Disease-free survival as a surrogate for overall survival in HR+/HER2- early breast cancer: A correlation analysis.

BACKGROUND: Overall survival (OS) is a universally accepted measure of clinical benefit; however, prolonged follow-up is needed to observe sufficient events. Disease-free survival (DFS) has been widely adopted as a primary endpoint for early breast cancer (EBC) trials, as follow-up is comparatively shorter. Here, we present an analysis evaluating DFS as a surrogate for OS for adjuvant treatment of hormone receptor-positive/human epidermal growth factor receptor 2-negative (HR+/HER2-) EBC. METHODS: A systematic literature review which included randomized controlled trials (RCTs) with ≥80% of adult patients with HR+/HER2- EBC was conducted. The RCTs evaluated various systemic therapeutic categories; key inclusion criteria included reporting of DFS and OS hazard ratios (HRs) and mature OS data. Spearman rank correlation and weighted linear regression analyses evaluated DFS and OS HR correlation. A scenario analysis tested base-case analysis robustness, and a parallel analysis using patient-level data was conducted. RESULTS: The base case (N = 14 RCTs) showed an unweighted Spearman coefficient of 0.81 between OS and DFS (weighted: 0.81), with 84% of the variability in OS explained by DFS differences (R2 from weighted regression). The surrogate threshold effect (Burzykowski T, Buyse M. Pharm Stat. 2006;5:173-186) was 0.82 for DFS/OS HR. Scenario analysis (n = 9 RCTs), which excluded chemotherapy trials, and patient-level analysis using FACE trial data were consistent with the base-case analysis. CONCLUSIONS: These analyses support DFS as a reliable surrogate endpoint for OS in adjuvant HR+/HER2- EBC trials. Using DFS as a surrogate measure will permit timelier access to novel treatments for patients with HR+/HER2- EBC.

Re-do cytoreductive surgery with hyperthermic intra-peritoneal chemotherapy (HIPEC): Risk factors and complications.

ABSTRACT: An effective multi-modal treatment option for patients with peritoneal surface metastatic malignancies has progressed and developed over the decades as cytoreductive surgery (CRS), and hyperthermic intra-peritoneal chemotherapy (HIPEC) delivers highly concentrated, heated chemotherapy drugs directly to the abdomen during surgery. Peritoneal metastasis and high staging abdominal malignancies were considered incurable and end up with the palliation only; the CRS+HIPEC combination approach increases the median survival rate and gives a better quality of life to these patients. It is a complicated surgery which poses a high rate of complications and challenges which are difficult to manage and requires a multi-disciplinary approach. The aim of this study is to elaborate the perioperative possible physiological changes, risk factors, and related complications after re-do HIPEC.

Health-related quality of life and economic burden of chronic lymphocytic leukemia in the era of novel targeted agents.

OBJECTIVE: To quantify the health-related quality of life (HRQoL) and economic burden of chronic lymphocytic leukemia (CLL). METHODS: Studies were searched through Embase, MEDLINE, PubMed, and Cochrane Library, as well as conference abstracts (1 January 2000-2 June 2019). RESULTS: Overall, 12 and 17 primary studies were included in the HRQoL and economic burden reviews, respectively. Patients with CLL reported impairment in various quality of life domains when compared with healthy controls, including fatigue, anxiety, physical functioning, social functioning, depression, sleep disturbance, and pain interference. Key factors associated with a negative impact on the HRQoL burden of CLL included female gender, increased disease severity, and the initiation of multiple lines of therapy. Economic burden was assessed for patients with CLL based on disease status and the treatment regimen received. The main cost drivers related to CLL were outpatient and hospitalization-related costs, primarily incurred as a result of chemo/chemoimmunotherapy, adverse events (AEs), and disease progression. Treatment with targeted agents, i.e. ibrutinib and venetoclax, was associated with lower medical costs than chemoimmunotherapy, although ibrutinib was associated with some increased AE costs related to cardiac toxicities. Cost studies of targeted agents were limited by short follow-up times that did not capture the full scope of treatment costs. CONCLUSIONS: CLL imposes a significant HRQoL and economic burden. Our systematic review shows that an unmet need persists in CLL for treatments that delay progression while minimizing AEs. Studies suggest targeted therapies may reduce the economic burden of CLL, but longer follow-up data are needed.

Case of nasopharyngeal teratoma: challenges in the management.

Teratomas are tumour with tissue or organ components resembling normal derivatives of more than one germ layer. The most common site of congenital teratoma is sacrococcygeal region. Teratomas in head and neck region are rarer. We report a 4-day-old male baby who presented with nasopharyngeal mass, which led to respiratory distress and feeding difficulty. It was managed with surgical excision with multidisciplinary approach.

Persistent respiratory distress in a neonate: a diagnostic dilemma.

We present a 17-day-old term, female baby who was referred to our centre for persistent respiratory distress. She was managed for pneumonia and pneumothorax at the primary care centre. On detailed clinical examination at admission, a possibility of congenital lobar emphysema (CLE) was considered. A CT chest was performed, and diagnosis of CLE was confirmed. The infant was managed with lobectomy. The respiratory distress settled within a few hours after the surgery, and the baby was discharged in stable condition.