Androgen therapy in inherited bone marrow failure syndromes: analysis from the Canadian Inherited Marrow Failure Registry.,British Journal of Haematology

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Androgen therapy in inherited bone marrow failure syndromes: analysis from the Canadian Inherited Marrow Failure Registry.
British Journal of Haematology ( IF 6.5 ) Pub Date : 2020-03-03 , DOI: 10.1111/bjh.16445
Albert Català ₁ , Salah S Ali ₂ , Geoffrey D E Cuvelier ₃ , MacGregor Steele ₄ , Robert J Klaassen ₅ , Conrad V Fernandez ₆ , Yves D Pastore ₇ , Sharon Abish ₈ , Meera Rayar ₉ , Lawrence Jardine ₁₀ , Vicky R Breakey ₁₁ , Josee Brossard ₁₂ , Roona Sinha ₁₃ , Mariana Silva ₁₄ , Lisa Goodyear ₁₅ , Jeffrey H Lipton ₁₆ , Bruno Michon ₁₇ , Catherine Corriveau-Bourque ₁₈ , Lillian Sung ₁₉ , Supanun Lauhasurayotin _{1,

20} , Bozana Zlateska ₁ , Michaela Cada ₁ , Yigal Dror _{1,

20,

21}

Affiliation

Division of Hematology/Oncology, Department of Pediatrics, Marrow Failure and Myelodysplasia Program, The Hospital for Sick Children, Toronto, ON, Canada.
Bone Marrow Transplantation and Cellular Therapy, Division of Hematology/Oncology, Department of Pediatrics, The Hospital for Sick Children, Toronto, ON, Canada.
Pediatric Hematology-Oncology-Blood and Marrow Transplantation, University of Manitoba, CancerCare Manitoba, Winnipeg, MB, Canada.
Alberta Children's Hospital, Calgary, AB, Canada.
Department of Pediatrics, Children's Hospital of Eastern Ontario, Ottawa, ON, Canada.
Pediatric Hematology/Oncology, IWK Health Centre, Halifax, NS, Canada.
CHU Sainte-Justine, Montreal, QC, Canada.
Pediatric Hematology Oncology, Montreal Children's Hospital, Montreal, QC, Canada.
Division of Hematology/Oncology, UBC & B.C. Children's Hospital, Vancouver, BC, Canada.
Children's Hospital, London Health Sciences Centre, London, ON, Canada.
Department of Pediatrics, McMaster University, Hamilton, ON, Canada.
Centre Hospitalier Universitaire, Sherbrooke, QC, Canada.
Royal University Hospital, Saskatoon, SK, Canada.
Kingston General Hospital, Kingston, ON, Canada.
Pediatric Hematology/Oncology, Janeway Child Health Centre, St. John's, NF, Canada.
Allogeneic Blood and Marrow Transplant Program, Princess Margaret Cancer Centre, University of Toronto, Toronto, ON, Canada.
Centre Hospitalier Universitaire de Quebec, Sainte-Foy, QC, Canada.
Pediatrics, University of Alberta, Edmonton, AB, Canada.
Division of Hematology/Oncology, Department of Pediatrics, Child and Population Health Sciences, Research Institute, The Hospital for Sick Children, Toronto, ON, Canada.
Genetics and Genome Biology Program, The Hospital for Sick Children, Toronto, ON, Canada.
Institute of Medical Science, University of Toronto, Toronto, ON, Canada.

Progressive cytopenia is a serious complication among paediatric patients with inherited bone marrow failure syndromes (IBMFS). Androgens have been used to improve blood counts in different bone marrow failure conditions. Little is known about efficacy and toxicity with new androgens (i.e., danazol) in different types of IBMFS. We identified 29 patients from the Canadian Inherited Marrow Failure Registry, who received oxymetholone or danazol. Sixteen (55%) had haematological response including patients with unclassified IBMFS (45%). Danazol showed a better toxicity profile and similar efficacy compared to oxymetholone. Androgens are an effective and safe option to ameliorate bone marrow failure in IBMFS.

中文翻译：

遗传性骨髓衰竭综合征中的雄激素治疗：来自加拿大遗传性骨髓衰竭登记处的分析。

进行性血细胞减少症是患有遗传性骨髓衰竭综合征（IBMFS）的小儿患者的严重并发症。雄激素已用于改善不同骨髓衰竭情况下的血球计数。对于新型雄激素（如达那唑）在不同类型的IBMFS中的功效和毒性知之甚少。我们从加拿大遗传性骨髓衰竭登记处确定了29例接受羟甲酮或达那唑的患者。十六名（55％）的血液学反应包括未分类IBMFS的患者（45％）。与羟甲烯龙相比，达那唑具有更好的毒性和相似的功效。雄激素是改善IBMFS中骨髓衰竭的有效且安全的选择。

更新日期：2020-03-03

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