Introduction: Mobilization regimes in pediatric patients at high risk for poor mobilization are not standardized across different institutions. We present a retrospective analysis of our experience with a high-dose granulocyte colony-stimulating factor (G-CSF) regime of 12 μg/Kg per body weight (BW) twice a day for 4 days used in high-risk patients.
Material And Methods: We report the results of all pediatric patients mobilized with high-dose G-CSF between January 1999 and February 2021 in our center. A successful mobilization was defined as a peripheral blood (PB) CD34 cell count of ≥10 CD34 cells/μl on the fifth day of mobilization immediately before leukapheresis. A minimum cell yield of ≥2 × 10 CD34 cells/Kg of BW was required for a successful collection.
Results: Of the 262 patients included in the analysis, mobilization failure was found in 27 (10.3%). In a univariate analysis, this was associated with age, weight, baseline diagnosis, and having undergone a previous mobilization cycle, the latter being the only factor that remained significantly associated in a multivariate analysis (P = 0.03). The 54 patients (20.6%) did not reach the minimum required CD34 cell yield. 50.4% of the patients reported adverse events (AEs) during the mobilization period, and 23 (9.1%) reported 3 or more concomitant AEs. However, all of them were mild and did not affect the mobilization schedule.
Conclusions: Although most high-risk pediatric patients are successfully mobilized with the high-dose G-CSF regime, this approach does not salvage all of them and significantly increases the presence of AEs in comparison to standard-dose regimes.
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http://dx.doi.org/10.1002/jca.21994 | DOI Listing |
Proc Natl Acad Sci U S A
January 2025
Cancer Biology & Genetics Program, Sloan Kettering Institute, New York, NY 10065.
Malignant peripheral nerve sheath tumors (MPNSTs) are aggressive sarcomas and the primary cause of mortality in patients with neurofibromatosis type 1 (NF1). These malignancies develop within preexisting benign lesions called plexiform neurofibromas (PNs). PNs are solely driven by biallelic loss eliciting RAS pathway activation, and they respond favorably to MEK inhibitor therapy.
View Article and Find Full Text PDFObjective: While the association of a syrinx with a tethered spinal cord in the context of VACTERL (vertebral defects [V], imperforate anus or anal atresia [A], cardiac malformations [C], tracheoesophageal defects [T] with or without esophageal atresia [E], renal anomalies [R], and limb defects [L]) association is known, the incidence of idiopathic syrinxes among these patients has not previously been reported. The authors aimed to characterize the incidence of syrinxes and the pattern of congenital anomalies in pediatric patients with VACTERL association, with a specific focus on the presence of idiopathic syrinxes in this population.
Methods: An institutional database was retrospectively queried for all pediatric patients with VACTERL association.
J Neurosurg
January 2025
2Department of Radiology, New York University Grossman School of Medicine, New York, New York.
Objective: The objective was to comprehensively investigate the clinical, molecular, and imaging characteristics and outcomes of H3 K27-altered diffuse midline glioma (DMG) in adults.
Methods: Retrospective chart and imaging reviews were performed in 111 adult patients with H3 K27-altered DMG from two tertiary institutions. Clinical, molecular, imaging, and survival characteristics were analyzed.
PLoS Negl Trop Dis
January 2025
Sustainable Sciences Institute, Managua, Nicaragua.
Background: Dengue virus, a major global health threat, consists of four serotypes (DENV1-4) that cause a range of clinical manifestations from mild to severe and potentially fatal disease.
Methods: This study, based on 19 years of data from the Pediatric Dengue Cohort Study and Pediatric Dengue Hospital-based Study in Managua, Nicaragua, investigates the relationship of serotype and immune status with dengue severity. Dengue cases were confirmed by molecular, serological, and/or virological methods, and study participants 6 months to 17 years old were followed during their hospital stay or as ambulatory patients.
Pain
February 2025
Department of Anesthesiology, Peking University People's Hospital, Beijing, China.
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