P-129: Quality of life reported outcomes in published research on AL Amyloidosis: a systematic literature review

Abstract

Background Recent advances in the treatment of AL Amyloidosis have considerably prolonged survival outcomes. Hence, disease and therapy-related quality of life are becoming important treatment endpoints. Patient reported outcome measures (PROMs) are increasingly considered the gold standard for evidence-based adjustments of treatment to enhance patients’ wellbeing. The objective of this study was to identify the QOL questionnaires (QOLQs) used in literature for AL Amyloidosis and assess their validity. Methods A systematic literature search of PubMed, Medline and EMBASE databases was undertaken to identify QOLQs used in research when assessing QOL in patients with AL Amyloidosis up to May 2021.The validity of these tools in this context were assessed in accordance with the COSMIN (Consensus-based Standards for the selection of health Measurement Instruments) quality criteria. Results 246 publications were originally identified and screened for relevance to the study by two independent investigators. Review of these reports excluded from further analysis 92 that were duplicates, 13 case-reports, 11 reports that were not relevant to AL Amyloidosis, 56 with no detail reference to QOL, 13 reporting QOL without using QOLQs or PROMs and 4 review articles. 57 reported studies met criteria, 47 observational studies and 10 prospective clinical trials reported QOL and PROM(s) results in AL Amyloidosis and could be analysed further. Thirteen different questionnaires were occasionally used to report QOL outcomes (SF-36; EQ-5D-3L; FACT-G; PROMIS-GH); HPRSS; DT; EORTC QLQ-C30; KCCQ-12; GAF; SWLS; STAI; CESD; MDASI). The most commonly (35/58 publications) used was the SF-36. None is AL Amyloidosis specific, but they have been traditionally applied in similar diseases (Multiple Myeloma) or its complications (Congestive Heart Failure). Literature data when open QOL questionnaires were used in AL Amyloidosis patients, identified QOL issues that severely impact their vitality, mobility, physical function, working capacity, family and social role, leisure activities, sleep quality and mental health. None of the QOLQs used succeeds to mirror effectively these Al Amyloidosis patient-raised issues (poor content validity). Also, none met COSMIN validation criteria, and only the PROMIS-GH and SF-36 showed acceptable internal consistency (Cronbach’s a>70). Conclusions This literature review showed that currently applied QOLQs /PROMs in observational and clinical trial studies on AL Amyloidosis are general questionnaires and lack validity evidence. Thus, there is need for the development of a new disease specific AL Amyloidosis QOLQ that can be used to represent quality of life issues and guide treatment adjustments and novel therapies’ approval. Recent advances in the treatment of AL Amyloidosis have considerably prolonged survival outcomes. Hence, disease and therapy-related quality of life are becoming important treatment endpoints. Patient reported outcome measures (PROMs) are increasingly considered the gold standard for evidence-based adjustments of treatment to enhance patients’ wellbeing. The objective of this study was to identify the QOL questionnaires (QOLQs) used in literature for AL Amyloidosis and assess their validity. A systematic literature search of PubMed, Medline and EMBASE databases was undertaken to identify QOLQs used in research when assessing QOL in patients with AL Amyloidosis up to May 2021.The validity of these tools in this context were assessed in accordance with the COSMIN (Consensus-based Standards for the selection of health Measurement Instruments) quality criteria. 246 publications were originally identified and screened for relevance to the study by two independent investigators. Review of these reports excluded from further analysis 92 that were duplicates, 13 case-reports, 11 reports that were not relevant to AL Amyloidosis, 56 with no detail reference to QOL, 13 reporting QOL without using QOLQs or PROMs and 4 review articles. 57 reported studies met criteria, 47 observational studies and 10 prospective clinical trials reported QOL and PROM(s) results in AL Amyloidosis and could be analysed further. Thirteen different questionnaires were occasionally used to report QOL outcomes (SF-36; EQ-5D-3L; FACT-G; PROMIS-GH); HPRSS; DT; EORTC QLQ-C30; KCCQ-12; GAF; SWLS; STAI; CESD; MDASI). The most commonly (35/58 publications) used was the SF-36. None is AL Amyloidosis specific, but they have been traditionally applied in similar diseases (Multiple Myeloma) or its complications (Congestive Heart Failure). Literature data when open QOL questionnaires were used in AL Amyloidosis patients, identified QOL issues that severely impact their vitality, mobility, physical function, working capacity, family and social role, leisure activities, sleep quality and mental health. None of the QOLQs used succeeds to mirror effectively these Al Amyloidosis patient-raised issues (poor content validity). Also, none met COSMIN validation criteria, and only the PROMIS-GH and SF-36 showed acceptable internal consistency (Cronbach’s a>70). This literature review showed that currently applied QOLQs /PROMs in observational and clinical trial studies on AL Amyloidosis are general questionnaires and lack validity evidence. Thus, there is need for the development of a new disease specific AL Amyloidosis QOLQ that can be used to represent quality of life issues and guide treatment adjustments and novel therapies’ approval.