Treatment satisfaction in 5q-spinal muscular atrophy under nusinersen therapy

Abstract

Background: Nusinersen was the first approved disease-modifying therapy for all 5q-spinal muscular atrophy (SMA) patients regardless of age or disease severity. Its efficacy in adults has recently been demonstrated in a large cohort by motor outcome measures, which were only partially suitable to detect changes in very mildly or severely affected patients. Patient-reported outcome measures (PROs) have been suggested as a valuable addition. Here, we aimed to assess treatment satisfaction and investigate whether it may be a useful PRO to monitor SMA patients. Methods: We enrolled 91 mainly adult 5q-SMA patients treated with nusinersen in a national, multicenter, cross-sectional observational study. 21 patients underwent longitudinal follow up. Patients’ satisfaction with treatment in four dimensions (global, effectiveness, convenience, side effects) was assessed by the Treatment Satisfaction Questionnaire for Medication German version 1.4 (TSQM-1.4©) and related to clinical parameters, motor scores, and treatment duration. Results: More than 90% of SMA patients were consistently satisfied over a median treatment duration of 10 months. Highest mean scores were observed in the dimensions ‘side effects,’ ‘global satisfaction,’ and ‘effectiveness’ (93.5 ± 14.8 versus 73.1 ± 21.0 and 64.8 ± 20.6, respectively). Patients’ satisfaction with the convenience of treatment was considerably lower (43.6 ± 20.2). Interestingly, satisfaction with the effectiveness was higher in ambulatory ( p = 0.014) compared with non-ambulatory patients and directly correlated to motor outcome measures. Five non-ambulatory patients withdrew from therapy. All of them presented with a deterioration of motor outcome measures and reported dissatisfaction with treatment effectiveness and convenience. Conclusion: Most patients were satisfied with nusinersen treatment effectiveness. Less severely affected patients indicated higher satisfaction. The TSQM-1.4© helped to identify therapy non-responders, who mainly addressed dissatisfaction with effectiveness and convenience. We suggest introducing the TSQM-1.4© as an additional PRO in SMA into clinical practice.

Assessment of Health-Related Quality of Life in Adult Spinal Muscular Atrophy Under Nusinersen Treatment—A Pilot Study

“…Although these data together with our own results allow for doubts regarding the efficacy of nusinersen treatment on QoL in adult SMA patients, they are contrary to recent studies reporting high satisfaction of patients under nusinersen treatment, especially regarding its effectiveness ( 33 , 34 ), and at least transiently reduced fatigue ( 35 ). Moreover, treatment adherence is generally high in this patient cohort despite the obvious burden of intrathecal treatment.…”

Validity and reliability of the German multidimensional fatigue inventory in spinal muscular atrophy

“…The underlying mechanisms are not fully understood and appear to be complex, involving psychological and somatic factors 4,5 . To evaluate treatment effectiveness and patient satisfaction, patient‐reported outcome measures (PRO), such as fatigue, have recently moved into focus 6,7 . They capture the patient’s subjective well‐being, which may differ from clinical parameters, but is no less important.…”

“…4,5 To evaluate treatment effectiveness and patient satisfaction, patientreported outcome measures (PRO), such as fatigue, have recently moved into focus. 6,7 They capture the patient's subjective well-being, which may differ from clinical parameters, but is no less important. PROs are often elusive and challenging to quantify.…”

Nusinersen: A Review in 5q Spinal Muscular Atrophy

“…Patient satisfaction with nusinersen was assessed using the Treatment Satisfaction Questionnaire for Medication Version 1.4 in 91 patients (aged 10–65 years with a genetic diagnosis of 5q SMA; 96% of patients had 5q SMA type 2 or 3) participating in a multicentre observational study [ 34 ]. Following a median treatment duration of 10 months, most patients were at least ‘somewhat satisfied’ with nusinersen (≈ 91%), its ability to prevent or treat the disease (> 90%) and the way it relieves symptoms (≈ 80%).…”

“…Following a median treatment duration of 10 months, most patients were at least ‘somewhat satisfied’ with nusinersen (≈ 91%), its ability to prevent or treat the disease (> 90%) and the way it relieves symptoms (≈ 80%). However, over half (≈ 55%; estimated from a figure) confirmed nusinersen was difficult to use, which likely reflects the need for multiple lumbar punctures [ 34 ].…”