Characterization of pulmonary function in 10–18 year old patients with Duchenne muscular dystrophy

the DELOS Study Group

Research output: Contribution to journalArticle

15 Citations (Scopus)

Abstract

Pulmonary function loss in patients with Duchenne muscular dystrophy (DMD) is progressive and leads to pulmonary insufficiency. The purpose of this study in 10–18 year old patients with DMD is the assessment of the inter-correlation between pulmonary function tests (PFTs), their reliability and the association with the general disease stage measured by the Brooke score. Dynamic PFTs (peak expiratory flow [PEF], forced vital capacity [FVC], forced expiratory volume in one second [FEV1]) and maximum static airway pressures (MIP, MEP) were prospectively collected from 64 DMD patients enrolled in the DELOS trial (ClinicalTrials.gov, number NCT01027884). Baseline PEF percent predicted (PEF%p) was <80% and patients had stopped taking glucocorticoids at least 12 months prior to study start. At baseline PEF%p, FVC%p and FEV1%p correlated well with each other (Spearman's rho: PEF%p–FVC%p: 0.54; PEF%p–FEV1%p: 0.72; FVC%p–FEV1%p: 0.91). MIP%p and MEP%p correlated well with one another (MIP%p–MEP%p: 0.71) but less well with PEF%p (MIP%p–PEF%p: 0.40; MEP%p–PEF%p: 0.41) and slightly better with FVC%p (MIP%p–FVC%p: 0.59; MEP%p–FVC%p: 0.74). The within-subject coefficients of variation (CV) for successive measures were 6.97% for PEF%p, 6.69% for FVC%p and 11.11% for FEV1%p, indicating that these parameters could be more reliably assessed compared to maximum static airway pressures (CV for MIP%p: 18.00%; MEP%p: 15.73%). Yearly rates of PFT decline (placebo group) were larger in dynamic parameters (PEF%p: −8.9% [SD 2.0]; FVC%p: −8.7% [SD 1.1]; FEV1%p: −10.2% [SD 2.0]) than static airway pressures (MIP%p: −4.5 [SD 1.3]; MEP%p: −2.8 [SD 1.1]). A considerable drop in dynamic pulmonary function parameters was associated with loss of upper limb function (transition from Brooke score category 4 to category 5). In conclusion, these findings expand the understanding of the reliability, correlation and evolution of different pulmonary function measures in DMD patients who are in the pulmonary function decline phase.

Original languageEnglish (US)
Pages (from-to)307-314
Number of pages8
JournalNeuromuscular Disorders
Volume27
Issue number4
DOIs
StatePublished - Apr 1 2017

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Duchenne Muscular Dystrophy
Vital Capacity
Lung
Respiratory Function Tests
Pressure
Forced Expiratory Volume
Upper Extremity
Glucocorticoids
Placebos

Keywords

  • Duchenne muscular dystrophy
  • forced vital capacity
  • peak expiratory flow
  • pulmonary function

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Neurology
  • Clinical Neurology
  • Genetics(clinical)

Cite this

Characterization of pulmonary function in 10–18 year old patients with Duchenne muscular dystrophy. / the DELOS Study Group.

In: Neuromuscular Disorders, Vol. 27, No. 4, 01.04.2017, p. 307-314.

Research output: Contribution to journalArticle

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title = "Characterization of pulmonary function in 10–18 year old patients with Duchenne muscular dystrophy",
abstract = "Pulmonary function loss in patients with Duchenne muscular dystrophy (DMD) is progressive and leads to pulmonary insufficiency. The purpose of this study in 10–18 year old patients with DMD is the assessment of the inter-correlation between pulmonary function tests (PFTs), their reliability and the association with the general disease stage measured by the Brooke score. Dynamic PFTs (peak expiratory flow [PEF], forced vital capacity [FVC], forced expiratory volume in one second [FEV1]) and maximum static airway pressures (MIP, MEP) were prospectively collected from 64 DMD patients enrolled in the DELOS trial (ClinicalTrials.gov, number NCT01027884). Baseline PEF percent predicted (PEF{\%}p) was <80{\%} and patients had stopped taking glucocorticoids at least 12 months prior to study start. At baseline PEF{\%}p, FVC{\%}p and FEV1{\%}p correlated well with each other (Spearman's rho: PEF{\%}p–FVC{\%}p: 0.54; PEF{\%}p–FEV1{\%}p: 0.72; FVC{\%}p–FEV1{\%}p: 0.91). MIP{\%}p and MEP{\%}p correlated well with one another (MIP{\%}p–MEP{\%}p: 0.71) but less well with PEF{\%}p (MIP{\%}p–PEF{\%}p: 0.40; MEP{\%}p–PEF{\%}p: 0.41) and slightly better with FVC{\%}p (MIP{\%}p–FVC{\%}p: 0.59; MEP{\%}p–FVC{\%}p: 0.74). The within-subject coefficients of variation (CV) for successive measures were 6.97{\%} for PEF{\%}p, 6.69{\%} for FVC{\%}p and 11.11{\%} for FEV1{\%}p, indicating that these parameters could be more reliably assessed compared to maximum static airway pressures (CV for MIP{\%}p: 18.00{\%}; MEP{\%}p: 15.73{\%}). Yearly rates of PFT decline (placebo group) were larger in dynamic parameters (PEF{\%}p: −8.9{\%} [SD 2.0]; FVC{\%}p: −8.7{\%} [SD 1.1]; FEV1{\%}p: −10.2{\%} [SD 2.0]) than static airway pressures (MIP{\%}p: −4.5 [SD 1.3]; MEP{\%}p: −2.8 [SD 1.1]). A considerable drop in dynamic pulmonary function parameters was associated with loss of upper limb function (transition from Brooke score category 4 to category 5). In conclusion, these findings expand the understanding of the reliability, correlation and evolution of different pulmonary function measures in DMD patients who are in the pulmonary function decline phase.",
keywords = "Duchenne muscular dystrophy, forced vital capacity, peak expiratory flow, pulmonary function",
author = "{the DELOS Study Group} and Thomas Meier and Christian Rummey and Mika Leinonen and Paolo Spagnolo and Mayer, {Oscar H.} and Buyse, {Gunnar M.} and G. Bernert and F. Knipp and Buyse, {G. M.} and N. Goemans and {Van den Hauwe}, M. and T. Voit and V. Doppler and T. Gidaro and Cuisset, {J. M.} and S. Coopman and U. Schara and S. Lutz and J. Kirschner and S. Borell and M. Will and D'Angelo, {M. G.} and E. Brighina and S. Gandossini and K. Gorni and E. Falcier and L. Politano and P. D'Ambrosio and A. Taglia and Verschuuren, {J. J.G.M.} and Straathof, {C. S.M.} and {V{\'i}lchez Padilla}, {J. J.} and {Muelas G{\'o}mez}, N. and T. Sejersen and M. Hovm{\"o}ller and Jeannet, {P. Y.} and C. Bloetzer and S. Iannaccone and D. Castro and G. Tennekoon and R. Finkel and C. B{\"o}nnemann and C. McDonald and E. Henricson and N. Joyce and S. Apkon and Richardson, {R. C.}",
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T1 - Characterization of pulmonary function in 10–18 year old patients with Duchenne muscular dystrophy

AU - the DELOS Study Group

AU - Meier, Thomas

AU - Rummey, Christian

AU - Leinonen, Mika

AU - Spagnolo, Paolo

AU - Mayer, Oscar H.

AU - Buyse, Gunnar M.

AU - Bernert, G.

AU - Knipp, F.

AU - Buyse, G. M.

AU - Goemans, N.

AU - Van den Hauwe, M.

AU - Voit, T.

AU - Doppler, V.

AU - Gidaro, T.

AU - Cuisset, J. M.

AU - Coopman, S.

AU - Schara, U.

AU - Lutz, S.

AU - Kirschner, J.

AU - Borell, S.

AU - Will, M.

AU - D'Angelo, M. G.

AU - Brighina, E.

AU - Gandossini, S.

AU - Gorni, K.

AU - Falcier, E.

AU - Politano, L.

AU - D'Ambrosio, P.

AU - Taglia, A.

AU - Verschuuren, J. J.G.M.

AU - Straathof, C. S.M.

AU - Vílchez Padilla, J. J.

AU - Muelas Gómez, N.

AU - Sejersen, T.

AU - Hovmöller, M.

AU - Jeannet, P. Y.

AU - Bloetzer, C.

AU - Iannaccone, S.

AU - Castro, D.

AU - Tennekoon, G.

AU - Finkel, R.

AU - Bönnemann, C.

AU - McDonald, C.

AU - Henricson, E.

AU - Joyce, N.

AU - Apkon, S.

AU - Richardson, R. C.

PY - 2017/4/1

Y1 - 2017/4/1

N2 - Pulmonary function loss in patients with Duchenne muscular dystrophy (DMD) is progressive and leads to pulmonary insufficiency. The purpose of this study in 10–18 year old patients with DMD is the assessment of the inter-correlation between pulmonary function tests (PFTs), their reliability and the association with the general disease stage measured by the Brooke score. Dynamic PFTs (peak expiratory flow [PEF], forced vital capacity [FVC], forced expiratory volume in one second [FEV1]) and maximum static airway pressures (MIP, MEP) were prospectively collected from 64 DMD patients enrolled in the DELOS trial (ClinicalTrials.gov, number NCT01027884). Baseline PEF percent predicted (PEF%p) was <80% and patients had stopped taking glucocorticoids at least 12 months prior to study start. At baseline PEF%p, FVC%p and FEV1%p correlated well with each other (Spearman's rho: PEF%p–FVC%p: 0.54; PEF%p–FEV1%p: 0.72; FVC%p–FEV1%p: 0.91). MIP%p and MEP%p correlated well with one another (MIP%p–MEP%p: 0.71) but less well with PEF%p (MIP%p–PEF%p: 0.40; MEP%p–PEF%p: 0.41) and slightly better with FVC%p (MIP%p–FVC%p: 0.59; MEP%p–FVC%p: 0.74). The within-subject coefficients of variation (CV) for successive measures were 6.97% for PEF%p, 6.69% for FVC%p and 11.11% for FEV1%p, indicating that these parameters could be more reliably assessed compared to maximum static airway pressures (CV for MIP%p: 18.00%; MEP%p: 15.73%). Yearly rates of PFT decline (placebo group) were larger in dynamic parameters (PEF%p: −8.9% [SD 2.0]; FVC%p: −8.7% [SD 1.1]; FEV1%p: −10.2% [SD 2.0]) than static airway pressures (MIP%p: −4.5 [SD 1.3]; MEP%p: −2.8 [SD 1.1]). A considerable drop in dynamic pulmonary function parameters was associated with loss of upper limb function (transition from Brooke score category 4 to category 5). In conclusion, these findings expand the understanding of the reliability, correlation and evolution of different pulmonary function measures in DMD patients who are in the pulmonary function decline phase.

AB - Pulmonary function loss in patients with Duchenne muscular dystrophy (DMD) is progressive and leads to pulmonary insufficiency. The purpose of this study in 10–18 year old patients with DMD is the assessment of the inter-correlation between pulmonary function tests (PFTs), their reliability and the association with the general disease stage measured by the Brooke score. Dynamic PFTs (peak expiratory flow [PEF], forced vital capacity [FVC], forced expiratory volume in one second [FEV1]) and maximum static airway pressures (MIP, MEP) were prospectively collected from 64 DMD patients enrolled in the DELOS trial (ClinicalTrials.gov, number NCT01027884). Baseline PEF percent predicted (PEF%p) was <80% and patients had stopped taking glucocorticoids at least 12 months prior to study start. At baseline PEF%p, FVC%p and FEV1%p correlated well with each other (Spearman's rho: PEF%p–FVC%p: 0.54; PEF%p–FEV1%p: 0.72; FVC%p–FEV1%p: 0.91). MIP%p and MEP%p correlated well with one another (MIP%p–MEP%p: 0.71) but less well with PEF%p (MIP%p–PEF%p: 0.40; MEP%p–PEF%p: 0.41) and slightly better with FVC%p (MIP%p–FVC%p: 0.59; MEP%p–FVC%p: 0.74). The within-subject coefficients of variation (CV) for successive measures were 6.97% for PEF%p, 6.69% for FVC%p and 11.11% for FEV1%p, indicating that these parameters could be more reliably assessed compared to maximum static airway pressures (CV for MIP%p: 18.00%; MEP%p: 15.73%). Yearly rates of PFT decline (placebo group) were larger in dynamic parameters (PEF%p: −8.9% [SD 2.0]; FVC%p: −8.7% [SD 1.1]; FEV1%p: −10.2% [SD 2.0]) than static airway pressures (MIP%p: −4.5 [SD 1.3]; MEP%p: −2.8 [SD 1.1]). A considerable drop in dynamic pulmonary function parameters was associated with loss of upper limb function (transition from Brooke score category 4 to category 5). In conclusion, these findings expand the understanding of the reliability, correlation and evolution of different pulmonary function measures in DMD patients who are in the pulmonary function decline phase.

KW - Duchenne muscular dystrophy

KW - forced vital capacity

KW - peak expiratory flow

KW - pulmonary function

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U2 - 10.1016/j.nmd.2016.12.014

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VL - 27

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JO - Neuromuscular Disorders

JF - Neuromuscular Disorders

SN - 0960-8966

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