scholarly journals Quality of life data for individuals affected by Spinal Muscular Atrophy: A baseline dataset from the Cure SMA Community Update Survey

2020 ◽  
Author(s):  
Lisa Belter ◽  
Rosángel Cruz ◽  
Jill Jarecki
Keyword(s):  
Quality Of Life ◽  
Spinal Muscular Atrophy ◽  
Functional Status ◽  
Outcome Measures ◽  
Muscular Atrophy ◽  
Work Productivity ◽  
Type I ◽  
Activity Impairment ◽  
Patient Reported

Abstract BACKGROUND Individuals and/or caregivers of individuals affected by Spinal Muscular Atrophy (SMA) completed the 2019 Cure SMA Community Update Survey, online, assessing health-related quality of life (HRQoL), loss of work productivity, and fatigue using the Health Utilities Index Questionnaire (HUI), the Work Productivity and Activity Impairment Questionnaire (WPAI), and the Patient Reported Outcomes Measurement Information System Fatigue Short Form (PROMIS Fatigue SF), respectively. The purpose was to collect baseline quality of life results among individuals affected by SMA using the above Patient Reported Outcome Measures (PROMs). RESULTS Of 666 surveys completed between March and May 2019, 478 were included in this analysis, accounting for duplicates, missing data, or deaths. The breakdown across SMA type I, II and III was 25%, 47% and 28%, respectively. Responses were characterized by current functional status/milestone, with subsets for “permanent ventilation,” “non-sitters,” “sitters,” “walk with support,” and “walk alone.” WPAI and HUI respondents included affected adults and caregivers. The PROMIS Fatigue SF was completed by the primary caregiver of affected children. Overall, those affected by a less severe form of SMA and with a higher functional status reported higher HRQoL and lower work productivity and activity impairment. All affected individuals reported higher fatigue levels than the general population. CONCLUSIONS This study offers useful insights into the burden of SMA among affected individuals and their caregivers. The results provide a baseline picture of the patient and caregiver experience with SMA in a post-treatment era from which to measure year-over-year changes in quality of life scores from new therapies and improved care. The WPAI demonstrates the significant impact of work productivity among SMA populations. Aspects of the HUI seem more appropriate to certain SMA sub-populations than others. Measures from the PROMIS Fatigue SF appear to under-represent the burden of fatigue often reported by SMA individuals and caregivers; this may, perhaps be due to a lack of sensitivity in the questions associated with fatigue in the SMA affected population, when compared with other studies on this topic. Overall, these results suggest the need for SMA-specific quality of life outcome measures to fully capture clinically meaningful change in the SMA population.

scholarly journals Quality of life data for individuals affected by Spinal Muscular Atrophy: A baseline dataset from the Cure SMA Community Update Survey

2020 ◽  
Author(s):  
Lisa Belter ◽  
Rosángel Cruz ◽  
Jill Jarecki
Keyword(s):  
Quality Of Life ◽  
Spinal Muscular Atrophy ◽  
Functional Status ◽  
Outcome Measures ◽  
Muscular Atrophy ◽  
Work Productivity ◽  
Type I ◽  
Activity Impairment ◽  
Patient Reported

Abstract BACKGROUND Individuals and/or caregivers of individuals affected by Spinal Muscular Atrophy (SMA) completed the 2019 Cure SMA Community Update Survey, online, assessing health-related quality of life (HRQoL), loss of work productivity, and fatigue using the Health Utilities Index Questionnaire (HUI), the Work Productivity and Activity Impairment Questionnaire (WPAI), and the Patient Reported Outcomes Measurement Information System Fatigue Short Form (PROMIS Fatigue SF), respectively. The purpose was to collect baseline quality of life results among individuals affected by SMA using the above Patient Reported Outcome Measures (PROMs). RESULTS Of 666 surveys completed between March and May 2019, 478 were included in this analysis, accounting for duplicates, missing data, or deaths. The breakdown across SMA type I, II and III was 25%, 47% and 28%, respectively. Responses were characterized by current functional status/milestone, with subsets for “permanent ventilation,” “non-sitters,” “sitters,” “walk with support,” and “walk alone.” WPAI and HUI respondents included affected adults and caregivers. The PROMIS Fatigue SF was completed by the primary caregiver of affected children. Overall, those affected by a less severe form of SMA and with a higher functional status reported higher HRQoL and lower work productivity and activity impairment. All affected individuals reported higher fatigue levels than the general population. CONCLUSIONS This study offers useful insights into the burden of SMA among affected individuals and their caregivers. The results provide a baseline picture of the patient and caregiver experience with SMA in a post-treatment era from which to measure year-over-year changes in quality of life scores from new therapies and improved care. The WPAI demonstrates the significant impact of work productivity among SMA populations. Aspects of the HUI seem more appropriate to certain SMA sub-populations than others. Measures from the PROMIS Fatigue SF appear to under-represent the burden of fatigue often reported by SMA individuals and caregivers; this may, perhaps be due to a lack of sensitivity in the questions associated with fatigue in the SMA affected population, when compared with other studies on this topic. Overall, these results suggest the need for SMA-specific quality of life outcome measures to fully capture clinically meaningful change in the SMA population.

A Prospective, Crossover Survey Study of Child- and Proxy-Reported Quality of Life According to Spinal Muscular Atrophy Type and Medical Interventions

2020 ◽  
Vol 35 (5) ◽  
pp. 322-330
Author(s):  
Meaghann S. Weaver ◽  
Rewais Hanna ◽  
Scott Hetzel ◽  
Karen Patterson ◽  
Alice Yuroff ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Spinal Muscular Atrophy ◽  
Muscular Atrophy ◽  
Survey Study ◽  
Type I ◽  
Family Impact ◽  
Proxy Report ◽  
Spinal Muscular Atrophy Type ◽  
Medical Interventions

Background: Spinal muscular atrophy is an autosomal-recessive, progressive neuromuscular disease associated with extensive morbidity. Children with spinal muscular atrophy have potentially increased life spans due to improved nutrition, respiratory support, and novel pharmaceuticals. Objectives: To report on the quality of life and family experience for children with spinal muscular atrophy with attentiveness to patient- and proxy-concordance and to stratify quality of life reports by spinal muscular atrophy type and medical interventions. Methods: A prospective, crossover survey study inclusive of 58 children (26 spinal muscular atrophy type I, 23 type II, 9 type III) and their family caregivers at a free-standing Midwestern children’s hospital. Twenty-eight families completed the 25-item PedsQL 3.0 Neuromuscular Module. Forty-four participants completed the 36-item PedsQL Family Impact Module and 47 completed the Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) questionnaire. Results: The PedsQL Family Impact Module demonstrated significant differences between spinal muscular atrophy types I and II in functioning domains including physical, emotional, social, and family relations ( P < .03). Child self-report and proxy report surveys demonstrated significant differences between spinal muscular atrophy types in the communication domains ( P < .003). Children self-reported their quality of life higher than proxy report of child quality of life. Gastrostomy tube ( P = .001) and ventilation support ( P = .029) impacted proxy-reported quality of life perspectives, whereas nusinersen use did not. Spinal surgery was associated with improved parental quality of life and family impact ( P < .03). Conclusions: The measurement and monitoring of quality of life for children with spinal muscular atrophy and their families represents an implementable priority for care teams.

scholarly journals Asthma control is associated with economic outcomes, work productivity and health-related quality of life in patients with asthma

2020 ◽  
Vol 7 (1) ◽  
pp. e000534
Author(s):  
Lulu K Lee ◽  
Karthik Ramakrishnan ◽  
Guilherme Safioti ◽  
Rinat Ariely ◽  
Michael Schatz
Keyword(s):  
Quality Of Life ◽  
Asthma Control ◽  
Work Productivity ◽  
Emergency Department Visits ◽  
Health Related Quality ◽  
Activity Impairment ◽  
Patient Reported ◽  
Related Quality ◽  
Health Related

BackgroundThe objective of this analysis was to examine the association between asthma control (based on Asthma Control Test (ACT) responses) and healthcare resource utilisation (HRU), work productivity and health-related quality of life (HRQoL) among a nationwide sample of US adults with a self-reported diagnosis of asthma and without comorbid chronic obstructive pulmonary disease.MethodsData were obtained from the 2015 and 2016 self-administered, internet-based National Health and Wellness Surveys. Patients were grouped by ACT score (≤15: poorly controlled; 16–19: partly controlled; 20–25: well-controlled asthma). Study outcomes included HRU (patient-reported healthcare provider visits, emergency department visits and hospitalisations during the previous 6 months); work productivity, measured using the Work Productivity and Activity Impairment-General Health Scale; HRU-associated costs and work productivity loss and HRQoL, measured using EuroQoL-5 Dimensions-5 Levels (EQ-5D-5L) and the Short Form Health Survey-36V.2 (SF-36V.2). Incremental differences in outcomes between groups were assessed using generalised linear models adjusted for covariates.ResultsOf 7820 eligible adults, 17.4% had poorly controlled, 20.1% partly controlled and 62.5% well-controlled asthma. Well-controlled asthma was associated with significantly lower HRU (p<0.001) and lower mean direct costs ($6012 vs $8554 and $15 262, respectively; p<0.001); well-controlled asthma was also associated with significantly lower mean scores for work absenteeism, work presenteeism, overall work impairment and activity impairment (all p<0.001), and lower mean indirect costs ($6353 vs $10 448 and $14 764, respectively; p<0.001). Clinically meaningful differences favouring well-controlled asthma were seen for all HRQoL measures, with statistically significantly higher adjusted mean EQ-5D-5L index and SF-6D Health Utilities Index scores (derived from SF-36V.2) for patients with well-controlled asthma compared with partly controlled or poorly controlled asthma (p<0.001).ConclusionsThe study demonstrates a clear relationship between asthma control and its impact on HRU, costs, work productivity and HRQoL. This will allow for better identification and management of patients with poorly controlled asthma.

scholarly journals AB0727 WORK OUTCOMES AND ASSOCIATED FACTORS IN ANKYLOSING SPONDYLITIS PATIENTS IN CHINA

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 1658.1-1658
Author(s):  
L. Tu ◽  
Y. Xie ◽  
Q. Lv ◽  
M. Yang ◽  
Z. Liao ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Ankylosing Spondylitis ◽  
Disease Duration ◽  
Productivity Loss ◽  
Work Productivity ◽  
Work Outcomes ◽  
Activity Impairment ◽  
Factors Associated ◽  
The Impact

Background:Poorer work productivity due to pain and functional impairment is commonly seen in ankylosing spondylitis (AS) patients, which may contribute to huge social economic burden. However, data about work outcomes and associated factors in Chinese AS patients were barely reported.Objectives:To assess work outcomes and identify factors associated with poor work productivity in patients with AS in China.Methods:A cross-sectional study was conducted in China. Adult patients (aged ≥ 18 years) fulfilled the 1984 New York modified criteria of AS were enrolled from rheumatology center from Jan 2017 to Aug 2017. All participants completed questionnaires about socio-demographic characteristics, disease characteristics, quality of life and the Work productivity and activity impairment questionnaire in AS (WPAI:SpA) to accesses the impact of chronic health conditions on job performance and productivity. Factors associated with work outcomes were evaluated.Results:A total of 91 patients with AS were included: 87.8% males, 78.02% employed, mean age and disease duration of 30 and 10 years respectively. The mean (SD) activity impairment of all patients was 48.57% (22.02%). For patients with employed work, mean (SD) absenteeism, presenteeism and work productivity loss were 10.22% (19.44%), 43.86% (22.48%) and 47.92% (25.81%) respectively. In multivariable analysis, activity impairment was associated with Bath Ankylosing Spondylitis Functional Index (BASFI) (P<0.01) and Ankylosing Spondylitis Quality of Life (ASQoL) (P<0.01). Absenteeism was associated with disease duration (P=0.03). Presenteeism was associated with disease duration (P=0.04), BASFI (P<0.01) and ASQoL (P<0.01). Work productivity loss was associated with BASFI (P<0.01) and ASQoL (P<0.01).Conclusion:Longer disease duration, reduced physical function and poorer quality of life are associated with reduced work productivity in Chinese AS patients.References:[1]Boonen A, van der Heijde D, Landewe R, Spoorenberg A, Schouten H, Rutten-van Molken M, et al. Work status and productivity costs due to ankylosing spondylitis: comparison of three European countries. Annals of the rheumatic diseases. 2002;61(5):429-37.[2]Martindale J, Shukla R, Goodacre J. The impact of ankylosing spondylitis/axial spondyloarthritis on work productivity. Best Pract Res Clin Rheumatol. 2015;29(3):512-23.[3]Castillo-Ortiz JD, Ramiro S, Landewe R, van der Heijde D, Dougados M, van den Bosch F, et al. Work Outcome in Patients With Ankylosing Spondylitis: Results From a 12-Year Followup of an International Study. Arthritis Care Res (Hoboken). 2016;68(4):544-52.[4]Sag S, Nas K, Sag MS, Tekeoglu I, Kamanli A. Relationship of work disability between the disease activity, depression and quality of life in patients with ankylosing spondylitis. J Back Musculoskelet Rehabil. 2018;31(3):499-505.[5]Goh Y, Kwan YH, Leung YY, Fong W, Cheung PP. A cross-sectional study on factors associated with poor work outcomes in patients with axial spondyloarthritis in Singapore. Int J Rheum Dis. 2019;22(11):2001-8.Disclosure of Interests:None declared

Patient Reported Outcome Measures of Health-Related Quality of Life and Asthenia after Thyroidectomy

2021 ◽  
Vol 264 ◽  
pp. 394-401
Author(s):  
Mary Kate Luddy ◽  
Rachel Vetter ◽  
Jessica Shank ◽  
Whitney Goldner ◽  
Anery Patel ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Outcome Measures ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Health Related Quality ◽  
Patient Reported ◽  
Related Quality ◽  
Health Related

scholarly journals AB0814 SUSTAINED IMPROVEMENTS IN PHYSICAL FUNCTION, QUALITY OF LIFE, AND WORK PRODUCTIVITY WITH IXEKIZUMAB IN PATIENTS WITH ACTIVE PSORIATIC ARTHRITIS AND PREVIOUS INADEQUATE RESPONSE TO TUMOUR NECROSIS FACTOR-INHIBITORS: 3-YEAR RESULTS FROM SPIRIT-P2 TRIAL

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 1709-1710
Author(s):  
A. M. Orbai ◽  
J. Gratacos-Masmitja ◽  
E. Dokoupilova ◽  
B. Combe ◽  
A. Constantin ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Physical Function ◽  
Work Productivity ◽  
Inadequate Response ◽  
Research Support ◽  
Activity Impairment ◽  
Sf 36 ◽  
Intent To Treat ◽  
Treat Population

Background:Ixekizumab (IXE), a high-affinity monoclonal antibody that selectively targets IL-17A, has shown improvements compared to placebo (PBO) not only in disease activity but also in various patient-reported outcomes (PROs) assessing physical function, quality of life (QoL), and work productivity in PsA patients treated for 24 weeks and sustained up to 52 weeks.1, 2Objectives:To report the effects of treatment with IXE on these PROs after up to 3 years of treatment.Methods:In SPIRIT-P2 (NCT02349295), a Phase 3 trial, 363 adult patients with active PsA and prior inadequate response or intolerance to 1 or 2 TNF inhibitors (TNFis) were randomized 1:1:1 to IXE 80 mg every 4 weeks (IXEQ4W; N=122) or every 2 weeks (IXEQ2W; N=123), or PBO (N=118) in the double-blind treatment period (Weeks 0-24). Both IXE regimens had a starting dose of 160 mg. Results are reported from a subset of the intent-to-treat population who were randomized to IXE at baseline (Week 0). The following PROs were assessed during Weeks 0-156: HAQ-DI (minimally clinically important difference [MCID] an improvement ≥0.35), medical outcomes survey Short Form-36 (SF-36) Physical and Mental Component Summary (PCS and MCS), European Quality of Life 5 Dimensions Visual Analog Scale (EQ-5D VAS), and Work Productivity and Activity Impairment Questionnaire-Specific Health Problem (WPAI-SHP; absenteeism, presenteeism, work productivity, and activity impairment). Missing values were imputed by observed analysis and modified baseline observation carried forward (mBOCF) for continuous data or by modified non-responder imputation (mNRI) for categorical data.Results:Mean baseline scores for SF-36 (PCS and MCS), EQ-5D VAS, and WPAI-SHP (Figure 1) and HAQ-DI (mean [SD]: IXEQ4W=1.2 [0.6]; IXEQ2W=1.2 [0.6]), indicated impaired physical function and QoL. The percentage of patients of who completed 156 weeks of the study in IXEQ4W and IXEQ2W arms were 57.4% (n=70) and 44.7% (n=55), respectively. Patients receiving IXE treatment up to 3 years reported sustained improvements in SF-36 (PCS and MCS), EQ-5D VAS, and WPAI-SHP (presenteeism, work productivity, and activity impairment) (Figure 1). Observed HAQ-DI mean change from baseline in IXEQ4W: -0.46 (0.62) and IXEQ2W: -0.48 (0.55). The percentage of IXE treated patients achieving MCID for HAQ-DI (improvement ≥0.35) was sustained at 3 years (Figure 2).Figure 1.Summary of Patient-Reported Outcomes presented as change from baseline at Week 156 (Observed and mBOCF): Intent-to-Treat Population (Patients Randomized to IXE at Baseline)Figure 2.Patients achieving HAQ-DI MCID Response up to Week 156 (Observed) and at Week 156 (mNRI) among patients with HAQ-DI≥0.35 at baseline: Intent-to-Treat Population (Patients Randomized to IXE at Baseline)Conclusion:Improvements in PROs, measuring physical and mental function, quality of life, and work productivity are maintained up to 3 years with IXE treatment in patients with active PsA who have had an inadequate response or intolerance to 1 or 2 TNFis.References:[1]Nash P, et al. Lancet. 2017;389(10086):2317-2327.[2]Genovese MC, et al. Rheumatology (Oxford). 2018;57(11):2001-2011.Disclosure of Interests:Ana-Maria Orbai Grant/research support from: Abbvie, Eli Lilly and Company, Celgene, Novartis, Janssen, Horizon, Consultant of: Eli Lilly; Janssen; Novartis; Pfizer; UCB. Ana-Maria Orbai was a private consultant or advisor for Sun Pharmaceutical Industries, Inc, not in her capacity as a Johns Hopkins faculty member and was not compensated for this service., Jordi Gratacos-Masmitja Grant/research support from: a grant from Pfizzer to study implementation of multidisciplinary units to manage PSA in SPAIN, Consultant of: Pfizzer, MSD, ABBVIE, Janssen, Amgen, BMS, Novartis, Lilly, Speakers bureau: Pfizzer, MSD, ABBVIE, Janssen, Amgen, BMS, Novartis, Lilly, Eva Dokoupilova Grant/research support from: Eli Lilly and Abbvie, Bernard Combe Grant/research support from: Novartis, Pfizer, Roche-Chugai, Consultant of: AbbVie; Gilead Sciences, Inc.; Janssen; Eli Lilly and Company; Pfizer; Roche-Chugai; Sanofi, Speakers bureau: Bristol-Myers Squibb; Gilead Sciences, Inc.; Eli Lilly and Company; Merck Sharp & Dohme; Pfizer; Roche-Chugai; UCB, Arnaud Constantin Grant/research support from: Study was sponsored by Sanofi Genzyme, Consultant of: Consulting fees from Abbvie, BMS, Celgene, Gilead, Janssen, Lilly, Novartis, Pfizer, Roche, Sanofi, UCB, Amanda M. Gellett Shareholder of: Eli Lilly and company, Employee of: Eli Lilly and company, Aubrey Trevelin Sprabery Shareholder of: Eli Lilly and Company, Employee of: Eli Lilly and Company, Julie Birt Shareholder of: Eli Lilly and Company, Employee of: Eli Lilly and Company, Vladimir Geneus Shareholder of: Eli Lilly and Company, Employee of: Eli Lilly and Company, Peter Nash Grant/research support from: AbbVie, Bristol-Myers Squibb, Celgene, Eli Lilly and Company, Gilead, Janssen, MSD, Novartis, Pfizer Inc, Roche, Sanofi, UCB, Consultant of: AbbVie, Bristol-Myers Squibb, Celgene, Eli Lilly, Gilead, Janssen, MSD, Novartis, Pfizer Inc, Roche, Sanofi, UCB, Speakers bureau: AbbVie, Bristol-Myers Squibb, Celgene, Eli Lilly, Gilead, Janssen, MSD, Novartis, Pfizer Inc, Roche, Sanofi, UCB

Trends in Hidradenitis Suppurativa Disease Severity and Quality of Life Outcome Measures: A Systematic Review (Preprint)

2021 ◽  
Author(s):  
Jalal Maghfour ◽  
Torunn Elise Sivesind ◽  
Cory A. Dunnick ◽  
Robert Paul Dellavalle
Keyword(s):  
Quality Of Life ◽  
Systematic Review ◽  
Disease Severity ◽  
Outcome Measures ◽  
Hidradenitis Suppurativa ◽  
Life Quality ◽  
Clinical Score ◽  
Validity And Reliability ◽  
Patient Reported

BACKGROUND While there has been an increase in the number of randomized, controlled trials (RCTs) evaluating treatment efficacy for HS, instrument measurements of disease severity and quality of life (QoL) are varied, making compilation of data and comparison between studies a challenge for clinicians. OBJECTIVE The aim of this review is to evaluate trends in disease severity scales and patient reported outcome measures used in RCTs assessing treatment interventions among HS patients. METHODS A primary systematic literature review was conducted in August 2020. PubMed/MEDLINE, Embase, Web of Science, and Cochrane databases were used to identify all articles published from January 1964 to July 2020. The study was prospectively registered with PROSPERO (ID: 209582). Twenty-five articles were included in the systematic review. RESULTS Sartorius and modified Sartorius scores (n=8), and Hidradenitis Suppurativa Clinical score (HiSCs) (n=8) were the most commonly used instruments for disease severity. Participants’ pain, followed by Dermatology Life Quality Index (DLQI), were the most common QoL measures used in the reviewed studies. CONCLUSIONS Heterogeneity of data characterizing both the validity and reliability of existing outcome measures hinders interpretation and translation of the results from RCTs into clinical practice. Many of the QoL measures identified were not specific to HS and may not be representative of all factors impacting patients.

Sign in / Sign up

Export Citation Format

Share Document