Robert Chiesa
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View article: Defining a <scp>CMV</scp> viral load threshold for pre‐emptive therapy in paediatric haematopoietic stem cell transplant recipients
Defining a <span>CMV</span> viral load threshold for pre‐emptive therapy in paediatric haematopoietic stem cell transplant recipients Open
Summary Cytomegalovirus (CMV) infection is a significant complication in paediatric haematopoietic stem cell transplant (HSCT), with substantial morbidity and mortality. While pre‐emptive treatment guided by CMV viral load thresholds helps…
View article: Safety and Diagnostic Utility of Brain Biopsy and Metagenomics in Decision-Making for Patients with Inborn Errors of Immunity (IEI) and Unexplained Neurological Manifestations
Safety and Diagnostic Utility of Brain Biopsy and Metagenomics in Decision-Making for Patients with Inborn Errors of Immunity (IEI) and Unexplained Neurological Manifestations Open
Unexplained neurological symptoms can pose a diagnostic challenge in patients with inborn errors of immunity (IEI) where the aetiology can be varied, and diverse pathologies may require contrasting treatments. Brain biopsy, the process of …
View article: Association of busulfan exposure and outcomes after HCT for patients with an inborn error of immunity
Association of busulfan exposure and outcomes after HCT for patients with an inborn error of immunity Open
Allogeneic hematopoietic cell transplantation (HCT) is a potentially curative treatment strategy for patients with inborn errors of immunities (IEIs). The objective of this study was to assess the optimal busulfan exposure before allogenei…
View article: Pediatric adapted risk index to predict 2-year transplant-related mortality post-HSCT in children
Pediatric adapted risk index to predict 2-year transplant-related mortality post-HSCT in children Open
Several attempts have been made to optimize pretransplant risk assessment to improve hematopoietic stem cell transplantation (HSCT) decision-making and to predict post-HSCT outcomes. However, the relevance of pretransplant risk assessment …
View article: Intention-to-treat outcomes utilising a stringent event definition in children and young people treated with tisagenlecleucel for r/r ALL through a national access scheme
Intention-to-treat outcomes utilising a stringent event definition in children and young people treated with tisagenlecleucel for r/r ALL through a national access scheme Open
CAR T-cell therapy has transformed relapsed/refractory (r/r) B-cell precursor acute lymphoblastic leukaemia (B-ALL) management and outcomes, but following CAR T infusion, interventions are often needed. In a UK multicentre study, we retros…
View article: CD19/CD22 targeting with cotransduced CAR T cells to prevent antigen-negative relapse after CAR T-cell therapy for B-cell ALL
CD19/CD22 targeting with cotransduced CAR T cells to prevent antigen-negative relapse after CAR T-cell therapy for B-cell ALL Open
CD19-negative relapse is a leading cause of treatment failure after chimeric antigen receptor (CAR) T-cell therapy for acute lymphoblastic leukemia. We investigated a CAR T-cell product targeting CD19 and CD22 generated by lentiviral cotra…
View article: Model Based Estimation of Posaconazole Tablet and Suspension Bioavailability in Hospitalized Children Using Real-World Therapeutic Drug Monitoring Data in Patients Receiving Intravenous and Oral Dosing
Model Based Estimation of Posaconazole Tablet and Suspension Bioavailability in Hospitalized Children Using Real-World Therapeutic Drug Monitoring Data in Patients Receiving Intravenous and Oral Dosing Open
Invasive fungal infections are a major cause of morbidity and mortality for immunocompromised patients. Posaconazole is approved for treatment and prophylaxis of invasive fungal infection in adult patients, with intravenous, oral suspensio…
View article: CD34<b>+</b>-selected stem cell boost can safely improve cytopenias following CAR T-cell therapy
CD34<b>+</b>-selected stem cell boost can safely improve cytopenias following CAR T-cell therapy Open
2][3][4] Prolonged cytopenias are an underrecognized toxicity of CAR T-cell therapy.For example, in the ELIANA study, 41% and 53% of patients demonstrated grade 3 to 4 thrombocytopenia and neutropenia, respectively, persisting beyond day 3…
View article: Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis
Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis Open
Allogeneic hematopoietic stem cell transplantation (HSCT) is a potentially curative treatment for patients affected by Wiskott-Aldrich syndrome (WAS). Reported HSCT outcomes have improved over time with respect to overall survival, but som…
View article: Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy
Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy Open
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) in early cerebral adrenoleukodystrophy can stabilize neurologic function and improve survival but has associated risks including transplant-related mortality (TRM), graft failu…
View article: Impact of in Vivo Lymphodepletion on Outcome in Children with Nonmalignant Disorders Receiving Peripheral Blood Stem Cell Transplantation
Impact of in Vivo Lymphodepletion on Outcome in Children with Nonmalignant Disorders Receiving Peripheral Blood Stem Cell Transplantation Open
Peripheral blood stem cell transplantation (PBSCT) with in vivo lymphodepletion can provide faster neutrophil recovery with limited risk of severe graft-versus-host disease (GVHD) in children with nonmalignant disorders (NMDs). We aimed to…
View article: Pathobiologic Mechanisms of Neurodegeneration in Osteopetrosis Derived From Structural and Functional Analysis of 14 ClC-7 Mutants
Pathobiologic Mechanisms of Neurodegeneration in Osteopetrosis Derived From Structural and Functional Analysis of 14 ClC-7 Mutants Open
ClC-7 is a chloride-proton antiporter of the CLC protein family. In complex with its accessory protein Ostm-1, ClC-7 localizes to lysosomes and to the osteoclasts' ruffled border, where it plays a critical role in acidifying the resorption…
View article: Hematopoietic cell transplantation in chronic granulomatous disease: a study of 712 children and adults
Hematopoietic cell transplantation in chronic granulomatous disease: a study of 712 children and adults Open
Chronic granulomatous disease (CGD) is a primary immunodeficiency resulting in life-threatening infections and inflammatory complications. Allogeneic hematopoietic cell transplantation (allo-HCT) can cure the disease, but the indication to…
View article: Cellular Therapies in Chronic Granulomatous Disease
Cellular Therapies in Chronic Granulomatous Disease Open
Allogeneic hematopoietic stem cell transplantation (HSCT) has become the main curative treatment in patients with chronic granulomatous disease (CGD). CGD is caused by inherited defects of the phagolysomal NADPH-oxidase, leading to a lifel…
View article: New insights into risk factors for transplant-associated thrombotic microangiopathy in pediatric HSCT
New insights into risk factors for transplant-associated thrombotic microangiopathy in pediatric HSCT Open
This study aimed to identify a risk profile for development of transplant-associated thrombotic microangiopathy (TA-TMA) in children undergoing hematopoietic stem cell transplantation (HSCT). Between 2013 and 2016, 439 children underwent 4…
View article: Modelling of neutrophil dynamics in children receiving busulfan or treosulfan for haematopoietic stem cell transplant conditioning
Modelling of neutrophil dynamics in children receiving busulfan or treosulfan for haematopoietic stem cell transplant conditioning Open
Aims Busulfan and treosulfan are cytotoxic agents used in the conditioning regime prior to paediatric haematopoietic stem cell transplantation (HSCT). These agents cause suppression of myeloid cells leaving patients severely immunocompromi…
View article: Transplant- and Disease-Related Outcomes of Allogeneic Hematopoietic Stem Cell Transplant in Patients with Cerebral Adrenoleukodystrophy Vary By Donor Cell Source, Conditioning Regimen, and Stage of Cerebral Disease
Transplant- and Disease-Related Outcomes of Allogeneic Hematopoietic Stem Cell Transplant in Patients with Cerebral Adrenoleukodystrophy Vary By Donor Cell Source, Conditioning Regimen, and Stage of Cerebral Disease Open
Early treatment with allogeneic hematopoietic stem cell transplantation (allo-HSCT) for cerebral adrenoleukodystrophy (CALD) has a beneficial effect on clinical indices of disease and long-term survival but is associated with immunologic r…
View article: Proposed Therapeutic Range of Treosulfan in Reduced Toxicity Pediatric Allogeneic Hematopoietic Stem Cell Transplant Conditioning: Results From a Prospective Trial
Proposed Therapeutic Range of Treosulfan in Reduced Toxicity Pediatric Allogeneic Hematopoietic Stem Cell Transplant Conditioning: Results From a Prospective Trial Open
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 treo…
View article: Delaying haematopoietic stem cell transplantation in children with viral respiratory infections reduces transplant‐related mortality
Delaying haematopoietic stem cell transplantation in children with viral respiratory infections reduces transplant‐related mortality Open
Summary 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 moni…
View article: Current status of umbilical cord blood transplantation in children
Current status of umbilical cord blood transplantation in children Open
Summary The first umbilical cord blood (UCB) transplantation was performed 30 years ago. UCB transplantation (UCBT) is now widely used in children with malignant and non‐malignant disorders who lack a matched family donor. UCBT affords a l…
View article: Adoptive T Cell Therapy Strategies for Viral Infections in Patients Receiving Haematopoietic Stem Cell Transplantation
Adoptive T Cell Therapy Strategies for Viral Infections in Patients Receiving Haematopoietic Stem Cell Transplantation Open
Adverse outcomes following virus-associated disease in patients receiving allogeneic haematopoietic stem cell transplantation (HSCT) have encouraged strategies to control viral reactivation in immunosuppressed patients. However, despite ti…
View article: Treatment dilemmas in asymptomatic children with primary hemophagocytic lymphohistiocytosis
Treatment dilemmas in asymptomatic children with primary hemophagocytic lymphohistiocytosis Open
Key Points Preemptive allogeneic stem cell transplantation improves prognosis of asymptomatic children genetically predisposed to primary HLH.
View article: Clinical T Cell Receptor Repertoire Deep Sequencing and Analysis: An Application to Monitor Immune Reconstitution Following Cord Blood Transplantation
Clinical T Cell Receptor Repertoire Deep Sequencing and Analysis: An Application to Monitor Immune Reconstitution Following Cord Blood Transplantation Open
Spectratyping assays are well recognized as the clinical gold standard for assessing the T cell receptor (TCR) repertoire in haematopoietic stem cell transplant (HSCT) recipients. These assays use length distributions of the hyper variable…