Background: Regenerative medicine using stem cell technology is an emerging field that is currently tested for inborn and acquired liver diseases.
Objective: This phase I/II prospective, open label, multicenter, randomized trial aimed primarily at evaluating the safety of Heterologous Human Adult Liver-derived Progenitor Cells (HepaStem) in pediatric patients with urea cycle disorders (UCDs) or Crigler-Najjar (CN) syndrome 6 months posttransplantation. The secondary objective included the assessment of safety up to 12 months postinfusion and of preliminary efficacy.
Methods: Fourteen patients with UCDs and 6 with CN syndrome were divided into 3 cohorts by body weight and intraportally infused with 3 doses of HepaStem. Clinical status, portal vein hemodynamics, morphology of the liver, de novo detection of circulating anti-human leukocyte antigen antibodies, and clinically significant adverse events (AEs) and serious adverse events to infusion were evaluated by using an intent-to-treat analysis.
Results: The overall safety of HepaStem was confirmed. For the entire study period, patient-month incidence rate was 1.76 for the AEs and 0.21 for the serious adverse events, of which 38% occurred within 1 month postinfusion. There was a trend of higher events in UCD as compared with CN patients. Segmental left portal vein thrombosis occurred in 1 patient and intraluminal local transient thrombus in a second patient. The other AEs were in line with expectations for catheter placement, cell infusion, concomitant medications, age, and underlying diseases.
Conclusions: This study led to European clinical trial authorization for a phase II study in a homogeneous patient cohort, with repeated infusions and intermediate doses.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1097/TP.0000000000002605 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Cross-Sectoral Comparisons of Process Quality Indicators of Health Care Across Residential Regions Using Restricted Mean Survival Time.
Med Care
November 2024
Institute of Clinical Biometrics, Center for Medical Data Science, Medical University of Vienna, Vienna, Austria.
Background: Practice guidelines recommend patient management based on scientific evidence. Quality indicators gauge adherence to such recommendations and assess health care quality. They are usually defined as adverse event rates, which may not fully capture guideline adherence over time.
View Article and Find Full Text PDFDiagnostic Value and Short-Term Prognosis Assessment of Copeptin in Non-ST-Elevation Acute Coronary Syndrome.
J Cardiovasc Transl Res
December 2024
Department of Clinical Laboratory, Henan Provincial People's Hospital, Zhengzhou, China.
This study explored the early diagnosis and prognostic value of copeptin in non-ST-segment elevation acute coronary syndrome (NSTE-ACS). 171 patients with chest pain or myocardial ischemia symptoms were enrolled. Patients with NSTE-ACS were further divided into the non-ST-elevation myocardial infarction (NSTEMI) and unstable angina (UA).
View Article and Find Full Text PDFLong-term use of proton pump inhibitors was associated with rapid progression to end stage kidney disease in a Korean nationwide study.
Sci Rep
December 2024
Department of Internal Medicine, Pusan National University School of Medicine, Busan, South Korea.
Proton pump inhibitors (PPIs) are among the most widely used drugs worldwide. However, their influence on the progression of end-stage kidney disease (ESKD) in established chronic kidney disease (CKD) cases is unclear. Using the Korean Health Insurance Review and Assessment database encoded by the Observational Medical Outcomes Partnership-Common Data Model (OMOP-CDM), patients with stage 3 or 4 CKD initiating PPIs or histamine-2 receptor antagonists (H2RAs) for over 90 days were enrolled from 2012 through 2021.
View Article and Find Full Text PDFNew-onset cardiovascular diseases post SARS-CoV-2 infection in an urban population in the Bronx.
Sci Rep
December 2024
Department of Radiology, Albert Einstein College of Medicine and Montefiore Medical Center, Bronx, New York, USA.
This study investigated the incidence of new-onset cardiovascular disorders up to 3.5 years post SARS-CoV-2 infection for 56,400 individuals with COVID-19 and 1,093,904 contemporary controls without COVID-19 in the Montefiore Health System (03/11/2020 to 07/01/2023). Outcomes were new incidence of major adverse cardiovascular event (MACE), arrhythmias, inflammatory heart disease, thrombosis, cerebrovascular disorders, ischemic heart disease and other cardiac disorders between 30 days and (up to) 3.
View Article and Find Full Text PDFPredictive nomogram of ultrasound indicators for the termination outcome of caesarean scar pregnancy.
Sci Rep
December 2024
Department of Ultrasound, The First Hospital of Hunan University of Chinese Medicine, Hunan University of Chinese Medicine, Changsha, 410021, Hunan, People's Republic of China.
To develop and validate a nomogram for predicting the risk of adverse events (intraoperative massive haemorrhage or retained products of conception) associated with the termination of Caesarean scar pregnancy (CSP). Data were retrospectively collected from patients diagnosed with CSP who underwent Dilation and Curettage (D&C) at two hospitals. This data was divided into internal and external cohorts for analysis.
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!