| DOI: 10.29090/psa.2024.02.24.148 | Pharm Sci Asia 2024; 51(2), 155-163 |
Realistic efficacy of oxymetholone compared with rabbit-antithymocyte globulin for severe acquired aplastic anemiaThitichaya Penthinapong1,2, Pirun Saelue3*, Warunsuda Sripakdee1, Thitima Doungngern1, Pimwara Tanvejsilp4
1 Department of Clinical Pharmacy, Faculty of Pharmaceutical Sciences, Prince of Songkla University, Hat Yai, Songkhla, Thailand
2 Pharmaceutical Care Training Center (PCTC), Faculty of Pharmacy, Chiang Mai University, Chiang Mai, Thailand
3 Clinical Hematology Unit, Division of Internal Medicine, Faculty of Medicine, Prince of Songkla University, Hat Yai, Songkhla, Thailand
4 Department of Social and Administrative Pharmacy, Faculty of Pharmaceutical Sciences, Prince of Songkla University, Hat Yai, Songkhla, Thailand
Although anti-thymocyte globulin with cyclosporine (rATG/CsA), bone marrow transplantation, and eltrombopag are the cornerstone treatment for severe and very severe aplastic anemia (SAA/vSAA), oxymetholone remains beneficial in ineligible patients. This study was conducted to compare the realistic effectiveness of oxymetholone with rATG/CsA in SAA/vSAA. The primary outcome was 2-year overall response rate (ORR). Factors associated with ORR were evaluated using a logistic model. The secondary outcomes were 3-, 6-, 12- and 18-month ORR and overall survival (OS). Kaplan-Meier estimates were calculated for OS and risk factors related to PFS were assessed using Cox proportional hazards models. A total of 47 eligible patients and 53 treatment periods were included. The ORR of rATG/CsA treatment was significantly higher than for oxymetholone (46.7 vs. 15.8%, p=0.025). With a median follow-up time of 20.4 months, OS among patients initially treated with rATG/CsA and oxymetholone were 78.6% and 28.5%, respectively (p=0.001). However, patients responding to oxymetholone had a longer median survival compared with patients having no response to rATG/CsA as front-line treatment (11.8 years and 2.9 years, respectively) although a statistically significant difference between the two groups was undetected (p=0.092). Severity of disease and response to treatment were significant predictors of mortality from AA. This study revealed the superiority of rATG/CsA in response and survival improvement. However, the patient’s situation, needing to start oxymetholone as front-line treatment, oxymetholone, still had improved survival among the responders. Patients unresponsive to oxymetholone within 3 months should be switched throughout the treatment.
Keyword:
Hematologic disorder, Acquired aplastic anemia, Oxymetholone, Androgens, Anti-thymocyte globulin, Immunosuppressive therapy
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