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Androgen therapy in inherited bone marrow failure syndromes: analysis from the Canadian Inherited Marrow Failure Registry.
Català, Albert; Ali, Salah S; Cuvelier, Geoffrey D E; Steele, MacGregor; Klaassen, Robert J; Fernandez, Conrad V; Pastore, Yves D; Abish, Sharon; Rayar, Meera; Jardine, Lawrence; Breakey, Vicky R; Brossard, Josee; Sinha, Roona; Silva, Mariana; Goodyear, Lisa; Lipton, Jeffrey H; Michon, Bruno; Corriveau-Bourque, Catherine; Sung, Lillian; Lauhasurayotin, Supanun; Zlateska, Bozana; Cada, Michaela; Dror, Yigal.
Afiliação
  • Català A; Division of Hematology/Oncology, Department of Pediatrics, Marrow Failure and Myelodysplasia Program, The Hospital for Sick Children, Toronto, ON, Canada.
  • Ali SS; Bone Marrow Transplantation and Cellular Therapy, Division of Hematology/Oncology, Department of Pediatrics, The Hospital for Sick Children, Toronto, ON, Canada.
  • Cuvelier GDE; Pediatric Hematology-Oncology-Blood and Marrow Transplantation, University of Manitoba, CancerCare Manitoba, Winnipeg, MB, Canada.
  • Steele M; Alberta Children's Hospital, Calgary, AB, Canada.
  • Klaassen RJ; Department of Pediatrics, Children's Hospital of Eastern Ontario, Ottawa, ON, Canada.
  • Fernandez CV; Pediatric Hematology/Oncology, IWK Health Centre, Halifax, NS, Canada.
  • Pastore YD; CHU Sainte-Justine, Montreal, QC, Canada.
  • Abish S; Pediatric Hematology Oncology, Montreal Children's Hospital, Montreal, QC, Canada.
  • Rayar M; Division of Hematology/Oncology, UBC & B.C. Children's Hospital, Vancouver, BC, Canada.
  • Jardine L; Children's Hospital, London Health Sciences Centre, London, ON, Canada.
  • Breakey VR; Department of Pediatrics, McMaster University, Hamilton, ON, Canada.
  • Brossard J; Centre Hospitalier Universitaire, Sherbrooke, QC, Canada.
  • Sinha R; Royal University Hospital, Saskatoon, SK, Canada.
  • Silva M; Kingston General Hospital, Kingston, ON, Canada.
  • Goodyear L; Pediatric Hematology/Oncology, Janeway Child Health Centre, St. John's, NF, Canada.
  • Lipton JH; Allogeneic Blood and Marrow Transplant Program, Princess Margaret Cancer Centre, University of Toronto, Toronto, ON, Canada.
  • Michon B; Centre Hospitalier Universitaire de Quebec, Sainte-Foy, QC, Canada.
  • Corriveau-Bourque C; Pediatrics, University of Alberta, Edmonton, AB, Canada.
  • Sung L; Division of Hematology/Oncology, Department of Pediatrics, Child and Population Health Sciences, Research Institute, The Hospital for Sick Children, Toronto, ON, Canada.
  • Lauhasurayotin S; Division of Hematology/Oncology, Department of Pediatrics, Marrow Failure and Myelodysplasia Program, The Hospital for Sick Children, Toronto, ON, Canada.
  • Zlateska B; Genetics and Genome Biology Program, The Hospital for Sick Children, Toronto, ON, Canada.
  • Cada M; Division of Hematology/Oncology, Department of Pediatrics, Marrow Failure and Myelodysplasia Program, The Hospital for Sick Children, Toronto, ON, Canada.
  • Dror Y; Division of Hematology/Oncology, Department of Pediatrics, Marrow Failure and Myelodysplasia Program, The Hospital for Sick Children, Toronto, ON, Canada.
Br J Haematol ; 189(5): 976-981, 2020 06.
Article em En | MEDLINE | ID: mdl-32128787
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.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Transtornos da Insuficiência da Medula Óssea / Androgênios Tipo de estudo: Etiology_studies Limite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Infant / Male / Middle aged País/Região como assunto: America do norte Idioma: En Revista: Br J Haematol Ano de publicação: 2020 Tipo de documento: Article País de afiliação: Canadá
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Transtornos da Insuficiência da Medula Óssea / Androgênios Tipo de estudo: Etiology_studies Limite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Infant / Male / Middle aged País/Região como assunto: America do norte Idioma: En Revista: Br J Haematol Ano de publicação: 2020 Tipo de documento: Article País de afiliação: Canadá