Evaluation of clinical and laboratory parameters of adult patients with SMA under treatment with nusinerse
- Conditions
- G12.1Other inherited spinal muscular atrophy
- Registration Number
- DRKS00015702
- Lead Sponsor
- Klinik für Neurologie Universitätsklinikum Essen
- Brief Summary
Between July 13, 2017, and May 1, 2019, 173 patients were screened, of whom 139 (80%) were eligible for data analysis. Of these, 124 (89%) were included in the 6-month analysis, 92 (66%) in the 10-month analysis, and 57 (41%) in the 14-month analysis; patients with missing baseline HFMSE scores were excluded from these analyses. Mean HFMSE scores were significantly increased compared with baseline at 6 months (mean difference 1·73 [95% CI 1·05-2·41], p<0·0001), 10 months (2·58 [1·76-3·39], p<0·0001), and 14 months (3·12 [2·06-4·19], p<0·0001). Clinically meaningful improvements (=3 points increase) in HFMSE scores were seen in 35 (28%) of 124 patients at 6 months, 33 (35%) of 92 at 10 months, and 23 (40%) of 57 at 14 months. To 14-month follow-up, the most frequent adverse effects among 173 patients were headache (61 [35%] patients), back pain (38 [22%]), and nausea (19 [11%]). No serious adverse events were reported.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete
- Sex
- All
- Target Recruitment
- 378
genetically confirmed 5q-SMA
Planned treatment with Nusinersen
Patient is able to give informed consent
Pregnancy
< 18 y.
Study & Design
- Study Type
- observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Change of Hammersmith functional Evaluation Score (HFMSE) at 6 and 12 months after treatment with nusinersen
- Secondary Outcome Measures
Name Time Method Change in Revised Upper Limb Module (RULM) score after 6 and 12 months treatment with nusinersen