ClinicoEconomics and Outcomes Research (Sep 2023)
Using a Modified Delphi Panel to Estimate Health Service Utilization for Patients with Advanced and Non-Advanced Systemic Light Chain Amyloidosis
Abstract
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 Broder3,* 1Department of Medicine, Mayo Clinic, Rochester, MN, USA; 2Department of Medicine, Indiana University School of Medicine; Director, Multiple Myeloma, Waldenstrom’s Disease and Amyloidosis Program, Indianapolis, IN, USA; 3PHAR (Partnership for Health Analytic Research), Beverly Hills, CA, USA; 4Amyloidosis Support Groups Inc, Wood Dale, IL, USA; 5Memorial Sloan-Kettering Cancer Center, New York, NY, USA; 6Multiple Myeloma and Amyloidosis Program, Columbia University Medical Center, New York, NY, USA; 7Department of Cardiovascular Medicine, Mayo Clinic, Rochester, MN, USA; 8Malignant Hematology, Scripps Clinic MD Anderson Cancer Center, La Jolla, CA, USA; 9Health Economics and Outcomes Research, Prothena Biosciences Ltd., South San Francisco, CA, USA; 10Department of Medicine, Hematology/Oncology, Weill Cornell Medical College, New York, NY, USA; 11Division of Multiple Myeloma, Department of Hematology and Hematopoietic Cell Transplantation, City of Hope, Duarte, CA, USA; 12Division of Cardiovascular Medicine, Stanford School of Medicine, Palo Alto, CA, USA; 13Division of Hematology, Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill, NC, USA*These authors contributed equally to this workCorrespondence: Michael S Broder, PHAR (Partnership for Health Analytic Research), 280 S Beverly Drive, Suite 404, Beverly Hills, CA, 90212, USA, Tel +1 310 858 9555, Email [email protected]: 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: consensus, hematology, outcomes research, mayo stage, cardiac failure
