PB0332 Hereditary Thrombotic Thrombocytopenic Purpura: A Survey of Canadian Treaters
Bibliographic record
Abstract
Background: hTTP is an ultra-rare disease characterized by microangiopathic hemolytic anemia, thrombocytopenia, and organ damage by microthrombi and caused by a defect in the ADAMTS13 gene.hTTP affects approximately 1 in 1,000,000 people and is expected to affect 38 Canadians.However, no Canadian data exist on the prevalence or treatment of this condition.Aims: The objective was to survey Canadian physicians on the prevalence, significance, and treatment of hTTP in Canada.Methods: We conducted an electronic survey (SurveyMonkey ® ) of physicians, mainly hematologists, to collect data on their hTTP patients.Participants were given 4 months to complete the survey between August 2021 and December 2021.The survey collected data on prevalence, disease burden and treatment of hTTP patients.Descriptive analysis was used to analyze the results.Results: We received 48 responses.Of these responses, 23 respondents (47.9%) have managed at least one hTTP patient in their careers.All respondents practiced in academic centres, with 83% treating adult patients.Over the course of their practice, the respondents have collectively managed 29 hTTP patients.Most physicians managed 1(57.9%) or 2(31.6%)hTTP patients.At the time of the survey, 14 patients were actively managed and 12/14(85.7%)had ADAMTS13 genetic testing done.11/14(78.6%)have experienced at least one acute episode of TTP and/or organ damage.6/14 (42.8%) of patients have had at least one stroke and 3/14(21.4%)have a permanent disability.Out of all the patients that were ever managed by the respondents, 2 have died because of disease complications (Figure 1).11/14 (78.6%) patients are on prophylactic plasma infusions.
Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.
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 | 0.002 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.000 | 0.000 |
| Bibliometrics | 0.002 | 0.005 |
| Science and technology studies | 0.002 | 0.000 |
| Scholarly communication | 0.001 | 0.000 |
| Open science | 0.001 | 0.001 |
| Research integrity | 0.001 | 0.000 |
| Insufficient payload (model declined to judge) | 0.004 | 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".