The cornerstone of life-saving therapy in immune mediated thrombotic thrombocytopenic purpura (iTTP) has been plasma exchange (PEX) combined with immunomodulatory strategies. Caplacizumab, a novel anti-von Willebrand factor nanobody, trialled in two multicentre, randomised-placebo-controlled trials leading to EU and FDA approval, has been available in the UK through a patient-access scheme. Data was collected retrospectively from 2018-2020 for 85 patients receiving caplacizumab, including 4 children, from 22 UK hospitals. Patient characteristics and outcomes in the real-world clinical setting were compared with caplacizumab trial endpoints and historical outcomes in the pre-caplacizumab era. 84/85 patients received steroid and rituximab alongside PEX; 26% required intubation. Median time to platelet count normalisation (3 days), duration of PEX (7 days) and hospital stay (12 days) was comparable with RCT data. Median duration of PEX and time from PEX initiation to platelet count normalisation was favourable compared with historical outcomes (p<0.05). TTP recurrence occurred in 5/85 patients; all with persistent ADAMTS13 activity <5iu/dL. Of 31 adverse events in 26 patients, 17/31 (55%) were bleeding episodes and 5/31 (16%) were thrombotic events (two unrelated to caplacizumab); mortality was 6% (5/85), with no deaths attributed to caplacizumab. In 4/5 deaths caplacizumab was introduced >48 hours after PEX initiation (3-21 days). This real-world evidence represents the first and largest series of TTP patients receiving caplacizumab outside clinical trials, including paediatric patients. Representative of true clinical practice, the findings provide valuable information for clinicians treating TTP globally.

BackgroundClinical data of 288 chronic rhinosinusitis patients were retrospectively analyzed to investigate the risk factors of clinical prognosis, aiming to provide clinical evidence for the diagnosis and treatment of chronic rhinosinusitis.Material/MethodsA total of 288 patients diagnosed with chronic rhinosinusitis in the Department of Otolaryngology of the First Affiliated Hospital of Xinjiang Medical University were recruited. Among all participants, 177 were male and 111 were female, aged from 22 to 83 years, (52±14) years on average. Subsequent follow-up was conducted to evaluate surgical efficacy. Influencing factors of clinical prognosis were analyzed by univariate and multivariate logistic regression analyses.ResultsAfter functional endoscopic sinus surgery by Messerklinger technique, 187 (64.9%) patients were fully recovered, 72 (25.0%) presented with improvement, and 28 (10.1%) were untreated. Univariate logistic regression analysis revealed that 11 variables were correlated with the clinical prognosis of chronic rhinosinusitis. Multivariate logistic regression analysis demonstrated that age, history of allergic rhinitis, severity of dysosmia, history of nasosinusitis surgery, and long-term use of nasal decongestant were the risk factors, whereas comprehensive therapy after surgery was a protective factor.ConclusionsMore emphasis should be placed upon the factors associated with the clinical prognosis of patients with chronic rhinosinusitis following undergoing endoscopic sinus surgery, offering consolidated evidence for the prevention and treatment of chronic rhinosinusitis.

Renal artery pseudoaneurysms after renal transplantation are extremely uncommon and are able to cause severe complications such as aneurysm rupture or renal allograft loss. Treatment often leads to transplant nephrectomy. We successfully treated a transplant renal artery pseudoaneurysm with covered stents, which resulted in well-preserved renal function.

Abstract.A majority of studies have indicated that microRNA-125b (miR-125b) is aberrantly expressed in various types of cancer. However, there are no studies on the expression and function of miR-125b in human laryngeal squamous cell carcinoma (LSCC). In the present study, miR-125b expression in LSCC sample tissues, corresponding adjacent non-neoplastic tissues, LSCC cell lines and a normal human keratinocyte cell line was measured using the reverse transcription-quantitative polymerase chain reaction. Following transfection with miR-125b mimics, the Cell Counting Kit-8, cell migration, cell invasion, western blotting and dual-luciferase reporter assays were performed on LSCC cell lines. According to the results, miR-125b was observed to be significantly downregulated in LSCC, and its expression was significantly associated with clinical stage and alcohol history. miR-125b was also observed to decrease cell growth, migra tion and invasion in LSCC cells by directly targeting signal transducer and activator of transcription 3. The results of the present study suggested that miR-125b may be a potential treatment target of LSCC in the future.

Chronic rhinosinusitis (CRS) is one of the most common types of respiratory disease and affects a large proportion of the population worldwide. The clinical differences between CRS with nasal polyps (CRSwNP) and CRS without nasal polyps (CRSsNP) facilitate studies on the development of polyps. In the present study, next-generation sequencing was performed to identify differentially expressed microRNAs (miRNAs/miRs) in CRSwNP vs. CRSsNP tissues and subsequently validated the expression of selected genes using reverse transcription-quantitative polymerase chain reaction analysis. In total, 6 miRNAs were identified to be downregulated in the CRSwNP samples compared with those in the CRSsNP samples. The predicted targets of these miRNAs were determined to be enriched in a number of signaling pathways, including the ErbB, Ras, cyclic adenosine monophosphate and Janus kinase (Jak)/signal transducer and activator of transcription (STAT) pathways. The expression of miR-4492 and that if its targets predicted by a bioinformatics analysis, tumor necrosis factor α (TNF-α) and interleukin (IL)-10, was validated in 96 clinical specimens. miR-4492 was downregulated and IL-10 was upregulated in CRSwNP vs. CRSsNP tissues, and an inverse correlation between miR-4492 and IL-10 was determined in CRS tissues; however no difference was identified in the expression of TNF-α between the different groups. The present results indicate that the miR-4492/IL-10 interaction in the Jak/STAT signaling pathway may be one of the key mechanisms in CRSwNP.