Rare Non-Cryptic Rearrangements Associated With Myeloid Neoplasms and Their Poor Prognostic Impact.

Background: rearrangements (r), associated with various hematologic malignancies, involve more than 30 partner genes. Despite their clinical significance, reports on the clinicopathological characteristics of rare r remain limited. We investigated the characteristics of patients with myeloid neoplasms harboring r among those identified as having 11p15 translocation in chromosomal analysis.

QuantiFERON monitor predicts early cytomegalovirus infection and viral burden in allogeneic hematopoietic stem cell transplantation.

Introduction: Cytomegalovirus (CMV) infection is a major cause of transplantation-related morbidity and mortality. This study assessed the utility of the QuantiFERON monitor (QFM; Qiagen) for the prediction of early CMV infection and viral burden.

Methods: QuantiFERON-CMV (QF-CMV; Qiagen) and QFM were measured at the post-allogeneic hematopoietic stem cell transplantation (HSCT) week 4.

Quadriceps recovery and pain relief in knee osteoarthritis rats by cog polydioxanone filament insertion.

Quadriceps muscles play a pivotal role in knee osteoarthritis (OA) progression and symptom manifestation, particularly pain. This research investigates the therapeutic effectiveness of muscle enhancement and support therapy (MEST), a recently developed device intended for intramuscular insertion of cog polydioxanone filaments, in quadriceps restoration to alleviate OA pain. Knee OA was induced in Sprague Dawley rats via monoiodoacetate injections.

Tuning the CO Reduction Selectivity of an Immobilized Molecular Ag Complex beyond CO.

The electrochemical reduction of carbon dioxide (CO) to produce fuels and chemicals has garnered significant attention. However, achieving control over the selectivity of the resulting products remains a challenging task, particularly within molecular systems. In this study, we employed a molecular silver complex immobilized on graphitized mesoporous carbon (GMC) as a catalyst for converting CO into CO, achieving an impressive selectivity of over 90% at -1.

Validation of the 2022 European LeukemiaNet risk stratification for acute myeloid leukemia.

This study aimed to validate the 2022 European LeukemiaNet (ELN) risk stratification for acute myeloid leukemia (AML). A total of 624 newly diagnosed AML patients from 1998 to 2014 were included in the analysis. Genetic profiling was conducted using targeted deep sequencing of 45 genes based on recurrent driver mutations.

Alleviation of peripheral sensitization by quadriceps insertion of cog polydioxanone filaments in knee osteoarthritis rats.

A recently established therapeutic strategy, involving the insertion of biodegradable cog polydioxanone filaments into the quadriceps muscles using the Muscle Enhancement and Support Therapy (MEST) device, has demonstrated significant efficacy in alleviating knee osteoarthritis (OA) pain. This study investigated changes in peripheral sensitization as the potential mechanism underlying MEST-induced pain relief in monoiodoacetate (MIA) induced OA rats. The results revealed that MEST treatment potently reduces MIA-induced sensitization of L3/L4 dorsal root ganglion (DRG) neurons, the primary nociceptor pathway for the knee joint.

Paired Electrosynthesis of Formaldehyde Derivatives from CO Reduction and Methanol Oxidation.

Utilizing CO -derived formaldehyde derivatives for fuel additive or polymer synthesis is a promising approach to reduce net carbon dioxide emissions. Existing methodologies involve converting CO to methanol by thermal hydrogenation, followed by electrochemical or thermochemical oxidation to produce formaldehyde. Adding to the conventional methanol oxidation pathway, we propose a new electrochemical approach to simultaneously generate formaldehyde derivatives at both electrodes by partial methanol oxidation and the direct reduction of CO .

The Effect of Evogliptin Tartrate on Controlling Inflammatory Pain.

: Evogliptin tartrate inhibits dipeptidyl peptidase-4 (DPP-4), boosting glucagon-like peptide 1 (GLP-1) secretion and improving insulin release and glucose tolerance, while also exerting anti-inflammatory effects. We investigated its anti-inflammatory and analgesic effects. : Forty male Sprague Dawley rats were divided into (N = 10 in each): (1) naïve, (2) complete Freund's adjuvant (CFA) inflammation + evogliptin tartrate (once for 10 mg/kg) (CFAE), (3) CFA + vehicle (same volume with normal saline with evogliptin tartrate/once) (CFAV), and (4) CFA + indomethacin (5 mg/mL/kg/1 time) (CFAI) groups.

Long-Term Efficacy and Safety of Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria and High Disease Burden: Real-World Data From Korea.

Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare hematologic disorder characterized by uncontrolled terminal complement activation. Eculizumab, a monoclonal antibody C5 inhibitor was introduced in Korea in 2009 and has been the standard treatment option for PNH.

Methods: This study assessed the long-term efficacy/safety of eculizumab in PNH using real-world data from the Korean Health Insurance Review and Assessment Service.

New Ternary Blend Strategy Based on a Vertically Self-Assembled Passivation Layer Enabling Efficient and Photostable Inverted Organic Solar Cells.

Herein, a new ternary strategy to fabricate efficient and photostable inverted organic photovoltaics (OPVs) is introduced by combining a bulk heterojunction (BHJ) blend and a fullerene self-assembled monolayer (C -SAM). Time-of-flight secondary-ion mass spectrometry - analysis reveals that the ternary blend is vertically phase separated with the C -SAM at the bottom and the BHJ on top. The average power conversion efficiency - of OPVs based on the ternary system is improved from 14.

A phase III, randomised, double-blind, multi-national clinical trial comparing SB12 (proposed eculizumab biosimilar) and reference eculizumab in patients with paroxysmal nocturnal haemoglobinuria.

Treatment of paroxysmal nocturnal haemoglobinuria (PNH) includes the monoclonal antibody eculizumab. This randomised, double-blind, multi-national cross-over Phase III study in PNH patients aimed to demonstrate the equivalence of the proposed eculizumab biosimilar SB12 and reference eculizumab (Soliris, ECU). PNH patients with lactate dehydrogenase (LDH) ≥1·5× upper limit of normal were randomised into treatment sequences SB12-ECU or ECU-SB12.

Clinical Significance of bZIP In-Frame CEBPA-Mutated Normal Karyotype Acute Myeloid Leukemia.

Purpose: We evaluated the characteristics of CCAAT/enhancer-binding protein α (CEBPA) mutations and the significance of a basic leucine zipper in-frame mutation (bZIPin-f) of CEBPA in patients with acute myeloid leukemia with a normal karyotype.

Materials And Methods: Based on updated knowledge of CEBPA mutations, we conducted next-generation sequencing analyses in a previously established real-world cohort.

Results: Among 78 of a total of 395 patients (19.

Biosimilars in rare diseases: a focus on paroxysmal nocturnal hemoglobinuria.

Biologics, a class of medicines grown in and purified from genetically engineered cell cultures, have transformed the management of many cancers and rare diseases, such as paroxysmal nocturnal hemoglobinuria. As prescription drug spending has increased and exclusivity periods have expired, manufacturers have developed biosimilars-biologics that may be more affordable and highly similar to a licensed biological therapeutic, with no clinically meaningful differences in terms of safety or efficacy. With biosimilars gaining regulatory approval around the globe and broadening patient access to biologics, this review aims to help rare disease healthcare providers familiarize themselves with biosimilars, understand their development and regulatory approval process, and address practical considerations that may facilitate their use.

Effects of -Mutant Type and Burden on the Phenotype of Myeloproliferative Neoplasms.

Somatic mutations occur in approximately 70% of patients with V617F-negative essential thrombocythemia (ET) and primary myelofibrosis (PMF). We evaluated the effects of the mutant type and burden on the phenotype of -mutated myeloproliferative neoplasms (MPN). Of the 510 patients with suspected or diagnosed MPN, all 49 patients detected with mutations were diagnosed with ET (n = 32) or PMF (n = 17).

Allogeneic hematopoietic cell transplantation can overcome the adverse prognosis indicated by secondary-type mutations in de novo acute myeloid leukemia.

Secondary-type mutations (STMs), namely SRSF2, SF3B1, U2AF1, ZRSR2, ASXL1, EZH2, BCOR, and STAG2, are more frequently detected in secondary acute myeloid leukemia (AML) than in de novo AML. Whether de novo AML with STMs should be differently managed is, however, unclear. In 394 patients diagnosed with de novo AML who had a normal karyotype, the genetic profiling via targeted deep sequencing of 45 genes revealed 59 patients carrying STMs (STM).

Eltrombopag in patients with chronic immune thrombocytopenia in Asia-Pacific, the Middle East, and Turkey: final analysis of CITE.

CITE was a prospective, noninterventional study in adult patients with chronic immune thrombocytopenia treated with eltrombopag under routine clinical care in Asia-Pacific, the Middle East, and Turkey. Data to assess eltrombopag usage, compliance, and outcomes were collected from May 2017 to December 2020. Platelet response was defined as platelet count ≥50 × 103/μL in the absence of rescue medications and splenectomy.

Early diagnosis of Gaucher disease in Korean patients with unexplained splenomegaly: a multicenter observational study.

Background: Gaucher disease (GD) is an autosomal recessive disorder characterized by excessive accumulation of glucosylceramide in multiple organs. This study was performed to determine the detection rate of GD in a selected patient population with unexplained splenomegaly in Korea.

Methods: This was a multicenter, observational study conducted at 18 sites in Korea between December 2016 and February 2020.