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High-dose Cyclophosphamide for Moderate to Severe Refractory Multiple Sclerosis: 2-Year Follow-up (Investigational New Drug No. 65863).
MedLine Citation:
PMID:  19770795     Owner:  NLM     Status:  In-Data-Review    
High-dose cyclophosphamide (HDC) is a chemotherapy treatment designed to eradicate autoreative B- and T-cells responsible for lymphocyte-mediated autoimmune illness while sparing the pluripotent blood stem cell of any ill effects. Multiple sclerosis (MS) is the most common inflammatory and demyelinating immune-mediated disorder of the central nervous system in young adults. Patients with moderate to severe, refractory MS, defined as an Expanded Disability Status Scale (EDSS) score of 3.5 or higher after two or more Food and Drug Administration-approved disease-modifying agents, received 200 mg/kg of cyclophosphamide over 4 days. For the next 2 years, quarterly EDSS score evaluations and biannual brain magnetic resonance imaging and neuro-ophthalmologic evaluations were obtained. Fifteen patients were evaluated for clinical response. During follow-up, one patient increased their baseline EDSS score by 1.0. EDSS score stability of decrease was realized in five of seven (71%) patients with relapsing-remitting MS and six of eight (75%) patients with secondary progressive MS. Four patients required additional immunomodulatory treatment after treatment. Treatment response was seen regardless of the baseline presence or absence of contrast lesion activity. HDC can effectively decrease symptoms, stop disease progression, and allow for disability regression in relapsing-remitting MS and secondary progressive MS patients. The most appropriate candidates for HDC, its duration of benefit, and the potential need for prophylactic preventative immune manipulation after HDC all require further investigation.
Douglas E Gladstone; Robert Peyster; Edward Baron; Sharon Friedman-Urevich; Patrick Sibony; Patricia Melville; Malcolm Gottesman
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Publication Detail:
Type:  Journal Article    
Journal Detail:
Title:  American journal of therapeutics     Volume:  18     ISSN:  1536-3686     ISO Abbreviation:  Am J Ther     Publication Date:  2011 Jan 
Date Detail:
Created Date:  2012-05-21     Completed Date:  -     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  9441347     Medline TA:  Am J Ther     Country:  United States    
Other Details:
Languages:  eng     Pagination:  23-30     Citation Subset:  IM    
1Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University, Baltimore, MD; Departments of 2Radiology, 3Ophthalmology, and 4Neurology, State University of New York Medical Center at Stony Brook, Stony Brook, NY; 5Department of Ophthalmology, Good Samaritan Hospital, Bay Shore, NY; and 6Department of Neurology, Winthrop University Hospital, Mineola, NY.
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