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Abstract
_% V( }9 v M5 yBackground
4 W& @& q9 G* b: g5 `Autism is a pervasive neurodevelopmental disorder. At present there are no defined
/ G5 [3 n; D6 i. f, b" r1 Cmechanisms of pathogenesis and therapy is mostly limited to behavioral interventions. Stem
1 W; A! G+ q8 s5 f3 z& O. mcell transplantation may offer a unique treatment strategy for autism due to immune and ) G/ c, j. v) @9 W* x: c
neural dysregulation observed in this disease. This non-randomized, open-label, single center
1 \, Z3 s2 q9 i/ W' p9 uphase I/II trial investigated the safety and efficacy of combined transplantation of human cord
. t _6 q2 R; N+ k% a: N0 Dblood mononuclear cells (CBMNCs) and umbilical cord-derived mesenchymal stem cells
$ l; }0 A+ \7 d. i+ g; {& u(UCMSCs) in treating children with autism.
8 A) A5 ~: y# y8 x. m. KMethods ; ~ h4 W- O. L: Q; a, J6 t
37 subjects diagnosed with autism were enrolled into this study and divided into three groups:
; v* x, l7 V# K8 RCBMNC group (14 subjects, received CBMNC transplantation and rehabilitation therapy),
& |8 E$ t, p. V& S/ N9 M" W& bCombination group (9 subjects, received both CBMNC and UCMSC transplantation and 0 `* {+ w+ ~3 R- _4 R, [4 q
rehabilitation therapy), and Control group (14 subjects, received only rehabilitation therapy).
$ o. M/ p3 G2 ?; R5 N% J- XTransplantations included four stem cell infusions through intravenous and intrathecal 1 b. r7 Y9 N/ {, i$ d
injections once a week. Treatment safety was evaluated with laboratory examinations and
: }4 z9 r% O! H0 Vclinical assessment of adverse effects. The Childhood Autism Rating Scale (CARS), Clinical 4 ^2 z! H1 Y3 ]* h2 ^
Global Impression (CGI) scale and Aberrant Behavior Checklist (ABC) were adopted to
2 x T' e# e; d, A& @4 gassess the therapeutic efficacy at baseline (pre-treatment) and following treatment. , o) N: ?0 e8 v& u
Results
3 V3 g4 Y* z- E- ]0 `There were no significant safety issues related to the treatment and no observed severe 7 I3 f: V9 N: m
adverse effects. Statistically significant differences were shown on CARS, ABC scores and 4 w5 f. r! W K8 m. P- f
CGI evaluation in the two treatment groups compared to the control at 24 weeks post-
" {+ [0 K( f7 Htreatment (p < 0.05).
* f2 d% c& G# m/ Z" |( |( n% n. nConclusions 3 C6 r# f: I- S- `
Transplantation of CBMNCs demonstrated efficacy compared to the control group; however,
! Q) }) T s- s p; tthe combination of CBMNCs and UCMSCs showed larger therapeutic effects than the
1 w# E) N; x8 x! \" g4 tCBMNC transplantation alone. There were no safety issues noted during infusion and the # f3 f4 v+ G6 J( x2 y
whole monitoring period. Trial registration
5 l/ }7 P, n6 e; K: NClinicalTrials.gov: NCT01343511, Title “Safety and Efficacy of Stem Cell Therapy in & A/ [' L8 e+ I$ T7 f
Patients with Autism”.
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发表于 2013-9-2 15:29
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发表于 2013-11-23 22:18
