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Comparative Study
. 2022 Feb 1:13:837335.
doi: 10.3389/fimmu.2022.837335. eCollection 2022.

Comparison of Hematopoietic Stem Cell Transplantation Outcomes Using Matched Sibling Donors, Haploidentical Donors, and Immunosuppressive Therapy for Patients With Acquired Aplastic Anemia

Yuanfeng Zhang  1   2 , Jiali Huo  1 , Li Liu  1 , Yuyan Shen  1 , Juan Chen  1 , Tingting Zhang  1 , Xin Chen  1 , Aiming Pang  1 , Donglin Yang  1 , Rongli Zhang  1 , Qiaoling Ma  1 , Weihua Zhai  1 , Yi He  1 , Jialin Wei  1 , Erlie Jiang  1 , Mingzhe Han  1 , Yizhou Zheng  1 , Sizhou Feng  1
Affiliations
Comparative Study

Comparison of Hematopoietic Stem Cell Transplantation Outcomes Using Matched Sibling Donors, Haploidentical Donors, and Immunosuppressive Therapy for Patients With Acquired Aplastic Anemia

Yuanfeng Zhang et al. Front Immunol. .

Abstract

We retrospectively compared the outcomes of 387 consecutive patients with acquired aplastic anemia (AA) who underwent hematopoietic stem cell transplantation (HSCT) with a fludarabine-based conditioning regimen from matched sibling donors (MSD) (n = 108) or haploidentical donors (HID) (n = 91) and immunosuppressive therapy (IST) (n = 188) from 2014 to 2020 at our hospital. Compared with HID-HSCT, MSD-HSCT had a lower incidence of graft failure (1% vs. 7%, p = 0.062), grade II-IV acute graft versus host disease (aGvHD) (16% vs. 35%, p = 0.001), and mild to severe chronic GvHD (cGvHD) (8% vs. 23%, p = 0.007), but an equivalent incidence of grade III-IV aGvHD (8% vs. 12%, p = 0.237) and moderate to severe cGvHD (3% vs. 9%, p = 0.076). HSCT had superior blood count recovery at 3, 6, and 12 months compared with IST (p < 0.001). The estimated 5-year overall survival (OS) of the MSD, HID, and IST groups were 86%, 72%, and 79% (p = 0.02), respectively; accordingly, the failure-free survival (FFS) rates were 85%, 68%, and 56%, respectively (p < 0.001). For patients aged ≤40 years, the OS rate was still significantly superior for MSD-HSCT receipients compared to HID-HSCT receipients (89% vs. 76%, p = 0.024) while the HID-HSCT recipients showed similar OS (76% vs. 78%, p = 0.166) but superior FFS (p = 0.047) when follow-up was longer than 14.5 months in contrast to IST. In a multivariate analysis, HID-HSCT and a conditioning regimen that included busulfan were adversely related to OS among patients who received allografts. In conclusion, MSD-HSCT was the frontline choice for patients with severe AA aged ≤40 years, while HID-HSCT was as effective as IST for patients without an MSD.

Keywords: aplastic anemia; haploidentical donor; immunosuppressive therapy; matched sibling donor; transplantation.

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Conflict of interest statement

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

Figures

Figure 1
Figure 1
Cumulative incidence (CI) of grade II–IV aGvHD (A), grade III–IV aGvHD (B), mild to severe cGvHD (C), and moderate to severe cGvHD (D) in MSD-HSCT and HID-HSCT patients.
Figure 2
Figure 2
Therapy-related mortality (TRM) (A), overall survival (OS) (B), failure-free survival (FFS) (C), and GvHD-free, failure-free survival (GFFS) (D) of MSD-HSCT, HID-HSCT, and IST patients.
Figure 3
Figure 3
Overall survival (OS) (A) and failure-free survival (FFS) (B) following MSD-HSCT, HID-HSCT, and IST for patients aged ≤40 years.
Figure 4
Figure 4
Overall survival (OS) of patients aged ≤40 years stratified by conditioning regimen in MSD (A) and HID (B) patients.
See this image and copyright information in PMC

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