Neuromyelitis Optica (NMO) is a demyelinating and degenerative disorder of the CNS affecting vision and spinal cord function. This disease is rare compared to Multiple Sclerosis (MS), but it is devastating and often leads to accumulating disability with a 5 year-mortality of approximately 30%. Survivors are typically left with severe morbidity secondary to blindness, quadriparesis and respiratory failure. No agent has been found to be highly effective in halting disease activity. Based on recent outcomes of stem cell transplant trials and reports in autoimmune diseases including MS, and based on the mechanisms of NMO, we anticipate that stem cell transplantation may provide lasting disease stability for NMO patients. The hypothesis of the present trial is that autologous hematopoetic stem cell transplantation in patients with NMO will provide lasting benefit in relapse prevention. Specifically, we anticipate a 50% reduction in the proportion of patients experiencing relapse over a three year period. We will be following patients for a total of five years after transplantation.
Patients who are deemed eligible will be enrolled and undergo a two stage transplant process followed by neurological assessments every 6 months for the following 5 years assessing EDSS, visual metrics, MRI, AQP-4 antibodies, MSFC and SF36.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
3
AHSCT Procedure: 1. Mobilization and Harvesting: * Cyclophosphamide * Rituximab * GSCF * Dexamethasone * Apheresis 2. Conditioning and Infusion (3-4 weeks after Mobilization and Harvesting): * Cyclophosphamide * MESNA * Rabbit ATG * Rituximab * Methylprednisolone * Stem Cell infusion * GSCF
Foothills Medical Centre, University of Calgary
Calgary, Alberta, Canada
Proportion relapse-free at three years
The proportion of surviving patients who are relapse-free at three years after transplant
Time frame: 3 years
Proportion relapse-free at five years
The proportion of surviving patients relapse-free at year five
Time frame: 5 years
Relapse count
Number of NMO relapse events
Time frame: Annually over 5 years
Disability progression
Time to progression of EDSS by one step
Time frame: Over 5 years
Retinal nerve fiber layer (RFNL) status
Change in RNFL by optical coherence tomography over trial
Time frame: 5 years
25 foot timed walk test
Change in 25 ft timed walk test over trial
Time frame: 5 years
PASAT
Annual and change from baseline to end of trial in Paced Auditory Serial Addition Test to assess cognitive function.
Time frame: Annually over 5 years
Hospitalization
Number of hospitalizations, days in hospital over trial period
Time frame: Over 5 years
Overall survival
Survival over trial period
Time frame: Over 5 years
Time to next relapse
Time to next relapse after transplant
Time frame: Over 5 years
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.