MADRID — High-dose immunosuppressive therapy with autologous hematopoietic stem cell transplant may be a promising new treatment option for multiple sclerosis, based on results that were obtained in a series of 56 Russian patients.
The treatment combination improved or stabilized the condition in 92% of the patients and resulted in long-term, progression-free survival in 83%, lead investigator Dr. Yury Shevchenko reported at the annual congress of the European Federation of Neurological Societies.
He presented full data for 48 patients, all of whom had 6–30 months of follow-up (mean of 19 months).
The patients' mean age was 32 years and their mean disease duration was 7 years. Of the 56 patients, 27 had secondary progressive disease, 10 had primary progressive, 18 had relapsing-remitting disease, and 1 had progressive-relapsing disease. Their mean EDSS (Expanded Disability Status Scale) score was 6.
All patients underwent the BEAM conditioning regimen before surgery. This regimen consisted of high-dose BCNU (carmustine) at 300 mg/m2 on day 6 before the stem cell transplant; etoposide at 200 mg/m2, cytarabine at 200 mg/m2 from day 5 to day 2 before transplant, and melphalan 140 mg/m2 on the day before transplant (Exp. Hematol. 2008;36:922–8).
Patients tolerated the conditioning and transplant very well, said Dr. Shevchenko of the Pirogov National Medical Surgical Center, Moscow.
Neutropenic fever occurred in 52%; thrombocytopenia in 50%; transient increases in liver enzymes in 46%; and enteritis in 18%. There were no transplant-related deaths.
Clinical improvement (defined as a decrease on the EDSS score of at least 0.5 points) occurred in 27 patients, and stabilization occurred in 17 patients, Dr. Shevchenko said. The published study noted that nine patients had “dramatic improvement.”
One patient with secondary progressive disease and another patient with relapsing-remitting disease worsened within 6 months of the transplant but then stabilized during follow-up.
MR imaging results were available for 37 patients. Of these, 16 had active lesions at baseline, and all of these became inactive during follow-up. Of the 21 patients without active lesions at baseline, 20 remained inactive and one developed an active lesion.
There was one death, the paper noted. This patient, with secondary progressive disease, deteriorated 12 months after transplantation. About 4 years after the procedure, she developed acute promyelocytic leukemia and died of a cerebral hemorrhage.