bNot sure if this was posted here, but thought it should be.
(you have to register to search abstracts - its free to register, then search on natalizumab.)
Presented at the 56th AAN Annual Meeting in San Francisco:
1. [P06.082] Single-Patient Study for the Emergency Use of Natalizumab (Antegran) in the Treatment of Pediatric Multiple Sclerosis
Authors: Shehzad Choudry, Shane Maxwell, Douglas R. Jeffery, Wisnton-Salem, NC, Michael Panzara, Boston, MA, E. Steve Roach, Winston-Salem, NC
Date/Time: Thursday, April 29, 2004 - 3:00 PM
Session Info: Poster: Multiple Sclerosis: Therapeutics I (3:00 PM - 7:00 PM)
[P06.082] Single-Patient Study for the Emergency Use of Natalizumab (Antegran) in the Treatment of Pediatric Multiple Sclerosis
Shehzad Choudry, Shane Maxwell, Douglas R. Jeffery, Wisnton-Salem, NC, Michael Panzara, Boston, MA, E. Steve Roach, Winston-Salem, NC
OBJECTIVE: To assess the safety and efficacy of monthly IV doses of natalizumab (Antegran) administered to a 5-year old patient with aggressive multiple sclerosis (MS)
BACKGROUND: Pediatric MS is uncommon and there have been no controlled trials of standard immunomodulatory therapy. The present report describes a 5 year old patient with onset at 18 months treated with natalizumab (Antegran) as rescue therapy. Antegran is a humanized monoclonal IgG4 antibody to the a4 integrin. The patient had an uncomplicated aseptic meningitis at 15 months. Three months later she developed a right hemiparesis and was diagnosed with acute disseminated encephalomyelitis. MRI scan showed multiple white matter lesions. Three months later she suffered a partial transverse myelitis and her MRI scan showed more new lesions. She then developed ataxia and subsequently a bilateral optic neuritis accompanied by further changes on MRI. Oligoclonal bands were negative but extensive evaluation failed to reveal a more likely diagnosis and the diagnosis of MS was later confirmed by brain biopsy. She was started on interferon beta-1a and titrated up to 21 mcg IM twice weekly. Despite treatment she continued to show evidence of aggressive inflammatory disease and required frequent IV methylprednisolone (MP) and eventually cyclophosphamide. She tolerated treatment poorly and progressed despite therapy. She suffered a severe right optic neuritis with complete loss of vision in her right eye and a cervical transverse myelitis. She was treated with IV MP and plasma exchange with only minimal recovery. At this point she remained quadriparetic and blind from her MS. She began treatment with Antegran on an emergency basis approved by the FDA and the IRB.
DESIGN/METHODS: Given her age and weight, she was initially dosed at 3 mg/kg monthly for four months and later at 6 mg/kg to achieve adequate drug serum concentrations. Therapeutic effect was assessed by MRI, relapse frequency, and EDSS. Safety assessments included exams, vital signs, adverse event monitoring, and laboratories as well as monitoring pharmacokinetics.
RESULTS: Following 29 weeks of therapy she has continued to improve clinically and is now fully ambulatory. Vision remains severely impaired with only partial recovery of vision in her right eye. MRI scans have shown a marked reduction in gadolinium enhancement and in new lesion formation with minimal disease activity. She developed an apparent IFN related hepatitis resulting in the cessation of IFN therapy. No elevation of liver function tests or other drug related adverse events has been apparent on Antegran alone.
CONCLUSIONS: The use of Antegran has been safe and effective for 29 weeks in a five year old patient with aggressive MS who failed to respond to standard immunomodulating therapy. Supported by: Biogen, Idec, and Elan Pharmaceuticals.
Category - MS and Related Diseases
SubCategory - Therapeutics
Thursday, April 29, 2004 3:00 PM