Effects of GBA1 Variants in Patients With Parkinson's Disease and Levodopa-Carbidopa Intestinal Gel: A Nation-Wide, Multicenter, Longitudinal, "Real-World" Study. The EPIC Study
These videos have been matched automatically. Contact us if they are not relevant.
These videos have been matched automatically. Contact us if they are not relevant.
View abstract on PubMed
Summary
This summary is machine-generated.Levodopa/carbidopa intestinal gel (LCIG) is safe for Parkinson's disease (PD) patients with GBA1 mutations, showing similar adverse event risks. However, GBA1-PD patients experienced less motor improvement and faster cognitive decline with LCIG.
Area Of Science
- Neurology
- Genetics
- Pharmacology
Background
- Levodopa/carbidopa intestinal gel (LCIG) is a treatment for advanced Parkinson's disease (PD).
- The efficacy and safety of LCIG in PD patients with GBA1 mutations (GBA-PD) are not well understood.
- GBA1 mutations are a significant genetic risk factor for PD.
Purpose Of The Study
- To assess the safety and effectiveness of LCIG in a large cohort of PD patients.
- To specifically evaluate the outcomes of LCIG in patients carrying GBA1 variants.
- To compare GBA-PD patients with non-carriers regarding LCIG treatment response.
Main Methods
- A multicenter, retrospective, longitudinal "real-world" study.
- Inclusion of consecutive advanced PD patients treated with LCIG across 31 Italian centers.
- Data collection at baseline, 1-year, 5-year, and last available follow-up, including GBA1 genotyping.
Main Results
- LCIG improved motor and non-motor symptoms, OFF-time, and dyskinesias in the overall PD population.
- GBA-PD patients showed no significant improvement in MDS-UPDRS parts I-III, less improvement in part IV, and faster cognitive decline compared to non-carriers.
- GBA1 mutation status did not predict adverse events or LCIG discontinuation.
Conclusions
- LCIG is a safe treatment option for advanced GBA-PD, irrespective of baseline cognitive status.
- GBA-PD patients may experience reduced efficacy in motor symptom management and a more rapid cognitive decline compared to non-carriers.
- GBA1 mutation status does not heighten the risk of adverse events or treatment discontinuation with LCIG.
These videos have been matched automatically. Contact us if they are not relevant.
These videos have been matched automatically. Contact us if they are not relevant.
Related Concept Videos
Neurodegenerative disorders, such as Parkinson's Disease (PD), involve the gradual and irreversible destruction of neurons in particular brain areas. These disorders exhibit standard features like proteinopathies, selective vulnerability of some neurons, and an interaction of intrinsic properties, genetics, and environmental influences in neural injury.
Parkinson's Disease is primarily a result of the loss of dopaminergic neurons in the substantia nigra pars compacta. The cornerstone of...
Neurodegenerative disorders are progressive diseases that cause irreversible damage and loss to neurons in specific brain areas. Examples of these disorders include Parkinson's disease, Alzheimer's disease, Multiple Sclerosis (MS), and Amyotrophic Lateral Sclerosis (ALS). These disorders share characteristics such as proteinopathies, selective neuronal vulnerability, and a complex interplay between genetic and environmental factors. The primary therapeutic goal for these conditions is...
Prokinetic agents are specialized medications that stimulate gastrointestinal (GI) motility, promoting food movement through the GI tract. Dopamine, an inhibitory neurotransmitter, plays a significant role in this process, reducing GI motility and indirectly controlling the speed of digestion. Dopamine receptor antagonists, such as metoclopramide and domperidone, offer a unique advantage as prokinetic agents. By blocking the dopamine receptors, these drugs increase GI motility, improving food...
Digestion begins with a cephalic phase that prepares the digestive system to receive food. When our brain processes visual or olfactory information about food, it triggers impulses in the cranial nerves innervating the salivary glands and stomach to prepare for food.
The cephalic phase is a conditioned or learned response to familiar foods. Our appetite or desire for a particular food modifies the preparatory responses directed by the brain. Individuals may produce more saliva and stomach...
Gastrointestinal or GI motility disorders are characterized by irregular gastrointestinal tract movements, disrupting food transit from the mouth to the anus. They are caused by damage or dysfunction in gut muscles or nerves. These disorders can cause symptoms such as severe constipation, diarrhea, abdominal pain, and swallowing difficulties. Disorders can affect any segment of the GI tract and range widely in severity, from common conditions like GERD to life-threatening conditions like...