TY - JOUR
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.
N1 - Publisher Copyright:
© 2017 The Authors
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
DO - 10.1016/j.nmd.2016.12.014
M3 - Article
C2 - 28189481
AN - SCOPUS:85011850773
SN - 0960-8966
VL - 27
SP - 307
EP - 314
JO - Neuromuscular Disorders
JF - Neuromuscular Disorders
IS - 4
ER -