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Table 1 PECOS criteria to outline the scope of the literature review

From: The clinical course of Duchenne muscular dystrophy in the corticosteroid treatment era: a systematic literature review

Population

Including males with DMD in North America

Exposures/comparators

Subgroup

Corticosteroid treatment

By age

By disease status at baseline

Outcomes

Clinical/functional measures measured over a minimum of 1 yeara

Pulmonary function tests: Forced vital capacity, peak expiratory flow

Assessment of cardiac function: Ejection fraction, left ventricular end diastolic dimension, shortening fraction

Key clinical outcomes

LOA

Scoliosis

Need for ventilatory support

Pulmonary dysfunction

Cardiac dysfunction/cardiomyopathy

Mortality

Study design

Prospective or retrospective studies

Case series

  1. DMD Duchenne muscular dystrophy, LOA loss of ambulation
  2. aOnly commonly reported functional assessments described in included studies are listed. Other functional assessments were searched (e.g. the 6-min walk test, North Star Ambulatory Assessment, Maximum inspiratory/expiratory pressure, Forced expiratory volume) but results to include in this manuscript were not identified