A Phase II Trial to Assess the Efficacy, Safety and Feasibility of 20[percnt] Subcutaneous Immunoglobulin in Patients with Myasthenia Gravis Exacerbation - Interim Analysis of Efficacy (P3.176)
Bibliographic record
Abstract
OBJECTIVE: A preliminary analysis of efficacy of 20[percnt] Subcutaneous immunoglobulin (SCIg) Hizentra@) in an ongoing study on patients with worsening myasthenia gravis (MG). BACKGROUND: Recent studies in immune-mediated neuromuscular diseases indicate that SCIg may be an effective alternative to intravenous immunoglobulin (IVIg). Patients on chronic IVIg switched to SCIg without any loss of efficacy and reported improved quality of life due to flexibility of their treatment schedule with SCIg. No prior studies have explored the efficacy of SCIg in MG. METHODS: This is an ongoing investigator-initiated, open label, multicenter, pilot study to assess the efficacy of SCIg in patients with moderately severe MG (MGFA Class II & III). Patients received 2gm/kg of SCIg in a flexible dosing regimen over four weeks. The primary outcome measure for efficacy was change in Quantitative MG (QMG) Scores from baseline to six weeks. Secondary measures included change in QMG Composite (QMGC), Manual Muscle Testing (MMT) and MG activities of daily living (MG ADL) scores. RESULTS: To date 13 patients have been screened with 10 deemed eligible and eight enrolled (5 males & 3 females; mean age 58 years, 6 seropositive, 2 seronegative). One patient was unable to infuse SCIG due to hand weakness and another withdrew consent. Most patients showed improvement in QMG scores (mean 19 vs 13; p<.05) from baseline as well as in secondary outcome measures (mean QMGC 19 vs 8; mean MMT 15 vs. 7; mean MG ADL 7 vs. 5). In general, peak improvement occurred during the third week of treatment. Mild and transient worsening occurred in one patient initially. CONCLUSIONS: Interim analysis suggests that SCIg may be effective in patients with MG exacerbation. This is an ongoing study and further data will be needed to corroborate these preliminary findings.
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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 | 0.003 | 0.002 |
| Meta-epidemiology (narrow) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.002 | 0.001 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 0.001 |
| Scholarly communication | 0.001 | 0.001 |
| Open science | 0.001 | 0.000 |
| Research integrity | 0.001 | 0.003 |
| Insufficient payload (model declined to judge) | 0.005 | 0.001 |
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".