The PROMiSe trial: Baseline data review and progress report

Jerry S. Wolinsky; Lorne Kastrukoff; Pierre Duquette; Mark Freedman; Paul O'Connor; Mark Debouverie; Catherine Lubetski; Gilles Edan; Etienne Roullet; Christian Confavreux; Alan Thompson; Lance Blumhardt; Stanley Hawkins; Thomas Scott; Daniel Wynn; Joana Cooper; Stephen Thurston; Stanton Elias; Clyde Markowitz; David Mattson; Aaron Miller; John Noseworthy; Elizabeth Shuster; Jonathan Carter; Fred Lublin; William Stuart; Michael Kaufman; Gary Birnbaum; Kottil Rammohan; Ruth Whitham; Cornelia Mihai; Steven Greenberg; Craig Smith; Mark Agius; Stan van den Noort; Lawrence Myers; James Nelson; Douglas Goodin; Barry Arnason; Khurram Bashir; Sharon Lynch

doi:10.1191/1352458504ms1034oa

The PROMiSe trial: Baseline data review and progress report

Jerry S. Wolinsky, Lorne Kastrukoff, Pierre Duquette, Mark Freedman, Paul O'Connor, Mark Debouverie, Catherine Lubetski, Gilles Edan, Etienne Roullet, Christian Confavreux, Alan Thompson, Lance Blumhardt, Stanley Hawkins, Thomas Scott, Daniel Wynn, Joana Cooper, Stephen Thurston, Stanton Elias, Clyde Markowitz, David MattsonAaron Miller, John Noseworthy, Elizabeth Shuster, Jonathan Carter, Fred Lublin, William Stuart, Michael Kaufman, Gary Birnbaum, Kottil Rammohan, Ruth Whitham, Cornelia Mihai, Steven Greenberg, Craig Smith, Mark Agius, Stan van den Noort, Lawrence Myers, James Nelson, Douglas Goodin, Barry Arnason, Khurram Bashir, Sharon Lynch

Research output: Contribution to journal › Article › peer-review

57 Scopus citations

Abstract

The PROMiSe trial is a multinational, multicentre, double-blind, placebo-controlled trial evaluating the effects of glatiramer acetate treatment over 3 years in patients with primary progressive multiple sclerosis (PPMS). A total of 943 patients were enrolled, and all those remaining on-study had completed at least 24 months as of October 2002. Baseline clinical and MRI characteristics and select correlations are reported here. A total of 3.9% of patients exhibited confirmed relapse over 1904 patient-years of exposure, indicating success of efforts to exclude relapsing MS types. Of the 26.3% of patients who have prematurely withdrawn from the study, only 36% discontinued after meeting the study primary endpoint of disease progression. The progression rate in patients in the low Expanded Disability Status Scale (EDSS) stratum (3.0-5.0) observed thus far is markedly lower than the 50% annual progression rate estimate used for determining size and statistical power of the trial; progression was observed in 16.1% of patients with 12 months of study exposure. These early findings raise some concern about the ability of the trial no demonstrate a significant treatment effect, and suggest that the short-term natural history of PPMS may not be as aggressive as previously assumed.

Original language	English (US)
Pages (from-to)	S65-S72
Journal	Multiple Sclerosis
Volume	10
Issue number	SUPPL. 1
DOIs	https://doi.org/10.1191/1352458504ms1034oa
State	Published - Jun 2004
Externally published	Yes

Keywords

Cerebrospinal fluid
Clinical trials
Diagnostic criteria
Magnetic resonance imaging
Primary progressive multiple sclerosis

ASJC Scopus subject areas

Neurology
Clinical Neurology

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10.1191/1352458504ms1034oa

Cite this

Wolinsky, J. S., Kastrukoff, L., Duquette, P., Freedman, M., O'Connor, P., Debouverie, M., Lubetski, C., Edan, G., Roullet, E., Confavreux, C., Thompson, A., Blumhardt, L., Hawkins, S., Scott, T., Wynn, D., Cooper, J., Thurston, S., Elias, S., Markowitz, C., ... Lynch, S. (2004). The PROMiSe trial: Baseline data review and progress report. Multiple Sclerosis, 10(SUPPL. 1), S65-S72. https://doi.org/10.1191/1352458504ms1034oa

Wolinsky, JS, Kastrukoff, L, Duquette, P, Freedman, M, O'Connor, P, Debouverie, M, Lubetski, C, Edan, G, Roullet, E, Confavreux, C, Thompson, A, Blumhardt, L, Hawkins, S, Scott, T, Wynn, D, Cooper, J, Thurston, S, Elias, S, Markowitz, C, Mattson, D, Miller, A, Noseworthy, J, Shuster, E, Carter, J, Lublin, F, Stuart, W, Kaufman, M, Birnbaum, G, Rammohan, K, Whitham, R, Mihai, C, Greenberg, S, Smith, C, Agius, M, van den Noort, S, Myers, L, Nelson, J, Goodin, D, Arnason, B, Bashir, K & Lynch, S 2004, 'The PROMiSe trial: Baseline data review and progress report', Multiple Sclerosis, vol. 10, no. SUPPL. 1, pp. S65-S72. https://doi.org/10.1191/1352458504ms1034oa

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abstract = "The PROMiSe trial is a multinational, multicentre, double-blind, placebo-controlled trial evaluating the effects of glatiramer acetate treatment over 3 years in patients with primary progressive multiple sclerosis (PPMS). A total of 943 patients were enrolled, and all those remaining on-study had completed at least 24 months as of October 2002. Baseline clinical and MRI characteristics and select correlations are reported here. A total of 3.9% of patients exhibited confirmed relapse over 1904 patient-years of exposure, indicating success of efforts to exclude relapsing MS types. Of the 26.3% of patients who have prematurely withdrawn from the study, only 36% discontinued after meeting the study primary endpoint of disease progression. The progression rate in patients in the low Expanded Disability Status Scale (EDSS) stratum (3.0-5.0) observed thus far is markedly lower than the 50% annual progression rate estimate used for determining size and statistical power of the trial; progression was observed in 16.1% of patients with 12 months of study exposure. These early findings raise some concern about the ability of the trial no demonstrate a significant treatment effect, and suggest that the short-term natural history of PPMS may not be as aggressive as previously assumed.",

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author = "Wolinsky, {Jerry S.} and Lorne Kastrukoff and Pierre Duquette and Mark Freedman and Paul O'Connor and Mark Debouverie and Catherine Lubetski and Gilles Edan and Etienne Roullet and Christian Confavreux and Alan Thompson and Lance Blumhardt and Stanley Hawkins and Thomas Scott and Daniel Wynn and Joana Cooper and Stephen Thurston and Stanton Elias and Clyde Markowitz and David Mattson and Aaron Miller and John Noseworthy and Elizabeth Shuster and Jonathan Carter and Fred Lublin and William Stuart and Michael Kaufman and Gary Birnbaum and Kottil Rammohan and Ruth Whitham and Cornelia Mihai and Steven Greenberg and Craig Smith and Mark Agius and {van den Noort}, Stan and Lawrence Myers and James Nelson and Douglas Goodin and Barry Arnason and Khurram Bashir and Sharon Lynch",

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AU - Wolinsky, Jerry S.

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AU - Duquette, Pierre

AU - Freedman, Mark

AU - O'Connor, Paul

AU - Debouverie, Mark

AU - Lubetski, Catherine

AU - Edan, Gilles

AU - Roullet, Etienne

AU - Confavreux, Christian

AU - Thompson, Alan

AU - Blumhardt, Lance

AU - Hawkins, Stanley

AU - Scott, Thomas

AU - Wynn, Daniel

AU - Cooper, Joana

AU - Thurston, Stephen

AU - Elias, Stanton

AU - Markowitz, Clyde

AU - Mattson, David

AU - Miller, Aaron

AU - Noseworthy, John

AU - Shuster, Elizabeth

AU - Carter, Jonathan

AU - Lublin, Fred

AU - Stuart, William

AU - Kaufman, Michael

AU - Birnbaum, Gary

AU - Rammohan, Kottil

AU - Whitham, Ruth

AU - Mihai, Cornelia

AU - Greenberg, Steven

AU - Smith, Craig

AU - Agius, Mark

AU - van den Noort, Stan

AU - Myers, Lawrence

AU - Nelson, James

AU - Goodin, Douglas

AU - Arnason, Barry

AU - Bashir, Khurram

AU - Lynch, Sharon

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KW - Clinical trials

KW - Diagnostic criteria

KW - Magnetic resonance imaging

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ER -

The PROMiSe trial: Baseline data review and progress report

Abstract

Keywords

ASJC Scopus subject areas

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