High-dose cyclophosphamide promising for severe refractory MS
Last Updated: 2006-10-13 16:15:23 -0400 (Reuters Health)

NEW YORK (Reuters Health) - In patients with moderate to severe refractory MS, high-dose cyclophosphamide may lead to disease stabilization and improved functionality and quality of life, according to early results of a study reported in the Archives of Neurology for October.

High-dose cyclophosphamide has demonstrated activity against autoimmune neurologic illnesses, but this study represents the first time it’s been tried in MS, first author Dr. Douglas E. Gladstone of the State University of New York at Stony Brook told Reuters Health.

He and colleagues determined the clinical response to cyclophosphamide (200 mg/kg over 4 days) in 12 patients with moderate to severe refractory MS that had progressed despite treatment with the two FDA-approved disease-modifying medications.

According to the team, cyclophosphamide was associated with "minimal morbidity and improved clinical outcomes. After high-dose cyclophosphamide, no patient met study criteria for disease progression."

During a median follow up of 15 months (range 6-24 months), no patients increased their entry level Expanded Disability Status Scale (EDSS) score by more than 1.0; five patients (42%) decreased their EDSS score by 1.0 or more.

"What was surprising and unexpected was that 40% of the patients actually had a measurable increase in their physical abilities," Dr. Gladstone said. "Some people had improvement in gait and many had improvement in vision and bladder control. Bladder control is one of those things that if it improves at all it’s a huge change in their quality of life," he noted.

Two of 11 patients had a single enhancing lesion at baseline; these lesions resolved after cyclophosphamide treatment. At one year, one patient showed one new enhancing lesion. Treatment response was seen regardless of the presence or absence of contrast lesion activity at baseline.

Overall, Dr. Gladstone said he’s "more than pleased with the results," adding "our goal is to study patients with less severe MS."

Arch Neurol 2006;63:1388-1393.