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Osipova 1 et al. (Abstract)

Cellular Therapy and Transplantation (CTT), Vol. 3, No. 9
doi: 10.3205/ctt-2010-No9-abstract26
© The Authors. This abstract is provided under the following license: Creative Commons Attribution 3.0 Unported

Abstract accepted for "4th Raisa Gorbacheva Memorial Meeting on Hematopoietic Stem Cell Transplantation",
Saint Petersburg, Russia, September 18–20, 2010

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Risk assessment of high-dose immunosuppressive therapy (HDIT) with hematopoietic stem cell transplantation (HSCT) in patients with multiple sclerosis (MS): A 10-year single center experience

Nina E. Osipova, Denis T. Muhamedzhanov, Vladimir N. Vavilov, Elena V. Babenko, Maria A. Estrina, Elena I. Darskaya, Boris V. Afanasyev

R.M. Gorbacheva Memorial Institute of Children Hematology and Transplantation, Pavlov State Medical University, Saint-Petersburg, Russia

Correspondence: Nina E. Osipova, R.M. Gorbacheva Memorial Institute of Children Hematology and Transplantation, Saint-Petersburg Pavlov State Medical University, 6/8, Tolstoy str., Saint-Petersburg, 197022, Russia, E-mail: ninamd13@spam is badmail.ru

Abstract

Purpose: To assess the risk factors of HDIT with HSCT in patients (pts) with MS.

Patients and methods: In our clinic we have performed 23 HSCT on MS pts since October 2000. There were 5 pts with primary progressive MS, 12 pts with secondary progressive MS, and 6 pts with a relapsing-remitting course of MS. The median age was 34.5 yrs (22–55 yrs); 11 pts were male and 12, female. The median follow-up was 4.5 yrs (1.5yrs–9 yrs 7 months). The median EDSS at the time of treatment was 5.7 (1.5–7.5), and at the last follow-up 5.4 (1.0–8.0). The majority of patients had had 2 or 3 previous lines of immunosuppressive and immunomodulation therapy. The median time from disease onset until HDIT was 6.8 yrs. We used myeloablative (BEAM) conditioning in 17 cases and a non-myeloablative regimen (fludarabine + melphalan) in 6 cases. In vivo T cell depletion with ATG 5 mg/kg on days +1 and +2 was performed in all patients.

Results: The mean agranulocytosis duration was 13 d. The main complications in the early post-transplant period were infections (19 pts), hemorrhage (12 pts), neurologic deterioration (7 pts), and serum sickness (10 pts). One patient died on day +8 from sepsis with multiple organ failure. In the early post-transplant period (D+100) general response (improvement + stabilization) was 81%.

At the last follow-up (June 2010), the general response was 62%. One pt died 2 years after HDIT in an accident (not responding to treatment).

There were no statistically valid differences between disease outcome and gender, age, course of MS, and conditioning regimen. The EDSS at HSCT (≤6.5) and the disease duration at the time of HSCT (≤3.5 yrs) were statistically significant factors.

Conclusions: HDIT with HSCT in MS is feasible procedure and a significant majority of pts may at least stabilize their disability. The risk of the procedure can be minimized by detailed individual risk assessment before the inclusion.

Keywords: multiple sclerosis, HDIT, HSCT, risk factors

 

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