Abstract
We conducted a prospective cohort study in newly diagnosed systemic light chain (AL) amyloidosis patients (N = 59) to study patient-reported outcomes (PROs) through the first year. The median age was 68 years with 42% female, 8% Black, and 78% lambda subtype. Organ involvement was cardiac in 66%, renal in 58%, with 25% having 3 or greater organs involved. Between baseline and 3 months, all PROMIS®-29 domain scores worsened by 0.4–4.1 points except anxiety which improved by 2.1 points. By 1 year, scores improved compared to the greatest decline at 3 months, most statistically significant for global physical health, physical function, and fatigue. On stage-adjusted survival analysis, in addition to baseline global physical and mental health, domains measuring physical function, fatigue, anxiety, depression, and social roles were associated with 1-year survival. At 1 year, PROMIS measures were associated with NT-proBNP changes and hematologic response. Among patients with an NT-proBNP response, the improvement was seen in physical function, social roles, global mental health, and anxiety. Among patients with an NT-proBNP progression, worsening was seen with anxiety, depression, sleep, and global mental health. Measuring and tracking PROs in patients with AL amyloidosis is important and these important outcomes can be used as correlative endpoints in clinical care/research.
Highlights
Systemic light chain (AL) amyloidosis is a plasma cell neoplasm associated with a multisystem disease with high early mortality and morbidity[1]
Of 61 patients enrolled between 2/1/2016 to 4/15/2019, 1 patient was ineligible based on inclusion criteria and 1 patient had localized instead of systemic amyloidosis
Previous work has shown that the anxiety and uncertainty about symptoms that are often associated with a delayed diagnosis may improve after receiving a diagnosis[18,19]
Summary
Systemic light chain (AL) amyloidosis is a plasma cell neoplasm associated with a multisystem disease with high early mortality and morbidity[1]. Mortality in the first year of newly diagnosed AL amyloidosis can be as high as 35–40%2,3. Stage is currently best determined using the cardiac biomarkers, N-terminal of the prohormone of brain natriuretic peptide (NT proBNP) and troponin T (TnT), and the free light chain burden as determined by the difference between the involved and uninvolved free light chains (dFLC)[4]. Current treatment of AL amyloidosis depends primarily on chemotherapy-based treatment of the amyloidogenic clone to lower the concentration of the involved free light chain. This treatment is suboptimal and has no effect on preformed amyloid fibrils for which endogenous clearance is slow. Despite toxicities, chemotherapy treatment is the mainstay of therapy along with supportive care
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