دورية أكاديمية
Sunitinib for metastatic progressive phaeochromocytomas and paragangliomas. results from FIRSTMAPPP, an academic, multicentre, international, randomised, placebo-controlled, double-blind, phase 2 trial
| العنوان: | Sunitinib for metastatic progressive phaeochromocytomas and paragangliomas. results from FIRSTMAPPP, an academic, multicentre, international, randomised, placebo-controlled, double-blind, phase 2 trial |
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| المؤلفون: | Baudin, Eric, Goichot, Bernard, Berruti, Alfredo, Hadoux, Julien, Moalla, Salma, Laboureau, Sandrine, Nölting, Svenja, de la Fouchardière, Christelle, Kienitz, Tina, Deutschbein, Timo, Zovato, Stefania, Amar, Laurence, Haissaguerre, Magalie, Timmers, Henri, Niccoli, Patricia, Faggiano, Antongiulio, Angokai, Moussa, Lamartina, Livia, Luca, Florina, Cosentini, Deborah, Hahner, Stefanie, Beuschlein, Felix, Attard, Marie, Texier, Matthieu, Fassnacht, Martin |
| المساهمون: | Baudin, Eric, Goichot, Bernard, Berruti, Alfredo, Hadoux, Julien, Moalla, Salma, Laboureau, Sandrine, Nölting, Svenja, de la Fouchardière, Christelle, Kienitz, Tina, Deutschbein, Timo, Zovato, Stefania, Amar, Laurence, Haissaguerre, Magalie, Timmers, Henri, Niccoli, Patricia, Faggiano, Antongiulio, Angokai, Moussa, Lamartina, Livia, Luca, Florina, Cosentini, Deborah, Hahner, Stefanie, Beuschlein, Felix, Attard, Marie, Texier, Matthieu, Fassnacht, Martin |
| بيانات النشر: | Elsevier |
| سنة النشر: | 2024 |
| المجموعة: | Sapienza Università di Roma: CINECA IRIS |
| مصطلحات موضوعية: | phaeochromocytoma, paraganglioma, sunitinib, firstmappp |
| الوصف: | Background: No randomised controlled trial has ever been done in patients with metastatic phaeochromocytomas and paragangliomas. Preclinical and first clinical evidence suggested beneficial effects of sunitinib. We aimed to evaluate the safety and efficacy of sunitinib in patients with metastatic phaeochromocytomas and paragangliomas. Methods: FIRSTMAPPP is a multicentre, international, randomised, placebo-controlled, double-blind, phase 2 trial done at 14 academic centres across four European countries. Eligible participants were adults (aged ≥18 years) with sporadic or inherited progressive metastatic phaeochromocytomas and paragangliomas. Patients were randomly assigned (1:1) to receive either oral sunitinib (37·5 mg per day) or placebo. Randomisation was stratified according to SDHB status (mutation present vs wild type) and number of previous systemic therapies (0 vs ≥1). Primary endpoint was the rate of progression-free survival at 12 months according to real-time central review (Response Evaluation Criteria in Solid Tumours version 1.1). On the basis of a two-step Simon model, we aimed for the accrual of 78 patients, assuming a 20% improvement of the 12-month progression-free survival rate from 20% to 40%, to conclude that sunitinib is effective. Crossover from the placebo group was allowed. This trial is registered with ClinicalTrials.gov, number NCT01371201, and is closed for enrolment. Findings: From Dec 1, 2011, to Jan 31, 2019, a total of 78 patients with progressive metastatic phaeochromocytomas and paragangliomas were enrolled (39 patients per group). 25 (32%) of 78 patients had germline SDHx variants and 54 (69%) had used previous therapies. The primary endpoint was met, with a 12-month progression-free survival in 14 of 39 patients (36% [90% CI 23-50]) in the sunitinib group. In the placebo group, the 12-month progression-free survival in seven of 39 patients was 19% (90% CI 11-31), validating the hypotheses of our study design. The most frequent grade 3 or 4 adverse events were asthenia (seven ... |
| نوع الوثيقة: | article in journal/newspaper |
| اللغة: | English |
| العلاقة: | info:eu-repo/semantics/altIdentifier/pmid/38402886; volume:403; issue:10431; firstpage:1; lastpage:10; numberofpages:10; journal:THE LANCET; https://hdl.handle.net/11573/1704606Test; info:eu-repo/semantics/altIdentifier/scopus/2-s2.0-85187118655 |
| DOI: | 10.1016/S0140-6736(23)02554-0 |
| الإتاحة: | https://doi.org/10.1016/S0140-6736Test(23)02554-0 https://hdl.handle.net/11573/1704606Test |
| حقوق: | info:eu-repo/semantics/closedAccess |
| رقم الانضمام: | edsbas.BD05BD48 |
| قاعدة البيانات: | BASE |
| DOI: | 10.1016/S0140-6736(23)02554-0 |
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