Early Tx Exposure May Prevent Progression in Pediatric Clinically Isolated Syndrome

boy child hospital
boy child hospital
Patients at the greatest risk of disability progression were older than 12 years, and had minimal exposure to DMTs.

Pediatric patients with clinically isolated syndrome who experience spinal or optic neuritis and have minimal exposure to disease-modifying treatments are most likely to have disability progression, according to findings presented at the 32nd Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), taking place in London September 14-17, 2016.

The authors, led by Pietro Iaffaldano, MD, of the University of Bari Aldo Moro in Bari, Italy, sought to identify predictive factors of disability progression in pediatric patients with a diagnosis of clinically isolated syndrome suggestive of multiple sclerosis.

The researchers used the Recursive Partitioning and Amalgamation (RECPAM) method to identify subgroups at different degrees of risk among 770 pediatric patients with clinically isolated syndrome. The patients were followed for up to 10 years to evaluate the risk of progression in Expanded Disability Status Scale (EDSS) score. An increase in EDSS score was deemed progression if the patient experienced a 1-point increase in EDSS score from baseline if baseline was 1-5.5; a 1.5-point increase from baseline if baseline was 0; or a 0.5-point increase from baseline if baseline was ≥6.

Three heterogeneous subgroups were identified using RECPAM analysis. Patients in Class 1, who were at the greatest risk of disability progression, were more likely to be older than 12 years at symptom onset compared with patients in Classes 2 and 3 (97% vs 79% and 92%, respectively; P =.002). Patients in Class 1 were also more likely to have an isolated spinal (33.3% vs 9.5% and 8.3%; P =.0003) or optic neuritis (30.3% vs 20.7% and 20.4%) symptom at onset; relapses after the initial attack (39.4% vs 6% and 0%; P <.001); and less exposure to disease-modifying treatments (54% vs 78.5% and 71.3%; P =.02) compared with patients in Classes 2 and 3. Further analysis suggested that exposure to disease modifying treatments was the most protective factor in the prevention of disability progression (HR=0.22, 95% Confidence Interval 0.13-0.38).

“This work represents an important step forward in identifying predictors of an unfavorable course in patients with pCIS and, for the first time, it supports a beneficial effect of an early DMT exposure in preventing disability accumulation in this population,” the authors concluded.

Disclosures: The authors report multiple disclosures. See the abstract for a complete list.

For more coverage of ECTRIMS 2016, go here. 


Iaffaldano P, Simone M, Lucisano G, et al. Early disease modifying treatment delays disability accumulation in patients with pediatric onset clinically isolated syndrome suggestive of multiple sclerosis. Presented at: ECTRIMS 2016. September 14-17, 2016; London, UK. Abstract 187.