In SMA, as the efficacy data collected on nusinersen and risdiplam have mainly concerned the pediatric population, there is as yet no consensus on treating adults. Between October 2020 and September 2021, the choice of treating adult patients with one of these two products was studied at several multidisciplinary team meetings (MDTs):
- the discussions involved 107 adults with SMA, aged 16 to 62, most of whom had worsening motor weakness in their upper and lower limbs, 47 with type II SMA, 57 with type III SMA, one with type I SMA and two with type IV SMA,
- only 45.8% of patients had specific expectations of treatment to improve or stabilize some of their functions.
- the MDTs evaluated the introduction of nusinersen treatment for 46 patients (mainly with type III SMA), which was validated for 32 of them,
- the choice of risdiplam was suggested for 54 patients (mainly with type II SMA, who were older and whose motor and respiratory functions were more severely affected) and accepted for 28 of them,
- a request to treat with one or the other treatment (without preference) was made for five patients and confirmed for four of them,
- for 12 patients, a change of treatment was studied, from risdiplam to nusinersen for one patient, and from nusinersen to risdiplam for the others, notably due to difficulties associated with intrathecal injection,
- In all, of the 107 patients, 72 were able to receive treatment, the majority with nusinersen. Negative opinions were based on a period of SMA stability, an intercurrent disease, too severe muscle weakness, absence of contraindication to intrathecal injections (for risdiplam)…
