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Open Access 01-12-2017 | Research

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

Authors: 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

Published in: Journal of Hematology & Oncology | Issue 1/2017

Abstract

Background

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.

Methods

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.

Results

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.

Conclusions

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

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Title: Upfront haploidentical transplant for acquired severe aplastic anemia: registry-based comparison with matched related transplant
Authors: 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
Publication date: 01-12-2017
Publisher: BioMed Central
Published in: Journal of Hematology & Oncology / Issue 1/2017
Electronic ISSN: 1756-8722
DOI: https://doi.org/10.1186/s13045-017-0398-y

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