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Purpose Amyloid transthyretin (ATTR) amyloidosis is a rare, progressive, and fatal disease. The ATTR Patient Symptom Survey (ATTR-PSS) was previously developed through literature review and concept elicitation input from clinicians and patients and revised after evaluation by a patient focus group. This study further evaluated the content validity of the ATTR-PSS through qualitative cognitive debriefing interviews with clinicians and patients. Methods Seven clinicians and 10 patients with ATTR amyloidosis were interviewed individually regarding their overall impressions, the clarity and appropriateness of the survey, relevance of concepts measured, and comprehensiveness and comprehensibility of items and response choice sets. Results Clinicians acknowledged the usefulness of the ATTR-PSS in research and clinical settings. They suggested minor modifications to the survey instructions, the addition of 3 symptoms, and the transfer of 10 conditions from the symptom list to 2 separate items. Patients found the ATTR-PSS to be easy to complete and relevant to their experiences. Their feedback resulted in modification to instruction text, edits to the description of 4 symptoms, removal of 1 symptom, and addition of 2 diagnoses. Conclusion The findings support the content validity of the ATTR-PSS as an appropriate measure of symptom frequency, severity, and impact in patients with wild-type and hereditary ATTR amyloidosis.

Purpose Cardiac dysfunction is common in amyloid light-chain (AL) amyloidosis, a rare disease caused by extracellular deposition of misfolded immunoglobulin light chains. This study aimed to examine economic/clinical disease burden in hospitalized cardiac amyloidosis patients. Patients and methods Cardiac amyloidosis patients ≥18 years old hospitalized between 2014 and 2016 were identified in claims if they had ≥1 inpatient claim consistent with amyloidosis and evidence of cardiac dysfunction. Descriptive statistics were reported. Results 3239 cardiac amyloidosis patients [1795 (55.4%) with concurrent renal disease] were identified. Mean (SD) length of stay was 8.3 (11.1) days. 25.2% were admitted to the intensive care unit. Mean overall hospitalization costs were USD$20,584. In-hospital mortality was 9.0% overall. 16.8% were readmitted within 30 days, with 11.2% dying in-hospital and a mean readmission cost of USD$18,536. Conclusion Hospitalization for cardiac amyloidosis is costly, with high rates of readmission and mortality. Opportunities exist to improve care.

Introduction Light-chain (AL) amyloidosis is a rare, progressive, and typically fatal disease. Health-related quality of life (HRQoL) has been shown to be a significant prognostic factor associated with clinical outcomes such as survival and response to treatment. A better understanding of how patterns of HRQoL may be prospectively associated with costly healthcare resource utilization, such as emergency department (ED) visits and inpatient hospitalizations, is warranted. Methods A secondary data analysis of a non-interventional, longitudinal online study of patients with AL amyloidosis (n = 224) was conducted. Negative binomial regression models were used to examine whether initial HRQoL scores (as measured by the SF-36v2® Health Survey [SF-36v2], where higher scores reflect better HRQoL) and changes in HRQoL were associated with the number of ED visits and inpatient hospitalizations during a 12-month period. Incidence rate ratios were interpreted by 5-point decrements in initial HRQoL scores and minimally important changes in HRQoL change scores. Results There were significant inverse associations between initial SF-36v2 scores and subsequent rates of ED visits and inpatient hospitalizations across all domains and summary components (p

Aims: Examine mortality and healthcare costs in Medicare beneficiaries with newly diagnosed immunoglobulin light chain (AL) amyloidosis. Patients & methods: Cases were identified in 2012–2015 Medicare 5% data with ≥1 inpatient/≥2 outpatient claims consistent with AL amyloidosis and ≥1 AL-specific treatment. Cases were matched 3:1 with disease-free controls. Descriptive statistics were reported. Results: A total of 249 (33.3%) cases were matched to 747 (66.7%) controls. A total of 19.7% of cases died within 1 year of follow-up versus 5.5% of controls; 30.6 versus 11.8% died within 2 years (p < 0.001). Mean (SD) costs in 1-year of follow-up were significantly higher among cases versus controls ($71,040 [65,766] vs $13,722 [27,493]; p Conclusion: Mortality was nearly four-times higher, and costs nearly five-times higher in beneficiaries with AL amyloidosis versus controls.

Aim: To estimate healthcare utilization and costs in amyloid light-chain (AL) amyloidosis. Patients & methods: AL amyloidosis patients were identified in 2007–2015 claims databases if they had ≥1 inpatient/≥2 outpatient claims consistent with AL amyloidosis and received ≥1 AL-specific treatment. Descriptive statistics were reported. Results: 50.1% (n = 3670) were admitted ≥1 time during the year, 11.3% (n = 827) ≥3 times. From 2007 to 2015, bortezomib use increased from 4.6 to 25.3%; melphalan use decreased from 18.9 to 2.0%; costs increased from 92,866 to $114,030. Among incident patients with at least 2 years of follow-up, healthcare utilization and costs decreased from first to second year post-diagnosis. Conclusion: AL chemotherapy-based prescribing practices changed. Total annual healthcare costs increased over time among AL amyloidosis patients.

Background Light chain (AL) amyloidosis is a rare, complex disease associated with significant morbidity and mortality. Delays in diagnosis are common and may have detrimental consequences on patients’ prognosis. Too little is known regarding the patient journey to diagnosis. Objective The objective of this study was to describe the patient-reported journey to a correct diagnosis for AL amyloidosis. Methods Using a mixed-methods approach, data were collected from clinician (n = 4) and patient (n = 10) interviews and a survey of community-based patients with AL amyloidosis (n = 341). Data were used to document the patient experience between the onset of symptoms and the receipt of a diagnosis. Results Delays in diagnosis were common. Qualitative and quantitative data indicated that initial symptoms were varied and similar to other more prevalent diseases. Two themes regarding the journey to diagnosis emerged: (1) barriers to an early diagnosis; and (2) the emotional toll of the journey. Time to diagnosis was heavily influenced by how patients interpreted their initial symptoms, whether they sought early medical help, and challenges associated with making differential diagnoses. Survey results indicate that patients with primary cardiac involvement were more likely to receive a delayed diagnosis than those with primary kidney involvement. Patients described mixed emotions associated with the eventual diagnosis of AL amyloidosis. Conclusions These data support a need for better early identification and support for patients seeking a diagnosis. Increasing clinician awareness may reduce the time to diagnosis. Additional research is needed to identify optimal diagnostic testing to reduce delays in treatment initiation and subsequent severe impacts on health.

Amyloid light-chain (AL) amyloidosis is a rare disease caused by extracellular deposition of misfolded immunoglobulin light chains. This study aimed to provide an up-to-date estimate of prevalence and incidence of AL amyloidosis in the United States. Using claims databases from years 2007 to 2015, adults ≥18 years old with AL amyloidosis were included if they had (1) at least 1 inpatient or 2 outpatient claims consistent with AL amyloidosis and (2) received 1 AL-specific treatment. Prevalence was calculated as the number of AL patients divided by the number of enrollees on June 30th of each calendar year. Incidence was calculated as the number of patients with AL who were disease-free and enrolled with a health plan for 1 year prior, divided by the number of enrollees with enrollment from July 1st of the previous year to June 30th of each calendar year. The prevalence of AL amyloidosis increased significantly between 2007 and 2015, from 15.5 cases per million in 2007 to 40.5 in 2015, an annual percentage change (APC) of 12% ( P P = .114) with no statistically significant increase. There was an increase in AL amyloidosis prevalence over a 9-year period coupled with stable incidence rates. Although there is no diagnosis code specific to AL amyloidosis and no validated method for identifying this condition using claims data, extrapolating from our data, there are at least 12 000 adults in the United States living with AL amyloidosis, and the number seems likely to rise.