Androgen therapy in inherited bone marrow failure syndromes: analysis from the Canadian Inherited Marrow Failure Registry

Albert Català, Salah Ali, Geoff D.E. Cuvelier, MacGregor Steele, Robert J. Klaassen, Conrad V. Fernandez, Yves Pastore, Sharon Abish, Meera Rayar, Lawrence Jardine, Vicky R. Breakey, Josée Brossard, Roona Sinha, Mariana Silva, Lisa Goodyear, Jeffrey H. Lipton, Bruno Michon, Catherine Corriveau‐Bourque, Lillian Sung, Supanun Lauhasurayotin, Bozana Zlateska, Michaela Cada, Yigal Dror
In: British Journal of Haematology · 2020 · vol. 189(5) , pp. 976–981 · doi:10.1111/bjh.16445 · PMID:32128787 · W3010413675
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Androgen therapy using oxymetholone or danazol showed haematological response in 55% of Canadian IBMFS patients, with danazol demonstrating a better toxicity profile and similar efficacy.

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Abstract

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.

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