Japanese researchers have compared two protocols, Japanese and European, for administering nusinersen in the treatment of SMA:
- for regulatory reasons, the Japanese protocol involves only two intrathecal injections of nusinersen as a loading dose, followed by injections every 6 months instead of every 4 months in the European protocol,
- 14 Japanese adults with SMA took part in an observational study which lasted 39 months, and comparative (with a European reference cohort),
- efficacy judged by changes in functional scores (RULM and HFMSE) was comparable in patients who were still ambulatory, whether Japanese or European,
- conversely, injections every 6 months proved less effective than every 4 months in non-ambulatory patients.
These data are likely to become somewhat obsolete with the new protocols currently being validated following the DEVOTE study, which is evaluating higher doses of nusinersen.
