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Treatment of the heart in Duchenne muscular dystrophy
Author(s) -
Baxter Peter
Publication year - 2006
Publication title -
developmental medicine and child neurology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.658
H-Index - 143
eISSN - 1469-8749
pISSN - 0012-1622
DOI - 10.1017/s0012162206000351
Subject(s) - muscular dystrophy , medicine , cardiomyopathy , dilated cardiomyopathy , duchenne muscular dystrophy , pediatrics , cardiology , heart failure
and prevention. Guidelines for screening and treatment have been proposed, and promoted by at least one patient group,6 without an evidence base. Screening will demonstrate abnormalities in a high percentage of patients, which may create anxiety and has resource implications. Different studies use different tests of cardiac function, and our reviewers recommend another, but it is unclear if these are all equivalent in clinical terms. Classically, screening is only justified if there is an effective treatment, but we do not know if ACE inhibitors are or are not effective. In terms of prevention, McGuigan comments that a more appropriate statistical analysis of the randomized trial phase of ACE inhibitors might allow a more reliable conclusion. If ACE inhibitors or steroids do help, the guidelines need rewriting. Clearly there are several important issues that need proper trials for answers. In the meantime, patients and their families need sufficient information to decide whether or not to accept screening. It reinforces the need for guidelines to specify the level of evidence on which they are based in a prominent position adjacent to their recommendations, as for example in the SIGN guidelines. In addition, SIGN emphasizes the need for consultation and consensus before guidelines are agreed, to avoid dominant groups or individuals having a disproportionate influence.7 Is it now time that just as the raw evidence is graded according to formal criteria, guidelines should be too?