ABSTRACT
Objective: The purpose of the study was to examine the effects of COVID-19 on SMA patient care and clinical trial management in the United States. Background: Spinal Muscular Atrophy (SMA) is an autosomal recessive neuromuscular disease characterized by progressive muscle weakness and atrophy. Early treatment has been shown to modify disease progression and is expected to alter phenotype. Despite strains to the healthcare system due to the ongoing COVID-19 pandemic, the diagnosis of SMA remains a medical emergency. Design/Methods: A sample of providers at known SMA treatment and clinical trial sites was invited by Cure SMA to complete an online survey that was open from November 24, 2020 through March 8, 2021. 48 complete responses were received (SMA treatment sites (Care): n=22;SMA treatment and clinical trial sites (Care & Trial): n=26). Results: 26.9% of Care & Trial Sites and 27.3% of Care Sites report clinic/facility appointment cancellation(s) or delays for SMA drug treatment during the pandemic (from March 2020 to time of survey completion);11.5% of Care & Trial Sites and 9.1% of Care Sites report similar cancellations within the month prior to survey completion. 96.2% of Care & Trial Sites and 86.4% of Care Sites report patient and/or families cancelled/delayed SMA-related appointments due to exposure concerns during the pandemic while 65.4% of Care & Trial Sites and 63.6% of Care Sites noted these cancellations within the month prior to survey completion. 26.9% of Care & Trial Sites reported having paused enrollment, and 34.6% reported notable recruitment delays. Prioritization of assessments, telemedicine, and protocol amendments were helpful strategies to maintain trial continuity. Conclusions: This research provided insight into effects of the COVID-19 pandemic on access to SMA management and clinical trials. Healthcare providers are encouraged to collaborate with SMA patients to mitigate ongoing exposure concerns while supporting improved SMA outcomes and trial participation.