Anti-CD20 monoclonal antibodies are also effective in the treatment of pediatric patients with multiple sclerosis. Although little used (with the exception of natalizumab) in people who receive a diagnosis before the age of 18 (around 4-8 percent of all patients), this category of disease-modifying drugs (DMTs) determines a benefit that materializes with a reduction in long-term disabilities.
Evidence, that emerged from a study presented at the last congress of the European Society for the Treatment and Research of Multiple Sclerosis (Ectrims), which could represent the basis for a change of approach in the management of the pediatric forms of the disease, with the possible extension of use of active ingredients already in use in the therapy of adult patients (ocrelizumab, alemtuzumab, ofatumumab).
Pediatric multiple sclerosis: monoclonal antibodies slow disability
To reach these conclusions, the researchers analyzed the data of over 280 patients (selected from the Italian, French and global MSBase registries) who developed symptoms of the disease before the age of 18. The sample was divided into two groups, based on the age at which they began therapy with monoclonal antibodies: 12-17 and 20-22 years. Two intervals that still allow us to define a young onset of the disease.
Once the differences between the different patients had been excluded, it was possible for the researchers to understand how the therapeutic choice influenced the development of the disability (assessed with the Edss scale).
It was thus recognized that patients who had started treatments between the ages of 12 and 17 (4 out of 10) had recorded an increase in disability of 0.4 points, compared to the 0.95 recorded among those who had been prescribed monoclonals later. An almost similar gap was also detected among slightly older patients (23-27 years).
The greatest impact on patients with moderate disabilities
“The slowing of disease progression was particularly evident in patients with moderate disability,” he explained Sifat Sharminbiostatistician and researcher in the clinical outcomes unit at the University of Melbourne, presenting the data at the Copenhagen congress.
What has already been observed in women therefore also applies to younger patients: regardless of gender. “These data highlight the importance of early therapy with highly effective drugs to preserve neurological function and reduce the progression of disability.”
Pediatric multiple sclerosis: a decisive decade for new therapies
Regulatory agencies currently authorize the use of few drugs to treat pediatric multiple sclerosis. Beyond interferon and glatiramer acetate, which have low efficacy, the use of teriflunomide, fingolimod and natalizumab.
For the other active ingredients there is a lack of data (efficacy and safety) detected in clinical trials conducted enrolling patients under 18. However, several phase 3 studies are underway to evaluate the safety, tolerability, pharmacological characteristics and the most adequate dosage of monoclonal antibodies in the pediatric population.
The first results are expected between the end of 2025 and 2026.
Towards new guidelines for the treatment of pediatric multiple sclerosis?
The data presented in the Danish capital follows by a few months those emerging from a study published in The Lancet child & adolescent healthconducted on over five thousand patients (including a portion selected from 178 Italian centers) with an average age at onset of the disease of 15 years.
In addition to anti-CD20 and authorized drugs, in this work (conducted by many clinicians also involved in the study presented in Copenhagen) the response to mitoxantrone, cladribine and autologous hematopoietic stem cell transplantation was detected. That is, almost all of the active ingredients that today make up the rich range of highly effective drugs used against the disease.
The conclusions are the same: “Children with relapsing-remitting multiple sclerosis must be treated early with highly effective therapy, before they develop serious neurological impairments”, is what the authors of the study put in black and white. A thought that Sharmin agreed with in the Danish capital: “It’s time to rethink the current guidelines for the treatment of pediatric multiple sclerosis.”
