Association between treatment toxicity and outcomes in oncology clinical trials

M. V. Abola, Vinay Prasad, A. B. Jena

Research output: Contribution to journalArticle

14 Citations (Scopus)

Abstract

BACKGROUND: Whether or not toxicity predicts clinical outcomes has long been a question regarding cancer treatments. While prior studies have focused on specific cancers, therapies, and toxicities, no comprehensive evidence exists on whether treatment toxicity predicts favorable outcomes.

METHODS: We abstracted treatment toxicity and clinical outcome data from a sample of phase III oncology randomized clinical trials (n = 99 trials). We investigated whether treatments with relatively greater toxicity compared with their controls had relatively higher, lower, or equivocal rates of clinical efficacy, measured by progression-free survival (PFS) and overall survival (OS). Several toxicities were assessed (all grades, grades III/IV, cutaneous rash, gastrointestinal toxicity, and myelosuppression).

RESULTS: Toxicity and efficacy were greater among treatments than controls (e.g. 3.5 instances of all-grade toxicity per patient in treatment arms versus 2.8 instances in controls, P <0.001; mean PFS of 9.1 months across treatment arms versus 7.1 months across controls, P <0.001; mean OS of 18.6 months across treatment arms versus 16.9 months across controls, P <0.001). Across trials, greater relative treatment toxicity was strongly associated with greater PFS in treatments versus controls (P <0.001), but not OS (P = 0.44). Although higher relative rates of myelosuppression and cutaneous rash among treatments were not associated with greater treatment efficacy, greater relative gastrointestinal toxicity among treatments was associated with greater relative PFS compared with controls (P = 0.007).

CONCLUSION: Across trials, treatments with relatively greater all-grade toxicity compared with controls are associated with relatively greater PFS but not OS.

Original languageEnglish (US)
Pages (from-to)2284-2289
Number of pages6
JournalAnnals of Oncology
Volume25
Issue number11
DOIs
StatePublished - Nov 1 2014
Externally publishedYes

Fingerprint

Clinical Trials
Disease-Free Survival
Therapeutics
Survival
Exanthema
Neoplasms
Randomized Controlled Trials

Keywords

  • clinical trials
  • treatment toxicity

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Association between treatment toxicity and outcomes in oncology clinical trials. / Abola, M. V.; Prasad, Vinay; Jena, A. B.

In: Annals of Oncology, Vol. 25, No. 11, 01.11.2014, p. 2284-2289.

Research output: Contribution to journalArticle

@article{c57f7c86cd934954a3ab8fbe37ad73e9,
title = "Association between treatment toxicity and outcomes in oncology clinical trials",
abstract = "BACKGROUND: Whether or not toxicity predicts clinical outcomes has long been a question regarding cancer treatments. While prior studies have focused on specific cancers, therapies, and toxicities, no comprehensive evidence exists on whether treatment toxicity predicts favorable outcomes.METHODS: We abstracted treatment toxicity and clinical outcome data from a sample of phase III oncology randomized clinical trials (n = 99 trials). We investigated whether treatments with relatively greater toxicity compared with their controls had relatively higher, lower, or equivocal rates of clinical efficacy, measured by progression-free survival (PFS) and overall survival (OS). Several toxicities were assessed (all grades, grades III/IV, cutaneous rash, gastrointestinal toxicity, and myelosuppression).RESULTS: Toxicity and efficacy were greater among treatments than controls (e.g. 3.5 instances of all-grade toxicity per patient in treatment arms versus 2.8 instances in controls, P <0.001; mean PFS of 9.1 months across treatment arms versus 7.1 months across controls, P <0.001; mean OS of 18.6 months across treatment arms versus 16.9 months across controls, P <0.001). Across trials, greater relative treatment toxicity was strongly associated with greater PFS in treatments versus controls (P <0.001), but not OS (P = 0.44). Although higher relative rates of myelosuppression and cutaneous rash among treatments were not associated with greater treatment efficacy, greater relative gastrointestinal toxicity among treatments was associated with greater relative PFS compared with controls (P = 0.007).CONCLUSION: Across trials, treatments with relatively greater all-grade toxicity compared with controls are associated with relatively greater PFS but not OS.",
keywords = "clinical trials, treatment toxicity",
author = "Abola, {M. V.} and Vinay Prasad and Jena, {A. B.}",
year = "2014",
month = "11",
day = "1",
doi = "10.1093/annonc/mdu444",
language = "English (US)",
volume = "25",
pages = "2284--2289",
journal = "Annals of Oncology",
issn = "0923-7534",
publisher = "Oxford University Press",
number = "11",

}

TY - JOUR

T1 - Association between treatment toxicity and outcomes in oncology clinical trials

AU - Abola, M. V.

AU - Prasad, Vinay

AU - Jena, A. B.

PY - 2014/11/1

Y1 - 2014/11/1

N2 - BACKGROUND: Whether or not toxicity predicts clinical outcomes has long been a question regarding cancer treatments. While prior studies have focused on specific cancers, therapies, and toxicities, no comprehensive evidence exists on whether treatment toxicity predicts favorable outcomes.METHODS: We abstracted treatment toxicity and clinical outcome data from a sample of phase III oncology randomized clinical trials (n = 99 trials). We investigated whether treatments with relatively greater toxicity compared with their controls had relatively higher, lower, or equivocal rates of clinical efficacy, measured by progression-free survival (PFS) and overall survival (OS). Several toxicities were assessed (all grades, grades III/IV, cutaneous rash, gastrointestinal toxicity, and myelosuppression).RESULTS: Toxicity and efficacy were greater among treatments than controls (e.g. 3.5 instances of all-grade toxicity per patient in treatment arms versus 2.8 instances in controls, P <0.001; mean PFS of 9.1 months across treatment arms versus 7.1 months across controls, P <0.001; mean OS of 18.6 months across treatment arms versus 16.9 months across controls, P <0.001). Across trials, greater relative treatment toxicity was strongly associated with greater PFS in treatments versus controls (P <0.001), but not OS (P = 0.44). Although higher relative rates of myelosuppression and cutaneous rash among treatments were not associated with greater treatment efficacy, greater relative gastrointestinal toxicity among treatments was associated with greater relative PFS compared with controls (P = 0.007).CONCLUSION: Across trials, treatments with relatively greater all-grade toxicity compared with controls are associated with relatively greater PFS but not OS.

AB - BACKGROUND: Whether or not toxicity predicts clinical outcomes has long been a question regarding cancer treatments. While prior studies have focused on specific cancers, therapies, and toxicities, no comprehensive evidence exists on whether treatment toxicity predicts favorable outcomes.METHODS: We abstracted treatment toxicity and clinical outcome data from a sample of phase III oncology randomized clinical trials (n = 99 trials). We investigated whether treatments with relatively greater toxicity compared with their controls had relatively higher, lower, or equivocal rates of clinical efficacy, measured by progression-free survival (PFS) and overall survival (OS). Several toxicities were assessed (all grades, grades III/IV, cutaneous rash, gastrointestinal toxicity, and myelosuppression).RESULTS: Toxicity and efficacy were greater among treatments than controls (e.g. 3.5 instances of all-grade toxicity per patient in treatment arms versus 2.8 instances in controls, P <0.001; mean PFS of 9.1 months across treatment arms versus 7.1 months across controls, P <0.001; mean OS of 18.6 months across treatment arms versus 16.9 months across controls, P <0.001). Across trials, greater relative treatment toxicity was strongly associated with greater PFS in treatments versus controls (P <0.001), but not OS (P = 0.44). Although higher relative rates of myelosuppression and cutaneous rash among treatments were not associated with greater treatment efficacy, greater relative gastrointestinal toxicity among treatments was associated with greater relative PFS compared with controls (P = 0.007).CONCLUSION: Across trials, treatments with relatively greater all-grade toxicity compared with controls are associated with relatively greater PFS but not OS.

KW - clinical trials

KW - treatment toxicity

UR - http://www.scopus.com/inward/record.url?scp=84934281919&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84934281919&partnerID=8YFLogxK

U2 - 10.1093/annonc/mdu444

DO - 10.1093/annonc/mdu444

M3 - Article

C2 - 25193993

AN - SCOPUS:84934281919

VL - 25

SP - 2284

EP - 2289

JO - Annals of Oncology

JF - Annals of Oncology

SN - 0923-7534

IS - 11

ER -