Search reports, ask a question, or jump to a page.
The problem
Intervention: Treatment with risdiplam (EVRYSDI) versus nusinersen (SPINRAZA), both disease-modifying therapies for SMA
Who it was tested with: 58 children treated with risdiplam in FIREFISH trial and 81 children treated with nusinersen in SHINE/ENDEAR cohort, all with Type 1 SMA.
Children or communities are compared with a group that did not receive the intervention, or followed over years. This is the design that best separates what the intervention did from everything else that changed.
"Matching-adjusted indirect comparisons (MAICs) were conducted, adjusting for age at first dose, disease duration and baseline Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score. Cox proportional-hazards models were used to compare survival and times to Hammersmith Infant Neurological Examination, Module 2 (HINE-2) and CHOP-INTEND responses, and time to first serious adverse event (SAE)." This design leverages longitudinal data from two trials to indirectly compare treatments while controlling for baseline differences, representing a quasi-experimental approach stronger than observational studies but limited compared to true head-to-head randomization.
Non-randomized comparison with statistical adjustment only for observed baseline characteristics; unmeasured confounding may exist. Different patient populations from two separate trials. Effective sample size reduced to 40.6 for risdiplam after matching.
Across countries and designs
1 study · 1 country
