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

Abstract

Systemic light-chain (AL) amyloidosis is a rare disorder caused by misfolding of light chains produced by clonal plasma cells. These misfolded light chains may deposit in any organ or tissue, with the heart, kidneys, gastrointestinal system and peripheral nerves being most commonly affected, causing significant impairment.Novel chemoimmunotherapy regimens and autologous stem cell transplantation (auto-SCT) have substantially improved survival, which ranges between months and years depending on the stage, degree of cardiac involvement, the fitness for intensive therapy and the response to treatment. Nevertheless, disease and treatment-related morbidity remain considerable, 1 necessitating both balancing treatment goals to quality-of-life (QoL) burden and supportive care interventions.Patient-reported outcome measures (PROMS), namely via QoL questionnaires (QLQs) addressed to patients, are regarded as a gold standard tool to report QoL outcomes. To date, there is no disease-specific validated tool to assess and monitor the QoL for AL amyloidosis. Current tools are not associated with disease stage, organ involvement or treatment response or toxicity.The aim of this study was to identify and evaluate the validity of the applied PROMs in literature as well as to summarise QoL outcomes for AL amyloidosis.

Development and initial validation of the AL‐PROfile patient‐reported outcome measure in light chain (AL) amyloidosis

“…Several options exist for measuring patient-reported outcomes (PRO) in AL amyloidosis, 5 with the Medical Outcomes Study 36-item Short-Form Health Survey (SF-36) being the most widely studied. The SF-36 has demonstrated content validity 6 and acceptable psychometric properties in this context.…”