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- Durno, C., et al.
(författare)
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Survival Benefit for Individuals With Constitutional Mismatch Repair Deficiency Undergoing Surveillance
- 2021
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Ingår i: Journal of Clinical Oncology. - : American Society of Clinical Oncology (ASCO). - 0732-183X .- 1527-7755. ; 39:25
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Tidskriftsartikel (refereegranskat)abstract
- PURPOSE Constitutional mismatch repair deficiency syndrome (CMMRD) is a lethal cancer predisposition syndrome characterized by early-onset synchronous and metachronous multiorgan tumors. We designed a surveillance protocol for early tumor detection in these individuals. PATIENTS AND METHODS Data were collected from patients with confirmed CMMRD who were registered in the International Replication Repair Deficiency Consortium. Tumor spectrum, efficacy of the surveillance protocol, and malignant transformation of low-grade lesions were examined for the entire cohort. Survival outcomes were analyzed for patients followed prospectively from the time of surveillance implementation. RESULTS A total of 193 malignant tumors in 110 patients were identified. Median age of first cancer diagnosis was 9.2 years (range: 1.7-39.5 years). For patients undergoing surveillance, all GI and other solid tumors, and 75% of brain cancers were detected asymptomatically. By contrast, only 16% of hematologic malignancies were detected asymptomatically (P < .001). Eighty-nine patients were followed prospectively and used for survival analysis. Five-year overall survival (OS) was 90% (95% CI, 78.6 to 100) and 50% (95% CI, 39.2 to 63.7) when cancer was detected asymptomatically and symptomatically, respectively (P = .001). Patient outcome measured by adherence to the surveillance protocol revealed 4-year OS of 79% (95% CI, 54.8 to 90.9) for patients undergoing full surveillance, 55% (95% CI, 28.5 to 74.5) for partial surveillance, and 15% (95% CI, 5.2 to 28.8) for those not under surveillance (P < .0001). Of the 64 low-grade tumors detected, the cumulative likelihood of transformation from low-to high-grade was 81% for GI cancers within 8 years and 100% for gliomas in 6 years. CONCLUSION Surveillance and early cancer detection are associated with improved OS for individuals with CMMRD.
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| 2. |
- Jendle, Johan, 1963-, et al.
(författare)
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The Cost-Effectiveness of an Advanced Hybrid Closed-Loop System in People with Type 1 Diabetes : a Health Economic Analysis in Sweden
- 2021
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Ingår i: Diabetes Therapy. - : Springer. - 1869-6953 .- 1869-6961. ; 12:11, s. 2977-2991
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Tidskriftsartikel (refereegranskat)abstract
- INTRODUCTION: Swedish National Diabetes Registry data show a correlation of improved glycemic control in people with type 1 diabetes (T1D) with increased use of diabetes technologies over the past 25 years. However, novel technologies are often associated with a high initial outlay. The aim of the present study was to evaluate the long-term cost-effectiveness of the advanced hybrid closed-loop (AHCL) MiniMed 780G system versus intermittently scanned continuous glucose monitoring (isCGM) plus self-injection of multiple daily insulin (MDI) or continuous subcutaneous insulin infusion (CSII) in people with T1D in Sweden.METHODS: Outcomes were projected over patients' lifetimes using the IQVIA CORE Diabetes Model (v9.0). Clinical data, including changes in glycated hemoglobin (HbA1c) and hypoglycemia rates, were sourced from observational studies and a randomized crossover trial. Modeled patients were assumed to receive the treatments for their lifetimes, with HbA1c kept constant following the application of treatment effects. Costs were accounted from a societal perspective and expressed in Swedish krona (SEK). Utilities and days off work estimates were taken from published sources.RESULTS: The MiniMed 780G system was associated with an improvement in life expectancy of 0.16 years and an improvement in quality-adjusted life expectancy of 1.95 quality-adjusted life years (QALYs) versus isCGM plus MDI or CSII. These clinical benefits were due to a reduced incidence and a delayed time to onset of diabetes-related complications. Combined costs were estimated to be SEK 727,408 (EUR 72,741) higher with MiniMed 780G, with treatment costs partially offset by direct cost savings from the avoidance of diabetes-related complications and indirect cost savings from the avoidance of lost workplace productivity. The MiniMed 780G system was associated with an incremental cost-effectiveness ratio of SEK 373,700 per QALY gained.CONCLUSIONS: Based on a willingness-to-pay threshold of SEK 500,000 per QALY gained, the MiniMed 780G system was projected to be cost-effective versus isCGM plus MDI or CSII for the treatment of T1D in Sweden.
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