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

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Journal of Hematology and Oncology

, 10:25

First Online: 21 January 2017Received: 15 July 2016Accepted: 12 January 2017DOI: 10.1186-s13045-017-0398-y

Cite this article as: Xu, LP., Jin, S., Wang, SQ. et al. J Hematol Oncol 2017 10: 25. doi:10.1186-s13045-017-0398-y


BackgroundHaploidentical 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.

MethodsWe 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.

ResultsEighty-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.

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

KeywordsUpfront Haploidentical transplantation Acquired severe aplastic anemia AbbreviationsAAAplastic anemia

aGVHDAcute graft-versus-host disease

ATGAntithymocyte globulin

BMBone marrow


CBMTRChinese Bone Marrow Transplantation Registry

CD25 mAbCD25 monoclonal antibody

cGVHDChronic graft-versus-host disease


CMV-CTLCytomegalovirus-specific cytotoxic T lymphocytes

CsACyclosporin A


EBVEpstein-Barr virus

ECOGEastern Cooperative Oncology Group

FDCFull donor chimerism

FFSFailure-free survival


GFFSGVHD-free, failure-free survival

HIDHaploidentical donor

HSCTHematopoietic stem cell transplantation

ISTImmunosuppressive therapy

KPSKarnofsky Performance Status Scale

MMFMycophenolate mofetil

MNCMononuclear cell

MRDMatched related donor


OSOverall survival

PBPeripheral blood

PTLDPost-transplant lymphoproliferative disorders

RBCRed blood cell

RRTRegimen-related toxicity

SAASevere aplastic anemia

TRMTransplantation-related mortality

Electronic supplementary materialThe online version of this article doi:10.1186-s13045-017-0398-y contains supplementary material, which is available to authorized users.

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Author: Lan-Ping Xu - Song Jin - Shun-Qing Wang - Ling-Hui Xia - Hai Bai - Su-Jun Gao - Qi-Fa Liu - Jian-Min Wang - Xin Wang - Mi

Source: https://link.springer.com/


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