Abstract:All 3 scoring options showed large longitudinal construct validity in the long-term follow-up. The GMFM-88 total and goal total scores revealed large changes in gross motor function earlier postoperatively than the GMFM-66 scores.
“…Muscle strength was evaluated by the physiotherapist. Grading was according to the gross motor function measure score sheet (GMFM‐88) . The GMFM‐88 is a standardized observational instrument designed and validated to measure change in gross motor function over time in children.…”
CPET is safe for pediatric Pompe patients. ERT may benefit exercise capacity in patients with less advanced disease. Individualized assessment by CPET, 6MWT, and motor function may help ERT adjustment by providing precise quantification of the response to treatment. Additional studies are needed to clarify the benefit of this assessment protocol.
“…Muscle strength was evaluated by the physiotherapist. Grading was according to the gross motor function measure score sheet (GMFM‐88) . The GMFM‐88 is a standardized observational instrument designed and validated to measure change in gross motor function over time in children.…”
CPET is safe for pediatric Pompe patients. ERT may benefit exercise capacity in patients with less advanced disease. Individualized assessment by CPET, 6MWT, and motor function may help ERT adjustment by providing precise quantification of the response to treatment. Additional studies are needed to clarify the benefit of this assessment protocol.
BackgroundStem cell therapy is a promising treatment for cerebral palsy, which refers to a category of brain diseases that are associated with chronic motor disability in children. Autologous MSCs may be a better cell source and have been studied for the treatment of cerebral palsy because of their functions in tissue repair and the regulation of immunological processes.MethodsTo assess neural stem cell–like (NSC-like) cells derived from autologous marrow mesenchymal stem cells as a novel treatment for patients with moderate-to-severe cerebral palsy, a total of 60 cerebral palsy patients were enrolled in this open-label, non-randomised, observer-blinded controlled clinical study with a 6-months follow-up. For the transplantation group, a total of 30 cerebral palsy patients received an autologous NSC-like cells transplantation (1-2 × 107 cells into the subarachnoid cavity) and rehabilitation treatments whereas 30 patients in the control group only received rehabilitation treatment.ResultsWe recorded the gross motor function measurement scores, language quotients, and adverse events up to 6 months post-treatment. The gross motor function measurement scores in the transplantation group were significantly higher at month 3 (the score increase was 42.6, 95% CI: 9.8–75.3, P=.011) and month 6 (the score increase was 58.6, 95% CI: 25.8–91.4, P=.001) post-treatment compared with the baseline scores. The increase in the Gross Motor Function Measurement scores in the control group was not significant. The increases in the language quotients at months 1, 3, and 6 post-treatment were not statistically significant when compared with the baseline quotients in both groups. All the 60 patients survived, and none of the patients experienced serious adverse events or complications.ConclusionOur results indicated that NSC-like cells are safe and effective for the treatment of motor deficits related to cerebral palsy. Further randomised clinical trials are necessary to establish the efficacy of this procedure.
“…The GMFM‐66 is one version, in which a child’s gross motor function is observed and tested against 66 standardized items. It has been found to be valid and reliable for children with CP and has good longitudinal construct validity 19–21 . GMFM‐66 scores were obtained using the Gross Motor Ability Estimator 22 .…”
Aim The aim of this study was to explore changes in motor function up to 10 years after selective dorsal rhizotomy (SDR).
Method The participants comprised 29 children (20 males, nine females) with bilateral spastic diplegia who were consecutively operated on at a median age of 4 years and 3 months and followed until a median age of 15 years. SDR was combined with physiotherapy and regular follow‐up visits. The distribution of preoperative Gross Motor Function Classification System (GMFCS) levels was as follows: I, n=1; II, n=7; III, n=8; IV, n=12; and V, n=1. Muscle tone in hip flexors, hip adductors, knee flexors, and plantar flexors was assessed with the modified Ashworth scale, passive range of motion in hip abduction, popliteal angle, maximum knee extension, dorsiflexion of the foot was measured with a goniometer, and gross motor function was assessed using the Gross Motor Function Measure (GMFM‐66). The results were compared with preoperative values, taking into account age at the time of SDR.
Results After 10 years, muscle tone in hip flexors, hip adductors, knee flexors and plantar flexors was normalized in 19, 24, 13 and 23 participants respectively; mean change in passive range of motion ranged from −2.0° to 8.6°, and the mean increase in GMFM‐66 was 10.6. Changes in GMFM‐66 were associated with preoperative GMFCS level and GMFM‐66 scores.
Interpretation Children who underwent SDR and physiotherapy and were regularly followed up by an experienced team showed improved gross motor function for up to 10 years postoperatively.
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