Ruxolitinib for pediatric acute and chronic graft-versus-host disease: a single-center retrospective study of efficacy and safety.

Graft-versus-host disease (GVHD) is a major concern for patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT). Ruxolitinib has been proven effective in treating adult steroid-refractory GVHD; however, studies on pediatric patients are relatively scarce. Thus, this single-center study evaluated the efficacy and safety of ruxolitinib in pediatric patients with steroid-refractory GVHD.

Disease burden, management strategies, and unmet needs in α-thalassemia due to hemoglobin H disease.

Alpha-thalassemia is an inherited blood disorder caused by impaired α-globin chain production, leading to anemia and other complications. Hemoglobin H (HbH) disease is caused by a combination of mutations generally affecting the expression of three of four α-globin alleles; disease severity is highly heterogeneous, largely driven by genotype. Notably, non-deletional mutations cause a greater degree of ineffective erythropoiesis and hemolysis, higher transfusion burden, and increased complication risks versus deletional mutations.

Ruxolitinib in treatment-naive or corticosteroid-refractory paediatric patients with chronic graft-versus-host disease (REACH5): interim analysis of a single-arm, multicentre, phase 2 study.

Background: Chronic graft-versus-host disease (GVHD) is a debilitating, and sometimes life threatening, complication of allogeneic haematopoietic stem-cell transplantation (HSCT). We aimed to investigate the activity, pharmacokinetics, and safety of ruxolitinib added to corticosteroids in paediatric patients (ie, <18 years) with moderate-to-severe chronic GVHD.

Methods: In this single-arm, phase 2 study, patients were recruited at 21 hospitals or clinics across 14 countries in Asia, Europe, and Canada.

Higher tumor mutational burden is associated with inferior outcomes among pediatric patients with neuroblastoma.

Introduction: Neuroblastoma is a pediatric malignancy with heterogeneous clinical outcomes. Our aim was to identify prognostic genetic markers for patients with neuroblastoma, who were treated with the Taiwan Pediatric Oncology Group (TPOG) neuroblastoma N2002 protocol, to improve risk stratification and inform treatment.

Methods: Our analysis was based on 53 primary neuroblastoma specimens, diagnosed pre-chemotherapy, and 11 paired tumor relapse specimens.

Feasibility of F-DOPA and F-FDG PET/CT for guiding decision-making for localized incidental neuroblastoma in infants under 18 months of age.

Background: Neuroblastoma varies widely in risk. Risk indicators in infants with incidental neuroblastoma refine treatment confidence for observation or intervention. The potential of functional imaging, particularly PET/CT, remains to be defined.

Terminal deoxynucleotidyl transferase expression in different subtypes of childhood B-cell acute lymphoblastic leukemia.

The lack of expression of terminal deoxynucleotidyl transferase (TdT) is frequently associated with KMT2A-rearranged subtype of pediatric acute lymphoblastic leukemia (ALL). However, this association has not been investigated extensively in the Asian population. A retrospective analysis of TdT expression in pediatric B-cell ALL (B-ALL) was performed in patients treated using the Taiwan Pediatric Oncology Group (TPOG) ALL 2002 and 2013 protocols.

Allele-specific polymerase chain reaction can determine the diplotype of NUDT15 variants in patients with childhood acute lymphoblastic Leukemia.

Mercaptopurine intolerance is an adverse effect of mercaptopurine administration in pediatric patients with acute lymphoblastic leukemia (ALL). NUDT15 variants have emerged as major determinants of mercaptopurine intolerance, especially in the Asian population. Two variants, c.

A microRNA signature for clinical outcomes of pediatric ALL patients treated with TPOG protocols.

MicroRNA (miRNA) expression is reportedly associated with clinical outcomes in childhood acute lymphoblastic leukemia (ALL). Here, we aimed at investigating whether miRNA expression is associated with clinical outcomes in pediatric ALL patients treated with the Taiwan Pediatric Oncology Group (TPOG) protocols. The expression of 397 miRNAs was measured using stem-loop quantitative real-time polymerase chain reaction miRNA arrays in 60 pediatric ALL patients treated with TPOG-ALL-93 or TPOG-ALL-97 VHR (very high-risk) protocols.

High frequency of heat shock protein 27 overexpression is a highly effective, high-coverage marker for minimal residual disease detection in children with B-cell acute lymphoblastic leukemia.

Background: Acute lymphoblastic leukemia (ALL) is the most common pediatric cancer. Minimal residual disease (MRD) detection is the most powerful prognostic tool for monitoring treatment efficacy and predicting clinical outcomes. We aimed to identify key leukemia-associated markers, the proportions of differential expression in patients, and the most effective marker combination for MRD detection by flow cytometry.

Surgical treatment confers prognostic significance in pediatric malignant mediastinal germ cell tumors.

Background: Primary malignant mediastinal germ cell tumors (GCTs) are rare pediatric tumors that have a poorer prognosis compared to GCTs occurring elsewhere in the body. The current study aimed to assess the prognostic factors and treatment outcomes of children with primary malignant mediastinal GCT in Taiwan.

Methods: The authors retrospectively reviewed children 0-18 years old who were newly diagnosed with primary malignant mediastinal GCT between January 1, 2005 and December 31, 2019 and were registered in the Taiwan Pediatric Oncology Group patient registry.

Corrigendum: Methylation Statuses of H19DMR and KvDMR at WT2 in Wilms Tumors in Taiwan.

[This corrects the article DOI: 10.1007/s12253-020-00802-6.].

Development of a Therapeutic Video Game With the MDA Framework to Decrease Anxiety in Preschool-Aged Children With Acute Lymphoblastic Leukemia: Mixed Methods Approach.

Background: Preschool-aged children with acute lymphoblastic leukemia (ALL) receive long-term treatment according to the Taiwan Pediatric Oncology Group (TPOG)-ALL 2013 protocol. Severe anxiety and noncompliance ahead of frequent invasive therapies leads to an increase in health care costs. Previous studies have shown that therapeutic video games (TVGs) can decrease the anxiety experienced by children who are ill.

Sequential Approach to Improve the Molecular Classification of Childhood Acute Lymphoblastic Leukemia.

Identification of specific leukemia subtypes is a key to successful risk-directed therapy in childhood acute lymphoblastic leukemia (ALL). Although RNA sequencing (RNA-seq) is the best approach to identify virtually all specific leukemia subtypes, the routine use of this method is too costly for patients in resource-limited countries. This study enrolled 295 patients with pediatric ALL from 2010 to 2020.

[Effects of Tropical Cyclones on Ozone Pollution in the Pearl River Delta in Autumn].

Based on the tropical cyclone track data in the northwest Pacific Ocean from 2015 to 2020, meteorological observation data, and ozone concentration monitoring data in the Pearl River Delta (PRD), the impacts of four tropical cyclones, namely the westbound tropical cyclone (type A), East China Sea tropical cyclone (type B), offshore tropical cyclone (type C), and offshore tropical cyclone (type D), on ozone concentration in the PRD were analyzed. The results showed that:under the influence of the type A tropical cyclone, the risk of regional ozone concentration exceeding the standard exhibited little change. Under the influence of the type B tropical cyclone, the risk of ozone exceeding the standard in the PRD was obviously increased.

Correlations between histological characterizations and methylation statuses of tumour suppressor genes in Wilms' tumours.

Wilms' tumour is a solid tumour that frequently occurs in children. Genetic changes in WT1 and epigenetic aberrations that affect imprinted control region 1 in WT2 loci are implicated in its aetiology. Moreover, tumour suppressor genes are frequently silenced by methylation in this tumour.

C1GALT1 expression predicts a favorable prognosis and suppresses malignant phenotypes via TrkA signaling in neuroblastoma.

Neuroblastoma (NB) is a childhood tumor derived from the sympathoadrenal lineage of the neural crest progenitor cells. Core 1 β1,3-galactosyltransferase (C1GALT1) controls the crucial step of GalNAc-type O-glycosylation, and its altered expression affects cancer behaviors. However, the role of C1GALT1 in NB tumors remains unclear.

Integration of immunohistochemistry, RNA sequencing, and multiplex ligation-dependent probe amplification for molecular classification of pediatric medulloblastoma.

Background: Medulloblastoma (MB) is commonly classified into four molecular groups, that is, WNT, SHH, group 3, and group 4, for prognostic and therapeutic purposes.

Methods: Here we applied immunohistochemistry (IHC) and RNA sequencing (RNA-seq) for the molecular classification of MB, and utilized multiplex ligation-dependent probe amplification (MLPA) to determine chromosomal alterations and specific gene amplifications.

Results: We retrospectively enrolled 37 pediatric MB patients.

Prognostic Value of Interim 18F-DOPA and 18F-FDG PET/CT Findings in Stage 3-4 Pediatric Neuroblastoma.

Purpose: This retrospective study aimed to determine the prognostic value of imaging parameters derived from midtherapy 18F-fluorodihydroxyphenylalanine (18F-DOPA) and 18F-FDG PET in pediatric patients with stage 3-4 neuroblastoma.

Methods: We enrolled 32 stage 3-4 pediatric neuroblastoma patients who underwent 18F-DOPA and 18F-FDG PET/CT scans before and after 3 chemotherapy cycles. We measured metabolic and volumetric parameters and applied a metabolic burden scoring system to evaluate the primary tumor extent and soft tissue metastases and that of bone/bone marrow involvement.

Acute pancreatitis in children with acute lymphoblastic leukemia correlates with L-asparaginase dose intensity.

Background: L-Asparaginase (L-Asp) is an important therapeutic for childhood acute lymphoblastic leukemia (ALL). Asparaginase-associated pancreatitis (AAP) is a severe complication of L-Asp related to the dosage. We investigated the incidence of, and risk factors for, AAP in pediatric patients with ALL.

Relationship between transfusion burden, healthcare resource utilization, and complications in patients with beta-thalassemia in Taiwan: A real-world analysis.

Background: This study utilized a population-based claims database to identify patients with beta-thalassemia and evaluate associations between transfusion burden, healthcare resource utilization (HCRU), and complications.

Study Design And Methods: Taiwan's National Health Insurance Research Database was used to identify patients with beta-thalassemia (ICD-10 D56.1) in 2016.