Androgen therapy in inherited bone marrow failure syndromes: analysis from the Canadian Inherited Marrow Failure Registry.
Adolescent
Adult
Androgens
/ adverse effects
Bone Marrow Failure Disorders
/ blood
Canada
/ epidemiology
Cell Lineage
Child
Child, Preschool
Combined Modality Therapy
Danazol
/ adverse effects
Disease Progression
Drug Substitution
Female
Hematopoietic Stem Cell Transplantation
Humans
Infant
Male
Middle Aged
Oxymetholone
/ adverse effects
Pancytopenia
/ drug therapy
Registries
Thrombocytopenia
/ drug therapy
Treatment Outcome
Virilism
/ chemically induced
Fanconi anaemia
androgens
bone marrow failure
danazol
dyskeratosis congenita
Journal
British journal of haematology
ISSN: 1365-2141
Titre abrégé: Br J Haematol
Pays: England
ID NLM: 0372544
Informations de publication
Date de publication:
06 2020
06 2020
Historique:
received:
17
08
2019
accepted:
21
11
2019
pubmed:
5
3
2020
medline:
28
1
2021
entrez:
5
3
2020
Statut:
ppublish
Résumé
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.
Substances chimiques
Androgens
0
Oxymetholone
L76T0ZCA8K
Danazol
N29QWW3BUO
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
976-981Informations de copyright
© 2020 British Society for Haematology and John Wiley & Sons Ltd.
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