Chemotherapy with stem cell transplantation is more effective than immunotherapy in sporadic late onset nemaline myopathy with monoclonal gammopathy
Bibliographic record
Abstract
Sporadic late onset nemaline myopathy (SLONM) is a rare proximal myopathy with an insidious onset and slow progression. Patients present initially with a proximal limb weakness. Most commonly dyspnea, dysphagia, facial muscle weakness, and cardiomyopathy develop with disease progression. If not treated, SLONM could result in 40% mortality within the first 12 months. In approximately half of the reported cases SLONM is associated with plasma cell disorders, mostly monoclonal gammopathy of unknown significance (MGUS) and anecdotally with multiple myeloma and light chain deposition disease [ 1 , 2 , 3 ]. SLONM associated with a monoclonal gammopathy (SLONM + MGUS) has an aggressive course of the disease, more severe weakness, earlier development of dysphagia and dyspnea, and overall carries an unfavourable prognosis [ 1 , 2 ]. Two approaches are used to treat SLONM + MGUS: (1) immunosuppressive therapy (steroids, steroid-sparing agents, intravenous immunoglobulins (IVIG), and plasmapheresis/plasma exchange) or (2) chemotherapy followed by autologous stem cell transplantation (ASCT). Due to the rare nature of the disease, the best treatment modality is unknown.
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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 | 0.001 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.000 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.000 |
| 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".