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One hundred thirty-four germ line PU.1 variants and the agammaglobulinemic patients carrying them.
Blood
January 2025
Division of Immunology and Allergy, Children's Hospital of Philadelphia; Department of Pediatrics, Perelman School of Medicine; Institute for Immunology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, United States.
Leukopoiesis is lethally arrested in mice lacking the master transcriptional regulator PU.1. Depending on the animal model, subtotal PU.
View Article and Find Full Text PDFNeeds Assessment to Guide the Development of a Pediatric Survivorship Program to Promote the Physical and Psychological Well-being of Children After Cancer Treatment.
Cancer Nurs
January 2025
Author Affiliations: The School of Nursing, The Hong Kong Polytechnic University (Dr Chung); and The Nethersole School of Nursing, Faculty of Medicine, The Chinese University of Hong Kong (Dr Li), Hong Kong Special Administrative Region, China; School of Nursing, School of Health and Social Care, Edinburgh Napier University, United Kingdom (Dr Ching); Hong Kong Sanatorium and Hospital (Ms Chiu); Paediatric Haematology and Oncology (Ms Mak) and Department of Paediatric and Adolescent Medicine (Dr Chan), Hong Kong Children's Hospital, Hong Kong Special Administrative Region, China.
Background: Studies have provided insights into the major challenges and needs of young children who survive cancer. Many such challenges are related to a lack of support during the patient's transition back to normal life. Survivorship programs are designed to help survivors cope with potential late effects of treatment and to provide psychosocial support.
View Article and Find Full Text PDFSecond Primary Neoplasms in Pediatric Cancer Survivors With Single Institution Experience From Turkey.
J Pediatr Hematol Oncol
January 2025
Department of Pediatric Oncology, Faculty of Medicine, Ege University.
Background: This study aims to establish the characteristics of second primary neoplasms (SPNs) and the long-term follow-up status of a tertiary pediatric oncology center.
Methods: Records of 1799 patients followed up in the pediatric oncology division between January 1981 and December 2022 were evaluated retrospectively.
Results: Thirty-four (1.
A 5-year Single-center Experience on the Use of Emicizumab Prophylaxis in Children With Severe Hemophilia A With and Without Factor VIII Inhibitors.
J Pediatr Hematol Oncol
January 2025
Department of Paediatric Haematology, NHS Foundation Trust, Birmingham Children's Hospital, Birmingham, UK.
Objective: Emicizumab promotes efficacious hemostasis in persons with hemophilia A persons with hemophilia A with and without inhibitors. Primary analyses of real-world data and clinical trials have shown emicizumab efficacy and safety; however, long-term data are limited.
Methods: This retrospective study was conducted to assess real-world long-term outcomes of pediatric patients on emicizumab in our hemophilia center between the period of February 2018 and September 2023.
Toward Equitable Access to Psychosocial Screening in Canada: Validation of the French-Canadian Psychosocial Assessment Tool.
Pediatr Blood Cancer
March 2025
Azrieli Research Centre, Centre Hospitalier Universitaire Sainte-Justine, Montréal, Québec, Canada.
Objective: The Psychosocial Assessment Tool (PAT) is a brief caregiver report, family-centered, psychosocial risk screening tool widely used in pediatrics and available in many languages. Although French is an official language of Canada, a French-Canadian version of the PAT has not yet been validated, which impedes access to this tool for family psychosocial screening. This study aimed to translate, adapt as necessary, and validate the French-Canadian version of the PAT.
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