Health-Related Quality of Life Instruments for Clinical Trials in AL Amyloidosis: Report from the Amyloidosis Forum HRQOL Working Group

References

• Merlini G, Dispenzieri A, Sanchorawala V, et al. Systemic immunoglobulin light chain amyloidosis. Nat Rev Dis Primers. 2018;4(1):38. doi:10.1038/s41572-018-0034-3
   PubMed Web of Science ®Google Scholar
• Gertz MA, Comenzo R, Falk RH, et al. Definition of organ involvement and treatment response in immunoglobulin light chain amyloidosis (AL): a consensus opinion from the 10th International symposium on amyloid and amyloidosis. Am J Hematol. 2005;79(4):319–328. doi:10.1002/ajh.20381
   PubMed Web of Science ®Google Scholar
• Quock TP, Yan T, Chang E, Guthrie S, Broder MS. Epidemiology of AL amyloidosis: a real-world study using US claims data. Blood Adv. 2018;2(10):1046–1053. doi:10.1182/bloodadvances.2018016402
   PubMed Web of Science ®Google Scholar
• Desport E, Bridoux F, Sirac C, et al. AL amyloidosis. Orphanet J Rare Dis. 2012;7:54. doi:10.1186/1750-1172-7-54
   PubMed Web of Science ®Google Scholar
• Muchtar E, Gertz MA, Kyle RA, et al. A modern primer on light chain amyloidosis in 592 patients with mass spectrometry-verified typing. Mayo Clin Proc. 2019;94(3):472–483. doi:10.1016/j.mayocp.2018.08.006
   PubMed Web of Science ®Google Scholar
• Schulman A, Connors LH, Weinberg J, et al. Patient outcomes in light chain (AL) amyloidosis: the clock is ticking from symptoms to diagnosis. Eur J Haematol. 2020;105(4):495–501. doi:10.1111/ejh.13472
   PubMed Web of Science ®Google Scholar
• Varga C, Comenzo RL. High-dose melphalan and stem cell transplantation in systemic AL amyloidosis in the era of novel anti-plasma cell therapy: a comprehensive review. Bone Marrow Transpl. 2019;54(4):508–518. doi:10.1038/s41409-018-0284-4
   PubMed Web of Science ®Google Scholar
• Sanchorawala V. High-dose melphalan and autologous peripheral blood stem cell transplantation in AL amyloidosis. Acta Haematol. 2020;143(4):381–387. doi:10.1159/000506498
   PubMed Web of Science ®Google Scholar
• Kastritis E, Palladini G, Minnema MC, et al. Daratumumab-based treatment for immunoglobulin light-chain amyloidosis. N Engl J Med. 2021;385(1):46–58. doi:10.1056/NEJMoa2028631
   PubMed Web of Science ®Google Scholar
• Lousada I, Comenzo RL, Landau H, Guthrie S, Merlini G. Light chain amyloidosis: patient experience survey from the Amyloidosis Research Consortium. Adv Ther. 2015;32(10):920–928. doi:10.1007/s12325-015-0250-0
   PubMed Web of Science ®Google Scholar
• McCausland KL, White MK, Guthrie SD, et al. Light chain (AL) amyloidosis: the journey to diagnosis. Patient. 2018;11(2):207–216. doi:10.1007/s40271-017-0273-5
   PubMedGoogle Scholar
• Lin HM, Seldin D, Hui AM, Berg D, Dietrich CN, Flood E. The patient’s perspective on the symptom and everyday life impact of AL amyloidosis. Amyloid. 2015;22(4):244–251. doi:10.3109/13506129.2015.1102131
   PubMed Web of Science ®Google Scholar
• Wilson IB, Cleary PD. Linking clinical variables with health-related quality of life. A conceptual model of patient outcomes. JAMA. 1995;273(1):59–65. doi:10.1001/jama.1995.03520250075037
   PubMed Web of Science ®Google Scholar
• Bayliss M, McCausland KL, Guthrie SD, White MK. The burden of amyloid light chain amyloidosis on health-related quality of life. Orphanet J Rare Dis. 2017;12(1):15. doi:10.1186/s13023-016-0564-2
   PubMed Web of Science ®Google Scholar
• Food and Drug Administration. Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. Guidance for Industry. Food and Drug Administration (US); 2009.
   Google Scholar
• Inaugural Amyloidosis Forum P, Lousada I. The amyloidosis forum: a public private partnership to advance drug development in AL amyloidosis. Orphanet J Rare Dis. 2020;15(1):268. doi:10.1186/s13023-020-01525-2
   PubMed Web of Science ®Google Scholar
• Lin HM, Gao X, Cooke CE, et al. Disease burden of systemic light-chain amyloidosis: a systematic literature review. Curr Med Res Opin. 2017;33(6):1017–1031. doi:10.1080/03007995.2017.1297930
   PubMed Web of Science ®Google Scholar
• D’Souza A, Myers J, Cusatis R, et al. Development of a conceptual model of patient-reported outcomes in light chain amyloidosis: a qualitative study. Qual Life Res. 2022;32:1083–1092. doi:10.1007/s11136-021-02943-w
   Google Scholar
• Food and Drug Administration. Patient-Focused Drug Development: Methods to Identify What is Important to Patients. Guidance for Industry. Food and Drug Administration (US); 2022.
   Google Scholar
• Food and Drug Administration. Patient-Focused Drug Development: Collecting Comprehensive and Representative Input. Guidance for Industry. Food and Drug Administration (US); 2020.
   Google Scholar
• Food and Drug Administration. Patient-focused drug development: Selecting, developing, or modifying fit-for-purpose clinical outcome assessments. Guidance for Industry [draft]. Food and Drug Administration (US): 2022.
   Google Scholar
• Lousada I. Considerations for Novel Endpoint Development in AL Amyloidosis. Presented at: Meeting of the Amyloidosis Forum; January 22, 2021. Available from: https://amyloidosisforum.org/considerations-for-novel-endpoint-development-in-al-amyloidosis/. Accessed January 22, 2021.
   Google Scholar
• Rizio AA, White MK, McCausland KL, et al. Treatment tolerability in patients with immunoglobulin light-chain amyloidosis. Am Health Drug Benefits. 2018;11(8):430–437.
   PubMed Web of Science ®Google Scholar
• Lousada I, Boedicker M. The impact of al amyloidosis: the patient experience. Hematol Oncol Clin North Am. 2020;34(6):1193–1203. doi:10.1016/j.hoc.2020.08.003
   PubMed Web of Science ®Google Scholar
• White MK, Bayliss MS, Guthrie SD, Raymond KP, Rizio AA, McCausland KL. Content validation of the SF-36v2® health survey with AL amyloidosis patients. J Patient Rep Outcomes. 2017;1(1):13. doi:10.1186/s41687-017-0020-7
   PubMedGoogle Scholar
• Maruish ME, Ed. User’s Manual for the SF-36v2 Health Survey. 3rd. Lincoln, RI: QualityMetric Incorporated; 2013.
   Google Scholar
• White MK, McCausland KL, Sanchorawala V, Guthrie SD, Bayliss MS. Psychometric validation of the SF-36 health survey in light chain amyloidosis: results from community-based and clinic-based samples. Patient Relat Outcome Meas. 2017;8:157–167. doi:10.2147/PROM.S146849
   PubMed Web of Science ®Google Scholar
• McCausland KL, Quock TP, Rizio AA, et al. Cardiac biomarkers and health-related quality of life in patients with light chain (AL) amyloidosis. Br J Haematol. 2019;185(5):998–1001. doi:10.1111/bjh.15693
   PubMed Web of Science ®Google Scholar
• Seldin DC, Anderson JJ, Sanchorawala V, et al. Improvement in quality of life of patients with AL amyloidosis treated with high-dose melphalan and autologous stem cell transplantation. Blood. 2004;104(6):1888–1893. doi:10.1182/blood-2004-01-0089
   PubMed Web of Science ®Google Scholar
• Sanchorawala V, McCausland KL, White MK, et al. A longitudinal evaluation of health-related quality of life in patients with AL amyloidosis: associations with health outcomes over time. Br J Haematol. 2017;179(3):461–470. doi:10.1111/bjh.14889
   PubMed Web of Science ®Google Scholar
• D’Souza A, Szabo A, Flynn KE, et al. Adjuvant doxycycline to enhance anti-amyloid effects: results from the dual Phase 2 trial. eClinicalMedicine. 2020;2:23.
   Google Scholar
• Hays RD, Spritzer KL, Schalet BD, Cella D. Promis®-29 v2.0 profile physical and mental health summary scores. Qual Life Res. 2018;27(7):1885–1891. doi:10.1007/s11136-018-1842-3
   PubMed Web of Science ®Google Scholar
• D’Souza A, Brazauskas R, Dispenzieri A, Panepinto J, Flynn KE. Changes in patient-reported outcomes in light chain amyloidosis in the first year after diagnosis and relationship to NT-proBNP change. Blood Cancer J. 2021;11(2):29. doi:10.1038/s41408-021-00412-8
   PubMed Web of Science ®Google Scholar
• D’Souza A, Magnus BE, Myers J, Dispenzieri A, Flynn KE. The use of PROMIS patient-reported outcomes (PROs) to inform light chain (AL) amyloid disease severity at diagnosis. Amyloid. 2020;27(2):111–118. doi:10.1080/13506129.2020.1713743
   PubMed Web of Science ®Google Scholar
• Shu J, Lo S, Phillips M, et al. Depression and anxiety in patients with AL amyloidosis as assessed by the SF-36 questionnaire: experience in 1226 patients. Amyloid. 2016;23(3):188–193. doi:10.1080/13506129.2016.1208081
   PubMed Web of Science ®Google Scholar
• Maurer MS, Dunnmon P, Fontana M, et al. Proposed cardiac end points for clinical trials in immunoglobulin light chain amyloidosis: report from the Amyloidosis Forum Cardiac Working Group. Circ Heart Fail. 2022;15(6):e009038. doi:10.1161/CIRCHEARTFAILURE.121.009038
   PubMed Web of Science ®Google Scholar
• Food and Drug Administration. Patient-focused drug development: Incorporating clinical outcome assessments into endpoints for regulatory decision-making. Guidance for Industry [draft]. Food and Drug Administration (US): 2023
   Google Scholar
• Benjamin K, Vernon MK, Patrick DL, Perfetto E, Nestler-Parr S, Burke L. Patient-reported outcome and observer-reported outcome assessment in rare disease clinical trials: an ISPOR COA Emerging Good Practices Task Force Report. Value Health. 2017;20(7):838–855. doi:10.1016/j.jval.2017.05.015
   PubMed Web of Science ®Google Scholar