Reduced intensity hematopoietic stem-cell transplantation across human leukocyte antigen barriers in a patient with congenital amegakaryocytic thrombocytopenia and monosomy 7
MacGregor Steele MD, FRCPC
Marrow Failure and Myelodysplasia Programme, and Blood and Marrow Transplantation Section, Division of Haematology and Oncology, Department of Paediatrics, The Hospital for Sick Children and the University of Toronto, Toronto, Ontario, Canada
Search for more papers by this authorJohann Hitzler MD, FRCPC, Dr. Med
Marrow Failure and Myelodysplasia Programme, and Blood and Marrow Transplantation Section, Division of Haematology and Oncology, Department of Paediatrics, The Hospital for Sick Children and the University of Toronto, Toronto, Ontario, Canada
Search for more papers by this authorJohn J. Doyle MD, FRCPC, FAAP
Marrow Failure and Myelodysplasia Programme, and Blood and Marrow Transplantation Section, Division of Haematology and Oncology, Department of Paediatrics, The Hospital for Sick Children and the University of Toronto, Toronto, Ontario, Canada
Search for more papers by this authorManuela Germeshausen PhD
Department of Pediatric Hematology and Oncology, Hannover Medical School, Hannover, Germany
Search for more papers by this authorConrad V. Fernandez Hon BSc, MD, FRCPC
Department of Pediatrics, IWK Health Centre, Dalhousie University, Halifax, Nova Scotia, Canada
Search for more papers by this authorKim Yuille RN, BScN
Marrow Failure and Myelodysplasia Programme, and Blood and Marrow Transplantation Section, Division of Haematology and Oncology, Department of Paediatrics, The Hospital for Sick Children and the University of Toronto, Toronto, Ontario, Canada
Search for more papers by this authorCorresponding Author
Yigal Dror MD
Marrow Failure and Myelodysplasia Programme, and Blood and Marrow Transplantation Section, Division of Haematology and Oncology, Department of Paediatrics, The Hospital for Sick Children and the University of Toronto, Toronto, Ontario, Canada
Division of Haematology/Oncology, The Hospital for Sick Children, 555 University Avenue, Toronto, Ont., Canada M5G 1X8.===Search for more papers by this authorMacGregor Steele MD, FRCPC
Marrow Failure and Myelodysplasia Programme, and Blood and Marrow Transplantation Section, Division of Haematology and Oncology, Department of Paediatrics, The Hospital for Sick Children and the University of Toronto, Toronto, Ontario, Canada
Search for more papers by this authorJohann Hitzler MD, FRCPC, Dr. Med
Marrow Failure and Myelodysplasia Programme, and Blood and Marrow Transplantation Section, Division of Haematology and Oncology, Department of Paediatrics, The Hospital for Sick Children and the University of Toronto, Toronto, Ontario, Canada
Search for more papers by this authorJohn J. Doyle MD, FRCPC, FAAP
Marrow Failure and Myelodysplasia Programme, and Blood and Marrow Transplantation Section, Division of Haematology and Oncology, Department of Paediatrics, The Hospital for Sick Children and the University of Toronto, Toronto, Ontario, Canada
Search for more papers by this authorManuela Germeshausen PhD
Department of Pediatric Hematology and Oncology, Hannover Medical School, Hannover, Germany
Search for more papers by this authorConrad V. Fernandez Hon BSc, MD, FRCPC
Department of Pediatrics, IWK Health Centre, Dalhousie University, Halifax, Nova Scotia, Canada
Search for more papers by this authorKim Yuille RN, BScN
Marrow Failure and Myelodysplasia Programme, and Blood and Marrow Transplantation Section, Division of Haematology and Oncology, Department of Paediatrics, The Hospital for Sick Children and the University of Toronto, Toronto, Ontario, Canada
Search for more papers by this authorCorresponding Author
Yigal Dror MD
Marrow Failure and Myelodysplasia Programme, and Blood and Marrow Transplantation Section, Division of Haematology and Oncology, Department of Paediatrics, The Hospital for Sick Children and the University of Toronto, Toronto, Ontario, Canada
Division of Haematology/Oncology, The Hospital for Sick Children, 555 University Avenue, Toronto, Ont., Canada M5G 1X8.===Search for more papers by this authorAbstract
Congenital amegakaryocytic thrombocytopenia (CAMT) is a rare inherited bone marrow failure syndrome that has the potential to progress to pancytopenia and acute myeloid leukemia. Hematopoietic stem-cell transplantation (HSCT) is presently the only curative treatment approach. We used a reduced intensity transplantation regimen in a CAMT patient with aplastic anemia and monosomy 7 who had no matched related donor. The patient had rapid and durable engraftment with minimal complications and is well 24 months post-transplantation. Thus, reduced intensity conditioning might be a feasible approach to stem-cell transplantation in patients with CAMT who do not have a related donor and who are at increased risk of toxicity from standard conditioning regimens. © 2005 Wiley-Liss, Inc.
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