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Record W2970900708 · doi:10.1080/21678707.2019.1651641

Recent developments with defibrotide for the treatment of hepatic veno-occlusive disease/sinusoidal obstruction syndrome

2019· article· en· W2970900708 on OpenAlexaboutno aff
Christine Duncan, Justine M. Kahn, Stephan A. Grupp, Paul G. Richardson

Bibliographic record

VenueExpert Opinion on Orphan Drugs · 2019
Typearticle
Languageen
FieldMedicine
TopicHematopoietic Stem Cell Transplantation
Canadian institutionsnot available
Fundersnot available
KeywordsDefibrotideMedicineHepatic veno-occlusive diseaseTransplantationDiseaseChemotherapyIntensive care medicineClinical trialHematopoietic stem cell transplantationSurgeryInternal medicine

Abstract

fetched live from OpenAlex

Introduction: Hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a potentially life-threatening condition associated with endothelial cell damage due to hematopoietic cell transplantation (HCT) conditioning or chemotherapy not associated with HCT. Defibrotide is approved to treat hepatic VOD/SOS with renal or pulmonary dysfunction post-HCT in the United States and Canada, and severe hepatic VOD/SOS in patients aged >1 month in the European Union.Areas covered: VOD/SOS results from prolonged endothelial cell activation from exposure to chemotherapy or radiotherapy. PubMed was searched for articles on defibrotide without publication date restrictions. In vitro evidence suggests defibrotide has a protective effect on endothelial cells. The efficacy of defibrotide for the treatment of VOD/SOS with multi-organ dysfunction has been shown in clinical trials. Defibrotide has a favorable safety profile comparable to best supportive care in patients with severe forms of VOD/SOS.Expert opinion: As defibrotide treatment for VOD/SOS is most effective when initiated early, medical teams should familiarize themselves with the risk factors for VOD/SOS development and be able to recognize early signs and symptoms. Preclinical data indicate that defibrotide has a protective effect on endothelial cells that may contribute to its efficacy in VOD/SOS and that may render the drug applicable for other indications.

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 distilled prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.

metaresearch head score (Codex)0.000
metaresearch head score (Gemma)0.000
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Observational · Consensus signal: none
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.502
Threshold uncertainty score0.533

Codex and Gemma teacher scores by category

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

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.

Opus teacher head0.018
GPT teacher head0.283
Teacher spread0.265 · 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 teacher head, not a consensus.

The models applied no category: nothing in the taxonomy fit this work.
Study designObservational
Domainnot available
GenreEmpirical

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

Citations5
Published2019
Admission routes1
Has abstractyes

Explore more

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