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

Authors :: Català A
Ali SS
Cuvelier GDE
Steele M
Klaassen RJ
Fernandez CV
Pastore YD
Abish S
Rayar M
Jardine L
Breakey VR
Brossard J
Sinha R
Silva M
Goodyear L
Lipton JH
Michon B
Corriveau-Bourque C
Sung L
Lauhasurayotin S
Zlateska B
Cada M
Dror Y
Source :: British journal of haematology [Br J Haematol] 2020 Jun; Vol. 189 (5), pp. 976-981. Date of Electronic Publication: 2020 Mar 03.
Publication Year :: 2020
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.<br /> (© 2020 British Society for Haematology and John Wiley & Sons Ltd.)

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