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. 2023 Sep 11:15:673-680.
doi: 10.2147/CEOR.S412079. eCollection 2023.

Using a Modified Delphi Panel to Estimate Health Service Utilization for Patients with Advanced and Non-Advanced Systemic Light Chain Amyloidosis

Morie Gertz #  1 , Rafat Abonour #  2 , Sarah N Gibbs #  3 , Muriel Finkel #  4 , Heather Landau #  5 , Suzanne Lentzsch #  6 , Grace Lin #  7 , Anuj Mahindra #  8 , Tiffany Quock #  9 , Cara Rosenbaum #  10 , Michael Rosenzweig #  11 , Surbhi Sidana #  12 , Sascha A Tuchman #  13 , Ronald Witteles #  12 , Irina Yermilov #  3 , Michael S Broder #  3
Affiliations

Using a Modified Delphi Panel to Estimate Health Service Utilization for Patients with Advanced and Non-Advanced Systemic Light Chain Amyloidosis

Morie Gertz et al. Clinicoecon Outcomes Res. .

Abstract

Purpose: Patients with diagnosed with systemic light chain (AL) amyloidosis at advanced Mayo stages have greater morbidity and mortality than those diagnosed at non-advanced stages. Estimating service use by severity is difficult because Mayo stage is not available in many secondary databases. We used an expert panel to estimate healthcare utilization among advanced and non-advanced AL amyloidosis patients.

Patients and methods: Using the RAND/UCLA modified Delphi method, expert panelists completed 180 healthcare utilization estimates, consisting of inpatient and outpatient visits, testing, chemotherapy, and procedures by disease severity and organ involvement during two treatment phases (the 1 year after starting first line [1L] therapy and 1 year following treatment [post-1L]). Estimates were also provided for post-1L by hematologic treatment response (complete or very good partial response [CR/VGPR], partial, no response or relapse [PR/NR/R]). Areas of disagreement were discussed during a meeting, after which ratings were completed a second time.

Results: During 1L therapy, 55% of advanced patients had ≥1 hospitalization and 38% had ≥2 admissions. Rates of hematopoietic stem cell transplant (HSCT) in advanced patients were 5%, while pacemaker or implantable cardioverter defibrillator (ICD) placement were 15%. During post-1L therapy, rates of hospitalization in advanced patients remained high (≥1 hospitalization: 20-43%, ≥2 hospitalizations: 10-20%), and up to 10% of advanced patients had a HSCT. Ten percent of these patients underwent pacemaker/ICD placement.

Conclusion: Experts estimated advanced patients, who would not be good candidates for HSCT, would have high rates of hospitalization (traditionally the most expensive type of healthcare utilization) and other health service use. The development of new treatment options that can facilitate organ recovery and improve function may lead to decreased utilization.

Keywords: cardiac failure; consensus; hematology; mayo stage; outcomes research.

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Conflict of interest statement

Morie Gertz reports personal fees from Ionis/Akcea, honorarium from Alnylym, personal fees from Prothena Biosceinces Ltd., Sanofi, Janssen, Aptitude Healthgrants and Ashfield, Juno, Physicians Education Resource, Johnson & Johnson, and Celgene, Research to Practice, and Sorrento, Development of educational materials for i3Health; personal fees for Data Safety Monitoring board from Abbvie. Rafat Abonour reports honoraria from Prothena Biosceinces Ltd. Sarah N. Gibbs is an employee of PHAR (Partnership for Health Analytic Research, LLC) which was paid by Prothena Biosciences Ltd. to conduct the research described in this manuscript and by Amgen, BioMarin Pharmaceuticals, Bristol Myers Squibb, Celgene, Delfi Diagnostics, Dompe, Eisai, Exact Sciences Corporation, Genentech, Gilead, GRAIL, Jazz, Kite, Nobelpharma, Novartis, Otsuka, Recordati, Regeneron, Sanofi US Services, Takeda Pharamceuticals USA to conduct research related to the work described in this manuscript. Muriel Finkel does not have disclosures to report. Heather Landau reports honorarium from Karyopharm, Pfizer Inc, Celgene, Juno, Prothena Biosceinces Ltd., Caelum Biosciences, Legend Biotech USA Inc, Takeda Pharmaceuticals, and Janssen Pharmaceuticals and research funding from Alexion Pharmaceuticals, Takeda Pharmaceuticals, Janssen Scientific Affairs, LLC and Prothena Biosciences Ltd. Suzanne Lentzsch reports research support from Zentalis and Sanofi. She is a member of DMCs of Janssen and BMS and participated in compensated Advisory Boards of BMS, Adaptive, Regeneron, Takeda, Janssen and GSK. She is a patent holder and owns royalties for CAEL-101. She also reports personal fees from Clinical Care Options, RedMedEd, PeerView, and Alexion outside the submitted work. Grace Lin reports research support from Pfizer, Ionis, Biotronik and advisory board participation for Boston Scientific. She also reports equity from HeartScreen Health, advisory board for Empallo, and research support from Anumana, outside the submitted work. Anuj Mahindra reports advisor/speaker honorarium from GSK, Epizyme, and Bristol Myers Squibb. Tiffany Quock reports employment at Prothena Biosciences Ltd. for the duration of work described in the manuscript. Cara Rosenbaum has participated on the advisory board for Takeda and Janssen and has received research support from Janssen, Karyopharm, GlaxoSmithKline, and Amgen. Michael Rosenzweig reports grants or contracts from Janssen and Pfizer, and payment or honoraria for lectures, presentations, speakers bureaus, manuscript writing or educational events from Janssen, Bristol Myers Squibb and Akcea and Takeda. Surbhi Sidana reports research funding from Magenta Therapeutics, BMS, Allogene, Janssen and consultancy fees from Magenta Therapeutics, BMS, Janssen, Sanofi, Oncopeptides, Takeda; consulting for Prothena Biosciences Ltd. Sascha Tuchman reports honoraria from Prothena Biosciences Ltd. Dr Sascha Tuchman also reports consulting fees from Prothena Biosciences Ltd., during the conduct of the study; grants and/or personal fees from Caelum, Sanofi, Janssen, BMS, Abbvie, and Karyopharm outside the submitted work. Ronald Witteles reports advisory board participation for Pfizer, Alnylam, Janssen, Ionis, BridgeBio, Astra Zeneca, Intellia, Regeneron, and Novo Nordisk. Irina Yermilov is an employee of PHAR (Partnership for Health Analytic Research, LLC) which was paid by Prothena Biosciences Ltd. to conduct the research described in this manuscript and by Amgen, BioMarin Pharmaceuticals, Bristol Myers Squibb, Celgene, Delfi Diagnostics, Dompe, Eisai, Exact Sciences Corporation, Genentech, Gilead, GRAIL, Jazz, Kite, Nobelpharma, Novartis, Otsuka, Recordati, Regeneron, Sanofi US Services, Takeda Pharamceuticals USA to conduct research related to the work described in this manuscript. Michael S. Broder is an employee of PHAR (Partnership for Health Analytic Research, LLC) which was paid by Prothena Biosceinces Ltd. to conduct the research described in this manuscript and by Amgen, BioMarin Pharmaceuticals, Bristol Myers Squibb, Celgene, Delfi Diagnostics, Dompe, Eisai, Exact Sciences Corporation, Genentech, Gilead, GRAIL, Jazz, Kite, Nobelpharma, Novartis, Otsuka, Recordati, Regeneron, Sanofi US Services, Takeda Pharamceuticals USA to conduct research related to the work described in this manuscript. The authors report no other conflicts of interest in this work.

Figures

Figure 1
Figure 1
RAND/UCLA modified Delphi panel process.
See this image and copyright information in PMC

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