Long‐term safety and treatment effects of cannabidiol in children and adults with treatment‐resistant epilepsies

Research Report URL: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6175436/

What does this paper’s abstract explain?

The abstract of this paper reports interim results on the safety and efficacy of cannabidiol (CBD) in an ongoing expanded-access program (EAP) for patients with treatment-resistant epilepsies (TREs) treated through December 2016. The objective of the paper is to evaluate the long-term safety and treatment effects of CBD in children and adults with TREs.

What are the main results of the paper?

The paper reports interim results on the safety and efficacy of cannabidiol (CBD) in patients with treatment-resistant epilepsies (TREs) who were enrolled in an ongoing expanded-access program (EAP) since 2014. The results showed that CBD was generally well-tolerated, with most adverse events (AEs) being mild or moderate in severity. The most common AEs were diarrhoea, somnolence, decreased appetite, and fatigue. CBD treatment was associated with a significant reduction in seizure frequency in most patients, with some patients achieving complete seizure freedom. The study provides important information on the long-term safety and treatment effects of CBD in patients with TREs.

What are the contributions of this paper?

This paper contributes to the evaluation of the long-term safety and treatment effects of cannabidiol (CBD) in children and adults with treatment-resistant epilepsies (TREs) through an ongoing expanded-access program (EAP). The interim results reported in this paper provide important information on the safety and efficacy of CBD as a potential treatment option for patients with TREs.

What does the introduction of this paper summarise?

The introduction of this paper provides background information on the use of cannabidiol (CBD) as a potential treatment option for patients with treatment-resistant epilepsies (TREs). It highlights the limitations of current treatments for TREs and the need for new and effective therapies. The paper describes an ongoing expanded-access program (EAP) that has been providing CBD to patients with TREs since 2014. The objective of the paper is to report interim results on the safety and efficacy of CBD in EAP patients treated through December 2016.

What literature does this paper provide?

This paper provides a literature survey of previous studies on the use of CBD in the treatment of epilepsy. The authors note that while there is growing interest in the use of CBD for epilepsy, there is still a lack of high-quality evidence to support its use. They highlight several studies that have reported positive results with CBD, including a randomized, double-blind, placebo-controlled trial of CBD in patients with Dravet syndrome, and an open-label study of CBD in patients with Lennox-Gastaut syndrome. However, they also note that some studies have reported negative or inconclusive results, and that more research is needed to fully understand the safety and efficacy of CBD for epilepsy.

What methods were used in this paper?

The paper reports interim results on the safety and efficacy of CBD in patients with treatment-resistant epilepsies (TREs) who were enrolled in an ongoing expanded-access program (EAP) that has been providing CBD since 2014. The methods used in this paper include:

• A retrospective analysis of data collected from patients enrolled in the EAP through December 2016.
• The primary outcome measures were adverse events (AEs) and changes in seizure frequency.
• Safety assessments were conducted at each visit, including physical and neurological examinations, vital signs, and laboratory tests.
• Efficacy assessments were based on changes in seizure frequency from baseline.
• Data were analysed using descriptive statistics.

What data was used in this paper?

The data used in this paper is from patients enrolled in an ongoing expanded-access program (EAP) that has been providing cannabidiol (CBD) to patients with treatment-resistant epilepsies (TREs) since 2014. The paper reports interim results on the safety and efficacy of CBD in EAP patients treated through December 2016. The methods used in this paper include a retrospective analysis of data collected from patients enrolled in the EAP.

What does the paper’s conclusion say?

The paper concludes that cannabidiol (CBD) is generally well-tolerated and effective in reducing seizure frequency in patients with treatment-resistant epilepsies (TREs). The study provides important information on the long-term safety and treatment effects of CBD in patients with TREs. However, further research is needed to fully understand the potential benefits and risks of CBD treatment in this patient population.

What limitations does this paper have?

The limitations of this paper include:

• The lack of a control group in the expanded access program (EAP) design, which limits the ability to draw definitive conclusions about the safety and efficacy of CBD in patients with TREs.
• The heterogeneity of patient populations and treatment protocols across the different EAP sites, which may affect the generalisability of the findings.
• The reliance on patient-reported outcomes and open-label design, which may introduce bias and affect the accuracy of the results.
• The limited information on the potential long-term effects of CBD treatment on cognitive function, behavior, and quality of life in patients with TREs.
• The lack of information on the potential interactions between CBD and other AEDs, which may affect the safety and efficacy of CBD treatment.

Overall, while the study provides important insights into the potential benefits and risks of CBD treatment in patients with TREs, further research is needed to fully understand the optimal use of CBD in this patient population.

What are the practical implications of this paper?

The practical implications of this paper are as follows:

• The interim results reported in this paper suggest that CBD may be a safe and effective treatment option for patients with treatment-resistant epilepsies (TREs).
• The findings of this paper may have important implications for the development of new treatments for TREs, as well as for the use of CBD in clinical practice.
• The results of this paper may also help to inform clinical guidelines and recommendations for the use of CBD in the treatment of TREs.
• The ongoing expanded-access program (EAP) described in this paper may provide a valuable resource for patients with TREs who are seeking alternative treatment options.

What future works are suggested in this paper?

The paper suggests that future research should focus on the following areas:

• Conducting randomised, double-blind, placebo-controlled trials to further evaluate the safety and efficacy of CBD in patients with TREs.
• Investigating the optimal dosing and administration of CBD for different types of epilepsies and patient populations.
• Exploring the potential interactions between CBD and other antiepileptic drugs (AEDs).
• Assessing the long-term effects of CBD treatment on cognitive function, behavior, and quality of life in patients with TREs.
• Investigating the potential use of CBD in combination with other therapies for the treatment of TREs.

These future research directions will help to further elucidate the potential benefits and risks of CBD treatment in patients with TREs and inform clinical practice.