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Abstract
- X. o ^, q) h ]$ e1 mBackground 2 M: [) o7 u) x6 O6 R
Autism is a pervasive neurodevelopmental disorder. At present there are no defined ; ^% }) x4 T& ?7 E% Z3 b4 q! ~6 P
mechanisms of pathogenesis and therapy is mostly limited to behavioral interventions. Stem 8 @# H/ c/ q* ]/ F0 ?& x
cell transplantation may offer a unique treatment strategy for autism due to immune and
$ B; E! L* N, S: c/ v' aneural dysregulation observed in this disease. This non-randomized, open-label, single center " |; G. |' A! D- q
phase I/II trial investigated the safety and efficacy of combined transplantation of human cord
* d- ^ u) T9 ]9 }blood mononuclear cells (CBMNCs) and umbilical cord-derived mesenchymal stem cells 2 K i* k% x4 Y+ t" \/ v+ F) {
(UCMSCs) in treating children with autism.
5 d( \& d4 s5 R! q( FMethods
4 j) R. ^! U9 Q4 m37 subjects diagnosed with autism were enrolled into this study and divided into three groups:
- @" l, E" C' h( A0 wCBMNC group (14 subjects, received CBMNC transplantation and rehabilitation therapy), $ J6 a8 X# E- B
Combination group (9 subjects, received both CBMNC and UCMSC transplantation and
, }" T+ A5 y. \4 ~+ D) ]7 J% ]0 Xrehabilitation therapy), and Control group (14 subjects, received only rehabilitation therapy).
. p$ o- \: Y# f4 ^, W6 OTransplantations included four stem cell infusions through intravenous and intrathecal 6 Z1 P# D, ?: \5 I
injections once a week. Treatment safety was evaluated with laboratory examinations and
1 y L2 m# @: Kclinical assessment of adverse effects. The Childhood Autism Rating Scale (CARS), Clinical / e0 E5 d& p/ i: y4 r
Global Impression (CGI) scale and Aberrant Behavior Checklist (ABC) were adopted to
$ s/ A0 u: [) Oassess the therapeutic efficacy at baseline (pre-treatment) and following treatment.
4 ? Q# t: T" \! zResults
; K5 g& {; v" f1 m. GThere were no significant safety issues related to the treatment and no observed severe 5 E1 h; [2 m* r. Z
adverse effects. Statistically significant differences were shown on CARS, ABC scores and
9 n1 Q/ u' K7 R$ i+ T" ~- sCGI evaluation in the two treatment groups compared to the control at 24 weeks post-' K, k' _" o- T9 e* P/ }
treatment (p < 0.05). * D0 _- m2 T: O- f1 M0 C1 V
Conclusions $ T8 E7 t3 T. J. Q9 f" H9 z4 V
Transplantation of CBMNCs demonstrated efficacy compared to the control group; however, 4 u+ f& I& h0 u! e' X1 V! N6 S3 E
the combination of CBMNCs and UCMSCs showed larger therapeutic effects than the
7 [+ \1 v4 L G% V0 H, gCBMNC transplantation alone. There were no safety issues noted during infusion and the
3 {# k: }! l2 V- ?whole monitoring period. Trial registration
$ N# n. @. t5 C& N% HClinicalTrials.gov: NCT01343511, Title “Safety and Efficacy of Stem Cell Therapy in
+ [, W( a B' z# g: d* G; q; YPatients with Autism”. 3 k5 w- ~8 {/ u: d; _8 Y
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发表于 2013-9-2 15:29
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发表于 2013-11-23 22:18
