Journal article
Androgen therapy in inherited bone marrow failure syndromes: analysis from the Canadian Inherited Marrow Failure Registry
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. …
Authors
Català A; Ali SS; Cuvelier GDE; Steele M; Klaassen RJ; Fernandez CV; Pastore YD; Abish S; Rayar M; Jardine L
Journal
British Journal of Haematology, Vol. 189, No. 5, pp. 976–981
Publisher
Wiley
Publication Date
June 2020
DOI
10.1111/bjh.16445
ISSN
0007-1048
Fields of Research (FoR)
Medical Subject Headings (MeSH)
AdolescentAdultAndrogensBone Marrow Failure DisordersCanadaCell LineageChildChild, PreschoolCombined Modality TherapyDanazolDisease ProgressionDrug SubstitutionFemaleHematopoietic Stem Cell TransplantationHumansInfantMaleMiddle AgedOxymetholonePancytopeniaRegistriesThrombocytopeniaTreatment OutcomeVirilism