Peter Lang
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View article: Human CART22.19 Therapy in Refractory Pediatric B-ALL: Insights from a Named-Patient Cohort
Human CART22.19 Therapy in Refractory Pediatric B-ALL: Insights from a Named-Patient Cohort Open
Background CD19-directed chimeric antigen receptor (CAR) T-cell therapies have transformed the treatment landscape for pediatric B-cell acute lymphoblastic leukemia (B-ALL), yet relapse driven by antigen escape remains a major limitation. …
View article: Improvements in health-related quality of life in patients with transfusion-dependent β-thalassemia after exagamglogene autotemcel
Improvements in health-related quality of life in patients with transfusion-dependent β-thalassemia after exagamglogene autotemcel Open
Transfusion-dependent β-thalassemia (TDT) can have negative impacts on a patient’s health-related quality of life (HRQoL). Exagamglogene autotemcel (exa-cel) is a one-time, ex vivo CRISPR-Cas9 gene–edited cell therapy for TDT shown in a ph…
View article: Deletion of the <scp> <i>HLA‐B</i> </scp> Gene in One of the Inherited Haplotypes in a Northern European Family
Deletion of the <span> <i>HLA‐B</i> </span> Gene in One of the Inherited Haplotypes in a Northern European Family Open
Targeted next generation sequencing‐based HLA typing of a 17‐year‐old female transplant patient showed homozygosity for the HLA‐B allele. The segregation analysis of HLA haplotypes of family members only allowed the conclusion that the B‐a…
View article: Hematopoietic stem cell transplantation in a newborn suffering from severe combined immunodeficiency and severe hemophilia A: a case report and review of the literature
Hematopoietic stem cell transplantation in a newborn suffering from severe combined immunodeficiency and severe hemophilia A: a case report and review of the literature Open
This is the first case report of a newborn suffering from both SCID and severe hemophilia A. HSCT is feasible in this situation without bleeding complications if an individual substitution regimen with FVIII is applied.
View article: High Incidence and Impact of Suspected Exocrine Pancreatic Insufficiency in Patients Post‐Hematopoietic Stem Cell Transplantation: A Single‐Center Prospective Observational Study
High Incidence and Impact of Suspected Exocrine Pancreatic Insufficiency in Patients Post‐Hematopoietic Stem Cell Transplantation: A Single‐Center Prospective Observational Study Open
Exocrine pancreatic insufficiency (EPI) is suspected but remains understudied in immunosuppressed conditions such as post‐hematopoietic stem cell transplantation (HSCT). This prospective observational study aimed to investigate the inciden…
View article: Multinational retrospective analysis of bridging therapy prior to chimeric antigen receptor t cells for relapsed/refractory acute lymphoblastic leukemia in children and young adults
Multinational retrospective analysis of bridging therapy prior to chimeric antigen receptor t cells for relapsed/refractory acute lymphoblastic leukemia in children and young adults Open
Anti-CD19 chimeric antigen receptor T cells (CAR) are a well-established treatment option for children and young adults suffering from relapsed/refractory B-lineage acute lymphoblastic leukemia. Bridging therapy is used to control disease …
View article: Everolimus with or without Mycophenolate Mofetil for Graft-versus-Host Disease Prophylaxis after Hematopoietic Stem Cell Transplantation in Children with Acute Kidney Injury: A Single-Center Retrospective Analysis
Everolimus with or without Mycophenolate Mofetil for Graft-versus-Host Disease Prophylaxis after Hematopoietic Stem Cell Transplantation in Children with Acute Kidney Injury: A Single-Center Retrospective Analysis Open
Hematopoietic stem cell transplantation (HSCT) serves as a therapeutic intervention for various pediatric diseases. Acute and chronic graft-versus-host disease (GVHD) are decisive determinants of successful allogeneic HSCT. The immunosuppr…
View article: Long-Term Outcomes and Quality of Life of High-Risk Neuroblastoma Patients Treated with a Multimodal Treatment Including Anti-GD2 Immunotherapy: A Retrospective Cohort Study
Long-Term Outcomes and Quality of Life of High-Risk Neuroblastoma Patients Treated with a Multimodal Treatment Including Anti-GD2 Immunotherapy: A Retrospective Cohort Study Open
Background: The incorporation of anti-GD2 antibodies such as ch14.18/SP2/0 into the multimodal treatment of high-risk neuroblastoma (HR-NB) patients has improved their outcomes. As studies assessing the long-term outcomes, long-term sequel…
View article: Elevated Soluble ACE2 Activity in Children and Adults After SARS‐CoV‐2 Exposure Irrespective of Laboratory‐Confirmed Infection
Elevated Soluble ACE2 Activity in Children and Adults After SARS‐CoV‐2 Exposure Irrespective of Laboratory‐Confirmed Infection Open
The pivotal role of the cell entry receptor ACE2 for SARS‐CoV‐2 infection is well‐established. When ACE2 is shed from cell surface into plasma as soluble ACE2 (sACE2), it can effectively neutralize SARS‐CoV‐2. This longitudinal prospective…
View article: Allogeneic Hematopoietic Stem Cell Transplantation in Pediatric Acute Myeloid Leukemia: Results of the AML BFM Study Group
Allogeneic Hematopoietic Stem Cell Transplantation in Pediatric Acute Myeloid Leukemia: Results of the AML BFM Study Group Open
Introduction: The considerable success in the transplant field has changed transplant indications for pediatric acute myeloid leukemia (AML). However, the risk stratification for allogeneic hematopoietic stem cell transplantation (allo-HSC…
View article: Durable Clinical Benefits with Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia
Durable Clinical Benefits with Exagamglogene Autotemcel for Transfusion-Dependent β-Thalassemia Open
Background: Exagamglogene autotemcel (exa-cel) is a non-viral cell therapy that reactivates fetal hemoglobin (HbF) via ex vivo CRISPR-Cas9 gene-editing of autologous CD34+ hematopoietic stem and progenitor cells at the erythroid-specific e…
View article: Investigation of iron oxide nanoparticle formation in a spray-flame synthesis process using laser-induced incandescence
Investigation of iron oxide nanoparticle formation in a spray-flame synthesis process using laser-induced incandescence Open
In this work, iron-oxide nanoparticle formation in the spray-flame synthesis (SFS) process of the standardized SpraySyn 2.0 burner was investigated in situ using laser-induced incandescence (LII). For the evaluation of these measurements, …
View article: Long‐term and landscape impacts of buffel grass on arid plant communities: Ecosystem shifts and acceleration by fire
Long‐term and landscape impacts of buffel grass on arid plant communities: Ecosystem shifts and acceleration by fire Open
Plant invasions drive biodiversity loss, transform ecosystems, and promote positive‐feedback cycles between invasion and fire. However, the long‐term impacts of invasive grasses across landscapes with diverse plant communities and interact…
View article: Irinotecan and temozolomide in combination with dasatinib and rapamycin versus irinotecan and temozolomide for patients with relapsed or refractory neuroblastoma (RIST-rNB-2011): a multicentre, open-label, randomised, controlled, phase 2 trial
Irinotecan and temozolomide in combination with dasatinib and rapamycin versus irinotecan and temozolomide for patients with relapsed or refractory neuroblastoma (RIST-rNB-2011): a multicentre, open-label, randomised, controlled, phase 2 trial Open
Background Neuroblastoma is the most common extracranial solid tumour in children. Relapsed or refractory neuroblastoma is associated with a poor outcome. We assessed the combination of irinotecan–temozolomide and dasatinib–rapamycin (RIST…
View article: Adenovirus‐ and cytomegalovirus‐specific adoptive T‐cell therapy in the context of hematologic cell transplant or HIV infection – A single‐center experience
Adenovirus‐ and cytomegalovirus‐specific adoptive T‐cell therapy in the context of hematologic cell transplant or HIV infection – A single‐center experience Open
Background Reactivation of viral infections, in particular cytomegalovirus (CMV) and adenovirus (ADV), cause morbidity and non‐relapse‐mortality in states of immune deficiency, especially after allogeneic hematopoietic cell transplantation…
View article: Everolimus and mycophenolate mofetil effectively prevent GvHD in children with severe acute kidney injury undergoing allogeneic HSCT
Everolimus and mycophenolate mofetil effectively prevent GvHD in children with severe acute kidney injury undergoing allogeneic HSCT Open
Allogeneic hematopoietic stem cell transplantation (HSCT) serves as a therapeutic intervention for various pediatric diseases. Acute kidney injury afflicts 21-84% of pediatric HSCT cases, significantly compromising clinical outcomes. This …
View article: Allogeneic hematopoietic cell transplantation for adult metachromatic leukodystrophy: a case series
Allogeneic hematopoietic cell transplantation for adult metachromatic leukodystrophy: a case series Open
Metachromatic leukodystrophy (MLD) is a rare genetic disorder caused by pathogenic variants of the ARSA gene, leading to a deficiency of the arylsulfatase A enzyme (ARSA) and consecutive accumulation of galactosylceramide-3-0-sulfate in th…
View article: Finding a balance in reduced toxicity hematopoietic stem cell transplantation for thalassemia: role of infused CD3+ cell count and immunosuppression
Finding a balance in reduced toxicity hematopoietic stem cell transplantation for thalassemia: role of infused CD3+ cell count and immunosuppression Open
We performed a retrospective analysis on 124 patients with transfusion-dependent thalassemia who were registered in the German pediatric registry for stem cell transplantation. All patients underwent first allogeneic hematopoietic stem cel…
View article: Influence of ATLG serum levels on CD3/CD19-depleted hematopoietic grafts and on immune recovery in pediatric haplo-HSCT
Influence of ATLG serum levels on CD3/CD19-depleted hematopoietic grafts and on immune recovery in pediatric haplo-HSCT Open
Anti–T lymphocyte globulin (ATLG) significantly reduces the risk of engraftment failure in allogeneic hematopoietic stem cell transplant (HSCT) but hampers posttransplant immune reconstitution. We hypothesized that in patients receiving ha…
View article: Quality of life and healthcare utilization during the COVID-19 pandemic are more restricted in chronically ill than in healthy children: a tertiary care children’s hospital experience
Quality of life and healthcare utilization during the COVID-19 pandemic are more restricted in chronically ill than in healthy children: a tertiary care children’s hospital experience Open
The global COVID-19 pandemic forced changes in everyday life of children and adolescents due to government containment measures, an altered healthcare accessibility and utilization, and public concern about SARS-CoV-2 transmission. Data on…
View article: Graft Manipulation
Graft Manipulation Open
Graft manipulation is performed to define and to optimize the volume and cellular composition of stem cell sources like apheresis products, bone marrow, or umbilical cord blood.
View article: A novel approach to guide GD2-targeted therapy in pediatric tumors by PET and [<sup>64</sup>Cu]Cu-NOTA-ch14.18/CHO
A novel approach to guide GD2-targeted therapy in pediatric tumors by PET and [<sup>64</sup>Cu]Cu-NOTA-ch14.18/CHO Open
Background: The tumor-associated disialoganglioside GD2 is a bona fide immunotherapy target in neuroblastoma and other childhood tumors, including Ewing sarcoma and osteosarcoma. GD2-targeting antibodies proved to be effective in neuroblas…
View article: Targeted therapies in retinoblastoma: GD2-directed immunotherapy following autologous stem cell transplantation and evaluation of alternative target B7-H3
Targeted therapies in retinoblastoma: GD2-directed immunotherapy following autologous stem cell transplantation and evaluation of alternative target B7-H3 Open
Background GD2-directed immunotherapy is highly effective in the treatment of high-risk neuroblastoma (NB), and might be an interesting target also in other high-risk tumors. Methods The German-Austrian Retinoblastoma Registry, Essen, was …