Therapeutic potential of human embryonic stem cell transplantation in patients with cerebral palsy

Journal of Translational Medicine

Jitender Kumar Barthakur¹  Anupama Gupta²  Geeta Shroff³ 

[1] Ministry of Home Affairs, Government of India, New Delhi, India;Pediatrician, Max Hospital, Saket, New Delhi, India;Nutech Mediworld, H-8, Green Park Extension, New Delhi 110016, India

关键词: R;    GMFCS-E &;    Transplantation;    Cerebral palsy;    Human embryonic stem cells;   

Others  :  1146731
DOI  :  10.1186/s12967-014-0318-7

 received in 2014-08-08, accepted in 2014-11-05,  发布年份 2014

【 摘 要 】

Background

The present study evaluated the efficacy and safety of human embryonic stem cell (hESC) therapy in patients with CP.

Materials and methods

This analysis included patients (30 days-18 yr) with documented diagnosis of CP. The study consisted of four treatment phases (T1, T2, T3, T4) separated by gap phases. Efficacy of hESC therapy was evaluated based on Gross Motor Function Classification Scores Expanded and Revised (GMFCS-E & R; 1-good to 5-bad).

Results

Ninety one patients were included and all received hESC therapy in T1, 66 patients returned for T2, 38 patients for T3, and 15 patients for T4. Overall, 30.2% patients achieved GMFCS-E & R score 1 during the study with different number of patients achieving GMFCS score 1 by the end of each treatment phase (T1: 6 [6.6%]; T2: 7 [10.6%]; T3: 11 [28.9%]; and T4: 5 [33.3%]). All patients in up to 2 yr (n = 10), 2-4 yr (n = 10), 4-6 yr (n = 9), and 6-12 yr (n = 8) age groups except one of the 5 patients in the age group of 12-18 yr transitioned from GMFCS-E & R score 5 to lower scores by end of T1. Most patients transitioned to GMFCS-E & R score 2 (n = 34) from higher scores by end of T2. Eleven patients achieved GMFCS-E & R score 1 by end of T3. No serious adverse events were observed.

Conclusion

Use of hESC therapy in patients with CP is effective and safe. hESC therapy has demonstrated significant improvement in GMFCS-E & R scale.

【 授权许可】

   
2014 Shroff et al.; licensee BioMed Central.

【 图 表 】

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