J. Lammers, S. Norman, J. Berthy, J. Bender
Powell Gene Therapy Center, University of Florida
Gainesville, Florida
ABSTRACT
Introduction: Children need stability, self-produced sensorimotor experiences, and variable practice to master developmental skills in supine, side-lying, and prone positions.
Objective: Evaluate the safety and feasibility of home-based caregiver-mediated exercise training with a novel Therapeutic Play Gym (TPG).
Methods: Nine children, age five months to five years with severe mobility impairment and their caregivers enrolled in the study. Childhood diagnoses included treated Spinal Muscular Atrophy (SMA) Type 0 and SMA Type I, untreated X-Linked Myotubular Myopathy (XLMTM) and Nemaline Rod Muscular Dystrophy. No child could lift their head in prone, roll, or sit independently at baseline. Six children were both ventilator and gastrostomy-tube dependent. All child-caregiver dyads completed six months of training and completed testing at baseline (BL), Month 3, and Month 6 end of study (EOS) timepoints. Outcome measures included the Neuromuscular Gross Motor Outcome (GRO), FUNctional Measure (FUNM)-an exploratory device-specific outcome, range of motion (ROM), degree of scoliosis curvature, exploratory Caregiver Impression of Change Questionnaire (CICQ), and an end of study interview. Eight dyads enrolled in the two-year long-term extension (LTE) study starting in June 2022.
End of Study Results: Participants collectively trained for 22,000 minutes with no device related adverse events. Outcomes demonstrated ability to capture significant change in function and quality of life: GRO scores from BL to EOS were p= 0.0264. EOS FUNM scores (attached) minus (not attached) to TPG were p= 0.0013, FUNM (not attached) baseline to EOS were p=0.0087. CICQ scores were also statistically significant at p< 0.0001. More timepoints are needed to assess the training effect on ROM and scoliosis curvature through the LTE study. Conclusion: Exercise training using the TPG is yielding promising results in terms of functional and quality of life improvements over a 6-month period. (If selected for the 2023 MDA Conference, the 12-month study results will be presented)