Stem cell transplantation

Comparison of efficacy and health-related quality of life of first-line haploidentical hematopoietic stem cell transplantation with unrelated cord blood infusion and first-line immunosuppressive therapy for acquired severe aplastic anemia

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Abstract

We retrospectively compared the efficacy and health-related quality of life (HRQoL) of (1) first-line haploidentical hematopoietic stem cell transplantation (haplo-HSCT, n = 146) combined with unrelated cord blood (UCB) infusion and (2) first-line immunosuppressive therapy (IST, n = 219) in acquired severe aplastic anemia (SAA) patients. At 6 months post treatment, 90.30% patients in the haplo-HSCT group and 18.78% patients in the IST group achieved normal blood routine (P < 0.0001). The time required to discontinue red blood cells and platelets transfusion in the IST group were longer than in the haplo-HSCT group (P < 0.0001). The estimated overall survival at 4 years was similar (80.1 ± 3.5% vs. 80.1 ± 3.0%, P = 0.726); the estimated failure-free survival (FFS) at 4 years was 77.8 ± 3.7% in the haplo-HSCT group and 48.0 ± 3.6% in the IST group (P < 0.0001). Patients treated with haplo-HSCT scored significantly better in the HRQoL than treated with IST (P < 0.0001). In the multivariate analysis, first-line haplo-HSCT was the favorable factor for FFS and HRQoL (P < 0.0001). These results suggest that first-line haplo-HSCT combined with UCB infusion might provide a better chance of success and HRQoL than first-line IST for SAA patients.

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Fig. 1: Patient overall survival (OS), as assessed using Kaplan–Meier analysis.
Fig. 2: Patient failure-free survival (FFS), as assessed using Kaplan–Meier analysis.

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Acknowledgements

Huiying Qiu, Xiaowen Tang, Yue Han, Chengcheng Fu, Zhengming Jin, Suning Chen, Xiaohui Hu, and Aining Sun provided partial patient data. This work was partially supported by grants from the National Natural Science Foundation of China (81730003), National Science and Technology Major Project (2017ZX09304021), National Key R&D Program of China (2019YFC0840604, 2017YFA0104502), Priority Academic Program Development of Jiangsu Higher Education Institutions (PAPD), Jiangsu Medical Outstanding Talents Project (JCRCA2016002), Jiangsu Provincial Key Medical Center (YXZXA2016002).

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DW designed the research; LL, YZ, WJ, and MM analyzed the data and wrote the paper; and all authors provided patient data and gave final approval for the paper.

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Correspondence to Miao Miao or Depei Wu.

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Liu, L., Zhang, Y., Jiao, W. et al. Comparison of efficacy and health-related quality of life of first-line haploidentical hematopoietic stem cell transplantation with unrelated cord blood infusion and first-line immunosuppressive therapy for acquired severe aplastic anemia. Leukemia 34, 3359–3369 (2020). https://doi.org/10.1038/s41375-020-0933-7

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