Efficacy of Oxymetholone in Severe and Nonsevere Acquired Aplastic Anemia: A Propensity Score Matching Analysis
Worachaya Pengthina,1 Pirun Saelue2 1Pharmaceutical Care Training Center, Department of Pharmaceutical Care, Faculty of Pharmacy, Chiang Mai University, Chiang Mai, Thailand; 2Clinical Hematology Unit, Division of Internal Medicine, Faculty of Medicine, Prince of Songkla University, Hat Yai, Songkhl...
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| Format: | Article |
| Language: | English |
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Dove Medical Press
2022-12-01
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| Series: | Journal of Blood Medicine |
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| Online Access: | https://www.dovepress.com/efficacy-of-oxymetholone-in-severe-and-nonsevere-acquired-aplastic-ane-peer-reviewed-fulltext-article-JBM |
| _version_ | 1811212878275936256 |
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| author | Pengthina W Saelue P |
| author_facet | Pengthina W Saelue P |
| author_sort | Pengthina W |
| collection | DOAJ |
| description | Worachaya Pengthina,1 Pirun Saelue2 1Pharmaceutical Care Training Center, Department of Pharmaceutical Care, Faculty of Pharmacy, Chiang Mai University, Chiang Mai, Thailand; 2Clinical Hematology Unit, Division of Internal Medicine, Faculty of Medicine, Prince of Songkla University, Hat Yai, Songkhla, ThailandCorrespondence: Pirun Saelue, Clinical Hematology Unit, Division of Internal Medicine, Faculty of Medicine, Prince of Songkla University, Hat Yai, Songkhla, 90110, Thailand, Tel +66 7445 1481 ; +66 81543 3031, Fax +66 7428 1457, Email pirun2118@hotmail.comBackground: Bone marrow transplantation, antithymocyte globulin/cyclosporine and eltrombopag are recommended as first-line therapy of severe aplastic anemia (SAA). However, androgens could be considered as front-line treatment among any patients ineligible for better methods although unsatisfactory efficacy is presented.Objective: This retrospective study aimed to evaluate response and survival rate of practical-based treatment with oxymetholone.Patients and Methods: This constituted an analysis of patients receiving a diagnosis of acquired aplastic anemia (AA) at the age of 15 or over and receiving oxymetholone between January 2004 and December 2018. Propensity Score Analysis (PSA) 1:1 matching was performed, according to sex, age and interval from first symptom to treatment. The primary outcome was one-year overall response (OR).Results: Seventy-four patients were successfully matched by PSA. The 1-year OR of oxymetholone in the nonsevere AA (nSAA) and SAA/very severe AA (vSAA) groups was 54.1 and 13.5%, respectively (P < 0.001). With median follow-up 2.7 years, the overall survival was 59.5% in nSAA and 37.8% in SAA/vSAA (P = 0.051). Median survival in nSAA and SAA/vSAA were 7.0 years and 1.8 years, respectively (P = 0.045). However, the responders of SAA/vSAA had longer survival than nonresponders of the nSAA group.Conclusion: These results revealed longer survival among the responders of patients with AA, even in the SAA/vSAA group. However, close monitoring of therapeutic responses is still performed. Switching therapy is necessary when remission is undetected after 6 months of oxymetholone treatment.Keywords: androgen, hematologic disease, real world practice, survival |
| first_indexed | 2024-04-12T05:36:42Z |
| format | Article |
| id | doaj.art-7a8b4b7ab8cd4b63901e65a2d50cf5c4 |
| institution | Directory Open Access Journal |
| issn | 1179-2736 |
| language | English |
| last_indexed | 2024-04-12T05:36:42Z |
| publishDate | 2022-12-01 |
| publisher | Dove Medical Press |
| record_format | Article |
| series | Journal of Blood Medicine |
| spelling | doaj.art-7a8b4b7ab8cd4b63901e65a2d50cf5c42022-12-22T03:45:49ZengDove Medical PressJournal of Blood Medicine1179-27362022-12-01Volume 1375376180149Efficacy of Oxymetholone in Severe and Nonsevere Acquired Aplastic Anemia: A Propensity Score Matching AnalysisPengthina WSaelue PWorachaya Pengthina,1 Pirun Saelue2 1Pharmaceutical Care Training Center, Department of Pharmaceutical Care, Faculty of Pharmacy, Chiang Mai University, Chiang Mai, Thailand; 2Clinical Hematology Unit, Division of Internal Medicine, Faculty of Medicine, Prince of Songkla University, Hat Yai, Songkhla, ThailandCorrespondence: Pirun Saelue, Clinical Hematology Unit, Division of Internal Medicine, Faculty of Medicine, Prince of Songkla University, Hat Yai, Songkhla, 90110, Thailand, Tel +66 7445 1481 ; +66 81543 3031, Fax +66 7428 1457, Email pirun2118@hotmail.comBackground: Bone marrow transplantation, antithymocyte globulin/cyclosporine and eltrombopag are recommended as first-line therapy of severe aplastic anemia (SAA). However, androgens could be considered as front-line treatment among any patients ineligible for better methods although unsatisfactory efficacy is presented.Objective: This retrospective study aimed to evaluate response and survival rate of practical-based treatment with oxymetholone.Patients and Methods: This constituted an analysis of patients receiving a diagnosis of acquired aplastic anemia (AA) at the age of 15 or over and receiving oxymetholone between January 2004 and December 2018. Propensity Score Analysis (PSA) 1:1 matching was performed, according to sex, age and interval from first symptom to treatment. The primary outcome was one-year overall response (OR).Results: Seventy-four patients were successfully matched by PSA. The 1-year OR of oxymetholone in the nonsevere AA (nSAA) and SAA/very severe AA (vSAA) groups was 54.1 and 13.5%, respectively (P < 0.001). With median follow-up 2.7 years, the overall survival was 59.5% in nSAA and 37.8% in SAA/vSAA (P = 0.051). Median survival in nSAA and SAA/vSAA were 7.0 years and 1.8 years, respectively (P = 0.045). However, the responders of SAA/vSAA had longer survival than nonresponders of the nSAA group.Conclusion: These results revealed longer survival among the responders of patients with AA, even in the SAA/vSAA group. However, close monitoring of therapeutic responses is still performed. Switching therapy is necessary when remission is undetected after 6 months of oxymetholone treatment.Keywords: androgen, hematologic disease, real world practice, survivalhttps://www.dovepress.com/efficacy-of-oxymetholone-in-severe-and-nonsevere-acquired-aplastic-ane-peer-reviewed-fulltext-article-JBMandrogenhematologic diseasereal world practicesurvival |
| spellingShingle | Pengthina W Saelue P Efficacy of Oxymetholone in Severe and Nonsevere Acquired Aplastic Anemia: A Propensity Score Matching Analysis Journal of Blood Medicine androgen hematologic disease real world practice survival |
| title | Efficacy of Oxymetholone in Severe and Nonsevere Acquired Aplastic Anemia: A Propensity Score Matching Analysis |
| title_full | Efficacy of Oxymetholone in Severe and Nonsevere Acquired Aplastic Anemia: A Propensity Score Matching Analysis |
| title_fullStr | Efficacy of Oxymetholone in Severe and Nonsevere Acquired Aplastic Anemia: A Propensity Score Matching Analysis |
| title_full_unstemmed | Efficacy of Oxymetholone in Severe and Nonsevere Acquired Aplastic Anemia: A Propensity Score Matching Analysis |
| title_short | Efficacy of Oxymetholone in Severe and Nonsevere Acquired Aplastic Anemia: A Propensity Score Matching Analysis |
| title_sort | efficacy of oxymetholone in severe and nonsevere acquired aplastic anemia a propensity score matching analysis |
| topic | androgen hematologic disease real world practice survival |
| url | https://www.dovepress.com/efficacy-of-oxymetholone-in-severe-and-nonsevere-acquired-aplastic-ane-peer-reviewed-fulltext-article-JBM |
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