Assessing risks of multiple sclerosis therapies

Journal of the Neurological Sciences

Volumn 332, Issue 1-2, 2013, Pages 59-65

  Parfenov, Valeriy ^a   Schluep, Myriam ^a   Du Pasquier, Renaud ^a  

^a LAUSANNE UNIVERSITY HOSPITAL   (Switzerland)

Author keywords

Complications; Drug approval; Monitoring; Multiple sclerosis; Safety; Treatment

Indexed keywords

ALEMTUZUMAB; BETA INTERFERON; DACLIZUMAB; FINGOLIMOD; FUMARIC ACID DIMETHYL ESTER; GLATIRAMER; LEFLUNOMIDE; MITOXANTRONE; NATALIZUMAB; OCRELIZUMAB; PLACEBO; TERIFLUNOMIDE;

ALLERGIC REACTION; ANXIETY; ARTICLE; CARDIOTOXICITY; CHEST TIGHTNESS; DEPRESSION; DRUG POTENTIATION; DYSPNEA; FLU LIKE SYNDROME; FLUSHING; HEART PALPITATION; HERPES SIMPLEX; HUMAN; HYPERTRANSAMINASEMIA; INJECTION SITE REACTION; LEUKOPENIA; LYMPHOCYTOPENIA; MULTIPLE SCLEROSIS; PATIENT MONITORING; PATIENT SAFETY; PHASE 3 CLINICAL TRIAL (TOPIC); PNEUMONIA; PRIORITY JOURNAL; PROGRESSIVE MULTIFOCAL LEUKOENCEPHALOPATHY; RHEUMATOID ARTHRITIS; RISK ASSESSMENT; RISK MANAGEMENT; SIDE EFFECT; URINARY TRACT INFECTION;

COMPLICATIONS; DRUG APPROVAL; MONITORING; MULTIPLE SCLEROSIS; SAFETY; TREATMENT;

HUMANS; IMMUNOLOGIC FACTORS; MULTIPLE SCLEROSIS; RISK ASSESSMENT;

EID: 84881476702     PISSN: 0022510X     EISSN: 18785883     Source Type: Journal    
DOI: 10.1016/j.jns.2013.06.013     Document Type: Article

References (94)

1. H.G. Eichler, E. Abadie, J.M. Raine, and T. Salmonson Safe drugs and the cost of good intentions NEJM 360 14 2009 1378 1380
2. E.U. Walther, and R. Hohlfeld Multiple sclerosis: side effects of interferon beta therapy and their management Neurology 53 8 Nov 10 1999 1622 1627
3. The IFNb Multiple Sclerosis Study Group Interferon beta-1bis effective in relapsing-remitting multiple sclerosis. I. Clinical results of a multicenter, randomized, double-blind, placebo controlled trial Neurology 43 1993 655 661
4. The IFNb Multiple Sclerosis Study Group and the UBC MS/MRI Analysis Group Interferon beta-1b in the treatment of multiple sclerosis: final outcome of the randomized controlled trial Neurology 45 1995 1277 1285
5. D.W. Paty, and D.K.B. Li The UBC MS/MRI Study Group, the IFNB Multiple Sclerosis Study Group. Interferon beta-1b is effective in relapsing-remitting multiple sclerosis. II. MRI analysis results of a multicenter, randomized, double-blind, placebo controlled trial Neurology 43 1993 662 667
6. L.D. Jacobs, D.L. Cookfair, and R.A. Rudick Intramuscular interferon beta-1a for disease progression in relapsing multiple sclerosis Ann Neurol 39 1996 285 294
7. PRISMS (Prevention of Relapses, Disability by Interferon b-1a Subcutaneously in Multiple Sclerosis) Study Group Randomised double-blind placebo-controlled study of interferonb-1a in relapsing/remitting multiple sclerosis Lancet 352 1998 1498 1504
8. European Study Group on Interferon b-1b in Secondary Progressive MS Placebo-controlled multicentre randomised trial of interferon b-1b in treatment of secondary progressive multiple sclerosis Lancet 352 1998 1491 1497
9. A.T. Reder, and G.C. Ebers Cross-sectional study assessing long-term safety of interferon-beta-1b for relapsing-remitting MS Neurology 74 23 Jun 8 2010 1877 1885
10. H.L. Tremlett, and J. Oger Elevated aminotransferases during treatment with interferon-beta for multiple sclerosis: actions and outcomes Mult Scler 10 2004 298 301
11. E.M. Yoshida, S.L. Rasmussen, and U.P. Steinbrecher Fulminant liver failure during interferon beta treatment of multiple sclerosis Neurology 56 2001 1416
12. Mathew Pulicken, Ayman Koteish, and Karen DeBusk Unmasking of autoimmune hepatitis in a patient with MS following interferon beta therapy Neurology 66 2006 1954
13. A.T. Reder, G.C. Ebers, and A. Traboulsee Cross-sectional study assessing long-term safety of interferon-b-1b for relapsing-remitting MS NEJM 360 14 2009 1378 1380
14. Y.D. Fragoso, and E.R. Frota Severe depression, suicide attempts, and ideation during the use of interferon beta by patients with multiple sclerosis Clin Neuropharmacol 33 6 Nov-Dec 2010 312 316
15. C. Borras, J. Rio, J. Porcel, M. Barrios, M. Tintore, and X. Montalban Emotional state of patients with relapsing-remitting MS treated with interferon beta-1b Neurology 52 1999 1636 1639
16. A. Boster, M.P. Bartoszek, C. O'Connell, D. Pitt, and M. Racke Efficacy, safety, and cost-effectiveness of glatiramer acetate in the treatment of relapsing-remitting multiple sclerosis Ther Adv Neurol Disord 4 5 Sep 2011 319 332
17. B.C. Kieseier, and O. Stuve A critical appraisal of treatment decisions in multiple sclerosis-old versus new Nat Rev Neurol 7 2011 255 262
18. T.D. Shenkenberg, and D. von Hoff Mitoxantrone: a new anticancer drug with significant clinical activity Ann Intern Med 105 1986 67 81
19. H.P. Hartung, and R. Gonsette Mitoxantrone in progressive multiple sclerosis: a placebo-controlled, double-blind, randomised, multicentre trial Lancet 360 9350 Dec 21-28 2002 2018 2025
20. J.J. Marriott, and J.M. Miyasaki Evidence report: the efficacy and safety of mitoxantrone (Novantrone) in the treatment of multiple sclerosis: report of the Therapeutics and Technology Assessment Subcommittee of the American Academy of Neurology Neurology 74 18 May 4 2010 1463 1470
21. J.R. Avasarala, A.H. Cross, D.B. Clifford, B.A. Singer, B.A. Siegel, and E.E. Abbey Rapid onset mitoxantrone-induced cardiotoxicity in secondary progressive multiple sclerosis Mult Scler 9 2003 59 62
22. F. Paul, J. Dörr, and J. Würfel Early mitoxantrone-induced cardiotoxicity in secondary progressive multiple sclerosis J Neurol Neurosurg Psychiatry 78 2007 198 200
23. S. Goffete, V. van Pesch, and J.L. Vanoverschelde Severe delayed heart failure in three multiple sclerosis patients previously treated with mitoxantrone J Neurol 252 2005 1217 1222
24. A. Hamzehloo, and M. Etemadifar Mitoxantrone-induced cardiotoxicity in patients with multiple sclerosis Arch Iran Med 9 2006 111 114
25. B.O. Khatri, M. Wroblewski, and J. Kramer Mitoxantrone in worsening secondary progressive multiple sclerosis: a prospective, open-label study Curr Ther Res 67 2006 55 65
26. A. Ostberg, F. Pittas, and B. Taylor Use of low-dose mitoxantrone to treat aggressive multiple sclerosis: a single centre open-label study using patient self-assessment and clinical measures of multiple sclerosis status Int Med J 35 2005 382 387
27. E. Kingwell, B. Leung, and S. Isserow Suspected cardiotoxicity during mitoxantrone treatment for multiple sclerosis in British Columbia, Canada Neurology 72 Suppl. 3 2009 A237 A238
28. S. Hum, and Y. Lapierre A clinical retrospective report on mitoxantrone treatment in active multiple sclerosis patients Neurology 72 Suppl. 3 2009 A237
29. V.C. Zingler, M. Nabauer, and K. Jahn Assessment of potential cardiotoxic side effects of mitoxantrone in patients with multiple sclerosis Eur Neurol 54 2005 28 33
30. C. Buttinelli, A. Clemenzi, and G. Borriello Mitoxantrone treatment in multiple sclerosis: a 5-year clinical and MRI follow-up Eur J Neurol 14 2007 1281 1287
31. E. Le Page, E. Leray, and G. Taurin Mitoxantrone as induction treatment in aggressive relapsing remitting multiple sclerosis: treatment response factors in a 5-year follow-up observational study of 100 patients J Neurol Neurosurg Psychiatry 79 2008 52 56
32. V. Rivera, A. Al-Sabbagh, and R. Bennett RENEW study update XVIII: ongoing evaluation of the safety and tolerability of mitoxantrone in worsening multiple sclerosis Mult Scler 14 Suppl. 1 2008 S175 [Abstract]
33. R.G. Ghalie, E. Mauch, and G. Edan A study of therapy related leukemia after mitoxantrone therapy for multiple sclerosis Mult Scler 8 2002 441 445
34. A.M. Vicari, F. Ciceri, and F. Folli Acute promyelocytic leukemia following mitoxantrone as single agent for the treatment of multiple sclerosis Leukemia 12 1998 441 442
35. K. Rammohan, M. Kita, and D.J. Lynn Post-marketing reports of acute leukemia in mitoxantrone-treated multiple sclerosis patients Mult Scler 14 Suppl. 1 2008 S174 [Abstract]
36. A. Pascual, I. Boscá, and J. Mallacia Secondary acute myeloid leukemia following long-term treatment with mitoxantrone in patients with multiple sclerosis Mult Scler 13 2007 S23 [Abstract]
37. Cordioli C, Cattaneo C, Capra R. Analysis of incidence, risk factors and prognosis of acute promyelocytic leukaemia related to mitoxantrone therapy in multiple sclerosis.
38. Neurology 68 Suppl. 1 2007 A276 [Abstract]
39. D.A. Woo, R.H. Collins, and H.S. Rossman Mitoxantrone associated leukemia in multiple sclerosis: case studies Int J MS Care 10 2008 41 46
40. I. Bosca, A.M. Pascual, and B. Casanova Four new cases of therapy-related acute promyelocytic leukemia after mitoxantrone Neurology 71 2008 457 458
41. M. Debouverie, L. Taillandier, and S. Pittion-Vouyovitch Clinical follow-up of 304 patients with multiple sclerosis three years after mitoxantrone treatment Mult Scler 13 2007 626 631
42. R. Tanasescu, M. Debouverie, and S. Pittion Acute myeloid leukaemia induced by mitoxantrone in a multiple sclerosis patient J Neurol 251 2004 762 763
43. S.A. Sadiq, M. Rammal, and G. Sara Chronic myeloid leukemia associated with mitoxantrone treatment in a patient with MS Mult Scler 14 2008 272 273
44. V. Martinelli, and E. Cocco Acute myeloid leukemia in Italian patients with multiple sclerosis treated with mitoxantrone Neurology 77 21 Nov 22 2011 1887 1895
45. D. Brassat, C. Recher, and E. Waubant Therapy-related acute myeloblastic leukemia after mitoxantrone treatment in a patient with MS Neurology 59 2002 954 955
46. D.E. Goodkin Therapy-related leukemia in mitoxantrone treated patients Mult Scler 9 2003 426
47. C. Cattaneo, C. Almici, and E. Borlenghi A case of acute promyelocytic leukaemia following mitoxantrone treatment of multiple sclerosis Leukemia 17 2003 985 986
48. C. Heesen, M. Bruegmann, and J. Gbdamosi Therapy related acute myelogenous leukaemia (t-AML) in a patient with multiple sclerosis treated with mitoxantrone Mult Scler 9 2003 213 214
49. B. Delisse, J. de Seze, and A. Mackowiak Therapy related acute myeloblastic leukaemia after mitoxantrone treatment in a patient with multiple sclerosis Mult Scler 10 2004 92
50. C.H. Polman, P.W. O'Connor, and E. Havrdova A randomized, placebo-controlled trial of natalizumab for relapsing multiple sclerosis N Engl J Med 354 2006 899 910
51. R.A. Rudick, W.H. Stuart, and P.A. Calabresi Natalizumab plus interferon beta-1a for relapsing multiple sclerosis N Engl J Med 354 2006 911 923
52. K. Hellwig, and R. Gold Progressive multifocal leukoencephalopathy and natalizumab J Neurol 258 11 Nov 2011 1920 1928
53. C. Bozic, L.M. Christiano, and R. Hyde Utilisation and safety of natalizumab in patients with relapsing multiple sclerosis Mult Scler 16 Suppl. 10 2010 S315
54. A.K. Bag, J.K. Curé, P.R. Chapman, G.H. Roberson, and R. Shah JC virus infection of the brain AJNR Am J Neuroradiol 31 9 2010 1564 1576
55. http://www.biogenidec.ch/medizinische-fachkreise.aspx?ID=10389 [accessed 27 March 2012]
56. R.J. Fox, and R.A. Rudick Risk stratification and patient counseling for natalizumab in multiple sclerosis Neurology 78 6 Feb 7 2012 436 437
57. S. Jilek, E. Jaquiéry, H.H. Hirsch, A. Lysandropoulos, M. Canales, and L. Guignard Immune responses to JC virus in patients with multiple sclerosis treated with natalizumab: a cross-sectional and longitudinal study Lancet Neurol 9 3 Mar 2010 264 272
58. L. Rinaldi, F. Rinaldi, and P. Perini No evidence of JC virus reactivation in natalizumab treated multiple sclerosis patients: an 18 month follow-up study J Neurol Neurosurg Psychiatry 81 12 Dec 2010 1345 1350
59. Y. Chen, and E. Bord Asymptomatic reactivation of JC virus in patients treated with natalizumab N Engl J Med 361 11 Sep 10 2009 1067 1074
60. R.M. Lonergan, M.J. Carr, and C.F. De Gascun Reactivation of BK polyomavirus in patients with multiple sclerosis receiving natalizumab therapy J Neurovirol August 2009
61. C. Ryschkewitsch, P. Jensen, J. Hou, G. Fahle, S. Fischer, and E.O. Major Comparison of PCR-southern hybridization and quantitative real-time PCR for the detection of JC and BK viral nucleotide sequences in urine and cerebrospinal fluid J Virol Methods 121 2004 217 221
62. C. De Gascun, M. Carr, and R.M. Lonergan Reactivation of latent viral infections in patients receiving natalizumab for the treatment of multiple sclerosis J Neurovirol 15 Suppl. 1 2009 22 23
63. L. Gorelik, and M. Lerner Anti-JC virus antibodies: implications for PML risk stratification Ann Neurol 68 3 Sep 2010 295 303
64. L. Kappos Natalizumab treatment for multiple sclerosis: updated recommendations for patient selection and monitoring Lancet Neurol 10 8 Aug 2011 745 758
65. N.F. Blair, B.J. Brew, and J.P. Halpern Natalizumab-associated PML identified in the presymptomatic phase using MRI surveillance Neurology 78 7 Feb 14 2012 507 508
66. S. Bezabeh, C.M. Flowers, C. Kortepeter, and M. Avigan Clinically significant liver injury in patients treated with natalizumab Aliment Pharmacol Ther 31 9 May 2010 1028 1035
67. A. Lisotti, F. Azzaroli, S. Brillanti, and G. Mazzella Severe acute autoimmune hepatitis after natalizumab treatment Dig Liver Dis 44 4 2012 356 357
68. TYSABRI prescribing information http://www.tysabri.com/en-US/tysb/site/ pdfs/TYSABRI-pi.pdf [Accessed 12/22/2011]
69. V. Brinkmann FTY720 (fingolimod) in multiple sclerosis: therapeutic effects in the immune and the central nervous system Br J Pharmacol 158 2009 1173 1182
70. J. Chun, and H.P. Hartung Mechanism of action of oral fingolimod (FTY720) in multiple sclerosis Clin Neuropharmacol 33 2010 91 101
71. L. Kappos, E.W. Radue, and P. O'Connor A placebo controlled trial of oral fingolimod in relapsing multiple sclerosis N Engl J Med 362 2010 387 401
72. Fingolimod prescribing information http://www.pharma.us.novartis.com [Accessed December 23, 2011]
73. G. Francis, L. Kappos, and P. O'Connor Lymphocytes and fingolimod: temporal pattern and relationship with infections Mult Scler 16 2010 S146
74. J.A. Cohen, and F. Barkhof Oral fingolimod or intramuscular interferon for relapsing multiple sclerosis N Engl J Med 362 5 Feb 4 2010 402 415
75. Antonio Uccelli, Francesca Ginocchio, and Giovanni Luigi Mancardi Primary varicella zoster infection associated with fingolimod treatment Neurology 76 2011 1023
76. M.C. Kowarik, and H.L. Pellkofer Differential effects of fingolimod (FTY720) on immune cells in the CSF and blood of patients with MS Neurology 76 14 Apr 5 2011 1214 1221
77. FDA Peripheral and CNS Advisory Committee Meeting Slides [Available at] http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/ Drugs/PeripheralandCentralNervousSystemDrugsAdvisoryCommittee/UCM216556.pdf [Accessed December 23, 2011]
78. European Medicines Agency Web site http://www.ema.europa.eu/ema/index. jsp?curl=pages/medicines/human/public-health-alerts/2012/01/human-pha-detail- 000050.jsp&mid=WC0b01ac058001d126 [Accessed March 5 2012]
79. FDA.gov Gilenya (fingolimod): Drug Safety Communication - Safety Review of a Reported Death After the First Dose http://www.fda.gov/Safety/MedWatch/ SafetyInformation/SafetyAlertsforHumanMedicalProducts/ucm284355.htm 12/20/2011 [Accessed 23/12/2011]
80. K.B. Conzett, and I. Kolm Melanoma occurring during treatment with fingolimod for multiple sclerosis: a case report Arch Dermatol 147 8 Aug 2011 991 992
81. H. Munier-Lehmann, P.O. Vidalain, F. Tangy, and Y.L. Janin On dihydroorotate dehydrogenases and their inhibitors and uses J Med Chem Mar 20 2013
82. Aubagio prescribing information http://products.sanofi.us/aubagio/ aubagio.pdf [Accessed 04/23/2013]
83. R.A. Linker, D.H. Lee, and S. Ryan Fumaric acid esters exert neuroprotective effects in neuroinflammation via activation of the Nrf2 antioxidant pathway Brain 134 2011 678 692
84. Tecfidera prescribing information http://www.tecfidera.com/pdfs/full- prescribing-information.pdf [Accessed 4/23/2013]
85. R. Gold, L. Kappos, D.L. Arnold, A. Bar-Or, G. Giovannoni, and K. Selmaj DEFINE study investigators. Placebo-controlled phase 3 study of oral BG-12 for relapsing multiple sclerosis N Engl J Med 367 12 Sep 20 2012 1098 1107
86. U. Ermis, J. Weis, and J.B. Schulz PML in a patient treated with fumaric acid N Engl J Med 368 17 2013 1657 1658
87. B.W. van Oosten, J. Killestein, F. Barkhof, C.H. Polman, and M.P. Wattjes PML in a patient treated with dimethyl fumarate from a compounding pharmacy N Engl J Med 368 17 2013 1658 1659
88. M.T. Sweetser, K.T. Dawson, and C. Bozic Manufacturer's response to case reports of PML N Engl J Med 368 17 2013 1659 1661
89. A. Deiß, I. Brecht, A. Haarmann, and M. Buttmann Treating multiple sclerosis with monoclonal antibodies: a 2013 update Expert Rev Neurother 13 3 Mar 2013 313 335 10.1586/ern.13.17
90. A. Ray Opinion: beyond debacle and debate: developing solutions in drug safety Nat Rev Drug Discov 8 October 2009 775 777
91. European Medicines Agency. Guideline on procedures for the granting of a marketing authorization under exceptional circumstances, pursuant to article 14 (8) of regulation (EC) No 726/2004 [EMEA web site] http://www.emea.europa.eu/ docs/en-GB/document-library/Regulatory-and-procedural-guideline/2009/10/ WC500004883.pdf [Accessed Janury 2012]
92. A. Bouchie Industry reneges on post-marketing trial commitments Nat Biotechnol 21 2003 718
93. J. Concato, N. Shah, and R.I. Horwitz Randomized, controlled trials, observational studies, and the hierarchy of research designs N Engl J Med 342 2000 1887 1892
94. A. Breckenridge, K. Woods, and J. Raine Monitoring the safety of licensed medicines Nat Rev Drug Discov 4 2005 541 543