A5011950454- Hematopoietic Stem Cell Transplantation
- Hemoglobinopathies and Related Disorders
- Acute Lymphoblastic Leukemia research
- CAR-T cell therapy research
- Acute Myeloid Leukemia Research
- Immunodeficiency and Autoimmune Disorders
- Lymphoma Diagnosis and Treatment
- Prenatal Screening and Diagnostics
- Cytomegalovirus and herpesvirus research
- Blood disorders and treatments
- Childhood Cancer Survivors' Quality of Life
- Neutropenia and Cancer Infections
- Parvovirus B19 Infection Studies
- Erythrocyte Function and Pathophysiology
- Glioma Diagnosis and Treatment
- Virus-based gene therapy research
- Cancer-related cognitive impairment studies
- Polyomavirus and related diseases
- Immune Cell Function and Interaction
- Renal Transplantation Outcomes and Treatments
- Adolescent and Pediatric Healthcare
- Chemotherapy-induced cardiotoxicity and mitigation
- Chronic Myeloid Leukemia Treatments
- Viral-associated cancers and disorders
- Chronic Lymphocytic Leukemia Research
Texas Children's Hospital
2017-2025
Baylor College of Medicine
2017-2025
Children's Cancer Center
2017-2025
Stanford University
2023-2024
Stanford Medicine
2024
Palo Alto University
2023
Lucile Packard Children's Hospital
2023
In-Q-Tel
2022
Houston Methodist
2018-2021
Methodist Hospital
2018-2021
ABSTRACT Background Immune effector cell (IEC) therapies, including chimeric antigen receptor (CAR)‐modified T‐cell therapy, have shown efficacy in pediatric B‐cell acute lymphoblastic leukemia (B‐ALL) and are being investigated for other malignancies. A common toxicity associated with IEC therapy is cytokine release syndrome (CRS), which can lead to cardiovascular decompensation due systemic inflammation. Data limited regarding adverse effects children. This study aims describe the effect...
Abstract Adoptive transfer of virus-specific T cells (VSTs) has been shown to be safe and effective in stem cell transplant recipients. However, the lack virus-experienced donor cord blood (CB) prevented development ex vivo expanded donor-derived VSTs for recipients this source. Here we evaluated feasibility safety expansion CB from 20% fraction unit pediatric patients receiving a single (CBT). In 2 clinical trials conducted at separate sites, manufactured CB-derived multivirus-specific...
Abstract The MECOM gene encodes multiple protein isoforms that are essential for hematopoietic stem cell self‐renewal and maintenance. Germline variants have been associated with congenital thrombocytopenia, radioulnar synostosis bone marrow failure; however, the phenotypic spectrum of ‐associated syndromes continues to expand novel pathogenic continue be identified. We describe eight unrelated patients who add previously known phenotypes genetic defects syndromes. As each subject presented...
Chronic active Epstein-Barr virus (EBV) disease (CAEBV) is characterized by high levels of EBV predominantly in T and/or natural killer cells with lymphoproliferation, organ failure due to infiltration tissues virus-infected cells, hemophagocytic lymphohistiocytosis, lymphoma. The more common Asia than the United States and Europe. Although allogeneic hematopoietic stem cell transplantation (HSCT) considered only curative therapy for CAEBV, its efficacy best treatment modality reduce...
Follow-up is integral for hematopoietic cell transplantation (HCT) care to ensure surveillance and intervention complications. We characterized the incidence of predictors being lost follow-up. Two-year survivors first allogeneic HCT (10,367 adults 3865 children) or autologous (7291 467 malignant/nonmalignant disorders between 2002 2013 reported Center International Blood Marrow Transplant Research were selected. The cumulative follow-up (defined as having missed 2 consecutive reporting...
There is no consensus on the best donor for children with nonmalignant disorders and immune deficiencies in absence of a matched related (MRD). We evaluated 2-year overall survival (OS) after umbilical cord blood transplantation (UCBT) patients from 2009 to 2020 enrolled prospective clinical trial using either 5/6 or 6/6 UCB as cell source. Patients receive fully ablative busulfan, cyclophosphamide, fludarabine without serotherapy. Fifty-five were enrolled, median age 5 months (range, 1-111...
Abstract Relapsed ALK ‐positive ALCL often is responsive to CRZ monotherapy. The subsequent role of allogeneic HCT after achieving second remission poorly understood. We report 6 children who underwent for relapsed . Age at transplant ranged from 10.7 22.6 years. Follow‐up 0.9 4.5 All patients engrafted. Three 4 that received a reduced‐toxicity conditioning regimen containing fludarabine, alemtuzumab, and low‐dose irradiation showed progressive mixed chimerism. Five remain in remission. One...
Background: Computer-based cognitive rehabilitation programs may help adolescent and young adult (AYA) patients with cancer-related impairment. This pilot study investigated the feasibility of as a preventive intervention for AYA receiving chemotherapy. Explorative objectives included correlation performance serum brain-derived neurotrophic factor (BDNF). Methods: prospective English-speaking 12-25 years age fist diagnosis cancer requiring Participants enrolled in arm participated...
There are sparse data on the long-term and late effects of hematopoietic cell transplantation (HCT) for sickle disease (SCD).
Background There are sparse data on long-term and late effects of hematopoietic cell transplantation (HCT) for sickle disease (SCD) Objectives To establish an international registry outcomes post-HCT SCD demonstrate the feasibility recruitment at a single site in US. Methods The STELLAR is designed to enroll patients ≥ 1-year post-HCT, their siblings without SCD, non-transplanted controls collect participant self-report health status practices using BMT survivor study surveys, HRQOL PROMIS...
Abstract The Blood and Marrow Transplant Clinical Trials Network (BMT CTN) 1507 leadership the data safety monitoring board (DSMB) established incremental entry criteria for children aged 5 to 14.99 years with sickle cell disease (SCD) enrolling in a phase 2 trial of HLA-haploidentical hematopoietic stem transplantation. First, enrollment was limited overt stroke first 10 participants (stage 4). Subsequently, DSMB reviewed interim results expanded eligibility include silent cerebral infarcts...