Benefits of glucocorticoids in non-ambulant boys/men with Duchenne muscular dystrophy: A multicentric longitudinal study using the Performance of Upper Limb test

Pane, Marika; Fanelli, Lavinia; Mazzone, Elena; Olivieri, Giorgia; D’Amico, Adele; Messina, Sonia; Scutifero, Marianna; Battini, Roberta; Petillo, Roberta; Frosini, Silvia; Sivo, Serena; Vita, Gianluca; Bruno, Claudio; Mongini, Tiziana; Pegoraro, Elena; Sanctis, Roberto De; Gardani, Alice; Berardinelli, Angela; Lanzillotta, Valentina; Carlesi, Adelina; Viggiano, Emanuela; Cavallaro, Fabio; Sframeli, Maria; Bello, Luca; Barp, Andrea; Bianco, F.; Bonfiglio, S; Rolle, Enrica; Palermo, Concetta; D'Angelo, Maria Grazia; Pini, Antonella; Iotti, Elena; Gorni, Ksenija; Baranello, Giovanni; Bertini, Enrico; Politano, Luisa; Sormani, Maria Pia; Mercuri, Eugenio

doi:10.1016/j.nmd.2015.07.009

Cited by 43 publications

(43 citation statements)

References 14 publications

(21 reference statements)

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“…[9,20,21] Furthermore, in response to the demand for monitoring motor function in the older subjects, the Performance of the Upper Limb (PUL), a functional scale dedicated to evaluating upper extremities in DMD has been developed, [22] (Mayhew et al under review) necessitating further evaluation in other cohorts of patients. [23][24][25][26] The aim of this prospective, longitudinal, multicentre natural history study was to provide novel information on outcome measures that can support drug development in DMD subjects…”

Section: Accepted Manuscriptmentioning

confidence: 99%

Respiratory and upper limb function as outcome measures in ambulant and non-ambulant subjects with Duchenne muscular dystrophy: A prospective multicentre study

Ricotti

Selby

Ridout

et al. 2019

Neuromuscular Disorders

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The field of translational research in Duchenne muscular dystrophy (DMD) has been transformed in the last decade by a number of therapeutic targets, mostly studied in ambulant patients. A paucity of studies focus on measures that capture the non-ambulant stage of the disease, and the transition between the ambulant and non-ambulant phase. In this prospective natural history study, we report the results of a comprehensive assessment of respiratory, upper limb function and upper limb muscle strength in a group of 89 DMD boys followed in 3 European countries, 81 receiving corticosteroids, spanning a wide age range (5-18 years) and functional abilities, from ambulant (n=60) to non-ambulant (n=29).

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Section: Accepted Manuscriptmentioning

confidence: 99%

Respiratory and upper limb function as outcome measures in ambulant and non-ambulant subjects with Duchenne muscular dystrophy: A prospective multicentre study

Ricotti

Selby

Ridout

et al. 2019

Neuromuscular Disorders

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“…There is general agreement that patients with DMD benefit from GC treatment [31,32], which has become the mainstay of neuromuscular management in DMD [33,34]. Specifically, the effect of GC on muscle strength has been shown to prolong ambulation by several years and more recently, continued treatment with GC after the patient becomes non-ambulant has also shown to reduce the risk of progressive scoliosis, stabilize pulmonary function [17,[35][36][37] and delay the loss of upper limb function [38]. An interesting finding of this work was the observation that previous GC use had no long-lasting effect on the decline in pulmonary function.…”

Section: Pulmonary Function Status and Evolution Is Not Influenced Bymentioning

confidence: 99%

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

Meier

Rummey

Leinonen

et al. 2017

Neuromuscular Disorders

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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.

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“…Three studies recommend the continuation of glucocorticoid therapy for the wheelchair-bound stage of the disease (Level of evidence: 3B, Class of Recommendation: B) 40,50,51 . For these patients, the aim of glucocorticoid therapy is to preserve heart, lung and upper limb function as much as possible 40,50,51 . The above recommendations are summarized in the Table 3.…”

Section: Should Corticosteroids Be Prescribed To Nonambulant Dmd Patimentioning

confidence: 99%

Brazilian consensus on Duchenne muscular dystrophy. Part 1: diagnosis, steroid therapy and perspectives

Araujo

Carvalho

Cavalcanti³

et al. 2017

Arq. Neuro-Psiquiatr.

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Significant advances in the understanding and management of Duchenne muscular dystrophy (DMD) took place since international guidelines were published in 2010. Our objective was to provide an evidence-based national consensus statement for multidisciplinary care of DMD in Brazil. A combination of the Delphi technique with a systematic review of studies from 2010 to 2016 was employed to classify evidence levels and grade of recommendations. Our recommendations were divided in two parts. We present Part 1 here, where we describe the guideline methodology and overall disease concepts, and also provide recommendations on diagnosis, steroid therapy and new drug treatment perspectives for DMD. The main recommendations: 1) genetic testing in diagnostic suspicious cases should be the first line for diagnostic confirmation; 2) patients diagnosed with DMD should have steroids prescribed; 3) lack of published results for phase 3 clinical trials hinders, for now, the recommendation to use exon skipping or read-through agents.Keywords: muscular dystrophy, Duchenne; practice guideline; consensus; diagnosis; genetic testing; drug therapy; glucocorticoids; utrophin. RESUMOAvanços na compreensão e no manejo da distrofia muscular de Duchenne (DMD) ocorreram desde a publicação de diretrizes internacionais em 2010. Nosso objetivo foi elaborar um consenso nacional baseado em evidências de cuidado multidisciplinar dos pacientes com DMD no Brasil. Utilizamos a técnica de Delphi combinada com revisão sistemática da literatura de 2010 a 2016 classificando níveis de evidência e graus de recomendação. Nossas recomendações foram divididas em duas partes. Apresentamos aqui a parte 1, descrevendo a metodologia utilizada e conceitos gerais da doença, e fornecemos recomendações sobre diagnóstico, tratamento com corticosteroides e novas perspectivas de tratamentos medicamentosos. As principais recomendações: 1) testes genéticos deveriam ser a primeira linha para confirmação de casos suspeitos; 2) pacientes com diagnóstico de DMD devem receber corticosteroides; 3) por enquanto, a falta de publicações de resultados dos ensaios clínicos de fase 3, dificulta recomendações de uso medicamentos que "saltam exons" ou "passam" por código de parada prematura.

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Benefits of glucocorticoids in non-ambulant boys/men with Duchenne muscular dystrophy: A multicentric longitudinal study using the Performance of Upper Limb test

Cited by 43 publications

References 14 publications

Respiratory and upper limb function as outcome measures in ambulant and non-ambulant subjects with Duchenne muscular dystrophy: A prospective multicentre study

Respiratory and upper limb function as outcome measures in ambulant and non-ambulant subjects with Duchenne muscular dystrophy: A prospective multicentre study

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

Brazilian consensus on Duchenne muscular dystrophy. Part 1: diagnosis, steroid therapy and perspectives

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