Publications by authors named "F Goldman"
Article Synopsis
- Patients with telomere biology disorders (TBD) commonly experience severe liver diseases, and while liver transplantation (LT) is debated for these cases, this study aimed to evaluate patient outcomes and management strategies related to TBD-associated liver disease.
- A total of 83 patients were analyzed, with 40 experiencing advanced liver disease; among them, 20 underwent LT, and notable factors like pulmonary issues and elevated bilirubin levels were linked to the advanced disease group.
- Results showed that LT recipients had a 73% one-year survival rate and improved respiratory conditions in over half of the patients post-transplant, suggesting that a TBD diagnosis should not prevent consideration for LT.
Wiskott-Aldrich syndrome (WAS) is a multifaceted monogenic disorder with a broad disease spectrum and variable disease severity and a variety of treatment options including allogeneic hematopoietic stem cell transplantation (HSCT) and gene therapy (GT). No reliable biomarker exists to predict disease course and outcome for individual patients. A total of 577 patients with a WAS variant from 26 countries and a median follow-up of 8.
View Article and Find Full Text PDFWhile the survival of children with cancer has improved over time, infection remains a major morbidity and mortality risk. We conducted a systematic literature review to determine the unmet needs in diagnosing infection in immunocompromised children with cancer. The comprehensive search strategy followed the guidelines established by the Cochrane Handbook for Systematic Reviews of Interventions and the Preferred Reporting Items for Systematic reviews and Meta-Analyses (PRISMA) 2020 statement, and spanned multiple bibliographic databases and other public sources from January 1, 2012 to June 23, 2022.
View Article and Find Full Text PDFBackground: The Primary Immune Deficiency Treatment Consortium (PIDTC) enrolled children in the United States and Canada onto a retrospective multicenter natural history study of hematopoietic cell transplantation (HCT).
Objective: We investigated outcomes of HCT for severe combined immunodeficiency (SCID).
Methods: We evaluated the chronic and late effects (CLE) after HCT for SCID in 399 patients transplanted from 1982 to 2012 at 32 PIDTC centers.
Aging is the consequence of intra- and extracellular events that promote cellular senescence. Dyskeratosis congenita (DC) is an example of a premature aging disorder caused by underlying telomere/telomerase-related mutations. Cells from these patients offer an opportunity to study telomere-related aging and senescence.
View Article and Find Full Text PDF