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Annals of hematology2025; doi: 10.1007/s00277-025-06460-5

Good response to oxymetholone in adult aplastic anemia.

Abstract: 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.
© 2025. The Author(s).
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Publication Date: 2025-06-14 PubMed ID: 40515826PubMed Central: 8440252DOI: 10.1007/s00277-025-06460-5Google Scholar: Lookup
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Summary

This research summary has been generated with artificial intelligence and may contain errors and omissions. Refer to the original study to confirm details provided. Submit correction.

The research article explores effective alternative treatment options for adult aplastic anemia, specifically through the use of oxymetholone, in Thailand due to the inaccessibility of stem cell transplantation and horse antithymocyte globulin (ATG).

Study Background and Purpose

  • In Thailand, the more common treatment options for aplastic anemia (AA) such as stem cell transplantation and antithymocyte globulin (ATG) are not readily available. This led the researchers to explore alternative therapies for these patients, resulting in a study specifically focusing on the effectiveness of oxymetholone as a treatment.

Methods of the Study

  • Conducting a cohort study from 2013 to 2023, the researchers used a sample of 110 adult AA patients that were treated solely with oxymetholone for a minimum of 30 days.
  • The response to oxymetholone treatment and the different factors influencing prognosis were evaluated after six months.

Study Participants and Treatment

  • The mean age of the study participants was 63.4 years and 58.2% of them were female.
  • Severe and very severe AA (SAA/VSAA) conditions were found in 64.5% and 3.6% of the participants respectively.
  • The initial daily dose of oxymetholone administered was 150 mg for 66.4% of the patients.

Results and Implications

  • Results showed that overall response was 56.4% (50.7% for SAA/VSAA).
  • Responding patients achieved transfusion independence at a median time of 11.8 weeks.
  • Failure to respond and subsequent deaths were accounted as no response. Seventeen (or 17.9%) patients discontinued treatment due to side effects, primarily hepatitis.
  • Androgenic side effects were evident in 55.5% of patients within the first month of treatment.
  • A higher baseline reticulocyte count, as well as the onset of oily skin and acne within the first two months, were identified as predictors of positive responses.
  • The SKAR scoring system, incorporating these three factors, demonstrated a high predictive accuracy with an area under the ROC curve of 0.87.
  • The overall survival rate at 5 years was 77.4%. The performance status and response status significantly impacted mortality, with responding patients demonstrating a 94.5% survival rate at 5 years.
  • In conclusion, androgens like oxymetholone can be a valuable treatment option for AA in regions like Thailand where other forms of treatment are less accessible. Additionally, a score based on reticulocyte counts and androgenic effects could potentially guide treatment decisions.

Cite This Article

APA
Chaipokam J, Rojnuckarin P. (2025). Good response to oxymetholone in adult aplastic anemia. Ann Hematol. https://doi.org/10.1007/s00277-025-06460-5

Publication

Annals of hematology
ISSN: 1432-0584
NlmUniqueID: 9107334
Country: Germany
Language: English

Researcher Affiliations

Chaipokam, Jindaratn
  • Department of Medicine, Sakon Nakhon Hospital, Sakon Nakhon, Thailand.
  • Faculty of Medicine, Kasetsart University, Bangkok, Thailand.
Rojnuckarin, Ponlapat
  • Excellence Center in Translational Hematology, Department of Medicine, Faculty of Medicine, Chulalongkorn University, Bangkok, 10330, Thailand. rojnuckarinp@gmail.com.

Conflict of Interest Statement

Declarations. Human ethics and consent to participate: The protocol was approved by the Sakon Nakhon Hospital Ethical Committee. All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional research committee and with the 1964 Helsinki declaration. After ethical committee approval, 111 patients were recruited after informed consent. No informed consent was required in 34 retrospectively reviewed cases according to our Institutional Review Board. Competing interests: The authors declare no competing interests.

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