Analysis of data collected by the German-speaking SMArtCARE observatory of 143 patients under 18 years of age with early-onset SMA who had not acquired sitting before starting nusinersen (Spinraza®) showed that:
- 88 children started nusinersen before the age of 2 years with a follow-up of 23.3 ± 12.9 months (cohort 1a), and 55 after the age of 2 years with a follow-up of 27.6 ± 11.0 months (cohort 1b); 44.8% of the patients had a follow-up of at least 38 months on treatment ;
- motor improvement at CHOP INTEND was more pronounced in children treated with nusinersen before the age of two, among whom four acquired standing and two walking;
- 24.5% of children acquired the ability to sit: 1/3 of cohort 1a and 10% of cohort 1b;
- significant motor improvements occurred within the first 14 months of treatment;
- the probability of respiratory assistance increased from 29.5% to 59.7% for cohort 1a and from 70.9% to 79% for cohort 1b;
- the probability of enteral nutrition increased from 23.9% to 52.5% for cohort 1a and from 43.6% to 52.5% for cohort 1b
This real-life study supports the need for early treatment of newborns and infants with type 1 SMA. It also confirms that while motor function and survival improve with nusinersen, bulbar and respiratory function do not.
