The impact on quality of life of diet restrictions and disease symptoms associated with phenylketonuria: a time trade-off and discrete choice experiment study.

Phenylketonuria (PKU) is a metabolic disorder leading to a deficiency in the metabolism of phenylalanine (Phe). Elevated Phe levels in the blood, tissue and brain may lead to emotional, cognitive, and physical symptoms in adults. To control blood Phe levels, most individuals with PKU need to follow a strict, life-long Phe-restricted diet. The main objective of this study was to estimate the impact of PKU-related disease symptoms and dietary restrictions on health-related quality of life (HRQoL). This study was designed as a web-based survey using the methods of Time Trade-Off (TTO) and Discrete Choice Experiment with duration (DCETTO) in a survey of the Swedish general population and population with PKU. Data were collected using questionnaires for TTO diet (n = 509), TTO symptoms (n = 507), and DCETTO (n = 1117). The disutility of diet restrictions ranged from 0.050 for a partially restricted diet without medical food to 0.193 for a fully restricted diet with medical food in the TTO (DCE: 0.043-0.108). The disutility of experiencing symptoms (emotional, cognitive and physical) ranged from 0.148 for mild symptoms to 0.593 for severe symptoms in the TTO (DCE: 0.122-1.522). The study shows that both diet and symptoms have a significant negative impact on HRQoL in PKU and that utility estimates are largely consistent across methods and samples.

Cost comparisons of endoscopic and surgical resection of stage T1 rectal cancer.

Background and study aims Management of T1 rectal cancer is complex and includes several resection methods, making cost comparisons challenging. The aim of this study was to compare costs of endoscopic and surgical resection and to investigate hypothetical cost scenarios for the treatment of T1 rectal cancer. Patients and methods Retrospective population-based cost minimization study on prospectively collected data on T1 rectal cancer patients treated using endoscopic submucosal dissection (ESD), transanal endoscopic microsurgery (TEM), open, laparoscopic, or robotic resection, in Skåne County, Sweden (2011-2017). The hypothetical cost scenarios were based on the distribution of high-risk features of lymph node metastases in a national cohort (2009-2017). Results Eighty-five patients with T1 RC undergoing ESD (n = 16), TEM (n = 17), open (n = 35), laparoscopic (n = 9), and robotic (n = 8) resection were included. ESD had a total 1-year cost of 5165 €â€Šand was significantly ( P  < 0.05) less expensive compared to TEM (14871€), open (21 453 €), laparoscopic (22 488 €) and robotic resection (26 562 €). Risk factors for lymph node metastases were seen in 68 % of 779 cases of T1 rectal cancers included in the national cohort. The hypothetical scenario of performing ESD on all T1 RC had the lowest total 1-year per patient cost compared to all other alternatives. Conclusions This is the first study analyzing total 1-year costs of endoscopic and surgical methods to resect T1 rectal cancer, which showed that the cost of ESD was significantly lower compared to TEM and surgical resection. In fact, based on hypothetical cost scenarios, ESD is still justifiable from a cost perspective even when all high-risk cases are followed by surgery in accordance to guidelines.

Clinical practice of BOTOX® treatment for overactive bladder syndrome in Sweden: an assessment of resource use and external validity.

OBJECTIVE: The objective of this study was to assess the resource use of treating overactive bladder (OAB) patients in real-world clinical practice and to evaluate whether there is external validity in the treatment of OAB in clinical practice. MATERIALS AND METHODS: The study included 55 patients suffering from OAB and treated with BOTOX® at two Swedish clinics. The study was conducted as an anonymized retrospective chart review study. RESULTS: The estimated yearly direct cost of BOTOX treatment was €902. The mean age of patients in the study was 60 years, and 85% were women. The severity of OAB before BOTOX treatment, given by the mean number of daily leakages, equalled 4.8. The median interval between treatments was 210 days. CONCLUSIONS: Patient characteristics in the real world were similar to those in the clinical trials, showing a high degree of external validity. Treatment intervals were longer in the real world than in clinical trials, indicating that treatment cost could be lower when patients are treated as observed in real-world clinical practice.

Fast-track access to urologic care for patients with macroscopic haematuria is efficient and cost-effective: results from a prospective intervention study.

BACKGROUND: The delay between onset of macroscopic haematuria and diagnosis of bladder cancer is often long. METHODS: We evaluated timely diagnosis and health-care costs for patients with macroscopic haematuria given fast-track access to diagnostics. During a 15-month period, a telephone hotline for fast-track diagnostics was provided in nine Swedish municipalities for patients aged ⩾50 years with macroscopic haematuria. The control group comprised 101 patients diagnosed with bladder cancer in the same catchment area with macroscopic haematuria who underwent regular diagnostic process. RESULTS: In all 275 patients who called 'the Red Phone' hotline were investigated, and 47 of them (17%) were diagnosed with cancer and 36 of those had bladder cancer. Median time from patient-reported haematuria to diagnosis was 29 (interquartile range (IQR) 14-104) days and 50 (IQR 27-165) days in the intervention and the control group, respectively (P=0.03). The median health-care costs were lower in the intervention group (655 (IQR 655-655) EUR) than in the control group (767 (IQR 490-1096) EUR) (P=0.002). CONCLUSIONS: Direct access to urologic expertise and fast-track diagnostics is motivated for patients with macroscopic haematuria to reduce diagnostic intervals and lower health-care expenditures.