PMID- 32014055 OWN - NLM STAT- MEDLINE DCOM- 20210311 LR - 20240328 IS - 1757-6512 (Electronic) IS - 1757-6512 (Linking) VI - 11 IP - 1 DP - 2020 Feb 3 TI - Therapeutic evidence of umbilical cord-derived mesenchymal stem cell transplantation for cerebral palsy: a randomized, controlled trial. PG - 43 LID - 10.1186/s13287-019-1545-x [doi] LID - 43 AB - BACKGROUND: Cerebral palsy (CP) is a syndrome of childhood movement and posture disorders. Clinical evidence is still limited and sometimes inconclusive about the benefits of human umbilical cord mesenchymal stem cells (hUC-MSCs) for CP. We conducted a randomized trial to evaluate the safety and efficacy of hUC-MSC transplantation concomitant with rehabilitation in patients with CP. METHODS: Eligible patients were allocated into the hUC-MSC group and control group. In addition to rehabilitation, the patients in the hUC-MSC group received four transfusions of hUC-MSCs intravenously, while the control group received a placebo. Adverse events (AEs) were collected for safety evaluation in the 12-month follow-up phase. Primary endpoints were assessed as activities of daily living (ADL), comprehensive function assessment (CFA), and gross motor function measure (GMFM) scales. In addition, cerebral metabolic activity was detected by (18)F-FDG-PET/CT to explore the possible mechanism of the therapeutic effects. Primary endpoint data were analyzed by ANOVA using SPSS version 20.0. RESULTS: Forty patients were enrolled, and 1 patient withdrew informed consent. Therefore, 39 patients received treatments and completed the scheduled assessments. No significant difference was shown between the 2 groups in AE incidence. Additionally, significant improvements in ADL, CFA, and GMFM were observed in the hUC-MSC group compared with the control group. In addition, the standard uptake value of (18)F-FDG was markedly increased in 3 out of 5 patients from the hUC-MSC group at 12 months after transplantation. CONCLUSIONS: Our clinical data showed that hUC-MSC transplantation was safe and effective at improving the gross motor and comprehensive function of children with CP when combined with rehabilitation. Recovery of cerebral metabolic activity might play an essential role in the improvements in brain function in patients with CP. The therapeutic window, transfusion route, and dosage in our study were considerable for reference in clinical application. TRIAL REGISTRATION: Chictr.org.cn, ChiCTR1800016554. Registered 08 June 2018-retrospectively registered. The public title was "Randomized trial of umbilical cord-derived mesenchymal stem cells for cerebral palsy." FAU - Gu, Jiaowei AU - Gu J AD - Department of Neonatology, The First Affiliated Hospital of Xi'an Jiaotong University, No.277 West Yanta Road, Xi'an, 710061, Shaanxi, People's Republic of China. AD - Affiliated Taihe Hospital of Hubei University of Medicine, No. 32 Southern Renmin Road, Shiyan, 422000, Hubei, People's Republic of China. FAU - Huang, Li AU - Huang L AD - Affiliated Taihe Hospital of Hubei University of Medicine, No. 32 Southern Renmin Road, Shiyan, 422000, Hubei, People's Republic of China. FAU - Zhang, Che AU - Zhang C AD - Department of Neonatology, The First Affiliated Hospital of Xi'an Jiaotong University, No.277 West Yanta Road, Xi'an, 710061, Shaanxi, People's Republic of China. AD - Affiliated Taihe Hospital of Hubei University of Medicine, No. 32 Southern Renmin Road, Shiyan, 422000, Hubei, People's Republic of China. FAU - Wang, Yong AU - Wang Y AD - Affiliated Taihe Hospital of Hubei University of Medicine, No. 32 Southern Renmin Road, Shiyan, 422000, Hubei, People's Republic of China. FAU - Zhang, Ruibo AU - Zhang R AD - Affiliated Taihe Hospital of Hubei University of Medicine, No. 32 Southern Renmin Road, Shiyan, 422000, Hubei, People's Republic of China. FAU - Tu, Ziliang AU - Tu Z AD - Affiliated Taihe Hospital of Hubei University of Medicine, No. 32 Southern Renmin Road, Shiyan, 422000, Hubei, People's Republic of China. FAU - Wang, Hengdong AU - Wang H AD - Affiliated Taihe Hospital of Hubei University of Medicine, No. 32 Southern Renmin Road, Shiyan, 422000, Hubei, People's Republic of China. FAU - Zhou, Xihui AU - Zhou X AD - Department of Neonatology, The First Affiliated Hospital of Xi'an Jiaotong University, No.277 West Yanta Road, Xi'an, 710061, Shaanxi, People's Republic of China. FAU - Xiao, Zhousheng AU - Xiao Z AD - Department of Medicine, University of Tennessee Health Science Center, Memphis, TN, 38163, USA. FAU - Liu, Zegan AU - Liu Z AD - Affiliated Taihe Hospital of Hubei University of Medicine, No. 32 Southern Renmin Road, Shiyan, 422000, Hubei, People's Republic of China. FAU - Hu, Xiang AU - Hu X AD - Shenzhen Beike Biotechnology Co., Ltd, No. 18 Keyuan Road, Hi-Tech Industrial Park South Area, Shenzhen, 518057, People's Republic of China. FAU - Ke, Zunchen AU - Ke Z AD - Shiyan City Disabled Persons' Federation, No. 12 Beijing Road, Shiyan, 422000, Hubei, People's Republic of China. FAU - Wang, Dabin AU - Wang D AD - Affiliated Taihe Hospital of Hubei University of Medicine, No. 32 Southern Renmin Road, Shiyan, 422000, Hubei, People's Republic of China. FAU - Liu, Li AU - Liu L AUID- ORCID: 0000-0003-1200-3068 AD - Department of Neonatology, The First Affiliated Hospital of Xi'an Jiaotong University, No.277 West Yanta Road, Xi'an, 710061, Shaanxi, People's Republic of China. liuli918@163.com. LA - eng GR - R01 AR071930/AR/NIAMS NIH HHS/United States PT - Journal Article PT - Randomized Controlled Trial PT - Research Support, Non-U.S. Gov't DEP - 20200203 PL - England TA - Stem Cell Res Ther JT - Stem cell research & therapy JID - 101527581 SB - IM MH - Activities of Daily Living MH - Cerebral Palsy/*therapy MH - Child, Preschool MH - Double-Blind Method MH - Female MH - Humans MH - Male MH - Mesenchymal Stem Cell Transplantation/*methods MH - Mesenchymal Stem Cells/*metabolism PMC - PMC6998370 OTO - NOTNLM OT - Activities of daily living OT - Cell transplantation OT - Cerebral palsy OT - Clinical trial OT - Mesenchymal stem cells OT - Umbilical cord COIS- The authors declare that they have no competing interests. EDAT- 2020/02/06 06:00 MHDA- 2021/03/12 06:00 PMCR- 2020/02/03 CRDT- 2020/02/05 06:00 PHST- 2019/10/11 00:00 [received] PHST- 2019/12/29 00:00 [accepted] PHST- 2019/12/12 00:00 [revised] PHST- 2020/02/05 06:00 [entrez] PHST- 2020/02/06 06:00 [pubmed] PHST- 2021/03/12 06:00 [medline] PHST- 2020/02/03 00:00 [pmc-release] AID - 10.1186/s13287-019-1545-x [pii] AID - 1545 [pii] AID - 10.1186/s13287-019-1545-x [doi] PST - epublish SO - Stem Cell Res Ther. 2020 Feb 3;11(1):43. doi: 10.1186/s13287-019-1545-x.