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Master Thesis

Revue systématique des essais thérapeutiques de phase-I/II chez les patients porteurs d’un sarcome d’Ewing réfractaire ou en rechute : état des connaissances actuelles et pistes pour optimiser les futures recherches

Abstract : Objectives: to analyze phase-II efficiency trials evaluating new treatments in refractory/relapsed Ewing sarcomas (ES) in order to better design future trials. Methods: we performed a systematic review of clinical trials registered on the five mains international registries or referenced in PubMed, between 2005 and 2018, using the following criterion: (Ewing sarcoma OR bone sarcoma OR sarcoma) AND (Phase-I or Phase-II). Results: among the 146 trials identified, 77 were phase-I/II trials, 67 phase-II trials and 2 phase-II/III trials evaluating targeted therapy (34%), chemotherapy (24%), immunotherapy (19%) or a combination of these 3 pharmaceutical classes (23%). Only 12% of Phase-I/II trials were appropriate in terms of age inclusion criteria for the epidemiology of ES recurrence. Primary outcome was mostly RR for 116/146 (79%), PFS for 23/146(16%) and OS for 4/146 (3%). Results were published for 60 trials enrolling 930 ES patients. RR was poor (9.7%; 15CR=1.7%, 68PR=7.9%), with 32/60 (53%) trials harboring a RR=0%. SD was the best response in 175/863 patients (20%). Most of the patients had experienced PD (n=580/863; 67%). The median PFS was of 1.9 months (range 1.3-14.7 months). The median OS was of 7.6 months (range 5-30 months). There were 11/60 (18%) published trials with results considered as positive. There was no improvement of RR or survival throughout time. Conclusion: the results obtained from phase-I/II trials opened in last 13 years in patients with recurrent/refractory ES are disappointing. Our analysis supports the need to develop internationally randomized phase-II trials across all age ranges with standardized primary endpoints as PFS.
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Master Thesis
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https://dumas.ccsd.cnrs.fr/dumas-02996451
Contributor : Université Paris Descartes - Scd <>
Submitted on : Monday, November 9, 2020 - 4:17:34 PM
Last modification on : Wednesday, February 17, 2021 - 3:19:23 AM

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  • HAL Id : dumas-02996451, version 1

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Arthur Félix. Revue systématique des essais thérapeutiques de phase-I/II chez les patients porteurs d’un sarcome d’Ewing réfractaire ou en rechute : état des connaissances actuelles et pistes pour optimiser les futures recherches. Médecine humaine et pathologie. 2019. ⟨dumas-02996451⟩

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