淀粉样变早期诊断和治疗的价值：腕管松解术中滑膜活检的初步研究

Q3 Medicine

Journal of Hand Surgery Global Online Pub Date : 2025-07-23 DOI:10.1016/j.jhsg.2025.100779

Trevor Ruesch BS , Nevil Khurana MS , Logan Hansen MD , Katiya Barkho BS , Julia Malewicz BS , Benjamin Brennan MS , Charles S. Day MD, MBA

{"title":"淀粉样变早期诊断和治疗的价值：腕管松解术中滑膜活检的初步研究","authors":"Trevor Ruesch BS , Nevil Khurana MS , Logan Hansen MD , Katiya Barkho BS , Julia Malewicz BS , Benjamin Brennan MS , Charles S. Day MD, MBA","doi":"10.1016/j.jhsg.2025.100779","DOIUrl":null,"url":null,"abstract":"<div><h3>Purpose</h3><div>The purpose of this study is to calculate the value of early diagnosis and treatment of transthyretin amyloidosis with tafamadis prior to the development of the symptoms of heart failure. In this pilot study of 51 patients, we present the validation of a published algorithm for the early identification of patients at risk for amyloidosis via tenosynovial biopsy during carpal tunnel release. In addition, by integrating clinical data from this pilot study with published predictive models, we aim to calculate the value of routine screening biopsies for transthyretin amyloidosis.</div></div><div><h3>Methods</h3><div>Patients presenting for carpal tunnel release surgery had a tenosynovial biopsy collected at the time of surgery. Cost information was gathered from hospital records. In conjunction with published models, five incremental cost effectiveness ratio equations were generated to assess the value of these screening biopsies.</div></div><div><h3>Results</h3><div>Of the 51 biopsied patients, six tested positive for amyloid, and one was started on tafamadis, a disease-modifying medication. Early diagnosis and treatment of patients with New York Heart Association class I (NYHA I) heart failure as opposed to NYHA IV results at a cost of $166,691.49 USD per quality adjusted life year (QALY). When treatment is initiated at NYHA class II stage compared with NYHA class IV, there is a cost of $155,977.22/QALY. For treatment at NYHA class III compared with NYHA class IV, the cost is $75,333.28/QALY.</div></div><div><h3>Conclusions</h3><div>This study validates the utility of previous criteria in identifying patients at high risk for systemic amyloidosis earlier in the disease course. Using the commonly accepted willingness to pay threshold of $50,000/QALY, early initiation of tafamadis does not represent a cost effective intervention. Routine biopsy of patients is not cost effective with the current cost of therapy and positivity rates of amyloidosis screening.</div></div><div><h3>Type of study/level of evidence</h3><div>Prognostic IB.</div></div>","PeriodicalId":36920,"journal":{"name":"Journal of Hand Surgery Global Online","volume":"7 5","pages":"Article 100779"},"PeriodicalIF":0.0000,"publicationDate":"2025-07-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Value of Early Diagnosis and Treatment of Amyloidosis: A Pilot Study of Synovial Biopsy During Carpal Tunnel Release\",\"authors\":\"Trevor Ruesch BS , Nevil Khurana MS , Logan Hansen MD , Katiya Barkho BS , Julia Malewicz BS , Benjamin Brennan MS , Charles S. Day MD, MBA\",\"doi\":\"10.1016/j.jhsg.2025.100779\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><h3>Purpose</h3><div>The purpose of this study is to calculate the value of early diagnosis and treatment of transthyretin amyloidosis with tafamadis prior to the development of the symptoms of heart failure. In this pilot study of 51 patients, we present the validation of a published algorithm for the early identification of patients at risk for amyloidosis via tenosynovial biopsy during carpal tunnel release. In addition, by integrating clinical data from this pilot study with published predictive models, we aim to calculate the value of routine screening biopsies for transthyretin amyloidosis.</div></div><div><h3>Methods</h3><div>Patients presenting for carpal tunnel release surgery had a tenosynovial biopsy collected at the time of surgery. Cost information was gathered from hospital records. In conjunction with published models, five incremental cost effectiveness ratio equations were generated to assess the value of these screening biopsies.</div></div><div><h3>Results</h3><div>Of the 51 biopsied patients, six tested positive for amyloid, and one was started on tafamadis, a disease-modifying medication. Early diagnosis and treatment of patients with New York Heart Association class I (NYHA I) heart failure as opposed to NYHA IV results at a cost of $166,691.49 USD per quality adjusted life year (QALY). When treatment is initiated at NYHA class II stage compared with NYHA class IV, there is a cost of $155,977.22/QALY. For treatment at NYHA class III compared with NYHA class IV, the cost is $75,333.28/QALY.</div></div><div><h3>Conclusions</h3><div>This study validates the utility of previous criteria in identifying patients at high risk for systemic amyloidosis earlier in the disease course. Using the commonly accepted willingness to pay threshold of $50,000/QALY, early initiation of tafamadis does not represent a cost effective intervention. Routine biopsy of patients is not cost effective with the current cost of therapy and positivity rates of amyloidosis screening.</div></div><div><h3>Type of study/level of evidence</h3><div>Prognostic IB.</div></div>\",\"PeriodicalId\":36920,\"journal\":{\"name\":\"Journal of Hand Surgery Global Online\",\"volume\":\"7 5\",\"pages\":\"Article 100779\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2025-07-23\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Journal of Hand Surgery Global Online\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S2589514125000994\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q3\",\"JCRName\":\"Medicine\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Journal of Hand Surgery Global Online","FirstCategoryId":"1085","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S2589514125000994","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q3","JCRName":"Medicine","Score":null,"Total":0}

引用次数: 0

摘要

目的探讨经甲状腺蛋白淀粉样变合并他他玛地斯在心衰症状出现前的早期诊断和治疗价值。在这项51例患者的初步研究中，我们提出了一种已发表的算法的验证，该算法可在腕管释放期间通过肌腱滑膜活检早期识别有淀粉样变风险的患者。此外，通过将该试点研究的临床数据与已发表的预测模型相结合，我们旨在计算甲状腺素淀粉样变性的常规筛查活检的价值。方法接受腕管松解手术的患者在手术时进行肌腱滑膜活检。从医院记录中收集了费用信息。结合已发表的模型，生成了五个增量成本效益比方程来评估这些筛查活检的价值。结果在51名接受活检的患者中，6名淀粉样蛋白检测呈阳性，1名患者开始服用一种名为他法马地的疾病缓解药物。早期诊断和治疗纽约心脏协会I级（NYHA I）心力衰竭患者与NYHA IV的结果相比，每个质量调整生命年（QALY）的成本为166,691.49美元。与NYHA IV期相比，在NYHA II期开始治疗的成本为155,977.22美元/QALY。与NYHA IV级相比，NYHA III级治疗的成本为75,333.28美元/QALY。结论：本研究验证了在病程早期识别全身性淀粉样变性高危患者的既往标准的有效性。使用普遍接受的50,000美元/QALY的支付意愿阈值，早期开始使用tafamadis并不代表具有成本效益的干预措施。以目前的治疗成本和淀粉样变性筛查的阳性率，常规活检患者并不具有成本效益。研究类型/证据水平预后IB。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

查看原文本刊更多论文

Value of Early Diagnosis and Treatment of Amyloidosis: A Pilot Study of Synovial Biopsy During Carpal Tunnel Release

Purpose

The purpose of this study is to calculate the value of early diagnosis and treatment of transthyretin amyloidosis with tafamadis prior to the development of the symptoms of heart failure. In this pilot study of 51 patients, we present the validation of a published algorithm for the early identification of patients at risk for amyloidosis via tenosynovial biopsy during carpal tunnel release. In addition, by integrating clinical data from this pilot study with published predictive models, we aim to calculate the value of routine screening biopsies for transthyretin amyloidosis.

Methods

Patients presenting for carpal tunnel release surgery had a tenosynovial biopsy collected at the time of surgery. Cost information was gathered from hospital records. In conjunction with published models, five incremental cost effectiveness ratio equations were generated to assess the value of these screening biopsies.

Results

Of the 51 biopsied patients, six tested positive for amyloid, and one was started on tafamadis, a disease-modifying medication. Early diagnosis and treatment of patients with New York Heart Association class I (NYHA I) heart failure as opposed to NYHA IV results at a cost of $166,691.49 USD per quality adjusted life year (QALY). When treatment is initiated at NYHA class II stage compared with NYHA class IV, there is a cost of $155,977.22/QALY. For treatment at NYHA class III compared with NYHA class IV, the cost is $75,333.28/QALY.

Conclusions

This study validates the utility of previous criteria in identifying patients at high risk for systemic amyloidosis earlier in the disease course. Using the commonly accepted willingness to pay threshold of $50,000/QALY, early initiation of tafamadis does not represent a cost effective intervention. Routine biopsy of patients is not cost effective with the current cost of therapy and positivity rates of amyloidosis screening.