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Abstract

Objective: To review our multicenter experience with cyclophosphamide in the treatment of children with multiple sclerosis (MS).
Methods: Retrospective chart review of children with MS treated with cyclophosphamide. Demographic, clinical, treatment, and MRI parameters were collected.
Results: We identified 17 children with MS treated with cyclophosphamide. All but one had worsening of Expanded Disability Status Scale scores or multiple relapses prior to treatment initiation. Children were treated with one of three regimens: 1) induction therapy alone; 2) induction therapy with pulse maintenance therapy; or 3) pulse maintenance therapy alone. Treatment resulted in a reduction in relapse rate and stabilization of disability scores assessed 1 year after treatment initiation in the majority of patients. Longer follow-up was available for most cases. Cyclophosphamide was well tolerated in most patients. However, side effects included vomiting, transient alopecia, osteoporosis, and amenorrhea. One patient developed bladder carcinoma that was successfully treated.
Conclusions: Cyclophosphamide is an option for the treatment of children with aggressive multiple sclerosis refractory to first-line therapies. Recommendations regarding patient selection, treatment administration, and monitoring are discussed.

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Published In

Neurology®
Volume 72Number 24June 16, 2009
Pages: 2076-2082
PubMed: 19439723

Publication History

Published online: May 13, 2009
Published in print: June 16, 2009

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Authors

Affiliations & Disclosures

N. Makhani, MD
From The Hospital for Sick Children (N.M., H.M.B., B.L.B.), University of Toronto, Canada; Partners Pediatric Multiple Sclerosis Center (M.P.G., T.C.), Massachusetts General Hospital for Children, Boston; Children's Hospital (M.P.G.), Boston; and Partners Multiple Sclerosis Center (L.S., T.C.), Brigham and Women's Hospital, Boston, MA.
M. P. Gorman, MD
From The Hospital for Sick Children (N.M., H.M.B., B.L.B.), University of Toronto, Canada; Partners Pediatric Multiple Sclerosis Center (M.P.G., T.C.), Massachusetts General Hospital for Children, Boston; Children's Hospital (M.P.G.), Boston; and Partners Multiple Sclerosis Center (L.S., T.C.), Brigham and Women's Hospital, Boston, MA.
H. M. Branson, MD
From The Hospital for Sick Children (N.M., H.M.B., B.L.B.), University of Toronto, Canada; Partners Pediatric Multiple Sclerosis Center (M.P.G., T.C.), Massachusetts General Hospital for Children, Boston; Children's Hospital (M.P.G.), Boston; and Partners Multiple Sclerosis Center (L.S., T.C.), Brigham and Women's Hospital, Boston, MA.
L. Stazzone, NP
From The Hospital for Sick Children (N.M., H.M.B., B.L.B.), University of Toronto, Canada; Partners Pediatric Multiple Sclerosis Center (M.P.G., T.C.), Massachusetts General Hospital for Children, Boston; Children's Hospital (M.P.G.), Boston; and Partners Multiple Sclerosis Center (L.S., T.C.), Brigham and Women's Hospital, Boston, MA.
B. L. Banwell, MD
From The Hospital for Sick Children (N.M., H.M.B., B.L.B.), University of Toronto, Canada; Partners Pediatric Multiple Sclerosis Center (M.P.G., T.C.), Massachusetts General Hospital for Children, Boston; Children's Hospital (M.P.G.), Boston; and Partners Multiple Sclerosis Center (L.S., T.C.), Brigham and Women's Hospital, Boston, MA.
T. Chitnis, MD
From The Hospital for Sick Children (N.M., H.M.B., B.L.B.), University of Toronto, Canada; Partners Pediatric Multiple Sclerosis Center (M.P.G., T.C.), Massachusetts General Hospital for Children, Boston; Children's Hospital (M.P.G.), Boston; and Partners Multiple Sclerosis Center (L.S., T.C.), Brigham and Women's Hospital, Boston, MA.

Notes

Address correspondence and reprint requests to Dr. Tanuja Chitnis, Partners Pediatric Multiple Sclerosis Center, Massachusetts General Hospital, ACC-708, 55 Fruit Street, Boston, MA 02114 [email protected].

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