Cost-Effectiveness of Defibrotide for the Treatment of Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome (VOD/SOS) with Multi-Organ Dysfunction (MOD) Post-Hematopoietic Stem Cell Transplantation (HSCT) in Canada
Notice bibliographique
Résumé
Abstract Introduction Hepatic VOD/SOS is a progressive, potentially life-threatening complication of conditioning for HSCT or of nontransplant-associated chemotherapy. Without treatment VOD/SOS with MOD (eg, renal and/or pulmonary dysfunction) may be associated with >80% mortality. Defibrotide (DF) is approved to treat hepatic VOD/SOS with renal and/or pulmonary dysfunction post-HSCT in the United States and Canada, and to treat severe hepatic VOD/SOS post-HSCT in patients aged >1 month in the European Union. The current analysis evaluates the cost-effectiveness of DF vs best supportive care (BSC) in Canada in patients with VOD/SOS with MOD post-HSCT. Methods A previously reported global Markov cost-utility model was adapted to reflect Canadian sources of care with regard to epidemiology, management costs, and survival expectancy; only direct medical costs were included. The model included an acute phase and a long-term phase, with a lifetime horizon. Transition probabilities in the acute phase were based on Phase 3 (Richardson et al, 2016) endpoints of Day +100 survival and complete response (CR). The model included 4 health states: severe VOD/SOS, CR, survival, and death. Survival in the long-term phase was extrapolated using Remberger et al (2011) and Ashton et al (2014). Hospital costs were calculated by taking the difference in time to CR in each arm in order to estimate the expected difference in hospital days between the BSC and DF arms. The severe VOD/SOS utility value was assumed to be the same as acute liver failure and end-stage liver disease scores (0.208), and the utility for CR was set to the age-matched general population. Costs and outcomes were discounted at 1.5% per year according to government guidance. Health effects were primarily expressed in terms of quality adjusted life years (QALYs). Results The difference in estimated costs between DF and BSC was $27,396 (Canadian dollars; Table). DF showed an increase in 1.5 QALYs versus BSC. The incremental cost-effectiveness ratio (ICER; cost per QALY gained) was $17,724. In the probabilistic sensitivity analysis, for willingness to pay of $30,000 and $50,000 per QALY gained, the probabilities of DF being cost-effective were 85.6% and 100%, respectively (Figure). Conclusion These results suggest that DF treatment for VOD/SOS with MOD represents cost-effective use of health resources in Canada, with an ICER estimate compared with BSC that was below the accepted willingness to pay threshold. Limitations of the analysis include longer-term extrapolation of clinical trial data and assumptions about resource utilization patterns. Results were driven by estimates of more rapid recovery, reduced length of stay, and improved Day +100 survival in DF-treated patients. Results were supported by the sensitivity analysis. Support: Jazz Pharmaceuticals. Disclosures Belsey: Jazz Pharmaceuticals: Consultancy. Kemadjou:Jazz Pharmaceuticals: Consultancy. Isaila:Jazz Pharmaceuticals: Employment, Other: Stock and stock options. Villa:Jazz Pharmaceuticals: Employment, Equity Ownership, Other: Stock and stock options.
Récupéré en direct depuis OpenAlex et désinversé. Les résumés ne sont pas conservés dans cette base de données : les index inversés représentent 8,6 Go des 9,3 Go de texte de la base, et le serveur dispose de 13 Go libres.
Comment cette classification a été obtenuedéplier
Prédiction machine sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Le volet Gemma est une étiquette directe du modèle pour chaque travail de la base, lue sur la notice réduite au titre. Le volet Codex est un classifieur appris des 10 348 étiquettes directes de Codex et calibré sur les taux pondérés de l'échantillon; les champs sans appui suffisant ne portent aucun appel Codex. Le mode candidate est l'union des deux volets; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont pas des étiquettes humaines.
Scores du classifieur distillé par catégorie (deux têtes)
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,002 | 0,007 |
| Méta-épidémiologie (sens strict) | 0,001 | 0,001 |
| Méta-épidémiologie (sens large) | 0,001 | 0,003 |
| Bibliométrie | 0,001 | 0,002 |
| Études des sciences et des technologies | 0,001 | 0,001 |
| Communication savante | 0,002 | 0,001 |
| Science ouverte | 0,002 | 0,001 |
| Intégrité de la recherche | 0,001 | 0,002 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,004 | 0,000 |
Scores machine (provisoires)
Les deux têtes enseignantes du modèle étudiant, lues sur ce travail. Un score ordonne la base pour la relecture; il n'affirme jamais une catégorie, et le statut de validation accompagne chaque rangée tel quel.
Scores de référence d'un modèle non mature (critères de maturité non atteints, 7 itérations). Un score ordonne; il n'affirme jamais une catégorie.
score_only:v0-immature-baseline · tel quel depuis la passe de notation : score_only signifie que le nombre peut ordonner les travaux, et qu'aucune étiquette de catégorie n'en découleClassification
machine, non validéePrédiction automatique; un appel candidat d’une seule source (Gemma direct ou Codex distillé), pas un consensus.
Le détail, modèle par modèle et score par score, se trouve en fin de page sous « Comment cette classification a été obtenue ».