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Safety and efficacy of propranolol for treatment of familial cerebral cavernous malformations (Treat_CCM): a randomised, open-label, blinded-endpoint, phase 2 pilot trial.

Author: , Al-Shahi SalmanRustam, BertaniGiulio A, DejanaElisabetta, LanfranconiSilvia, LatiniRoberto, MeessenJennifer M T A, PalliniRoberto, ScolaElisa

Randomized Controlled TrialA clinical trial that randomly assigns participants to treatment and control groups to evaluate the effects of an intervention.
Clinical Trial, Phase IIA Phase II trial is a trial that further evaluates the efficacy and safety of a new drug or treatment, whose safety has been confirmed to some extent in a Phase I trial, in a small number of patients.
Journal ArticleAn article published in an academic or professional journal that reports original research or scholarly analysis.
Research Support, Non-U.S. Gov'tResearch funding provided by government agencies outside the United States.

Overview

This study investigates the safety and efficacy of prolonged propranolol treatment in reducing the risk of symptomatic intracerebral hemorrhage or focal neurological deficit in people with familial cerebral cavernous malformations, based on observations suggesting its potential benefit.
Paper Details　
Original Abstract of the Article :
Observations in people with cerebral cavernous malformations, and in preclinical models of this disorder, suggest that the β-blocker propranolol might reduce the risk of intracerebral haemorrhage. We aimed to evaluate the safety and efficacy of prolonged treatment with propranolol to reduce the inci...See full text at original site
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引用元：
https://doi.org/10.1016/S1474-4422(22)00409-4

データ提供：米国国立医学図書館（NLM）

Propranolol for Familial Cerebral Cavernous Malformations: A Promising Oasis in the Desert of Brain Disorders

Cerebral cavernous malformations (CCMs), like treacherous sandstorms in a desert, can lead to devastating neurological complications. This study, a randomized, open-label, blinded-endpoint, phase 2 pilot trial, explores the potential of propranolol, a beta-blocker, in reducing the risk of intracerebral hemorrhage in patients with familial CCMs. The researchers aimed to assess the safety and efficacy of long-term propranolol treatment in these patients. The study is ongoing and has not yet yielded definitive results. However, the preliminary findings suggest that propranolol may be a safe and potentially effective treatment option for patients with familial CCMs.

A New Path in the Desert of CCMs: Exploring Propranolol

This research, like a camel venturing into uncharted territory, explores the potential of propranolol in treating familial CCMs. The study's findings, while preliminary, offer a glimmer of hope for patients with this challenging condition. Further research is needed to definitively establish the efficacy of propranolol in reducing the risk of intracerebral hemorrhage in these patients.

Navigating the Desert of Brain Disorders: Seeking New Solutions

This study serves as a reminder that ongoing research is crucial for finding new and effective treatments for complex brain disorders. The exploration of propranolol as a potential treatment option for familial CCMs highlights the importance of continuously seeking novel therapies. As we navigate the vast desert of brain disorders, we must remain optimistic and persistent in our pursuit of better treatments.

Dr. Camel's Conclusion

This study, like a camel traversing the shifting sands of brain disorders, explores the potential of propranolol in treating familial cerebral cavernous malformations. While the results are still preliminary, they offer a beacon of hope for finding new and effective treatments for this challenging condition.

Date :
  1. Date Completed 2022-12-16
  2. Date Revised 2023-03-15
Further Info :

Pubmed ID

36403580

DOI: Digital Object Identifier

10.1016/S1474-4422(22)00409-4

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Article Analysis
・Side Effects of propranolol (cardiovascular)
SNS
PICO Info
in preparation
Languages

English

Positive IndicatorAn AI analysis index that serves as a benchmark for how positive the results of the study are. Note that it is a benchmark and requires careful interpretation and consideration of different perspectives.

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