anthonyyazdaniml/gliner-biomed-curated-corpus huggingface dataset:
The t(1;19) (q23;p13) TCF3::PBX1 is a well-described, recurring chromosomal abnormality in B-acute lymphoblastic leukaemia (B-ALL) that has historically been associated with a worse prognosis in paediatric patients. Gene expression profiling has demonstrated that TCF3::PBX1 results in a distinct subtype of B-ALL, leading to its recognition in the most recent WHO and ICC classifications. Though initially believed to be a poor prognostic sign in the adult population, emerging evidence suggests its presence may instead be intermediate or even favourable in B-ALL. However, adults with TCF3::PBX1 are typically younger and often qualify for treatment with paediatric-inspired regimens. Thus, the prognostic significance in this population remains unclear. This translocation appears to be very rare in older adults with B-ALL and its predictive and prognostic nature in this population is unknown. Herein, we explore a case of this translocation occurring in a patient in her 70s. She initially presented to the emergency department with abdominal pain and thrombocytopenia and was subsequently diagnosed with B-ALL. In addition to t(1;19) (q23;p13), a pathologic mutation in the CBL gene was identified. CBL mutations have been implicated in cancer progression and are mostly described in paediatric B-ALL. She was treated with modified Ph-negative EWALL induction (Vincristine, Idarubicin, dexamethasone) and achieved a complete remission. However, she subsequently experienced an early relapse and was refractory to targeted therapy with blinatumomab. After treatment with inotuzumab ozogamicin, she achieved a second complete remission. Unfortunately, she then suffered a central nervous system (CNS) relapse and passed away from complications of her disease. This case serves as an example of the heterogeneous nature of B-ALL. It demonstrates that patients with ostensibly favourable prognostic factors may experience poor response rates to traditional chemotherapy as well as targeted salvage agents. It also illustrates the challenges of treating B-ALL in the elderly population.
BACKGROUND: Chronic cough (CC) is common in the general population of China, creating a difficult-to-ignore public health burden. However, there is a lack of research on the nationwide prevalence and disease burden of CC in the Chinese population. We aim to use an insurance claims database to assess the prevalence and the corresponding economic burden owing to CC in China.
METHODS: This was a retrospective observational study based on an administrative medical insurance database in 2015, 2016 and 2017, from nine cities in North, South, East, South-West, and North-West regions of China. The study population was Chinese adults (≥ 18 years old) who had been identified as CC patients. Descriptive data analyses were used in statistical analysis.
RESULTS: A total of 44,472, 55,565, and 56,439 patients with mean ages of 53.2 (16.3) years were identified as patients with CC in 2015, 2016, and 2017, respectively. Of these, 55.24% were women. In addition, 8.90%, 9.46%, and 8.37% of all patients in 2015, 2016, and 2017, who had applied for medical insurance, had CC, respectively, with a three-year average probability of 8.88%. The median number of outpatient visits within a calendar year was 27 per year due to any reason during the period of 2015-2017. The median medical cost of each patient per year increased from 935.30 USD to 1191.47 USD from 2015 to 2017.
CONCLUSION: CC is common among medical insurance users, with a substantial utilization of medical resources, highlighting the huge burden of CC in China.
In recent years, nonoperative treatment has challenged appendectomy as a first-line treatment of acute uncomplicated appendicitis by demonstrating its safety and short-term efficacy in adults. Several randomized controlled trials comparing appendectomy to antibiotics alone in children with acute uncomplicated appendicitis have been reported from other countries. These studies reveal that non-operative management of appendicitis is a safe treatment for appendicitis and is associated with a significantly lower risk of complications than appendectomy. However, the success rate of nonoperative management was 75% at 1year. Chronic appendicitis has been described as a possible cause of persistent right lower quadrant (RLQ) pain and laparoscopic appendectomy has been shown to benefit a large number of children，but there are no high-quality clinical trials. There are more than 200 million children in China. According to preliminary clinical data, the incidence of pediatric appendicitis in China is higher than Europe and America. Due to the Chinese parents have different cultures and educational backgrounds, more high-quality clinical trials are needed to guide parents to choose appropriate treatment for chronic appendicitis in children. Therefore, the investigators propose a multi-institutional trial in which patients and their families choose between antibiotics therapy (AT group) or laparoscopic appendectomy (LAAPT group) to chronic appendicitis in children aged 3-15 years. The investigators hypothesize that laparoscopic appendectomy will be successful in 90% of patients at 1 year follow-up and will be associated with fewer disability days, higher quality of life scores, and higher pain reduce scores than antibiotic therapy. This study will enroll 200 patients, age 3-15 years, with chronic appendicitis at 8-10 hospitals.
Androgen deprivation therapy (ADT) is routinely used as first-line treatment for locally advanced and metastatic prostate cancer. However, it is associated with many side effects such as increased fat mass and reduced skeletal muscle mass, cardiorespiratory fitness and metabolic health. Ultimately, these negative changes induced by ADT contribute to reduced health-related quality of life (HRQoL). This study evaluates whether 3-months of exercise can mitigate treatment toxicity in prostate cancer patients initiating ADT.

In a parallel groups, prospective, randomised controlled design, participants were randomly allocated to a standard care control group or standard care plus exercise group. The exercise group completed 3-months of combined aerobic and resistance training. The standard care group did not receive any supervised exercise or specific physical activity recommendations. Outcomes were assessed at baseline, 3-month (post-intervention), and 6-month (follow-up) endpoints.
ALX-0061 225 mg every 2 weeks from baseline through Week 12. The last injection with study drug was administered at the Week 10 visit.
CF is a gentic disease resulting from mutations in a 230 kb gene on chromosome 7 known as the cystic fibrosis transmembrane conductance regulator (CFTR). Study subjects with CF manifest pathological changes in a variety or organs that express CFTR. The lungs are frequently affected, the sequelae being chronic infections and airway inflammation. The principal goal of both treatment of subjects with CF is to slow the chronic deterioration of lung function.

Study subjects will be randomized to receive either study drug or placebo (1.5% NaCl) by inhalation via a PARI eFlow nebulizer. Each subject will complete 28 days of daily dosing. All study patients will be followed for safety, pharmacokinetics, clinical and microbiologic activity for 56 days post completion of study treatment. For the two lower doses (70 mg and 140 mg): patients received drug for 28 days, followed by a 28 day safety evaluation. For 560 mg: patients received drug for 28 days, followed by a 56 day safety evaluation. The total study period will be up to 84 days, with screening visit occurring within the preceding 14 days prior to study day 1. Patients will be clinically evaluated during the first 48 hours post first study dose and weekly for the 28 day treatment period and during the follow up visits at study days 35, 42, 49, 56, 70 and 85 days to determine safety and tolerability, pharmacokinetics (PK) and clinical and microbiologic activity.

Clinical laboratory parameters, audiology testing, clinical adverse events and pulmonary function will be evaluated for all study subjects in order to determine the qualitative and quantitative safety and tolerability of Arikayce™ compared to placebo. Serum, urine and sputum specimens will be collected at periodic intervals to assess PK. Additionally, sputum samples will be collected to determine changes in bacterial density. Pulmonary function testing and CFQ-R measurements will be assessed at selected time points throughout the study. An exploratory evaluation of a Cystic Fibrosis Symptom Diary (CFSD) will also be implemented. Arikace™,Arikayce™, Liposomal Amikacin for Inhalation (LAI), and Amikacin Liposome Inhalation Suspension (ALIS) may be used interchangeably throughout this study and other studies evaluating amikacin liposomal inhalation suspension.
Leprosy, caused by the bacterium Mycobacterium leprae, is known to primarily affect the skin and peripheral nerves. We present a rare case of leprosy initially manifesting as demyelinating polyneuropathy. A 46-year-old female presented with progressive weakness, tingling, and numbness in her extremities. Nerve conduction studies revealed evidence of demyelination, prompting further investigations. Skin slit-skin smears confirmed the diagnosis of leprosy, with the presence of acid-fast bacilli. The patient was subsequently started on multidrug therapy, leading to significant clinical improvement. This case highlights the importance of considering leprosy as a differential diagnosis in patients presenting with demyelinating polyneuropathy, especially in endemic regions.
A capsule for therapeutically active substances has a wall which is of a material soluble in the stomach or intestine, e.g. gelatine. The capsule is closed by a cover and forms a holder for the therapeutical material; the interior being divided by >=1 partition into >=2 or more compartments. The separate compartments may contain similarly active therapeutical substances or alternatively, the medicaments contained may differ in their effect. The solubility of the materials of the outer capsule and of the covers is different. Alternatively the speed at which the material dissolve may be the same. The contents of the separate compartments may be the same in quantity or they may be different. The capsule allows the same or differing materials to be released into the alimentary duct, at different intervals of time in the same or in widely differing quantities.
Aq. shampoo, improving props of hair (e.g. lustre) contg. a surfactant, pref. a salt of a sulphonated or sulphated 12-21C fatty alcohol or its ether, and opt. 0.5-5 wt.% of a foam-reinforcing agent and a hair-cosmetic agent having a softening point 50 degrees C, is improved in that the hair-cosmetic agent (a resin, wax or oil) is kept in dispersion by means of a thickening and suspending agent (pref. 0.5-2 wt.% of a carboxylated acrylic polymer), and the hair-cosmetic agent is deposited on the hair when the shampoo is diluted with water during the shampooing. By this method various cosmetic agents can be uniformly deposited on hair.
Cancer patients often experience anemia due to the disease itself, chemotherapy, or both. Quality of life is also affected, due in part to the fatigue associated with anemia. Previous studies with epoetin alfa have suggested that achieving a higher hemoglobin level may improve quality of life and help patients live longer. This is a randomized, double-blind, placebo-controlled, multicenter study to assess the effect of treatment with epoetin alfa in reducing or preventing anemia and transfusions in patients receiving non-platinum chemotherapy for non-myeloid cancers. The study also aims to determine whether changes in erythropoietin and hemoglobin levels after 2 weeks, serum ferritin (iron) levels after 2 weeks and changes in hemoglobin and developing red blood cells after either 2 or 4 weeks predict responsiveness to epoetin alfa. There will be 2 treatment groups: one group will receive subcutaneous epoetin alfa injections 3 times per week (starting at 150 units per kilogram, adjusted if needed to a maximum of 300 units per kilogram) and the other group will receive an equal volume of matching subcutaneous placebo. Treatment is to continue for 12 to 24 weeks (3 to 6 chemotherapy cycles), plus 4 weeks post-chemotherapy. Patients in the study will be observed a year after the study ends for survival data. The primary measure of effectiveness will be determined by the number of patients who are transfused, depending on primary tumor type (solid or blood) and level of hemoglobin (above or below 10.5 grams per deciliter). Additional measures of effectiveness include the change in hemoglobin, hematocrit, and developing red blood cells from the start of the study until study completion, and the change in quality of life. Safety evaluations (incidence and severity of adverse events, laboratory tests, vital signs and physical examinations) will be performed throughout the study. The hypothesis of the study is that epoetin alfa will be superior to placebo in reducing the need for transfusions and in improving anemia and quality of life. 150 units per kilogram of epoetin alfa (or placebo) 3 times weekly subcutaneously for 4 weeks, then continuing this dose or 300 units per kilogram, depending on developing red blood cell counts and/or hemoglobin. Study duration is 12 to 24 weeks (3 to 6 cycles) plus 4 weeks post-chemotherapy.
BACKGROUND: Clinical trials for rare diseases often include multiple endpoints that capture the effects of treatment on different disease domains. In many rare diseases, the primary endpoint is not standardized across trials. The win ratio approach was designed to analyze multiple endpoints of interest in clinical trials and has mostly been applied in cardiovascular trials. Here, we applied the win ratio approach to data from COMET, a phase 3 trial in late-onset Pompe disease, to illustrate how this approach can be used to analyze multiple endpoints in the orphan drug context.
METHODS: All possible participant pairings from both arms of COMET were compared sequentially on changes at week 49 in upright forced vital capacity (FVC) % predicted and six-minute walk test (6MWT). Each participant's response for the two endpoints was first classified as a meaningful improvement, no meaningful change, or a meaningful decline using thresholds based on published minimal clinically important differences (FVC ± 4% predicted, 6MWT ± 39 m). Each comparison assessed whether the outcome with avalglucosidase alfa (AVA) was better than (win), worse than (loss), or equivalent to (tie) the outcome with alglucosidase alfa (ALG). If tied on FVC, 6MWT was compared. In this approach, the treatment effect is the ratio of wins to losses ("win ratio"), with ties excluded.
RESULTS: In the 2499 possible pairings (51 receiving AVA × 49 receiving ALG), the win ratio was 2.37 (95% confidence interval [CI], 1.30-4.29, p = 0.005) when FVC was compared before 6MWT. When the order was reversed, the win ratio was 2.02 (95% CI, 1.13-3.62, p = 0.018).
CONCLUSION: The win ratio approach can be used in clinical trials of rare diseases to provide meaningful insight on treatment benefits from multiple endpoints and across disease domains.
INTRODUCTION: Constriction band syndrome (CBS) is a congenital limb anomaly frequently associated with clubfoot. Clubfeet in CBS patients may be associated with peroneal nerve dysfunction in the involved lower extremity; however, the etiology of this neuromuscular dysfunction is not clear. We sought to characterize the distribution of constriction bands on lower extremities with clubfoot and determine if neuromuscular deficit (NMD), defined here as having absent ankle dorsiflexion, was associated with ipsilateral proximal bands. Our secondary aim was to compare the treatment and outcomes of clubfeet with NMD to those without NMD.
METHODS: We performed a retrospective review of all patients with CBS and clubfoot presenting to our facility between January 1, 1998 and December 31, 2018. Treatment with the Ponseti method, at least 1 year of follow-up at this facility, and a detailed physical exam describing lower extremity neuromuscular function and the presence and location of constriction bands were required for inclusion in the study cohort.
RESULTS: Twenty children with 26 clubfeet were included. Forty-six percent (12/26) of the clubfeet had NMD. Clubfeet with and without NMD had ipsilateral thigh or leg constriction bands at similar rates [42% (5/12) vs. 43% (6/14), P =0.106], and the majority (7/12) of clubfeet with NMD did not have an ipsilateral thigh or leg band. While children with an NMD clubfoot tended toward more casts, relapses, and surgical procedures, these differences did not reach statistical significance. The use of a daytime AFO beyond age four was higher in the NMD clubfeet [58% (7/12) vs. 14% (2/14), P =0.04].
CONCLUSION: Clubfeet with neuromuscular deficits may occur in the absence of proximal ipsilateral constriction bands, suggesting they may be caused by mechanisms other than direct damage from visible constriction bands to underlying nerves. They can also coexist with arthrogrypotic conditions. Clubfeet with an NMD tended toward more casts, relapses, and surgeries than those without NMD, but these differences did not reach statistical significance. These patients often elect long-term use of a daytime AFO.
The invention relates to medicinal application of microcystic toxins-LR, in particular to application of the microcystic toxins-LR in preparation of medicines for preventing and treating pulmonary fibrosis, belonging to the technical field of medicines. The microcystic toxins-LR is proved to be capable of improving the state of pulmonary fibrosis caused by bleomycin. The microcystic toxins-LR can inhibit the mRNA expression rise, caused by the bleomycin, of pulmonary fibrosis-related molecules such as transforming growth factor (TGF)-beta, tumor necrosis factor (TNF)-alpha, IL-1beta, IL-6, NF-kB and ET-1. The microcystic toxins-LR can inhibit the mRNA expression rise, caused by the bleomycin, of lung tissue vascular endothelial growth factor (VEGF). The microcystic toxins-LR interferes and relieves rat pulmonary fibrosis, induced by the bleomycin, by using ways such as a TGF-beta/Smad pathway and angiogenesis inhibition. A new way is provided for preparing the medicines for preventing and/or treating the pulmonary fibrosis disease.
A purified and modified phytase enzyme from Escherichia coli K12 appA phytase is provided. The enzyme has phytase activity and improved thermal tolerance as compared with the wild-type enzyme. In addition, the enzyme has improved protease stability at low pH. Glycosylation of the modified phytase provided a further improved enzyme having improved thermal tolerance and protease stability. The enzyme can be produced from native or recombinant host cells and can be used to aid in the digestion of phytase where desired. In particular, the phytase of the present invention can be used in foodstuffs to improve the feeding value of phytase rich ingredients.
Live vaccine against theileriosis in cattle consists of cattle lymphoid cells infested with Schizota of the attenuated strain Theileria annulata KTa No. 7. Pref. the vaccine is administered as a dose contg. 0.1-3 million living, infested clles. The vaccine is prepd. by growing T. annulata CY-1 (isolated from the mite Hyalomma detritum) on cattle lymphoid cells in a nutrient medium for 20-100 days, esp. in 6-12 passages over 60-80 days at 37.5-38.5 degrees C. The schizota are then collected and frozen in presence of a suitable cryoprotectant, esp. glycerine, at is not >-60 degrees C. The vaccine has high immunogenicity and is 10 times cheaper than known vaccines. It is only weakly reactogenic and induces no side effects in vaccinated cattle.
Pregnant women in the experimental group were administered pre-tests (Personal Information Form, Psychosocial Health Assessment Scale in Pregnancy and Wijma Birth Expectancy/ Experience Scale - A Version) just before watching the guided imagery video. In the first interview with pregnant women, video viewing days and hours were determined by giving information about the guided imagery video. Pregnant women were then shown the guided imagery video consisting of a total of 12 weeks, once a day for 15 minutes and 50 seconds. Immediately after the 12-week period was completed, post-tests (Psychosocial Health Evaluation Scale in Pregnancy and Wijma Birth Expectancy/ Experience Scale - A Version) were administered to the pregnant women.
Fibroblasts undergo metabolic reprogramming after contact with cancer cells in tumor microenvironment, producing lactate to provide a metabolic substrate for neighboring tumor cells. The exchange of lactate between cancer cells and fibroblasts via monocarboxylate transporters (MCTs) is known as the lactate shuttle. Colorectal cancer cells may establish a metabolic coupling akin to the lactate shuttle in collaboration with cancer-associated fibroblasts (CAFs) to augment their invasive and migratory capabilities. However, the specific phenomena and underlying mechanisms are not clear. In this study, we investigated the phenomena and explored the correlation and possible mechanism between CAFs and the invasion and migration of colorectal cancer cells by using two different co-culture models. The results showed that colorectal cancer cells established a lactate metabolic coupling with fibroblasts through the oxidative stress effect, triggering the metabolic reprogramming process of themselves and those of fibroblasts. In addition, lactate enhanced the invasion and migration of colorectal cancer by stabilizing the protein expression levels of nuclear factor kappa-B (NF-κB) and hypoxia-inducible factor-1α (HIF-1α). Blocking oxidative stress and lactate metabolic coupling with reactive oxygen species removers and MCT1-specific inhibitors, respectively, could effectively suppress metastasis in colorectal cancer. These findings suggest that targeting the lactate metabolic coupling between tumor cells and CAFs will offer a new strategy to combat colorectal cancer.
Epidemic evidence and animal studies suggested that hypothyroidism is related with maternal pregnancy outcome, offspring cognitive ability. Negro's study found improved maternal health in those screened in early trimester; while Lazarus et al. reported no significant offspring IQ score at 3yrs in universal screening in early trimester. Currently, it lacks evidence of universal pre-conception screening for maternal pregnancy outcome and offspring cognitive development, motivation ability, attention. China Governmental Health Department has included thyroid function screening into preconception in 2011. The investigators hypothesize that pre-conception effective treatment may be helpful for decrease possible negative effects on maternal and offspring health, instead of early trimester.