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. 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

Neurology

University of California at San Francisco
SUNY Buffalo
Biogen IDEC
Oregon Health and Science University
G. V. (Sonny) Montgomery Veterans Affairs Medical Center
Univ. of Mississipi Medical Center
Cleveland Clinic Foundation
Women and Children's Hospital of Buffalo
Georgetown University
Walter Reed National Military Medical Center
University of Colorado Anschutz Medical Campus
Slocum-Dickson Medical Group
University of Colorado Denver
Kaiser Permanente

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

26 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
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

Access to Document

10.1177/135245859800400604

Cite this

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

@article{358468413f864c58959a014ba799612f,

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",

volume = "4",

pages = "480--486",

journal = "Multiple Sclerosis",

issn = "1352-4585",

publisher = "SAGE Publications Ltd",

number = "6",

}

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

TY - JOUR

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.

KW - Clinical trials

KW - Multiple sclerosis

KW - Outcome measures

UR - https://www.scopus.com/pages/publications/0032408995

U2 - 10.1177/135245859800400604

DO - 10.1177/135245859800400604

M3 - Article

C2 - 9987756

AN - SCOPUS:0032408995

SN - 1352-4585

VL - 4

SP - 480

EP - 486

JO - Multiple Sclerosis

JF - Multiple Sclerosis

IS - 6

ER -

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

Abstract

Keywords

Access to Document

Other files and links

Fingerprint

Cite this