A5041199901- CAR-T cell therapy research
- Cytomegalovirus and herpesvirus research
- Polyomavirus and related diseases
- Viral-associated cancers and disorders
- Acute Lymphoblastic Leukemia research
- Virus-based gene therapy research
- Lymphoma Diagnosis and Treatment
- Platelet Disorders and Treatments
- Hematopoietic Stem Cell Transplantation
- Acute Myeloid Leukemia Research
- Neuroblastoma Research and Treatments
- Myeloproliferative Neoplasms: Diagnosis and Treatment
- Childhood Cancer Survivors' Quality of Life
- Cancer therapeutics and mechanisms
- Immunodeficiency and Autoimmune Disorders
- Integrated Circuits and Semiconductor Failure Analysis
- Immune Cell Function and Interaction
- Blood disorders and treatments
- Full-Duplex Wireless Communications
- Hume's philosophy and hair distribution
- Complement system in diseases
- Renal Transplantation Outcomes and Treatments
- Parkinson's Disease Mechanisms and Treatments
- Literature, Culture, and Criticism
- Pharmaceutical Economics and Policy
Cincinnati Children's Hospital Medical Center
2017-2025
University of Cincinnati Medical Center
2019-2025
University of Cincinnati
2004-2024
Immune Deficiency Foundation
2020
University of Virginia
2008-2013
University of Virginia Health System
2009-2011
Parkinson's disease, the most common adult neurodegenerative movement disorder, demonstrates a brain-wide pathology that begins pre-clinically with alpha-synuclein aggregates ("Lewy neurites") in processes of gut enteric and vagal motor neurons. Rostral progression into substantia nigra death dopamine neurons produces impairment phenotype yields clinical diagnosis. The vast majority disease occurs sporadically, current models sporadic (sPD) can utilize directly infused or systemic...
BK polyomavirus (BKPyV) infection is a major complication of hematopoietic stem cell transplant (HSCT) and solid organ (SOT). Treatment options are limited, poorly effective, have significant toxicities. Cellular therapy using T cells directed against BKPyV an emerging therapy, we report efficacy in controlling BKPyV-associated disease highly immunocompromised patients. Virus-specific (VSTs) were manufactured either blood from the patient's donor (donor-derived VSTs) or unrelated donors...
Abstract Infection with adenoviruses is a common and significant complication in pediatric patients after allogeneic hematopoietic stem cell transplantation. Treatment options traditional antivirals are limited by poor efficacy toxicities. T-cell reconstitution critical for the management of adenoviral infections, but it generally takes place months Ex vivo–generated virus-specific T cells (VSTs) an alternative approach viral control can be rapidly generated from either donor or healthy...
Abstract Viral infections are a major source of morbidity and mortality in the context immune deficiency immunosuppression following allogeneic hematopoietic cell (allo-HCT) solid organ transplantation (SOT). The pharmacological treatment viral is challenging often complicated by limited efficacy, development resistance, intolerable side effects. A promising strategy to rapidly restore antiviral immunity adoptive transfer virus-specific T cells (VST). This therapy involves isolation ex vivo...
Autologous CD19-directed chimeric antigen receptor T lymphocyte (CAR-T) therapy is an approved and effective treatment for the management of patients with refractory multiply relapsed B cell precursor acute lymphoblastic leukemia (B-ALL). Experience using this in pediatric extramedullary (EM) disease limited, part because these have frequently been excluded from clinical trials owing to concerns increased risk immune effector cell-associated neurotoxicity syndrome (ICANS). We infused 7 or...
Infections with double-stranded DNA viruses are a common complication after hematopoietic stem cell transplantation (HSCT) and cause significant morbidity mortality in the post-transplantation period. Both donor-derived (DD) third-party (TP) virus-specific T cells (VSTs) have shown efficacy safety viral management following HSCT children young adults. Owing to greater degree of HLA matching between recipient donor, DD VSTs potentially persist longer circulation compared TP VSTs, because they...
ABSTRACT Epstein–Barr virus (EBV)‐positive smooth muscle tumors (SMTs) are rare seen in immunocompromised patients. There is no clear standard of care for the management and treatment EBV‐SMTs. Patients often treated with chemotherapy, surgery, and/or radiation. Additional options include antiretroviral treatment, reduction immunosuppression, EBV‐directed virus‐specific T cells (VSTs). This report describes regimens outcomes eight patients EBV‐associated SMTs. Although consensus EBV‐SMTs has...
Infections with double-stranded DNA viruses are a significant cause of morbidity and mortality in pediatric patients following allogeneic hematopoietic stem cell transplantation (HSCT). Virus-specific T-cell therapies (VSTs) have been shown to be an effective treatment for infections adenovirus, BK virus, cytomegalovirus (CMV), Epstein-Barr virus (EBV). To date, prophylactic regimens prevent or mitigate these using conventional antiviral medications provide suboptimal response rates. Here we...
Progressive multifocal leukoencephalopathy (PML) is a progressive and generally fatal demyelinating neurologic disease that occurs in profoundly immunocompromised patients due to infection with the human polyomavirus JC virus (JCPyV). Treatment options are limited largely focused on restoring T cell immunity, outcomes historically poor. Control of JCPyV setting an patient by adoptive transfer third-party specific cells (VSTs) has been described small number cases. To investigate treatment...
Summary Burkitt lymphoma arising in paediatric post‐solid‐organ transplantation‐Burkitt (PSOT‐BL) is a clinically aggressive malignancy and rare form of post‐transplant lymphoproliferative disorder (PTLD). We evaluated 35 patients diagnosed with PSOT‐BL at 14 medical centres the United States. Median age organ transplantation was 2.0 years (range: 0.1–14) diagnosis 8.0 1–17). All but one patient had late onset (≥2 post‐transplant), median interval from transplant to 4.0 0.4–12). Heart ( n =...
Summary Venetoclax (VEN) combined with hypomethylating agents (HMA) decitabine or azacitidine is used for adult acute myeloid leukaemia (AML), but its application in paediatric, adolescent and young (AYA) AML lacks prospective studies. We performed a retrospective chart review of paediatric AYA patients treated HMA + VEN at Cincinnati Children's Hospital Medical Centre. Twenty‐seven received 30 treatment courses relapsed/refractory (R/R, n = 21) newly diagnosed ( 9) due to ineligibility...
Bridging therapy (BT) given during the period between T-cell collection and initiation of lymphodepleting chemotherapy is indicated for most children with B-cell acute lymphoblastic leukemia (B-ALL) undergoing treatment tisagenlecleucel (tisa-cel), a CD19-directed chimeric antigen receptor (CAR-T) therapy. Both conventional agents directed antibody-based therapies such as antibody-drug conjugates bispecific engagers have been used systemic forms BT. The purpose this retrospective study was...
Vulvovaginal graft-versus-host disease (GVHD) is an underdiagnosed and poorly recognized complication of hematopoietic stem cell transplantation (HSCT). Previous studies have reported findings restricted to predominantly adult populations. We report a case series pediatric young vulvovaginal GVHD, which was identified in 19 patients (median age, 11.8 years; range, 2.4 21.9 years) out total 302 female who underwent over 8-year period at HSCT center. The majority had concomitant nongenital...
Abstract Background Pediatric Epstein‐Barr virus–negative monomorphic post solid organ transplant lymphoproliferative disorder [EBV(−)M‐PTLD] comprises approximately 10% of M‐PTLD. No large multi‐institutional pediatric‐specific reports on treatment and outcome are available. Methods A retrospective review recipients diagnosed with EBV(−)M‐PTLD aged ≤21 years between 2001 2020 in 12 centers the United States Kingdom was performed, including demographics, staging, treatment, outcomes data....
Abstract Children with trisomy 18 are surviving longer and undergoing more aggressive life‐sustaining therapy. This report describes two patients hepatoblastoma (HB) successfully resected in the setting of significant pulmonary hypertension. Forty‐four previously published cases association between HB reviewed. With careful multidisciplinary preoperative planning, successful resection children who have hypertension is feasible. Because increasing prevalence, need for timely liver tumor will...
Chimeric antigen receptor T cells (CAR-T) are an effective and potentially durable treatment for refractory multiply relapsed B-cell acute lymphoblastic leukemia. Neurotoxicity is frequent after CAR-T cell therapy. Mechanisms driving neurotoxicity incompletely understood, symptoms can range from transient mild to severe life-threatening. Providers have exercised caution in providing patients with neurological comorbidities or extramedullary disease. Here, we report three prior significant...
CD19-directed chimeric antigen receptor T lymphocytes (CAR-T) have led to durable remissions in children with refractory and/or multiply relapsed B-lymphoblastic leukemia. For those who relapse or lose B-cell aplasia post CAR-T, the role of CAR-T reinfusion is unclear. We report two cases remission tisagenlecleucel despite failure achieve maintain aplasia, and compare these six additional received multiple infusions at our institution. Our experience suggests that safe may be a definitive...