Longer-term Follow-up of Stem Cell Transplant (SCT) in MS.

TitleLonger-term Follow-up of Stem Cell Transplant (SCT) in MS.
Publication TypeJournal Article
AuthorsKraft GH, Bowen JD, Cui J Y, McSweeney PA, Sullivan KM, Pavletic S, Maravilla KR, Al-Omaishi J, Corboy JR, Derrington D, DiPersio J, Georges GE, Holmberg LA, LeMaistre FC, Openshaw H, Ryan K, Sunderhaus J, Storek J, Zunt JR, Nash RA
JournalInternational Journal of MS Care

One year ago, we presented our early experience with High-Dose Immunosuppressive Therapy (HDIT) and SCT rescue for treatment of severe, high-risk MS patients. This presentation will evaluate the longer-term (up to three years) data from those subjects, along with shorter-term results of additional patients. Autologous peripheral blood stem cells were mobilized with G-CSF (16 µg/kg/day) and CD34 selected. HDIT consisted of TBI (800 cGy), cyclophosphamide (120 mg/kg) and ATGAM (90 mg/kg). Eligibility included an Extended Disability Status Scale (EDSS) score from 5.0 to 8.0 and an increase of 1.0 or more points in previous year. Twenty-one patients had failed previous therapy with interferon beta and fifteen had failed multiple therapies including Copaxone, prednisone, or methotrexate in addition to interferon. Twenty-six patients (secondary progressive = 18, primary progressive = 7, relapsing-remitting = 1), median age 41 (27–60) years were enrolled. Median EDSS at HDIT was 7.0 (5.0–8.0). Median follow-up was 12 (3-36) months. The median number of apheresis procedures was 3 (2-5; a back-up autologous graft was collected). One of the 26 patients died (day +53). She received rabbit ATG instead of ATGAM and developed CMV disease and EBV-PTLD. With current follow-up, the majority of patients remain neurologically stable with unchanged EDSS. Five patients have had an increase in EDSS of at least 1.0 point. Only two patients had enhancing lesions on brain MRI at one year after SCT. No patient has required further treatment with interferon beta or Copaxone after SCT.


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