Eculizumab in AQP4-IgG-positive relapsing neuromyelitis optica spectrum disorders: An open-label pilot study

The Lancet Neurology

Volumn 12, Issue 6, 2013, Pages 554-562

  Pittock, Sean J ^a   Lennon, Vanda A ^a,c   McKeon, Andrew ^a   Mandrekar, Jay ^b   Weinshenker, Brian G ^a   Lucchinetti, Claudia F ^a   O'Toole, Orna ^a   Wingerchuk, Dean M ^d  

^a MAYO CLINIC   (United States)

^b Mayo Clinic   (United States)

^c MAYO GRADUATE SCHOOL   (United States)

^d MAYO CLINIC   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AQUAPORIN 4 ANTIBODY; COMPLEMENT COMPONENT C5; ECULIZUMAB; MENINGOCOCCUS VACCINE;

ADULT; ADVERSE OUTCOME; AGED; ARTICLE; BRAIN FUNCTION; CLINICAL ARTICLE; CONTROLLED STUDY; DRUG EFFECT; DRUG EFFICACY; DRUG SAFETY; DRUG TOLERANCE; DRUG WITHDRAWAL; EXPANDED DISABILITY STATUS SCALE; FOLLOW UP; HUMAN; MENINGOCOCCEMIA; MOBILIZATION; MULTICENTER STUDY; MYELITIS; MYELOOPTIC NEUROPATHY; NEUROLOGIC EXAMINATION; OPTIC NEURITIS; OUTCOME ASSESSMENT; PILOT STUDY; PRIORITY JOURNAL; QUESTIONNAIRE; SCREENING; STERILE MENINGITIS; TREATMENT DURATION; UNITED STATES; VISUAL ACUITY;

ADOLESCENT; ADULT; AGED; ANTIBODIES, MONOCLONAL; ANTIBODIES, MONOCLONAL, HUMANIZED; AQUAPORIN 4; BIOLOGICAL MARKERS; FEMALE; FOLLOW-UP STUDIES; HUMANS; IMMUNOGLOBULIN G; MALE; MIDDLE AGED; NEUROMYELITIS OPTICA; PILOT PROJECTS; PROSPECTIVE STUDIES; RECURRENCE; YOUNG ADULT;

EID: 84877919003     PISSN: 14744422     EISSN: 14744465     Source Type: Journal    
DOI: 10.1016/S1474-4422(13)70076-0     Document Type: Article

References (45)

1. Wingerchuk DM, Lennon VA, Lucchinetti CF, Pittock SJ, Weinshenker BG The spectrum of neuromyelitis optica. Lancet Neurol 2007, 6:805-815.
2. Wingerchuk DM, Hogancamp WF, O'Brien PC, Weinshenker BG The clinical course of neuromyelitis optica (Devic's syndrome). Neurology 1999, 53:1107-1114.
3. Wingerchuk DM, Pittock SJ, Lucchinetti CF, Lennon VA, Weinshenker BG A secondary progressive clinical course is uncommon in neuromyelitis optica. Neurology 2007, 68:603-605.
4. Cross SA, Salomao DR, Parisi JE, et al. Paraneoplastic autoimmune optic neuritis with retinitis defined by CRMP-5-IgG. Ann Neurol 2003, 54:38-50.
5. Keegan BM, Pittock SJ, Lennon VA Autoimmune myelopathy associated with collapsin response-mediator protein-5 immunoglobulin G. Ann Neurol 2008, 63:531-534.
6. Lennon VA, Wingerchuk DM, Kryzer TJ, et al. A serum autoantibody marker of neuromyelitis optica: distinction from multiple sclerosis. Lancet 2004, 364:2106-2112.
7. Lennon VA, Kryzer TJ, Pittock SJ, Verkman AS, Hinson SR IgG marker of optic-spinal multiple sclerosis binds to the aquaporin-4 water channel. J Exp Med 2005, 202:473-477.
8. Roemer SF, Parisi JE, Lennon VA, et al. Pattern-specific loss of aquaporin-4 immunoreactivity distinguishes neuromyelitis optica from multiple sclerosis. Brain 2007, 130:1194-1205.
9. Pittock SJ, Weinshenker BG, Lucchinetti CF, Wingerchuk DM, Corboy JR, Lennon VA Neuromyelitis optica brain lesions localized at sites of high aquaporin 4 expression. Arch Neurol 2006, 63:964-968.
10. Waters PJ, McKeon A, Leite MI, et al. Serologic diagnosis of NMO: a multicenter comparison of aquaporin-4-IgG assays. Neurology 2012, 78:665-671.
11. Palace J, Leite MI, Nairne A, Vincent A Interferon beta treatment in neuromyelitis optica: increase in relapses and aquaporin 4 antibody titers. Arch Neurol 2010, 67:1016-1017.
12. Shimizu J, Hatanaka Y, Hasegawa M, et al. IFNbeta-1b may severely exacerbate Japanese optic-spinal MS in neuromyelitis optica spectrum. Neurology 2010, 75:1423-1427.
13. Tanaka M, Tanaka K, Komori M Interferon-beta(1b) treatment in neuromyelitis optica. Eur Neurol 2009, 62:167-170.
14. Jacob A, Hutchinson M, Elsone L, et al. Does natalizumab therapy worsen neuromyelitis optica?. Neurology 2012, 79:1065-1066.
15. Barnett MH, Prineas JW, Buckland ME, Parratt JD, Pollard JD Massive astrocyte destruction in neuromyelitis optica despite natalizumab therapy. Mult Scler 2012, 18:108-112.
16. Kleiter I, Hellwig K, Berthele A, et al. Failure of natalizumab to prevent relapses in neuromyelitis optica. Arch Neurol 2012, 69:239-245.
17. Min JH, Kim BJ, Lee KH Development of extensive brain lesions following fingolimod (FTY720) treatment in a patient with neuromyelitis optica spectrum disorder. Mult Scler 2012, 18:113-115.
18. Cree BA, Lamb S, Morgan K, Chen A, Waubant E, Genain C An open label study of the effects of rituximab in neuromyelitis optica. Neurology 2005, 64:1270-1272.
19. Jacob A, Weinshenker BG, Violich I, et al. Treatment of neuromyelitis optica with rituximab: retrospective analysis of 25 patients. Arch Neurol 2008, 65:1443-1448.
20. Mandler RN, Ahmed W, Dencoff JE Devic's neuromyelitis optica: a prospective study of seven patients treated with prednisone and azathioprine. Neurology 1998, 51:1219-1220.
21. Costanzi C, Matiello M, Lucchinetti CF, et al. Azathioprine: tolerability, efficacy, and predictors of benefit in neuromyelitis optica. Neurology 2011, 77:659-666.
22. Jacob A, Matiello M, Weinshenker BG, et al. Treatment of neuromyelitis optica with mycophenolate mofetil: retrospective analysis of 24 patients. Arch Neurol 2009, 66:1128-1133.
23. Hinson SR, Pittock SJ, Lucchinetti CF, et al. Pathogenic potential of IgG binding to water channel extracellular domain in neuromyelitis optica. Neurology 2007, 69:2221-2231.
24. Hinson SR, Roemer SF, Lucchinetti CF, et al. Aquaporin-4-binding autoantibodies in patients with neuromyelitis optica impair glutamate transport by down-regulating EAAT2. J Exp Med 2008, 205:2473-2481.
25. Vincent T, Saikali P, Cayrol R, et al. Functional consequences of neuromyelitis optica-IgG astrocyte interactions on blood-brain barrier permeability and granulocyte recruitment. J Immunol 2008, 181:5730-5737.
26. Hinson SR, Romero MF, Popescu BF, et al. Molecular outcomes of neuromyelitis optica (NMO)-IgG binding to aquaporin-4 in astrocytes. Proc Natl Acad Sci USA 2012, 109:1245-1250.
27. Saadoun S, Waters P, Bell BA, Vincent A, Verkman AS, Papadopoulos MC Intra-cerebral injection of neuromyelitis optica immunoglobulin G and human complement produces neuromyelitis optica lesions in mice. Brain 2010, 133:349-361.
28. Rother RP, Rollins SA, Mojcik CF, Brodsky RA, Bell L Discovery and development of the complement inhibitor eculizumab for the treatment of paroxysmal nocturnal hemoglobinuria. Nat Biotechnol 2007, 25:1256-1264.
29. Rollins SA, Sims PJ The complement-inhibitory activity of CD59 resides in its capacity to block incorporation of C9 into membrane C5b-9. J Immunol 1990, 144:3478-3483.
30. Wingerchuk DM, Lennon VA, Pittock SJ, Lucchinetti CF, Weinshenker BG Revised diagnostic criteria for neuromyelitis optica. Neurology 2006, 66:1485-1489.
31. Kurtzke JF Rating neurologic impairment in multiple sclerosis: an expanded disability status scale (EDSS). Neurology 1983, 33:1444-1452.
32. Hauser SL, Dawson DM, Lehrich JR, et al. Intensive immunosuppression in progressive multiple sclerosis. A randomized, three-arm study of high-dose intravenous cyclophosphamide, plasma exchange, and ACTH. N Engl J Med 1983, 308:173-180.
33. Weinshenker BG, O'Brien PC, Petterson TM, et al. A randomized trial of plasma exchange in acute central nervous system inflammatory demyelinating disease. Ann Neurol 1999, 46:878-886.
34. Keegan M, Pineda AA, McClelland RL, Darby CH, Rodriguez M, Weinshenker BG Plasma exchange for severe attacks of CNS demyelination: predictors of response. Neurology 2002, 58:143-146.
35. McKeon A, Fryer JP, Apiwattanakul M, et al. Diagnosis of neuromyelitis spectrum disorders: comparative sensitivities and specificities of immunohistochemical and immunoprecipitation assays. Arch Neurol 2009, 66:1134-1138.
36. Hillmen P, Hall C, Marsh JC, et al. Effect of eculizumab on hemolysis and transfusion requirements in patients with paroxysmal nocturnal hemoglobinuria. N Engl J Med 2004, 350:552-559.
37. Hillmen P, Young NS, Schubert J, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med 2006, 355:1233-1243.
38. Fitzpatrick AM, Mann CA, Barry S, Brennan K, Overell JR, Willison HJ An open label clinical trial of complement inhibition in multifocal motor neuropathy. J Peripher Nerv Syst 2011, 16:84-91.
39. Dmytrijuk A, Robie-Suh K, Cohen MH, Rieves D, Weiss K, Pazdur R FDA report: eculizumab (Soliris) for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Oncologist 2008, 13:993-1000.
40. Eculizumab (Soliris) US Food and Drug Administration, (accessed Oct 15, 2012). http://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ucm273089.htm.
41. Ram S, Lewis LA, Rice PA Infections of people with complement deficiencies and patients who have undergone splenectomy. Clin Microbiol Rev 2010, 23:740-780.
42. Lucchinetti CF, Mandler RN, McGavern D, et al. A role for humoral mechanisms in the pathogenesis of Devic's neuromyelitis optica. Brain 2002, 125:1450-1461.
43. Hinson SR, McKeon A, Fryer JP, Apiwattanakul M, Lennon VA, Pittock SJ Prediction of neuromyelitis optica attack severity by quantitation of complement-mediated injury to aquaporin-4-expressing cells. Arch Neurol 2009, 66:1164-1167.
44. Barnum SR Complement biosynthesis in the central nervous system. Crit Rev Oral Biol Med 1995, 6:132-146.
45. Woodruff TM, Nandakumar KS, Tedesco F Inhibiting the C5-C5a receptor axis. Mol Immunol 2011, 48:1631-1642.