A5078683084- Hematopoietic Stem Cell Transplantation
- Acute Myeloid Leukemia Research
- Hemoglobinopathies and Related Disorders
- Acute Lymphoblastic Leukemia research
- Prenatal Screening and Diagnostics
- Childhood Cancer Survivors' Quality of Life
- CAR-T cell therapy research
- Neutropenia and Cancer Infections
- Blood groups and transfusion
- Parvovirus B19 Infection Studies
- Neuroblastoma Research and Treatments
- Cancer Genomics and Diagnostics
- Glioma Diagnosis and Treatment
- Blood disorders and treatments
- Chronic Myeloid Leukemia Treatments
- Telomeres, Telomerase, and Senescence
- PI3K/AKT/mTOR signaling in cancer
- Chronic Lymphocytic Leukemia Research
- Cancer therapeutics and mechanisms
- Immunotherapy and Immune Responses
- Virus-based gene therapy research
- Immune Cell Function and Interaction
- Iron Metabolism and Disorders
- Platelet Disorders and Treatments
- Myeloproliferative Neoplasms: Diagnosis and Treatment
University Hospital Regensburg
2016-2025
University of Regensburg
2016-2025
Cornell University
2000-2024
University College London
2024
Magna Graecia University
2024
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
2024
Bambino Gesù Children's Hospital
2024
Heinrich Heine University Düsseldorf
2024
Düsseldorf University Hospital
2024
Ospedale Maggiore
2024
Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are severe monogenic diseases with potentially life-threatening manifestations. BCL11A is a transcription factor that represses γ-globin expression fetal hemoglobin in erythroid cells. We performed electroporation of CD34+ hematopoietic stem progenitor cells obtained from healthy donors, CRISPR-Cas9 targeting the erythroid-specific enhancer. Approximately 80% alleles at this locus were modified, no evidence off-target...
Sinusoidal obstruction syndrome, also known as veno-occlusive disease (SOS/VOD), is a potentially life threatening complication that can develop after hematopoietic cell transplantation. Although SOS/VOD progressively resolves within few weeks in most patients, the severe forms result multi-organ dysfunction and are associated with high mortality rate (>80%). Therefore, careful attention must be paid to allow an early detection of SOS/VOD, particularly drugs have now proven effective...
Numbers of Hematopoietic cell transplantation (HCT) in Europe and collaborating countries continues to rise with 48,512 HCT 43,581 patients, comprising 19,798 (41%) allogeneic 28,714 (59%) autologous, reported by 700 centers 51 during 2019. Main indications were myeloid malignancies 10,764 (25%), lymphoid 27,895 (64%), nonmalignant disorders 3173 (7%). A marked growth CAR-T cellular therapies from 151 2017 1134 patients 2019 is observed. This year's analyses focus on changes over 30 years....
Chimeric antigen receptor (CAR) T cells are a novel class of anti-cancer therapy in which autologous or allogeneic engineered to express CAR targeting membrane antigen. In Europe, tisagenlecleucel (Kymriah™) is approved for the treatment refractory/relapsed acute lymphoblastic leukemia children and young adults as well relapsed/refractory diffuse large B-cell lymphoma, while axicabtagene ciloleucel (Yescarta™) high-grade lymphoma primary mediastinal lymphoma. Both agents genetically CD19....
Several commercial and academic autologous chimeric antigen receptor T-cell (CAR-T) products targeting CD19 have been approved in Europe for relapsed/refractory B-cell acute lymphoblastic leukemia, high-grade lymphoma mantle cell lymphoma. Products other diseases such as multiple myeloma follicular are likely to be by the European Medicines Agency near future.The Society Blood Marrow Transplantation (EBMT)-Joint Accreditation Committee of ISCT EBMT (JACIE) Haematology Association...
The advances in hematopoietic cell transplantation (HCT) over the last decade have led to a transplant-related mortality below 15%. Hepatic sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD) is life-threatening complication of HCT that belongs group diseases increasingly identified as transplant-related, systemic endothelial diseases. In most cases, SOS/VOD resolves within weeks; however, severe results multi-organ dysfunction/failure with rate >80%. A timely diagnosis critical...
For over two decades, the EBMT has updated recommendations on indications for haematopoietic cell transplantation (HCT) practice based clinical and scientific developments in field. This is eighth special report HCT haematological diseases, solid tumours immune disorders. Our aim to provide general guidance according prevailing countries centres. In order inform patient decisions, these must be considered conjunction with risk of disease, procedure non-transplant strategies, including...
Total body irradiation (TBI) before allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with acute lymphoblastic leukemia (ALL) is efficacious, but long-term side effects are concerning. We investigated whether preparative combination chemotherapy could replace TBI such patients.FORUM a randomized, controlled, open-label, international, multicenter, phase III, noninferiority study. Patients ≤ 18 years at diagnosis, 4-21 HSCT, complete remission pre-HSCT, and an...
Abstract Hematopoietic-cell transplantation (HCT) is widely used for acquired and congenital disorders of the hematopoietic system. Number transplants performed in Europe associated countries continues to rise with 47,468 HCT 42,901 patients [19,630 allogeneic (41%) 27,838 autologous (59%)] reported by 701 centers 50 2018. Main indications were myeloid malignancies 10,679 (25%; 97% allogeneic), lymphoid 27,318 (64%; 20% solid tumors 1625 (4%; 2.9% nonmalignant 3063 (7%; 81% allogeneic). This...
The Worldwide Network of Blood and Marrow Transplantation (WBMT) pursues the mission promoting hematopoietic cell transplantation (HCT) for instance by evaluating activities through member societies, national registries individual centers. In 2016, 82,718 first HCT were reported 1,662 teams in 86 195 World Health Organization states representing a global increase 6.2% autologous 7.0% allogeneic bringing total to 1,298,897 procedures. Assuming frequency 84,000/year, 1.5 million performed 2019...
Hematopoietic cell transplantation (HCT) is widely used for acquired and congenital disorders of the hematopoietic system. Number transplants done in Europe associated countries continues to rise with 45,418 HCT 41,100 patients [(17,155 allogeneic (42%) 23,945 autologous (58%)] reported by 683 centers 50 2017. Main indications were myeloid malignancies 10,147 (25%; 96% allogeneic), lymphoid 26,488 (64%; 19% solid tumors 1,607 (3.9%; 2% nonmalignant 2,667 (7%; 81% allogeneic). Trends donor...
Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to reactivate fetal hemoglobin synthesis by means of ex vivo clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 gene editing autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) at the erythroid-specific enhancer region
Abstract In 2021, 47,412 HCT (19,806 (42%) allogeneic and 27,606 (58%) autologous) in 43,109 patients were reported by 694 European centers. 3494 received advanced cellular therapies, 2524 of which CAR-T treatments, an additional 3245 DLI. Changes compared to the previous year treatment (+35%), +5.4%, autologous +3.9%, more pronounced non-malignant disorders. Main indications for myeloid malignancies 10,745 (58%), lymphoid 5127 (28%) disorders 2501 (13%). 22,129 (90%) solid tumors 1635 (7%)....
BackgroundExagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to reactivate fetal hemoglobin synthesis through ex vivo clustered regularly interspaced short palindromic repeats (CRISPR)–Cas9 gene editing of the erythroid-specific enhancer region BCL11A in autologous CD34+ hematopoietic stem and progenitor cells (HSPCs).MethodsWe conducted an open-label, single-group, phase 3 study exa-cel patients 12 35 years age with transfusion-dependent β-thalassemia β0/β0, β0/β0-like,...
Oncogenic Kit mutations are found in somatic gastrointestinal (GI) stromal tumors (GISTs) and mastocytosis. A mouse model for the study of constitutive activation oncogenesis has been produced by a knock-in strategy introducing exon 11-activating mutation into genome based on case human familial GIST syndrome. Heterozygous mutant V558 Δ /+ mice develop symptoms disease eventually die from pathology GI tract. Patchy hyperplasia Kit-positive cells is evident within myenteric plexus entire...
Abstract Ependymoma with YAP1‐MAMLD1 fusion is a rare, recently described supratentorial neoplasm of childhood, few cases published so far. We report on 15 pediatric patients ependymomas carrying fusions, their characteristic histopathology, immunophenotype and molecular/cytogenetic, radiological clinical features. The was documented by RT‐PCR/Sanger sequencing, tumor genomes were studied molecular inversion probe (MIP) analysis. Significant copy number alterations identified GISTIC (Genomic...