Intraventricular Baclofen for Treatment of Severe Dystonia Associated with Glutaryl-CoA Dehydrogenase Deficiency (GA1): Report of Two Cases
| Autor: | Kevin A. Strauss, Anne M. Ranney, Malgosia A. Kokoszka, Saadi Ghatan |
|---|---|
| Rok vydání: | 2016 |
| Předmět: |
congenital
hereditary and neonatal diseases and abnormalities Pain relief Glutaric aciduria type 1 Enteral administration 03 medical and health sciences chemistry.chemical_compound 0302 clinical medicine otorhinolaryngologic diseases medicine Case Series Glutaryl-CoA dehydrogenase deficiency Depression (differential diagnoses) Dystonia business.industry medicine.disease nervous system diseases Baclofen Neurology chemistry 030220 oncology & carcinogenesis Anesthesia Anxiety Neurology (clinical) medicine.symptom business 030217 neurology & neurosurgery |
| Zdroj: | Movement Disorders Clinical Practice. 3:296-299 |
| ISSN: | 2330-1619 |
| Popis: | Two individuals with intractable generalized dystonia secondary to glutaric aciduria type 1 (GA1) were treated with continuous intraventricular baclofen (IVB) infusion. On IVB of 220 μg/day, one 10‐year‐old girl had an 85% reduction in dystonia, from Barry‐Albright Dystonia Scale (BADS) score 30.7 to 4.5 (maximum score: 32) at 30 postoperative months. Her enteral dystonia medications were reduced >60%, and she discontinued medications for pain, anxiety, and depression. A second GA1 patient, age 23, experienced a more modest 18% reduction in dystonia (BADS decrease from 29.7 to 24.3) on IVB of 1,665 μg/day at 14 postoperative months. He substantially reduced his enteral dystonia medications and reported meaningful pain relief. These cases demonstrate that IVB may be a palliative option in the intractable dystonia of GA1. Our provisional observations suggest that IVB may be more beneficial in younger GA1 patients. |
| Databáze: | OpenAIRE |
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