Expert consensus on the off-label use in China of drugs for rare hematologic diseases (2024 edition).

Drug package inserts are a crucial foundation for clinical medication practices and serve as the legal basis for guiding rational drug use and ensuring patient safety and efficacy. As rare disease treatments evolve, current package inserts often need to meet the clinical requirements for treating such conditions, frequently resulting in off-label drug use. This consensus is derived from discussions between Guangdong Pharmaceutical Association Hematologic Rare Diseases Group experts.

Unveiling the role of gut microbiota in curcumin metabolism using antibiotic-treated mice.

Curcumin might exert its therapeutic effects by interacting with gut microbiota. However, the role of gut microbiota in curcumin metabolism in vivo remains poorly understood. To address this, we used antibiotics to deplete gut microbiota and compared curcumin metabolism in control and antibiotic-treated mice.

Hierarchical Porous Reduced Graphene Oxide/Poly(l-lactic acid) Fiber Films: The Influence of Recrystallization on Strength.

Electrospinning technology for fabricating nanofiber films and the Hummer method for synthesizing graphene oxide (GO), along with subsequent reduction, have been significantly advanced, demonstrating immense potential for large-scale industrial applications. Nanofibrous films loaded with reduced graphene oxide (rGO) have been widely explored for their applications in electromagnetic shielding, the biomedical fields, and pollutant adsorption. However, fragile mechanical performance of electrospun fibers with limited surface post-treatment methods has somewhat hindered their further industrial development.

Systemic immune-inflammation index, neutrophil-to-lymphocyte ratio, and platelet-to-lymphocyte ratio in patients with type 2 diabetes at different stages of diabetic retinopathy.

Aim: To investigate systemic immune-inflammation index (SII), neutrophil-to-lymphocyte ratio (NLR), and platelet-to-lymphocyte ratio (PLR) levels in patients with type 2 diabetes at different stages of diabetic retinopathy (DR).

Methods: This retrospective study included 141 patients with type 2 diabetes mellitus (DM): 45 without diabetic retinopathy (NDR), 47 with non-proliferative diabetic retinopathy (NPDR), and 49 with proliferative diabetic retinopathy (PDR). Complete blood counts were obtained, and NLR, PLR, and SII were calculated.

Dietary tangeretin improved antibiotic-associated diarrhea in mice by enhancing the intestinal barrier function, regulating the gut microbiota, and metabolic homeostasis.

Antibiotic-associated diarrhea is mediated by antibiotic treatment and is usually caused by the disruption of the intestinal barrier, gut microbiota, and metabolic balance. To identify a dietary strategy that can mitigate the side effects of antibiotics, this study investigated the effect of tangeretin on antibiotic-associated diarrhea in C57BL/6 mice. The results revealed that dietary tangeretin significantly ameliorated symptoms of antibiotic-associated diarrhea, as evidenced by the decreased diarrhea status scores, the reduced fecal water content, the decreased caecum/body weight ratio, and the alleviated colonic tissue damage.

Detection and identification of imperatorin metabolites in rat, dog, monkey, and human liver microsomes by ultra-high-performance liquid chromatography combined with high-resolution mass spectrometry and Compound Discoverer software.

Imperatorin, a furanocoumarin that widely exists in many umbelliferous herbs, has been demonstrated to have a variety of pharmacological effects, including anti-inflammatory, antiosteoporosis, and antitumor activities. The purpose of this study was to investigate the metabolism of imperatorin using liver microsomes. The metabolites were generated by individually incubating imperatorin with rat, dog, monkey, and human liver microsomes.

Review of recent advances on health benefits, microbial transformations, and authenticity identification of Pericarpium bioactive compounds.

The extensive health-promoting effects of Pericarpium (CRP) have attracted researchers' interest. The difference in storage time, varieties and origin of CRP are closely related to the content of bioactive compounds they contain. The consitituent transformation mediated by environmental microorganisms (bacteria and fungi) and the production of new bioactive components during the storage process may be the main reason for 'the older, the better' of CRP.

Production Technology and Functionality of Bioactive Peptides.

Bioactive peptides are specific protein fragments that prove health-promoting potential for humans. The bioactivities include antimicrobial, antioxidant, anticancer, immunomodulatory activities, etc. Hence, bioactive peptides' production technology and processes have attracted excessive attention, especially concerning peptides' synthesis, separation, identification, and functionality.

Can SpRY recognize any PAM in human cells?

The application of clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated proteins (Cas) can be limited due to a lack of compatible protospacer adjacent motif (PAM) sequences in the DNA regions of interest. Recently, SpRY, a variant of Cas9 (SpCas9), was reported, which nearly completely fulfils the PAM requirement. Meanwhile, PAMs for SpRY have not been well addressed.

Structure and Properties of Organogels Prepared from Rapeseed Oil with Stigmasterol.

This work used the natural ingredient stigmasterol as an oleogelator to explore the effect of concentration on the properties of organogels. Organogels based on rapeseed oil were investigated using various techniques (oil binding capacity, rheology, polarized light microscopy, X-ray diffraction, and Fourier transform infrared spectroscopy) to better understand their physical and microscopic properties. Results showed that stigmasterol was an efficient and thermoreversible oleogelator, capable of structuring rapeseed oil at a stigmasterol concentration as low as 2% with a gelation temperature of 5 °C.

Prediction of Tacrolimus Dose/Weight-Adjusted Trough Concentration in Pediatric Refractory Nephrotic Syndrome: A Machine Learning Approach.

Purpose: Tacrolimus (TAC) is a first-line immunosuppressant for patients with refractory nephrotic syndrome (NS). However, there is a high inter-patient variability of TAC pharmacokinetics, thus therapeutic drug monitoring (TDM) is required. In this study, we aimed to employ machine learning algorithms to investigate the impact of clinical and genetic variables on the TAC dose/weight-adjusted trough concentration (C/D) in Chinese children with refractory NS, and then develop and validate the TAC C/D prediction models.

A patient with severe congenital neutropenia harbors a missense ELANE mutation due to paternal germline mosaicism.

Background: Clinical and genetic characteristics of ELANE mutation of a 3-year-old male who had a severe congenital neutropenia (SCN) were examined. We then investigated whether CRISPR/Cas9-mediated gene editing could correct the mutation.

Procedure: The proband underwent extensive clinical assessments, such as exome sequencing and bioinformatics analysis, so that pathogenic genes could be identified.

Early Prediction of Tacrolimus-Induced Tubular Toxicity in Pediatric Refractory Nephrotic Syndrome Using Machine Learning.

Tacrolimus(TAC)-induced nephrotoxicity, which has a large individual variation, may lead to treatment failure or even the end-stage renal disease. However, there is still a lack of effective models for the early prediction of TAC-induced nephrotoxicity, especially in nephrotic syndrome(NS). We aimed to develop and validate a predictive model of TAC-induced tubular toxicity in children with NS using machine learning based on comprehensive clinical and genetic variables.

Simultaneous determination of erythrocyte methotrexate polyglutamates by a novel and simple HPLC-MS/MS method with stable isotope-labeled internal standards.

Low-dose methotrexate is the first-line therapy for juvenile idiopathic arthritis. In vivo, methotrexate is converted into a series of methotrexate polyglutamates whose intracellular levels contribute significantly to its efficacy and toxicity. In this study, a novel high-performance liquid chromatography-tandem mass spectrometry method was developed and validated to simultaneously determine erythrocyte methotrexate polyglutamates using stable isotope-labeled internal standards.

Comparison of Pregnancy Outcomes Between Single-Morula Embryo Transfer and Single-Blastocyst Transfer in Fresh IVF/ICSI Cycles.

BACKGROUND This study investigated the effectiveness and feasibility of day 4 (D4) morula embryo transfer (ET) in comparison with day 5 (D5) blastocyst ET, with regards to their clinical data, laboratory test results, and pregnancy outcomes. MATERIAL AND METHODS This retrospective cohort study enrolled 1070 patients, including 178 cases in group D4 and 892 cases in group D5. The endpoint was live birth rate after fresh embryo transfer.

Early Prediction of Clinical Response to Etanercept Treatment in Juvenile Idiopathic Arthritis Using Machine Learning.

Background And Aims: At present, there is a lack of simple and reliable model for early prediction of the efficacy of etanercept in the treatment of juvenile idiopathic arthritis (JIA). This study aimed to generate and validate prediction models of etanercept efficacy in patients with JIA before administration using machine learning algorithms based on electronic medical record (EMR).

Materials And Methods: EMR data of 87 JIA patients treated with etanercept between January 2011 and December 2018 were collected retrospectively.

Effect of serum 25-hydroxyvitamin D levels on sperm quality and assisted reproductive technology outcomes for men of infertile Chinese couples.

Introduction: Vitamin D insufficiency/deficiency is highly prevalent in China, but its effect on sperm quality and outcomes of assisted reproductive technology (ART) in men from infertile couples is not clear. This study aimed to explore the association of serum 25-hydroxyvitamin D (25OHD) levels and semen parameters, as well as ART outcomes for men from infertile couples.

Materials And Methods: This study recruited 1308 men from infertile couples who attended the Reproductive Medicine Center of Peking University Third Hospital for first time from February to August 2019 (NCT03804294).

Gestational Perfluorooctanoic Acid Exposure Inhibits Placental Development by Dysregulation of Labyrinth Vessels and uNK Cells and Apoptosis in Mice.

Perfluorooctanoic acid (PFOA) is a widely used perfluorinated compound and known to cause developmental toxicity which includes the increase of resorbed embryo, decrease of fetal survival, and fetal growth retardation. Nevertheless, whether it is associated with alteration of placental development remains unknown. Pregnant mice were gavaged with 0, 2.

Roles of Vitamin D in Reproductive Systems and Assisted Reproductive Technology.

Vitamin D, an essential steroid hormone in the human body, plays an important role in not only the regulation of calcium and phosphorus metabolism, but also in various physiological processes, such as cell differentiation and apoptosis, inflammation, and insulin resistance. Vitamin D receptors are widely distributed in male and female reproductive systems, suggesting that vitamin D is essential for fertility. Because vitamin D deficiency is highly prevalent around the world, this review aims to discuss the potential functions of vitamin D in male and female reproductive systems and the associations between vitamin D and assisted reproductive technology (ART) outcomes.

Kidney podocyte-associated gene polymorphisms affect tacrolimus concentration in pediatric patients with refractory nephrotic syndrome.

Few studies have investigated the correlation between pharmacogenomics and tacrolimus pharmacokinetics in patients with nephrotic syndrome (NS). This study evaluated the influences of genetic polymorphisms of metabolic enzymes, transporters, and podocyte-associated proteins on tacrolimus concentration in Chinese pediatric patients with refractory NS. A total of 167 pediatric patients with refractory NS were included from July 2013 to December 2017.

Early and Accurate Prediction of Clinical Response to Methotrexate Treatment in Juvenile Idiopathic Arthritis Using Machine Learning.

Accurately predicting the response to methotrexate (MTX) in juvenile idiopathic arthritis (JIA) patients before administration is the key point to improve the treatment outcome. However, no simple and reliable prediction model has been identified. Here, we aimed to develop and validate predictive models for the MTX response to JIA using machine learning based on electronic medical record (EMR) before and after administering MTX.

Delayed development influences the outcome of different grades of D5 and D6 blastocysts during freeze-thaw cycle.

To analyze the effects of blastocysts on the 5th day (D5) and 6th day (D6) of frozen-thawed blastocyst transplantation on pregnancy outcome and provide evidence for further improvement of the strategy. This study included transfers from the Reproductive Medicine Center of the Second Affiliated Hospital of Wenzhou Medical University during freeze-thaw cycles from January 2016 to December 2017. They were divided into D5 group (1616 cases) and D6 group (619 cases) according to blastocyst formation and development.

Development and validation of a simple and sensitive high-resolution LC/MS method for determination of PF-04620110 in dog plasma: Application to a pharmacokinetic study.

In this study, a more sensitive and reliable quantitative method based on ultra-high performance liquid chromatography coupled with Q-Exactive-Orbitrap-MS in full-mass scan was developed and validated for the determination of PF-04620110 in dog plasma. After protein precipitation with acetonitrile, the sample separations were carried out on an Acquity BEH C column with 1 mm ammonium acetate in water and acetonitrile containing 0.1% acetic acid as mobile phase, at a flow rate of 0.

Light enhanced room temperature resistive NO sensor based on a gold-loaded organic-inorganic hybrid perovskite incorporating tin dioxide.

A material is described for sensing NO in the gas phase. It has an architecture of type Au/MASnI/SnO (where MA stands for methylammonium cation) and was fabricated by first synthesizing Au/MASnI and then crystallizing SnO on the surface by calcination. The physical and NO sensing properties of the composite were examined at room temperature without and with UV (365 nm) illumination, and the NO-sensing mechanism was studied.