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β细胞替代治疗的经济学。

Economics of Beta-Cell Replacement Therapy.

机构信息

Department of Bioengineering and iBB - Institute for Bioengineering and Biosciences, Instituto Superior Técnico, Universidade de Lisboa, Lisbon, Portugal.

The Discoveries Center for Regenerative and Precision Medicine, Lisbon Campus, Instituto Superior Técnico, Universidade de Lisboa, Lisbon, Portugal.

出版信息

Curr Diab Rep. 2019 Aug 2;19(9):75. doi: 10.1007/s11892-019-1203-9.

DOI:10.1007/s11892-019-1203-9
PMID:31375935
Abstract

PURPOSE OF REVIEW

Type 1 diabetes impacts 1.3 million people in the USA with a total direct lifetime medical cost of $133.7 billion. Management requires a mix of daily exogenous insulin administration and frequent glucose monitoring. Decision-making by the individual can be burdensome.

RECENT FINDINGS

Beta-cell replacement, which involves devices protecting cells from autoimmunity and allo-rejection, aims at restoring physiological glucose regulation and improving clinical outcomes in patients. Given the significant burden of T1D in the healthcare systems, cost-effectiveness analyses can drive innovation and policymaking in the area. This review presents the health economics analyses performed for donor-derived islet transplantation and the possible outcomes of stem cell-derived beta cells. Long-term cost-effectiveness of islet transplantation depends on the engraftment of these transplants, and the expenses and thresholds assumed by healthcare systems in different countries. Early health technology assessment analyses for stem cell-derived beta-cell replacement suggest manufacturing optimization is necessary to reduce upfront costs.

摘要

目的综述

美国有 1337 亿美元的直接终生医疗费用,影响了 130 万 1 型糖尿病患者。管理需要结合日常外源性胰岛素给药和频繁的血糖监测。个人的决策可能会带来负担。

最新发现

β细胞替代疗法包括保护细胞免受自身免疫和同种异体排斥的设备,旨在恢复生理葡萄糖调节,改善患者的临床结果。鉴于 T1D 在医疗保健系统中的巨大负担,成本效益分析可以推动该领域的创新和决策制定。本综述介绍了用于供体衍生胰岛移植的健康经济学分析以及干细胞衍生β细胞的可能结果。胰岛移植的长期成本效益取决于这些移植的植入情况,以及不同国家的医疗保健系统所承担的费用和阈值。干细胞衍生β细胞替代物的早期卫生技术评估分析表明,有必要进行制造优化以降低前期成本。

相似文献

1
Economics of Beta-Cell Replacement Therapy.β细胞替代治疗的经济学。
Curr Diab Rep. 2019 Aug 2;19(9):75. doi: 10.1007/s11892-019-1203-9.
2
Stem cells and beta cell replacement therapy: a prospective health technology assessment study.干细胞与β细胞替代疗法:一项前瞻性健康技术评估研究
BMC Endocr Disord. 2018 Jan 30;18(1):6. doi: 10.1186/s12902-018-0233-7.
3
Monitoring of beta cell replacement outcomes.β细胞替代治疗效果的监测。
Panminerva Med. 2016 Mar;58(1):59-71. Epub 2016 Jan 13.
4
Using the cost-effectiveness of allogeneic islet transplantation to inform induced pluripotent stem cell-derived β-cell therapy reimbursement.利用同种异体胰岛移植的成本效益来为诱导多能干细胞衍生的β细胞疗法报销提供依据。
Regen Med. 2015 Nov;10(8):959-73. doi: 10.2217/rme.15.59. Epub 2015 Nov 13.
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Local Immunomodulatory Strategies to Prevent Allo-Rejection in Transplantation of Insulin-Producing Cells.局部免疫调节策略预防胰岛素分泌细胞移植中的同种异体排斥反应。
Adv Sci (Weinh). 2021 Sep;8(17):e2003708. doi: 10.1002/advs.202003708. Epub 2021 Jul 14.
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The state of the art of islet transplantation and cell therapy in type 1 diabetes.1型糖尿病胰岛移植与细胞治疗的现状
Acta Diabetol. 2016 Oct;53(5):683-91. doi: 10.1007/s00592-016-0847-z. Epub 2016 Feb 29.
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Death and dysfunction of transplanted β-cells: lessons learned from type 2 diabetes?移植β细胞的死亡和功能障碍:从 2 型糖尿病中吸取的教训?
Diabetes. 2014 Jan;63(1):12-9. doi: 10.2337/db12-0364.
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Pancreas Islet Transplantation for Patients With Type 1 Diabetes Mellitus: A Clinical Evidence Review.1型糖尿病患者的胰岛移植:临床证据综述
Ont Health Technol Assess Ser. 2015 Sep 1;15(16):1-84. eCollection 2015.
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Stem cell-based immunomodulation in type 1 diabetes: beyond the regenerative approach.基于干细胞的 1 型糖尿病免疫调节:超越再生方法。
Curr Pharm Des. 2011;17(29):3229-42. doi: 10.2174/138161211798157595.
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In search of a surrogate: engineering human beta cell lines for therapy.寻找替代物:工程化人类β细胞系用于治疗
Trends Endocrinol Metab. 2014 Aug;25(8):378-80. doi: 10.1016/j.tem.2014.05.006. Epub 2014 Jun 20.

引用本文的文献

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The efficiency of stem cell differentiation into functional beta cells for treating insulin-requiring diabetes: Recent advances and current challenges.干细胞分化为功能性β细胞治疗胰岛素依赖型糖尿病的效率:最新进展和当前挑战。
Endocrine. 2024 Oct;86(1):1-14. doi: 10.1007/s12020-024-03855-8. Epub 2024 May 10.
2
Combinatorial islet protective therapeutic approaches in β-cell transplantation: Rationally designed solutions using a target product profile.β细胞移植中的组合式胰岛保护治疗方法:基于目标产品概况的合理设计解决方案。
FASEB Bioadv. 2023 Jun 2;5(7):287-304. doi: 10.1096/fba.2023-00029. eCollection 2023 Jul.
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本文引用的文献

1
Navigating Two Roads to Glucose Normalization in Diabetes: Automated Insulin Delivery Devices and Cell Therapy.在糖尿病中实现血糖正常化的两条道路:自动化胰岛素输送装置和细胞疗法。
Cell Metab. 2019 Mar 5;29(3):545-563. doi: 10.1016/j.cmet.2019.02.007.
2
Recapitulating endocrine cell clustering in culture promotes maturation of human stem-cell-derived β cells.在培养中重现内分泌细胞的簇集促进了人干细胞衍生的β细胞的成熟。
Nat Cell Biol. 2019 Feb;21(2):263-274. doi: 10.1038/s41556-018-0271-4. Epub 2019 Feb 1.
3
Concise Review: Lessons Learned from Islet Transplant Clinical Trials in Developing Stem Cell Therapies for Type 1 Diabetes.
Ethics of Early Clinical Trials of Bio-Artificial Organs.
生物人工器官早期临床试验的伦理问题。
Transpl Int. 2022 Jul 6;35:10621. doi: 10.3389/ti.2022.10621. eCollection 2022.
4
Process parameter development for the scaled generation of stem cell-derived pancreatic endocrine cells.规模化生成干细胞来源的胰腺内分泌细胞的工艺参数开发。
Stem Cells Transl Med. 2021 Nov;10(11):1459-1469. doi: 10.1002/sctm.21-0161. Epub 2021 Aug 13.
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Stem Cell-Based Clinical Trials for Diabetes Mellitus.基于干细胞的糖尿病临床研究。
Front Endocrinol (Lausanne). 2021 Feb 26;12:631463. doi: 10.3389/fendo.2021.631463. eCollection 2021.
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Regenerative medicine clinical readiness.再生医学临床准备就绪。
Regen Med. 2021 Mar;16(3):309-322. doi: 10.2217/rme-2020-0178. Epub 2021 Feb 24.
7
Insulin-Producing Cell Transplantation Platform for Veterinary Practice.用于兽医实践的胰岛素产生细胞移植平台。
Front Vet Sci. 2020 Feb 12;7:4. doi: 10.3389/fvets.2020.00004. eCollection 2020.
简明综述:从胰岛移植临床试验中吸取的经验教训,为 1 型糖尿病的干细胞治疗开发提供参考。
Stem Cells Transl Med. 2019 Mar;8(3):209-214. doi: 10.1002/sctm.18-0156. Epub 2018 Nov 12.
4
Dissolvable Microcarriers Allow Scalable Expansion And Harvesting Of Human Induced Pluripotent Stem Cells Under Xeno-Free Conditions.可溶解微载体在无动物源条件下允许规模化扩增和收获人诱导多能干细胞。
Biotechnol J. 2019 Apr;14(4):e1800461. doi: 10.1002/biot.201800461. Epub 2018 Nov 12.
5
Pluripotent Stem Cell-Derived Pancreatic Progenitors and β-Like Cells for Type 1 Diabetes Treatment.多能干细胞衍生的胰腺祖细胞和 β 样细胞治疗 1 型糖尿病。
Physiology (Bethesda). 2018 Nov 1;33(6):394-402. doi: 10.1152/physiol.00026.2018.
6
Stem Cell-Derived Pancreatic Progenitor Cells Have Now Been Transplanted into Patients: Report from IPITA 2018.干细胞来源的胰腺祖细胞现已被移植到患者体内:来自2018年国际胰腺移植受者协会(IPITA)的报告。
Am J Transplant. 2018 Jul;18(7):1581-1582. doi: 10.1111/ajt.14954.
7
Prevalence of Diagnosed Diabetes in Adults by Diabetes Type - United States, 2016.按糖尿病类型划分的美国成年人确诊糖尿病患病率,2016年
MMWR Morb Mortal Wkly Rep. 2018 Mar 30;67(12):359-361. doi: 10.15585/mmwr.mm6712a2.
8
The journey of islet cell transplantation and future development.胰岛细胞移植的历程与未来发展。
Islets. 2018 Mar 4;10(2):80-94. doi: 10.1080/19382014.2018.1428511. Epub 2018 Feb 5.
9
Why Are Diabetes Medications So Expensive and What Can Be Done to Control Their Cost?为什么糖尿病药物如此昂贵,如何控制其成本?
Curr Diab Rep. 2017 Sep;17(9):71. doi: 10.1007/s11892-017-0893-0.
10
Randomised, prospective, medico-economic nationwide French study of islet transplantation in patients with severely unstable type 1 diabetes: the STABILOT study protocol.随机、前瞻性、全国性的法国医学经济学胰岛移植研究在严重不稳定 1 型糖尿病患者中的应用:STABILOT 研究方案。
BMJ Open. 2017 Feb 20;7(2):e013434. doi: 10.1136/bmjopen-2016-013434.