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。
Androgen therapy in inherited bone marrow failure syndromes: analysis from the Canadian Inherited Marrow Failure Registry.
机构信息
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.
出版信息
Br J Haematol. 2020 Jun;189(5):976-981. doi: 10.1111/bjh.16445. Epub 2020 Mar 3.
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.
进行性细胞减少症是儿童遗传性骨髓衰竭综合征（IBMFS）患者的严重并发症。雄激素已被用于改善不同骨髓衰竭情况下的血细胞计数。对于不同类型的 IBMFS 中新型雄激素（即达那唑）的疗效和毒性知之甚少。我们从加拿大遗传性骨髓衰竭登记处确定了 29 名接受羟甲烯龙或达那唑治疗的患者。16 名（55%）患者有血液学反应，包括未分类 IBMFS 患者（45%）。与羟甲烯龙相比，达那唑具有更好的毒性特征和相似的疗效。雄激素是改善 IBMFS 骨髓衰竭的有效且安全的选择。
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