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The article focuses on a study which examines cardiac biomarkers as well as the health-related quality of life in patients with light chain (AL) amyloidosis, which is a rare and often fatal disease caused by misfolded light chains, that form soluble toxic aggregates that deposit as fibrils in tissue and organs. Information about disease progression and cardiac response to treatment is presented.

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 ( HRQoL) 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 HRQoL during clinic visits. Analysis of variance was used to compare pre- and post-treatment HRQoL 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 HRQoL 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 HRQoL assessments in every physician visit or treatment may provide valuable insights for treating rare conditions like AL amyloidosis. [ABSTRACT FROM AUTHOR]