Is there a representative quality‐of‐life questionnaire for patients with AL amyloidosis?—systematic literature review

Abstract

SummarySystemic AL amyloidosis is an incurable condition with various presentations and may cause multiple complications related to organ involvement. As survival has improved, disease and therapy‐related quality of life (QoL) is becoming an increasingly important treatment endpoint. We review the literature summarising the utilised QoL questionnaires (QLQs) and assess their validity according to COSMIN (Consensus‐based Standards for the Selection of Health Measurement Instruments) standards. Thirteen retrospective observational studies and thirty‐two prospective clinical trials were analysed. Most QLQs are generic or only validated in populations with distinct complications of the disease. None meet ‘strong evidence’ for validation in this context. There is a need to develop a disease‐specific QLQ, which could inform treatment choices and facilitate the approval of novel therapies.