We evaluated objective and subjective swallowing function outcomes in patients with dysphagia treated for head and neck cancer (HNC) and identified risk factors for poor swallowing outcomes. Patients undergoing videofluoroscopic swallowing studies (VFSS) between January 2016 and March 2021 were divided into four groups according to primary tumor sites; post-treatment dysphagia was assessed. The penetration–aspiration scale (PAS) and bolus residue scale (BRS) were used to objectively assess swallowing function through VFSS. The Functional Oral Intake Scale (FOIS) was used for subjective analyses of swallowing statuses. To account for potential confounding, important covariates were adjusted for in logistic regression models. Oropharyngeal tumors were significantly more likely to have poor PAS and BRS scores than oral cavity tumors, and the patients with nasopharyngeal tumors were significantly less likely to have poor FOIS scores. Old age, having multiple HNCs, and a history of radiotherapy were associated with an increased odds of poor PAS scores (for all types of swallows), poor BRS scores (for semiliquid and solid swallows), and poor FOIS scores, respectively. This indicates using only subjective assessments may not allow for accurate evaluations of swallowing function in patients treated for HNC. Using both objective and subjective assessments may allow for comprehensive evaluations.

Purpose Taiwan launched reimbursement of prophylactic coagulation factor replacement therapy (CFRT) for patients with severe hemophilia type A (severe PWHA) in 2014. However, since then, the effectiveness of prophylactic CFRT in real-world practice has not been evaluated thoroughly. This study aimed to evaluate the effectiveness of prophylactic CFRT in severe PWHA cases on the outcome of bleeding risks. Patients and Methods We included male, severe PWHA cases from a nationwide, population-based database in Taiwan. Given that the database lacked details of the dosing regimen for prophylactic CFRT, we applied group-based trajectory modeling using the proportion of days covered (PDC) by CFRT from 2014 to 2015 in order to classify patients. A high PDC level corresponded to a greater proportion of time under CFRT, thus implying that the patient was probably receiving prophylactic therapy. We followed up patients from January 01, 2016 until occurrence of any bleeding events, death or December 31st 2017. Results We identified a total of 420 severe PWHA and classified them into high- (n = 88), medium- (n = 181) and low- (n = 151) PDC groups. The mean (±SD) PDC values of the three groups were 0.78 (±0.1), 0.40 (±0.1) and 0.12 (±0.1), respectively. Using Cox regression models with propensity score adjustment, we found patients with medium- (hazard ratio: 0.69; 95% CI: 0.56–0.89) or high-PDC (0.45; 0.36–0.68) under CFRT had reduced risks of any bleeding, compared to the low PDC group. Conclusion The findings demonstrated the effectiveness of prophylactic CFRT in the prevention of bleeding events in real-life severe PWHA.

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Purpose Development and evaluation of a drug-safety signal detection system integrating data-mining tools in longitudinal data is essential. This study aimed to construct a new triage system using longitudinal data for drug-safety signal detection, integrating data-mining tools, and evaluate adaptability of such system. Patients and Methods Based on relevant guidelines and structural frameworks in Taiwan’s pharmacovigilance system, we constructed a triage system integrating sequence symmetry analysis (SSA) and tree-based scan statistics (TreeScan) as data-mining tools for detecting safety signals. We conducted an exploratory analysis utilizing Taiwan’s National Health Insurance Database and selecting two drug classes (sodium-glucose co-transporter-2 inhibitors (SGLT2i) and non-fluorinated quinolones (NFQ)) as chronic and episodic treatment respectively, as examples to test feasibility of the system. Results Under the proposed system, either cohort-based or self-controlled mining with SSA and TreeScan was selected, based on whether the screened drug had an appropriate comparator. All detected alerts were further classified as known adverse drug reactions (ADRs), events related to other causes or potential signals from the triage algorithm, building on existing drug labels and clinical judgement. Exploratory analysis revealed greater numbers of signals for NFQ with a relatively low proportion of known ADRs; most were related to indication, patient characteristics or bias. No safety signals were found. By contrast, most SGLT2i signals were known ADRs or events related to patient characteristics. Four were potential signals warranting further investigation. Conclusion The proposed system facilitated active and systematic screening to detect and classify potential safety signals. Countries with real-world longitudinal data could adopt it to streamline drug-safety surveillance.

Objectives: Neoadjuvant (neo-adj) therapy is a treatment given prior to surgery of patients with early breast cancer (eBC). It makes breast-conserving surgery possible to be replaced with mastectomy. As shown in NeoSphere clinical trials, by receiving neo-adj treatment, it is possible to achieve pathological complete response (pCR). Pertuzumab as an add on to trastuzumab is a targeted therapy which is also used for neo-adj treatment of eBC patients with HER2+ mutation. The objective of this study is to analyze the cost effectiveness of pertuzumab+trastuzumab against traztuzumab for neo-adj treatment of eBC in Turkish healthcare environment. Methods: A Markov model consisting of six health states (event-free, locoregional recurrence, remission, metastatic-not-progressed, metastatic-progressed and death) is used to perform health economic analysis. Clinical data from NeoSphere trial is used as clinical inputs and long-term outcomes of neo-adj treatment are estimated based on meta-analysis. Time horizon is selected as 50 years and all costs are calculated from Turkish payer perspective. Results: For the base scenario, incremental cost and QALY is found to be 3.894 TRY and 0,28, respectively. ICER was calculated as 13.759 TRY which is highly cost-effective in an environment where the CE threshold is w170.000 TRY. The result is supported with different scenarios and probabilistic sensitivity analysis. Conclusions: This study concludes that pertzumab+trastuzumab combination provides better clinical benefits by being highly cost-effective against trastuzumab treatment in the neo-adj setting of eBc patients. It is expected that pertuzumab treatment in neo-adj setting would result in better long term outcomes both for the patients and the payer in Turkish healthcare environment.