title: High-dose cyclophosphamide in the treatment of multiple sclerosis.
abstract: High dose cyclophosphamide (HDC) has been successfully used for the treatment of a variety of autoimmune diseases. In this study, we sought to determine whether the use of high dose cyclophosphamide provided stabilization of relapsing remitting MS (RRMS), secondary progressive MS (SPMS), or primary progressive MS (PPMS). The parameters evaluated were EDSS scores, lesion load and brain volumes by MRI and frequency of relapses. Twenty-three patients underwent immunoablative therapy with HDC and were followed for 3.5 years. Nine were relapsing remitting (RRMS), 11 secondary progressive (SPMS), and 3 primary progressive (PPMS). Four of 9 RRMS have had no clinical progression up to 3.5 years following treatment. Three of 9 patients maintained a normal neurologic examination with improved EDSS scores. Seven of the nine RRMS patients had reduction in flare frequency which was maintained for 3.5 years following treatment or no immunomodulating agents. Subgroup analysis in the RRMS patients of lesion load and brain parenchymal volume revealed a favorable trend in these parameters which did not reach statistical significance. The treatment was generally ineffective for SPMS and failed in the 2 PPMS patients. HDC was well tolerated, demonstrated a good safety profile and had minimal adverse effects. These results along with previous reports suggest that early use of HDC therapy in RRMS is promising.
authors.affiliation: Drexel University College of Medicine-Neurology, Philadelphia, Pennsylvania 19102, USA. Robert.schwartzman@drexelmed.edu
authors.forename: Robert Jay
authors.initials: RJ
authors.lastname: Schwartzman
authors.forename: Nicole
authors.initials: N
authors.lastname: Simpkins
authors.forename: Guillermo M
authors.initials: GM
authors.lastname: Alexander
authors.forename: Erin
authors.initials: E
authors.lastname: Reichenberger
authors.forename: Kristine
authors.initials: K
authors.lastname: Ward
authors.forename: Noah
authors.initials: N
authors.lastname: Lindenberg
authors.forename: David
authors.initials: D
authors.lastname: Topolsky
authors.forename: Pam
authors.initials: P
authors.lastname: Crilley
mesh_terms: D000328:Adult; D000368:Aged; D003520:Cyclophosphamide / Q000008:administration & dosage*; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D008297:Male; D008875:Middle Aged; D009103:Multiple Sclerosis / Q000188:drug therapy* / Q000473:pathology / Q000523:psychology; D011788:Quality of Life / Q000523:psychology; D016896:Treatment Outcome
chemical_list: D003520:Cyclophosphamide