RESUMO
OBJECTIVE: To evaluate for disparities in surgical care among US children with hepatoblastoma (HB) and hepatocellular carcinoma (HCC). STUDY DESIGN: In this retrospective National Cancer Database study (2004-2015), children aged <18 years with HB or HCC were included. Multivariable mixed-effects logistic regression was used to evaluate the association of sociodemographic factors (age, sex, race and ethnicity, insurance status, income, proximity to treating hospital) with the odds of undergoing surgical treatment after adjusting for disease-related factors (tumor size, metastasis, comorbidities) and hospital-level effects. Subgroup analyses by tumor histology were performed. RESULTS: A total of 811 children were included (HB: 80.9%; HCC: 19.1%), of which 610 (75.2%) underwent surgical treatment. Following adjustment, decreased odds of undergoing surgical treatment were associated with Black race (OR: 0.46 vs White, 95% CI [95% CI]: 0.26-0.80, P = .01), and having Medicaid (OR: 0.58 vs private, 95% CI: 0.38-0.88, P = .01) or no insurance (OR: 0.33 vs private, 95% CI: 0.13-0.80, P = .02). In children with HB, Black race was associated with decreased odds of undergoing surgical treatment (OR: 0.47 vs White, 95% CI: 0.25-0.89, P = .02). In children with HCC, Medicaid (OR: 0.10 vs private, 95% CI: 0.03-0.35, P < .001), or no insurance status (OR: 0.10 vs private, 95% CI: 0.01-0.83, P = .03) were associated with decreased odds of undergoing surgical treatment. Other than metastatic disease, no additional factors were associated with likelihood of surgical treatment in any group. CONCLUSIONS: Black race and having Medicaid or no insurance are independently associated with decreased odds of surgical treatment in children with HB and HCC, respectively. These children may be less likely to undergo curative surgery for their liver cancer.
Assuntos
Carcinoma Hepatocelular , Disparidades em Assistência à Saúde , Hepatoblastoma , Neoplasias Hepáticas , Humanos , Hepatoblastoma/cirurgia , Carcinoma Hepatocelular/cirurgia , Neoplasias Hepáticas/cirurgia , Neoplasias Hepáticas/patologia , Masculino , Feminino , Criança , Estudos Retrospectivos , Pré-Escolar , Lactente , Estados Unidos , Disparidades em Assistência à Saúde/estatística & dados numéricos , Disparidades em Assistência à Saúde/etnologia , Adolescente , Fatores Sociodemográficos , Medicaid/estatística & dados numéricos , Fatores Socioeconômicos , Bases de Dados FactuaisRESUMO
OBJECTIVE: To evaluate the effect of the RAS-MAPK pathway inhibitor trametinib on medically refractory chylous effusions in 3 hospitalized patients with Noonan syndrome. STUDY DESIGN: Pharmacologic MEK1/2 inhibition has been used to treat conditions associated with Noonan syndrome, given that activation of RAS-MAPK pathway variants leads to downstream MEK activation. We describe our experience with 3 patients with Noonan syndrome (owing to variants in 3 distinct genes) and refractory chylous effusions treated successfully with MEK inhibition. A monitoring protocol was established to standardize medication dosing and monitoring of outcome measures. RESULTS: Subjects demonstrated improvement in lymphatic leak with additional findings of improved growth and normalization of cardiac and hematologic measurements. Trametinib was administered safely, with only moderate skin irritation in 1 subject. CONCLUSIONS: Improvements in a variety of quantifiable measurements highlight the potential utility of MEK1/2 inhibition in patients with Noonan syndrome and life-threatening lymphatic disease. Larger, prospective studies are needed to confirm efficacy and assess long-term safety.