Patient registries collect information about the health status of people with a certain disease. They are particularly important for rare diseases such as Lambert-Eaton myasthenic syndrome (LEMS), bringing together a small patient population and gathering information about how the disease affects them.
Researchers in academia and pharmaceutical companies can then use this information to develop potential new treatments for the disease. Below is more information about the European LEMS registry.
The registry began in 2010 and recruited patients for five years and then continued monitoring them until 2018.
It was non-interventional, meaning that patients did not receive any treatments or undergo testing outside their usual maintenance.
What were the registry’s goals?
The registry was created to collect more information about the natural history and progression of LEMS.
It also aimed to investigate the safety of Firdapse, observe the safety outcomes of other treatments for comparison, and gather more information about the long-term safety and effectiveness of different treatments.
The setup of the registry was a requirement as part of the approval of Firdapse by the European Medicines Agency.
Who could participate?
The registry was open to all patients with LEMS, regardless of whether or not they were taking Firdapse. Patients would, however, need to be able to travel to one of 29 medical centers located in four different countries: Germany, Italy, France, and Spain.
What information did the registry collect?
The registry collected different data to monitor any changes in LEMS progression, including information about any LEMS-specific treatments that patients had taken before the registry and were receiving at different times throughout the registry.
The registry also collected assessment information routinely used for LEMS monitoring, including quantitative myasthenia gravis scores, compound muscle action potential tests, muscle strength assessments, tests for impaired balance or coordination (ataxia), reflex tests, observation of any symptoms related to trouble with the autonomic nervous system, electrocardiograms, and tests of breathing ability.
Patients also answered questionnaires about their general health and daily functions, such as the ability to walk, climb stairs, and stand up.
What do preliminary data show?
A research article published in 2015 showed that 69 patients had enrolled in the registry by that time. Thirty-six of these were male and 32 were female. One patient’s gender was not identified. The patients’ ages at enrollment ranged from 27 to 84, with a mean average age of 61.5. Eighteen of the patients had cancer, including 11 with lung cancer.
Sixty-five percent of patients were taking either Firdapse (43%) or a similar medication, Ruzurgi (22%). Most patients had mild to moderate loss of daily function with 39% of patients having moderate anxiety and/or depression.
Researchers have published no further results from the registry so far. However, they included updated safety information in the New Drug Application for Firdapse to the U.S. Food and Drug Administration. In the application, a total of 106 patients had enrolled in the registry by the cutoff date of September 2017. Of those patients, 78 had received Firdapse at some point.