. Jeff Ginsberg MD, FRCPC Professor5,
  3. Holger J. Schünemann MD, PhD, MSc, FACP, FCCP Professor6,7,
  4. Ralph M. Meyer MD, FRCPC Director Professor8,9 and
  5. Richard Lottenberg MD, FACP Professor10
  1. Issac Odame MB, ChB, FRCPCH, FRCPath, FRCPC1 and
  2. Deborah Rund MD2

Published Online: 4 FEB 2009

DOI: 10.1002/9781444300857.ch31

Evidence-Based Hematology

Evidence-Based Hematology

How to Cite

Odame, I. and Rund, D. (2008) Evidence-Based Treatment of Thalassemia Major, in Evidence-Based Hematology (eds M. A. Crowther, J. Ginsberg, H. J. Schünemann, R. M. Meyer and R. Lottenberg), Wiley-Blackwell, Oxford, UK. doi: 10.1002/9781444300857.ch31

Editor Information

  1. 3

    Division of Hematology, McMaster University, Hamilton, Ontario, Canada

  2. 4

    St. Joseph's Hospital, Hamilton, Ontario, Canada

  3. 5

    Department of Medicine, McMaster University, Hamilton, Canada

  4. 6

    CLARITY Research Group, Department of Epidemiology, Italian National Cancer Institute Regina Elena, Rome, Italy

  5. 7

    McMaster University, Hamilton, Canada

  6. 8

    National Cancer Institute of Canada, Clinical Trials Group, Edith Eisenhauer Chair in Clinical Cancer Research, Queen's University, Kingston, Ontario, Canada

  7. 9

    Departments of Oncology and Medicine, Queen's University, Kingston, Ontario, Canada

  8. 10

    Division of Hematology/Oncology, Department of Medicine, University of Florida College of Medicine, Gainesville, Florida, USA

Author Information

  1. 1

    Division of Hematology/Oncology, The Hospital for Sick Children, Toronto, Ontario, Canada

  2. 2

    Hematology Department, Hebrew University, Hadassah University Hospital, Ein Kerem, Jerusalem, Israel

Publication History

  1. Published Online: 4 FEB 2009
  2. Published Print: 27 JUN 2008

ISBN Information

Print ISBN: 9781405157476

Online ISBN: 9781444300857



  • thalassemia major;
  • evidence-based findings on treatment of transfusion-dependent β-thalassemia major;
  • grading of evidence and recommendations;
  • clinical trials on desferrioxamine (DFO);
  • desferrioxamine and deferiprone combinations;
  • risks of splenectomy;
  • splenectomy in transfused patients?;
  • transplants from HLA-identical related donors;
  • transplants from HLA-unrelated donors;
  • cord blood transplantation


This chapter contains sections titled:

  • Introduction

  • Literature-search strategy

  • Grading of evidence and recommendations

  • In patients on chronic transfusion therapy, what is the target hemoglobin that should be maintained?

  • What is optimal iron chelation therapy for patients on chronic transfusion?

  • What are the indications for splenectomy in transfused patients?

  • What is the role of hematopoietic stem cell transplantation in the management of patients with transfusion-dependent β-thalassemia major?

  • Transplants from HLA-identical related donors

  • Transplants from HLA-unrelated donors

  • What role do fetal hemoglobin-inducing therapies have in the management of β-thalassemia major?

  • References