Small Patients, Big Dilemmas: A Critical Review of the TERIKIDS Study

During the BINCA Journal Club, Dr. Rodrigo Martínez analyzed the TERIKIDS clinical trial on pediatric multiple sclerosis.

On March 26, in our second edition of the BINCA Journal Club of the year, Dr. Rodrigo Martínez Espinosa, Sanofi LATAM Medical Manager and professor at La Salle University, led an enriching session focused on the clinical study "Safety and efficacy of teriflunomide in pediatric multiple sclerosis (TERIKIDS)", published in The Lancet Neurology. This phase 3, double-blind, multicenter, placebo-controlled trial evaluated the safety and efficacy of teriflunomide—an oral immunomodulatory agent—in children and adolescents diagnosed with relapsing-remitting multiple sclerosis.

Dr. Rodrigo began by offering a concise overview of multiple sclerosis, before delving into the methodological design of the study. He highlighted the ethical and methodological complexities introduced by the inclusion of an open-label extension, used as a “rescue strategy” for patients with high MRI activity or clinical relapse. While ethically justified, this design choice raises concerns about potential bias and reduced statistical power. This set the stage for a central question: To what extent should statistical outcomes dictate clinical decisions when observable patient benefits are present?

One of the session’s most compelling discussions centered on the ethical implications of the trial design. Dr. Rodrigo firmly raised a critical concern: Is it ethically acceptable to administer a placebo to children suffering from an active inflammatory disease such as multiple sclerosis? “We’re dealing with pediatric patients carrying a significant disease burden, who initially receive no active treatment,” he pointed out. While the study allowed participants to switch to the active drug upon relapse or disease progression seen on MRI, this only occurred after clear signs of worsening. Moreover, patients who transitioned to the open-label phase after receiving placebo displayed different clinical and statistical outcomes compared to those treated with teriflunomide from the beginning—complicating both the efficacy assessment and interpretation of the drug’s actual impact. The conversation underscored the delicate balance between scientific rigor and the ethical imperative to safeguard vulnerable research populations, emphasizing the crucial role of ethics committees in pediatric research.

The session also explored how patient dropout impacts the interpretation of results. “Data interpretation must go beyond p-values—we need to consider clinical context and the consistency of observed effects,” Dr. Rodrigo stressed.

The discussion concluded with a forward-looking reflection on the current landscape and future of pediatric neuroimmunology research. Advances in oral immunomodulatory therapies were reviewed, and the importance of case reports as valuable evidence in areas with limited clinical trials was reaffirmed.

You can watch the full session at: https://www.facebook.com/share/v/1BmUbJErLk/ 

The Bioethics and Neuroethics Group at Universidad Anáhuac (BINCA) is a flagship initiative of CADEBI. Since October 2014, it has fostered critical reflection and interdisciplinary research.

Composed of students, academics, and researchers in the health sciences, BINCA focuses on the bioethical study of health-related disciplines through a comprehensive, multidisciplinary lens.