A critical review of clinical trials in systemic lupus erythematosus

Mary Alison Mahieu*, V. Strand, L. S. Simon, P. E. Lipsky, Rosalind Ramsey-Goldman

*Corresponding author for this work

Research output: Contribution to journalReview article

18 Citations (Scopus)

Abstract

One challenge in caring for patients with systemic lupus erythematosus (SLE) is a paucity of approved therapeutics for treatment of the diverse disease manifestations. In the last 60 years, only one drug, belimumab, has been approved for SLE treatment. Critical evaluation of investigator initiated and pharma-sponsored randomized controlled trials (RCTs) highlights barriers to successful drug development in SLE, including disease heterogeneity, inadequate trial size or duration, insufficient dose finding before initiation of large trials, handling of background medications, and choice of primary endpoint. Herein we examine lessons learned from landmark SLE RCTs and subsequent advances in trial design, as well as discuss efforts to address limitations in current SLE outcome measures that will improve detection of true therapeutic responses in future RCTs.

Original languageEnglish (US)
Pages (from-to)1122-1140
Number of pages19
JournalLupus
Volume25
Issue number10
DOIs
StatePublished - Sep 1 2016

Fingerprint

Systemic Lupus Erythematosus
Clinical Trials
Randomized Controlled Trials
Therapeutics
Pharmaceutical Preparations
Research Personnel
Outcome Assessment (Health Care)

Keywords

  • Systemic lupus erythematosus
  • clinical trials
  • outcome measures

ASJC Scopus subject areas

  • Rheumatology

Cite this

Mahieu, Mary Alison ; Strand, V. ; Simon, L. S. ; Lipsky, P. E. ; Ramsey-Goldman, Rosalind. / A critical review of clinical trials in systemic lupus erythematosus. In: Lupus. 2016 ; Vol. 25, No. 10. pp. 1122-1140.
@article{a81393c2ba9f46e183ed2b43fae0bec5,
title = "A critical review of clinical trials in systemic lupus erythematosus",
abstract = "One challenge in caring for patients with systemic lupus erythematosus (SLE) is a paucity of approved therapeutics for treatment of the diverse disease manifestations. In the last 60 years, only one drug, belimumab, has been approved for SLE treatment. Critical evaluation of investigator initiated and pharma-sponsored randomized controlled trials (RCTs) highlights barriers to successful drug development in SLE, including disease heterogeneity, inadequate trial size or duration, insufficient dose finding before initiation of large trials, handling of background medications, and choice of primary endpoint. Herein we examine lessons learned from landmark SLE RCTs and subsequent advances in trial design, as well as discuss efforts to address limitations in current SLE outcome measures that will improve detection of true therapeutic responses in future RCTs.",
keywords = "Systemic lupus erythematosus, clinical trials, outcome measures",
author = "Mahieu, {Mary Alison} and V. Strand and Simon, {L. S.} and Lipsky, {P. E.} and Rosalind Ramsey-Goldman",
year = "2016",
month = "9",
day = "1",
doi = "10.1177/0961203316652492",
language = "English (US)",
volume = "25",
pages = "1122--1140",
journal = "Lupus",
issn = "0961-2033",
publisher = "SAGE Publications Ltd",
number = "10",

}

A critical review of clinical trials in systemic lupus erythematosus. / Mahieu, Mary Alison; Strand, V.; Simon, L. S.; Lipsky, P. E.; Ramsey-Goldman, Rosalind.

In: Lupus, Vol. 25, No. 10, 01.09.2016, p. 1122-1140.

Research output: Contribution to journalReview article

TY - JOUR

T1 - A critical review of clinical trials in systemic lupus erythematosus

AU - Mahieu, Mary Alison

AU - Strand, V.

AU - Simon, L. S.

AU - Lipsky, P. E.

AU - Ramsey-Goldman, Rosalind

PY - 2016/9/1

Y1 - 2016/9/1

N2 - One challenge in caring for patients with systemic lupus erythematosus (SLE) is a paucity of approved therapeutics for treatment of the diverse disease manifestations. In the last 60 years, only one drug, belimumab, has been approved for SLE treatment. Critical evaluation of investigator initiated and pharma-sponsored randomized controlled trials (RCTs) highlights barriers to successful drug development in SLE, including disease heterogeneity, inadequate trial size or duration, insufficient dose finding before initiation of large trials, handling of background medications, and choice of primary endpoint. Herein we examine lessons learned from landmark SLE RCTs and subsequent advances in trial design, as well as discuss efforts to address limitations in current SLE outcome measures that will improve detection of true therapeutic responses in future RCTs.

AB - One challenge in caring for patients with systemic lupus erythematosus (SLE) is a paucity of approved therapeutics for treatment of the diverse disease manifestations. In the last 60 years, only one drug, belimumab, has been approved for SLE treatment. Critical evaluation of investigator initiated and pharma-sponsored randomized controlled trials (RCTs) highlights barriers to successful drug development in SLE, including disease heterogeneity, inadequate trial size or duration, insufficient dose finding before initiation of large trials, handling of background medications, and choice of primary endpoint. Herein we examine lessons learned from landmark SLE RCTs and subsequent advances in trial design, as well as discuss efforts to address limitations in current SLE outcome measures that will improve detection of true therapeutic responses in future RCTs.

KW - Systemic lupus erythematosus

KW - clinical trials

KW - outcome measures

UR - http://www.scopus.com/inward/record.url?scp=84982946509&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84982946509&partnerID=8YFLogxK

U2 - 10.1177/0961203316652492

DO - 10.1177/0961203316652492

M3 - Review article

VL - 25

SP - 1122

EP - 1140

JO - Lupus

JF - Lupus

SN - 0961-2033

IS - 10

ER -