Treatments with a risk of liver injury, fatigue, headache and nausea were 1.6 times less likely to be chosen than treatments with a risk of diarrhoea and rash. Similarly, participants were 4.3 times and 5.8 times more likely to choose stable or improved (vs worse) breathing function, respectively. Participants ( n = 65) were 4.8 times and 8.1 times more likely to choose a treatment with stable or improved (vs worse) motor function, respectively. A clustered conditional logit model was used to estimate treatment preferences. Participants were recruited via patient advocacy associations to complete an online survey. Lastly, an attribute described whether a treatment had evidence of treatment effectiveness in different SMA types.
Treatment risks and side effects related to currently available treatments including risk of liver injury, fatigue, headache, nausea, diarrhoea and rash were described.
The mode of treatment administration (intrathecal injection, single intravenous infusion or regular oral therapy) was described. The attributes described improvements or worsening in motor and breathing function. The DCE described choice questions in terms of attributes and levels combined using a D-efficient design. This survey built on the design of a similar study undertaken in the UK. MethodsĪ discrete choice experiment (DCE) survey was developed to elicit the preferences of adult patients and caregivers regarding different treatment aspects of SMA. The aim of this study was to examine treatment preferences of patients and caregivers of patients with Type 2 and non-ambulatory Type 3 SMA in the Netherlands, Belgium, Finland, Ireland and Portugal. Spinal muscular atrophy (SMA) is a rare neuromuscular disease that affects motor neurons, resulting in progressive skeletal muscle weakness and atrophy.