Il trattamento delle paralisi cerebrali infantili: sperimentazioni cliniche e ricadute assistenziali di nuovi approcci terapeutici e riabilitativi The treatment of infant cerebral palsy: clinical experimental trials of new therapeutic approaches and outcomes on health care

Background In the last decades, several treatment approaches have been used to improve upper limb function in hemiplegic CP. Only recently has Constraint Induced Movement Therapy (CIMT) emerged as a treatment approach for children with hemiplegic CP with the aim of reversing the behavioral suppression of movement in the affected upper limb. To date, evidence on this treatment has been very poor and limited, since all currently available trials reveal methodological limitations and a need for additional research to support the application of this treatment technique. Aim The thesis aims at exploring the safety and efficacy of a new treatment approach, Constraint Induced Movement Therapy has been studied and compared to a bimanual intensive rehabilitation approach and to traditional rehabilitation program. Methods This thesis presents the planning and development of a national mustisite clinical trial started in 2006 and carried out in collaboration with 21 Italian rehabilitations centers belonging to the Italian Group for Cerebral Palsy (GIPCI). The effectiveness and safety of CIMT combined with an intensive rehabilitation program was compared to 2 comparison groups: one treated with an intensive rehabilitation program and the other with standard treatment. Patients with hemiplegic cerebral palsy, aged between 2 and 8 years, who have never undergone constraint therapy have been recruited. Primary outcome measures include 2 major domains: UE motor ability (QUEST) and hand function assessment evaluating both grip function and spontaneous use of the affected side (Besta Scale). Secondary outcome measures concern overall function, behavior, compliance and satisfaction with treatment program of both child and family. Patients’ follow-up assessment was performed at 3, 6 and 12 months after the end of treatment. Collected data have been analyzed through univariate and multivariate statistical analysis. The experimental phase was preceded by a standardized analysis or primary outcome measures (QUEST and Besta Scale) in order to evaluate the agreement among the assessors of all participating centers. All evaluators scored the same 84 video-recorded tests each (42 QUEST and 42 Besta Scales administered to 42 children, 2 per each participating center). The analysis evaluated the inter-rater agreement and the reliability of primary outcome measures. During the clinical trial all the professionals involved in the project have been interviewed to explore their opinion on conducting clinical research in the field of rehabilitation and in their daily practice. Results 105 children have been recruited (39 undergoing CIMT, 33 undergoing bimanual intensive rehabilitation program and 33 undergoing traditional rehabilitation program). The main results demonstrate a significant effect of both intensive programs on the upper limb function. CIMT results particularly effective in ameliorating the grasp function, while bimanual intensive rehabilitation program results particularly efficacious in activity of daily living tasks. The traditional treatment does not show a significant modification of upper limb function. The patterns of brain plasticity and the process cortical reorganization following injury seem to play a crucial role in upper limb function modifications after intense treatment. Secondary outcome measures show the importance of the intensity of treatment and follow-up to reduce family stress and behavioral disturbances in children. The agreement analysis demonstrated an overall good inter-rater agreement and the reliability of both scales in assessing hand and upper limb function. 68 professionals have been involved in the survey regarding research in rehabilitation. The main results show the importance of conducting research in rehabilitation not only to obtain evidence for new therapeutic approaches, but also to standardize the practices and to share and improve the ability to use new and standardized assessment tools. Conclusions The researches conducted and the results obtained seem particularly important for the current rehabilitation practice and for the organization of rehabilitation programs in the dedicated health care service. Further research is needed on these issues, since, if these results will be confirmed they could dramatically change the approach to children with hemiplegic cerebral palsy and modify sensitively their disease’s natural history.