Severe gastrointestinal problems in Duchenne muscular dystrophy: A case series

•Gastrointestinal problems can be very serious in more advanced disease stages of Duchenne muscular dystrophy, even life threatening. Until now, a variety of treatment options are considered, however an overview is lacking.•We describe the longitudinal course of 6 patients with Duchenne muscular dys...

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Veröffentlicht in:Neuromuscular disorders : NMD 2024-07, Vol.40, p.31-37
Hauptverfasser: Blokhuis, AM, Tytgat, KMAJ, Groothuis, JT, Houwen-van Opstal, SLS
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Sprache:eng
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Zusammenfassung:•Gastrointestinal problems can be very serious in more advanced disease stages of Duchenne muscular dystrophy, even life threatening. Until now, a variety of treatment options are considered, however an overview is lacking.•We describe the longitudinal course of 6 patients with Duchenne muscular dystrophy and gastrointestinal problems requiring admission. Besides we have looked at the time frame of non-invasive ventilation (NIV), and radiographic abnormalities in relation to the admission.•We provide an overview of the different therapeutic options for gastrointestinal problems in Duchenne muscular dystrophy. Due to improved supportive care, survival of patients with Duchenne muscular dystrophy (DMD) has increased significantly. Consequently, new challenges emerge in adult patients with DMD. In clinical practice we increasingly see patients with serious, even life-threatening, gastrointestinal (GI) problems in advanced disease stages. Little is known about the longitudinal course of GI problems and the appropriate management. We present a case-series of six adult patients with DMD with (recurrent) GI problems that required hospital admission. The most prevalent reported serious GI symptoms were gastrointestinal pseudo-obstruction, (sub)ileus and gastric dilatation. Besides, an overview is presented of the therapeutic options for GI problems in DMD. The current study provides insight in possible treatment options, however, there is a clear need for more research and an integral guideline on treatment of GI problems in adult patients with DMD in order to reduce associated morbidity and mortality.
ISSN:0960-8966
1873-2364
1873-2364
DOI:10.1016/j.nmd.2024.05.006