-
British Journal of Haematology Jun 2020Progressive cytopenia is a serious complication among paediatric patients with inherited bone marrow failure syndromes (IBMFS). Androgens have been used to improve blood...
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.
Topics: Adolescent; Adult; Androgens; Bone Marrow Failure Disorders; Canada; Cell Lineage; Child; Child, Preschool; Combined Modality Therapy; Danazol; Disease Progression; Drug Substitution; Female; Hematopoietic Stem Cell Transplantation; Humans; Infant; Male; Middle Aged; Oxymetholone; Pancytopenia; Registries; Thrombocytopenia; Treatment Outcome; Virilism
PubMed: 32128787
DOI: 10.1111/bjh.16445 -
British Journal of Haematology Oct 2019
Topics: Adult; Aged; Aged, 80 and over; Androgens; Danazol; Female; Humans; Male; Middle Aged; Myelodysplastic Syndromes; Oxymetholone; Retrospective Studies; Thrombocytopenia; Treatment Outcome; Young Adult
PubMed: 31368111
DOI: 10.1111/bjh.16121