RESUMO
Here we report efficacy, pharmacokinetics, and safety data obtained in treatment-naive, pediatric patients with newly diagnosed advanced MDS receiving azacitidine in the AZA-JMML-001 study. The primary endpoint was response rate (proportion of patients with complete response [CR], partial response [PR], or marrow CR, sustained for ≥4 weeks). Of the 10 patients enrolled, one had an unconfirmed marrow CR and none had confirmed responses after three cycles; the study was therefore closed after stage 1. Azacitidine was well tolerated. The lack of efficacy of azacitidine in pediatric patients with newly diagnosed advanced MDS highlights the need for effective new treatments in these patients.
Assuntos
Transplante de Células-Tronco Hematopoéticas , Leucemia Mieloide Aguda , Síndromes Mielodisplásicas , Humanos , Criança , Azacitidina/efeitos adversos , Leucemia Mieloide Aguda/tratamento farmacológico , Síndromes Mielodisplásicas/tratamento farmacológico , Síndromes Mielodisplásicas/diagnóstico , Resultado do Tratamento , Transplante de Células-Tronco Hematopoéticas/efeitos adversosRESUMO
OBJECTIVES: To develop a cultural and trauma-informed mental health self-management program for immigrant Latina survivors of adverse childhood experiences with depression or anxiety symptoms. METHOD: Guided by Barrera's five-stage process for cultural adaptation, we collaborated with multiple stakeholders including clinical psychologists, community health workers, and Latina immigrant women with a history of adverse childhood experiences and depression or anxiety to transform a chronic disease self-management program to be trauma informed, culturally appropriate, and focus on self-management of depression and anxiety symptoms. RESULTS: Adaptations included translating program materials to Spanish, education on how early life adversity and trauma may impact mental health, virtual delivery, more frequent and shorter sessions, and addition of graphics and written prompts in workbook materials. For the facilitator's manual, culturally relevant vignettes and guidance were added to guide participants through activities and adapt sessions based on participant needs. CONCLUSIONS: Barrera's five-stage process was useful for adapting a program to be both trauma and culturally informed for an underserved population disproportionately affected by trauma and limited access to mental health services. The adaptation demonstrated acceptability with Latina immigrant women and the promise of utilizing unlicensed personnel and technology for increasing the reach of mental health support. (PsycInfo Database Record (c) 2024 APA, all rights reserved).
Assuntos
Anemia Aplástica , Soro Antilinfocitário , Ciclosporina , Imunossupressores , Humanos , Soro Antilinfocitário/uso terapêutico , Anemia Aplástica/tratamento farmacológico , Ciclosporina/uso terapêutico , Criança , Imunossupressores/uso terapêutico , Cavalos , Animais , Masculino , Coelhos , Feminino , Adolescente , Pré-Escolar , Resultado do Tratamento , Lactente , Estudos RetrospectivosRESUMO
Frogs are an ecologically diverse and phylogenetically ancient group of anuran amphibians that include important vertebrate cell and developmental model systems, notably the genus Xenopus. Here we report a high-quality reference genome sequence for the western clawed frog, Xenopus tropicalis, along with draft chromosome-scale sequences of three distantly related emerging model frog species, Eleutherodactylus coqui, Engystomops pustulosus, and Hymenochirus boettgeri. Frog chromosomes have remained remarkably stable since the Mesozoic Era, with limited Robertsonian (i.e., arm-preserving) translocations and end-to-end fusions found among the smaller chromosomes. Conservation of synteny includes conservation of centromere locations, marked by centromeric tandem repeats associated with Cenp-a binding surrounded by pericentromeric LINE/L1 elements. This work explores the structure of chromosomes across frogs, using a dense meiotic linkage map for X. tropicalis and chromatin conformation capture (Hi-C) data for all species. Abundant satellite repeats occupy the unusually long (~20 megabase) terminal regions of each chromosome that coincide with high rates of recombination. Both embryonic and differentiated cells show reproducible associations of centromeric chromatin and of telomeres, reflecting a Rabl-like configuration. Our comparative analyses reveal 13 conserved ancestral anuran chromosomes from which contemporary frog genomes were constructed.