尽管近年来取得了进展，但骨髓衰竭（BMF）和骨髓性肿瘤引起的细胞减少的治疗仍然具有挑战性。雄激素促进血细胞的更新和成熟，可能在这些病症中有益。本文报告了雄激素在血液学疾病中作为单一药物的系统评价。共纳入了46项研究，主要为回顾性研究，使用不同类型和剂量的雄激素：12项关于后天性再生障碍性贫血（AA），11项关于遗传性BMF，17项关于骨髓增生异常综合征（MDS），以及7项关于骨髓纤维化。遗传性BMF的反应范围为50%至70%，后天性AA和MDS的反应范围为40%至50%，而对于依赖输血的贫血，仅有有限的证据表明雄激素有益。副作用主要包括男性化和肝酶升高，而白血病进展的风险仍然存在争议。版权所有 © 2023 Elsevier Ltd.

Despite recent advancements, treatment of cytopenia due to bone marrow failures (BMF) and myeloid neoplasms remains challenging. Androgens promote renewal and maturation of blood cells and may be beneficial in these forms. Here we report a systematic review of androgens use as single agent in hematologic conditions. Forty-six studies, mainly retrospective with various androgen types and doses, were included: 12 on acquired aplastic anemia (AA), 11 on inherited BMF, 17 on myelodysplastic syndromes (MDS), and 7 on myelofibrosis. Responses ranged from 50 to 70% in inherited BMF, 40-50% in acquired AA and MDS, while very limited evidence emerged for myelofibrosis. In acquired AA, response was associated with presence of non-severe disease; in MDS androgens were more effective on thrombocytopenia or mild to moderate anemia, whilst limited benefit was observed for transfusion dependent anemia. Toxicity profile mainly consisted of virilization and liver enzyme elevation, whilst the risk of leukemic evolution remains controversial.Copyright © 2023. Published by Elsevier Ltd.