A ground-breaking study – the largest of its kind globally – has found children with multiple sclerosis (MS) have better outcomes if treated early and with the same high-efficacy therapies as adults.

There are a limited number of therapies approved for children with MS, with only one considered to be of high-efficacy – meaning highly effective.

However, a Royal Melbourne Hospital (RMH) observational study has determined that paediatric patients should be treated with the same high-efficacy treatments offered to adults as early in their diagnosis as possible to avoid the onset of significant disability.

“We found that patients who were treated with high-efficacy disease-modifying therapies during the initial phases of their disease benefitted the most compared to patients who were not treated,” Dr Sifat Sharmin, a Research Fellow at the Royal Melbourne Hospital’s Neuroimmunology Centre, and University of Melbourne’s Department of Medicine, said.

“Based on our findings we recommend that patients with paediatric-onset multiple sclerosis should be treated early in the disease course, when the disability is still minimal, to preserve neurological capacity before it’s damaged.”

The observational study analysed global data of more than 5000 people diagnosed with MS during childhood over the last 30 years – including from MSBase, a large international registry encompassing 41 countries, and a national registry in Italy, where the disease is highly prevalent.

It compared the strength of treatment with the severity of the disease later in life, and concluded patients treated with the most effective treatments early on in their diagnosis were less likely to experience disability worsening. These disease-modifying therapies include highly effective antibodies that change the way in which an individual’s immune system behaves.

The findings were published in the prestigious journal the Lancet Child and Adolescent Health this week.

The research also confirmed that any treatment – including low-efficacy treatments – was better than no treatment.

Dr Sharmin, who led the study, said because paediatric-onset MS was a rare disease – about four to eight per cent of MS patients are diagnosed before age 18 – it wasn’t as well investigated.

“This is the largest study of its kind for paediatric MS,” she said.

“We hope this may have some policy implications so children with MS can access the most effective therapies as early as possible.”

MS is a chronic condition that occurs when the immune system attacks the brain and spinal cord. There is currently no cure for the condition.

Allie won't let MS slow her down

Allie was just 12 years' old when she was diagnosed with MS.

After falling and hitting her head, and having persistent double vision, Allie was referred to the Royal Children’s Hospital (RCH) by her optometrist.

Multiple scans and tests later, Allie was told she had MS.

“I didn’t know what it meant,” Allie recalls.

“It was a bit scary at first. There weren’t many kids that had MS. But as I got older it’s just become my day to day – it doesn’t affect me at all. I don’t try and dwell on it.”

Now 19, and in the care of the RMH, Allie is focused on her exciting job as an apprentice aerospace manufacturer engineer for a major aviation company.

“Basically I make the wings [for planes],” Allie said.

“It’s a lot of hard labour. But I knew I could never sit and do a 9-5 job. I hate sitting down and I used to love making things.

“It’s the best thing to happen [to me] – I feel like I have something to work towards.”

Allie said she was grateful for the care she’s received from a young age - which has enabled her to live life to its fullest.

“It’s really eye opening how lucky I am to be in Australia and have this treatment. If I was in another country… I feel very lucky I have this support system,” she said.

Dr Sifat Sharmin
Dr Sifat Sharmin hopes her research will influence how children with MS are treated.
Mobile Stroke Unit with Ambulance Victoria paramedic and the RMH Stroke team
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