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

Albert CatalàSalah S AliGeoffrey D E CuvelierMacGregor SteeleRobert J KlaassenConrad V FernandezYves D PastoreSharon AbishMeera RayarLawrence JardineVicky R BreakeyJosee BrossardRoona SinhaMariana SilvaLisa GoodyearJeffrey H LiptonBruno MichonCatherine Corriveau-BourqueLillian SungSupanun LauhasurayotinBozana ZlateskaMichaela CadaYigal Dror
Published in: British journal of haematology (2020)
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.
Keyphrases
  • bone marrow
  • mesenchymal stem cells
  • oxidative stress
  • newly diagnosed
  • multiple sclerosis
  • ejection fraction
  • intensive care unit
  • stem cells
  • prognostic factors
  • data analysis