Cost-utility analysis of romiplostim for the treatment of chronic primary immune thrombocytopenia in China.

This study aimed to assess the cost-utility of romiplostim (ROMI) compared to eltrombopag (EPAG) as a second-line treatment for chronic primary immune thrombocytopenia (cITP) in Chinese adults. A decision tree-embedded Markov model with a lifetime horizon was used to estimate the quality-adjusted life years (QALYs) and costs for ROMI versus EPAG from the perspective of the Chinese health care system. The model was driven by platelet response with a 4-week cycle.

A comprehensive value-based method for new nuclear medical service pricing: with case study of radium [223] bone metastases treatment.

Importance: Innovative nuclear medicine services offer substantial clinical value to patients. However, these advancements often come with high costs. Traditional payment strategies do not incentivize medical institutes to provide new services nor determine the fair price for payers.

Development and initial validation of the career resilience instrument for CDC emergency responders in China within the context of public health emergencies: based on a survey conducted in Shanghai.

Background: China faces various public health emergencies, and emergency responders at the Centers for Disease Control and Prevention (CDC emergency responders) are a mainstay in responding to public health emergencies. Career resilience can help CDC emergency responders to effectively respond to and recover from public health emergencies, but there is no specific measurement instrument available. In this study, we aimed to develop and conduct an initial validation of the career resilience instrument for CDC emergency responders in China within the context of public health emergencies from a process perspective.

Mutant in a Multiplex Family: A Dominant Variant Co-Segregated With Moderate Malformations.

is a well-documented disease-related gene. Several -recessive variants have been reported to cause Ehlers-Danlos syndrome (EDS). To the best of our knowledge, no dominant variant that causes human disease has been reported.

MECOM-related disorder: Radioulnar synostosis without hematological aberration due to unique variants.

Purpose: The etiology for a considerable proportion of patients with congenital radioulnar synostosis (RUS) remains unclear. This study aimed to investigate the genetic cause of RUS without a known cause.

Methods: Patients with RUS were investigated.

Case series of congenital pseudarthrosis of the tibia unfulfilling neurofibromatosis type 1 diagnosis: 21% with somatic NF1 haploinsufficiency in the periosteum.

Up to 84% of patients with congenital pseudarthrosis of the tibia (CPT) present with neurofibromatosis type 1 (NF1) (NF1-CPT). However, the etiology of CPT not fulfilling the NIH diagnostic criteria for NF1 (non-NF1-CPT) is not well understood. Here, we collected the periosteum tissue from the pseudarthrosis (PA) site of 43 non-NF1-CPT patients and six patients with NF1-CPT, together with the blood or oral specimen of trios (probands and unaffected parents).

A genotype and phenotype analysis of SMAD6 mutant patients with radioulnar synostosis.

Background: SMAD6 variants have been reported in patients with radioulnar synostosis (RUS). This study aimed to investigate the genotypes and phenotypes for a large cohort of patients with RUS having mutant SMAD6.

Methods: Genomic DNA samples were isolated from 251 RUS sporadic patients (with their parents) and 27 RUS pedigrees.

Network Meta-analysis of the Therapeutic Effects of Hypoglycemic Drugs and Intensive Lifestyle Modification on Impaired Glucose Tolerance.

Purpose: In the Guidelines for the Prevention and Control of Type 2 Diabetes in China (2017 edition), intensive lifestyle interventions are recommended for preventing the progression of impaired glucose tolerance (IGT) to type 2 diabetes mellitus. Acarbose and metformin can also be considered if intensive lifestyle modification has been ineffective for 6 months. But the effects of intensive lifestyle modification and glucose-lowering drug interventions that work best in the population with IGT are unclear.

Cost-effectiveness analysis of hemodialysis plus hemoperfusion versus hemodialysis alone in adult patients with end-stage renal disease in China.

Background: This study evaluates the cost-effectiveness of hemodialysis (HD) plus hemoperfusion (HP) with HD alone in adult patients with end-stage renal disease (ESRD) in China.

Methods: A Markov model was constructed to assess the cost-effectiveness of interventions over a lifetime horizon. Model parameters were informed by the HD/HP trial, the first randomized, open-label multicenter trial comparing survival outcomes and incidence of cardiovascular disease (CVD) for HD + HP versus HD alone, and supplemented by published literature and expert opinion.

Survival Outcomes of Hemoperfusion and Hemodialysis versus Hemodialysis in Patients with End-Stage Renal Disease: A Systematic Review and Meta-Analysis.

Background: The maintenance treatment of ESRD puts a great burden on individual patients, society, and the country of China as a whole. Hemoperfusion plus hemodialysis (HP + HD) in ESRD patients can yield modified outcomes such as prolonged life expectancy and improved dialysis quality and quality of life. This study aimed to systematically analyze the effect of HP + HD on the overall survival (OS) rates of ESRD patients and to provide support for clinical decision-making.

Disorder of Sexual Development Males With XYY in Blood Have Exactly X/XY/XYY Mosaicism in Gonad Tissues.

Y chromosome represents masculinization. The extra Y chromosome of XYY patients usually leads to over-masculinization phenotypes. The occurrence of several DSD cases with XYY in blood is controversial.

Economic evaluation of olmesartan/amlodipine fixed-dose combination for hypertension treatment in China.

To evaluate the cost-effectiveness of olmesartan/amlodipine fixed-dose combination vs olmesartan and amlodipine free combination, amlodipine single drug, and valsartan/amlodipine fixed-dose combination in the treatment of hypertensive patients from payer perspective in China. A Markov model was constructed, which included five health states of hypertensive patients who are aged 35-84 years at baseline and free of cardiovascular disease. Clinical data were obtained from a network meta-analysis.

Management of Benign Tracheal Stenosis by Small-diameter Tube-assisted Bronchoscopic Balloon Dilatation.

Background: A limitation of bronchoscopic balloon dilatation (BBD) is that airflow must be completely blocked for as long as possible during the operation. However, the patient often cannot hold his or her breath for a long period affecting the efficacy of the procedure. In this study, we used an extra-small-diameter tube to provide assisted ventilation to patients undergoing BBD and assessed the efficacy and safety of this technique.