Chaipokam Jindaratn, Rojnuckarin Ponlapat
Department of Medicine, Sakon Nakhon Hospital, Sakon Nakhon, Thailand.
Faculty of Medicine, Kasetsart University, Bangkok, Thailand.
Ann Hematol. 2025 Jun 14. doi: 10.1007/s00277-025-06460-5.
In Thailand, stem cell transplantation and horse antithymocyte globulin (ATG) are not accessible for most adult aplastic anemia (AA) patients. Alternative therapies are required. We conducted a cohort study of 110 adult AA patients treated with oxymetholone alone for at least 30 days from 2013 to 2023. Response at month 6 and prognostic factors were evaluated. The mean age was 63.4 years old and 58.2% were female. Severe and very severe AA (SAA/VSAA) comprised 64.5% and 3.6%, respectively. The initial oxymetholone daily dose was 150 mg in 66.4%. The overall response was 56.4% (50.7% for SAA/VSAA), with a median time to transfusion independence of 11.8 weeks. Deaths were regarded as no response. Seventeen (17.9%) patients discontinued the treatment due to side effects, especially hepatitis (15/17). Androgenic side effects (55.5%) mostly occurred within the first month. Multivariate analysis identified that baseline reticulocyte count > 10 × 10/L (adjusted odds ratio [OR] 7.3, 95% confidence interval [CI] (2.55-21.11), oily skin (OR 4.93, 95%CI 1.50-16.26) and acne (OR 9.78, 95%CI 2.11-45.28) occurring within 2 months were predictive for responses. The SKAR scoring system using these three factors showed an area under the ROC curve of 0.87 (95%CI 0.80-0.92). The 5-year overall survival rate was 77.4%. Poor performance status (p < 0.001) and response status (p < 0.001) significantly impacted mortality. Responding patients demonstrated 94.5% 5-year survival. In conclusion, androgen is a useful treatment option for AA in Thailand. The score based on reticulocytes and androgenic effects could predict the response and potentially help decision-making.
在泰国，大多数成年再生障碍性贫血（AA）患者无法获得干细胞移植和马抗胸腺细胞球蛋白（ATG）。需要其他治疗方法。我们对2013年至2023年期间110例仅接受司坦唑醇治疗至少30天的成年AA患者进行了一项队列研究。评估了第6个月时的反应和预后因素。平均年龄为63.4岁，女性占58.2%。重度和极重度AA（SAA/VSAA）分别占64.5%和3.6%。66.4%的患者初始司坦唑醇每日剂量为150毫克。总体缓解率为56.4%（SAA/VSAA为50.7%），输血独立的中位时间为11.8周。死亡被视为无反应。17例（17.9%）患者因副作用停药，尤其是肝炎（15/17）。雄激素副作用（55.5%）大多发生在第一个月内。多变量分析确定，基线网织红细胞计数>10×10⁹/L（调整后的优势比[OR]为7.3，95%置信区间[CI]为(2.55 - 21.11)）、油性皮肤（OR为4.93，95%CI为1.50 - 16.26）以及2个月内出现痤疮（OR为9.78，95%CI为2.11 - 45.28）可预测反应。使用这三个因素的SKAR评分系统显示ROC曲线下面积为0.87（95%CI为0.80 - 0.92）。5年总生存率为77.4%。较差的体能状态（p < 0.001）和反应状态（p < 0.001）对死亡率有显著影响。有反应的患者5年生存率为94.5%。总之，雄激素是泰国AA的一种有效治疗选择。基于网织红细胞和雄激素效应的评分可预测反应，并可能有助于决策。
