P 882. Four-Level Release (Rideau) and Glucocorticoid Therapy in Patients with DMD—Additive Positive Effect on Ambulation

Corinna Stoltenburg; Claudia Weiß; Dilan Bayram; Julia Funk; Susanne Lebek

doi:10.1055/s-0038-1676016

Neuropediatrics, Table of Contents

Neuropediatrics 2018; 49(S 02): S1-S69
DOI: 10.1055/s-0038-1676016

Posters

Therapy Strategies and Free Topics

Georg Thieme Verlag KG Stuttgart · New York

P 882. Four-Level Release (Rideau) and Glucocorticoid Therapy in Patients with DMD—Additive Positive Effect on Ambulation

Authors

Corinna Stoltenburg

¹Charité – Universitätsmedizin Berlin, Neuropädiatrie, Berlin, Germany
Claudia Weiß

¹Charité – Universitätsmedizin Berlin, Neuropädiatrie, Berlin, Germany
Dilan Bayram

¹Charité – Universitätsmedizin Berlin, Neuropädiatrie, Berlin, Germany
Julia Funk

²Charité – Universitätsmedizin Berlin, Orthopädie, Berlin, Germany
Susanne Lebek

²Charité – Universitätsmedizin Berlin, Orthopädie, Berlin, Germany

Abstract

Full Text

Background: Duchenne muscular dystrophy (DMD) is among the most frequent genetic muscular diseases in childhood. Despite promising new therapeutic approaches, there is still no cure for most patients. Glucocorticoid therapy is an inherent part of international therapeutical guidelines due to the positive effect on maintenance of ambulation. Nevertheless, in our daily routine, we still experience parents’ reserve due to fear of side effects.

Rideau first described a positive effect of early contracture-releasing surgery of lower limbs (four-level release) on maintenance of ambulation in 1983. However, it does still not constitute an element of current therapeutic guidelines.

All patients at the SPZ of the Charité with DMD and an appropriate indication were offered four-level release according to Rideau as well as medication with glucocorticoids.

Aim and Scientific Issue: The aim of this study was the critical evaluation of our therapeutic approach toward patients with DMD. Notably, we examined the effect of glucocorticoid therapy and four-level release on the maintenance of ambulation.

Methods: We examined our patient cohort with DMD from 2013 to 2017 by means of a comprehensive retrospective data analysis.

Results: At the SPZ of the Charité, 91 patients with DMD were treated in the period mentioned earlier. Of these, 46 (50.5%) were treated with glucocorticoids (prednisone or deflazacort) at least temporarily, and 31 (34.1%) received surgical four-level release.

The positive effect of a medication with glucocorticoids could be reproduced in our patient cohort: Median age of loss of ambulation was 12 years in treated patients versus 9 years in untreated patients.

Additionally, we could confirm a delay of 2 years by four-level release in our cohort: Median age of loss of ambulation in operated patients was 12 years and 10 years in not operated patients.

Furthermore, we found an additive effect of both therapies: Patients without four-level release and without glucocorticoid therapy became wheelchair bound at the median age of 9 years, while patients with four-level release and glucocorticoid therapy lost ambulation at 14 years on average.

Conclusion: Four-level release surgery and glucocorticoid therapies constitute positive additive effects. Both therapies should be offered to patients fulfilling the indications. Adding the four-level release to international therapeutic guidelines for DMD should be discussed.