| Literature DB >> 32128787 |
Albert Català1, Salah S Ali2, Geoffrey D E Cuvelier3, MacGregor Steele4, Robert J Klaassen5, Conrad V Fernandez6, Yves D Pastore7, Sharon Abish8, Meera Rayar9, Lawrence Jardine10, Vicky R Breakey11, Josee Brossard12, Roona Sinha13, Mariana Silva14, Lisa Goodyear15, Jeffrey H Lipton16, Bruno Michon17, Catherine Corriveau-Bourque18, Lillian Sung19, Supanun Lauhasurayotin1,20, Bozana Zlateska1, Michaela Cada1, Yigal Dror1,20,21.
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.Entities:
Keywords: Fanconi anaemia; androgens; bone marrow failure; danazol; dyskeratosis congenita
Year: 2020 PMID： 32128787 DOI： 10.1111/bjh.16445
Source DB: PubMed Journal: Br J Haematol ISSN： 0007-1048 Impact factor: 6.998