e22081 Background: Targeting the hedgehog (HH) protein family pathway has consistently shown activity in locally advanced and metastatic BCC. Itraconazole is an affordable agent with known toxicity profile that demonstrates in vitro HH signaling inhibition, suggesting a possible role in BCC treatment. Initial results of a phase 2 study designed to investigate efficacy of Itraconazole as neoadjuvant treatment in patients with resectable BCC are reported. Methods: Eligible pts had biopsy-proven resectable BCC, ECOG PS 0-3, adequate organ function and measurable disease. Dosing of I consisted of 200mg twice daily for 60 days followed by surgery. Simon two-stage design was used to test a null rate of 5% vs. 20% (power = 0.80; α = 0.05, unilateral). If ≥1 of 13 pts in stage 1 have objective response (OR) according to RECIST 1.1 criteria, 14 more pts will be enrolled. If ≥4 of 27 pts have OR, the treatment is worthy of further study. Secondary endpoints include safety and change in Ki-67 and Gli1. Results: Thirteen pts with 14 measurable lesions enrolled from Jan/2018 to Feb/2019; 1 pt was not evaluable, but was included in safety analysis. Demographics and outcomes are summarized in Table. After 60 days of treatment, all patients had R0 resection as primary planned and 1 (8.3%) PR was observed. There were no progressions during the study. OR rate was 8.3%. No grade 3 AE or SAE were reported. 6 pts had grade 1 or 2 AEs at least possibly related to I including depression, headache, GPT elevation, diarrhea, abdominal pain and bilirubin elevation. Conclusions: Itraconazole showed initial anti-tumor activity in pts with resectable BCC, no safety concerns were observed. Study will proceed to Simon’s second stage. Clinical Trial Information: NCT03972748 , 47011715.0.0000.5327 . [Table: see text]
e16549 Background: Treatment of Urothelial Cancer (UC) is rapidly evolving with innovative biomarker-based approaches. Alterations in the gene encoding fibroblast growth factor receptor (FGFR) are frequent in UC and are an emerging focus for targeted therapy. However, real-world data on FGFR prevalence, as well as clinicopathological characteristics and outcomes of patients (pts) with advanced UC in Latin America is lacking. Methods: LACOG 1518 is a multicenter multinational study that retrospectively included pts diagnosed with metastatic or relapsed UC between Jan-2016 and Dec-2019. Clinicopathological characteristics, outcomes and treatment patterns were retrieved from medical records. Archival tumor samples were analyzed for FGFR alterations by NGS. Results: Two hundred thirteen pts were included across 24 centers in 4 Latin American countries. Median age was 68 years (range, 31-90), most pts (75%) had PS 0-1, and 83 (39%) pts had clinical stage IV at diagnosis. Bladder was the primary site of disease in 190 (89%) pts, 92 (43.2%) pts had histological variants of UC - 46 (21.6%) pts with adenocarcinoma differentiation and 16 (7.5%) with squamous-cell differentiation. Patients with relapsed locoregional disease not candidates for curative therapy comprised 10.3% of the sample. Among pts with metastatic disease, visceral metastases were present in 99 (51%) pts. FGFR alterations eligible for treatment with FGFR inhibitors (FGFRi) were identified in 29 (14.8%) out of 196 pts tested and included 29 mutations (14 S249C, 7 Y373C, 5 R248C and 3 G370C) and 13 fusions (8 FGFR3-TACC3, 2 FGFR3-BAIAP2L1, 2 FGFR2-BICC1 and 1 FGFR2-CASP7). Other FGFR alterations were also present in 6 (3.1%) pts. Median overall survival since initial diagnosis and advanced disease were 31.6 months (95% CI, 25.5 – 37.6) and 15.2 months (95% CI, 12.4 – 17.9), respectively. Median time to treatment failure to the first treatment line was 3.9 months (95% CI, 3.0 – 4.4). Regarding treatment patterns, 175 (82.1%) pts received first line treatment: 127 (72.6%) combined chemotherapy, 29 (16.6%) immunotherapy alone, 15 (8.6%) single agent chemotherapy, and 4 (2.3%) chemo-immunotherapy. Gemcitabine-platinum combinations were the backbone of choice in 93.7% of pts receiving combined chemotherapy. Pembrolizumab and atezolizumab were the chosen first-line agents for 10.3% and 4% of the treated pts, respectively. Considering all treatment lines, 75 (35.2%) and 3 (1.4%) pts were exposed to immunotherapy and FGFRi in the course of their disease, respectively. Conclusions: The prevalence of FGFR alterations in advanced UC in Latin America is similar to those described in other regions. Our data suggest limited access to FGFR inhibitors for the treatment of advanced UC in Latin America.
Gastrointestinal neuroectodermal tumor is a rare neoplasm that affects young population and is associated with poor prognosis. Previously named Clear cell sarcoma-like tumor of the gastrointestinal tract, due to the similar phenotypic pattern, advances in the molecular characterization of this tumor helped to differentiate it as a separate disease. Despite this, the small number of cases reported only suggests a reserved prognosis and isolated treatment recommendations based on local experiences. This case report illustrates a 37-year-old patient with constitutional symptoms diagnosed with metastatic disease treated with an aggressive approach involving preoperative chemotherapy followed by metastasis resection, with no evidence of disease recurrence after two years of follow-up.
TPS272 Background: Treatment of metastatic castration-resistant prostate cancer (mCRPC) has been steadily evolving during the last decade, but access remains a significant issue in low- and middle-income countries (LMICs). As novel therapies emerge and translate into clinical practice, the gap in treatment patterns between patients who do or do not have access to these therapies is expected to increase. The resulting disparities in outcomes are likely to be more noticeable in countries with pronounced inequality, such as Brazil. In Brazil, while a minority of patients have private insurance and have access to nearly all available treatment options, approximately 75% of patients depend on the public health system, which is unable to afford most of the recent treatment innovations for mCRPC, such as novel hormonal agents (NHA) or PARP inhibitors. LACOG 1818 was developed to investigate and compare outcomes of patients with mCRPC treated at private and public hospitals in Brazil. Methods: LACOG 1818 (NCT04962919) is a retrospective multicentric study investigating disparities in cancer treatment and survival among public and private institutions in Brazil. Patients with mCRPC diagnosed within January 2014 and December 2017 will be included and their data will be abstracted from medical records. Primary endpoint is cause-specific survival, comparing patients from private and public institutions. We estimate that 299 events are needed to detect a hazard ratio of 0.75 indicating a lower risk of prostate cancer-related death for patients treated at private institutions with a power of 80% and a bicaudate significance level of 10%. Considering a follow up of 24 months and a median cause-specific survival of 20 months in patients from public institutions, 590 patients are planned to be included. Secondary endpoints include describing comorbidities, sociodemographic and clinicopathological characteristics of patients with mCRPC; comparing overall survival of patients with mCRPC treated at public and private institutions; comparing treatments patterns of patients with mCRPC treated at public and private institutions; and describing skeleton-related complications, bone-directed treatments, and admission rates. From January 2020 to August 2022, 244 of planned 590 patients have been enrolled in 7 Brazilian centers. Additional 12 centers are still planned to open before 2023. Results are expected in the first semester of 2023. Clinical trial information: NCT04962919 .
Gastrointestinal neuroectodermal tumor is a rare neoplasm that affects young population and is associated with poor prognosis. Previously named Clear cell sarcoma-like tumor of the gastrointestinal tract, due to the similar phenotypic pattern, advances in the molecular characterization of this tumor helped to differentiate it as a separate disease. Despite this, the small number of cases reported only suggests a reserved prognosis and isolated treatment recommendations based on local experiences. This case report illustrates a 37-year-old patient with constitutional symptoms diagnosed with metastatic disease treated with an aggressive approach involving preoperative chemotherapy followed by metastasis resection, with no evidence of disease recurrence after two years of follow-up.
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