Brightline-1: phase II/III trial of the MDM2–p53 antagonist BI 907828 versus doxorubicin in patients with advanced DDLPS

References

• Farid M , NgeowJ. Sarcomas associated with genetic cancer predisposition syndromes: a review. Oncologist21(8), 1002–1013 (2016).
   PubMed Web of Science ®Google Scholar
• Stiller CA , TramaA, SerrainoDet al. Descriptive epidemiology of sarcomas in Europe: report from the RARECARE project. Eur. J. Cancer49(3), 684–695 (2013).
   PubMed Web of Science ®Google Scholar
• Ducimetiere F , LurkinA, Ranchere-VinceDet al. Incidence of sarcoma histotypes and molecular subtypes in a prospective epidemiological study with central pathology review and molecular testing. PLOS ONE6(8), e20294 (2011).
   PubMed Web of Science ®Google Scholar
• Orphanet . Prevalence and incidence of rare diseases: bibliographic data (2022). Available at: www.orpha.net/orphacom/cahiers/docs/GB/Prevalence_of_rare_diseases_by_alphabetical_list.pdf (Accessed 22June2022).
   Google Scholar
• Gronchi A , MiahAB, DeiTos APet al. Soft tissue and visceral sarcomas: ESMO-EURACAN-GENTURIS Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann. Oncol.32(11), 1348–1365 (2021).
   PubMed Web of Science ®Google Scholar
• Sbaraglia M , BellanE, DeiTos AP. The 2020 WHO classification of soft tissue tumours: news and perspectives. Pathologica113(2), 70–84 (2021).
   PubMed Web of Science ®Google Scholar
• Lee ATJ , ThwayK, HuangPH, JonesRL. Clinical and molecular spectrum of liposarcoma. J. Clin. Oncol.36(2), 151–159 (2018).
   PubMed Web of Science ®Google Scholar
• Singer S , AntonescuCR, RiedelE, BrennanMF. Histologic subtype and margin of resection predict pattern of recurrence and survival for retroperitoneal liposarcoma. Ann. Surg.238(3), 358–370; discussion 370–351 (2003).
   PubMed Web of Science ®Google Scholar
• Henricks WH , ChuYC, GoldblumJR, WeissSW. Dedifferentiated liposarcoma: a clinicopathological analysis of 155 cases with a proposal for an expanded definition of dedifferentiation. Am. J. Surg. Pathol.21(3), 271–281 (1997).
   PubMed Web of Science ®Google Scholar
• Ghadimi MP , Al-ZaidT, MadewellJet al. Diagnosis, management, and outcome of patients with dedifferentiated liposarcoma systemic metastasis. Ann. Surg. Oncol.18(13), 3762–3770 (2011).
   PubMed Web of Science ®Google Scholar
• Tirumani SH , TirumaniH, JagannathanJPet al. Metastasis in dedifferentiated liposarcoma: predictors and outcome in 148 patients. Eur. J. Surg. Oncol.41(7), 899–904 (2015).
   PubMed Web of Science ®Google Scholar
• Gronchi A , ColliniP, MiceliRet al. Myogenic differentiation and histologic grading are major prognostic determinants in retroperitoneal liposarcoma. Am. J. Surg. Pathol.39(3), 383–393 (2015).
   PubMed Web of Science ®Google Scholar
• Thway K , JonesRL, NoujaimJet al. Dedifferentiated liposarcoma: updates on morphology, genetics, and therapeutic strategies. Adv. Anat. Pathol.23(1), 30–40 (2016).
   PubMed Web of Science ®Google Scholar
• Tucci JJ , DashtiNK, CatesJMM. A proposed staging system for improved prognostication of MDM2-amplified liposarcoma. Am. J. Surg. Pathol.45(1), 101–107 (2021).
   PubMed Web of Science ®Google Scholar
• Vos M , BoeveWC, van GinhovenTMet al. Impact of primary tumor location on outcome of liposarcoma patients, a retrospective cohort study. Eur. J. Surg. Oncol.45(12), 2437–2442 (2019).
   PubMed Web of Science ®Google Scholar
• Gamboa AC , GronchiA, CardonaK. Soft-tissue sarcoma in adults: an update on the current state of histiotype-specific management in an era of personalized medicine. CA Cancer J. Clin.70(3), 200–229 (2020).
   PubMed Web of Science ®Google Scholar
• Dalal KM , KattanMW, AntonescuCR, BrennanMF, SingerS. Subtype specific prognostic nomogram for patients with primary liposarcoma of the retroperitoneum, extremity, or trunk. Ann. Surg.244(3), 381–391 (2006).
   PubMed Web of Science ®Google Scholar
• Italiano A , ToulmondeM, CioffiAet al. Advanced well-differentiated/dedifferentiated liposarcomas: role of chemotherapy and survival. Ann. Oncol.23(6), 1601–1607 (2012).
   PubMed Web of Science ®Google Scholar
• Langmans C , CornillieJ, van CannTet al. Retrospective analysis of patients with advanced liposarcoma in a tertiary referral center. Oncol. Res. Treat.42(7–8), 396–404 (2019).
   PubMed Web of Science ®Google Scholar
• Savina M , LeCesne A, BlayJYet al. Patterns of care and outcomes of patients with METAstatic soft tissue SARComa in a real-life setting: the METASARC observational study. BMC Med.15(1), 78 (2017).
   PubMedGoogle Scholar
• Jones RL , FisherC, Al-MuderisO, JudsonIR. Differential sensitivity of liposarcoma subtypes to chemotherapy. Eur. J. Cancer41(18), 2853–2860 (2005).
   PubMed Web of Science ®Google Scholar
• Carvalho C , SantosRX, CardosoSet al. Doxorubicin: the good, the bad and the ugly effect. Curr. Med. Chem.16(25), 3267–3285 (2009).
   PubMed Web of Science ®Google Scholar
• Prathumsap N , ShinlapawittayatornK, ChattipakornSC, ChattipakornN. Effects of doxorubicin on the heart: from molecular mechanisms to intervention strategies. Eur. J. Pharmacol.866, 172818 (2020).
   PubMed Web of Science ®Google Scholar
• Swain SM , WhaleyFS, EwerMS. Congestive heart failure in patients treated with doxorubicin: a retrospective analysis of three trials. Cancer97(11), 2869–2879 (2003).
   PubMed Web of Science ®Google Scholar
• Bill KL , GarnettJ, MeauxIet al. SAR405838: a novel and potent inhibitor of the MDM2:p53 axis for the treatment of dedifferentiated liposarcoma. Clin. Cancer Res.22(5), 1150–1160 (2016).
   PubMed Web of Science ®Google Scholar
• Binh MB , Sastre-GarauX, GuillouLet al. MDM2 and CDK4 immunostainings are useful adjuncts in diagnosing well-differentiated and dedifferentiated liposarcoma subtypes: a comparative analysis of 559 soft tissue neoplasms with genetic data. Am. J. Surg. Pathol.29(10), 1340–1347 (2005).
   PubMed Web of Science ®Google Scholar
• Conyers R , YoungS, ThomasDM. Liposarcoma: molecular genetics and therapeutics. Sarcoma2011, 483154 (2011).
   PubMedGoogle Scholar
• Rekhi B , KarnikN, AgrawalR, ShettyO, PatkarS. Detection of MDM2 gene amplification on tissue microarray-based Fluorescence In-Situ Hybridization (FISH) in well-differentiated and dedifferentiated liposarcomas, displaying a wide morphological spectrum: a validation study at a tertiary cancer referral centre. Indian J. Pathol. Microbiol.65(1), 65–75 (2022).
   PubMed Web of Science ®Google Scholar
• Singer S , SocciND, AmbrosiniGet al. Gene expression profiling of liposarcoma identifies distinct biological types/subtypes and potential therapeutic targets in well-differentiated and dedifferentiated liposarcoma. Cancer Res.67(14), 6626–6636 (2007).
   PubMed Web of Science ®Google Scholar
• Nishio J , NakayamaS, NabeshimaK, YamamotoT. Biology and management of dedifferentiated liposarcoma: state of the art and perspectives. J. Clin. Med.10(15), 3230 (2021).
   PubMed Web of Science ®Google Scholar
• Schoffski P . Established and experimental systemic treatment options for advanced liposarcoma. Oncol. Res. Treat.45, 525–543 (2022).
   PubMed Web of Science ®Google Scholar
• Cornillie J , WozniakA, LiHet al. Anti-tumor activity of the MDM2-TP53 inhibitor BI-907828 in dedifferentiated liposarcoma patient-derived xenograft models harboring MDM2 amplification. Clin. Transl. Oncol.22(4), 546–554 (2020).
   PubMed Web of Science ®Google Scholar
• Gounder M , PatelMR, YamamotoNet al. 1548P A phase Ia/Ib, dose-escalation/expansion study of the MDM2–p53 antagonist BI 907828 in patients (pts) with advanced/metastatic sarcoma. Ann. Oncol.32(Suppl. 5), S1124–S1125 (2021).
   Google Scholar
• LoRusso P , GounderMM, PatelMRet al. A phase I dose-escalation study of the MDM2-p53 antagonist BI 907828 in patients (pts) with advanced solid tumors. J. Clin. Oncol.39(Suppl. 15), Abstr3016 (2021).
   Google Scholar
• Gounder MM , YamamotoN, PatelMRet al. A phase Ia/Ib, dose-escalation/expansion study of the MDM2–p53 antagonist BI 907828 in patients with solid tumors, including advanced/metastatic liposarcoma (LPS). J. Clin. Oncol.40(Suppl. 16), Abstr3004 (2022).
   Web of Science ®Google Scholar
• LoRusso P , GounderM, PatelMRet al. A Phase I dose-escalation study of the MDM2-p53 antagonist BI 907828 in patients with advanced solid tumors. Poster presented at the American Society of Clinical Oncology (ASCO) Annual Meeting, Virtual Format. (June 4–8, 2021).
   Google Scholar
• Yamamoto N , HafezN, TolcherAWet al. A phase Ia/Ib, dose-escalation/expansion study of BI 907828 in combination with BI 754091 (ezabenlimab) and BI 754111 in patients (pts) with advanced solid tumors. J. Clin. Oncol.40(Suppl. 16), Abstr3095 (2022).
   PubMed Web of Science ®Google Scholar
• Minasian L , RosenO, AuclairDet al. Optimizing dosing of oncology drugs. Clin. Pharmacol. Ther.96(5), 572–579 (2014).
   PubMed Web of Science ®Google Scholar
• Jenkins M , StoneA, JennisonC. An adaptive seamless phase II/III design for oncology trials with subpopulation selection using correlated survival endpoints. Pharm. Stat.10(4), 347–356 (2011).
   PubMed Web of Science ®Google Scholar
• Lehmacher W , WassmerG. Adaptive sample size calculations in group sequential trials. Biometrics55(4), 1286–1290 (1999).
   PubMed Web of Science ®Google Scholar
• Bretz F , KoenigF, BrannathW, GlimmE, PoschM. Adaptive designs for confirmatory clinical trials. Stat. Med.28(8), 1181–1217 (2009).
   PubMed Web of Science ®Google Scholar