title: The patient's perspective on the symptom and everyday life impact of AL amyloidosis.
abstract: BACKGROUND
  This study aimed to understand the symptomatic impact of amyloid light-chain (AL) amyloidosis from the patient's perspective.
  
  
  METHODS
  Four data sources were included: a literature review, review of online patient blogs, expert clinician interviews and patient interviews. Patients were recruited through the Amyloidosis Foundation and physician referral. Phone interviews were conducted and included open-ended concept elicitation questions. Thematic analysis was performed to identify symptoms and impacts. Descriptive statistics were used to characterize the sample. A conceptual model was developed depicting the impact of disease and treatment.
  
  
  RESULTS
  Two hundred seventy abstracts were identified; 10 articles were deemed relevant. No qualitative studies were identified, and only three studies included patient-reported measures. Ten patients completed interviews (mean age 61 [±8]; 7 male). Over 25 signs/symptoms were identified, including fatigue, weakness, dyspnea, neuropathy, edema, dizziness/lightheadedness, anorexia, diarrhea and constipation. Impacts included reduced physical and social functioning, and emotional impacts, including frustration, anxiety and depression. Findings from the blogs and expert interviews were consistent with patient reports.
  
  
  CONCLUSIONS
  Symptoms can vary widely, but a core set of symptoms were common across patients. The conceptual model derived from this study can be used to ensure a patient-centered approach to drug development.
authors.affiliation: a Millennium Pharmaceuticals, Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Limited , Cambridge , MA , USA .
authors.forename: Huamao Mark
authors.initials: HM
authors.lastname: Lin
authors.affiliation: b Boston Medical Center , Boston , MA , USA , and.
authors.forename: David
authors.initials: D
authors.lastname: Seldin
authors.affiliation: a Millennium Pharmaceuticals, Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Limited , Cambridge , MA , USA .
authors.forename: Ai-Min
authors.initials: AM
authors.lastname: Hui
authors.affiliation: a Millennium Pharmaceuticals, Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Limited , Cambridge , MA , USA .
authors.forename: Deborah
authors.initials: D
authors.lastname: Berg
authors.affiliation: c ICON PRO , Gaithersburg , MD , USA.
authors.forename: C Noelle
authors.initials: CN
authors.lastname: Dietrich
authors.affiliation: c ICON PRO , Gaithersburg , MD , USA.
authors.forename: Emuella
authors.initials: E
authors.lastname: Flood
mesh_terms: D000368:Aged; D058227:Amyloidogenic Proteins / Q000378:metabolism*; D000686:Amyloidosis / Q000378:metabolism / Q000503:physiopathology* / Q000523:psychology; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D011788:Quality of Life
chemical_list: D058227:Amyloidogenic Proteins
keywords: Burden; conceptual model; health-related quality of life; interview; qualitative