ObjectivesTo provide a reference for the evaluation procedures of genetic testing technology applicable to China by combining the existing evaluation frameworks and procedures for genetic testing techniques globally, and also put forward design suggestions for the construction of evaluation procedures in China.MethodsThe literature research method was primarily used to summarize different evaluation progress, as well as put forward design suggestions.ResultsAt present, numerous developed countries have organized genetic testing technology evaluation projects. The various evaluation frameworks developed were based on the ACCE or HTA framework. The evaluation and decision-making procedures were similar in general, including topic selection, evaluation implementation, results reporting and making recommendations. However, there still remained difficulties such as limited evidence and uncertainty in decision-making.ConclusionsTo establish the procedures of genetic testing technology applicable in China, the following specific procedures are recommended: selecting target genetic testing technology topics; analyzing necessity and feasibility of target testing technology evaluation; evaluating and reviewing the evidence; applying results and decision-making transformation; developing regular review and revision mechanisms.
ObjectivesTo initially construct a scientific, reasonable and precision medicine technology value judgment framework suitable for China’s national conditions based on expert consultation method, so as to provide scientific value judgment system support for China's medical insurance decision-making.MethodsThe preliminary evaluation indicator system for precision medicine technology value was established by using literature analysis and expert consultation method, and the direct weighting method was used to determine the indicators weight.ResultsAfter two rounds of expert consultation, an indicator system suitable for the value judgment of precision medicine technology in China was constructed, including 5 primary indicators (health needs, health effects, economics, innovation and suitability) and 14 secondary indicators. Each indicator was weighted according to importance.ConclusionsA set of precision medicine value judgment indicator system suitable for China has been initially established, which lays a certain foundation for further measurement research of the indicator system and provides a scientific basis for medical insurance decision-making.
With the increasingly prominent contradiction between limited health resources and the growing population, priority setting of health research, as a response, has received widespread attention from health systems worldwide. As the results of priority setting at different levels increase year by year, some questions in the results reporting are also constantly emerging. For example, the process of producing the results is vague, too dependent on individual subjective judgment, the participation of individual stakeholder groups is limited or lack of voice, unable to identify potential conflicts of interest, and so on. It does not only seriously affect the effectiveness and rationality of the results themselves, but also create intangible obstacles to their promotion and adoption. In 2019, BMC Medical Research Methodology published ‘Reporting guideline for priority setting of health research (REPRISE)’, which makes uniform specifications for more comprehensive and consistent reporting of results in priority areas. This paper interpreted the background, formulation process and key contents of the REPRISE guideline, with an aim to promote the application of the reporting guideline in China.
ObjectiveTo systematically review the economics evaluation studies on the early screening or diagnosis of primary immunodeficiency diseases (PID). MethodsWeb of Science, CRD, PubMed, The Cochrane Library, CNKI and WanFang Data databases were electronically searched to collect the economics evaluation studies on the early screening or diagnosis of PID from inception to July 1st, 2022. Two reviewers independently screened literature, extracted data and assessed the risk of bias of included studies, then, a descriptive systematic review was performed. ResultsA total of 10 studies focusing on SCID were included. The results showed that under a relatively high threshold, the early screening and diagnosis of SCID were cost-effective, which can reduce severe infections in patients and treatment costs while improving patient’s survival. ConclusionCurrent evidence shows that early diagnosis of PID can reduce costs and improve benefits. Due to limited quality and quantity of the included studies, more high quality studies are needed to verify above conclusion.
Health technology assessment (HTA) is becoming more and more popular recently. For populations in China that share at least half of the global disease burden of liver cancer, it is extremely vital to give rise to an efficient secondary prevention strategy. The China central government launched liver cancer screening program in rural areas in 2005, and then extended to populations in urban in 2012. The studies of health technology assessment of liver cancer screening are based on available evidence, from an HTA perspective, aims to evaluate performance of liver screening, economic burden and cost-effectiveness and some other issues, in order to raise suggestions for possible directions in research and public health program related to liver cancer screening in China.
The main task of Campbell Collaboration is to collaborate with Cochrane Collaboration so as to produce high quality evidence for the social welfare, education, justice and criminal, international development policy and other social sciences. This article systematically introduces Campbell Collaboration, its origins, achievements and development, with the purpose of allowing more scholars understand evidence-based ideas and methods of social science, providing evidence-based methodology basis for China's social policy.
Objective To review the methods of repair and reconstruction of the large segmental bone tumor defect in distal tibia. Methods The related literature of repair and reconstruction of the large segmental bone tumor defect in disatal tibia was reviewed and analyzed in terms of the reserved ankle joint and the non-reserved ankle joint. Results For large segmental bone tumor defect in distal tibia, the conventional allograft bone transplantation, vascularized autologous fibular transplantation, vascularized fibular allograft, inactivated tumor regeneration, distraction osteogenesis, and bone transport techniques can be selected, and the membrane-induced osteogenesis, artificial tumor stem prosthesis, three-dimensional printed metal trabecular prosthesis, ankle arthrodesis, artificial tumor ankle joint placement surgery are gradually to be applied to repair the bone defect. Moreover, due to its long survival time, the function of reconstructed bone tumor defect in the distal tibia has also received increasing attention. Conclusion Although the ideal methods has not yet been developed, great progress has been achieved in repair and reconstruction of the large segmental bone tumor defect in the distal tibia. Recently, with the appearance of three-dimensional printing and various preoperative simulation techniques, personalized and precise therapy could become ture, but therapies for the large segmental bone tumor defect in the distal tibia still need to be further explored.
ObjectiveTo systematically review the efficacy and safety of different tyrosine kinase inhibitors (TKIs) in the treatment of chronic myelocytic leukemia (CML).MethodsPubMed, EMbase, The Cochrane Library, CBM, WanFang Data and CNKI databases were electronically searched to collect randomized controlled trials (RCTs) of nilotinib, dasatinib, flumatinib and imatinib for CML from inception to August, 2020. Two reviewers independently screened literature, extracted data and assessed the risk of bias of included studies; network meta-analysis was then performed using Stata 15.0 software and R 3.4.0 software.ResultsA total of 8 RCTs involving 2 775 patients were included. Compared with other TKIs, flumatinib had higher 3-month early molecular response and 1-year progression free survival, and the incidence of serious side effects was relatively low. Major molecular response and complete cytogenetic response were significantly superior to imatinib, and had the same or similar effects to other second-generation TKIs.ConclusionsCurrent evidence shows that flumartinib in the treatment of CML is obviously superior to imatinib, has the same or similar effect with other second generation TKIs. Due to limited quality and quantity of the included studies, more high quality studies are needed to verify above conclusions.
ObjectiveTo explore the optimal conditions of rat model of hyperuricemia (HUA) induced by different doses of potassium oxanate (PO) combined with adenine, and to provide reference for the treatment of HUA.MethodsMale Sprague-Dawley rats (220-240 g body weight) were divided into normal control group, potassium oxanate (1000, 1500 mg/kg) and adenine (0, 50, 100 mg/kg) combined model groups, with 8 rats in each group. After 5 weeks of intragastric administration, blood were collected from tail vein of rats every week, and serum uric acid, creatinine and blood urea nitrogen level were measured. At the 6th week, the changes of the pathological characteristics, expression of inflammatory and fibrosis-related factors in the kidneys were observed.ResultsIn the 1500 mg/kg potassium oxanate combined with 100 mg/kg adenine group, rats died after 2 weeks of molding, and the survival rate at the 6th week was 62.5%; but there was no significant difference between the other groups and the normal control group in survival rate (P>0.05). Compared with the normal group, the level of serum uric acid in each model group increased significantly after 1 week of molding (P<0.05), but recovered to the pre-model level after stopping intragastric administration in week 6. After 5 weeks, in model groups the levels of serum creatinine and blood urea nitrogen were higher than those in the normal control group; and the inflammation and fibrosis-related factors mRNA and protein expression of kidney tissue in model groups increased with the increase of ademine dose, and there was a significant difference in the PO 1 000 mg/kg with adenine 100 mg/kg group, PO 1 500 mg/kg with Adenine 50 mg/kg group compared to the normal control group (P<0.05). The results of renal anatomy and histology testing in rats showed that with the increased of the dosage of PO and adenine in the model groups, the increase of white deposition of renal medulla, tubulointerstitial fibrosis, and tubular epithelial cell necrosis was found, and the glomerular atrophy aggravated. Compared with the indexes in the normal control group, the expression levels of inflammation and fibrosis related genes and proteins in the 50 mg/kg adenine combined with 1 500 mg/kg PO group were higher, and inflammatory cell infiltration and fibrosis were observed, which was consistent with the clinical manifestation of hyperuricemia induced renal injury.ConclusionPO (1500 mg/kg) combined with adenine (50 mg/kg) can establish a stable hyperuricemic nephropathy model in rats.
ObjectiveTo systematically review the pharmacoeconomics of hemodialysis and peritoneal dialysis in patients with end-stage renal disease. MethodsCRD, NICE, CADTH, HITAP, NECA, IWIQG, ISPOR, PubMed, EMbase, CNKI, and WanFang Data databases were electronically searched to collect pharmacoeconomic studies on the hemodialysis and peritoneal dialysis in the treatment of end-stage renal disease from inception to December 2020. Two reviewers independently screened literature, extracted data, and assessed the risk of bias of included studies. Then, the conclusions of research models, pharmacoeconomic evaluation results, and sensitivity analysis were summarized. ResultsA total of 15 pharmacoeconomic studies were included, among which 9 studies used the Markov state transition model, and 6 were observational studies. From the perspective of health outcomes, peritoneal dialysis had cost-effectiveness advantages over hemodialysis in the treatment of end-stage renal disease under the condition of a clear threshold. ConclusionsCurrent evidence shows that compared with hemodialysis, peritoneal dialysis has certain cost-effectiveness advantages in the treatment of end-stage renal disease. Due to limited quality and quantity of the included studies, more high-quality studies are required to verify the above conclusions.