723P - Response to systemic therapy in fumarate hydratase (FH) mutated papillary renal cell carcinoma (pRCC): Is there a winner?

Background

Hereditary Leiomyomatosis and RCC (HLRCC) is a rare autosomal dominantly inherited disorder which confers susceptibility to develop cutaneous, uterine leiomyomas and type 2 pRCC, caused by germline mutation in FH. Data on efficacy of systemic treatment (TT) in FH muted (FHmut) pRCC population is limited.

Methods

All FH mutated metastatic pRCC patients (pts) treated by systemic TT from June 2006 to February 2020 were included in this multicentric study. Clinical, laboratory and survival data were retrospectively reviewed. Disease control rate (DCR) was defined as complete response (CR) + partial response (PR) + stable disease (SD) (CR+PR+SD). Objective response (OR) was defined as CR+PR. Time to treatment failure (TTF), defined as time from TT initiation to discontinuation for any reason, was reported. Overall survival (OS) was defined as time from TT initiation to the date of last follow-up (FUP) or death. OS and TTF were estimated by Kaplan-Meier (K-M).

Results

Twenty pts with metastatic pRCC FHmut were included in the analysis. Median age was 35 (20.1 – 60.3) years, 10 (50%) were male, and 14 (70%) had prior nephrectomy. Performance status was ≥ 80% in 60%. IMDC risk group was good, intermediate and poor in 25%, 30%, and 15% respectively (3 missing); and 45% had a family story of kidney cancer. At the time of analysis, 45% pts were dead. Median OS was 41.6 months (95%IC: 30.13-53.20). Eight (40%) pts received at ≥3 lines of systemic TT. Type of systemic TT and efficacy results are described in the table. Median FUP was 41.6 months (95%IC: 25.4; 95.2). Table: 723P

Efficacy results according to type of systemic TT

Conclusions

We reported response to systemic TT from a large cohort of FHmut metastatic pRCC. The long OS observed in this rare and aggressive disease raises the interest of the role of new systemic TTs in this population. Antiangiogenics (AA) showed the highest DCR.

Clinical trial identification

Editorial acknowledgement

Legal entity responsible for the study

The authors.

Funding

Has not received any funding.

Disclosure

E. Colomba-Blameble: Honoraria (self), Honoraria (institution), Advisory/Consultancy, Research grant/Funding (self), Travel/Accommodation/Expenses: IPSEN; Honoraria (self), Advisory/Consultancy, Travel/Accommodation/Expenses: BMS; Honoraria (institution): Sanofi; Advisory/Consultancy, Travel/Accommodation/Expenses: Novartis; Honoraria (self), Advisory/Consultancy, Travel/Accommodation/Expenses: Pfizer; Honoraria (institution), Advisory/Consultancy: GSK. L. Crouzet: Honoraria (institution), Advisory/Consultancy, Travel/Accommodation/Expenses: janssen; Honoraria (institution), Advisory/Consultancy, Travel/Accommodation/Expenses: Ipsen; Honoraria (institution), Advisory/Consultancy, Travel/Accommodation/Expenses: Astellas; Travel/Accommodation/Expenses: Novartis. B. Laguerre: Honoraria (self): Pfizer; Honoraria (self): AstraZeneca; Honoraria (self), Travel/Accommodation/Expenses: Janssen; Honoraria (self): Ipsen; Honoraria (self): Roche; Honoraria (self), Travel/Accommodation/Expenses: MSD; Travel/Accommodation/Expenses: BMS; Honoraria (self): Novartis. C. Thibault: Honoraria (self), Travel/Accommodation/Expenses: Pfizer; Honoraria (self), Research grant/Funding (self), Travel/Accommodation/Expenses: AstraZeneca; Honoraria (self), Advisory/Consultancy: Sanofi; Honoraria (self), Advisory/Consultancy, Travel/Accommodation/Expenses: Janssen; Honoraria (self): Astellas. C. Vicier: Travel/Accommodation/Expenses: Astellas; Travel/Accommodation/Expenses: Pfizer; Travel/Accommodation/Expenses: Roche; Research grant/Funding (institution): BMS. G. de Velasco: Honoraria (self): Pfizer; Honoraria (self), Advisory/Consultancy: Novartis; Honoraria (self), Advisory/Consultancy: Roche; Honoraria (self), Advisory/Consultancy: BMS; Honoraria (self), Advisory/Consultancy: Ipsen. A. Fléchon: Honoraria (self), Travel/Accommodation/Expenses: Pfizer; Honoraria (self), Travel/Accommodation/Expenses: BMS; Honoraria (self), Travel/Accommodation/Expenses: Novartis; Honoraria (self), Travel/Accommodation/Expenses: Ipsen. C. Saldana: Honoraria (self), Advisory/Consultancy, Travel/Accommodation/Expenses: Ipsen; Honoraria (self), Advisory/Consultancy, Travel/Accommodation/Expenses: BMS; Honoraria (institution), Advisory/Consultancy, Travel/Accommodation/Expenses: Sanofi; Honoraria (self), Advisory/Consultancy, Travel/Accommodation/Expenses: Pfizer. B. Escudier: Honoraria (self), Advisory/Consultancy, Research grant/Funding (self), Travel/Accommodation/Expenses: BMS; Honoraria (self), Advisory/Consultancy, Travel/Accommodation/Expenses: Ipsen; Honoraria (self), Advisory/Consultancy, Speaker Bureau/Expert testimony, Travel/Accommodation/Expenses: Pfizer; Honoraria (self), Honoraria (institution), Advisory/Consultancy, Research grant/Funding (self), Travel/Accommodation/Expenses: Novartis; Honoraria (self), Advisory/Consultancy: Eusa Pharma; Honoraria (self), Advisory/Consultancy: AVEO; Advisory/Consultancy, Travel/Accommodation/Expenses: Roche; Honoraria (self), Advisory/Consultancy, Travel/Accommodation/Expenses: Genetec. L. Albiges: Honoraria (self), Honoraria (institution), Advisory/Consultancy, Travel/Accommodation/Expenses: Pfizer; Honoraria (institution), Advisory/Consultancy, Research grant/Funding (institution), Travel/Accommodation/Expenses: BMS; Honoraria (institution), Advisory/Consultancy, Speaker Bureau/Expert testimony, Research grant/Funding (institution), Travel/Accommodation/Expenses: Ipsen; Advisory/Consultancy, Travel/Accommodation/Expenses: Roche; Honoraria (institution), Advisory/Consultancy, Travel/Accommodation/Expenses: Novartis; Honoraria (institution), Advisory/Consultancy, Travel/Accommodation/Expenses: Sanofi. All other authors have declared no conflicts of interest.