Few reports have examined whether prophylactic allogeneic hematopoietic cell transplantation (HCT) for X-linked lymphoproliferative syndrome type 1 (XLP1) improves the prognosis. We compared the prognosis of symptomatic probands and affected siblings in the same family. Twenty-two cases (10 probands and 12 affected siblings) in Japan, the United Kingdom, and the United States were analyzed. The overall survival (OS) rate at 5 years after diagnosis was 70.0% in probands and 91.7% in affected siblings (p = 0.0789). The prognosis of patients who developed symptoms of XLP1 before HCT and those who did not was also compared. The 5-year probability of OS from the time of diagnosis in asymptomatic patients (100%) was significantly better than that in symptomatic patients (66.7%). These results suggested that early HCT as soon as the diagnosis is made improves the prognosis in asymptomatic XLP1 patients.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1016/j.clim.2022.108993 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Delivery of Prime editing in human stem cells using pseudoviral NanoScribes particles.
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 PDFImpact of automated nucleic acid extraction platforms on plasma Cytomegalovirus DNA loads quantitated by real-time PCR normalized to the 1st WHO international standard.
Enferm 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.
The Burden of Multiple Myeloma in China: Trends from 1990 to 2021 and Forecasts for 2050.
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 PDFDonor Type Does Not Impact Late Graft Failure Following Reduced Intensity Allogeneic Hematopoietic Cell Transplantation with Post-Transplant Cyclophosphamide Based Graft-Versus-Host Disease Prophylaxis.
Transplant Cell Ther
January 2025
Dana-Farber Cancer Institute, Division of Transplantation and Cellular Therapy, Boston, MA. Electronic address:
Background: Post-transplant cyclophosphamide (PTCy) is a commonly used graft-vs-host disease (GVHD) prophylaxis, particularly in the setting of haploidentical (haplo) hematopoietic cell transplantation (HCT). The rate of graft failure has been reported to be as high as 12-20% in haplo-HCT recipients using PTCy. The objective of this study was to determine if donor type influenced the risk of late graft failure following RIC HCT using PTCy-based GVHD prophylaxis.
View Article and Find Full Text PDFOutcomes of Allogeneic Hematopoietic Stem Cell Transplantation in Patients Aged 70 Years and Older: A Systematic Review and Meta-analysis.
Transplant 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 PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!