A longitudinal evaluation of health‐related quality of life in patients with AL amyloidosis: associations with health outcomes over time

Summary Light chain ( AL ) amyloidosis is a rare disease associated with significant, irreversible organ dysfunction and high case fatality. An observational study was conducted to assess health‐related quality of life ( HRQ oL) in patients treated for AL amyloidosis between 1994 and 2014 with both high dose melphalan and stem cell transplantation ( HDM / SCT ) or non‐ SCT chemotherapy regimens. The SF ‐36v1 ® Health Survey ( SF ‐36) was administered to assess HRQ oL during clinic visits. Analysis of variance was used to compare pre‐ and post‐treatment HRQ oL within each treatment group to an age‐ and gender‐adjusted general population ( GP ) normative sample. Cox proportional hazard models were fit to examine associations between pre‐treatment levels of HRQ oL and mortality within 1 and 5 years after initiating specific treatment regimens ( HDM / SCT : n = 402; non‐ SCT chemotherapy regimens: n = 172). Among patients who received HDM / SCT , there were significant improvements following treatment in vitality, social functioning, role‐emotional and mental health. Worse pre‐treatment SF ‐36 physical component scores were associated with a greater risk of mortality in both treatment groups and follow‐up periods ( P ≤ 0·005 for both). [Correction added on 20 October 2017, after first online publication: This P value has been corrected]. Using HRQ oL assessments in every physician visit or treatment may provide valuable insights for treating rare conditions like AL amyloidosis.