High-Dose Immunosuppressive Therapy Using Carmustine, Etoposide, Cytarabine, and Melphalan (BEAM) + Thymoglobulin Followed by Syngeneic or Autologous Hematopoietic Cell Transplantation for Patients With Autoimmune Neurologic Diseases
I. Evaluate the safety of high-dose carmustine, etoposide, Ara-c (cytarabine) and melphalan
(BEAM) and Thymoglobulin (antithymocyte globulin) as a high-dose immunosuppressive treatment
(HDIT) regimen in patients with severe, refractory neurological autoimmune disease.
I. Evaluate disease responses and the duration of response to HDIT and autologous
hematopoietic stem cell transplantation (HSCT).
II. Determine the efficacy and safety of G-CSF (filgrastim) and prednisone or
cyclophosphamide for hematopoietic stem mobilization in patients with neurological
Patients receive carmustine intravenously (IV) on day -6, etoposide IV and cytarabine IV
twice daily (BID) on days -5 to -2, melphalan IV on day -1, and antithymocyte globulin IV on
days -2 and -1. Patients then undergo autologous or syngeneic peripheral blood stem cell
transplant on day 0. Patients also receive prednisone orally (PO) once daily (QD) on days
7-21, followed by 2 week taper.
After completion of study treatment, patients are followed up at 3 months, 1 year, and then
annually thereafter for up to 5 years.
Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Incidence of grades 4-5 regimen-related toxicity as assessed by the Regimen Related Toxicity Scale
Using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v.3.0. The development of a grade 4 to 5 toxicity of any of the included major organ systems within the first 28 days after transplant will be defined as regimen-related toxicity.
Within 28 days post-transplant
Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium
United States: Institutional Review Board
|Fred Hutchinson Cancer Research Center||Seattle, Washington 98109|
|Swedish Neuroscience Institute||Seattle, Washington 98122|
|Colorado Blood Cancer Institute||Denver, Colorado 80218|