A5043877705- Hematopoietic Stem Cell Transplantation
- Acute Myeloid Leukemia Research
- Acute Lymphoblastic Leukemia research
- Childhood Cancer Survivors' Quality of Life
- Immune Cell Function and Interaction
- CAR-T cell therapy research
- Mesenchymal stem cell research
- Renal Transplantation Outcomes and Treatments
- T-cell and B-cell Immunology
- Neutropenia and Cancer Infections
- Hemoglobinopathies and Related Disorders
- Pancreatic and Hepatic Oncology Research
- Multiple Myeloma Research and Treatments
- Blood disorders and treatments
- Immunotherapy and Immune Responses
- Chronic Lymphocytic Leukemia Research
- Neonatal Respiratory Health Research
- Polyomavirus and related diseases
- Immunodeficiency and Autoimmune Disorders
- Virus-based gene therapy research
- Interstitial Lung Diseases and Idiopathic Pulmonary Fibrosis
- Chronic Myeloid Leukemia Treatments
- Clinical Nutrition and Gastroenterology
- Neuroblastoma Research and Treatments
- Pancreatic function and diabetes
Icahn School of Medicine at Mount Sinai
2016-2025
Mount Sinai Hospital
2018-2025
Tisch Cancer Institute
2017-2025
University of Michigan
2012-2024
University of Oklahoma Health Sciences Center
2024
Children's Healthcare of Atlanta
2024
National Institutes of Health
2024
University of Central Oklahoma
2024
Cancer Institute (WIA)
2019-2023
Huntsman Cancer Institute
2023
Pompe disease is a progressive metabolic neuromuscular disorder resulting from deficiency of lysosomal acid alpha-glucosidase (GAA). Infantile-onset characterized by cardiomyopathy, respiratory and skeletal muscle weakness, early death. The safety efficacy recombinant human (rh) GAA were evaluated in 18 patients with rapidly progressing infantile-onset disease.Patients diagnosed at 6 months age younger exhibited severe cardiomyopathy. Patients received IV infusions rhGAA 20 mg/kg (n = 9) or...
Highlights•Staging GVHD symptoms varies among BMT centers because of different practices.•Different staging practices make comparing results between studies difficult.•Standardized detailed guidance was developed by a consortium.•Increased uniformity may improve clinical trial reproducibility.AbstractAcute graft-versus-host disease (GVHD) remains leading cause morbidity and nonrelapse mortality after allogeneic hematopoietic cell transplantation. The greatly transplant is frequently not...
Next-generation sequencing of the hypervariable V3 region 16s rRNA gene isolated from serial stool specimens collected 31 patients receiving allogeneic stem cell transplantation (SCT) was performed to elucidate variations in composition intestinal microbiome course SCT. Metagenomic analysis complemented by strain-specific enterococcal PCR and indirect assessment bacterial load liquid chromatography-tandem mass spectrometry urinary indoxyl sulfate. At time admission, showed a predominance...
<h3>Importance</h3> Cancer is caused by a diverse array of somatic and germline genomic aberrations. Advances in sequencing technologies have improved the ability to detect these molecular aberrations with greater sensitivity. However, integrating them into clinical management an individualized manner has proven challenging. <h3>Objective</h3> To evaluate use integrative genetic counseling assessment treatment children young adults cancer. <h3>Design, Setting, Participants</h3> Single-site,...
No plasma biomarkers are associated with the response of acute graft-versus-host disease (GVHD) to therapy after allogeneic hematopoietic stem-cell transplantation.We compared 12 in obtained a median 16 days initiation from 10 patients complete by day 28 and progressive GVHD during therapy. The lead biomarker, suppression tumorigenicity 2 (ST2), was measured at beginning treatment for 381 first month transplantation three independent sets totaling 673 determine association this biomarker...
Several international recommendations address the assessment of graft-versus-host disease (GvHD) after hematopoietic cell transplantation (HCT). This position statement by GvHD experts from European Society for Blood and Marrow Transplantation (EBMT), National Institutes Health (NIH) Center International Transplant Research (CIBMTR) reviews existing guidelines both acute chronic GvHD, addresses potential confusions that arise in daily practice proposes consensus definitions many key terms....
Highlights•High-risk graft-versus-host disease patients are less likely to respond steroids than standard-risk patients.•Patients with high-risk have higher risks of mortality and transplant-related patients.•Refined risk score better predicts outcomes published scores.•Patients candidates for novel treatment approaches.•Patients studies investigating toxic therapy.AbstractTo develop a acute (GVHD) score, we examined the GVHD clinical stage grade 1723 at onset systemic steroids. Using...
Abstract Purpose: Tisagenlecleucel is an anti-CD19 chimeric antigen receptor (CAR19) T-cell therapy approved for the treatment of children and young adults with relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL). Patients Methods: We evaluated cellular kinetics tisagenlecleucel, effect patient factors, humoral immunogenicity, manufacturing attributes on its kinetics, exposure-response analysis efficacy, safety pharmacodynamic endpoints in 79 patients across two studies...
BACKGROUND. No laboratory test can predict the risk of nonrelapse mortality (NRM) or severe graft-versus-host disease (GVHD) after hematopoietic cellular transplantation (HCT) prior to onset GVHD symptoms.
Severe (grade 3-4) acute graft-versus-host disease (AGVHD) is a major cause of death after unrelated-donor (URD) hematopoietic cell transplant (HCT), resulting in particularly high mortality HLA-mismatched transplantation. There are no approved agents for AGVHD prevention, underscoring the critical unmet need novel therapeutics. ABA2 was phase II trial to rigorously assess safety, efficacy, and immunologic effects adding T-cell costimulation blockade with abatacept calcineurin inhibitor...
High allelic ratio (HAR)
PURPOSE: Patients with advanced myeloid malignancies who experience relapse after allogeneic bone marrow transplantation (BMT) have a poor prognosis. Long-term survival chemotherapy alone, second myeloablative transplant, or donor leukocyte infusions (DLIs) alone is unusual. DLIs may minimal effectiveness in disease because adequate cellular responses are not able to develop the presence of bulky, fast-growing disease. A strategy was used debulk before administration granulocyte...
Graft-versus-host disease (GVHD), the major complication of allogeneic bone marrow transplantation, affects skin, liver, and gastrointestinal tract. There are no plasma biomarkers specific for any acute GVHD target organ. We used a large-scale quantitative proteomic discovery procedure to identify biomarker candidates skin validated lead candidate, elafin, with enzyme-linked immunosorbent assay in samples from 492 patients. Elafin was overexpressed biopsies. Plasma concentrations elafin were...