Found 54 Presentations For Request "bladder"

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Urothelial cancer

1764P - The vanishing clinical value of PD-L1 status as predictive biomarker in first-line treatment of urothelial carcinoma of the bladder

Presentation Number
1764P
Speakers
  • Alexander Tamalunas (Munich, Germany)
Date
Mon, 12.09.2022

Abstract

Background

Since the introduction of immune checkpoint inhibitors (ICI) for systemic treatment of metastatic urothelial carcinoma of the bladder (UCB) in 2017, the clinical value of programmed cell death 1 ligand 1 (PD-L1) as biomarker remains controversial. In 2018 European Medicines Agency (EMA) restricted approval of first-line pembrolizumab and atezolizumab in Cisplatin-ineligible patients to PD-L1 positive tumors, defined as either combined positive score (CPS) >/=10 or immune cell (IC) score 2/3. We aimed to address the clinical significance of PD-L1 positivity in patients with advanced UCB.

Methods

Patients with advanced UCB were prospectively enrolled, following radical cystectomy (RC) from January 2018 to December 2020 at our tertiary referral center. Clinical outcome defined as progression free survival (PFS) and overall survival (OS) on systemic treatment was analyzed using the χ2-test, Mann-Whitney-U-test, Kaplan-Meier method and log-rank test.

Results

PD-L1-status was analyzed as CPS and IC-score in 141 patients (43.5%) with high-risk (pT3–pT4 and/or N+) or metastatic UCB. While median PFS was 17.5 months (95%CI 11.0-24.1) for PD-L1+ patients who received ICI in 1-L, PD-L1- or PD-L1+ patients who received chemotherapy in 1-L had PFS of 3.8 (IQR 2.3-5.2) and 3.4 months (95%CI 2.9-3.9), respectively.Regardless of treatment, mean OS for PD-L1+ patients was 23.7 months (95%CI 14.9-32.5) vs. 18.2 (95%CI 5.7-30.7) for PD-L1- patients (p=0.592). Median OS with 1-L ICI was 27.2 months (95%CI 12.7-41.7) vs. 17.2 months (95%CI 10.9-23.5) with 1-L chemotherapy (p=0.311).

Conclusions

We could show that PFS largely depended on the kind of treatment given to patients, but observed no difference in OS regardless of treatment or PD-L1 status. However, the fact that PD-L1 negative patients responded to anti-PD-L1 therapy, underlines the need for reliable predictive biomarkers for agents targeting this pathway.

Legal entity responsible for the study

The authors.

Funding

Has not received any funding.

Disclosure

All authors have declared no conflicts of interest.

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Urothelial cancer

1771P - Genomic characteristics and clinical outcomes of HRAS-mutated urothelial bladder cancer

Presentation Number
1771P
Speakers
  • Jesus Antonio Ocejo Gallegos (Miami, United States of America)
Date
Mon, 12.09.2022

Abstract

Background

HRAS gene encodes the GTPase HRAS protein, a member of the RAS superfamily, which is enriched in high-grade urothelial bladder carcinoma (UBC). We aimed to characterize genomic and clinical outcomes of patients with HRAS mutations (HRASmt) as a possible prognostic and therapeutic biomarker.

Methods

A total of 3,426 UBC tissue samples underwent molecular profiling at Caris Life Sciences utilizing NGS of DNA. MAP kinase pathway activation and the likelihood of a tumor’s response to anti-PD(L)1 therapy were measured via MPAS and IFN signature, respectively. Wilcoxon, Fisher’s exact were used for statistical significance. Overall survival (OS) was calculated from the date of tissue collection or the start of treatment until the last contact from insurance claims. All comparisons were conducted between HRAS mutated tumors and the entire UBC general cohort (GC).

Results

HRASmt were detected in 107 patients with metastatic UBC (3.12%). Of those, 70% were detected in primary and 30% from metastatic sites. HRASmt were associated with lower TMB (5 vs 8 mut/Mb, q<0.01) and higher PD-L1 expression (55.3% vs 39.1%, q<0.01). HRASmt tumors harbored less TP53 (25.5% vs 60.1%, q<0.05), RB1 (5.3% vs 22.1%, q<0.05), FGFR3 (1.9% vs 13.9%, q<0.05), and KMT2D (14.0% vs 25.4%, q<0.05), but more BRAF mutations (14.0% vs 2.1%, q<0.05). The most frequent HRAS point mutation involved Q61 (47.6%). HRAS-Q61 mutant tumors showed higher MPAS scores (2.21 vs 0.6, q<0.001), enrichment in pathways (all q<0.05) such as EMT (0.86 vs 0.83), TGFb (0.91 vs 0.89), angiogenesis (0.78 vs 0.75) and notch signaling (0.82 vs 0.81). Patients with HRAS-Q61 point mutations experienced worse OS and prognosis compared to HRASwt tumors when treated with checkpoint inhibitors (HR 1.45, 95% 1.01-2.10; p <0.05).

Conclusions

HRAS-Q61 point mutations represent a clinically relevant mutation in UBC, as evidenced by its association with worse clinical outcomes compared to HRASwt tumors. Our transcriptomic analysis suggests HRAS-Q61 mutations were associated with lower response to immunotherapy and leads to MAPK activation, as well as modulation of important TME pathways, possibly indicating a distinct biological and clinical phenotype. Our study provides rationale for therapeutic HRAS targeting in UBC.

Legal entity responsible for the study

The authors.

Funding

Has not received any funding.

Disclosure

G. Lopes: Financial Interests, Personal, Stocks/Shares: Lucence Diagnostics; Financial Interests, Personal, Advisory Role: Pfizer, AstraZeneca. All other authors have declared no conflicts of interest.

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Urothelial cancer

1745P - Phase II study of neoadjuvant chemotherapy with 4 cycles of dose dense MVAC followed by radical surgery in Korean patients with MIBC and locally advanced urothelial carcinoma of bladder (NCT04047693)

Presentation Number
1745P
Speakers
  • Kwonoh Park (Yangsan, Korea, Republic of)
Date
Mon, 12.09.2022

Abstract

Background

Neoadjuvant chemotherapy (NAC) followed by radical cystectomy (RC) is standard for patients with muscle-invasive bladder cancer (MIBC). When previous western studies using retrospective or phase II studies showed that dd-MVAC (dose dense methotrexate, vinblastine, doxorubicin and cisplatin) for NAC was beneficial, while none was reported in Asian population. Also, there has been no study related with dd-MVAC in locally advanced urothelial carcinoma (UC). This prospective phase II study aimed to evaluate efficacy and safety of dd-MVAC in Korean MIBC or locally advanced UC patients.

Methods

Patients with MIBC (cT2-4aN0M0) or locally advanced UC (cTanyN1-3M0) eligible for RC were prospectively enrolled. The participants were treated with four cycles of dd-MVAC with pegfilgrastim every 2 weeks. The primary end point was pathologic partial response (pPR, < ypT2N0). Secondary end points were relapse-free survival (RFS) and overall survival (OS).

Results

Of 24 enrolled patients between DEC 2019 and AUG 2021, 23 were evaluable (12% cT2N0, 44% cT3-4aN0, and 44% cTanyN1-3). Eighteen patients (78%) completed four cycles of dd-MVAC, while remaining 5 patients showed early discontinuation. Dose modification (91%) and dose delay (70%) were occurred, finally, dose intensity of dd-MVAC was 79%. Nineteen patients underwent RC except 4 patients who declined. Among 19 patients, 13 (68%) were neobladder as urinary diversion, and case of 90-days readmission was none. Seven patients [37% (95% CI, 16-58)] achieved < ypT2N0. With median follow-up of 22.8 months (95% CI, 7.1-38.5), RFS was 13.5 months and OS was 23.1 months (95% CI, 17.1-29.1). According to pPR, RFS showed numerical trend (pPR, not reached, non-pPR, 11.8 months, P = 0.059) and OS showed significant difference (pPR, not reached, non-pPR 22.2 months, P=0.034).

Conclusions

Our study showed substantial efficacy and safety, despite of including patients with locally advanced UC. Patients with < ypT2N0 showed improved OS. Considering that NAC regimen using immune check-point inhibitors do not have robust evidence, dd-MVAC could still be a promising option as NAC in Asian patients with UC.

Clinical trial identification

NCT04047693, Initial release: 08-May-2019.

Legal entity responsible for the study

The authors.

Funding

Has not received any funding.

Disclosure

All authors have declared no conflicts of interest.

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Urothelial cancer

1777P - 13 genes panel validation to assess minimal residual disease through urinary tumor DNA in muscle-invasive bladder cancer patients

Presentation Number
1777P
Speakers
  • Gianluigi De Renzi (Rome, Italy)
Date
Mon, 12.09.2022

Abstract

Background

Muscle-invasive bladder cancer (MIBC) is an aggressive disease with a 5-year mortality rate of 40–50%. Standard therapy includes neoadjuvant chemotherapy (NAC) followed by radical cystectomy (RC). Unfortunately, almost 40% of the patients experience a disease recurrence after RC, due to the persistence of micro-metastatic disease (minimal residual disease, MRD). In those cases, markers predicting MRD persistence would be essential to enhance individual patient outcomes, as preoperatively determined histopathological variables are still insufficient to predict pCR. We here validated an urinary liquid biopsy test to be used for a non-invasive MRD assessment.

Methods

Our panel consist of 13 genes mutated at a rate > 7% in MIBC cases, including: KRAS, HRAS, PIK3CA, TP53, TERT, FGFR3, ERCC2, ERBB2, ATM, RB1, AKT1, CDKN1A and CDKN2A. To validate this panel, 20 MIBC treatment-naïve patients candidate to RC were enrolled and compared to 10 healthy donors. All patients provided samples (paired blood, urine and tissue) prior to RC. FFPE DNA, circulating tumor DNA (ctDNA) and urinary tumor DNA (utDNA) have been extracted through Maxwell RSC system from tissue, blood and urine samples. FFPE DNA, ctDNA and utDNA quantity and quality were assessed before sequencing, performed using Ion Torrent NGS technology.

Results

18/20 tumor tissue samples showed detectable mutations. The most frequently mutated genes in tissue samples were TERT (12/20; 60.0%) and TP53 (9/20; 45.0%). Mutations occurred less frequently (<20.0%) in the other 11 genes. We found that 88.9% of mutations found in tumor tissue were also identified in utDNA, whereas all the mutations found in utDNA were also detected in paired tumor tissues (concordance rate 90%). Conversely, the concordance between paired tissues and plasma samples was 40.5%.

Conclusions

In MIBC mutant DNA is detected more frequently in urine compared to plasma. These preliminary data pave the way for deeper investigations to define whether a non-invasive MRD assessment can avoid needless and risky surgical procedures, allowing the prediction of the MRD status, personalizing therapeutic choice and ultimately improving patients’ quality of life.

Legal entity responsible for the study

Sapienza University.

Funding

Sapienza University.

Disclosure

All authors have declared no conflicts of interest.

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Biomarkers (agnostic)

100P - Co-mutational landscape of key fibroblast growth factor receptor (FGFR) alterations in intra-hepatic cholangiocarcinoma (iCCA), bladder cancer (BC) and glioma

Presentation Number
100P
Speakers
  • Andrea Necchi (Milan, Italy)
Date
Sat, 10.09.2022

Abstract

Background

Selective tyrosine kinase inhibitors targeting FGFR1–4 genomic alterations (GAs) are in development or have been granted FDA-accelerated approval for FGFR-altered cancers (i.e. advanced iCCA and BC). Understanding FGFR inhibitor resistance mechanisms is increasingly relevant; the genomic co-mutational landscape influencing inhibitor response should be studied comprehensively.

Methods

iCCA, BC and glioma solid tissue samples underwent hybrid capture-based comprehensive genomic profiling (CGP; Foundation Medicine, Inc., Cambridge, MA, USA) to assess all classes of GAs. Tumour mutational burden (TMB; non-driver somatic mutations per megabase) was determined on up to 1.1 megabases of sequenced DNA and microsatellite instability (MSI) on up to 114 loci.

Results

Most common FGFR GAs for iCCA, BC and glioma were FGFR2 rearrangements (REs; 9.3%; n = 618/6,641), FGFR3 short variants (SVs; 13.6%; 1,051/7,739) and FGFR1 SVs (2.1%; 239/11,550), respectively. FGFR2 RE-mutant (mt) iCCAs were significantly less likely to be TMB-high (0.5% vs 4.0%; P=1.3x10-7) and MSI-high (0.3% vs 1.4%; P=0.03) vs wildtype (wt). FGFR3 SV-mt BC was significantly less likely to be TMB-high (24.7% vs 34.9%; P=3.5x10-11) and more likely to be MSI-high (1.6% vs 0.7%; P=0.004) vs wt. Across the cohort (Table), TP53 and EGFR were significantly depleted in FGFR-mt tumours vs wt. In iCCA and BC, ERBB2 and KRAS were depleted, whilst BAP1 was enriched. For iCCA and glioma, IDH1 and TERT were depleted; in glioma, ATRX and H3-3A were both enriched. CDKN2A/B, TERT and STAG2 had contrasting trends between BC and glioma.

% iCCA (n = 6,641) BC (n = 7,739) Glioma (n = 11,550)
FGFR2 RE FGFR3 SV FGFR1 SV
Mt Wt P Mt Wt P Mt Wt P
ATRX 0.5 0.6 1 1 1 1 25.1 15.3 9.1x10-5
BAP1 31.1 11.9 2.7x10-32 4.2 2.1 0.0002 0 0.2 1
CDKN2A/B 29.8 30.8 0.6 63.8 32.7 9.0x10-81 20.1 45.9 1.6x10-16
EGFR 0.8 2.3 0.01 0.8 4.8 2.3x10-12 1.7 28.5 1.7x10-28
ERBB2 0 5.6 3.7x10-15 6.8 17.5 1.1x10-21 0.4 0.2 0.38
H3-3A 0 0.1 1 0.4 0.3 0.56 26.8 3.1 6.4x10-39
IDH1 1.1 15.8 1.4x10-33 0.1 0.3 0.51 4.2 21.4 1.2x10-13
KRAS 1.3 22 1.2x10-50 1.4 6.8 6.2x10-15 2.9 2.1 0.36
NF1 0.6 2.9 0.0002 2.1 3 0.11 26.8 15.3 7.6x10-6
STAG2 0.3 0.3 1 20.7 5.8 5.5x10-49 0.4 2.6 0.035
TERT 2.3 6.6 2.7x10-6 81.6 70.3 5.2x10-15 12.1 58.6 1.3x10-49
TP53 10.8 35.8 3.2x10-42 29.5 66.6 3.9x10-114 13 39.6 3.3x10-19

Conclusions

Appropriate combination therapy may differ between FGFR-altered tumours. CGP can inform molecular-based patient stratification for future clinical trials, next-generation FGFR inhibitor development and combination therapy for FGFR-altered tumours. Prof. Necchi and Dr Murugesan are co-first authors.

Editorial acknowledgement

Research support for third-party writing assistance for this abstract, furnished by Stephen Salem, BSc, of Health Interactions, was provided by F. Hoffmann-La Roche Ltd., Basel, Switzerland.

Legal entity responsible for the study

F. Hoffmann-La Roche Ltd.

Funding

F. Hoffmann-La Roche Ltd.

Disclosure

A. Necchi: Financial Interests, Personal, Member, Steering Committee member: Astellas, AstraZeneca, Bayer, Clovis Oncology, F. Hoffmann-La Roche Ltd., Janssen, Merck; Non-Financial Interests, Institutional, Research Grant: AstraZeneca, BMS, Gilead Sciences, Inc., Ipsen, Merck; Non-Financial Interests, Personal, Leadership Role: Global Society of Rare Genitourinary Tumors (GSRGT); Non-Financial Interests, Personal, Principal Investigator, Coordinating Principal Investigator: Incyte; Non-Financial Interests, Personal, Principal Investigator, Local Principal Investigator: Pfizer; Non-Financial Interests, Personal, Funding, Research support for third-party writing assistance for this abstract, furnished by Stephen Salem, BSc, of Health Interactions, was provided by F. Hoffmann-La Roche Ltd., Basel, Switzerland: F. Hoffmann-La Roche Ltd. K. Murugesan: Financial Interests, Personal, Stocks/Shares: F. Hoffmann-La Roche Ltd.; Non-Financial Interests, Personal, Funding, Research support for third-party writing assistance for this abstract, furnished by Stephen Salem, BSc, of Health Interactions, was provided by F. Hoffmann-La Roche Ltd., Basel, Switzerland: F. Hoffmann-La Roche Ltd.; Financial Interests, Personal, Full or part-time Employment: Foundation Medicine, Inc. T. Burn: Financial Interests, Personal, Full or part-time Employment, Previous employment: Incyte Corporation; Non-Financial Interests, Personal, Funding, Research support for third-party writing assistance for this abstract, furnished by Stephen Salem, BSc, of Health Interactions, was provided by F. Hoffmann-La Roche Ltd., Basel, Switzerland. F. Hoffmann-La Roche Ltd.; Financial Interests, Personal, Stocks/Shares: Incyte Corporation, Tyra Biosciences; Financial Interests, Personal, Full or part-time Employment: Tyra Biosciences. O. Gjoerup: Financial Interests, Personal, Stocks/Shares: F. Hoffmann-La Roche Ltd.; Non-Financial Interests, Personal, Funding, Research support for third-party writing assistance for this abstract, furnished by Stephen Salem, BSc, of Health Interactions, was provided by F. Hoffmann-La Roche Ltd., Basel, Switzerland: F. Hoffmann-La Roche Ltd.; Financial Interests, Personal, Full or part-time Employment: Foundation Medicine, Inc. R. Greenstein: Financial Interests, Personal, Stocks/Shares: F. Hoffmann-La Roche Ltd.; Non-Financial Interests, Personal, Funding, Research support for third-party writing assistance for this abstract, furnished by Stephen Salem, BSc, of Health Interactions, was provided by F. Hoffmann-La Roche Ltd., Basel, Switzerland: F. Hoffmann-La Roche Ltd.; Financial Interests, Personal, Full or part-time Employment: Foundation Medicine, Inc. J.A. López: Financial Interests, Personal, Stocks/Shares: F. Hoffmann-La Roche Ltd.; Financial Interests, Personal, Full or part-time Employment: F. Hoffmann-La Roche Ltd.; Non-Financial Interests, Personal, Funding, Research support for third-party writing assistance for this abstract, furnished by Stephen Salem, BSc, of Health Interactions, was provided by F. Hoffmann-La Roche Ltd., Basel, Switzerland: F. Hoffmann-La Roche Ltd. M. Montesion: Financial Interests, Personal, Stocks/Shares: F. Hoffmann-La Roche Ltd.; Non-Financial Interests, Personal, Funding, Research support for third-party writing assistance for this abstract, furnished by Stephen Salem, BSc, of Health Interactions, was provided by F. Hoffmann-La Roche Ltd., Basel, Switzerland: F. Hoffmann-La Roche Ltd.; Financial Interests, Personal, Full or part-time Employment: Foundation Medicine, Inc. H. Nimeiri: Financial Interests, Personal, Stocks/Shares: F. Hoffmann-La Roche Ltd.; Non-Financial Interests, Personal, Funding, Research support for third-party writing assistance for this abstract, furnished by Stephen Salem, BSc, of Health Interactions, was provided by F. Hoffmann-La Roche Ltd., Basel, Switzerland: F. Hoffmann-La Roche Ltd.; Financial Interests, Personal, Full or part-time Employment: Foundation Medicine, Inc. A.R. Parikh: Non-Financial Interests, Personal, Funding, Research support for third-party writing assistance for this abstract, furnished by Stephen Salem, BSc, of Health Interactions, was provided by F. Hoffmann-La Roche Ltd., Basel, Switzerland: F. Hoffmann-La Roche Ltd.; Financial Interests, Personal and Institutional, Advisory Role, Consultancy services: Foundation Medicine, Inc. S. Roychowdhury: Non-Financial Interests, Personal, Funding, Research support for third-party writing assistance for this abstract, furnished by Stephen Salem, BSc, of Health Interactions, was provided by F. Hoffmann-La Roche Ltd., Basel, Switzerland: F. Hoffmann-La Roche Ltd.; Financial Interests, Personal, Funding, Advisory Board: AbbVie, Bayer, Merck; Financial Interests, Personal, Funding, Honoraria: IDT DNA Technologies; Financial Interests, Personal and Institutional, Funding, Advisory Board, Research Grant: Incyte, QED Therapeutics. S. Schwemmers: Financial Interests, Personal, Stocks/Shares: F. Hoffmann-La Roche Ltd.; Financial Interests, Personal, Full or part-time Employment: F. Hoffmann-La Roche Ltd.; Non-Financial Interests, Personal, Funding, Research support for third-party writing assistance for this abstract, furnished by Stephen Salem, BSc, of Health Interactions, was provided by F. Hoffmann-La Roche Ltd., Basel, Switzerland: F. Hoffmann-La Roche Ltd. I.M. Silverman: Financial Interests, Personal, Full or part-time Employment, Previous employment: Incyte Corporation; Non-Financial Interests, Personal, Funding, Research support for third-party writing assistance for this abstract, furnished by Stephen Salem, BSc, of Health Interactions, was provided by F. Hoffmann-La Roche Ltd., Basel, Switzerland: F. Hoffmann-La Roche Ltd. A. Vogel: Financial Interests, Personal, Advisory Board, Speaker, consultancy and advisory role: AstraZeneca, Bayer, BMS, BTG, Daiichi Sankyo, Eisai, Eli Lilly and Company, F. Hoffmann-La Roche Ltd., GSK, Imaging Equipment Ltd (AAA), Incyte, Ipsen, Merck, MSD, Pierre Fabre, Sanofi, Servier, Sirtex, Terumo; Non-Financial Interests, Personal, Funding, Research support for third-party writing assistance for this abstract, furnished by Stephen Salem, BSc, of Health Interactions, was provided by F. Hoffmann-La Roche Ltd., Basel, Switzerland: F. Hoffmann-La Roche Ltd.

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Urothelial cancer

1746P - Disease-free survival (DFS) and distant metastasis-free survival (DMFS) as surrogates for overall survival (OS) in adjuvant treatment of muscle-invasive bladder cancer (MIBC)

Presentation Number
1746P
Speakers
  • Cora N. Sternberg (New York, United States of America)
Date
Mon, 12.09.2022

Abstract

Background

OS is the gold standard efficacy measure in oncology, and historically a primary endpoint for majority of randomized controlled trials (RCTs) of adjuvant chemotherapy in MIBC. Despite prolonged times to observe mature OS, there is a lack of well-established surrogates that can enable earlier evaluation of efficacy.

Methods

DFS and DMFS were assessed as potential surrogates for OS using individual patient data from 9 RCTs (median follow-up: 3.5-14.6 years) comparing cisplatin-based combination chemotherapy vs observation +/- deferred chemotherapy at relapse. Strength of patient-level associations between DFS/DMFS and OS was measured by Spearman’s (ρ) rank correlation and derived from copula functions. Strength of trial-level associations between treatment effects on the surrogates and OS was measured by coefficient of determination (R2) and derived from regression, the robustness of which tested by cross validation. Primary analyses used all patients (n=1075) from all RCTs. Sensitivity analyses were performed with respect to trial follow-up and among lymph-node negative (N0) patients (n=607).

Results

At the patient level, both DFS and DMFS were strongly correlated with OS, and at the trial level, correlation was moderate for DFS, but much stronger for DMFS (Table). For both surrogates, truncating events beyond 7 years had a modest impact on both correlations (<0.01 change in ρ, <0.05 change in R2, with narrower 95% CIs), whereas limiting the focus to the N0 subgroup of patients strengthened both correlations (Table). The regression model’s 95% prediction intervals for OS hazard ratios contained the observed OS hazard ratios for all RCTs in cross-validation.

Surrogate Analysis Patient level, ρ [95% CI] Trial level, R2 [95% CI]
DFS Primary 0.89 [0.87, 0.90] 0.69 [0.34, 1.00]
N0 subgroup 0.93 [0.92, 0.95] 0.96 [0.91, 1.00]
DMFS Primary 0.91 [0.89, 0.92] 0.90 [0.74, 1.00]
N0 subgroup 0.94 [0.92, 0.95] 0.94 [0.82, 1.00]

Conclusions

DFS and particularly DMFS are potentially important surrogates for OS for patients with MIBC treated with adjuvant cisplatin-based chemotherapy; predictions from DFS are subject to higher uncertainty than those from DMFS.

Legal entity responsible for the study

IDDI.

Funding

Bristol Myers Squibb.

Disclosure

C. Sternberg: Financial Interests, Personal, Other, Honoraria or consultation fees: Astellas Pharma, AstraZeneca, Bayer, Genzyme, Gilead, Medscape, Janssen, Bristol Myers Squibb, Merck, MSD, Pfizer, Roche, Impact, Pharma, Sanofi-Genzyme, UroToday, CCO Clinical, NCI. P. Squifflet: Financial Interests, Personal, Full or part-time Employment: IDDI; Financial Interests, Institutional, Funding, Current research: Bristol Myers Squibb. S. Burdett, D. Fisher, J. Tierney: Financial Interests, Personal and Institutional, Funding, Current research: Bristol Myers Squibb. E.D. Saad: Financial Interests, Personal, Full or part-time Employment: IDDI; Financial Interests, Institutional, Funding, Current research: Bristol Myers Squibb. M. Kurt: Financial Interests, Personal, Full or part-time Employment, Employee of Bristol Myers Squibb since June-2018: Bristol Myers Squibb; Financial Interests, Personal, Stocks/Shares, Owns restricted shares of Bristol Myers Squibb since 2019: Bristol Myers Squibb; Non-Financial Interests, Project Lead: Bristol Myers Squibb. S. Teitsson: Financial Interests, Personal, Full or part-time Employment: Bristol Myers Squibb; Financial Interests, Personal, Stocks/Shares: Brystol Myers Squibb. J.R. May: Financial Interests, Personal, Full or part-time Employment: Bristol Myers Squibb; Financial Interests, Personal, Stocks/Shares: Bristol Myers Squibb. A. Zhegalik: Financial Interests, Personal, Other: Personal fees, outside the current submitted work. M.E. Buyse: Financial Interests, Personal, Officer, Chief Scientific Officer: IDDI; Financial Interests, Personal, Invited Speaker, Board Member: CluePoints; Financial Interests, Personal, Stocks/Shares: IDDI, CluePoints. All other authors have declared no conflicts of interest.

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Urothelial cancer

1778P - Comparative genomic alterations (GA) landscape in urothelial carcinoma of the bladder (UCB) in patients of South Asian ancestry (SAS)

Presentation Number
1778P
Speakers
  • Philippe E. Spiess (Tampa, United States of America)
Date
Mon, 12.09.2022

Abstract

Background

UCB has a variable prevalence worldwide. We queried whether UCB arising in pts with SAS ancestry would feature unique genomic features when compared with patients (pts) of other, predominantly European, descent.

Methods

6,107 Global clinically advanced UCB cases underwent comprehensive genomic profiling (CGP) that included ancestry classification using a proprietary ancestry calculation algorithm. All classes of GA were determined on either primary or metastatic disease biopsies using a 324-gene hybrid-capture based method which also calculates tumor mutational burden (TMB) and determines microsatellite status (MSI).

Results

Overall, 70 (1.3%) of patients with UCB were of SAS ancestry. In comparison with the Global pts, the gender distribution was similar, but pts of SAS were older (p<.0001). The GA/tumor were also similar. In addition to lower TERT GA (57% v 77%; P=.0002), pts of SAS had lower frequency of ‘targetable’ GA, including FGFR3 (8.6% v 18.4%; p=.038) as well as non-significantly lower frequency of PIK3CA (17.1% v 22.6%) and ERBB2 (11.4% v 17.9%), PTEN (1.4% v 4.4%) and TSC1 (7.1% v 9.2%). In contrast, pts of SAS had higher frequency of potential immune-oncology (IO) biomarkers, including higher mean TMB (15.5 v 10.3 mut/Mb; p=.002) and TMB > 10 mut/Mb (43.8% v 36.6%; NS).

UCB Global UCB SAS P Value
Cases 1,760 70
Male/Female 74%/26% 74%/26%
Mean age 71 77 <.0001
GA/tumor 8.17 8.78 NS
CDKN2A 38.1% 32.8% NS
TP53 60.6% 68.3% NS
RB1 20.1% 20.1% NS
TERT 77.4% 57.1% =.0002
ARID1A 24.8% 18.4% NS
FGFR3 18.4% 8.6% =.038
PIK3CA 22.6% 17.1% NS
ERBB2 17.9% 11.4% NS
PTEN 4.4% 1.4% NS
TSC1 9.2% 7.1% NS
BRCA1 2.1% 2.8% NS
BRCA2 2.6% 2.8% NS
MTAP 25.0% 15.7% =.09
CD274 1.2% 0% NS
MDM2 9.0% 10.0% NS
MSI High 0.7% 0% NS
Mean TMB 10.3 15.4 =.002
TMB≥10 mut/Mb 36.6% 43.8% NS
TMB20 mut/Mb 12.4% 12.5% NS
.

Conclusions

At less than 2.5 cases per 100,000 population, UCB is an extremely uncommon disease in SAS pts. However, when UCB occurs in the advanced setting, it seems to have unique features when compared to UCB in North America and Western Europe, including significantly reduced opportunities for targeted therapies, but potentially higher chance for response to IO-based therapy.

Legal entity responsible for the study

The authors.

Funding

Foundation Medicine Inc.

Disclosure

P. Grivas: Financial Interests, Personal, Advisory Board: AstraZeneca, Merck, Bristol Myers Squibb, Clovis Oncology, EMD Serono, Seattle Genetics, Pfizer, Janssen, Genzyme, Mirati Therapeutics, Exelixis, Genentech/Roche, GlaxoSmithKline, Immunomedics, Dyania Health, Infinity Pharmaceuticals, QED Therapeutics, 4D Pharma PLC; Financial Interests, Personal, Other, consulting: Foundation Medicine; Financial Interests, Institutional, Invited Speaker: Pfizer, Clovis Oncology, Bavarian Nordic, Immunomedics, Bristol Myers Squibb, Debiopharm Group, Merck, QED Therapeutics, GlaxoSmithKline, Mirati Therapeutics. A. Necchi: Financial Interests, Institutional, Research Grant: Merck, AstraZeneca, Ipsen, BMS, Gilead; Financial Interests, Personal, Invited Speaker: Roche, Janssen, Bayer, Astellas, AstraZeneca, Merck, Clovis Oncology; Financial Interests, Invited Speaker: Incyte, Pfizer; Non-Financial Interests, Leadership Role: Global society of Rare Genitourinary Tumors (GSRGT). N.A. Danziger: Financial Interests, Personal, Full or part-time Employment: Foundation Medicine Inc.; Financial Interests, Personal, Stocks/Shares: F. Hoffman La-Roche Ltd. D.C. Pavlick, H. Tukachinsky, R. P. Graf: Financial Interests, Personal, Full or part-time Employment: Foundation Medicine Inc.; Financial Interests, Personal, Stocks/Shares: Roche. J.S. Ross: Financial Interests, Personal, Full or part-time Employment, Medical Director: Foundation Medicine; Financial Interests, Personal, Stocks/Shares: Roche Holdings, Tango Therapeutics, Celsius Therapeutics. All other authors have declared no conflicts of interest.

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Urothelial cancer

1762P - MERINOS: Metastatic non muscle invasive urothelial carcinoma - An observational study

Presentation Number
1762P
Speakers
  • Mathilde Haberstich (Paris, France)
Date
Mon, 12.09.2022

Abstract

Background

Common evolution of non-muscle invasive bladder cancer (NMIBC) is local recurrence or progression to muscle invasive bladder cancer (MIBC). Metastatic disease usually occurs only after progression to muscle invasive disease. However, some patients (pts) with NMIBC might develop metastases without progression to MIBC (mNMIBC). Yet, characteristics and clinical evolution of this atypical presentation of bladder cancer are unknown.

Methods

In this retrospective, multicentric French study, we aimed to describe clinical characteristics of mNMIBC (synchronous or metachronous). Pts with a history of MIBC (proven or suspected, histologically or radiologically), upper tract urothelial carcinoma or intra-diverticular bladder tumour were excluded. Survival was assessed using Kaplan Meier and Cox regression model.

Results

From 2005 to 2021, 70 pts were included from 17 French hospitals. Patients were mostly men (83%); median age at diagnosis for NMIBC was 63,8 years. The majority (93% of pts) had a high or very high risk NMIBC. Overall, 63% received intravesical BCG instillations and 21% of them were BCG-unresponsive; 24% of the cohort had a cystectomy. 19% of the pts had an upfront mNMIBC whereas 81% had metachronous mNMIBC. The median time to metastatic progression was 22.2 months [19,8; 54 months]. 21% of pts had lymph nodes only metastatic disease. The median overall survival from metastatic diagnosis was 27 months [16,6; NR]. The first line treatment was platinum-based chemotherapy for 84% of pts (cisplatin for 44%, and carboplatin for 40%). The objective response rate was 67%(standard deviation 0.32) (CR: 43% – PR: 24%) and the median time to treatment failure was 12.5 months [8,3; 14,7].

Conclusions

This study highlights that NMIBC can evolve to metastatic disease (upfront or metachronous presentation) without proven progression to MIBC. Objective response to platinum-based chemotherapy in the present mNMIBC cohort compared favourably with historical data in pts with metastatic urothelial carcinoma. A molecular profiling is ongoing to better understand the oncogenesis of this aggressive subtype of NMIBC.

Legal entity responsible for the study

AP-HP.

Funding

FONCER.

Disclosure

G. Pignot: Financial Interests, Personal, Expert Testimony: Janssen, Pfizer, Roche; Financial Interests, Personal, Invited Speaker: Janssen, Astellas, BMS, Ipsen, Bayer; Financial Interests, Institutional, Invited Speaker: MSD. M. Cancel: Financial Interests, Personal, Invited Speaker: Janssen, Sanofi; Financial Interests, Institutional, Research Grant: Ipsen; Financial Interests, Institutional, Invited Speaker: Janssen. D. Maillet: Financial Interests, Other: MSD, Pfizer, Astellas. S. Oudard: Financial Interests, Personal, Advisory Board, consultancy, advisory role: Sanofi; Financial Interests, Personal, Invited Speaker, public speaking and advisory role: Janssen; Financial Interests, Personal, Advisory Board, advisory role and public speaking: AstraZeneca, MSD, Merck, Astellas, Ipsen, Pfizer, Roche, Genetech; Financial Interests, Personal, Advisory Board, Advirory board and public speaking: BMS, Bayer, Novartis. D. Pouessel: Financial Interests, Institutional, Research Grant: MSD; Financial Interests, Personal, Advisory Role: AstraZeneca, Pfizer, Merck, MSD, Astellas; Financial Interests, Speaker’s Bureau: AstraZeneca, Pfizer, MSD, BMS, Astellas, Janssen, Ipsen; Financial Interests, Personal, Other: Pfizer, MSD. C. Serrate: Financial Interests, Personal, Invited Speaker: AstraZeneca; Financial Interests, Personal, Advisory Board: Pfizer; Financial Interests, Personal, Other, formation: Janssen. L. Campedel: Financial Interests, Personal, Advisory Role: Pfizer, BMS, MSD. C. Dumont: Financial Interests, Personal, Advisory Role: Pfizer, Bristol Myers Squibb, Astellas Pharma; Financial Interests, Personal, Other: Ipsen, Pfizer, MSD, Janssen. D. Borchiellini: Financial Interests, Personal, Advisory Board: Astellas, AstraZeneca, Bayer, Bristol Myer Squibb, Ipsen, Janssen, Merck, Pfizer; Financial Interests, Institutional, Advisory Board: MSD; Financial Interests, Institutional, Invited Speaker, Clinical Research: Astellas, AstraZeneca, Bayer, Bristol Myer Squibb, Exelixis, Infinity, Janssen, MSD, Roche, Taiho Oncology. P. Barthelemy: Financial Interests, Personal, Advisory Board: BMS, MSD, Merck, Pfizer, Ipsen, Bayer, Janssen Cilag, Astellas, Novartis, Amgen; Financial Interests, Personal, Invited Speaker: AstraZeneca, Seagen. E. Colomba: Financial Interests, Other: Ipsen, BMS, Pfizer; Financial Interests, Advisory Role: Eisai, MSD, Ipsen, Pfizer, GSK, Tesaro, Sanofi, Janssen, Clovis, BMS. O. Huillard: Financial Interests, Personal, Invited Speaker: Sanofi, Ipsen, Novartis; Financial Interests, Personal, Advisory Board: Janssen, Bristol Myers Squibb, AstraZeneca, Pfizer, Eisai, Bayer. H.J. Boyle: Financial Interests, Personal, Advisory Role: Sanofi, Novartis, Janssen, Ipsen; Financial Interests, Personal, Other: Pfizer, BMS, Pfizer, Janssen, Astellas, Sanofi, Ipsen. F. Constans Schlurmann: Financial Interests, Personal, Advisory Board: BMS, Ipsen, MSD, Pfizer. F. Audenet: Financial Interests, Personal, Invited Speaker: VitaDX; Financial Interests, Personal, Advisory Board: UroDiag. C. Thibault: Financial Interests, Personal, Advisory Board: Janssen, Astellas, Sanofi, Ipsen, Pfizer, Merck, MSD, BMS, AstraZeneca; Financial Interests, Institutional, Funding: AstraZeneca, Sanofi. All other authors have declared no conflicts of interest.

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Developmental therapeutics

462P - TYRA-300: FGFR3 selective and gatekeeper agnostic

Presentation Number
462P
Speakers
  • Jacqueline Starrett (Carlsbad, United States of America)
Date
Mon, 12.09.2022

Abstract

Background

There is a critical unmet need for next generation targeted therapies with improved efficacy and tolerability for patients with metastatic urothelial carcinoma. Resistance to FGFR targeted therapies can be mediated by an acquired gatekeeper mutation, yet no approved therapies are available in this setting. TYRA-300 is an FGFR3-selective inhibitor agnostic to the gatekeeper mutation, with less hyperphosphatemia mediated by FGFR1 inhibition than pan-FGFR inhibitors in preclinical models.

Methods

TYRA-300 was evaluated in enzymatic assays and cell lines driven by FGFR3 fusions and mutations, as well as a CRISPR-engineered gatekeeper-mutant cell line. In vivo tumor growth inhibition with TYRA-300 was tested in several FGFR3-driven xenograft models.

Results

In enzymatic assays, TYRA-300 maintained potency against the gatekeeper mutants V555L/M, while approved pan-FGFR inhibitors lost at least 30-fold potency compared to wild-type IC50s. These data were confirmed in an FGFR3::TACC3 human bladder cancer cell line engineered to express the V555M gatekeeper mutation. TYRA-300 caused in vivo tumor regression in an FGFR3::TACC3 bladder cancer xenograft model as well as an FGFR3 S249C bladder cancer xenograft model when dosed either once or twice daily. In the FGFR3::TACC3-V555M xenograft model, a 77% inhibition of tumor growth was observed with TYRA-300 while a 12% tumor growth inhibition was observed with erdafitinib. TYRA-300 shows significant FGFR3 isoform selectively in a panel of Ba/F3 cell lines expressing FGFR1-4, in contrast to approved pan-FGFR inhibitors. In support of TYRA-300’s selectivity profile, a single oral dose of TYRA-300 did not substantially elevate phosphate levels in rats, while erdafitinib did increase phosphate. RNA sequencing analysis of in vitro and in vivo treated tumors identified potential biomarkers of activity that can be followed to assess TYRA-300 in future clinical studies.

Conclusions

TYRA-300 is currently under development for patients with FGFR3-altered urothelial carcinoma. TYRA-300 retains efficacy in the presence of a gatekeeper resistance mutation. Importantly, TYRA-300 is selective for FGFR3, and thus may limit toxicities observed with pan-FGFR inhibitors.

Legal entity responsible for the study

Tyra Biosciences, Inc.

Funding

Tyra Biosciences, Inc.

Disclosure

J. Starrett, E. Allen, A.M. Balcer, D.C. Bensen, I. Hoffman, R.L. Hudkins, S. Iyer, M. Neal, P. Patel: Financial Interests, Personal, Full or part-time Employment: Tyra Biosciences; Financial Interests, Personal, Stocks/Shares: Tyra Biosciences. T. Burn: Financial Interests, Personal, Full or part-time Employment: Tyra Biosciences; Financial Interests, Personal, Stocks/Shares: Tyra Biosciences, Incyte. K. Nelson: Financial Interests, Personal, Full or part-time Employment: Tyra Biosciences; Financial Interests, Personal, Stocks/Shares: Tyra Biosciences, Qpex Biopharma. C. Occhino: Financial Interests, Personal, Full or part-time Employment: Tyra Biosciences; Financial Interests, Personal, Stocks/Shares: Tyra Biosciences, Johnson & Johnson. R.V. Swanson: Financial Interests, Personal, Full or part-time Employment: Tyra Biosciences; Financial Interests, Personal, Stocks/Shares: Tyra Biosciences, Johnson & Johnson, Cidara, Antlia; Financial Interests, Personal, Advisory Role: Cidara; Financial Interests, Personal, Advisory Board: Aro. Q. Ye: Financial Interests, Personal, Full or part-time Employment: Tyra Biosciences; Financial Interests, Personal, Stocks/Shares: Tyra Biosciences.

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Urothelial cancer

1757P - Preliminary results from AVENANCE, an ongoing, noninterventional real-world, ambispective study of avelumab first-line (1L) maintenance treatment in patients (pts) with locally advanced or metastatic urothelial carcinoma (la/mUC)

Presentation Number
1757P
Speakers
  • Philippe Barthelemy (Strasbourg, France)
Date
Mon, 12.09.2022

Abstract

Background

In the JAVELIN Bladder 100 trial, patients with la/mUC that had not progressed after 1L platinum-based chemotherapy (CTx) had significantly prolonged overall survival (OS) with avelumab 1L maintenance + best supportive care (BSC) vs BSC only. In the AVENANCE study (NCT04822350), we investigated efficacy and safety in patients with la/muC treated with avelumab 1L maintenance in France.

Methods

In this ongoing study, eligible pts have la/mUC that did not progress after 1L platinum-based CTx and previous, ongoing, or planned avelumab maintenance. The primary endpoint is OS; secondary endpoints include progression-free survival (PFS), duration of treatment (DOT), and safety. Pts who started avelumab ≥6 months prior to data cutoff (January 31, 2022) were analyzed.

Results

The analysis included 267 pts (of 500 planned). Median follow-up was 11.4 months (range, 0-25.0). Median age was 73.1 years (Q1-Q3, 66.7-77.9). At start of 1L CTx (excluding pts with missing data), disease stage was metastatic in 237 pts (90.5%; visceral in 193 [81.8%]) and locally advanced in 25 (9.5%). ECOG performance status was 0-1 in 177 (85.5%) and 2-3 in 30 (14.5%). 1L CTx was gemcitabine + carboplatin in 152 (58.0%), gemcitabine + cisplatin in 82 (31.3%), and other CTx in 28 (10.7%). Median number of cycles was 5 (range, 1-10). Response to 1L CTx was complete response in 56 pts (21.6%), partial response in 144 (55.6%), and stable disease in 52 (20.1%). Median DOT with avelumab was 5.6 months (95% CI, 4.9-7.5). At data cutoff, 100 pts (37.5%) remained on treatment. The 12-month OS rate was 64.1% (95% CI, 57.1%-70.3%) and median PFS from start of avelumab was 5.7 months (95% CI, 5.1-7.9). Treatment-emergent adverse events (AEs) occurred in 142 pts (53.2%) with serious AEs in 58 (21.7%); 59 pts (22.1%) had an AE leading to treatment discontinuation.

Conclusions

These first real-world data for avelumab 1L maintenance in pts with la/mUC from AVENANCE support the findings of the JAVELIN Bladder 100 trial, and confirm the clinical activity and safety of avelumab in a heterogenous population.

Clinical trial identification

NCT04822350.

Editorial acknowledgement

Editorial support was provided by Abhijith Thippeswamy of ClinicalThinking, and was funded by Pfizer as part of an alliance between Pfizer and Merck (CrossRef Funder ID: 10.13039/100009945).

Legal entity responsible for the study

This study was sponsored by Pfizer as part of an alliance between Pfizer and Merck (CrossRef Funder ID: 10.13039/100009945).

Funding

This study was sponsored by Pfizer as part of an alliance between Pfizer and Merck (CrossRef Funder ID: 10.13039/100009945).

Disclosure

P. Barthelemy: Financial Interests, Personal, Advisory Board: BMS, MSD, Merck, Pfizer, Ipsen, Bayer, Janssen Cilag, Astellas, Novartis, Amgen; Financial Interests, Personal, Invited Speaker: AstraZeneca, Seagen. C. Thibault: Financial Interests, Institutional, Research Grant: AstraZeneca, Sanofi; Financial Interests, Personal, Advisory Role: AstraZeneca, MSD, BMS, Pfizer, Merck, Ipsen; Financial Interests, Personal, Speaker’s Bureau: Sanofi, AstraZeneca, Janssen, Astellas, MSD, BMS, IPSEN. J. Eymard: Financial Interests, Personal and Institutional, Research Grant: Pfizer, Ipsen, BMS; Financial Interests, Personal, Advisory Role: Astellas, Sanofi, Janssen, Ipsen. A. Ravaud: Financial Interests, Institutional, Research Grant: Pfizer, Merck, Ipsen; Financial Interests, Personal, Advisory Role: Pfizer, Merck, Ipsen, BMS, AstraZeneca, Eisai; Financial Interests, Personal, Other, Travelling et housing for meetings: Pfizer, Merck, Ipsen, BMS, AstraZeneca, Eisai. A. Flechon: Financial Interests, Personal, Other, Transportation: Merck, Pfizer, MSD, Gilead, Janssen. D. Pouessel: Financial Interests, Institutional, Research Grant: MSD; Financial Interests, Personal, Advisory Role: AstraZeneca, Pfizer, Merck, MSD, Astellas; Financial Interests, Personal, Speaker’s Bureau: AstraZeneca, Pfizer, MSD, BMS, Astellas, Janssen, Ipsen; Financial Interests, Personal, Other, Support for attending meetings and/or travel: Pfizer, Merck. E. Nicolas: Financial Interests, Institutional, Advisory Role: Merck. G. Denechere: Financial Interests, Personal, Full or part-time Employment: Pfizer. M. Solbes: Financial Interests, Personal, Full or part-time Employment: Merck Santé SAS, Lyon, France, an affiliate of Merck KGaA. P. Lambert: Financial Interests, Personal, Full or part-time Employment: Pfizer. Y. Loriot: Financial Interests, Personal, Advisory Board: Merck Kga, Pfizer, Gilead, Seattle Genetics, Tahio; Financial Interests, Personal, Other, lectures, Advisory Boards: MSD, AstraZeneca, Astellas, Janssen; Financial Interests, Personal, Other, Lectures, Advisory Boards: Roche, BMS; Financial Interests, Institutional, Research Grant: Janssen, Sanofi, MSD, Roche, Celsius; Financial Interests, Institutional, Invited Speaker: Janssen, Pfizer, Janssen, MSD, Janssen, Exelexis, AstraZeneca, Pfizer, Merck Kga, BMS, Astellas, Gilead, INCYTE; Financial Interests, Invited Speaker: MSD, Astellas, Gilead/Immunomedics, Tahio; Financial Interests, Personal, Invited Speaker: Basilea; Non-Financial Interests, Member: ESMO, ASCO, AACR; Non-Financial Interests, Other, Scientific Committee: ARC. All other authors have declared no conflicts of interest.

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Investigational immunotherapy

776P - Early mortality in patients with cancer treated with immunotherapy in routine practice

Presentation Number
776P
Speakers
  • Jacques Raphael (London, Canada)
Date
Mon, 12.09.2022

Abstract

Background

Early cross-over of Kaplan-Meier survival curves seen in clinical trials of immunotherapy (IO) suggests that some patients with cancer treated with IO are at risk for Early Mortality (EM). To prevent potential harm and guide best practice, we sought to estimate the proportion of patients who die soon after starting IO in the real world setting and to examine potential demographic and disease-related factors associated with EM.

Methods

We conducted a retrospective cohort study using linked health administrative data from Ontario, Canada. EM was defined as death from any cause within 60 days of IO initiation. Patients with solid tumours (melanoma, lung, bladder, head and neck or kidney cancer) treated with IO between 2011 and 2020 were included. Multivariable logistic regression was used to identify factors associated with EM. Sensitivity analyses assessed EM at 30 and 90 days.

Results

A total of 7,127 patients treated with IO were evaluated. Median age was 67 (IQR 60-74), 42% were of female sex, and 58% had lung cancer. EM at 60 days was 15% (1,076/7,127) overall with the highest mortality observed in bladder and head and neck tumors (»21% each). In multivariable analysis, older patients, patients with history of recent hospital admission or emergency department visit, receipt of chemotherapy or radiation therapy prior to IO, stage 4 disease at diagnosis, lower hemoglobin (<10 g/dl), higher white blood cell count (>11,000/mm3) and higher baseline Edmonton Symptoms Assessment System (ESAS) scores (>=2) were at a higher risk of dying within 60 days post IO start. Conversely, patients with lung and kidney cancer (compared to melanoma), lower neutrophil-to-lymphocytes ratio (NLR<5), who received steroids after IO start, and with higher body-mass index (BMI>=25) were less likely to die within 60 days post IO initiation. In a sensitivity analysis, the 30 and 90-day mortality were 7% (519/7,127) and 22% (1,583/7,127), respectively with comparable adjusted analyses results.

Conclusions

EM is common among patients treated with IO in the real-world setting and is associated with several patient and tumor characteristics. Development of a validated tool to predict EM in this setting may facilitate better patient selection for treatment with IO in routine clinical practice.

Clinical trial identification

N/A

Editorial acknowledgement

N/A

Legal entity responsible for the study

The authors.

Funding

Medical Oncology Research Fund, London Regional Cancer Program.

Disclosure

J. Raphael: Financial Interests, Personal, Advisory Board: Lilly, AstraZeneca, Merck, Novartis; Financial Interests, Personal, Invited Speaker: Roche. All other authors have declared no conflicts of interest.

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Renal cancer

1454P - Optimizing ipilimumab in RCC: Results from SAKK 07/17 (CM-980) nivolumab (N) + ipilimumab (Ipi) in mRCC

Presentation Number
1454P
Speakers
  • Frank Stenner-Liewen (Basel, Switzerland)
Date
Mon, 12.09.2022

Abstract

Background

N + Ipi is approved for 1st line treatment in advanced RCC patients (pts) with an intermediate or poor risk score (IMDC). The CheckMate 214 study (CM-214) with 4 cycles of Ipi (1mg/kg/q3w ) and N (3mg/kg/q2w) showed superior outcomes for N+Ipi compared to Sunitinib. SAKK 07/17 (CM-980) is a prospective single-stage single-arm multicenter phase II trial with 10 swiss centres and a total of 74 pts examining the treatment-related adverse events (TRAEs) by individualizing Ipi applications while preserving overall efficacy and outcome.

Methods

74 pts with metastatic clear cell RCC and all IMDC risks entered the study for 1st or 2nd line (after TKI) therapy from 12-2017 to 6-2019. 68 pts were evaluable for analysis. In cohort 1 (C1) pts. started treatment with N (240mg q2w until w20 and 480 mg q4w thereafter). After 2 weeks Ipi 1mg/kg/q6w was introduced. As soon as a radiographic complete or partial response (CR, PR) was observed, Ipi was stopped and N was continued until progression or a maximum of 2 years. In cohort 2 (C2) (pts 33-74) Ipi-rechallenge was allowed. The primary endpoint was investigator assessed objective response rate (ORR) per RECIST with a H0 ORR of 20% (one-sided significance level 0.05). Secondary endpoints included PFS, DOR, TTF, OS, AEs and TRAEs.

Results

From 68 pts, 61 received N+Ipi in 1st and 7 in 2nd line. Median age was 66 y (range 42-86). The median Ipi dose per patient was 2.5 (range 1-16). At a median follow-up of 42.5 (C1) and 20.4 (C2) months (mo) the pooled ORR was 42.6% (90% CI 32.4%-53.3%, p < 0.001 (one-sided)). The frequency of Grade ≥3 AEs and TRAEs were 77.3% and 47%, the median PFS was 8.7 mo (95% CI 6.0-11.8), median OS was 41 mo (95% CI 38.2-n.r.).

Conclusions

SAKK 07/17 was the first study to examine and report the impact of reducing Ipi in the treatment of mRCC. Here we report ORR, TRAE, PFS, OS. Despite, longer intervals of Ipi q6w and an overall lower dose of Ipi, a 47% rate of Grade 3/4 TRAE was observed. The frequency of TRAE (47%) in SAKK 07/17 was similar to those in CM-214 (46%). With 42.6% ORR, SAKK07/17 is comparable to ORR of the CM-214 trial (42.1, 39.1 and 28.8% according to risk group). Despite a similar safety profile and similar ORR, a shorter PFS and OS compared to CM-214 does not encourage routine Ipi delays or reductions.

Clinical trial identification

NCT03297593.

Legal entity responsible for the study

The authors.

Funding

BMS.

Disclosure

F. Stenner-Liewen: Financial Interests, Institutional, Advisory Board: Roche, BMS, MSD, Pfizer, IPSEN; Financial Interests, Institutional, Invited Speaker, Translational research support within a study: BMS; Non-Financial Interests, Principal Investigator, Trial PI: BMS. R. Cathomas: Financial Interests, Institutional, Advisory Board: AstraZeneca, BMS, Bayer, MSD, Roche, Sanofi, Astellas, Ipsen, Merck, Pfizer, Debiopharm; Financial Interests, Personal, Advisory Board: Janssen; Financial Interests, Personal, Invited Speaker: Janssen; Financial Interests, Institutional, Invited Speaker: Astellas. C.A. Rothermundt: Financial Interests, Institutional, Advisory Board: BMS, Roche, MSD, Merck, Novartis, Pfizer. J. Beyer: Financial Interests, Personal, Other, Co-Sponsored Educational Event: Astellas; Financial Interests, Personal, Other, Co-Sponsored Educational Event: Bayer; Financial Interests, Personal, Other, Co-Sponsored Educational Event: Janssen; Financial Interests, Personal, Other, Co-Sponsored Educational Event: Ipsen; Financial Interests, Personal, Other, Co-Sponsored Educational Event: Merck; Financial Interests, Personal, Other, Co-Sponsored Educational Event: Pfizer; Financial Interests, Personal, Other, Co-Sponsored Educational Event: Roche; Financial Interests, Personal, Other, Co-Sponsored Educational Event: AstraZeneca; Financial Interests, Personal, Other, Co-Sponsored Educational Event: Sanofi; Financial Interests, Personal, Other, Co-Sponsored Educational Event: Novartis; Financial Interests, Personal, Other, Co-Sponsored Educational Event: Bristol Myers Squibb; Financial Interests, Personal, Other, Co-Sponsored Educational Event: Amgen; Financial Interests, Personal, Other, Co-Sponsored Educational Event: Lilly. M. Kueng: Financial Interests, Personal, Advisory Board: BMS, MSD. A. Lorch: Financial Interests, Personal, Advisory Board: BMS, Merck, Pfizer, MSD, Astellas, Janssen, Roche, Ipsen, Bayer; Non-Financial Interests, Principal Investigator: PI in several phase II and III trials in the field of urooncology; Non-Financial Interests, Member: ASCO, DGHO, DKG, AIO, EAU, MAGIC, Swiss Ethics Committee, SAKK, SAKK Scientific Committee, EAU Guideline Bladder Cancer, Onkopedia Guideline Testis Cancer, Bladder cancer, German S3 Guidelines Testis Cancer, Wilsede School of Oncology; Non-Financial Interests, Other, Invited advanced training talks: SAMO Switzerland. D.R. Berthold: Financial Interests, Institutional, Invited Speaker: Astellas, Janssen; Financial Interests, Institutional, Advisory Board: MSD, Ipsen, Bayer, BMS, Roche. H. Läubli: Financial Interests, Institutional, Advisory Board: BMS, MSD. All other authors have declared no conflicts of interest.

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