A5047995450- Hematopoietic Stem Cell Transplantation
- Acute Lymphoblastic Leukemia research
- Acute Myeloid Leukemia Research
- Immune Cell Function and Interaction
- Immunodeficiency and Autoimmune Disorders
- Viral-associated cancers and disorders
- Chronic Lymphocytic Leukemia Research
- Prenatal Screening and Diagnostics
- Hormonal and reproductive studies
- Polyomavirus and related diseases
- CNS Lymphoma Diagnosis and Treatment
- Lymphoma Diagnosis and Treatment
- Childhood Cancer Survivors' Quality of Life
- Neonatal Respiratory Health Research
- Cystic Fibrosis Research Advances
- Adolescent and Pediatric Healthcare
- Pharmacological Effects and Toxicity Studies
- HIV Research and Treatment
- Blood disorders and treatments
- Chronic Myeloid Leukemia Treatments
- Neutropenia and Cancer Infections
- Genetics and Neurodevelopmental Disorders
- Cytomegalovirus and herpesvirus research
- Pharmaceutical studies and practices
- Ion Transport and Channel Regulation
Atara Biotherapeutics (United States)
2022-2024
Pharmacyclics (United States)
2022-2023
AbbVie (United States)
2022-2023
UCSF Benioff Children's Hospital
2011-2021
University of California, San Francisco
2012-2021
Memorial Sloan Kettering Cancer Center
2011
Karolinska University Hospital
2009
Karolinska Institutet
2009
Hvidovre Hospital
2009
Vanderbilt University
2004
Abstract Chronic graft-versus-host disease (cGVHD) and late acute (L-aGVHD) are understudied complications of allogeneic hematopoietic stem cell transplantation in children. The National Institutes Health Consensus Criteria (NIH-CC) were designed to improve the diagnostic accuracy cGVHD better classify (GVHD) syndromes but have not been validated patients <18 years age. objectives this prospective multi-institution study determine: (1) whether NIH-CC could be used diagnose pediatric...
To present primary and final analyses from the randomized, double-blind, placebo-controlled, phase III iNTEGRATE study, which evaluated safety efficacy of ibrutinib with prednisone in previously untreated patients chronic graft-versus-host disease (cGVHD).Patients (age ≥ 12 years) newly diagnosed moderate or severe cGVHD, requiring systemic corticosteroid therapy, no prior treatment for cGVHD were randomly assigned 1:1 to receive 420 mg once daily plus prednisone, starting at 1 mg/kg placebo...
Patients with Epstein-Barr virus (EBV)-positive posttransplant lymphoproliferative disease (EBV+ PTLD) in whom initial treatment fails have few options and historically low median overall survival (OS) of 0.7 months after allogeneic hematopoietic cell transplant (HCT) 4.1 solid organ (SOT). Tabelecleucel is an off-the-shelf, EBV-specific cytotoxic T-lymphocyte immunotherapy for EBV+ PTLD. Previous single-center experience showed responses patients PTLD HCT or SOT. We now report outcomes from...
In mice, the transcription factor, PLZF, controls development of effector functions in invariant NKT cells and a subset cell-like, γδ T cells. Here, we show that human lymphocytes, addition to cells, PLZF was also expressed large percentage CD8+ CD4+ Furthermore, found be all NK Importantly, donor lacking functional these various lymphocyte populations were altered. Therefore, contrast appears control and/or function wide variety lymphocytes represent more than 10% total PBMCs....
A prospective multicenter study was conducted to characterize the pharmacokinetics (PK) and pharmacodynamics (PD) of fludarabine plasma (f-ara-a) intracellular triphosphate (f-ara-ATP) in children undergoing hematopoietic cell transplantation (HCT) receiving with conditioning. Plasma peripheral blood mononuclear cells (PBMCs) were collected over course therapy for quantitation f-ara-a f-ara-ATP. Nonlinear mixed-effects modeling used develop PK model, including identification covariates...
The presence of increasing host chimerism or persistent mixed (MC) after hematopoietic stem cell transplantation for leukemia in children is a predictor relapse. To reduce the risk relapse, we prospectively studied post-transplantation chimerism-based immunotherapy (IT) using fast withdrawal immunosuppression (FWI) and donor lymphocyte infusions (DLI) with early MC. Forty-three hematologic malignancies at 2 institutions were enrolled this study from 2009 until 2012 followed mean 42 (SD, 10)...
Chronic graft-versus-host disease (cGVHD) is a potentially life-threatening complication of allogeneic hematopoietic stem cell transplantation. Clinical data surrounding cGVHD therapies in younger children are limited and critically needed. Primary endpoints were to determine the recommended pediatric equivalent dose (RPED) assess pharmacokinetics (PK) safety. Secondary included overall response rate (ORR; comprising complete partial response) according 2014 National Institutes Health...
Abstract Autosomal dominant neurohypophyseal diabetes insipidus (ADNDI) is a defect in free water conservation caused by mutations the single gene that encodes both vasopressin (VP) and its binding protein, neurophysin II (NP II). Most of human this have been portion encoding NP molecule; resultant abnormal products are believed to cause cellular toxicity as improperly folded precursor molecules accumulate endoplasmic reticulum. We identified new American kindred with ADNDI found novel...
Hizentra® (IGSC 20 %) is a % liquid IgG product approved for subcutaneous administration in adults and children 2 years of age older who have primary immunodeficiency disease (PIDD). There limited information about the use IGSC very young including those less than 5 age. A retrospective chart review involved 88 PIDD infants received Hizentra®. The mean at start was 34 months (range to 59 months). administered weekly 86 (two additional twice three times infusions, respectively) included an...
Abstract We describe five cases of children who completed chemotherapy for infantile acute lymphoblastic leukemia (ALL) and soon after were diagnosed with severe T‐cell, non‐HIV immunodeficiency, varying B‐cell NK‐cell depletion. There was near absence CD3 + , CD4 CD8 cells. All patients developed multiple, primarily opportunistic infections. Unfortunately, four died, although one successfully treated by hematopoietic stem cell transplantation. These immunodeficiencies appeared to be...
This retrospective analysis comprises 10-year experience with early posttransplant mixed chimerism-based preemptive intervention. Out of 104 patients, 51 received immunotherapy. Their outcomes were similar to patients achieving full donor chimerism spontaneously. Among receiving intervention, 5-year event-free survival was identical in and without pretransplant residual disease, respectively (68% [95% confidence interval (CI) 38-98%] vs. 69% CI 54-85%] log-rank = 0.4). In who immunotherapy,...
The purpose of this study was to review the preclinical and clinical literature relevant efficacy safety anabolic androgen steroid therapy for palliative treatment severe weight loss associated with chronic diseases. Data sources were published identified from Medline database January 1966 December 2000, bibliographic references, textbooks. Reports trials selected. Study designs results extracted trial reports. Statistical evaluation or meta-analysis combined not attempted. Androgenic...