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Outcomes of Haematopoietic Stem Cell Transplant Therapy Are Better in Patients With Early-Stage MS: Presented at ECTRIMS

By Chris Berrie

AMSTERDAM, the Netherlands -- October 26, 2011 -- High-dose immunosuppressive therapy with autologous haematopoietic stem cell transplantation (HSCT) is effective in patients with multiple sclerosis (MS), but outcomes are significantly better in patients undergoing transplantation at early stages of the disease.

Findings were presented here October 20 at the 5th Joint Triennial Congress of the European and Americas Committees for Treatment and Research in Multiple Sclerosis (ECTRIMS/ACTRIMS).

Over the past 10 years, high-dose immunosuppressive therapy plus HSCT has been increasingly used as a therapeutic option for patients with MS. However, patient selection criteria for this treatment remain unclear.

“Our previous results [with HSCT] in the progressive, late stage [of MS] were not very good, and so we decided to compare this with an early strategy,” said Denis A. Fedorenko, MD, Department of Haematology and Cellular Therapy, National Pirogov Medical Surgical Centre, Moscow, Russia.

The study included 186 patients with MS, of which 79 had secondary progressive disease, 28 had primary progressive, 5 had progressive-relapsing, and 74 had relapsing-remitting.

A total of 67 patients underwent early HSCT and 119 underwent late/salvage HSCT, but efficacy analysis at 12 months post transplant was performed in 142 patients. The median follow-up duration was 34 months (range, 1.5-143 months).

Most of the transplantation procedures were well tolerated. There was 1 death from sepsis with multiple organ failure was registered.

Neurological improvement was observed in 19 of the 46 patients who received early HSCT and stabilisation was observed in 26 patients. One patient in the early group progressed.

In the late/salvage HSCT group, neurological improvement was observed in 42 of the 96 patients, stabilisation was observed in 42 patients, and 12 patients progressed.

At long-term follow-up, overall clinical response (neurological improvement or stabilisation) was registered in 35 patients in the early HSCT group. Four patients progressed at different time-points after HSCT.

Among 78 patients in the late HSCT group, overall long-term clinical response was observed in 52 patients. A total of 26 patients progressed at different time-points after HSCT and 1 patient died after disease progression at 3 years post transplant.

The estimated 5-year progression-free rates were 87% in the early HSCT group and 70% in late/salvage HSCT group.

“Our results show that the treatment outcome is significantly better in the early group, compared with the late and salvage group,” said Dr. Fedorenko. However, he noted the need for further studies to better define the most beneficial timing for patients and to determine the true role of this approach in the treatment of patients with MS.
[Presentation title: Early Versus Late/ Salvage Autologous Haematopoietic Stem Cell Transplantation (AHSCT) in Multiple Sclerosis Patients. Abstract P477]

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