Are We Getting It Right? A Scoping Review of Outcomes Reported in Cell Therapy Clinical Studies for Cerebral Palsy

Abstract

Cell therapies are an emergent treatment for cerebral palsy (CP) with promising evidence demonstrating efficacy for improving gross motor function. However, families value improvements in a range of domains following intervention and the non-motor symptoms, comorbidities and complications of CP can potentially be targeted by cell therapies. We conducted a scoping review to describe all outcomes that have been reported in cell therapy studies for CP to date, and to examine what instruments were used to capture these. Through a systematic search we identified 54 studies comprising 2066 participants that were treated with a range of cell therapy interventions. We categorized the reported 53 unique outcome instruments and additional descriptive measures into 10 categories and 12 sub-categories. Movement and Posture was the most frequently reported outcome category, followed by Safety, however Quality of Life, and various prevalent comorbidities and complications of CP were infrequently reported. Notably, many outcome instruments used do not have evaluative properties and thus are not suitable for measuring change following intervention. We provide a number of recommendations to ensure that future trials generate high-quality outcome data that is aligned with the priorities of the CP community.

Allogeneic mesenchymal stem cells may be a viable treatment modality in cerebral palsy

“…According to a systematic review that listed all findings after cell therapy in CP and the measurement tools used, 2066 participants have undergone various cell therapy interventions in 54 trials[ 43 ]. Movement and posture were the most frequently reported outcome categories, followed by safety, although quality of life and various common comorbidities and complications associated with CP have rarely been reported[ 43 ].…”

Editorial Highlights from the Comorbidities and Complications of Cerebral Palsy Special Issue

“…With the rising interest in stem cell infusions, a scoping review of the outcome measures used post-stem cell infusions showed that most measures did not have the requisite measurement properties to validly measure a change following the intervention. The authors’ recommendations about the outcome measure selection include ensuring that the captured outcomes aligned with the priorities of the cerebral palsy community, such as quality of life [ 4 ]. Another paper focused on biomarkers and the role of inflammation in cerebral palsy, which are being increasingly studied.…”