Hematopoietic stem cell transplantation (HSCT) is a potentially life-saving therapy for patients with malignant and nonmalignant disease states. Transplant has been associated with high treatment-related morbidity and mortality, therefore limiting its usefulness in patients with baseline liver dysfunction. In the event that a patient with hepatic insufficiency is selected for HSCT, dosage adjustments may be considered; however, no reliable endogenous biomarkers can serve as a guide for adjustments. There is no clear standard or guideline for how to approach these patients, and most adjustments are made empirically on the basis of expert opinion. This article offers practical advice and outlines our personal approaches to provide dosing recommendations for commonly-used preparative agents in the setting of hepatic impairment with the aim to optimize dosing for this patient population.
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http://dx.doi.org/10.1016/j.bbmt.2014.01.029 | DOI Listing |
Nat Commun
January 2025
CIRI, Centre International de Recherche en Infectiologie Univ Lyon, Inserm, U1111, Université Claude Bernard Lyon 1, CNRS, UMR5308, ENS de Lyon, F-69007, Lyon, France.
Prime Editing can rewrite genes in living cells by allowing point mutations, deletions, or insertion of small DNA sequences with high precision. However, its safe and efficient delivery into human stem cells remains a technical challenge. In this report, we engineer Nanoscribes, virus-like particles that encapsidate ribonucleoprotein complexes of the Prime Editing system and allow their delivery into recipient cells.
View Article and Find Full Text PDFEnferm Infecc Microbiol Clin (Engl Ed)
January 2025
Microbiology Service, Clinic University Hospital, INCLIVA Health Research Institute, Valencia, Spain; CIBER de Enfermedades Infecciosas, Instituto de Salud Carlos III, Madrid, Spain; Department of Microbiology, School of Medicine, University of Valencia, Valencia, Spain. Electronic address:
Introduction: The extent to which commercially available nucleic acid extraction platforms impact the magnitude of Cytomegalovirus (CMV) DNA loads measured in plasma specimens by 1st WHO standard-normalized real-time PCR assays is uncertain.
Methods: This retrospective study compares the performance of Abbott m2000sp, Qiagen QIAsymphony SP, and KingFisher Flex platforms using plasma samples from allogeneic hematopoietic stem cell transplant recipients and plasma spiked with the CMV AD169 strain. The Abbott RealTime CMV PCR assay was used for CMV DNA quantitation.
Cancer Lett
January 2025
Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Peking University, Beijing 100044, China; Collaborative Innovation Center of Hematology, Soochow University, Suzhou 215006, China. Electronic address:
This study updates the disease burden of multiple myeloma (MM) in thirty-three provincial administrative units in China from 1990 to 2021 and forecast the disease burden for 2050. Data from the 2021 Global Burden of Disease (GBD) database was used for analysis. In 2021, there were an estimated 17,250 new MM cases and 12,984 deaths in China.
View Article and Find Full Text PDFTransplant Cell Ther
January 2025
University of Kansas Medical Center, Kansas City, KS.
Background: Allogeneic hematopoietic cell transplantation (allo-HCT) is a potential cure for many hematological malignancies. Historically, older adults were not considered eligible for allo-HCT due to increased toxicity and mortality concerns. This systematic review and meta-analysis aim to explore the outcomes of allo-HCT in patients aged 70 years or older.
View Article and Find Full Text PDFCell Rep
January 2025
Department of Surgery, University of California, San Francisco, San Francisco, CA 94143, USA; Eli & Edythe Broad Center for Regeneration Medicine, University of California, San Francisco, San Francisco, CA 94143, USA; Department of Bioengineering & Therapeutic Sciences, University of California, San Francisco, San Francisco, CA 94158, USA. Electronic address:
The most severe form of α-thalassemia results from loss of all four copies of α-globin. Postnatally, patients face challenges similar to β-thalassemia, including severe anemia and erythrotoxicity due to the imbalance of β-globin and α-globin chains. Despite progress in genome editing treatments for β-thalassemia, there is no analogous curative option for α-thalassemia.
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