12 Cost-Benefit Analysis of Newborn Screening for Severe Combined Immune Deficiency: literature review and results transferability
Notice bibliographique
Résumé
Abstract Background The purpose of screening for Severe Combined Immune Deficiency (SCID) is to enable timely diagnosis and treatment for this condition. Untreated SCID is uniformly fatal by 2 years of age. Hematopoietic stem cell transplantation is an effective treatment for SCID, and the success rate depends on the age at which it is performed. Earlier treatment improves survival, long term quality of life and decreases costs of treating patients, specifically by shortening hospitalization days. Screening, however, carries short-term implementation costs, that could potentially be a barrier to adding SCID to the newborn screening (NBS) panels. Objectives This literature review aimed to evaluate the cost-effectiveness of NBS for SCID and perform basic economic analysis review on available published sources. We also assessed the published results and clinical inputs for transferability between different centers. Design/Methods We conducted a systematic search of medical electronic databases: Google Scholar, Ovid, Medline, PubMed, CINAHL, EMBASE, the Cochrane Library, Science Citation Index and Evidence-Based Medicine and hand searched related references. We used the Preferred Reporting Items for Systematic review and Meta-analyses (PRISMA-2009) statement to report the findings. We extracted the details of individual study characteristics from each publication, assessed study quality, evaluated the effect sizes and assessed the influence of study design on the estimated effect size. The presence of small effect sizes was investigated using Funnel plots and Egger’s tests. Search terms included: newborn, SCID, newborn screening, cost-effectiveness, cost-benefit, cost-effectiveness analysis, cost-utility analysis, medical costs, the value of a statistical life, quality-adjusted life-years (QALYs) We included cross-sectional, case-control, and cohort studies that have been published in peer-reviewed journals, data from regional/national surveys. Results 298 records identified through database searching, 192 records removed. A total of 106 articles were found to be eligible for screening, 72 sources were excluded after abstracts review. Forty-four full -text articles were assessed for eligibility, and 14 were excluded (lack of relevance, misleading abstract). Thirty articles were included in the final literature review. We were looking for Level I evidence studies as a high-quality randomized trial or prospective study, sensible costs and alternatives, values obtained from many studies with multiway sensitivity analyses, a systematic review of Level I RCTs and Level I studies. A comparative economic analysis was performed on reviewed sources to determine the average cost-benefit of NBS for SCID among different centers. We used standard conversion to calculate total health costs and charges in US dollars. An average cost of screening for SCID per sample varies between 3.0 -6.0 US$, and at present, there are no known missed cases in SCID NBS programs. The average cost of treatment and QALY were the most common variables used in all reviewed sources and presented in Table 1. Charges for hospital care were more than 5 times higher for late-diagnosed cases of SCID compare to the early diagnosed cases (within the first 2 months of life). These results found to be none-specific to the particular countries, and have high potential transferability among different centers. Conclusion Our literature review analysis supports the cost-effectiveness of NBS for SCID. The opportunity of early treatment is a strong economic rationale for the addition of SCID screening to NBS programs.
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 distillée sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Apprise à partir de 10 348 étiquettes directes de Codex et de 10 348 étiquettes directes de Gemma. Le mode candidate est l'union des têtes enseignantes seuillées; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont ni des étiquettes humaines ni des étiquettes directes de modèles de pointe.
Scores Codex et Gemma par catégorie
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,001 | 0,001 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,001 | 0,000 |
| Bibliométrie | 0,000 | 0,002 |
| Études des sciences et des technologies | 0,001 | 0,000 |
| Communication savante | 0,000 | 0,000 |
| Science ouverte | 0,000 | 0,000 |
| Intégrité de la recherche | 0,000 | 0,001 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,000 | 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 tête enseignante, 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 ».