Background: The cytogenetics of acute myeloid leukemia (AML) increases exponentially with age. Adolescent and young adult (AYA) patients have specific psychosocial and other challenges, influencing their ability to access appropriate treatment. Therefore, in allogeneic hematopoietic stem cell transplantation (allo-HSCT) for AML, inferior outcomes would be observed in AYA patients compared to children.
Methods: We defined the age range of AYA patients as 15 to 29 years. Sixty-three patients who underwent allo-HSCT from 1998 to 2020 at Chang Gung Children Hospital were enrolled in this study. Overall survival was the time duration from HSCT to death from any cause. Disease-free survival was the time duration from HSCT to the last follow-up or first event (failure to achieve complete remission, relapse, secondary malignancy, or death from any cause).
Results: Thirty-seven (59%) patients were <15 years of age during allo-HSCT, and 26 (41%) were 15 to 29 years of age. The median age during allo-HSCT was 6.3 years for those <15 years of age compared with 15.7 years for AYA patients. The median follow-up period was 2.2 years after hematopoietic stem cell transplantation for patients <15 years old and 3.8 years after hematopoietic stem cell transplantation for AYA patients. Univariate analysis revealed no significant difference in the 5-year overall survival or disease-free survival among all patients.
Conclusions: Several distinct AML subtypes could be amenable to treatment deintensification and targeted therapies. Furthermore, we found that children and AYA patients who underwent allo-HSCT for AML had similar survival.
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http://dx.doi.org/10.1016/j.transproceed.2021.09.022 | DOI Listing |
Pediatr Blood Cancer
January 2025
Department of Pediatrics, Benioff Children's Hospitals, University of California, San Francisco, California, USA.
Background: Thrombopoietin receptor agonists (TPO-RAs) have demonstrated efficacy in treating clinically significant thrombocytopenia, including chemotherapy-induced thrombocytopenia in adults. However, data regarding their safety and efficacy in pediatric, adolescents, and young adult (AYA) patients with hematologic malignancies are limited.
Methods: We retrospectively identified 15 pediatric and AYA patients aged 25 years or younger with hematologic malignancies treated with a TPO-RA at UCSF Benioff Children's Hospitals between 2015 and 2023.
Target Oncol
January 2025
College of Medicine and Public Health, Flinders University, Adelaide, SA, Australia.
Background: Tumour mutational burden (TMB) is an established biomarker for patients treated with immune checkpoint inhibitors (ICIs). The optimal TMB cut-off is uncertain. It is also uncertain whether there is a sharp TMB threshold or a more graduated change in clinical outcomes as TMB increases.
View Article and Find Full Text PDFInt J Retina Vitreous
January 2025
New England Eye Center, Tufts Medical Center, Tufts University School of Medicine, Boston, MA, USA.
Purpose: To assess the repeatability of a microperimetry methodology for quantifying visual function changes in the junctional zone of eyes with geographic atrophy (GA) in the clinical trial context.
Methods: A post hoc analysis of the OAKS phase III trial was conducted, which enrolled patients with GA secondary to age-related macular degeneration. Microperimetry using a standard 10 - 2 fovea centered grid was performed at baseline and follow-up visits.
Int J Mol Sci
December 2024
Istituto di Ricerca Pediatrica "Città della Speranza", 35128 Padua, Italy.
Currently, risk stratification for pediatric Hodgkin lymphoma is based on clinical factors such as stage, bulk, and systemic symptoms. Novel minimally invasive biomarkers could enhance both prognosis and treatment strategies. Therefore, the plasma extracellular vesicles' microRNA profile was characterized by small RNA sequencing in 36 classical Hodgkin lymphoma cases and these findings were confirmed in an extended cohort of 86 patients by RT-qPCR.
View Article and Find Full Text PDFEClinicalMedicine
October 2024
Centre for Psychedelic Research, Division of Psychiatry, Department Brain Sciences, Imperial College London, United Kingdom.
Background: Psilocybin therapy (PT) produces rapid and persistent antidepressant effects in major depressive disorder (MDD). However, the long-term effects of PT have never been compared with gold-standard treatments for MDD such as pharmacotherapy or psychotherapy alone or in combination.
Methods: This is a 6-month follow-up study of a phase 2, double-blind, randomised, controlled trial involving patients with moderate-to-severe MDD.
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