PF703 LONG‐TERM FOSTAMATINIB TREATMENT OF ADULTS WITH IMMUNE THROMBOCYTOPENIA (ITP) DURING THE PHASE 3 CLINICAL TRIAL PROGRAM
Bibliographic record
Abstract
Background: Fostamatinib (Tavalisse®), an oral SYK inhibitor was approved in 2018 for the treatment of adults with chronic ITP who had an insufficient response to a prior treatment. In two randomized, double‐blind studies, 102 patients received fostamatinib and 48 received placebo; in the open‐label extension study, 123 received fostamatinib, including 44 from the placebo group. Aims: This abstract provides an update on 146 patients who have had up to 3 years of fostamatinib treatment. Methods: Adults with persistent/chronic ITP who had failed ≥1 prior therapy and had ≥3 platelet counts <30,000/μL at screening, were treated with fostamatinib at 100 mg BID, which increased to 150 mg BID based on tolerability, if platelets were <50,000/μL after 1 month. An overall response (post‐hoc analysis) was defined as achieving ≥1 platelet count of ≥50,000/μL within the first 12 weeks of treatment. Analyses excluded counts within 4 weeks of rescue therapy. Results: At the time of analysis, 42 (29%) of the 146 patients who received fostamatinib were continuing treatment. Reasons for treatment discontinuation included lack of response (41%), adverse events (AEs) (18%) and other (12%). An overall response occurred in 64/146 (44%) patients. Responders had reduced need for rescue medications and fewer bleeding episodes. The median platelet count in overall responders remained above 50,000/μL for more than 36 months. The most common AEs were diarrhea, hypertension and nausea, which were manageable with targeted treatment, dose modifications, or treatment withdrawal. Serious adverse events (SAE) were reported in 27% of patients and were possibly related to fostamatinib in 6%. SAEs led to treatment discontinuation in 3% of patients. Summary/Conclusion: 44% of patients had an overall response to fostamatinib. Fostamatinib was well tolerated for an extended period of time. No new safety signals and no cumulative toxicity was detected during long‐term treatment with fostamatinib (up to 41 months). image
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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 | 0.002 | 0.001 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.001 |
| Bibliometrics | 0.000 | 0.000 |
| Science and technology studies | 0.000 | 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) | 0.003 | 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".