P.131 Outcomes of Nusinersen in adult and pediatric cohorts of spinal muscular amyotrophy after two years of usage at the CHU de Québec-Université Laval
Bibliographic record
Abstract
Background: Nusinersen is approved for spinal muscular amyotrophy type I,II,III and is available for adult and pediatric populations since January 2019 at the CHU de Québec-UL. Methods: Patients who received at least one dose of nusinersen between January 2019 and March 2021 were included. Required information was gathered from patients’ charts. Results: Ten adults (70 injections) and 23 infants and children (202 injections) were included. No child but two adults stopped the therapy for personal reasons. Tests were performed at 0, 6, 18, 24 months when possible to assess efficacy, tolerability and quality of life perception (Table 1). Preliminary data shows stabilization or improvement for many tests. Conclusions: Nusinersen is well tolerated. Favorable effects were observed in both populations. A monitoring is still imperative for an objective assessment and for evaluation of the most relevant tests. Long term benefits remain to be demonstrated but results are encouraging.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.001 | 0.002 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.001 |
| Bibliometrics | 0.000 | 0.001 |
| Science and technology studies | 0.001 | 0.000 |
| Scholarly communication | 0.001 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.001 |
| Insufficient payload (model declined to judge) | 0.002 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".