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<p>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&#xa0;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 &gt; 10 × 10<sup>9</sup>/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 (<i>p</i> &lt; 0.001) and response status (<i>p</i> &lt; 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.</p>

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Good response to oxymetholone in adult aplastic anemia

  • 期刊论文

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  • 发表日期：

    2025年6月14日

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  • Jindaratn Chaipokam,
  • Ponlapat Rojnuckarin

摘要

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 × 109/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.