Jia Chen, Yabo Mei, Liu He, Zhenlan Du, Guosheng Xing, Xue Du, Liping Cheng, Xiaofei Wei, Danhua Zhao, Yanan Gu, Qiuping Li, Zhichun Feng
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引用次数: 0
Abstract
Background: Preclinical evidence supports the potential use of human umbilical cord blood-derived mononuclear cells (hUCB-MNCs) for preterm infants with preterm birth-associated complications (PBAC). Allogeneic hUCB-MNCs could extend this therapeutic option to infants without autologous cord blood available at birth.
Methods: This phase I, open-label, single-arm, and single-center study enrolled 10 preterm infants (gestational age [GA] 27.1 ± 0.7 weeks) with two or more PBAC. All received a single intravenous infusion of allogeneic, partially or fully human leukocyte antigen (HLA)-mismatched hUCB-MNCs without preconditioning or immunosuppression. The median cell was (3.51 ± 0.93) × 106 cells/kg. Safety, adverse events, and clinical outcomes were assessed over a 2-year follow-up period.
Results: All infants survived intensive care unit discharge. Four infants were lost to follow-up for parental reasons, with no in-hospital adverse events noted, their long-term outcomes remain unconfirmed. No infusion-related adverse events, acute reactions, or graft-versus-host disease (GVHD) were detected. One case of cerebral palsy was diagnosed during follow-up and was unrelated to cell infusion. Five of the six infants with evaluable long-term data had no respiratory, developmental, or neurological complications. Plasma inflammatory cytokines exhibited exploratory changes after infusion, without confirmed causality. All infants with follow-up data reached age-appropriate growth at 12 and 24 months of corrected age.
Conclusions: No major infusion-related adverse events were observed in preterm infants with PBAC receiving allogeneic hUCB-MNC infusion. Six infants completed 2-year follow-up and four were lost to follow-up. Constrained by small sample size and attrition, the results need further verification in larger controlled trials.
Trial registration: Chinese Clinical Trial Registry: ChiCTR-OPN-15006932, ChiCTR2000035227.
期刊介绍：
Stem Cells International is a peer-reviewed, Open Access journal that publishes original research articles, review articles, and clinical studies in all areas of stem cell biology and applications. The journal will consider basic, translational, and clinical research, including animal models and clinical trials.
Topics covered include, but are not limited to: embryonic stem cells; induced pluripotent stem cells; tissue-specific stem cells; stem cell differentiation; genetics and epigenetics; cancer stem cells; stem cell technologies; ethical, legal, and social issues.
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