SMArtCARE Real-World Data on Drug Treatment for Spinal Muscular Atrophy

Based on a better understanding of the molecular genetics of spinal muscular atrophy (SMA), targeted treatments for SMA have been developed in preclinical and clinical studies. To date, three different drugs are available in Germany for the treatment of patients with SMA. Approval of orphan drugs often occurs with limited evidence. Therefore, post-market surveillance and data capture are crucial to evaluate outcomes and the effect of treatment after prolonged treatment periods and in a broad spectrum of patients. SMArtCARE is a disease-specific registry to collect longitudinal data on all available SMA patients in German speaking countries. Data are collected during routine patient visits as real-world-outcome data. To evaluate motor function, standardized outcome measures are performed. Further, data on respiratory function, nutrition, orthopedic symptoms and genetic test results are collected in detail. Thus, data from the SMArtCARE registry may also contribute to regulatory purposes.

Regarding the treatment with nusinersen, our data collection comprises 745 patients: 470 pediatric patients (41% SMA type 1, 32% SMA type 2, 27% SMA type 3) and 275 adult patients. Baseline data and SMN2 copy number reflect the broad spectrum of disease severity. Under treatment with nusinersen, improvements in motor function are observed in all subgroups with varying extent depending on the age of the patients, SMA type, and baseline motor function.

Real-world data from the SMArtCARE registry make a relevant contribution to assessing treatment effects in a large cohort of SMA patients. This is crucial to develop treatment recommendations and thus to improve care of SMA patients.

No conflict of interest has been declared by the author(s).

Publication History

Article published online:
28 October 2021