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Prophylaxis in the haemophilia population

2010· review· en· W1748661727 on OpenAlexaff
Victor S. Blanchette

Bibliographic record

VenueHaemophilia · 2010
Typereview
Languageen
FieldMedicine
TopicHemophilia Treatment and Research
Canadian institutionsSickKids Foundation
Fundersnot available
KeywordsMedicineHaemophiliaHaemophilia APopulationPediatricsRegimenVon Willebrand diseaseCoagulationVon Willebrand factorIntensive care medicineInternal medicineEnvironmental health

Abstract

fetched live from OpenAlex

SUMMARY: Prophylaxis is recommended as preventive therapy for young boys with severe haemophilia in countries where safe factor concentrates are available. This recommendation is supported by results from a randomized, controlled study that compared on-demand therapy with full-dose prophylaxis (Manco-Johnson MJ, Abshire TC, Shapiro AD et al. N Engl J Med 2007;357:535). It is important to distinguish primary vs. secondary prophylaxis. Primary prophylaxis refers to preventive treatment started before the onset of joint damage, whereas secondary prophylaxis refers to treatment started after joint damage has occurred. Whereas the benefits of primary prophylaxis are well documented, data relating to secondary prophylaxis are limited, especially in the adolescent/adult haemophilia population. Failure of prophylaxis may relate to several variables, including: (i) underlying status of the joints; (ii) poor compliance; (iii) participation in high-risk activities and (iv) unfavourable pharmacokinetics (PK), i.e., too rapid elimination of infused coagulation factors. There is evidence that the risk of joint bleeding in individuals with severe haemophilia A relates to time spent with factor levels < 1% (Collins PW, Blanchette VS, Fischer K et al. J Thromb Haemost 2009;7:413); this variable is strongly influenced by frequency of factor infusions and the individual's PK profile. Key ongoing questions relating to prophylaxis include: (1) what is the optimal regimen for initiating primary prophylaxis; (2) role of prophylaxis in the adolescent/young adult haemophilia population and (3) role of prophylaxis in individuals with severe von Willebrand's disease and other rare inherited coagulation disorders. The role of novel long-acting factor concentrates for prophylaxis will also need to be evaluated.

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 imitation

Not 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.

metaresearch head score (Codex)0.001
metaresearch head score (Gemma)0.003
Version: metacan-v3-hybrid-931329e0061cValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Systematic review · Consensus signal: none
GenreCandidate signal: Review · Consensus signal: none
Teacher disagreement score0.006
Threshold uncertainty score0.019

Distilled classifier scores by category (both heads)

CategoryCodexGemma
Metaresearch0.0010.003
Meta-epidemiology (narrow)0.0000.000
Meta-epidemiology (broad)0.0000.000
Bibliometrics0.0000.001
Science and technology studies0.0000.000
Scholarly communication0.0010.000
Open science0.0000.000
Research integrity0.0010.001
Insufficient payload (model declined to judge)0.0060.001

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.

Opus teacher head0.084
GPT teacher head0.398
Teacher spread0.314 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it

Classification

machine, unvalidated

Machine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.

The models applied no category: nothing in the taxonomy fit this work.
Study designSystematic review
Domainnot available
GenreReview

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".

Quick stats

Citations74
Published2010
Admission routes1
Has abstractyes

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