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Abstract Details

Autologous Hematopoietic Stem Cell Transplantation in Neuromyelitis Optica
MS and Related Diseases
P02 - (-)
151
BACKGROUND: Neuromyelitis Optica (NMO) is an immune-mediated neurodegenerative disease of the central nervous system characterized by frequent demyelinating/necrotizing attacks of optic nerves and spinal cord leading to significant morbidity and mortality. Treatments are highly toxic and only minimally effective. Fifty percent of patients are blind in one eye or require a mobility aide within 5 years, with a 5-year survival of 68%. The immunological features of NMO, coupled with its severity make it an ideal candidate for a trial of AHSCT in an effort to achieve remission and freedom from immunosuppressive medications.
DESIGN/METHODS: Patients 18-65 with one relapse in 12 months and two in 24 months despite immunotherapy and EDSS < 6.5 are eligible provided there are no contraindicated medical conditions including pregnancy or previous malignancies. Patients undergo non-ablative stem cell mobilization and infusion using cyclophosphamide, rituximab and ATG. Outcomes including EDSS, NMO antibody titers, optical coherence tomography and MRI are done every 6-12 months for 5 years. Ten subjects are required to provide 80% power for our primary outcome.
RESULTS: Presently, two NMO patients have undergone transplantation. A 28F was transplanted in May 2011 with pre-transplant annualized relapse rate (ARR) of 5 and EDSS 4.5, now 0 and 2.0 respectively. A 36F was transplanted in April 2012 with pre-transplant ARR of 5, now 0. Neither maintenance nor rescue NMO therapy has been required. Updated results will be provided in March 2013.
CONCLUSIONS: Thus far, this transplant regimen is well tolerated with dramatic clinical improvement in the short-term. Additional patients and longer follow-up will determine if these benefits persist.
Authors/Disclosures
Jodie Burton, MD, FAAN
PRESENTER 		Dr. Burton has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Roche. Dr. Burton has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Burton has received personal compensation in the range of $0-$499 for serving as a Consultant for Horizon. The institution of Dr. Burton has received research support from Roy and Joan Allen Professorship for Sight. Dr. Burton has a non-compensated relationship as a Advisor with CADTH that is relevant to AAN interests or activities. Dr. Burton has a non-compensated relationship as a Advisor with Alexion that is relevant to AAN interests or activities. Dr. Burton has a non-compensated relationship as a Advisor with Horizon that is relevant to AAN interests or activities. Dr. Burton has a non-compensated relationship as a 好色先生al Chair with EMD Serono that is relevant to AAN interests or activities.
No disclosure on file
Douglas L. Arnold, MD, FAAN (Montreal Neurological Institute, McGill Univ) Dr. Arnold has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen. Dr. Arnold has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for BMS. Dr. Arnold has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Frequency Therapeutics. Dr. Arnold has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Merck. Dr. Arnold has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Novartis. Dr. Arnold has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Roche. Dr. Arnold has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Sanofi. Dr. Arnold has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Shionogi. Dr. Arnold has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Xfacto communications. Dr. Arnold has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Eli Lilly. Dr. Arnold has received personal compensation in the range of $500-$4,999 for serving as a Consultant for EMD Serono. Dr. Arnold has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biohaven. Dr. Arnold has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Find therapeutics. Dr. Arnold has received personal compensation in the range of $500-$4,999 for serving as a Consultant for GSK. Dr. Arnold has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Idorsia. Dr. Arnold has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Kiniksa. Dr. Arnold has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Clario.
Fiona E. Costello, MD (Fiona Costello Professional Corporation) Dr. Costello has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Dr. Costello has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Roche. Dr. Costello has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Costello has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion. Dr. Costello has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Accure Therapeutics. Dr. Costello has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for the Sumaira Foundation.
No disclosure on file
Luanne Metz, MD (Foothills Hospital) The institution of Dr. Metz has received research support from Multiple Sclerosis Society of Canada. The institution of Dr. Metz has received research support from Calgary Health Trust. The institution of Dr. Metz has received research support from Government of Alberta.