Outcome tools used for ambulatory children with cerebral palsy: responsiveness and minimum clinically important differences

Abstract

This prospective longitudinal multicenter study of ambulatory children with cerebral palsy (CP) examined changes in outcome tool score over time, tool responsiveness, and used a systematic method for defining minimum clinically important differences (MCIDs). Three hundred and eighty‐one participants with CP (Gross Motor Function Classification System [GMFCS] Levels I–III; age range 4–18y, mean age 11y [SD 4y 4mo]; 265 diplegia, 116 hemiplegia; 230 males, 151 females). At baseline and follow‐up at least 1 year later, Functional Assessment Questionnaire, Gross Motor Function Measure, Pediatric Quality of Life Inventory, Pediatric Outcomes Data Collection Instrument, Pediatric Functional Independence Measure, temporal–spatial gait parameters, and oxygen cost were collected. Adjusted standardized response means determined tool responsiveness for nonsurgical (n=292) and surgical (n=87) groups at GMFCS Levels I to III. Most scores reaching medium or large effect sizes were for GMFCS Level III. Nonsurgical group change scores were used to calculate MCID thresholds for ambulatory children with CP. These values were verified by examining participants who changed GMFCS levels. Tools measuring function were responsive when a change large enough to cause a change in GMFCS level occurred. MCID thresholds assess change in study populations over time, and serve as the basis for designing prospective intervention studies.

Effectiveness of motor learning coaching in children with cerebral palsy: a randomized controlled trial

“…The significant increases in GMFM-66 over 3 months (Table 2) in both groups suggest that both treatments may have been beneficial, as changes 41.5 are considered clinically meaningful. 23 We noted that 6-month retention of gross motor function improvements was maintained only following motor learning coaching treatment, as GMFM-66 declined following neurodevelopment treatment ( Table 2). This suggests that true motor learning occurred with motor learning coaching; the 3-month treatment course may be where new motor functions were gained and practiced, but not incorporated or optimized, as shown by no immediate change in ME.…”

Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease

“…No SAEs occurred. At Week 52, there was a change score of +7.89 on the GMAE, which would be considered a clinically important difference of moderate effect size [17], and would be considered statistically significant because of non-overlapping 95% CIs. In subanalyses of change on individual dimensions of the GMFM-66, the patient demonstrated improvement in Dimension B (Sitting), Dimension D (Standing), and Dimension E (Walking, Running, and Jumping).…”

Assessing methods to specify the target difference for a randomised controlled trial: DELTA (Difference ELicitation in TriAls) review

“…The view of the parents of children who were being treated was often used in studies in paediatric care along with clinical and/or child assessment. [314][315][316] Similarly, Rockwood and colleagues 312 used the combined judgement of the patient and the caregiver as well as the clinician's assessment. Basoglu and colleagues 291 used one instrument that was administered by a clinician, but it was unclear whether the patient or the clinician completed other instruments.…”