GOAL: Gait Outcome Assessment List

Background/Purpose: Ambulatory children with cerebral palsy (CP) undergo different interventions to enhance physiological functions, improve their gait, and in the end help them better participate in activities of daily life. To understand the needs and individual goals of these patients and their parents a new instrument, the GOAL, was introduced by Narayanan and Coworkers. The reliability of the German version (GV) of GOAL was to be assessed.

Methods: The original questionnaire was translated following the guidelines of transcultural adaption of health-related quality of life measurements. After finalizing the German version, 91 patients (classified by the Gross Motor Function Classification System (GMFCS); 32 level I, 27 level II, and 32 level III) and/or their parents were asked to complete the questionnaire consisting of 48 items across 6 domains.

Results: The mean total score was 57.3 (SD 16.1). The test-retest reliability proved to be acceptable. As proposed, the GOAL score decreased the higher the patient’s GMFCS level. Age (subgroups older and younger than 10 years) does not seem to have a major effect on the total scoring. Also a bilateral versus a unilateral CP did not seem to affect the score significantly. Children seem to better judge themselves than parents do (mean parents: 74.27, mean child: 82.18; p-value = 0.0002).

Conclusion: The GOAL questionnaire GV has sufficient reliability as a proxy and patient reported outcome measurement of health status and well-being of ambulatory children with CP.

Disclosure

This study was supported by Ionis Pharmaceuticals, Inc. and Biogen.

Authors Disclosures

R.F. receives grant support to his institution from the NIH, MDA (US), and clinical trial support from Ionis, Biogen, Cytokinetics, Catabasis, Sarepta, Summit, Bristol-Myers-Squibb, Lilly and ReveraGen; serves as a consultant to Ionis, Biogen, Roche, Novartis, AveXis, Lilly, Catabasis, Sarepta; serves on the medical/scientific advisory board to the MDA (US), CureSMA, SMA Foundation, Parent Project MD, SMA Reach (UK), SMA Europe, TREAT-NMD; and has served on the DSMB for Roche and AveXis clinical trials.

N.K. serves on the National Advisory Board for Biogen, on National Advisory Boards, and as a consultant for AveXis, Catalyst, Cytokinetics, Marathon, PTC, and Sarepta, in an advisory capacity to CureSMA and MGFA, and receives clinical trial support/participates in clinical trials with Biogen.

E.M. serves on Advisory Boards for Roche, Ionis, Biogen, Avexis, and Novartis for SMA studies; is PI of ongoing Ionis/Biogen and Roche trials; and receives funding from Italian Telethon, Famiglie SMA Italy, and SMA Europe.

F.M. has received consulting fees from PTC Therapeutics, Sarepta Therapeutics, BioMarin, Roche, Biogen, Italfarmaco, Avexis, Pfizer, Trivorsan, and Catabasis and receives support from the National Institute of Health Research Biomedical Research Centre at Great Ormond Street Hospital for Children NHS Foundation Trust and University College London. He was the UK principal investigator in the ENDEAR study.

C.A.C. is a consultant for AveXis, Novartis, and Roche and received grants from the Department of Defense and the SMA Foundation.

B.T.D. is a scientific advisory board consultant for AveXis, Biogen, Marathon Pharmaceuticals, Sarepta, Cytokinetics, PTC Therapeutics, and Roche; advisor for Ionis Pharmaceuticals, Inc.; and receives research support from the National Institutes of Health/National Institute of Neurological Disorders and Stroke, the Slaney Family Fund for SMA and the SMA Foundation.

H.T. has nothing to disclose.

J.M. receives support from Eunice Kennedy Shriver National Institute for Child Health and Human Development (NICHD) 1K01HD084690–01A1, serves as a consultant to Ionis Pharmaceuticals, Inc., and has served on advisory boards for Biogen and Roche.

J.S., C.F.B., and E.S. are employees of and hold stock/stock options in Ionis Pharmaceuticals Inc.

Z.J.Z., S.G., and W.F. are employees of and hold stock/stock options in Biogen.