Treatment of Severe Multiple Sclerosis (MS) with High Dose Immunosuppressive Therapy (HDIT) and Autologous Stem Cell Transplantation (SCT).

TitleTreatment of Severe Multiple Sclerosis (MS) with High Dose Immunosuppressive Therapy (HDIT) and Autologous Stem Cell Transplantation (SCT).
Publication TypeJournal Article
AuthorsNash RA, Kraft GH, Bowen JD, Pavletic S, Al-Omaishi G, McSweeney PA, Corboy JR, Openshaw H, Storek J, Holmberg LA, Zunt JR, Prather K, Ryan K, Sullivan KM
JournalMultiple Sclerosis

Objectives: To evaluate the safety and therapeutic efficacy of HDIT and autologous CD341-selected SCT for severe multiple sclerosis. Methods: Autologous peripheral blood stem cells (PBSC) were mobilized with G-CSF (16 mg/kg/day) and CD34 selected. HDIT consisted of total body irradiation (800 cGy), cyclophosphamide (120 mg/kg) and horse anti thymocyte globulin (ATG) (90 mg/kg). Eligibility required that patients have an EDSS from 5.0 to 8.0, and deterioration of one or more points over the previous year. Results: Twenty patients with secondary progressive (15), primary progressive (4), or relapsing-remitting (1) with a median age of 43 (range, 27–60) years were enrolled to the study. The median EDSS at HDIT was 7.0 (range, 5.0–8.0). The median follow-up has been 5 (range, 1–24) months. Patient 4 had an MS flare during administration of G-CSF for mobilization. Patient 9, the only patient who received rabbit ATG because of a positive skin test to horse ATG, developed an EBV-associated post-transplant lymphoproliferative disorder and died at day 53. Patient 16 had sustained fevers and had an increase in the baseline EDSS of 7.5 to 8.5 at 3 months. Thirteen patients were considered evaluable at 3 or more months after transplant. At last evaluation, 2 patients had an increase of EDSS of at least 0.5 points, 4 patients had a decrease in EDSS of at least 0.5 points and 7 patients have remained stable. Importantly, no new or enhancing lesions have been observed on MRI of the brain except for patient 4 with the flare of MS. Conclusions: Toxicities from HDIT to date have been manageable and transient. We have added prednisone to the mobilization regimen to prevent G-CSF related neurological toxicity and prohibited the use of rabbit ATG. Further follow-up is required to fully assess outcome.

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