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Bringing the HEET: The Argument for High-Efficacy Early Treatment for Pediatric-Onset Multiple Sclerosis
Marisa McGinley1, Ian T Rossman2
1Mellen Center for Multiple Sclerosis Treatment and Research, Cleveland Clinic, 9500 Euclid Avenue U10, Cleveland, OH, 44195, USA.
Insights
Pediatric-onset multiple sclerosis (POMS) presents unique challenges. A new high-efficacy early treatment (HEET) strategy using potent DMTs may offer better outcomes for pediatric MS patients.
Area of Science:
- Neurology
- Immunology
- Pediatrics
Background:
- Pediatric-onset multiple sclerosis (POMS) is a rare but distinct form of MS.
- POMS presents different diagnostic and treatment challenges compared to adult-onset MS.
- POMS often exhibits more active disease than adult-onset MS.
Purpose of the Study:
- To review clinical and radiographic presentations of POMS.
- To explore differences in natural history and long-term outcomes between POMS and adult-onset MS.
- To introduce and evaluate the high-efficacy early treatment (HEET) strategy for POMS.
Main Methods:
- Review of clinical and radiographic presentations in POMS.
- Comparison of POMS and adult-onset MS natural histories and outcomes.
- Analysis of available disease-modifying therapies (DMTs) for POMS.
- Introduction of the HEET strategy for POMS treatment.
Main Results:
- POMS is rarer than adult-onset MS but often presents with more active disease.
- Current guidelines often recommend off-label, lower-efficacy DMTs for POMS.
- The HEET strategy proposes using high-efficacy, intravenously infused DMTs as first-line therapy.
Conclusions:
- The HEET strategy offers a novel approach to managing POMS.
- High-efficacy early treatment may be more beneficial for POMS than current guidelines suggest.
- Prospective studies are needed to validate the HEET strategy, with treatment decisions made collaboratively.
Abstract:
Pediatric-onset multiple sclerosis (POMS) is rarer than adult-onset disease, and represents a different diagnostic and treatment challenge to clinicians. We review POMS clinical and radiographic presentations, and explore important differences between POMS and adult-onset MS natural histories and long-term outcomes. Despite having more active disease, current treatment guidelines for patients with POMS endorse the off-label use of lower-efficacy disease-modifying therapies (DMTs) as first line. We review the available MS DMTs, their evidence for use in POMS, and the contrasting treatment strategies of high-efficacy early treatment and escalation therapy. We introduce a new treatment approach, the "high-efficacy early treatment", or HEET strategy, based on using directly observed, high-efficacy intravenously infused DMTs as first-line therapies. Like other proposed POMS treatment strategies, HEET will need to be prospectively studied, and all treatment decisions should be determined by an experienced neurologist, the patient, and his/her parents.
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