Multiple Sclerosis Clinical Trial
Autologous Stem Cell Transplant for Neurologic Autoimmune Diseases
This phase II trial studies the side effects and how well carmustine, etoposide, cytarabine and melphalan together with antithymocyte globulin before a peripheral blood 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 peripheral blood 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.
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.
Patients with an autoimmune disorder of the central or peripheral nervous system will be eligible; this will include:
Primary Central Nervous System (CNS) vasculitis
Autoimmune peripheral neuropathy (anti-Hu [Anna-1], anti-GM1 [GD1b], anti-MAG, anti-ganglioside, anti-sulfatide)
Autoimmune cerebellar degeneration
Gait Ataxia with Late age Onset Polyneuropathy (GALOP)
Stiff Person Syndrome
Chronic Inflammatory Demyelinating Polyneuropathy
Lambert-Eaton myasthenic syndrome
Human T-cell lymphotropic virus (HTLV)-1-associated myelopathy (HAM) / tropical spastic paraparesis (TSP)
Other central or peripheral nervous system autoimmune diseases as approved by study neurologists and the Fred Hutchinson Cancer Research Center (FHCRC) faculty at Patient Care Conference (PCC)
Patients must satisfy the criteria for a diagnosis of one of the severe neurological autoimmune disorders outlined
Patients age =< 70 years
Evidence of disease activity as outlined (e.g. gadolinium enhancement on magnetic resonance imaging of the brain or clinical progression)
Patients must have failed at least 2 lines of standard therapy as outlined for the specific diseases
DONOR: Sibling of any patient enrolled on this protocol proven by ABO typing, human leukocyte antigen (HLA) typing and variable number tandem repeat (VNTR) analysis to be syngeneic with the patient (e.g. identical twin)
DONOR: Willing to undergo multiple apheresis procedures (except donors < 12 years who will undergo bone marrow harvests)
Age >= 71 years
Pregnancy or expressed plans to become pregnant within 1 year of the procedure
Patients who are serologically positive for human immunodeficiency virus (HIV)
Patients with pulmonary, cardiac, hepatic or renal impairment that would limit their ability to receive cytoreductive therapy and compromise their survival; this should include patients with any of the following:
Severe pulmonary dysfunction associated with a carbon monoxide diffusing capacity (DLCO) (corrected for hemoglobin) < 60%, or requires supplemental oxygen; patients who are unable to perform pulmonary function test (because of underlying disease) will be excluded if the oxygen saturation is < 92% on room air
Uncontrolled malignant arrhythmias, or clinical evidence of congestive heart failure (New York class III-IV) or ejection fraction < 50%
Renal disease with estimated glomerular filtration rate (GFR) by creatinine clearance or iothalamate clearance < 50 ml/min/1.73 m^2 body surface area
Serum glutamate pyruvate transaminase (SGPT)/aspartate aminotransferase (AST) > 3 times normal or direct bilirubin greater than 2.5 mg/dL on two repeated tests
Active uncontrolled infection
Demonstrated lack of compliance with prior medical care
Patients whose life expectancy is limited by illness other than their neurological condition
Patients with evidence of myelodysplasia
Active malignancy (excluding localized squamous cell or basal cell carcinoma of the skin)
DONOR: Inadequate documentation that donor and recipient are syngeneic
DONOR: Donors who do not fulfill criteria as apheresis donors as established by institutional guidelines
DONOR: Concordant for autoimmune neurological disease(s) as determined by neurological evaluation
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There are 3 Locations for this study
Denver Colorado, 80218, United States More Info
Seattle Washington, 98109, United States More Info
Seattle Washington, 98122, United States More Info
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