Introduction: The use of peripheral blood progenitor cells (PBPCs) as a source for hematopoietic stem cell transplantation (HSCT) in pediatric healthy donors is still under debate. The risk of a central venous catheter (CVC) placement and catheter-related complications continue to be the main arguments to discourage its use.
Methods: we present a retrospective analysis of 140 PBPC collections in pediatric patients and donors, describing adverse events (AE) related to CVCs as well as the influence of catheterrelated variables on the efficiency of the leukapheresis.
Results: 14 CVC-related AEs were recorded (10%). The most common was fever in 5 patients, 4 of which had a catheter-related bacteriemia. Thrombotic events were only observed in 3 patients with active malignancy. A healthy donor presented a moderate bleeding after catheter withdrawal that resolved with local measures, and none of the rest presented any AE. Regarding variables related to the development of AEs, the subject group (patient or donor) was the only one significantly associated ( < 0.0001). Of interest, efficiency was also related to catheter location, being worse in those located in the femoral vein than in into the jugular or the subclavian veins ( < 0.05). In a multivariate analysis, the only variable significantly associated was catheter size (beta 0.238, < 0.01).
Discussion: Placing a CVC for PBPC collection in pediatric subjects is overall safe; CVC-related complications in pediatric healthy donors are very rare. Furthermore, we should try to place catheters of the largest caliber possible, since the efficiency of the collection is related to this variable.
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http://dx.doi.org/10.3389/fped.2023.1131905 | DOI Listing |
J Transl Med
January 2025
Scientia Clinical Research and Prince of Wales Clinical School, University of New South Wales, Sydney, NSW, 2052, Australia.
Background: A novel anti-human epidermal growth factor receptor 2 (HER2) antibody-drug conjugate (ADC) GQ1001 was assessed in patients with previously treated HER2 positive advanced solid tumors in a global multi-center phase Ia dose escalation trial.
Methods: In this phase Ia trial, a modified 3 + 3 study design was adopted during dose escalation phase. Eligible patients were enrolled, and GQ1001 monotherapy was administered intravenously every 3 weeks.
Implement Sci
January 2025
University of California, Los Angeles, Los Angeles, CA, 90025, USA.
Background: Remaking Recess (RR) is a school-based evidence-based peer social engagement intervention for autistic students. RR involves direct training and coaching with educators; however, educators face several barriers to implementation at both the individual- and organizational-levels. This protocol paper describes a multi-site study that will test whether an educator-level implementation strategy, coaching, with or without a school-level implementation strategy, school-based teams, will maximize educators' use (fidelity and sustainment) of RR for autistic students and their peers who are socially-isolated, rejected, or peripheral and may need additional support during recess.
View Article and Find Full Text PDFOrphanet J Rare Dis
January 2025
Department of Cardiac Physiology, National Cerebral and Cardiovascular Center Research Institute, 6-1 Kishibe-Shimmachi, Suita, Osaka, 564-8565, Japan.
Background: Transient receptor potential cation channel subfamily V member 2 (TRPV2) functions as a stretch-sensitive calcium channel, with overexpression in the sarcolemma of skeletal and cardiac myocytes leading to detrimental calcium influx and triggering muscle degeneration. In our previous pilot study, we showed that tranilast, a TRPV2 inhibitor, reduced brain natriuretic peptide levels in two patients with muscular dystrophy and advanced heart failure. Building on this, we performed a single-arm, open-label, multicenter study herein to evaluate the safety and efficacy of tranilast in the treatment of advanced heart failure in patients with muscular dystrophy.
View Article and Find Full Text PDFBMC Gastroenterol
January 2025
Division of Gastroenterology, Department of Internal Medicine, Showa University Fujigaoka Hospital, Yokohama, Kanagawa, Japan.
Background: Endoscopic ultrasound-guided transmural drainage (EUS-TD) is widely performed to treat postoperative peripancreatic fluid collection (POPFC). Recent reports on EUS-TD lack a consensus on stent selection. This study aimed to assess the efficacy of EUS-TD for POPFC using an external drainage-based approach.
View Article and Find Full Text PDFBMC Gastroenterol
January 2025
Department of Gastroenterology, Jiangxi Provincial Key Laboratory of Digestive Diseases, Jiangxi Clinical Research Center for Gastroenterology, Digestive Disease Hospital, The First Affiliated Hospital, Jiangxi Medical College, Nanchang University, No.17 Yongwaizheng Street, Nanchang, Jiangxi, 330006, China.
Background: Endoscopic gallbladder-preserving cholecystolithotomy (EGPC) has become an alternative option for treating cholecystolithiasis. However, developing a new method of EGPC in which the gallbladder wall is not damaged remains a challenge. This study introduced a new EGPC method called endoscopic retrograde cholangiopancreatography (ERCP) combined with extracorporeal shock wave lithotripsy (ESWL), which preserves the integrity of the gallbladder wall in the treatment of cholecystolithiasis complicated with choledocholithiasis.
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