2024 Volume 41 Issue 5 Pages 759-764
Recent advances in the field of neurological diseases have led to an increase in treatable conditions and significant changes in treatment approaches. Among the diseases characterized by autonomic dysfunction, hereditary ATTR amyloidosis and Parkinson disease have shown notable treatment progress.
Hereditary ATTR amyloidosis is caused by abnormal folding of the transthyretin (TTR) protein, leading to amyloid deposits in tissues and organs. Autonomic dysfunction, such as cardiovascular, gastrointestinal, and urinary issues, is a prominent early symptom. Treatment options have evolved from liver transplantation to TTR–stabilizing drugs like tafamidis, which prevents TTR misfolding and amyloid formation, and RNA interference therapies like patisiran and vutrisiran, which reduce TTR production. These disease–modifying therapies have shown improvements in autonomic symptoms, gastrointestinal function, and quality of life.
Parkinson disease treatment initially focused on motor symptoms, but recent advancements have addressed non–motor symptoms, including autonomic dysfunctions such as orthostatic hypotension and gastrointestinal issues. Deep brain stimulation (DBS) has improved autonomic functions like heart rate variability and gastrointestinal motility. Continuous Levodopa–Carbidopa Intestinal Gel (LCIG) therapy stabilizes blood levels of levodopa, reducing fluctuations in symptoms and improving autonomic functions such as constipation and orthostatic hypotension. These are considered disease–modifying therapies as they offer more comprehensive management of Parkinson disease.
Future treatment prospects for autonomic diseases include gene therapy, stem cell therapy, and advanced deep brain stimulation techniques. Research on immunotherapy and antibody therapy targeting α–synuclein in Parkinson disease and other neurodegenerative diseases is ongoing, despite challenges in clinical trials. These advancements hold promise for further improving the quality of life for patients with autonomic dysfunction.
In conclusion, the continuous evolution of treatment methods for hereditary ATTR amyloidosis and Parkinson disease represents a significant step forward in managing autonomic dysfunction. The development and implementation of disease–modifying therapies are pivotal in this progress. Ongoing research and clinical trials are expected to bring even more innovative therapies to the forefront, offering hope for better management and improved outcomes for patients suffering from these debilitating conditions.
