Haploidentical HSCT vs immunosuppressive therapy for severe aplastic anemia in patients aged 40-65 years: a
Sheng-Nan Gao1, Jin-Song Jia1, Xiao-Jun Huang1
1Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Cell and Gene Therapy for Hematologic Malignancies, Peking University, Beijing, China.
Abstract:
Severe aplastic anemia (SAA) is a life-threatening hematological disorder, and the optimal treatment for patients aged ≥40 years remains controversial. This retrospective study aims to conduct a comparative analysis of the efficacy of immunosuppressive therapy (IST) versus haploidentical hematopoietic stem cell transplantation (haplo-HSCT) specifically in elderly patients. To minimize selection bias, propensity score matching (PSM) was performed to pair samples based on age and Eastern Cooperative Oncology Group score, thereby addressing treatment responses and survival outcomes while ensuring balanced baseline characteristics between the 2 groups. The study reveals that while overall survival (OS) rates were similar (69.6% ± 13.3% vs. 68.6% ± 12.1%, P = 0.80), haplo-HSCT significantly outperformed IST in terms of failure-free survival (FFS) rates (68.7% ± 12.1% vs. 46.5% ± 20.2%, P = 0.025). Multivariate Cox regression analysis confirmed that treatment modality and disease severity were independent predictors of FFS. These findings support haplo-HSCT as a viable first-line treatment option for appropriately selected elderly SAA patients, providing evidence for treatment options in patients with SAA aged 40 and above.
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