Evaluation of Progression-free Survival As a Surrogate Endpoint for Overall Survival in Locally Advanced or Metastatic Differentiated Thyroid Cancer: a Systematic Review

Overview

Journal Endocrine

Specialty Endocrinology

Date 2023 Sep 13

PMID 37702900

Authors

Shijie Yang

Jing Zhan

Xiequn Xu

Affiliations

Soon will be listed here.

Abstract

Purpose: Patients with locally advanced or metastatic differentiated thyroid cancer (DTC) have a variable prognosis, and the development of more effective treatment strategies is an important research topic. Overall survival (OS) is the gold standard for research endpoints in randomized controlled trials (RCTs), but observing an OS benefit requires the inclusion of a large number of patients and a long follow-up period. In this study, we aimed to investigate whether progression-free survival (PFS) could be used as a surrogate endpoint for OS in locally advanced or metastatic DTC clinical trials.

Materials And Methods: We conducted a search in the PubMed and EMBASE databases to include all RCTs of locally advanced or metastatic DTC and extracted survival data. A weighted linear regression analysis was performed to explore the correlation between PFS benefit and OS benefit by taking the logarithm of the hazard ratios (HRs) of PFS and OS for each trial with a base of 10 and weighted by the number of patients in each RCT.

Results: Seven RCTs, including 1410 patients, were included. At the trial level, PFS benefit was weakly correlated with OS benefit (R = 0.210, 95% CI: 0.000-0.811) and did not meet the statistical criteria for the surrogate endpoint.

Conclusion: This study does not support PFS as a surrogate endpoint for OS in locally advanced or metastatic DTC clinical trials.

Trial Registration: PROSPERO Identifier: CRD42022334898.

References

Li M, Dal Maso L, Vaccarella S . Global trends in thyroid cancer incidence and the impact of overdiagnosis. Lancet Diabetes Endocrinol. 2020; 8(6):468-470. DOI: 10.1016/S2213-8587(20)30115-7. View

Lim H, Devesa S, Sosa J, Check D, Kitahara C . Trends in Thyroid Cancer Incidence and Mortality in the United States, 1974-2013. JAMA. 2017; 317(13):1338-1348. PMC: 8216772. DOI: 10.1001/jama.2017.2719. View

Schlumberger M, Leboulleux S . Current practice in patients with differentiated thyroid cancer. Nat Rev Endocrinol. 2020; 17(3):176-188. DOI: 10.1038/s41574-020-00448-z. View

Jonklaas J, Sarlis N, Litofsky D, Ain K, Bigos S, Brierley J . Outcomes of patients with differentiated thyroid carcinoma following initial therapy. Thyroid. 2007; 16(12):1229-42. DOI: 10.1089/thy.2006.16.1229. View

Shoup M, Stojadinovic A, Nissan A, Ghossein R, Freedman S, Brennan M . Prognostic indicators of outcomes in patients with distant metastases from differentiated thyroid carcinoma. J Am Coll Surg. 2003; 197(2):191-7. DOI: 10.1016/S1072-7515(03)00332-6. View

Durante C, Haddy N, Baudin E, Leboulleux S, Hartl D, Travagli J . Long-term outcome of 444 patients with distant metastases from papillary and follicular thyroid carcinoma: benefits and limits of radioiodine therapy. J Clin Endocrinol Metab. 2006; 91(8):2892-9. DOI: 10.1210/jc.2005-2838. View

Brose M, Smit J, Lin C, Tori M, Bowles D, Worden F . Multikinase Inhibitors for the Treatment of Asymptomatic Radioactive Iodine-Refractory Differentiated Thyroid Cancer: Global Noninterventional Study (RIFTOS MKI). Thyroid. 2022; 32(9):1059-1068. DOI: 10.1089/thy.2022.0061. View

Savina M, Gourgou S, Italiano A, Dinart D, Rondeau V, Penel N . Meta-analyses evaluating surrogate endpoints for overall survival in cancer randomized trials: A critical review. Crit Rev Oncol Hematol. 2018; 123:21-41. DOI: 10.1016/j.critrevonc.2017.11.014. View

Fiteni F, Westeel V, Pivot X, Borg C, Vernerey D, Bonnetain F . Endpoints in cancer clinical trials. J Visc Surg. 2014; 151(1):17-22. DOI: 10.1016/j.jviscsurg.2013.10.001. View

10.

Gion M, Perez-Garcia J, Llombart-Cussac A, Sampayo-Cordero M, Cortes J, Malfettone A . Surrogate endpoints for early-stage breast cancer: a review of the state of the art, controversies, and future prospects. Ther Adv Med Oncol. 2021; 13:17588359211059587. PMC: 8640314. DOI: 10.1177/17588359211059587. View

11.

Buyse M, Molenberghs G . Criteria for the validation of surrogate endpoints in randomized experiments. Biometrics. 1998; 54(3):1014-29. View

12.

Buyse M, Molenberghs G, Burzykowski T, Renard D, Geys H . The validation of surrogate endpoints in meta-analyses of randomized experiments. Biostatistics. 2003; 1(1):49-67. DOI: 10.1093/biostatistics/1.1.49. View

13.

Xie W, Halabi S, Tierney J, Sydes M, Collette L, Dignam J . A Systematic Review and Recommendation for Reporting of Surrogate Endpoint Evaluation Using Meta-analyses. JNCI Cancer Spectr. 2019; 3(1):pkz002. PMC: 6649812. DOI: 10.1093/jncics/pkz002. View

14.

Moher D, Liberati A, Tetzlaff J, Altman D . Preferred reporting items for systematic reviews and meta-analyses: the PRISMA statement. BMJ. 2009; 339:b2535. PMC: 2714657. DOI: 10.1136/bmj.b2535. View

15.

Higgins J, Altman D, Gotzsche P, Juni P, Moher D, Oxman A . The Cochrane Collaboration's tool for assessing risk of bias in randomised trials. BMJ. 2011; 343:d5928. PMC: 3196245. DOI: 10.1136/bmj.d5928. View

16.

Leboulleux S, Bastholt L, Krause T, De La Fouchardiere C, Tennvall J, Awada A . Vandetanib in locally advanced or metastatic differentiated thyroid cancer: a randomised, double-blind, phase 2 trial. Lancet Oncol. 2012; 13(9):897-905. DOI: 10.1016/S1470-2045(12)70335-2. View

17.

Brose M, Nutting C, Jarzab B, Elisei R, Siena S, Bastholt L . Sorafenib in radioactive iodine-refractory, locally advanced or metastatic differentiated thyroid cancer: a randomised, double-blind, phase 3 trial. Lancet. 2014; 384(9940):319-28. PMC: 4366116. DOI: 10.1016/S0140-6736(14)60421-9. View

18.

Schlumberger M, Tahara M, Wirth L, Robinson B, Brose M, Elisei R . Lenvatinib versus placebo in radioiodine-refractory thyroid cancer. N Engl J Med. 2015; 372(7):621-30. DOI: 10.1056/NEJMoa1406470. View

19.

Lin Y, Qin S, Li Z, Yang H, Fu W, Li S . Apatinib vs Placebo in Patients With Locally Advanced or Metastatic, Radioactive Iodine-Refractory Differentiated Thyroid Cancer: The REALITY Randomized Clinical Trial. JAMA Oncol. 2021; 8(2):242-250. PMC: 8678901. DOI: 10.1001/jamaoncol.2021.6268. View

20.

Del Paggio J, Berry J, Hopman W, Eisenhauer E, Prasad V, Gyawali B . Evolution of the Randomized Clinical Trial in the Era of Precision Oncology. JAMA Oncol. 2021; 7(5):728-734. PMC: 7995135. DOI: 10.1001/jamaoncol.2021.0379. View