Resolution of Fenfluramine-associated Pulmonary Arterial Hypertension in Lennox-gastaut Syndrome: A Case Report and Literature Review
Abstract number :
1.542
Submission category :
7. Anti-seizure Medications / 7D. Drug Side Effects
Year :
2024
Submission ID :
1427
Source :
www.aesnet.org
Presentation date :
12/7/2024 12:00:00 AM
Published date :
Authors :
Presenting Author: Steven Wolf, MD – Boston Children's of New York
Patricia McGoldrick, NP, MSN, MPA, FAES – Boston Children's Health Physicians
Richard Wang, BS – New York Medical College School of Medicine
Marissa Petchpradub, BS – New York Medical College
Ariel Sacknovitz, MS – New York Medical College
Rebecca Strafella, MS – New York Medical College
Rationale: Fenfluramine is a serotonin agonist medication originally approved for weight loss before being withdrawn over concerns for pulmonary arterial hypertension (PAH) and cardiac valvulopathy. In recent years, interest in fenfluramine at lower doses has re-emerged for the treatment of drug-resistant epilepsy (DRE). Here, we present a case of a patient with Lennox-Gastaut Syndrome (LGS) and DRE treated with fenfluramine, with subsequent development of subclinical PAH that resolved upon discontinuation.
Methods: A 4-year old female with LGS and DRE managed with a vagus nerve stimulator (VNS) and felbamate was screened by echocardiogram and started on fenfluramine due to persistent seizures. At 6 months post-initiation, the patient underwent a routine follow up echocardiogram, which showed development of asymptomatic PAH. The patient was weaned from fenfluramine, and at 2 months post-cessation of fenfluramine, echocardiogram showed resolution of PAH. The patient was subsequently restarted on fenfluramine due to persistent refractory seizures, and follow up echocardiogram at 2 months showing no evidence of recurrence of PAH. Fenfluramine dosage was increased to the maximum for weight of 14.08 mg/day. At last follow-up, no signs or symptoms of PAH were found.
Results: We present a case of LGS with DRE treated with fenfluramine that was complicated by PAH until cessation of fenfluramine. Subsequent retrial of fenfluramine was tolerated without re-development of PAH. This is, to our knowledge, the first pediatric patient to develop cardiac abnormalities during treatment with fenfluramine. The child was rechallenged with fenfluramine after normal echocardiograms were obtained, and continues to be closely followed. Fenfluramine has otherwise been demonstrated to be efficacious and safe in DRE in several randomized controlled trials and cohort studies at lower doses than those originally utilized for adult weight loss. With appropriate screening and monitoring, fenfluramine should be considered as an adjunct treatment option for DRE in select pediatric patients.
Conclusions: Interest in fenfluramine has been renewed in recent years for its potential in treating DRE. Screening and monitoring is necessary to detect cases of rare cardiopulmonary complications, and cessation and appropriate re-trial are options for management of such complications when they occur.
Funding: none
Anti-seizure Medications