Background Prognosis of gallbladder cancer (GBC) has not changed in the past 20 years. Comprehensive genomic profiling (CGP) carries potential to determine the actionability for multiple targets, including ERBB2, ERBB3, MET, ROSI, FGFR, and PIK3. This study evaluates the role of CGP and targeted therapies.
Methods This is a multicenter, prospective, single-arm study. All consecutive patients of unresectable and/or metastatic GBC of age ≥18 years were enrolled. Hybrid capture-based CGP was performed by Foundation Medicine CDx. All patients received first-line chemotherapy with gemcitabine–cisplatin regimen. Patients with ERBB2/3 amplification received trastuzumab with capecitabine or nab-paclitaxel, and patients with MET amplification were treated with crizotinib. For ERBB2/3 mutations, lapatinib plus capecitabine regimen was used.
Results Fifty patients were studied with a median age of 56 years (range 26–83) and a male-to-female ratio of 1:1.6. ERBB2 and ERBB3 amplification was seen in 9 (18%) and 2 (4%) patients, respectively. Four patients with ERBB2 amplification received trastuzumab and/or lapatinib, showed partial response, and maintained response beyond 12 weeks. One patient had mixed response, whereas two patients progressed on trastuzumab and lapatinib. Three patients with ERBB3 mutations showed response to lapatinib–capecitabine. One patient with MET amplification responded to crizotinib for 4 weeks. PIK3 mutations were present in 14% of cases and were independent of ERBB aberrations.
Conclusion GBC is enriched in 28% of patients with ERBB2 and ERBB3 amplifications and/or mutations. Responses are seen with lapatinib in concurrent ERBB2 mutation and amplification. ERBB3 mutation showed response to lapatinib. MET and PIK3 are new findings in GBC, which may be targeted.
Aims Adolescent and young adults (AYAs), children with cancer, and their guardians have unique psychosocial morbidities adversely effecting quality of life (QOL). This is measurable using patented tools. We analyzed epidemiological and clinicopathological patterns of solid organ cancers in this subgroup. We also assessed psychosocial morbidity and changes in QOL faced by them.
Methods All patients aged 2 to 39 years, newly diagnosed with cancer from April 2017 to March 2019 were included. Clinical history, diagnosis, staging, treatment, outcomes, and follow-up were recorded. The National Comprehensive Cancer Network (NCCN) distress thermometer and European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ C-30) were used to assess psychosocial morbidity of AYAs, children ≥ 12 years, and parents of children < 12 years. Pediatric Quality of Life Inventory (Peds QL) version 3.0 was used for children < 12 years. Data was analyzed using descriptive statistics.
Results A total of 571 patients (512 AYAs, 59 children) were enrolled. Median age was 30 years with male predominance (58.1%). Most cases (98.6%) were absent from school or work. Carcinoma breast was the most common in females (29.3%) and non-Hodgkin lymphoma in males (12.6%). 91.06% had overall NCCN distress score ≥ 4. Also, 73.81 and 79.49% had “quite a bit” or “very much” responses on functional and symptom scales, respectively, in EORTC QLQ C-30 questionnaire. Peds QL version 3.0 revealed total score ranging from 276 to 523 for each patient.
Conclusion AYAs and children with cancer are extremely vulnerable to psychological stress and morbidity. Use of well-established tools help in assessing their mental status and timely psychiatric referral can be initiated.
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