Clinical implications of maximal respiratory pressure determinations for individuals with duchenne muscular dystrophy

Hahn, Andreas; Bach, John R.; Delaubier, Anne; Renardel-Irani, Alice; Guillou, C.; Rideau, Y

doi:10.1016/s0003-9993(97)90001-0

Cited by 126 publications

(118 citation statements)

References 22 publications

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“…We confirmed that vital capacity related directly to respiratory muscle strength, a finding previously reported in polymyositis 18 and Duchenne muscular dystrophy 19 but not noted in other studies of neuromuscular diseases. 4 5 We have expanded the existing data by showing for the first time that P 0.1 and P 0.1 /PImax increased progressively with declining respiratory muscle strength, a finding likely to reflect the compensatory rise in respiratory drive and muscle effort in an attempt to maintain alveolar ventilation.…”

Section: Discussionsupporting

confidence: 92%

Patterns and predictors of sleep disordered breathing in primary myopathies

Ragette

2002

Thorax

236

138

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Background: Sleep disordered breathing (SDB) is common in neuromuscular diseases but its relationship to respiratory function is poorly defined. A study was undertaken to identify distinct patterns of SDB, to clarify the relationships between SDB and lung and respiratory muscle function, and to identify daytime predictors for SDB at its onset, for SDB with continuous hypercapnic hypoventilation, and for diurnal respiratory failure. Methods: Upright and supine inspiratory vital capacity (IVC, % predicted), maximal inspiratory muscle pressure (PImax), respiratory drive (P 0.1 ), respiratory muscle effort (P 0.1 /PImax), and arterial blood gas tensions were prospectively compared with polysomnography and capnometry (PtcCO 2 ) in 42 patients with primary myopathies. Results: IVC correlated with respiratory muscle function and gas exchange by day and night. SDB evolved in three distinct patterns from REM hypopnoeas, to REM hypopnoeas with REM hypoventilation, to REM/non-REM (continuous) hypoventilation, and preceded diurnal respiratory failure. SDB correlated with IVC and PImax which yielded highly predictive thresholds for SDB onset (IVC <60%, PImax <4.5 kPa), SDB with continuous hypoventilation (IVC <40%, PImax <4.0 kPa), and SDB with diurnal respiratory failure (IVC <25%, PImax <3.5 kPa). Conclusion: Progressive ventilatory restriction in neuromuscular diseases correlates with respiratory muscle weakness and results in progressive SDB which, by pattern and severity, can be predicted from daytime lung and respiratory muscle function.

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Section: Discussionsupporting

confidence: 92%

Patterns and predictors of sleep disordered breathing in primary myopathies

Ragette

2002

Thorax

236

138

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“…At this early stage the MRI studies indicate that the patients had low‐volume breathing compared to the controls. It has been proposed that expiratory muscles are affected early in DMD, which would lead to larger residual volumes 24, 25. However, we observed that the lung area at residual volume was reduced indicating a change in the chest wall compliance in the patients 26.…”

Section: Discussioncontrasting

confidence: 51%

Respiratory magnetic resonance imaging biomarkers in Duchenne muscular dystrophy

Mankodi

Kovacs

Norato

et al. 2017

Ann Clin Transl Neurol

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ObjectiveTo examine the diaphragm and chest wall dynamics with cine breathing magnetic resonance imaging (MRI) in ambulatory boys with Duchenne muscular dystrophy (DMD) without respiratory symptoms and controls.MethodsIn 11 DMD boys and 15 controls, cine MRI of maximal breathing was recorded for 10 sec. The lung segmentations were done by an automated pipeline based on a Holistically‐Nested Network model (HNN method). Lung areas, diaphragm, and chest wall motion were measured throughout the breathing cycle.ResultsThe HNN method reliably identified the contours of the lung and the diaphragm in every frame of each dataset (~180 frames) within seconds. The lung areas at maximal inspiration and expiration were reduced in DMD patients relative to controls (P = 0.02 and <0.01, respectively). The change in the lung area between inspiration and expiration correlated with percent predicted forced vital capacity (FVC) in patients (r s = 0.75, P = 0.03) and was not significantly different between groups. The diaphragm position, length, contractility, and motion were not significantly different between groups. Chest wall motion was reduced in patients compared to controls (P < 0.01).InterpretationCine breathing MRI allows independent and reliable assessment of the diaphragm and chest wall dynamics during the breathing cycle in DMD patients and controls. The MRI data indicate that ambulatory DMD patients breathe at lower lung volumes than controls when their FVC is in the normal range. The diaphragm moves normally, whereas chest wall motion is reduced in these boys with DMD.

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“…The importance of respiratory abnormalities is well understood in later stages of DMD, but early subclinical deterioration of respiratory muscles with a mean annual decline in %pMIP and %pMEP of approximately 4% and %pFVC of approximately 5% has only recently been recognized 3, 31. %pMIP and %pMEP assess progression of DMD regardless of ambulatory status and are the most sensitive indicators of decreasing respiratory muscle strength, with %pMEP being the first pulmonary parameter to decline in patients with DMD 39, 40. Thus, the observed relative stability of respiratory muscle function over >3 years observed in this study is important supporting evidence for the clinical efficacy of eteplirsen above and beyond endurance, ambulation, and muscle function that is measured by the 6MWT 19…”

Section: Discussionmentioning

confidence: 99%

Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

Mendell

Goemans²,

Lowes

et al. 2016

Annals of Neurology

453

392

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ObjectiveTo continue evaluation of the long‐term efficacy and safety of eteplirsen, a phosphorodiamidate morpholino oligomer designed to skip DMD exon 51 in patients with Duchenne muscular dystrophy (DMD). Three‐year progression of eteplirsen‐treated patients was compared to matched historical controls (HC).MethodsAmbulatory DMD patients who were ≥7 years old and amenable to exon 51 skipping were randomized to eteplirsen (30/50mg/kg) or placebo for 24 weeks. Thereafter, all received eteplirsen on an open‐label basis. The primary functional assessment in this study was the 6‐Minute Walk Test (6MWT). Respiratory muscle function was assessed by pulmonary function testing (PFT). Longitudinal natural history data were used for comparative analysis of 6MWT performance at baseline and months 12, 24, and 36. Patients were matched to the eteplirsen group based on age, corticosteroid use, and genotype.ResultsAt 36 months, eteplirsen‐treated patients (n = 12) demonstrated a statistically significant advantage of 151m (p < 0.01) on 6MWT and experienced a lower incidence of loss of ambulation in comparison to matched HC (n = 13) amenable to exon 51 skipping. PFT results remained relatively stable in eteplirsen‐treated patients. Eteplirsen was well tolerated. Analysis of HC confirmed the previously observed change in disease trajectory at age 7 years, and more severe progression was observed in patients with mutations amenable to exon skipping than in those not amenable. The subset of patients amenable to exon 51 skipping showed a more severe disease course than those amenable to any exon skipping.InterpretationOver 3 years of follow‐up, eteplirsen‐treated patients showed a slower rate of decline in ambulation assessed by 6MWT compared to untreated matched HC. Ann Neurol 2016;79:257–271

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Clinical implications of maximal respiratory pressure determinations for individuals with duchenne muscular dystrophy

Cited by 126 publications

References 22 publications

Patterns and predictors of sleep disordered breathing in primary myopathies

Patterns and predictors of sleep disordered breathing in primary myopathies

Respiratory magnetic resonance imaging biomarkers in Duchenne muscular dystrophy

Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

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