Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2022 Sep;77(3):419-424.
doi: 10.1007/s12020-022-03128-2. Epub 2022 Jul 23.

New endpoints in adrenocortical carcinoma studies: a mini review

Matthieu Faron  1   2 , Livia Lamartina  3 , Segolene Hescot  4 , Sophie Moog  3 , Frederic Deschamps  5 , Charles Roux  5 , Rosella Libe  6   7 , Jerome Durand-Labrunie  8 , Abir Al Ghuzlan  9 , Julien Hadoux  3 , Eric Baudin  3
Affiliations
Review

New endpoints in adrenocortical carcinoma studies: a mini review

Matthieu Faron et al. Endocrine. 2022 Sep.

Abstract

Purpose: Adrenocortical carcinoma (ACC) is a very rare and aggressive malignant disease. Therefore, overall survival (OS) has long been considered as the best endpoint. Yet, a unique endpoint is not optimal to take into account the heterogeneity in tumor profile and the diversification of therapeutic option. The purpose of this mini review was to describe endpoints used in the past, present and future in the field of ACC.

Methods: Pubmed and Clinicaltrial.gov were used to identify relevant studies.

Results: Before year 2000 only three endpoints were regularly used: OS, recurrence-free survival (RFS) and response rate. These endpoints were used because ACC was seen as a homogeneous diseases with a high recurrence rate and low rate of long-term survival. Since 2000; along with the apparition of new class of drug, progression-free survival (PFS) has been more and more used. Other endpoints as "time to chemotherapy" or "Progression-free survival 2" were used to evaluate multimodal therapies or treatment with a delayed action. Finally, there is a hope that in the near future, quality of life along with other patient-reported outcomes may be used more frequently.

Conclusion: While OS and PFS are currently the most used endpoints in ACC, new endpoints are needed to better take into account the challenges offered by different situations and treatment strategies.

Keywords: Adrenocortical carcinoma; Endpoints; PRO; QOL; Surrogate.

PubMed Disclaimer

Comment in

References

    1. FDA-NIH Biomarker Working Group. BEST (Biomarkers, EndpointS, and Other Tools) Resource (Food and Drug Administration (US), Silver Spring (MD), 2016)
    1. T. Burzykowski, G. Molenberghs, M. Buyse, H. Geys, D. Renard, Validation of surrogate end points in multiple randomized clinical trials with failure time end points. J. R. Stat. Soc.: Ser. C. (Appl. Stat.) 50, 405–422 (2001). https://doi.org/10.1111/1467-9876.00244 - DOI
    1. M. Buyse, G. Molenberghs, T. Burzykowski, D. Renard, H. Geys, The validation of surrogate endpoints in meta-analyses of randomized experiments. Biostatistics 1, 49–67 (2000). https://doi.org/10.1093/biostatistics/1.1.49 - DOI - PubMed
    1. M. Fassnacht, M. Terzolo, B. Allolio, E. Baudin, H. Haak, A. Berruti et al. Combination chemotherapy in advanced adrenocortical carcinoma. N. Engl. J. Med. 366, 2189–2197 (2012). https://doi.org/10.1056/NEJMoa1200966 - DOI - PubMed
    1. M. Terzolo, M. Fassnacht, P. Perotti, R. Libe, A. Lacroix, D. Kastelan et al. Results of the ADIUVO study, the first randomized trial on adjuvant mitotane in adrenocortical carcinoma patients. J. Endocr. Soc. 5, A166–A167 (2021). https://doi.org/10.1210/jendso/bvab048.336 - DOI - PMC

LinkOut - more resources