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. 2021 Jan-Dec:30:963689721994808.
doi: 10.1177/0963689721994808.

Co-Transplantation of Haploidentical Stem Cells and a Dose of Unrelated Cord Blood in Pediatric Patients with Thalassemia Major

Xiaodong Wang  1 , Xiaoling Zhang  1 , Uet Yu  1 , Chunjing Wang  1 , Chunlan Yang  1 , Yue Li  1 , Changgang Li  1 , Feiqiu Wen  1 , Chunfu Li  2   3 , Sixi Liu  1
Affiliations

Co-Transplantation of Haploidentical Stem Cells and a Dose of Unrelated Cord Blood in Pediatric Patients with Thalassemia Major

Xiaodong Wang et al. Cell Transplant. 2021 Jan-Dec.

Abstract

Allogeneic stem cell transplantation is a cure for patients suffering from thalassemia major (TM). Historically, patients were limited by the selection of donors, while the advancement of haploidentical stem cell transplantation (haplo-SCT) has greatly expanded the donor pool. However, the outcomes of haplo-SCT in TM recipients vary between different programs. In this study, we retrospectively studied 73 pediatric TM patients (median age, 7 years; range, 3 to 14 years) who underwent haplo-cord transplantation. Both the estimated overall survival and transfusion-free survival were 95.26% (CI 95.77% to 96.23%). Neither primary nor secondary graft failures were observed. The median follow-up period was 811 days (range, 370 to 1433 days). Median neutrophil and platelet engraftment times were 22 days (range, 8 to 48 days) and 20 days (range, 8 to 99 days), respectively. Acute graft-versus-host disease (aGVHD) was observed in 52% of patients and of these, 25% developed grade III to IV aGVHD. Cord blood engraftment was associated with delayed immune recovery and increased aGVHD severity. Viral DNAemia occurred in a relatively high proportion of patients but only 7% of patients developed CMV disease, while another 7% of patients had post-transplantation lymphoproliferative disorder. Long-term complication outcomes were good. Only one patient developed extensive chronic GVHD. No surviving patients were reliant on blood transfusion by the time this manuscript was submitted. This is one of the largest studies on the outcomes of pediatric TM patients who received stem cell transplantations from alternative donors. The haplo-cord program is safe and practical for TM patients that do not have matched donors.

Keywords: cord blood transplant; haploidentical stem cell transplantation; thalassemia.

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Conflict of interest statement

Declaration of Conflicting Interests: The author(s) declared no potential conflicts of interest with respect to the research, authorship, and/or publication of this article.

Figures

Fig. 1.
Fig. 1.
Engraftment of patients that received haploidentical grafts or cord blood. (A) Neutrophil and (B) platelet recovery in patients with haploidentical (Haplo) or cord blood (CB) engraftments (mean ± SD, Haplo vs. CB; (A) 21 ± 4 days vs. 30 ± 8 days and (B) 17 ± 7 days vs. 45 ± 25 days; ****p < 0.001, Mann-Whitney U test).
Fig. 2.
Fig. 2.
The estimated three-year overall survival (OS) and transfusion-free survival (TFS) for pediatric thalassemia major patients who received haplo-cord transplantations.
Fig. 3.
Fig. 3.
Acute graft-versus-host disease (aGVHD) severity and viral DNAemia rates in patients engrafted with haploidentical grafts (Haplo) or cord blood (CB). (A) The severity of aGVHD in patients with Haplo or CB engraftments (****p < 0.001, Fisher’s exact test). (B) The number of patients with post-hematopoietic stem cell transplantation viral DNAemia in patients that received Haplo or CB engraftments (****p < 0.001, Fisher’s exact test).
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  • [Chinese guideline for diagnosis and treatment of transfusion dependent β-thalassemia (2022)].
    Red Blood Cell Diseases (Anemia) Group, Chinese Society of Hematology, Chinese Medical Association. Red Blood Cell Diseases (Anemia) Group, Chinese Society of Hematology, Chinese Medical Association. Zhonghua Xue Ye Xue Za Zhi. 2022 Nov 14;43(11):889-896. doi: 10.3760/cma.j.issn.0253-2727.2022.11.002. Zhonghua Xue Ye Xue Za Zhi. 2022. PMID: 36709178 Free PMC article. Chinese. No abstract available.

References

    1. John MJ, Jyani G, Jindal A, Mashon RS, Mathew A, Kakkar S, Bahuguna P, Prinja S. Cost effectiveness of hematopoietic stem cell transplantation compared with transfusion chelation for treatment of thalassemia major. Biol Blood Marrow Transplant. 2018;24(10):2119–2126. - PubMed
    1. Galanello R, Origa R. Beta-thalassemia. Orphanet J Rare Dis. 2010;5:11. - PMC - PubMed
    1. Mohamed SY. Thalassemia major: transplantation or transfusion and chelation. Hematol Oncol Stem Cell Ther. 2017;10(4):290–298. - PubMed
    1. Airoldi I, Bertaina A, Prigione I, Zorzoli A, Pagliara D, Cocco C, Meazza R, Loiacono F, Lucarelli B, Bernardo ME, Barbarito G, et al. γδ T-cell reconstitution after HLA-haploidentical hematopoietic transplantation depleted of TCR-αβ+/CD19+ lymphocytes. Blood. 2015;125(15):2349–2358. - PMC - PubMed
    1. Xu LP, Wu DP, Han MZ, Huang H, Liu QF, Liu DH, Sun ZM, Xia LH, Chen J, Wang HX, Wang C, et al. A review of hematopoietic cell transplantation in China: data and trends during 2008-2016. Bone Marrow Transplant. 2017;52(11):1512–1518. - PubMed

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