Longterm Followup of Quality of Life in Patients with Cryopyrin-associated Periodic Syndrome Treated with Canakinumab, an Anti-interleukin 1β Monoclonal Antibody
Bibliographic record
Abstract
To the Editor: Cryopyrin-associated periodic syndrome (CAPS) is an inherited autoinflammatory disorder that comprises a clinical spectrum of 3 distinct phenotypes increasing in severity: familial cold autoinflammatory syndrome, Muckle-Wells syndrome (MWS), and chronic infantile neurological cutaneous and articular syndrome (CINCA). Urticarial rash, fever, arthralgia, and intense fatigue are the main clinical signs1. CINCA is the most severe form, with mental and physical disability. Some patients are difficult to classify because they present intermediate phenotypes. The effect of CAPS on quality of life is significant in all phenotypes, with limitation in ability to work (78%) and in participation in outdoor activities (95%)2. The NOD-like receptor 3 (NLPR3) gene mutation is responsible for overproduction of interleukin 1β (IL-1β)3. Three IL-1 inhibitors have shown efficacy in CAPS: anakinra, rilonacept, and canakinumab4. The approval of canakinumab by the US Food and Drug Administration was based on a 48-week, double-blinded study showing rapid and sustained remission of symptoms under treatment5. Our objective with this monocentric longitudinal study was to evaluate quality of life and socioprofessional effects of … Address correspondence to Dr. C. Marsaud, Department of Pediatrics and Pediatric Rheumatology, Bicêtre University Hospital, Paris XI University, 78 rue du Général Leclerc, 94270 Le Kremlin Bicêtre, Paris, France. E-mail: celine.marsaud{at}bct.aphp.fr
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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 | 0.004 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.000 |
| 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 | 0.001 | 0.001 |
| Insufficient payload (model declined to judge) | 0.001 | 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".