A5012936889- Hematopoietic Stem Cell Transplantation
- Cytomegalovirus and herpesvirus research
- Lysosomal Storage Disorders Research
- Polyomavirus and related diseases
- Acute Lymphoblastic Leukemia research
- Virus-based gene therapy research
- Immune Cell Function and Interaction
- Human-Automation Interaction and Safety
- T-cell and B-cell Immunology
- Mesenchymal stem cell research
- Immunodeficiency and Autoimmune Disorders
- Acute Myeloid Leukemia Research
- Metabolism and Genetic Disorders
- Transplantation: Methods and Outcomes
- Neutrophil, Myeloperoxidase and Oxidative Mechanisms
- Neurogenetic and Muscular Disorders Research
- Blood disorders and treatments
- Childhood Cancer Survivors' Quality of Life
- Neonatal Respiratory Health Research
- Peroxisome Proliferator-Activated Receptors
- Autoimmune and Inflammatory Disorders Research
- Blood groups and transfusion
- Parvovirus B19 Infection Studies
- Immunotherapy and Immune Responses
- Biomedical Research and Pathophysiology
Duke Children's Hospital & Health Center
2008-2025
Duke Medical Center
2014-2024
Duke University
2013-2024
Bluebird Bio (United States)
2024
Dana-Farber/Boston Children's Cancer and Blood Disorders Center
2024
Harvard University
2024
Boston Children's Hospital
2024
University of Minnesota Medical Center
2024
Massachusetts General Hospital
2024
Marcus (United States)
2020-2022
Preliminary studies using directed-donor ex vivo expanded human mesenchymal stem cells (hMSCs) have shown promise in the treatment of acute graft-versus-host disease (aGVHD). However, their production is cumbersome and standardization difficult. We describe first experience a premanufactured, universal donor, formulation hMSCs (Prochymal) children (n = 12; 10 boys; 9 Caucasian; age range: 0.4-15 years) with treatment-resistant grade III IV aGVHD who received therapy on compassionate use...
Gene therapy with elivaldogene autotemcel (eli-cel) consisting of autologous CD34+ cells transduced lentiviral vector containing
Invasive pulmonary aspergillosis is a life-threatening mycosis that only affects patients with immunosuppression, chemotherapy-induced neutropenia, transplantation, or congenital immunodeficiency. We studied the clinical, genetic, histological, and immunological features of 2 unrelated without known immunodeficiency who developed extrapulmonary invasive at ages 8 18. One patient died age 12 progressive intra-abdominal aspergillosis. The other had presented candidiasis 9, central nervous...
Highlights•There is an unmet need for effective therapy adenovirus infection in hematopoietic cell transplant recipients•Brincidofovir has potent antiviral activity against vitro•Rapid decreases viremia were observed after initiation of brincidofovir twice weekly•Early intervention with may provide clinical benefit viremiaAbstractAdenovirus immunocompromised patients contributes to significant morbidity and mortality, especially allogeneic transplantation (HCT). Brincidofovir (BCV, CMX001)...
Steroid-refractory acute graft-versus-host disease (SR-aGVHD) following hematopoietic cell transplantation (HSCT) is associated with poor clinical outcomes. Currently, there are no safe and effective therapies approved for use in the pediatric population under age of 12 years. Accordingly, an urgent need new treatments that safe, well tolerated, managing this debilitating potentially fatal complication HSCT. In early phase trials, mesenchymal stromal cells (MSCs) have demonstrated efficacy...
Cerebral adrenoleukodystrophy (CALD) is a rapidly progressing, often fatal neurodegenerative disease caused by mutations in the ABCD1 gene, resulting deficiency of ALD protein. Clinical benefit has been reported following allogeneic hematopoietic stem cell transplantation (HSCT). We conducted large multicenter retrospective chart review to characterize natural history CALD, describe outcomes after HSCT, and identify predictors treatment outcomes. Major functional disabilities (MFDs) were...
Allogeneic stem cell transplantation (SCT) is considered effective in preventing disease progression patients with Hurler syndrome (HS). Unrelated umbilical cord blood (UCB) grafts are suggested as an alternative to bone marrow (BM) or peripheral cells (PBSC). We studied 93 HS receiving UCB graft analyze risk factors for outcomes. The median time from diagnosis transplant was 4.6 months, follow-up 29 and number of nucleated CB infused 7.6 x 10(7)/kg. Most the received 1 2 HLA disparate...
Background Banked unrelated donor umbilical cord blood ( CB ) has improved access to hematopoietic stem cell transplantation for patients without a suitably matched donor. In resource‐limited environment, ensuring that the public inventory is enriched with high‐quality units CBUs addressing needs of diverse group priority. Identification characteristics correlating higher CBU quality could guide operational strategies increase yield banked . Study Design and Methods Characteristics 5267...
Transplantation-related mortality (TRM) is high after HLA-mismatched umbilical cord blood (UCB) transplantation (UCBT). In utero, exposure to noninherited maternal antigen (NIMA) recognized by the fetus, which induces T regulator cells that haplotype. It plausible UCBTs in recipients are matched donor NIMAs may alleviate some of excess associated with this treatment. To explore concept, we used marginal matched-pair Cox regression analysis compare outcomes 48 NIMA-matched (ie, NIMA UCB unit...
Allogeneic hematopoietic stem cell transplantation (HSCT) can retard the progression of early infantile Krabbe disease (EIKD). Superior outcomes are achieved if HSCT is performed before onset symptoms; however, little information available about long-term in surviving patients. We now describe functional presymptomatic infants who underwent for EIKD at ≤ 2 months age. Records 19 patients age from 1996 to 2010 were reviewed. Long-term compared between those transplanted < 30 days and ≥ life....
Clinical outcomes in children with steroid-refractory acute graft-versus-host disease (SR-aGVHD) are generally poor, a high mortality rate and limited therapeutic options. Here we report our updated investigational experience mesenchymal stromal cell (MSC) therapy remestemcel-L multicenter expanded access protocol (ClinicalTrials.gov identifier NCT00759018) 241 aGVHD who failed to respond steroids or without other secondary tertiary immunosuppressive therapies. A total of grade B-D SR-aGVHD...
Adrenoleukodystrophy (ALD) is an X-linked disorder caused by a defect in the metabolism of long chain fatty acids leading to demyelination, neurodegeneration, and death. The disease typically presents young boys adolescent boys. Allogeneic bone marrow transplantation has been used halt progression disease. However, many patients lack suitable HLA- matched related donors must rely on unmatched for source stem cells. purpose this study was evaluate outcomes unrelated donor umbilical cord blood...
Autoimmune cytopenias are a recognized complication of hematopoietic stem cell transplant (HSCT), and considered to be feature chronic graft-versus-host disease (cGVHD). We report on cohort very young infants (≤3 months age) receiving HSCT from unrelated donor umbilical cord blood for genetic disorders who developed posttransplant autoimmune at an increased rate compared older aged controls. These received conditioning regimen consisting busulfan, cyclophosphamide, antithymocyte globulin...