Autologous Peripheral Blood Stem Cell Transplant for Neurologic Autoimmune Diseases

  • End date
    Jun 30, 2033
  • participants needed
  • sponsor
    Fred Hutchinson Cancer Center
Updated on 11 July 2022
blood stem cell transplant
white blood cell count
antithymocyte globulin
white blood cells
biomarker analysis
spinal cord
autoimmune disease
cell counts
gait ataxia
peripheral neuropathy
encephalitis autoimmune
multiple sclerosis
neuromyelitis optica
stiff person syndrome
syngeneic bone marrow transplantation
cerebellar degeneration
variable number tandem repeat
hematopoietic stem cell transplantation
abo typing
spinal cord disease


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 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.

Condition Autoimmune Disease, Neurologic Autoimmune Disease, Autologous Transplant Autoimmune, Multiple Sclerosis Transplant, MS Stem Cell Transplant, Multiple Sclerosis Stem Cell Transplant, Stiff Person Syndrome, HCT for Neurologic Autoimmune Disorders, CIDP Transplant, Myasthenia Gravis Transplant, Autoimmune Nervous System Disorder, Central Nervous System Vasculitis, Cerebellar Degeneration, Chronic Inflammatory Demyelinating Polyneuropathy, Lambert Eaton Myasthenic Syndrome, Myasthenia Gravis, Neuromyelitis Optica, Opsoclonus Myoclonus Syndrome, Rasmussen Subacute Encephalitis
Treatment anti-thymocyte globulin, laboratory biomarker analysis, cytarabine, etoposide, prednisone, melphalan, autologous hematopoietic stem cell transplantation, peripheral blood stem cell transplantation, carmustine, Syngeneic Bone Marrow Transplantation
Clinical Study IdentifierNCT00716066
SponsorFred Hutchinson Cancer Center
Last Modified on11 July 2022


Yes No Not Sure

Inclusion Criteria

Patients with an autoimmune disorder of the central or peripheral nervous system will be eligible; this will include
Primary Central Nervous System (CNS) vasculitis
Rasmussen's encephalitis
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
Myasthenia Gravis
Lambert-Eaton myasthenic syndrome
Human T-cell lymphotropic virus (HTLV)-1-associated myelopathy (HAM) / tropical spastic paraparesis (TSP)
Opsoclonus/myoclonus (anti-Ri)
Neuromyelitis optica
Multiple sclerosis
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
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)

Exclusion Criteria

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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