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01.12.2017 | Research | Ausgabe 1/2017 Open Access

Journal of Hematology & Oncology 1/2017

Upfront haploidentical transplant for acquired severe aplastic anemia: registry-based comparison with matched related transplant

Journal of Hematology & Oncology > Ausgabe 1/2017
Lan-Ping Xu, Song Jin, Shun-Qing Wang, Ling-Hui Xia, Hai Bai, Su-Jun Gao, Qi-Fa Liu, Jian-Min Wang, Xin Wang, Ming Jiang, Xi Zhang, De-Pei Wu, Xiao-Jun Huang
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Electronic supplementary material

The online version of this article (doi:10.​1186/​s13045-017-0398-y) contains supplementary material, which is available to authorized users.



Haploidentical donor (HID) hematopoietic stem cell transplantation (HSCT) is an alternative treatment method for severe aplastic anemia (SAA) patients lacking suitable identical donors and those who are refractory to immunosuppressive therapy (IST). The current study evaluated the feasibility of upfront haploidentical HSCT in SAA patients.


We conducted a multicenter study based on a registry database. One hundred fifty-eight SAA patients who underwent upfront transplantation between June 2012 and September 2015 were enrolled.


Eighty-nine patients had haploidentical donors (HIDs), and 69 had matched related donors (MRDs) for HSCT. The median times for myeloid engraftment in the HID and MRD cohorts were 12 (range, 9–20) and 11 (range, 8–19) days, with a cumulative incidence of 97.8 and 97.1% (P = 0.528), respectively. HID recipients had an increased cumulative incidence of grades II–IV acute graft-versus-host disease (aGVHD) (30.3 vs. 1.5%, P < 0.001), grades III–IV aGVHD (10.1 vs. 1.5%, P = 0.026), and chronic GVHD (cGVHD) (30.6 vs. 4.4%, P < 0.001) at 1 year but similar extensive cGVHD (3.4 vs. 0%, P = 0.426). The three-year estimated overall survival (OS) rates were 86.1 and 91.3% (P = 0.358), while the three-year estimated failure-free survival (FFS) rates were 85.0 and 89.8% (P = 0.413) in the HID and MRD cohorts, respectively. In multivariate analysis, survival outcome for the entire population was significantly adversely associated with increased transfusions and poor performance status pre-SCT. We did not observe differences in primary engraftment and survival outcomes by donor type.


Haploidentical SCT as upfront therapy was an effective and safe option for SAA patients, with favorable outcomes in experienced centers.

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Additional file 1: Table S1. Univariate analysis of factors associated with survival outcomes. Table S2. Univariate analysis of factors associated with II–IV aGVHD and III–IV aGVHD. Figure S1a. The cumulative incidence of 28-day neutrophil engraftment:HID97.75 ± 0.03%, MRD97.10 ± 0.05% (P = 0.528). Figure S1b. The cumulative incidence of platelet engraftment: HID96.63 ± 0.05%, MRD 95.65 ± 0.08% (P = 0.989). Figure S2a. The cumulative incidence of II–IV aGVHD: HID 30.34 ± 0.24%, MRD1.45 ± 0.02% (P < 0.001). Figure S2b. The cumulative incidence of III–IV aGVHD: HID10.11 ± 0.10%, MRD1.45 ± 0.02% (P = 0.026). Figure S3a. The cumulative incidence of cGVHD (P < 0.001). Figure S3b. The cumulative incidence of extensive cGVHD (P = 0.426). (DOCX 42 kb)
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