With the arrival of three innovative treatments (Spinraza, Zolgensma and Evrysdi), multidisciplinary team meetings (MTM) were set up in France in 2017 and then rolled out nationwide in 2019 to define the best treatment strategy for children who have just been diagnosed with SMA or who do not yet have treatment.
The main features of this decision-making process specific to France are as follows:
- national structure: around twenty experts (neopaediatricians, geneticists, etc.), under the auspices of the FILNEMUS network, meet twice a month via a secure platform,
- standardisation of files: the assessment is based on a set of standardised medical data, including genetics, anti-AAV9 status, CHOP-INTEND/HINE2 motor scores, and two videos (spontaneous motor skills and motor skills in a bathing situation),
- decision-making algorithm: the therapeutic opinion is based on a decision tree, developed over time and regularly updated,
- therapeutic alliance: the MTM’s opinion is advisory and the final decision rests with the family and the referring physician, the latter rarely deviating from the MTM’s opinion (less than 10%).
With 227 cases treated (i.e. all cases of SMA types 0 to III), this French model has proven its effectiveness in choosing the most appropriate treatment option for children with SMA.
