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Androgen therapy in inherited bone marrow failure syndromes: analysis from the Canadian Inherited Marrow Failure Registry
Author(s) -
Català Albert,
Ali Salah S.,
Cuvelier Geoffrey D. E.,
Steele MacGregor,
Klaassen Robert J.,
Fernandez Conrad V.,
Pastore Yves D.,
Abish Sharon,
Rayar Meera,
Jardine Lawrence,
Breakey Vicky R.,
Brossard Josee,
Sinha Roona,
Silva Mariana,
Goodyear Lisa,
Lipton Jeffrey H.,
Michon Bruno,
CorriveauBourque Catherine,
Sung Lillian,
Lauhasurayotin Supanun,
Zlateska Bozana,
Cada Michaela,
Dror Yigal
Publication year - 2020
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.16445
Subject(s) - cytopenia , medicine , danazol , bone marrow failure , bone marrow , bone marrow aplasia , endometriosis , biology , genetics , stem cell , haematopoiesis
Summary 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.