Myelofibrosis (MF) is associated with several constitutional symptoms. Currently, there are few therapeutic options for MF. Jaktinib, a novel, small-molecule inhibitor of JAK, is currently being studied for its potential to treat MF. This phase 2 trial investigated efficacy and safety of jaktinib in the treatment of MF patients. The primary end point was the proportion of patients with ≥35% reduction in spleen volume (SVR35, proportion of patients with ≥35% reduction in spleen volume) at week 24. The secondary end points included improvement of anemia, rates of symptom response, and safety profile. Between January 8, 2019 and August 29, 2020, 118 patients were recruited and treated with either jaktinib 100 mg BID or 200 mg QD. At week 24, 54.8% (34/62) of patients in the 100 mg BID group and 31.3% (15/48) in the 200 mg QD group achieved SVR35 (p = .0199). Jaktinib treatment increased hemoglobin level to ≥20 g/L in 35.6% (21/59) of patients with hemoglobin ≤100 g/L at baseline. The proportion of patients who achieved a ≥50% improvement in total symptom score at week 24 was 69.6% (39/56) in the BID group and 57.5% (23/40) in the QD group. The most common ≥ grade 3 hematological treatment-emergent adverse events (TEAEs; ≥ 10%) were anemia (100 mg BID: 24.2%, 200 mg QD: 28.8%), thrombocytopenia (16.7%, 11.5%), and neutropenia (3.0%, 11.5%). All non-hematological TEAEs were mild. These results indicate that jaktinib can shrink the spleen, improve anemia, and other clinical symptoms with good tolerability.
To prolong erythromycin (EM) release and prevent the side effects of EM, a Pluronic F-127 diacrylate macromer (PF127) was synthesized and then self-assembled into micelles with their hydrophobic cores loaded with EM. The EM-loaded micelles were mixed with a photoinitiator to form the EM/PF127 hydrogels rapidly under a low-intensity UV light. Afterward, the hydrogel properties, antibacterial performance, and cytotoxicity of this novel hybrid hydrogel were investigated. The results show that the EM/PF127 hydrogel had a rapid gelation time. The sustained release of EM reduced its side effects. With controlled antibacterial activity, the use of EM would be safer and more efficient. What is more, the EM/PF127 hydrogel showed a slight cytotoxicity, and this suggests great potential application as antibacterial hydrogels in the prevention of postoperative infection. V C 2014 Wiley Periodicals, Inc. J. Appl. Polym. Sci. 2014, 131, 40438.
Introduction:The development of inhibitors against factor FIX (FIX) is the most serious complication of FIX replacement therapy in haemophilia B (HB) patients. Currently, only few cohorts of HB inhibitor patients have been reported worldwide.Aim: This Chinese nationwide study of HB inhibitor patients explored their risk factors for FIX inhibitor development and experience on their management.
Methods:We retrospectively analysed patient characteristics, F9 genotypes, treatment strategies and outcomes of HB inhibitor patients registered to the Chinese National Registry and Patient Organization Registry.Results: Forty-four unique HB inhibitor patients were identified in 4485 unique HB patients registered by year 2021 to the two Registries. Inhibitor diagnosis were usually delayed and the low prevalence (.98%) may suggest some inhibitor patients were not identified. Their median age at inhibitor diagnosis was 7.5 (IQR, 3.0-14.8) years. Most
Introduction
Women and girls with haemophilia (WGH) may have spontaneous/traumatic bleeding similar to that in males with haemophilia, and in addition excessive bleeding during menstruation and delivery.
Aim
To characterize WGH in China and provide guidance for better management.
Methods
We retrospectively analysed the characteristics of WGH registered in the Haemophilia Treatment Center Collaborative Network of China (HTCCNC) Registry, including demographics, diagnosis and treatment, bleeding characteristics, obstetrical and gynaecological experiences, and surgical history.
Results
A total of 61 females had confirmed haemophilia. Diagnosis and treatment were typically delayed, longer in mild haemophilia than in severe and moderate. The most frequently reported bleeding manifestations were haemarthrosis in severe and moderate patients, and cutaneous bleeding in mild patients. Among 45 postmenarcheal WGH, 21 (46.7%) had history of heavy menstrual bleeding, but only three received treatments. Prenatal diagnosis and management of perinatal haemorrhage were inadequate. Of 34 deliveries in 30 women, nine deliveries were complicated by postpartum haemorrhage, and 22 offspring carried mutations causing haemophilia. Forty‐four surgical procedures were performed in 29 patients. Those procedures receiving preoperative coagulation factors coverage were significantly less likely to have excessive bleeding than those who did not (P = .003).
Conclusion
This is the first and largest study describing WGH in China. There are currently deficiencies in the identification, diagnosis, and management of these patients. Improving health insurance policies, establishing haemophilia centres, and multidisciplinary teams for bleeding and perinatal or perioperative management will help reduce morbidity and mortality.
BackgroundTelomeres undergo shortening with each cell division, which could be accelerated by increase obesity and is also related to endocrinology systems. In this study, we aimed to examine the complex association between telomere, C-peptide, and obesity as well as chronic inflammation in a large population-based cross-sectional survey.MethodsWe used data from a community-based population study, where around 1,382 participants were recruited and had telomere length measured. The association of telomere length with C-peptide was studied using multiple linear regression models. We also examined if obesity, measured by body mass index (BMI), and inflammation could affect this observed association.ResultsAround 48% of these participants were men and 52% were women. The average ages were 51.7 years old for men and 49.1 years old for women. After controlling for age and sex, 1 U increase of telomere length was associated with −0.17 (−0.28, −0.06) unit decrease of C-peptide. Additionally controlling for BMI, the association magnitude was decreased to −0.13 (−0.23, −0.04). Further adjusting for inflammation biomarker did not change the effect estimates.ConclusionLonger telomere was associated with lower levels of C-peptide. This association could be attenuated by adjusting for obesity.
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