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

• Published in: British journal of haematology Vol. 189; no. 5; pp. 976 - 981
• Main Authors: 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, Corriveau‐Bourque, Catherine, Sung, Lillian, Lauhasurayotin, Supanun, Zlateska, Bozana, Cada, Michaela, Dror, Yigal
• Format: Journal Article
• Language: English
• Published: England Blackwell Publishing Ltd 01.06.2020
• Subjects: Adolescent; Adult; Androgens; Androgens - adverse effects; Androgens - therapeutic use; Bone marrow; bone marrow failure; Bone Marrow Failure Disorders - blood; Bone Marrow Failure Disorders - drug therapy; Bone Marrow Failure Disorders - genetics; Bone Marrow Failure Disorders - therapy; Canada - epidemiology; Cell Lineage; Child; Child, Preschool; Combined Modality Therapy; danazol; Danazol - adverse effects; Danazol - therapeutic use; Disease Progression; Drug Substitution; dyskeratosis congenita; Fanconi anaemia; Female; Hematology; Hematopoietic Stem Cell Transplantation; Humans; Infant; Male; Middle Aged; Oxymetholone - adverse effects; Oxymetholone - therapeutic use; Pancytopenia - drug therapy; Pancytopenia - etiology; Registries; Thrombocytopenia - drug therapy; Thrombocytopenia - etiology; Toxicity; Treatment Outcome; Virilism - chemically induced; Canada; bone marrow failure; Fanconi anaemia; dyskeratosis congenita; androgens; danazol
• ISSN: 0007-1048; 1365-2141; 1365-2141
• DOI: 10.1111/bjh.16445

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.