Pregabalin was found to significantly reduce both pain and itch in patients with recessive dystrophic epidermolysis bullosa, according to a new study.
"Despite the study's small sample size, the results demonstrated a significant decrease in pain levels with pregabalin use compared to baseline, as opposed to the placebo use," noted lead author Margarita Calvo, MD, PhD, of the Pontificia Universidad Católica de Chile.
The randomized, double-blind crossover trial, published in JAMA Dermatology, researchers recruited 10 patients with recessive dystrophic epidermolysis bullosa (RDEB) aged 8 to 40 years who had evidence of neuropathic pain and itch. The participants received both pregabalin (50 to 300 mg/day) and matched placebo in randomized sequences over 24 weeks.
Dr. Calvo and colleagues found that pregabalin reduced mean pain scores by 1.9 points on a 10-point visual analog scale (VAS) compared with 0.1 points for placebo. Itch scores showed a modest but significant reduction of 0.9 points with pregabalin vs 0.1 points with placebo.
Half of pregabalin users achieved a 30% or greater reduction in pain levels, while 30% reported a 50% or greater reduction. The mean pregabalin maintenance dose was 186.1 mg/day and was generally well-tolerated with only minor adverse effects reported.
"The trial represent[s] the first evidence for pregabalin’s efficacy in RDEB-associated neuropathic pain and itch, potentially offering a new treatment option for this rare genetic condition where pain management has traditionally posed substantial challenges," said Dr. Calvo.
In an interview with this news organization, she discussed dosing strategies for patients with RDEB who were already using pregabalin off-label.
Gradual Dose Titration: Start with a low dose and gradually increasing every 2 to 5 days, based on patient tolerance and efficacy. This approach helps minimize side effects while allowing the patient to adjust to the medication.
Regular Follow-ups: Conduct frequent follow-up appointments to monitor pain levels, side effects, and overall functioning. Adjustments to the dosing regimen can be made based on these assessments.
Monitor for Side Effects: Patients should be educated on potential side effects such as sedation, dizziness, and cognitive impairment. Monitoring for these effects, especially during the titration phase is crucial, she said.
There were recruitment challenges, as many of the eligible patients were already using pregabalin off-label and declined the required washout period. Larger studies are needed to validate the preliminary results.
Although the study demonstrated significant pain and itch reduction, no improvement in quality-of-life scores was observed. Dr. Calvo suggested additional outcome measures for future trials: “I would recommend the following":
Functional Assessment Tools: Measure functional outcomes related to daily activities and social interactions to evaluate how symptom relief translates into improved functioning.
Patient-Reported Outcome Measures: Utilize measures that focus on the broader impact of symptoms on mental health and emotional well-being, such as the Patient Health Questionnaire (PHQ-9) for depression and the Generalized Anxiety Disorder 7-item scale (GAD-7).
Patient Satisfaction Surveys: Gather qualitative data through surveys or interviews to assess patients’ perceptions of treatment efficacy, their satisfaction with symptom management, and any changes in their overall health status.
Multidimensional Pain Scales: Implement more comprehensive pain assessment tools that evaluate not only pain intensity but also the quality and impact of pain on various aspects of life (e.g., the McGill Pain Questionnaire),” she underscored.
Conflict of interest disclosures can be found in the study.
