| Objective: To evaluate the therapeutic effect and safety of mouse nerve growthfactor(mNGF) in the treatment of childhood spinal muscular atrophy(SMA).Method: A prospective, self-control and randomised crossover multicentre studywas conducted（ChiCTR-TRC-10001093）. A total of48cases of SMA childrencollected from November2010to November2011in pediatric of PLA GeneralHospital, neurology department of Beijing Children Hospital and Capital pediatricinstitute were randomly divided into two groups. A group received mNGF injectionfor24weeks firstly and then under observation for12weeks; B group firstly underobservation for12weeks and then received mNGF injection for24weeks. Thechanges of clinical symptom and physical signs, electromyogram(EMG), PediatricEvaluation of Disability Inventory(PEDI) was observed before and after treatment.Menawhile, the drug side effect was observed and recorded. In A group,9patientshave been finished follow-up,14patients are currently in progress, and none of themdrop out; in B group,6patients have been finished follow-up,16patients are underit, and3patients lost to it. The data of15patients finished all follow-up wasstatistically analyzed.Result: All15patients have completed evaluation of therapeutic effect and security.The muscular tension and muscle strength was enhanced. EMG parametes(includingthe nerve conduction velocity and the amplitude of Compound muscle actionpotential)were improved a little but there was no significant difference comparedwith before and after24weeks treatment. The self-care value of functional skills inSMA type Ⅲ patients was improved significantly compared with SMA typeⅡ patients before and after24weeks treatment(P＜0.05); the caregiver assitance scalesafter treatment of24weeks were improved significantly compared with the scales of12weeks(P＜0.05). There was no side effect correlated with mNGF therapy forSMA patients.Conclusion: Mouse nerve growth factor in the preliminary treatment of childhoodspinal muscular atrophy got a positive effect, high safety, worthy of promotion. |
