Comparing the ability of various compositive outcomes to discriminate treatment effects in MS clinical trials

D. E. Goodkin; R. L. Priore; K. E. Wende; M. Campion; D. N. Bourdette; R. M. Herndon; J. S. Fischer; L. D. Jacobs; D. L. Cookfair; R. A. Rudick; J. R. Richert; A. M. Salazar; C. V. Granger; J. H. Simon; J. J. Alam; D. M. Bartoszak; J. Braiman; C. M. Brownscheidle; M. E. Coats; S. L. Cohan; D. S. Dougherty; R. P. Kinkel; M. K. Mass; F. E. Munschauer; P. M. Pullicino; B. J. Scherokman; B. Weinstock-Guttman; R. H. Whitham

doi:10.1177/135245859800400604

Comparing the ability of various compositive outcomes to discriminate treatment effects in MS clinical trials

D. E. Goodkin, R. L. Priore, K. E. Wende, M. Campion, D. N. Bourdette, R. M. Herndon, J. S. Fischer, L. D. Jacobs, D. L. Cookfair, R. A. Rudick, J. R. Richert, A. M. Salazar, C. V. Granger, J. H. Simon, J. J. Alam, D. M. Bartoszak, J. Braiman, C. M. Brownscheidle, M. E. Coats, S. L. CohanD. S. Dougherty, R. P. Kinkel, M. K. Mass, F. E. Munschauer, P. M. Pullicino, B. J. Scherokman, B. Weinstock-Guttman, R. H. Whitham

Neurology

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

25 Scopus citations

Abstract

We compared the ability of the Kurtzke Expanded Disability Status Scale (EDSS) and a composite outcome of non-physician-based measures of time to ambulate 25 feet (TA) and manual dexterity (the Box and Block Test [BBT], and 9-Hole Peg Test [9HPT]) to discriminate treatment effects in the Phase III study of interferon beta-la. A log-rank comparison of Kaplan-Meier curves by treatment group showed the non-physician-based composite of BET, 9HPT, and TA was of comparable sensitivity (P = 0.013) in discriminating sustained treatment failure as the EDSS alone (P = 0.029). The composite of BBT, 9HPT, TA, and EDSS was more sensitive (P = 0.009) in discriminating sustained treatment failure than the EDSS alone. Compositive outcomes of the EDSS and non-physician-based measures of manual dexterity and timed ambulation provide an appealing strategy to reduce the number of patients required to discriminate treatment effects in MS clinical trials.

Original language	English (US)
Pages (from-to)	480-486
Number of pages	7
Journal	Multiple Sclerosis
Volume	4
Issue number	6
DOIs	https://doi.org/10.1177/135245859800400604
State	Published - 1998

Keywords

Clinical trials
Multiple sclerosis
Outcome measures

ASJC Scopus subject areas

Neurology
Clinical Neurology

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10.1177/135245859800400604

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Goodkin, D. E., Priore, R. L., Wende, K. E., Campion, M., Bourdette, D. N., Herndon, R. M., Fischer, J. S., Jacobs, L. D., Cookfair, D. L., Rudick, R. A., Richert, J. R., Salazar, A. M., Granger, C. V., Simon, J. H., Alam, J. J., Bartoszak, D. M., Braiman, J., Brownscheidle, C. M., Coats, M. E., ... Whitham, R. H. (1998). Comparing the ability of various compositive outcomes to discriminate treatment effects in MS clinical trials. Multiple Sclerosis, 4(6), 480-486. https://doi.org/10.1177/135245859800400604

Goodkin, DE, Priore, RL, Wende, KE, Campion, M, Bourdette, DN, Herndon, RM, Fischer, JS, Jacobs, LD, Cookfair, DL, Rudick, RA, Richert, JR, Salazar, AM, Granger, CV, Simon, JH, Alam, JJ, Bartoszak, DM, Braiman, J, Brownscheidle, CM, Coats, ME, Cohan, SL, Dougherty, DS, Kinkel, RP, Mass, MK, Munschauer, FE, Pullicino, PM, Scherokman, BJ, Weinstock-Guttman, B & Whitham, RH 1998, 'Comparing the ability of various compositive outcomes to discriminate treatment effects in MS clinical trials', Multiple Sclerosis, vol. 4, no. 6, pp. 480-486. https://doi.org/10.1177/135245859800400604

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title = "Comparing the ability of various compositive outcomes to discriminate treatment effects in MS clinical trials",

abstract = "We compared the ability of the Kurtzke Expanded Disability Status Scale (EDSS) and a composite outcome of non-physician-based measures of time to ambulate 25 feet (TA) and manual dexterity (the Box and Block Test [BBT], and 9-Hole Peg Test [9HPT]) to discriminate treatment effects in the Phase III study of interferon beta-la. A log-rank comparison of Kaplan-Meier curves by treatment group showed the non-physician-based composite of BET, 9HPT, and TA was of comparable sensitivity (P = 0.013) in discriminating sustained treatment failure as the EDSS alone (P = 0.029). The composite of BBT, 9HPT, TA, and EDSS was more sensitive (P = 0.009) in discriminating sustained treatment failure than the EDSS alone. Compositive outcomes of the EDSS and non-physician-based measures of manual dexterity and timed ambulation provide an appealing strategy to reduce the number of patients required to discriminate treatment effects in MS clinical trials.",

keywords = "Clinical trials, Multiple sclerosis, Outcome measures",

author = "Goodkin, {D. E.} and Priore, {R. L.} and Wende, {K. E.} and M. Campion and Bourdette, {D. N.} and Herndon, {R. M.} and Fischer, {J. S.} and Jacobs, {L. D.} and Cookfair, {D. L.} and Rudick, {R. A.} and Richert, {J. R.} and Salazar, {A. M.} and Granger, {C. V.} and Simon, {J. H.} and Alam, {J. J.} and Bartoszak, {D. M.} and J. Braiman and Brownscheidle, {C. M.} and Coats, {M. E.} and Cohan, {S. L.} and Dougherty, {D. S.} and Kinkel, {R. P.} and Mass, {M. K.} and Munschauer, {F. E.} and Pullicino, {P. M.} and Scherokman, {B. J.} and B. Weinstock-Guttman and Whitham, {R. H.}",

year = "1998",

doi = "10.1177/135245859800400604",

language = "English (US)",

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T1 - Comparing the ability of various compositive outcomes to discriminate treatment effects in MS clinical trials

AU - Goodkin, D. E.

AU - Priore, R. L.

AU - Wende, K. E.

AU - Campion, M.

AU - Bourdette, D. N.

AU - Herndon, R. M.

AU - Fischer, J. S.

AU - Jacobs, L. D.

AU - Cookfair, D. L.

AU - Rudick, R. A.

AU - Richert, J. R.

AU - Salazar, A. M.

AU - Granger, C. V.

AU - Simon, J. H.

AU - Alam, J. J.

AU - Bartoszak, D. M.

AU - Braiman, J.

AU - Brownscheidle, C. M.

AU - Coats, M. E.

AU - Cohan, S. L.

AU - Dougherty, D. S.

AU - Kinkel, R. P.

AU - Mass, M. K.

AU - Munschauer, F. E.

AU - Pullicino, P. M.

AU - Scherokman, B. J.

AU - Weinstock-Guttman, B.

AU - Whitham, R. H.

PY - 1998

Y1 - 1998

N2 - We compared the ability of the Kurtzke Expanded Disability Status Scale (EDSS) and a composite outcome of non-physician-based measures of time to ambulate 25 feet (TA) and manual dexterity (the Box and Block Test [BBT], and 9-Hole Peg Test [9HPT]) to discriminate treatment effects in the Phase III study of interferon beta-la. A log-rank comparison of Kaplan-Meier curves by treatment group showed the non-physician-based composite of BET, 9HPT, and TA was of comparable sensitivity (P = 0.013) in discriminating sustained treatment failure as the EDSS alone (P = 0.029). The composite of BBT, 9HPT, TA, and EDSS was more sensitive (P = 0.009) in discriminating sustained treatment failure than the EDSS alone. Compositive outcomes of the EDSS and non-physician-based measures of manual dexterity and timed ambulation provide an appealing strategy to reduce the number of patients required to discriminate treatment effects in MS clinical trials.

AB - We compared the ability of the Kurtzke Expanded Disability Status Scale (EDSS) and a composite outcome of non-physician-based measures of time to ambulate 25 feet (TA) and manual dexterity (the Box and Block Test [BBT], and 9-Hole Peg Test [9HPT]) to discriminate treatment effects in the Phase III study of interferon beta-la. A log-rank comparison of Kaplan-Meier curves by treatment group showed the non-physician-based composite of BET, 9HPT, and TA was of comparable sensitivity (P = 0.013) in discriminating sustained treatment failure as the EDSS alone (P = 0.029). The composite of BBT, 9HPT, TA, and EDSS was more sensitive (P = 0.009) in discriminating sustained treatment failure than the EDSS alone. Compositive outcomes of the EDSS and non-physician-based measures of manual dexterity and timed ambulation provide an appealing strategy to reduce the number of patients required to discriminate treatment effects in MS clinical trials.

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KW - Outcome measures

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JO - Multiple Sclerosis

JF - Multiple Sclerosis

IS - 6

ER -

Comparing the ability of various compositive outcomes to discriminate treatment effects in MS clinical trials

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