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Quality of life in long term ventilated adult patients with Duchenne muscular dystrophy

Published on Aug 1, 2019in Neuromuscular Disorders2.612
· DOI :10.1016/j.nmd.2019.06.599
Grazia Crescimanno7
Estimated H-index: 7
(National Research Council),
Francesca Greco4
Estimated H-index: 4
+ 2 AuthorsOreste Marrone22
Estimated H-index: 22
(National Research Council)
Abstract
Abstract The aim of this study was to evaluate quality of life (QoL) and its possible determinants in patients affected by Duchenne muscular dystrophy (DMD) in late stages of their disease, when non-invasive ventilation (NIV) is already established. Forty-eight DMD patients who were treated by NIV were enrolled. QoL was assessed by the Individualized Neuromuscular Quality of Life (INQoL) questionnaire. By this questionnaire, different aspects of QoL were assessed on a scale from 0 (best) to 100 (worst). In addition, motor and respiratory function tests were performed. Dysautonomia symptoms, sleep quality, sleepiness, anxiety, and depression were evaluated by validated questionnaires. The global INQoL score was 42.8 ± 19, reflecting a moderately altered QoL. The physical health domain was heavily impaired while the psychosocial domain was only mildly affected. Independence had the highest scores (81.1 ± 21.2), proving to be the most affected item. On multivariate analysis, maximal inspiratory pressure and Pittsburgh Sleep Quality Index, but not daily duration of NIV therapy, predicted global INQoL score. Respiratory impairment and sleep quality were independent predictors of poor QoL in DMD patients under NIV. Sleep quality in DMD is often disregarded, while it should be carefully addressed to ensure a better QoL.
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References46
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#1Vivienne Travlos (University of Notre Dame Australia)H-Index: 2
#2Jenny Downs (Curtin University)H-Index: 23
Last. Shane Patman (University of Notre Dame Australia)H-Index: 14
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Abstract The physical and social challenges associated with neuromuscular disorders may impact mental wellbeing in non-ambulant youth during the more vulnerable period of adolescence. This cross-sectional survey investigated non-ambulant youths’ mental wellbeing and relationships with physical health, participation and social factors. The conceptual model was the International Classification of Functioning, Disability and Health (ICF). Thirty-seven youth aged 13–22 years old (mean age 17.4 years...
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#1Oscar H. MayerH-Index: 16
#2Andrea AlivertiH-Index: 1
Last. Thomas MeierH-Index: 40
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#1Brittany L. Adler (JHUSOM: Johns Hopkins University School of Medicine)H-Index: 2
#2James W. Russell (UMB: University of Maryland, Baltimore)H-Index: 26
Last. Zsuzsanna H. McMahanH-Index: 7
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Objective. Autonomic dysfunction is a known complication of systemic sclerosis (SSc) that can affect vascular tone, gastrointestinal (GI) motility, heart rate, and blood pressure control. We sought to quantify autonomic symptom burden in SSc, and to define the characteristics of patients with SSc and autonomic dysfunction. Methods. Patients with SSc were consecutively recruited during routine clinical visits at the Johns Hopkins Scleroderma Center and asked to complete the Composite Autonomic Sy...
4 CitationsSource
#1David J. Birnkrant (Case Western Reserve University)H-Index: 17
#2Katharine Bushby (Newcastle University)H-Index: 24
Last. Leanne M. Ward (Children's Hospital of Eastern Ontario)H-Index: 38
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Summary A coordinated, multidisciplinary approach to care is essential for optimum management of the primary manifestations and secondary complications of Duchenne muscular dystrophy (DMD). Contemporary care has been shaped by the availability of more sensitive diagnostic techniques and the earlier use of therapeutic interventions, which have the potential to improve patients' duration and quality of life. In part 2 of this update of the DMD care considerations, we present the latest recommendat...
99 CitationsSource
#1David J. Birnkrant (Case Western Reserve University)H-Index: 17
#2Katharine Bushby (Newcastle University)H-Index: 24
Last. David R. Weber (URMC: University of Rochester Medical Center)H-Index: 13
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Summary Since the publication of the Duchenne muscular dystrophy (DMD) care considerations in 2010, multidisciplinary care of this severe, progressive neuromuscular disease has evolved. In conjunction with improved patient survival, a shift to more anticipatory diagnostic and therapeutic strategies has occurred, with a renewed focus on patient quality of life. In 2014, a steering committee of experts from a wide range of disciplines was established to update the 2010 DMD care considerations, wit...
102 CitationsSource
#1Grazia Crescimanno (National Research Council)H-Index: 7
#2Oreste Marrone (National Research Council)H-Index: 22
2 CitationsSource
#1Vivienne Travlos (University of Notre Dame Australia)H-Index: 2
#2Shane Patman (University of Notre Dame Australia)H-Index: 14
Last. Jenny Downs (Curtin University)H-Index: 23
view all 5 authors...
Abstract Objective To investigate quality of life (QOL) and psychosocial well-being in youth with neuromuscular disorders (NMDs) who are wheelchair users. Data Sources MEDLINE, Embase, CINAHL, and PsycINFO (January 2004–April 2016) and reference lists of retrieved full-text articles. Study Selection Peer-reviewed studies were included when data describing self-reported QOL and psychosocial well-being could be separately understood for those using wheelchairs and 12 to 22 years of age. There were...
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#1Ilene L. Hollin (Johns Hopkins University)H-Index: 10
#2Holly L. Peay (RTP: Research Triangle Park)H-Index: 17
Last. John F. P. Bridges (Johns Hopkins University)H-Index: 31
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Objective This study quantifies caregiver and patient preferences for a therapeutic agent with demonstrated pulmonary benefits for Duchenne muscular dystrophy (DMD). Caregivers and patient differences are also explored. Methods A best-worst scaling survey (BWS) was administered to caregivers and patients. Across 9 profiles, respondents selected the best and worst attributes. Utility scores were estimated using mixed logistic regression. Results Respondents indicated greatest preference for thera...
14 CitationsSource
#1Benedikt Schoser (LMU: Ludwig Maximilian University of Munich)H-Index: 53
#2Edward Fong (Stanford University)H-Index: 1
Last. Peter Young (WWU: University of Münster)H-Index: 39
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Respiratory muscle strength is a proven predictor of long-term outcome of neuromuscular disease (NMD), including amyotrophic lateral sclerosis, Duchenne muscular dystrophy, and spinal muscular atrophy. Maximal inspiratory pressure (MIP), a sensitive measure of respiratory muscle strength, one of several useful tests of respiratory muscle strength, is gaining interest as a therapeutic clinical trial endpoint for NMD. In this comprehensive review we investigate the use of MIP as a measure of respi...
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#1Toshio SaitoH-Index: 14
#2Mitsuru KawaiH-Index: 1
Last. Saburo SakodaH-Index: 47
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Abstract The national muscular dystrophy wards database of Japan lists 118 long-term Duchenne muscular dystrophy (DMD) patients who were at least 40 years old as of October 1, 2013. To elucidate the clinical features of DMD patients aged 40 years and older, we obtained gene analysis and muscle biopsy findings, as well as medical condition information. Ninety-four of the registered patients consented to participate, of whom 55 meeting genetic or biochemical criteria confirming DMD were analyzed. ...
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Cited By1
Newest
#1Grazia Crescimanno (National Research Council)H-Index: 7
#2Francesca GrecoH-Index: 3
Last. Oreste Marrone (National Research Council)H-Index: 22
view all 6 authors...
Abstract Objective In stable neuromuscular patients under long-term non-invasive ventilation (NIV), subjective sleep quality may be predicted by chronic hypoventilation, as assessed by base excess (BE), and %N3 sleep stage duration. In this study, we explored how other variables, closely associated with self-reported health complaints, contributed to subjective sleep quality in adult patients with Duchenne muscular dystrophy (DMD). Methods This is a secondary analysis of a study about quality of...
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