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A longitudinal evaluation of health-related quality of life in patients with AL amyloidosis: associations with health outcomes over time.

ABSTRACT: Light chain (AL) amyloidosis is a rare disease associated with significant, irreversible organ dysfunction and high case fatality. An observational study was conducted to assess health-related quality of life (HRQoL) in patients treated for AL amyloidosis between 1994 and 2014 with both high dose melphalan and stem cell transplantation (HDM/SCT) or non-SCT chemotherapy regimens. The SF-36v1^® Health Survey (SF-36) was administered to assess HRQoL during clinic visits. Analysis of variance was used to compare pre- and post-treatment HRQoL within each treatment group to an age- and gender-adjusted general population (GP) normative sample. Cox proportional hazard models were fit to examine associations between pre-treatment levels of HRQoL and mortality within 1 and 5 years after initiating specific treatment regimens (HDM/SCT: n = 402; non-SCT chemotherapy regimens: n = 172). Among patients who received HDM/SCT, there were significant improvements following treatment in vitality, social functioning, role-emotional and mental health. Worse pre-treatment SF-36 physical component scores were associated with a greater risk of mortality in both treatment groups and follow-up periods (P ? 0·005 for both). [Correction added on 20 October 2017, after first online publication: This P value has been corrected]. Using HRQoL assessments in every physician visit or treatment may provide valuable insights for treating rare conditions like AL amyloidosis.

SUBMITTER: Sanchorawala V

PROVIDER: S-EPMC5697591 | biostudies-literature | 2017 Nov

REPOSITORIES: biostudies-literature

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A longitudinal evaluation of health-related quality of life in patients with AL amyloidosis: associations with health outcomes over time.

Sanchorawala Vaishali V McCausland Kristen L KL White Michelle K MK Bayliss Martha S MS Guthrie Spencer D SD Lo Stephen S Skinner Martha M

British journal of haematology 20170829 3

Light chain (AL) amyloidosis is a rare disease associated with significant, irreversible organ dysfunction and high case fatality. An observational study was conducted to assess health-related quality of life (HRQoL) in patients treated for AL amyloidosis between 1994 and 2014 with both high dose melphalan and stem cell transplantation (HDM/SCT) or non-SCT chemotherapy regimens. The SF-36v1<sup>®</sup> Health Survey (SF-36) was administered to assess HRQoL during clinic visits. Analysis of varia ...[more]

PMID: 28850697

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Project description:Systemic AL (light chain) amyloidosis is a rare protein misfolding disorder associated with plasma cell dyscrasia affecting various organs leading to organ dysfunction and failure. The Amyloidosis Forum is a public-private partnership between the Amyloidosis Research Consortium and the US Food and Drug Administration Center for Drug Evaluation and Research with the goal of accelerating the development of effective treatments for AL amyloidosis. In recognition of this goal, 6 individual working groups were formed to identify and/or provide recommendations related to various aspects of patient-relevant clinical trial endpoints. This review summarizes the methods, findings, and recommendations of the Health-Related Quality of Life (HRQOL) Working Group. The HRQOL Working Group sought to identify existing patient-reported outcome (PRO) assessments of HRQOL for use in clinical trials and practice deemed relevant across a broad spectrum of patients with AL amyloidosis. A systematic review of the AL amyloidosis literature identified 1) additional signs/symptoms not currently part of an existing conceptual model, and 2) relevant PRO instruments used to measure HRQOL. The Working Group mapped content from each identified instrument to areas of impact in the conceptual model to determine which instrument(s) provide coverage of relevant concepts. The SF-36v2® Health Survey (SF-36v2; QualityMetric Incorporated, LLC) and Patient-Reported Outcomes Measurement Information System-29 Profile (PROMIS-29; HealthMeasures) were identified as instruments relevant to patients with AL amyloidosis. Existing evidence of reliability and validity was evaluated with a recommendation for future work focused on estimating clinically meaningful within-patient change thresholds for these instruments. For sponsors, the context of use-including specific research objectives, trial population, and investigational product under study-should inherently drive selection of the appropriate PRO instrument and endpoint definitions to detect meaningful change and enable patient-focused drug development.

Project description:ObjectiveTo describe headache characteristics over time in patients with traumatic brain injury (TBI).SettingPatients enrolled and followed at the National Institutes of Health Clinical Center between 2011 and 2020.ParticipantsThere were 147 patients with TBI, with 74 mild TBI (mTBI), 49 moderate (modTBI), 24 severe (sTBI), and 20 individuals without brain injury (IWBIs).DesignRegular surveys of headache characteristics in patients with TBI were conducted. Patients were enrolled as early as 30 days post-injury and followed up to 5 years, for 419 total visits and 80 patients with multiple return visits.Main measuresSurveys of headache characteristics, including headache severity, were measured on a 0- to 10-point Likert scale and headache frequency quantified as headaches per month. Patients with migraine-type headaches ( n = 39) were identified by a clinician-administered tool. Functional outcomes were measured using the Glasgow Outcome Scale-Extended (GOS-E) and quality of life by the Satisfaction with Life Scale (SWLS) and the 36-item Short Form Survey (SF-36).ResultsAt their initial visit, patients with TBI had more severe and frequent headaches than IWBIs (median 5 vs 2.5, P < .001; median 2 vs 0.2, P < .001), as did patients with mTBI compared with modTBI/sTBI (all P ≤ .01). Migraines were associated with lower SWLS and SF-36 scores. Migraines and young age were associated with higher headache severity and frequency across time points. Longitudinally, time post-injury correlated with improvement in headache severity and frequency without differences by injury severity. However, time post-injury did not correlate with improvement in headache characteristics in a patient subgroup with moderate/severe headaches.ConclusionOur findings suggest that patients with mild, moderate, or severe TBI see improvement in headaches over time. However, patients should be counseled that improvement is modest and seen more in patients with milder headache symptoms. Patients with migraine headaches in particular are at risk for worse headache characteristics with greater impact on quality of life.

Dataset Information

A longitudinal evaluation of health-related quality of life in patients with AL amyloidosis: associations with health outcomes over time.

Publications

A longitudinal evaluation of health-related quality of life in patients with AL amyloidosis: associations with health outcomes over time.

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