Improved outcomes using unmanipulated haploidentical hematopoietic stem cells combined with third‐party umbilical cord blood transplantation for non‐malignant diseases in children: The experience of a single center

• Published in: Pediatric transplantation 2021-06, Vol.25 (4), p.e13995-n/a
• Main Authors: Tang, Xiangfeng, Yu, Zhang, Ping, Liu, Lu, Wei, Jing, Yuanfang, Cao, Xiuyan
• Format: Article
• Language: English
• Subjects: Adolescent; Bone Marrow Failure Disorders - mortality; Bone Marrow Failure Disorders - therapy; bone marrow failure syndromes; Child; Child, Preschool; children; Cord blood; Cord Blood Stem Cell Transplantation - methods; Female; Follow-Up Studies; haploidentical hematopoietic stem cell transplantation; Hematopoietic Stem Cell Transplantation - methods; Hematopoietic stem cells; Humans; inborn errors of metabolism; Infant; Kaplan-Meier Estimate; Leukocytes (neutrophilic); Male; Metabolism, Inborn Errors - mortality; Metabolism, Inborn Errors - therapy; non‐malignant diseases; Pediatrics; primary immunodeficiency disease; Primary Immunodeficiency Diseases - mortality; Primary Immunodeficiency Diseases - therapy; Retrospective Studies; Stem cell transplantation; Stem cells; Transplantation, Haploidentical - methods; Treatment Outcome; Umbilical cord; Viremia
• ISSN: 1397-3142; 1399-3046
• DOI: 10.1111/petr.13995

Background Unmanipulated haploid HSCT for SAA has resulted in improved outcomes over recent years. However, studies related to unmanipulated haploid HSCs combined with tp‐UCB transplantation for other types of NMD are rare. Accordingly, we present the outcomes of 109 pediatric patients with life‐threatening NMD undergoing unmanipulated haploid HSCs combined with tp‐UCB transplantation. Procedure We retrospectively investigated 109 pediatric patients with life‐threatening NMD treated with unmanipulated haploid HSCs combined with tp‐UCB transplantation in a single center. Results The median days of neutrophil and platelet engraftment were +13 and +22 days, respectively. None of the cases experienced PGF. The incidence rates for grade I‐II, III‐IV aGVHD and cGVHD were 44.9%, 24.8%, and 9.3%, respectively. The incidence rates of CMV and EBV viremia were 46.7% and 39.4%, respectively. The median follow‐up duration was 997 days. In total, 106 patients survived, including 104 cases with FFS and 2 cases with SGF. Three patients died. The 5‐year TRM, OS, and FFS were 2.8%, 97.2%, and 96.2%, respectively. Conclusion The results of unmanipulated haploid HSCs combined with tp‐UCB in pediatric patients with life‐threatening NMD were promising. However, further research is now needed to determine specific factors that might influence the engraftment of HSCs.