How to assess new drugs for neuropathies: Advances in trial design and methodology

Current Opinion in Neurology

Volumn 21, Issue 5, 2008, Pages 519-526

  Van Doorn, Pieter A ^a   Merkies, Ingemar S J ^a  

^a ERASMUS MEDICAL CENTER   (Netherlands)

Author keywords

Chronic inflammatory demyelinating polyradiculoneuropathy; Clinimetrics; Guillain Barre syndrome; Polyneuropathy; Randomized controlled trial

Indexed keywords

ASCORBIC ACID; AZATHIOPRINE; CYCLOPHOSPHAMIDE; CYCLOSPORIN; IMMUNOGLOBULIN; IMMUNOGLOBULIN M; IMMUNOSUPPRESSIVE AGENT; METHOTREXATE; METHYLPREDNISOLONE; MYCOPHENOLIC ACID; PLACEBO; PREDNISOLONE;

AUTOIMMUNE DISEASE; CHRONIC INFLAMMATORY DEMYELINATING POLYNEUROPATHY; CLINICAL TRIAL; COMBINATION CHEMOTHERAPY; DIABETIC NEUROPATHY; DISEASE COURSE; DISEASE SEVERITY; DRUG EFFICACY; FOLLOW UP; GUILLAIN BARRE SYNDROME; HEREDITARY MOTOR SENSORY NEUROPATHY; HUMAN; IMMUNOTHERAPY; MONOCLONAL IMMUNOGLOBULINEMIA; MONOTHERAPY; MOTOR NEUROPATHY; OUTCOME ASSESSMENT; PATIENT CARE; PERIPHERAL NEUROPATHY; PLASMAPHERESIS; PROGNOSIS; QUALITY OF LIFE; RANDOMIZED CONTROLLED TRIAL; REVIEW; SHORT COURSE THERAPY; TREATMENT DURATION; TREATMENT OUTCOME;

ANIMALS; DISEASE PROGRESSION; HUMANS; PERIPHERAL NERVOUS SYSTEM DISEASES; RANDOMIZED CONTROLLED TRIALS AS TOPIC; RESEARCH DESIGN; TREATMENT OUTCOME;

EID: 53549084034     PISSN: 13507540     EISSN: None     Source Type: Journal    
DOI: 10.1097/WCO.0b013e32830f1e3b     Document Type: Review

References (63)

1. Martyn CN, Hughes RA. Epidemiology of peripheral neuropathy. J Neural Neurosurg Psychiatry 1997; 62:310-318.
2. Mold JW, Vesely SK, Keyl BA, ef al. The prevalence, predictors, and consequences of peripheral sensory neuropathy in older patients. J Am Board Fam Pract 2004; 17:309-318.
3. Gregg EW, Sorlie P, Paulose-Ram R, et al. Prevalence of lower-extremity disease in the US adult population ≥40 years of age with and without diabetes: 1999-2000 national health and nutrition examination survey. Diabetes Care 2004; 27:1591-1597.
4. England JD, Asbury AK. Peripheral neuropathy. Lancet 2004; 363:2151-2161.
5. European Federation of Neurological Societies/Peripheral Nerve Society Guideline on management of multifocal motor neuropathy. Report of a joint task force of the European Federation of Neurological Societies and the Peripheral Nerve Society. J Peripher Nerv Syst 2006; 11:1-8.
6. Dyck PJ, Norell JE, Tritschler H, et al. Challenges in design of multicenter trials: end points assessed longitudinally for change and monotonicity. Diabetes Care 2007; 30:2619-2625. The paper gives very practical advice on how to improve the design for multicenter studies in diabetic neuropathy.
7. Reilly MM, de Jonghe P, Pareyson D. 136th ENMC International Workshop: Charcot-Marie-Tooth disease type 1A (CMT1A), 8-10 April 2005, Naarden, The Netherlands. Neuromuscul Disord 2006; 16:396-402.
8. Hughes RA, Swan AV, Raphael JC, et al. Immunotherapy for Guillain-Barré syndrome: a systematic review. Brain 2007; 130:2245-2257. Meta-analysis on the effect of IVIg, steroids and plasma exchange in GBS.
9. Ziegler D. Treatment of diabetic neuropathy and neuropathic pain: how far have we come? Diabetes Care 2008; 31 (Suppl 2):S255-S261.
10. Asbury AK, Cornblath DR. Assessment of current diagnostic criteria for Guillain-Barré syndrome. Ann Neural 1990; 27 (Suppl):S21-S24.
11. van der Meché FG, van Doom PA, Meulstee J, Jennekens FG. Diagnostic and classification criteria for the Guillain-Barré syndrome. Eur Neural 2001; 45:133-139.
12. Research criteria for diagnosis of chronic inflammatory demyelinating polyneuropathy (CIDP). Report from an Ad Hoc Subcommittee of the American Academy of Neurology AIDS Task Force. Neurology 1991; 41:617-618.
13. Mehndiratta MM, Hughes RA. Corticosteroids for chronic inflammatory demyelinating polyradiculoneuropathy. Cochrane Database Syst Rev 2002:CD002062.
14. Mehndiratta MM, Hughes RA, Agarwal P. Plasma exchange for chronic inflammatory demyelinating polyradiculoneuropathy. Cochrane Database Syst Rev 2004:CD003906.
15. van Schaik I, Winer JB, de Haan R, Vermeulen M. Intravenous immunoglobulin for chronic inflammatory demyelinating polyradiculoneuropathy. Cochrane Database Syst Rev 2002:CD001797.
16. Hughes RA, Donofrio P, Bril V, et al. Intravenous immune globulin (10% caprylate-chromatography purified) for the treatment of chronic inflammatory demyelinating polyradiculoneuropathy (ICE study): a randomised placebocontrolled trial, Lancet Neural 2008; 7:136-144. Largest RCT in CIDP that clearly showed the effect of IVIg. The responseconditional cross-over design and the long-term follow-up further make this an extraordinary paper in CIDP.
17. van Schaik I, van den Berg LH, de Haan R, Vermeulen M. Intravenous immunoglobulin for multifocal motor neuropathy. Cochrane Database Syst Rev 2005:CD004429.
18. European Federation of Neurological Societies/Peripheral Nerve Society Guideline on management of paraproteinemic demyelinating neuropathies. Report of a joint task force of the European Federation of Neurological Societies and the Peripheral Nerve Society. J Peripher Nerv Syst 2006; 11:9-19.
19. Streiner DL, Norman GR. Health measurement scales: a practical guide to their development and use. 2nd ed. New York: Oxford University Press; 1998.
20. Hobart JC, Cano SJ, Zajicek JP, Thompson AJ. Rating scales as outcome measures for clinical trials in neurology: problems, solutions, and recommendations. Lancet Neural 2007; 6:1094-1105. Paper that clearly describes practical issues related to the use of outcomes measures in RCT. It is shown that improvements in the scientific rigor of rating scales can improve the chances to obtain correct conclusions about the effectiveness of treatments.
21. Benatar M. Basic principles of epidemiology and biostatistics. In: Benetar M, editor. Neuromuscular disease. Evidence and analysis in clinical neurology. Totowa, New Jersey: Humana Press; 2006. pp. 3-40.
22. Guyatt G, Sackett D, Adachi J, et al. A clinician's guide for conducting randomized trials in individual patients. Can Med Assoc J 1988; 139: 497-503.
23. Mahon J, Laupacis A, Donner A, Wood T. Randomised study of n of 1 trials versus standard practice. Br Med J 1996; 312:1069-1074.
24. European Federation of Neurological Societies/Peripheral Nerve Society Guideline on management of chronic inflammatory demyelinating polyradiculoneuropathy. Report of a joint task force of the European Federation of Neurological Societies and the Peripheral Nerve Society. J Peripher Nerv Syst 2005; 10:220-228.
25. Hahn AF. Guillain-Barré syndrome. Lancet 1998; 352:635-641.
26. Hughes RA, Cornblath DR. Guillain-Barré syndrome. Lancet 2005; 366: 1653-1666.
27. Koller H, Schroeter M, Kieseier BC, Hartung HP. Chronic inflammatory demyelinating polyneuropathy - update on pathogenesis, diagnostic criteria and therapy. Curr Opin Neural 2005; 18:273-278.
28. Merkies IS, Lauria G. 131st ENMC international workshop: selection of outcome measures for peripheral neuropathy clinical trials, 10-12 December 2004, Naarden, The Netherlands. Neuromuscul Disord 2006; 16:149-156.
29. Ruts L, van Koningsveld R, Jacobs BC, van Doorn PA. Determination of pain and response to methylprednisolone in Guillain-Barré syndrome. J Neural 2007; 254:1318-1322. This paper acknowledges that pain is an important issue in GBS. Various different symptoms of pain can be distinguished, and presumably need different treatment approaches.
30. Hughes RA, Newsom-Davis JM, Perkin GD, Pierce JM. Controlled trial prednisolone in acute polyneuropathy. Lancet 1978; 2:750-753.
31. Hughes RA, Wijdicks EF, Barohn R, et al. Practice parameter: immunotherapy for Guillain-Barré syndrome: report of the Quality Standards Subcommittee of the American Academy of Neurology. Neurology 2003; 61:736-740.
32. Randomised trial of plasma exchange, intravenous immunoglobulin, and combined treatments in Guillain-Barré syndrome. Plasma Exchange/Sandoglobulin Guillain-Barre Syndrome Trial Group. Lancet 1997; 349:225-230.
33. van Koningsveld R, Schmitz PI, van der Meché FG, et al. Effect of methylprednisolone when added to standard treatment with intravenous immunoglobulin for Guillain-Barre syndrome: randomised trial. Lancet 2004; 363:192-196.
34. Garssen MP, van Koningsveld R, van Doom PA, et al. Treatment of Guillain-Barré syndrome with mycophenolate mofetil: a pilot study. J Neural Neurosurg Psychiatry 2007; 78:1012-1013.
35. Halstead SK, Zitman FM, Humphreys PD, et al. Eculizumab prevents antiganglioside antibody-mediated neuropathy in a murine model. Brain 2008; 131:1197-1208. Important paper that provides evidence that complement inhibition is important in murine model for GBS.
36. Ruts L, van Koningsveld R, van Doorn PA. Distinguishing acute-onset CIDP from Guillain-Barré syndrome with treatment related fluctuations. Neurology 2005; 65:138-140.
37. Hahn AF, Bolton CF, Pillay N, et al. Plasma-exchange therapy in chronic inflammatory demyelinating polyneuropathy. A double-blind, sham-controlled, cross-over study. Brain 1996; 119:1055-1066.
38. Hughes RA, Swan AV, van Doorn PA. Cytotoxic drugs and interferons for chronic inflammatory demyelinating polyradiculoneuropathy. Cochrane Database Syst Rev 2004:CD003280.
39. Piepers S, van den Berg-Vos R, van der Pol WL, et al. Mycophenolate mofetil as adjunctive therapy for MMN patients: a randomized, controlled trial. Brain 2007; 130:2004-2010. RCT showing that mycophenolate does not have an additional effect to IVIg in patients with MMN.
40. Leger JM, Chassande B, Musset L, et al. Intravenous immunoglobulin therapy in multifocal motor neuropathy: a double-blind, placebo-controlled study. Brain 2001; 124:145-153.
41. Niermeijer JM, Eurelings M, van der Linden MW, et al. Intermittent cyclo-phosphamide with prednisone versus placebo for polyneuropathy with IgM monoclonal gammopathy. Neurology 2007; 69:50-59. This RCT did not show a positive effect of cyclophosphamide and steroids in IgM MGUS. This could be due to the selection of the primary outcome criterion that was based on an assessment scale not sensitive for a predominant sensory neuropathy.
42. Sommer C, Lauria G. Skin biopsy in the management of peripheral neuropathy. Lancet Neural 2007; 6:632-642. Very good overview on the use of skin biopsies in peripheral neuropathies.
43. Young P, de Jonghe P, Stogbauer F, Butterfass-Bahloul T. Treatment for Charcot-Marie-Tooth disease. Cochrane Database Syst Rev 2008: CD006052.
44. Pareyson D, Schenone A, Fabrizi GM, ef al. A multicenter, randomized, double-blind, placebo-controlled trial of long-term ascorbic acid treatment in Charcot-Marie-Tooth disease type 1A (CMT-TRIAAL): the study protocol [EudraCT no.: 2006-000032-27]. Pharmacol Res 2006; 54:436-441.
45. Steyerberg EW, Bossuyt PM, Lee KL Clinical trials in acute myocardial infarction: should we adjust for baseline characteristics? Am Heart J 2000; 139:745-751.
46. Hernandez AV, Steyerberg EW, Habbema JD. Covariate adjustment in randomized controlled trials with dichotomous outcomes increases statistical power and reduces sample size requirements. J Clin Epidemiol 2004; 57:454-460.
47. Murray GD, Barer D, Choi S, ef al. Design and analysis of phase III trials with ordered outcome scales: the concept of the sliding dichotomy. J Neurotrauma 2005; 22:511-517.
48. Goodman SN, SIadky JT. A Bayesian approach to randomized controlled trials in children utilizing information from adults: the case of Guillain-Barré syndrome. Clin Trials 2005; 2:305-310.
49. Durand MC, Porcher R, Orlikowski D, et al. Clinical and electrophysiological predictors of respiratory failure in Guillain-Barré syndrome: a prospective study. Lancet Neurol 2006; 5:1021-1028.
50. van Koningsveld R, Steyerberg EW, Hughes RA, et al. A clinical prognostic scoring system for Guillain-Barré syndrome. Lancet Neurol 2007; 6:589-594. The EGOS is a very simple clinical scoring system for patients with GBS offering the opportunity to accurately predict the chance to walk unaided after 6 months.
51. Merkies IS, Schmitz PI, van der Meché FG, van Doom PA. Comparison between impairment and disability scales in immune-mediated polyneuropathies. Muscle Nerve 2003; 28:93-100.
52. Merkies IS, Schmitz PI. Getting closer to patients: the INCAT Overall Disability Sum Score relates better to patients' own clinical judgement in immunemediated polyneuropathies. J Neurol Neurosurg Psychiatry 2006; 77:970-972.
53. World Health Organization. International classification of functioning, disability, and health. Geneva, Switzerland: World Health Organization; 2001.
54. Aaronson NK. Quality of life: what is it? How should it be measured? Oncology (Williston Park) 1988; 2:69-76, 64.
55. Lunn MP, Leger JM, Merkies IS, et al. 151st ENMC international workshop: Inflammatory Neuropathy Consortium, 13th-15th April 2007, Schiphol, The Netherlands. Neuromuscul Disord 2008; 18:85-89.
56. van Nes SI, Faber CG, Merkies ISJ. Outcome measures in immune-mediated neuropathies: the need to standardize their use and to understand the clinimetric essentials. J Peripher Nerv Syst 2008; 13:136-147. This paper gives an overview on the use of various outcome measures in immunemediated neuropathies. It additionally discusses the use of modem methodology to assess outcome in these conditions.
57. Bond TG, Fox CM. Applying the Rasch model: fundamental measurement for the human sciences. New York: Lawrence Erlbaum Associates; 2001.
58. Hays RD, Morales LS, Reise SP. Item Response Theory and health outcomes measurement in the 21st century. Med Care 2000; 38:1128-1142.
59. Jaeschke R, Singer J, Guyatt GH. Measurement of health status. Ascertaining the minimal clinically important difference. Control Clin Trials 1989; 10:407-415.
60. Juniper EF. Quality of life questionnaires: does statistically significant = clinically important? J Allergy Clin Immunol 1998; 102:16-17.
61. Sloan J, Symonds T, Vargas-Chanes D, Fndley B. Practical guidelines for assessing the clinical significance of health-related quality of life changes within clinical trials. Drug Inform J 2003; 37:23-31.
62. Kulkami AV. Distribution-based and anchor-based approaches provided different interpretability estimates for the Hydrocephalus Outcome Questionnaire. J Clin Epidemiol 2006; 59:176-184.
63. Cohen J. Statistical power analysis for the behavioural sciences. New York: Academic Press; 1977.