TY - JOUR
T1 - Psychopharmaceutical treatment for neurobehavioral problems in Duchenne muscular dystrophy
T2 - a descriptive study using real-world data
AU - Weerkamp, Pien M.M.
AU - Geuens, Sam
AU - Collin, Philippe
AU - Goemans, Nathalie
AU - Vermeulen, R. Jeroen
AU - De Waele, Liesbeth
AU - Hendriksen, Jos G.M.
AU - Klinkenberg, Sylvia
N1 - Funding Information:
This work is generated within the European Reference Network for Neuromuscular Diseases. We genuinely thank Duchenne Parent Project Netherlands for their support to J.H. and S.G. and the Kan-Go! Fund for their financial support to S.G. during this research project. Also, we gratefully acknowledge support to P.W. and J.H. from the European Union Horizon 2020 Framework Programme research and innovation program ‘Brain Involvement iN Dystrophinopathies’ (grant agreement, 847826).
Publisher Copyright:
© 2023
PY - 2023/7/1
Y1 - 2023/7/1
N2 - Patients with Duchenne muscular dystrophy (DMD) are at risk to develop neurobehavioral problems. Evidence on how to treat these difficulties is scarce. This descriptive study reports the clinical experience with psychopharmaceutical treatment in 52 patients with DMD. Electronic patient files were searched for patients with DMD that had been treated with psychopharmaceuticals between 2008 and 2022. Information about neurobehavioral symptoms, type of medication, side effects, and behavioral changes were collected. Two independent clinicians used the clinical global impression scale (CGI) to assess severity of the neurobehavioral problems before and the change in symptoms after treatment. Descriptive statistics were used. Our results include 52 males with DMD (mean age 11 years) treated with psychopharmaceuticals of which 55.8% had four or more comorbid neurobehavioral symptoms. The clinical condition was much improved on the GCI in 54.2% treated with methylphenidate, in 38.9% of the patients treated with fluoxetine, and in 22.2% treated with risperidone. Minimal effects and side effects were also reported. In conclusion, patients with DMD may experience severe neurobehavioral symptoms interfering with learning and/or development. Treatment with psychopharmaceuticals can improve these neurobehavioral symptoms, but further research is needed to gain better insights in psychopharmaceutical treatment in patients with DMD.
AB - Patients with Duchenne muscular dystrophy (DMD) are at risk to develop neurobehavioral problems. Evidence on how to treat these difficulties is scarce. This descriptive study reports the clinical experience with psychopharmaceutical treatment in 52 patients with DMD. Electronic patient files were searched for patients with DMD that had been treated with psychopharmaceuticals between 2008 and 2022. Information about neurobehavioral symptoms, type of medication, side effects, and behavioral changes were collected. Two independent clinicians used the clinical global impression scale (CGI) to assess severity of the neurobehavioral problems before and the change in symptoms after treatment. Descriptive statistics were used. Our results include 52 males with DMD (mean age 11 years) treated with psychopharmaceuticals of which 55.8% had four or more comorbid neurobehavioral symptoms. The clinical condition was much improved on the GCI in 54.2% treated with methylphenidate, in 38.9% of the patients treated with fluoxetine, and in 22.2% treated with risperidone. Minimal effects and side effects were also reported. In conclusion, patients with DMD may experience severe neurobehavioral symptoms interfering with learning and/or development. Treatment with psychopharmaceuticals can improve these neurobehavioral symptoms, but further research is needed to gain better insights in psychopharmaceutical treatment in patients with DMD.
U2 - 10.1016/j.nmd.2023.05.011
DO - 10.1016/j.nmd.2023.05.011
M3 - Article
C2 - 37453172
SN - 0960-8966
VL - 33
SP - 619
EP - 626
JO - Neuromuscular Disorders
JF - Neuromuscular Disorders
IS - 7
ER -