This phase II trial studies the side effects and how well carmustine, etoposide, cytarabine and melphalan together with antithymocyte globulin before a stem cell transplant works in treating patients with autoimmune neurologic disease that did not respond to previous therapy. In autoimmune neurological diseases, the patient's own immune system 'attacks' the nervous system which might include the brain/spinal cord and/or the peripheral nerves. Giving high-dose chemotherapy, including carmustine, etoposide, cytarabine, melphalan, and antithymocyte globulin, before a stem cell transplant weakens the immune system and may help stop the immune system from 'attacking' a patient's nervous system. When the patient's own (autologous) stem cells are infused into the patient they help the bone marrow make red blood cells, white blood cells, and platelets so the blood counts can improve.
OUTLINE: 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 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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
53
Given IV
Undergo autologous or syngeneic stem cell transplantation
Given IV
Given IV
Given IV
Correlative studies
Given IV
Undergo autologous or syngeneic stem cell transplantation
Given PO
Undergo syngeneic bone marrow transplantation
Colorado Blood Cancer Institute
Denver, Colorado, United States
Fred Hutch/University of Washington Cancer Consortium
Seattle, Washington, United States
Swedish Medical Center-First Hill
Seattle, Washington, United States
Incidence of grades 4-5 regimen-related toxicity
Assessed by the Regimen Related Toxicity Scale. Using the National Cancer Institute Common Terminology Criteria for Adverse Events version 3.0. The development of a grade 4 to 5 toxicity of any of the included major organ systems within the first 365 days after transplant will be defined as regimen-related toxicity.
Time frame: Up to 1 year post-transplant
Transplant-related mortality
Defined as death within the first 100 days of transplant due to transplant-related complications.
Time frame: Within 100 days post-transplant
Disease responses
Assessed by clinical, laboratory and radiologic evaluation
Time frame: Up to 5 years
Engraftment kinetics
Monitored for engraftment kinetics of granulocytes, platelets and red cells post-transplant.
Time frame: Over first 60 days post-transplant
Number of subjects achieving greater than or equal to 4.0 x 10^6 CD34+ cells/kg, after up to two peripheral blood stem cell mobilizations
Efficacy of peripheral blood stem cell mobilization as evaluated by total number of harvested CD34+cells/kg, for autologous transplant.
Time frame: Baseline to post mobilization, assessed up to 20 days after starting final mobilization (up to two mobilizations)
Number of subjects with an exacerbation of autoimmune disease symptoms secondary to G-CSF (filgrastim) during peripheral blood stem cell mobilization
Subjects are evaluated by standardized clinical neurologic tests specific to autoimmune disease type.
Time frame: Baseline to post mobilization, assessed up to 20 days after starting final mobilization (up to two mobilizations)
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