Comparable Outcomes and Health-Related Quality of Life for Severe Aplastic Anemia: Haploidentical Combined With a Single Cord Blood Unit vs Matched Related Transplants.

Meiqing Lei,Xiaoli Li,Limin Liu,Qingyuan Wang,Aining Sun,Suning Chen,Xiaowen Tang,Yue Han,Huiying Qiu,Chengcheng Fu,Huifen Zhou,Yanming Zhang,Miao Miao,Zhengming Jin,Wenjing Jiao,Depei Wu,Qi Qu

doi:10.3389/fonc.2021.714033

Abstract

We retrospectively compared the outcomes and health-related quality of life (HRQoL) of severe aplastic anemia (SAA) patients who received haploidentical hematopoietic stem cell transplantation with a single unrelated cord blood unit (Haplo-cord HSCT) (n = 180) or matched related donor (MRD)-HSCT (n = 128). After propensity score matching, we were able to match 88 patients in each group and to compare the outcomes between the two matched-pair groups. Haplo-cord recipients exhibited a longer median days for neutrophil engraftment (12 vs 11, P = 0.001) and for platelet engraftment (15 vs 13, P = 0.003). Haplo-cord recipients a high cumulative incidence of grades II–IV acute graft-versus-host disease (GVHD) (29.8 vs 14.0%, P = 0.006), while similar III–IV acute GVHD, total chronic GVHD, and moderate to severe chronic GVHD at four-year (all P < 0.05). Among the Haplo-cord HSCT and MRD-HSCT groups, the four-year GVHD-free/failure-free survival rates were 73.5% and 66.9% (P = 0.388) respectively, and the overall survival rates were 81.5% and 77.2% (P = 0.484), respectively. Similar comparable results also were observed between the corresponding first-line, older or younger than 40 years old subgroups. The Haplo-cord HSCT group exhibited higher scores in the physical component summary, physical functioning, general health and social functioning than the MRD-HSCT group (all P < 0.05). In the multivariate analysis, young age and Haplo-cord HSCT were favorable factors for HRQoL, while moderate to severe cGVHD was associated with lower HRQoL. These results suggest that for SAA patients, Haplo-cord HSCT could achieve at least comparable efficacy and HRQoL to MRD-HSCT.

Highlights

Acquired severe aplastic anemia (SAA) is a kind of bone marrow (BM) failure syndromes mainly caused by immune destruction of hemopoietic stem and progenitor cells [1, 2]
More male donors were included in the Haplo-cord HSCT group than the matched related donor (MRD)-HSCT group (P = 0.013), which contributed to the difference in the donor-recipient sex match between the Haplo-cord HSCT and MRD-HSCT groups
graft-versus-host disease (GVHD) was a common complication after engraftment, and it might be directly related to survival and quality of life of the survivors, especially in severe cases

Summary

INTRODUCTION

Acquired severe aplastic anemia (SAA) is a kind of bone marrow (BM) failure syndromes mainly caused by immune destruction of hemopoietic stem and progenitor cells [1, 2]. No direct comparison was performed including the HRQoL in SAA patients treated with Haplo-cord HSCT and MRD-HSCT. The propensity score that indicated the HLA status for each patient was calculated based on a multivariate logistic regression model In this model, patient and donor age, female donor into male recipient, year of transplant (from January, 2014), disease status, and graft source between the two groups were used as covariates. Haplo-cord HSCT, haploidentical hematopoietic stem cell transplantation with unrelated cord blood infusion; MRD-HSCT, matched related donor hematopoietic stem cell transplantation; SAA, severe aplastic anemia; vSAA, very SAA; PNH, paroxysmal nocturnal haemoglobinuria; ECOG,eastern cooperative oncology group scale; RBC, red blood cell; PLT, paltelet; SF, serum ferritin; HCT-CI, hematopoietic stem cell transplantation-comorbidity index; BM, bone marrow; PB, peripheral blood; MNCs, mononuclear cells; TNCs, total nucleated cells. The bold values were statistically significant (P < 0.05)

RESULTS

DISCUSSION

ETHICS STATEMENT