Real-world treatment patterns, clinical outcomes, and health care resource utilization in advanced unresectable hepatocellular carcinoma.

BACKGROUND: The incidence of advanced unresectable hepatocellular carcinoma (HCC) is increasing in developed countries and the prognosis of advanced HCC remains poor. Real-world evidence of treatment patterns and outcomes can highlight the unmet clinical need. METHODS: We conducted a retrospective population-based cohort study of patients with advanced unresectable HCC diagnosed in Alberta, Canada (2008-2018) using electronic medical records and administrative claims data. A chart review was conducted on patients treated with systemic therapy to capture additional information related to treatment. RESULTS: A total of 1,297 advanced HCC patients were included of whom 555 (42.8%) were recurrent cases and the remainder were unresectable at diagnosis. Median age at diagnosis was 64 (range 21-94) years and 82.1% were men. Only 274 patients (21.1%) received first-line systemic therapy and, of those, 32 patients (11.7%) initiated second-line therapy. Nearly all of the patients received sorafenib (>96.4%) in first-line, and these patients had considerably higher median survival (12.23 months; 95% CI 10.72-14.10) compared with patients not treated with systemic therapy (2.66 months; 95% CI 2.33-3.12; log-rank p <0.001). Among patients treated with systemic therapy, overall survival was higher for recurrent cases, patients with Child-Pugh A functional status, and patients with HCV or multiple known HCC risk factors (p <0.05). CONCLUSIONS: In a Canadian real-world setting, patients who received systemic therapy had greater survival than those who did not, but outcomes were universally poor. These results underscore the need for effective front-line therapeutic options.

Real-World Treatment Patterns, Clinical Outcomes, and Health Care Resource Utilization in Extensive-Stage Small Cell Lung Cancer in Canada.

The prognosis for extensive-stage small cell lung cancer (ES-SCLC) is poor. Real-world evidence can highlight the unmet clinical need within this population. We conducted a population-based cohort study of ES-SCLC patients diagnosed in a large Canadian province (2010-2018) using electronic medical records and administrative claims data. In all, 1941 ES-SCLC patients were included, of which 476 (25%) were recurrent cases. Median age at diagnosis was 70 years (range: 39-94) and 50.2% were men. Of the 1941 ES-SCLC patients, 29.5% received chemotherapy and radiotherapy, 17.0% chemotherapy alone, 8.7% radiotherapy alone, and 44.8% received best supportive care. Chemotherapy was initiated by 46.5%, 8.5%, and 1.4% of first-, second-, and third-line patients, with lower uptake for recurrent cases. Median survival from first-, second-, and third-line chemotherapy was 7.82 months (95% CI: 7.50-8.22), 5.72 months (95% CI: 4.90-6.87), and 3.83 months (95% CI: 2.99-4.60). Among patients who received first-line therapy, the 2-year and 5-year survival was 7.3% (95% CI: 5.7-9.2) and 2.9% (95% CI: 1.8-4.5). In conclusion, initiation of first-line treatment in ES-SCLC was low with significant attrition in subsequent lines. These results underscore the need for effective front-line treatments and highlight the potential for novel therapies to improve patient outcomes.

Glucagon-like peptide-1 receptor agonists compared with basal insulins for the treatment of type 2 diabetes mellitus: a systematic review and meta-analysis.

AIMS: Since 2005, several glucagon-like peptide-1 receptor agonists (GLP-1 RAs) have been approved to treat people with type 2 diabetes. These agents are considered for use at the same point in the treatment paradigm as basal insulins. A comprehensive comparison of these drug classes, therefore, can help inform treatment decisions. This systematic review and meta-analysis assessed the clinical efficacy and safety of GLP-1 RAs compared with basal insulins. MATERIALS AND METHODS: MEDLINE, EMBASE, CENTRAL and PubMed databases were searched. Randomized clinical trials (RCTs) of ≥16 weeks' duration comparing GLP-1 RAs vs basal insulins in adults with type 2 diabetes inadequately controlled with oral antihyperglycemic drugs were included. Data on the change from baseline to 26 weeks (±10 weeks) of treatment in hemoglobin A1c (HbA1c) and weight, as well as the proportion of patients experiencing hypoglycaemia, were extracted. Fixed-effect pairwise meta-analyses were conducted where data were available from ≥2 studies. RESULTS: Fifteen RCTs were identified and 11 were meta-analysed. The once-weekly GLP-1 RAs, exenatide long acting release (LAR) and dulaglutide, led to greater, statistically significant mean HbA1c reductions vs basal insulins (exenatide: -0.31% [95% confidence interval -0.42, -0.19], dulaglutide: -0.39% [-0.49, -0.29]) whilst once-daily liraglutide and twice-daily exenatide did not (liraglutide: 0.06% [-0.06, 0.18], exenatide: 0.01% [-0.11, 0.13]). Mean weight reduction was seen with all GLP-1 RAs while mean weight gain was seen with basal insulins. Interpretation of the analysis of hypoglycaemia was limited by inconsistent definitions and reporting. Because of the limited number of available studies sensitivity analyses to explore heterogeneity could not be conducted. CONCLUSIONS: Although weight reduction is seen with all GLP-1 RA's, only the once-weekly agents, exenatide LAR and dulaglutide, demonstrate significant HbA1c reductions when compared to basal insulins.

The Logistics and Coordination of Respiratory Syncytial Virus Immunoprophylaxis Use Among US Pediatric Specialists.

This study was conducted to survey US pediatric specialists about administration of respiratory syncytial virus (RSV) immunoprophylaxis, communication patterns among physicians and parents, and barriers to access. Separate surveys were sent to neonatologists, pediatricians, pediatric pulmonologists, and pediatric cardiologists. Most physicians (≥93.5%) routinely recommended immunoprophylaxis to high-risk children. Most respondents (≥71.8%) reported that >50.0% of eligible infants and young children received each monthly dose throughout the RSV season, with the first dose most commonly administered before discharge from the birth hospitalization. To ensure receipt of subsequent doses, specialists frequently scheduled a follow-up visit at the end of the current appointment. All specialists reported insurance denials as the biggest obstacle to the administration of immunoprophylaxis to high-risk children. These findings may be used to improve adherence to immunoprophylaxis by enhancing education and physician-parent communications about severe RSV disease prevention, and by reducing known barriers to use of this preventive therapy.

Examining factors associated with self-management skills in teenage survivors of cancer.

BACKGROUND: Monitoring long-term health of teenage cancer survivors is dependent on successful transition from pediatric to adult long-term follow-up (LTFU) care. This study identified factors associated with self-management skills (SMSs), an important correlate of successful transition. METHODS: Data were collected from a cross-sectional survey conducted at three Canadian hospitals between July 2011 and January 2012. The sample included 184 childhood cancer survivors aged between 15 and 19 years. Independent factors included demographic- and illness-related factors. The outcome of interest was SMSs, measured using the SMSs scale, with higher scores indicating more SMSs. RESULTS: More SMSs were associated positively with older age (ß = 1.2, 95 % confidence interval (CI) = 0.1 to 2.4), being female (ß = 4.6, 95 % CI = 1.9 to 7.4), and having a non-married parent (ß = 5.2, 95 % CI = 0.04 to 10.4). There was a negative association between SMSs and having had a central nervous system tumor (CNS) compared to having leukemia (ß = -7.9, 95 % CI = -13.5 to -2.2). CONCLUSIONS: Younger, male, and CNS tumor survivors lack SMSs. Future research is needed to explore the extent and nature of associations between SMSs and parents' marital status. IMPLICATIONS FOR CANCER SURVIVORS: Younger, male, and CNS tumor survivors should be targeted for interventions in order to ensure that adequate SMSs are attained before completion of transition.

Perceived Risk of Severe Respiratory Syncytial Virus Disease and Immunoprophylaxis Use Among US Pediatric Specialists.

This study was conducted to assess the perception of US pediatric specialists of respiratory syncytial virus (RSV) disease risk and determine their clinical practices regarding immunoprophylaxis for high-risk children. Separate surveys were sent to neonatologists, pediatricians, pediatric pulmonologists, and pediatric cardiologists. Data were collected using structured questions requiring quantitative responses. Most neonatologists and pediatricians (>82.7%) reported a high clinical need for RSV immunoprophylaxis in preterm infants <32 weeks' gestational age. Pediatric pulmonologists and pediatric cardiologists suggested that health conditions indicative of chronic lung disease of prematurity and hemodynamically significant congenital heart disease, respectively, confer eligibility for RSV immunoprophylaxis. Agreement with the changes in the 2014 American Academy of Pediatrics guidance for RSV immunoprophylaxis was mixed among respondents from the 4 specialties. Survey findings may provide a basis to improve education about risk for severe RSV disease and evaluate changes in physician use of RSV immunoprophylaxis based on the 2014 guidance.

Factors associated with childhood cancer survivors' knowledge about their diagnosis, treatment, and risk for late effects.

BACKGROUND: While most children with cancer survive their initial disease, cancer therapy places them at risk for late effects (LE). Knowledge of their diagnosis, treatment, and LE risk may motivate survivors to attend long-term follow-up care. The aims of this study were to examine knowledge of cancer history and future risks, and to identify factors associated with such knowledge, in a cohort of childhood cancer survivors. METHODS: Survivors (i.e., patients finished cancer treatment, regardless of time since completion) aged 15 to 26 years from three Canadian cancer centers were invited to complete a questionnaire that assessed knowledge of cancer history and potential LE of treatments, including five specific LE known to have considerable long-term health impact. Clinical data were extracted from hospital records and used to validate participants' answers. RESULTS: Of 250 participants, 16 (6%) were unable to name their cancer, 79 (32%) had partial or no knowledge of their therapy, and 83 (33%) were unaware of at least some of their risks for LE. Decreasing age (OR for increase in age = 1.2 (1.1-1.4)), having had a renal tumor compared to leukemia (OR = 0.3 (0.1-0.9)), and lacking knowledge about treatment (OR = 0.4 (0.2-0.9)) were associated with lack of knowledge of LE. Of the five, the most and least familiar LE was LE associated with impaired pulmonary function and risk of second malignancy, respectively. CONCLUSION: This study highlights knowledge deficits in survivors, specifically regarding their risk for LE. IMPLICATIONS FOR CANCER SURVIVORS: Findings can be utilized to target survivors at risk for knowledge deficits.

Exploring Cancer Worry in Adolescent and Young Adult Survivors of Childhood Cancers.

PURPOSE: Adolescent and young adult (AYA) survivors of cancers in childhood experience cancer worry, defined as concerns about cancer-related issues such as relapse and late effects of treatment. Cancer worry is an important determinant of successful transition to long-term follow-up care. The primary aim of this study was to identify patient-, cancer-, and treatment-related factors associated with cancer worry in AYA survivors. A secondary aim was to explore and understand inappropriate cancer worry (e.g., worry of developing a late effect when not at risk) in this population. METHODS: Two hundred and fifty AYA survivors, aged 15­26 years, completed a 6-item Cancer Worry Scale. Selection of factors potentially associated with cancer worry was guided by literature and expert opinion for inclusion in univariable and multivariable regression analyses. RESULTS: Female survivors reported significantly more cancer worry than males did (b=-9.4; 95% CI -14.4 to -4.5; p < 0.001). Survivors treated with the most intensive therapies reported more cancer worry compared with those who received the least intensive therapies (b=-18.5; 95% CI -31.2 to -5.9; p = 0.004). Thirty-one percent of participants had inappropriate worry regarding infertility and/or secondary malignancy. CONCLUSIONS: In AYA survivors, female sex and higher treatment intensity were associated with increased cancer worry. Inappropriate worry was prevalent among survivors and may contribute to unnecessary distress. These findings can help identify survivors who are more likely to worry and support the development of appropriate services to reduce the effect of cancer worry on survivor well-being.