Introduction:Little is known about the clinical characteristics of von Willebrand disease (VWD) patients in China, the impact of Covid-19 on them and their genetic mutation. Aim:To describe the clinical characteristics of a group of VWD patients in China, the impact of Covid-19 on them and their genetic mutation.Methods: An online survey using a self-designed questionnaire was conducted among patients within a WeChat group of VWD patients in China. Data were analysed using t-test, the Chi-square test, Fisher's exact test and rank sum test.Results: Data from a total of 96 patients were collected. Several important findings are yielded. Above all, type 3 patients accounted for over half of the surveyed patients.Secondly, a surprising rate (>40%) of patients had experience of being misdiagnosed.Thirdly, treatment regimens were dominated by cryoprecipitate, blood-derived FVIII and plasma, and only a small percentage of patients received prophylaxis. Fourthly, we identified 17 new von Willebrand factor (VWF) mutant genes which merit further investigation. Additionally, Covid-19 was found to pose some challenges for the patients. Conclusion:In China, the high rates of type 3 patients and misdiagnosis suggest that most of the VWD patients may never be diagnosed in China. When it comes to diagnosis and treatment, there is a large gap between developing countries like China and developed countries.
BackgroundLittle is known about the intestinal flora of primary immune thrombocytopenia (ITP) patients in relation to those of healthy people. AimTo explore the structural and functional differences of intestinal flora between ITP patients and healthy controls.MethodsFeces from ITP patients and healthy controls were studied by 16S rRNA and metagenomic techniques at phylum, genus, species or functional levels. Blood samples were collected for the detection of IL-2 and IL-4 concentrations.ResultThe following changes in ITP patients were found: a decrease of Bacteroidetes phylum, an increase of Proteobacteria phylum and alterations of ten genera and 1045 species. Genera of Phascolarctobacterium and Romboutsia were correlated with IL-2 levels. Species of C. freundii, C. rodentium, and C. youngae were negatively correlated with bleeding scores, and S. infantis was positively related to platelet counts. Functionally, the intestinal flora of ITP patients changed mainly in terms of motility, chemotaxis, membrane transport, and metabolism. ConclusionsThe mechanism underlying the functional and structural changes of the intestinal flora in ITP patients may be related to inflammation and immunity, suggesting a new target for ITP treatment.
Objective: The purpose is to clarify the overall situation of clinical related to primary immune thrombocytopenia (ITP), to evaluate the difference between published clinical trials and unpublished trials, and to evaluate the relevant information of trial publication. Methods: Search the ClinicalTrials.gov database on March 20, 2022 to identify ITP clinical trials and obtain relevant data. Publications in PubMed were searched using standardized methods to identify the publication of completed clinical trials. Results: Of 341 trials identified, interventional trials were the most common (74.2%, n=253). Interventional trials and observational trials differ in the main research content (odds ratio (OR) 0.06, 95% confidence interval (CI) 0.03-0.015, P=0.000). In terms of published articles, there are few trials involving non drugs (OR 0.23, CI 0.08-0.64, P = 0.005). There were fewer trials with less than 10 participants (OR 1.52, CI 1.06-2.20, P = 0.024). Of the 167 completed trials, 93 (55.7%) were published and 48 (28.7%) uploaded results. Conclusion: If the main research content involves drugs, trials with a larger number of people are more likely to be published. The publication rate of ITP clinical trials is high, but the submission rate of database results is low. Therefore, more attention should be paid to the submission of clinical trial results in the later stage.
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