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Avery A Rizio,1 Kristen L McCausland,1 Michelle K Carty,1 Ansgar Conrad,2 Tiffany P Quock2 1QualityMetric Incorporated, LLC, Johnston, RI, USA; 2Prothena Biosciences Inc, South San Francisco, CA, USACorrespondence: Avery A Rizio, QualityMetric Incorporated, LLC, 1301 Atwood Avenue, Suite 216E, Johnston, RI, 02919, USA, Tel +1 401-903-4631, Email arizio@qualitymetric.comPurpose: The purpose of this secondary data analysis was to examine the association between hematologic response and health-related quality of life (HRQoL) among patients with light-chain (AL) amyloidosis.Patients and Methods: Data for this secondary analysis were collected through a non-interventional, longitudinal, online self-report survey of patients with AL amyloidosis. Patients completed an initial online survey, with follow-up surveys administered 1, 6, 12, 18, and 24 months after completion of the initial survey. The online survey included an assessment of patients’ most recent self-reported hematologic response status. Eight domains and 2 summary components of HRQoL were evaluated with the SF-36v2® Health Survey. A series of logistic regression models were used to examine the association between self-reported hematologic response at 24 months (dichotomized as new or maintained complete hematologic response; less than a complete response) and change in HRQoL from baseline to 24 months (dichotomized as meaningful worsening; improvement or preservation).Results: For all measured domains of HRQoL except physical functioning, there was no statistically significant relationship between meaningful worsening in HRQoL and hematologic response status at 24 months. Patients without a complete hematologic response had an odds of experiencing meaningful worsening of HRQoL that was similar to that of patients with a complete hematologic response.Conclusion: Among patients with AL amyloidosis, change in HRQoL was generally not associated with hematologic response. Achieving a complete hematologic respons

Meaghan O’Connor,1 Kristen Hsu,2 Lynne Broderick,1 Kristen L McCausland,1 Kaitlin LaGasse,1 Sabrina Rebello,2 Michelle Carty,1 Isabelle Lousada2 1QualityMetric Incorporated LLC, Johnston, RI, USA; 2Amyloidosis Research Consortium, Newton, MA, USACorrespondence: Kristen Hsu, Amyloidosis Research Consortium, 320 Nevada Street, Suite 210, Newton, MA, 02460, USA, Tel +1-617-467-5170, Email khsu@arci.orgPurpose: Patients with transthyretin amyloidosis (ATTR) experience a wide variety of symptoms and impacts on health-related quality of life (HRQoL). However, the lack of an ATTR-specific patient-reported outcome (PRO) measure has made consistent measurement of HRQoL in ATTR challenging. This paper describes the development of a conceptual model and subsequent content for the Transthyretin Amyloidosis – Quality of Life Questionnaire (ATTR-QOL), an ATTR-specific PRO measure.Methods: This was a cross-sectional, non-interventional, US-based study. The study design included three stages: 1) a targeted literature review followed by qualitative data collection with patients and experts; 2) development of a conceptual model and PRO measure; and 3) review of the PRO measure using a modified Delphi method, translatability assessment, and interviews with patients and experts. Revisions were made to the measure after each round of review.Results: Forty-four patients and 29 experts participated in this study. The conceptual model included two primary concepts of interest: symptoms (cardiac, neuropathic–peripheral, neuropathic–autonomic, and other) and impacts (eg, physical, role, and mental/emotional functioning). Seventy-two items were created (32 symptoms; 40 impacts) to align with the model. A recall period of one month was selected based on participant input.Conclusion: The ATTR-QOL was created with significant patient involvement and guidance from a multidisciplinary group of experts. The mix of patient and clinical perspectives helped to ensure a balanced representation

Avery A Rizio,1 Kristen L McCausland,1 Michelle K White,1 Tiffany P Quock2 1QualityMetric Incorporated, LLC, Johnston, RI, USA; 2Prothena Biosciences Inc, South San Francisco, CA, USACorrespondence: Avery A RizioQualityMetric Incorporated, LLC, 1301 Atwood Avenue, Suite 216E, Johnston, RI, 02919, USATel +1 401-903-4631Email arizio@qualitymetric.comBackground: Light chain (AL) amyloidosis is a rare and progressive disease that affects myriad organs and systems. Patients with cardiac involvement have the highest risk of death. This report compiles findings across three cohorts of patients with AL amyloidosis to understand patterns of employment and work impacts.Methods: Data came from three cohorts recruited through patient advocacy organizations in the US. Patients in Cohort 1 completed the SF-36v2® Health Survey (SF-36v2), the Work Productivity and Activity Impairments – Specific Health Problem (WPAI) questionnaire, and the 12-item Kansas City Cardiomyopathy Questionnaire (KCCQ-12). The relationship between work impacts (WPAI scores) and HRQoL (SF-36v2 scores) was investigated using multivariable logistic regression and summarized according to cardiac severity using New York Heart Association (NYHA) classes estimated from KCCQ-12 scores. Changes in employment, days of missed work, and long-term disability due to AL amyloidosis were summarized for patients diagnosed in the past 24 months and stratified by NYHA class (Cohort 2). Findings were contextualized using patient interviews (Cohort 3).Results: Work-related impacts, especially reduced productivity, were common among patients with AL amyloidosis. WPAI scores were significantly related to HRQoL (p< 0.05 for all models). Among patients with cardiac involvement, the greatest degree of work impacts was observed for those in NYHA class 3 or 4. Changes in employment, missed work, and long-term disability were common among newly diagnosed patients, especially among those in NYHA class 3 or 4. Patient interviews s

Michelle K White,1 Kristen L McCausland,1 Vaishali Sanchorawala,2 Spencer D Guthrie,3 Martha S Bayliss1 1Optum, Johnston, RI, 2Department of Medicine, Section of Hematology/Oncology, Amyloidosis Center, Boston University School of Medicine, Boston, MA, 3Medical Affairs, Prothena Biosciences Inc, South San Francisco, CA, USA Background: Light chain (AL) amyloidosis, a rare and life-threatening protein misfolding disorder, causes organ damage and severely impacts health-related quality of life (HRQoL). No patient-reported outcome (PRO) HRQoL measure has been validated for use in an AL amyloidosis patient population, leaving a gap for researchers conducting observational studies and clinical trials for drug development. The SF-36 Health Survey (SF-36) has been the most frequently used PRO in AL amyloidosis studies to date, and early qualitative validation studies support its use in this population. The aim of this study was to assess the psychometric properties of the SF-36 among patients with AL amyloidosis. Methods: Data from community-based (n=341) and clinic-based (n=1,438) observational studies were used to document the psychometric properties of the SF-36 in this disease population. Reliability was estimated using internal consistency (Cronbach’s alpha) and test–retest reliability (intraclass correlation). Convergent validity, known-groups validity, and the ability to detect change were assessed with available criterion variables. Results: Scale reliability (Cronbach’s alpha  ≥0.780 for all scores) and test–retest reliability (intraclass correlation coefficients ≥0.731 for all) were acceptable. Scale convergent validity was supported by strong correlations with conceptually related measures. Mean SF-36 scores varied by response to treatment (P<0.05 for all scores) and a self-reported measure of disease severity (P<0.001 for all scores). Data indicate that the SF-36 is sensitive to chan