Current use of androgens in bone marrow failure disorders: a report from the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation
Year: 2023
Type: article
Study design: Observational Study
Abstract: Androgens represent the historical therapeutic backbone of bone marrow failure (BMF) syndromes. However, their role has rarely been analyzed in a prospective setting, and systematic and long-term data regarding their usage, effectiveness and toxicity in both acquired and inherited BMF are currently unavailable. Here, taking advantage of a unique disease-specific international dataset, we retrospectively analyzed the largest cohort so far of BMF patients who received androgens before or in the absence of an allogeneic hematopoietic cell transplantation (HCT), re-evaluating their current use in these disorders. We identified 274 patients across 82 European Society for Blood and Marrow Transplantation (EBMT) affiliated centers: 193 with acquired (median age 32 years) and 81 with inherited (median age 8 years) BMF. With a median duration of androgen treatment of 5.6 and 20 months, respectively, complete and partial remission rates at 3 months were 6% and 29% in acquired and 8% and 29% in inherited disorders. Five-year overall survival and failure-free survival (FFS) were respectively 63% and 23% in acquired and 78% and 14% in inherited BMF. Androgen initiation after second-line treatments for acquired BMF, and after >12 months post diagnosis for inherited BMF were identified as factors associated with improved FFS in multivariable analysis. Androgen use was associated with a manageable incidence of organ-specific toxicity, and low rates of solid and hematologic malignancies. Sub-analysis of transplant-related outcomes after exposure to these compounds showed probabilities of survival and complications similar to other transplanted BMF cohorts. This study delivers a unique opportunity to track androgen use in BMF syndromes and represents the basis for general recommendations on this category of therapeutics on behalf of the Severe Aplastic Anemia Working Party of the EBMT.
Source: Haematologica
Authors: Simona Pagliuca, Austin G. Kulasekararaj, Dirk-Jan Eikema, Brian Piepenbroek, Raheel Iftikhar, Tariq Mahmood Satti, Morag Griffin, Marica Laurino, Alphan Kupesiz, Yves Bertrand, Bruno Fattizzo, Ibrahim Yakoub-Agha, Mahmoud Aljurf, Paola Corti, Erika Massaccesi, Bruno Lioure, Marisa Calabuig, Matthias Klammer, Emel Unal, Depei Wu, Patrice Chevallier, Edouard Forcade, John A. Snowden, Hakan Ozdogu, Antonio Risitano, Régis Peffault De Latour
Institutions: Centre National de la Recherche Scientifique, Centre Hospitalier Régional et Universitaire de Nancy, King's College London, King's College Hospital NHS Foundation Trust, European Society for Blood and Marrow Transplantation, Leiden University, Leeds Teaching Hospitals NHS Trust, Ospedale Policlinico San Martino, Akdeniz University, Institut d’Hématologie et d’Oncologie Pédiatrique, Hôpital Debrousse, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Inserm, Université de Lille, Centre Hospitalier Universitaire de Lille, Institute for Translational Research in Inflammation, King Faisal Specialist Hospital & Research Centre, Azienda Ospedaliera San Gerardo, University of Milano-Bicocca, Istituto Giannina Gaslini, Institut de Cancérologie Strasbourg, Hospital Clínico Universitario de Valencia, St George's Hospital, Ankara University, First Affiliated Hospital of Soochow University, Centre Hospitalier Universitaire de Nantes, Centre Hospitalier Universitaire de Bordeaux, Sheffield Teaching Hospitals NHS Foundation Trust, Başkent University, Azienda Ospedaliera S.Giuseppe Moscati, Assistance Publique – Hôpitaux de Paris, Hôpital Saint-Louis
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: gold
Estimated APC paid: $2,157
OpenAlex ID: W4377011771