Changes in patient-reported outcomes in light chain amyloidosis in the first year after diagnosis and relationship to NT-proBNP change

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 (R)-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.

Automated summary

In a prospective cohort study of newly diagnosed systemic light chain (AL) amyloidosis patients, patient-reported outcomes (PROs) were found to fluctuate in the first year of treatment, with domains related to 1-year survival including physical function, fatigue, anxiety, depression, and social roles. The study underscores the importance of measuring and tracking PROs in clinical care and research.