Survival, and time to an advanced disease state or progression, of untreated patients with moderately severe multiple sclerosis in a multicenter observational database: Relevance for design of a clinical trial for high dose immunosuppresive therapy with autologous hematopoietic stem cell transplantation

Multiple Sclerosis

Volumn 12, Issue 2, 2006, Pages 174-179

  Daumer, Martin ^a   Griffith, L M ^b   Meister, W ^a   Nash, R A ^c   Wolinsky, J S ^d  

^a Sylvia Lawry Centre for Multiple Sclerosis Research   (Germany)

^b NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES   (United States)

^c UNIVERSITY OF WASHINGTON   (United States)

^d UNIVERSITY OF TEXAS MEDICAL SCHOOL   (United States)

Author keywords

Autologus hematopoietic cell transplantation; Immunosuppresive therapy; Multiple sclerosis; Survival; Time to progression

Indexed keywords

IMMUNOSUPPRESSIVE AGENT; PLACEBO;

ADJUVANT THERAPY; ARTICLE; AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION; CLINICAL TRIAL; CONFIDENCE INTERVAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; DISEASE COURSE; DISEASE SEVERITY; DRUG MEGADOSE; EXPANDED DISABILITY STATUS SCALE; FEMALE; HUMAN; IMMUNOSUPPRESSIVE TREATMENT; MAJOR CLINICAL STUDY; MALE; MORTALITY; MULTICENTER STUDY; MULTIPLE SCLEROSIS; OBSERVATIONAL STUDY; PROBABILITY; RISK BENEFIT ANALYSIS; SURVIVAL RATE;

CLINICAL TRIALS; COMBINED MODALITY THERAPY; DATABASES, FACTUAL; DISABLED PERSONS; DISEASE PROGRESSION; FEMALE; FOLLOW-UP STUDIES; HUMANS; IMMUNOSUPPRESSIVE AGENTS; MALE; MULTIPLE SCLEROSIS; MULTIPLE SCLEROSIS, RELAPSING-REMITTING; PROPORTIONAL HAZARDS MODELS; REGRESSION ANALYSIS; RESEARCH DESIGN; STEM CELL TRANSPLANTATION; SURVIVAL ANALYSIS; TIME FACTORS; TRANSPLANTATION, AUTOLOGOUS;

EID: 33645800200     PISSN: 13524585     EISSN: None     Source Type: Journal    
DOI: 10.1191/135248506ms1256oa     Document Type: Article

References (36)

1. Atkins H, Freedman M, Bowman M, Bar-Or A, Cheynier R, Corsini R et al. Immune events following immunoablative therapy for the treatment of multiple sclerosis (MS). Biol Blood Marrow Transplant 2004; 10 (Supplement 1): 62.
2. Burt RK, Cohen BA, Russell E, Spero K, Joshi J, Oyama Y et al. Hematopoietic stem cell transplantation for progressive multiple sclerosis: failure of a total body irradiation-based conditioning regimen to prevent disease progression in patients with high disability scores. Blood 2003; 102: 2373-78.
3. Fassas A, Kazis A. High dose immunosuppression and autologous hematopoietic stem cell rescue for severe multiple sclerosis. J Hematother Stem Cell Res 2003; 12: 701-11.
4. Fassas A, Kimiskidis VK. Autologous hematopoietic stem cell transplantation in the treatment of multiple sclerosis: rationale and clinical experience. J Neurol Sci 2004; 223: 53-58.
5. Fassas A, Kimmiskidis VK. Stem cell transplantation for multiple sclerosis: what is the evidence? Blood Rev 2003; 17: 233-40.
6. Fassas A, Nash RA. Multiple sclerosis. Best Pract Res Clin Haematol 2004; 17: 247-62.
7. Fassas A, Passweg JR, Anagnastopoulos A, Kazis A, Kozak T, Havrdova E et al. Hematopoetic stem cell transplantation for multiple sclerosis. A retrospective multicenter study. J Neurol 2002; 249: 1088-97.
8. Freedman MS, Atkins HL. Suppressing immunity in advancing MS. Too much too late, or too late for much? Neurology 2004; 62: 168-69.
9. Muraro PA, Ingoni RC, Martin R. Hematopoetic stem cell transplantation for multiple sclerosis: current status and future challenges. Curr Opin Neurol 2003; 16: 299-305.
10. Nash RA, Bowen JD, McSweeney PA, Pavletic SZ, Maravilla KR, Park M-S et al. High-dose immunosuppressive therapy and autologous peripheral blood stem cell transplantation for severe multiple sclerosis. Blood 2003; 102: 2364-72.
11. Saccardi R, Mancardi GL, Solari A, Bosi A, Bruzzi P, Di Bartolomeo P et al. Autologous HSCT for severe progressive MS in a multicenter trial: impact on disease activity and quality of life. Blood 2005; 105: 2601-607.
12. Saiz A, Blanco Y, Carreras E, Berenguer J, Rovira M, Pujol T et al. Clinical and MRI outcome after autologous hematopoetic stem cell transplantation in MS. Neurology 2004; 62: 282-84.
13. Tyndall A, Gratwohl A. The use of high dose immunoablative therapy with hematopoetic stem cell support therapy in the treatment of severe autoimmune diseases. Int J Hematol 2002; 76 (Supplement 1): 218-22.
14. Kurtzke JF. Rating neurologic impairment in multiple sclerosis: an expanded disability status scale (EDSS). Neurology 1983; 33: 1444-52.
15. Hohlfeld R. How promising is haematopoetic stem cell transplantation in multiple sclerosis? J Neurol 2002; 249: 1147-49.
16. Lucchinetti CF, Bruck W, Lassmann H. Pathology and pathogenesis of MS. In McDonald WI, Noseworthy JH eds. Multiple sclerosis, second edition. Philadelphia, USA: Butterworth-Heinemann-Elsevier Science, 2003: 93-113.
17. Lucchinetti C, Bruck W, Parisi J, Scheithauer B, Rodriguez M, Lassmann H. Heterogeneity of multiple sclerosis lesions: implications for the pathogenesis of demyelination. Ann Neurol 2000; 47: 707-17.
18. Paty DW, Arnold DL. The lesions of multiple sclerosis. N Engl J Med 2002; 346: 199-200.
19. Goodin DS, Frohman EM, Garmany GP, Halper J, Likosky WH, Lublin FD et al. Disease modifying therapies in multiple sclerosis. Report of the Therapeutics and Technology Assessment Subcommittee of the American Academy of Neurology and the MS Council for Clinical Practice Guidelines. Neurology 2002; 58: 169-78.
20. Hartung HP, Gonsette R, Konig N, Kwiecinski H, Guseo A, Morrissey SP et al. Mitoxantrone in progressive multiple sclerosis: a placebo-controlled, double-blind, randomized, multicentre trial. Lancet 2002; 360: 2018-25.
21. Rieckmann P, Toyka KV. Multiple Sclerosis Therapy Consensus Study Group. Escalating immunotherapy of multiple sclerosis. New aspects and practical application. J Neurol 2004; 251: 1329-39.
22. Emanuel EJ, Wendler D, Grady C. What makes clinical research ethical? JAMA 2000; 283: 2701-11.
23. Bredeson CN, Pavletic SZ. Considerations when designing a clinical trial of haematopoietic stem cell transplantation for autoimmune disease. Best Pract Res Clin Haematol 2004; 17: 327-43.
24. Noseworthy J, Kappos L, Daumer M. Competing interests in multiple sclerosis research. Lancet 2003; 361: 350-51.
25. Benedikz J, Stefansson M, Guomundsson J, Jonasdottir A, Fossdal R, Gulcher J et al. The natural history of untreated multiple sclerosis in Iceland. A total population-based 50 year prospective study. Clin Neurol Neurosurg 2002; 104: 208-10.
26. Confavreux C, Vuksic S, Moreau T, Adeleine P. Relapses and progression of disability in multiple sclerosis. N Engl J Med 2000; 343: 1430-38.
27. Rudge P. The value of natural history studies of multiple sclerosis. Brain 1999; 122: 591-92.
28. Runmarker B, Anderson O. Prognostic factors in a multiple sclerosis incidence cohort with twenty-five years of follow-up. Brain 1993; 116: 117-34.
29. Weinshenker BG, Bass B, Rice GPA, Noseworthy J, Carriere W, Baskerville J et al. The natural history of multiple sclerosis: a geographically based study. 1. Clinical course and disability. Brain 1989; 112: 133-46.
30. Pittock SJ, Mayr WT, McClelland RL, Jorgensen NW, Weigand SD, Noseworthy JH et al. Change in MS-related disability in a population-based cohort. Neurology 2004; 62: 51-59.
31. Tyndall A, Saccardi R. Haematopoietic stem cell transplantation in the treatment of severe autoimmune disease: results from phase I/II studies, prospective randomized trials and future direction. Clin Exp Immunol 2005; 141: 1-9.
32. Brønnum-Hansen H, Koch-Henriksen N, Stenager E. Trends in survival and cause of death in Danish patients with multiple sclerosis. Brain 2004; 127: 844-50.
33. Jaeschke R, Singer J, Guyatt GH. Measurement of health status: ascertaining the minimal clinically important difference. Control Clin Trials 1989; 10: 407-15.
34. Wells GA, Tugwell P, Kraag GR, Baker PR, Groh J, Redelmeier DA. Minimum important differences between patients with rheumatoid arthritis: the patient's perspective. J Rheumatol 1993; 20: 557-60.
35. Noseworthy JH. The challenge of long-term studies in multiple sclerosis: use of pooled data, historical controls, and observational studies to determine efficacy. In Cohen JA, Rudick RA eds. Multiple sclerosis therapeutics, second edition. London, UK: Martin Dunitz, Taylor and Francis Group, 2003: 277-88.
36. Weinshenker BG. Databases in MS research: pitfalls and promises. Mult Scler 1999; 5: 206-11.