10 Year Musculoskeletal Outcomes with Tailored Primary Prophylaxis: The Canadian Hemophilia Prophylaxis Study.
Bibliographic record
Abstract
Abstract High-dose prophylaxis for severe hemophilia A [25–40 factor (F) VIII Units (u) kg−1 on alternate days] reduces arthropathy, but does so at great cost. (NEJM2007; 357: 535–544). Less intensive prophylaxis regimens might be cost-effective while reducing the need for indwelling venous access devices and their attendant complications. Tailored prophylaxis starts with FVIII, 50 u kg−1 weekly (Step1). If bleeding frequency is unacceptable (4 joint/soft tissue bleeds or 5 bleeds into a joint on a step of protocol), or if a target joint develops (≥3 bleeds into a single index joint-ankle, elbow or knee) over a consecutive 3-month period the dose of FVIII is escalated to 30 u kg−1 twice weekly (Step 2); a third escalation to 25 u kg−1 on alternate days if bleeding frequency remains unacceptable (Step3). Objectives: To assess the degree of joint damage in the 6 index joints for subjects receiving tailored prophylaxis. Methods: We studied an inception cohort of boys ages 1–2.5 years with severe hemophilia A (<2% FVIII) and no inhibitors treated with tailored prophylaxis. Patients were followed at 6 monthly intervals and parents have kept detailed diaries. Joint damage was measured using a validated physiotherapy joint score and function was assessed using the Child Health Assessment Questionnaire (CHAQ) Results: 54 boys were enrolled from 11 Canadian hemophilia treatment centers. The total follow-up is 3423 patient months (mean 62.2 months, median 54 months and range 5–117 months). 16 (29.6%) boys have been escalated to Step 2 and 14 (25.9%) have escalated to Step 3. 11 of the escalations were due to target joint bleeding. Subjects on study had a mean of 1.4 joint bleeds/patient-year. Physiotherapy Scores Maximum Possible Score Median Observed Range Ankles 56 2 0–20 Elbows 48 0 0–13 Knees 56 0 0–15 All Joints Swelling 18 0 0–4 Muscle Atrophy 18 0 0–3 CHAQ 3 0 0–2 89% of participants were compliant with the primary prophylaxis protocol. Non compliant subjects had the poorest outcomes. No treatment related adverse events have occurred. Conclusion: Tailored primary prophylaxis should be considered as a valid option for long term prophylaxis in young boys with severe hemophilia A. Maximum Possible Score Median Observed Range Ankles 56 2 0–20 Elbows 48 0 0–13 Knees 56 0 0–15 All Joints Swelling 18 0 0–4 Muscle Atrophy 18 0 0–3 CHAQ 3 0 0–2
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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) | 0.001 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.001 |
| Bibliometrics | 0.001 | 0.002 |
| Science and technology studies | 0.002 | 0.001 |
| Scholarly communication | 0.001 | 0.000 |
| Open science | 0.001 | 0.001 |
| Research integrity | 0.001 | 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".