Oana Ciocârlie

Genome-edited, donor-derived allogeneic anti-CD19 chimeric antigen receptor T cells in paediatric and adult B-cell acute lymphoblastic leukaemia: results of two phase 1 studies

Reuben Benjamin, Oana Mirci‐Danicar, Athos Gianella-Borradori, Florence Binlich et autres

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Therapy of Paediatric B-ALL with a Fast Off Rate CD19 CAR Leads to Enhanced Expansion and Prolonged CAR T Cell Persistence in Patients with Low Bone Marrow Tumour Burden, and Is Associated with a Favourable Toxicity Profile

Sara Ghorashian, Anne Marijn Kramer, Shimobi Onuoha, Gary Wright et autres

Introduction: The CARPALL study (NCT02443831) employed a novel CD19CAR (CAT-41BBz CAR) with a faster off rate than the Kymriah FMC63-41BBz CAR (CAT 3.1x10-3s-1, FMC 6.8 x 10-5s-1), with equivalent on-rate (CAT 2.2 x 105, FMC 2.1 x 105). We herein report updated …

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Proposed Therapeutic Range of Treosulfan in Reduced Toxicity Pediatric Allogeneic Hematopoietic Stem Cell Transplant Conditioning: Results From a Prospective Trial

Robert Chiesa, Joseph F. Standing, Robert B. Winter, Zohreh Nademi et autres

Treosulfan is given off‐label in pediatric allogeneic hematopoietic stem cell transplant. This study investigated treosulfan's pharmacokinetics (PKs), efficacy, and safety in a prospective trial. Pediatric patients (n = 87) receiving treosulfan‐fludarabine conditioning were followed for at least 1 year posttransplant. PKs were …

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Delaying haematopoietic stem cell transplantation in children with viral respiratory infections reduces transplant‐related mortality

Giorgio Ottaviano, Giovanna Lucchini, Judith Breuer, Juliana M. Furtado‐Silva et autres

Viral respiratory infections (VRIs) contribute to the morbidity and transplant-related mortality (TRM) after allogeneic haematopoietic stem cell transplantation (HSCT) and strategies to prevent and treat VRIs are warranted. We monitored VRIs before and after transplant in children undergoing allogeneic HSCT with nasopharyngeal …

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Enhanced CAR T cell expansion and prolonged persistence in pediatric patients with ALL treated with a low-affinity CD19 CAR

Sara Ghorashian, Anne Marijn Kramer, Shimobi Onuoha, Gary Wright et autres

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Preliminary Data on Safety, Cellular Kinetics and Anti-Leukemic Activity of UCART19, an Allogeneic Anti-CD19 CAR T-Cell Product, in a Pool of Adult and Pediatric Patients with High-Risk CD19+ Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia

Reuben Benjamin, Charlotte Graham, Deborah Yallop, Agnieszka Jóźwik et autres

Abstract Background UCART19 is an allogeneic, genetically modified CAR T-cell product (anti-CD19 scFv- 41BB-CD3ζ) manufactured from healthy donor T cells, in which TRAC and CD52 genes have been knocked out to allow its administration in non-HLA matched patients (pts). Aims Preliminary safety/anti …

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Hematopoietic Stem Cell Transplantation as Treatment for Patients with DOCK8 Deficiency

Susanne Aydin, Alexandra F. Freeman, Waleed Al–Herz, Hamoud Al‐Mousa et autres

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Contrast-enhanced ultrasound for multiple liver lesions after bone marrow transplant in a child with leukaemia: Multifocal focal nodular hyperplasia

MV Verhagen, Oana Ciocârlie, Paul Humphries, Tom A. Watson

We present a case of a two-year-old girl in which liver lesions were characterised on contrast-enhanced ultrasound as multifocal focal nodular hyperplasia. This child had previously undergone haematopoietic stem cell transplantation for juvenile myelomonocytic leukaemia and was suspected to have hepatobiliary graft …

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A Novel Low Affinity CD19CAR Results in Durable Disease Remissions and Prolonged CAR T Cell Persistence without Severe CRS or Neurotoxicity in Patients with Paediatric ALL

Sara Ghorashian, Anne Marijn Kramer, Sarah J. Albon, Gary Wright et autres

Abstract Introduction: Published studies of CD19 CAR T cells have shown unprecedented response rates in ALL but with a 23-27% incidence of severe Cytokine Release Syndrome (CRS) and 27-50% incidence of severe neurotoxicity which may limit broader application. We developed a novel …

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INSULIN DEPENDENT DIABETES AND ENTEROPATHY ASSOCIATED WITH IPEX CURED BY SELECTIVE DONOR ENGRAFTMENT WITHIN T-REGULATORY CELL COMPARTMENT, FOLLOWING HAEMATOPOIETIC STEM CELL TRANSPLANTATION

Oana Ciocârlie

Dendritic cell reconstitution is associated with relapse-free survival and acute GVHD severity in children after allogeneic stem cell transplantation

Markus C. Elze, Oana Ciocârlie, Annekathrin Heinze, Stephan Kloeß et autres

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Myeloid and Plasmacytoid Dendritic Cells: Reference Ranges in the Peripheral Blood of Healthy Children

Oana Ciocârlie, Annekathrin Heinze, Markus C. Elze, Stephan Kloeß et autres

To date, few publications report on dendritic cells values in healthy children and mostly are found as control groups in studies focused on either allergic and autoimmune diseases or malignancies. This report provides an overview of 8 publications regarding absolute dendritic cells …

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