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人同种异体AB0/Rh血型相同的脐带血细胞治疗青少年脑瘫患者。

Human allogeneic AB0/Rh-identical umbilical cord blood cells in the treatment of juvenile patients with cerebral palsy.

作者信息

Romanov Yury A, Tarakanov Oleg P, Radaev Sergey M, Dugina Tamara N, Ryaskina Svetlana S, Darevskaya Anna N, Morozova Yana V, Khachatryan William A, Lebedev Konstantin E, Zotova Nelli S, Burkova Anna S, Sukhikh Gennady T, Smirnov Vladimir N

机构信息

Laboratory of Human Stem Cells, National Cardiology Research Center, Moscow, Russian Federation.

Cord Blood Bank "CryoCenter", Moscow, Russian Federation.

出版信息

Cytotherapy. 2015 Jul;17(7):969-78. doi: 10.1016/j.jcyt.2015.02.010. Epub 2015 Mar 17.

DOI:10.1016/j.jcyt.2015.02.010
PMID:25791070
Abstract

BACKGROUND AIMS

The term "cerebral palsy" (CP) encompasses many syndromes that emerge from brain damage at early stages of ontogenesis and manifest as the inability to retain a normal body position or perform controlled movements. Existing methods of CP treatment, including various rehabilitation strategies and surgical and pharmacological interventions, are mostly palliative, and there is no specific therapy focused on restoring injured brain function.

METHODS

During a post-registration clinical investigation, the safety and efficacy of intravenous infusion of allogeneic human leukocyte antigen (HLA)-unmatched umbilical cord blood (UCB) cells were studied in 80 pediatric patients with cerebral palsy and associated neurological complications. Patients received up to 6 intravenous infusions of AB0/Rh-identical, red blood cell-depleted UCB cells at an average dose of 250 × 10(6) viable cells per infusion.

RESULTS

Patients were followed for 3-36 months, and multiple cell infusions did not cause any adverse effects. In contrast, in most patients who received four or more UCB cell infusions, positive dynamics related to significant improvements in neurological status and/or cognitive functions were observed.

CONCLUSIONS

The results confirm that multiple intravenous infusions of allogeneic AB0/Rh-identical UCB cells may be a safe and effective procedure and could be included in treatment and rehabilitation programs for juvenile patients with cerebral palsy.

摘要

背景目的

“脑瘫”(CP)一词涵盖了许多在个体发育早期因脑损伤而出现的综合征,表现为无法保持正常体位或进行可控运动。现有的脑瘫治疗方法,包括各种康复策略以及手术和药物干预,大多是姑息性的,且尚无专注于恢复受损脑功能的特异性疗法。

方法

在一项注册后临床研究中,对80例患有脑瘫及相关神经并发症的儿科患者进行了静脉输注非匹配人类白细胞抗原(HLA)的脐带血(UCB)细胞的安全性和有效性研究。患者接受了多达6次静脉输注AB0/Rh血型相同、去除红细胞的UCB细胞,每次输注的平均剂量为250×10⁶个活细胞。

结果

对患者进行了3至36个月的随访,多次细胞输注未引起任何不良反应。相反,在大多数接受四次或更多次UCB细胞输注的患者中,观察到与神经状态和/或认知功能显著改善相关的积极变化。

结论

结果证实,多次静脉输注AB0/Rh血型相同的异体UCB细胞可能是一种安全有效的治疗方法,可纳入脑瘫青少年患者的治疗和康复方案中。

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