A5029816641- Hematopoietic Stem Cell Transplantation
- Acute Lymphoblastic Leukemia research
- CAR-T cell therapy research
- Acute Myeloid Leukemia Research
- Childhood Cancer Survivors' Quality of Life
- Immune Cell Function and Interaction
- Chronic Myeloid Leukemia Treatments
- Mesenchymal stem cell research
- T-cell and B-cell Immunology
- Virus-based gene therapy research
- Immunodeficiency and Autoimmune Disorders
- Polyomavirus and related diseases
- Prenatal Screening and Diagnostics
- Transplantation: Methods and Outcomes
- Renal Transplantation Outcomes and Treatments
- Cancer Genomics and Diagnostics
- Chronic Lymphocytic Leukemia Research
- Blood disorders and treatments
- Cytomegalovirus and herpesvirus research
- Neuroblastoma Research and Treatments
- Neutropenia and Cancer Infections
- Hemoglobinopathies and Related Disorders
- Immunotherapy and Immune Responses
- Lymphoma Diagnosis and Treatment
- Neonatal Respiratory Health Research
Goethe University Frankfurt
2016-2025
University Hospital Frankfurt
2016-2025
Frankfurt Cancer Institute
2021-2025
Institute of Clinical Cancer Research
2021-2025
Deutschen Konsortium für Translationale Krebsforschung
2021-2025
Children's Clinical University Hospital
2003-2023
Uniwersytecki Szpital Dziecięcy
2003-2023
Klinik und Poliklinik für Kinder- und Jugendmedizin
2010-2021
St. Jude Children's Research Hospital
2003-2019
Children's Hospital of Los Angeles
2019
In a single-center phase 1-2a study, the anti-CD19 chimeric antigen receptor (CAR) T-cell therapy tisagenlecleucel produced high rates of complete remission and was associated with serious but mainly reversible toxic effects in children young adults relapsed or refractory B-cell acute lymphoblastic leukemia (ALL).We conducted 2, single-cohort, 25-center, global study pediatric adult patients CD19+ ALL. The primary end point overall rate (the incomplete hematologic recovery) within 3...
Purpose Blinatumomab is a bispecific T-cell engager antibody construct targeting CD19 on B-cell lymphoblasts. We evaluated the safety, pharmacokinetics, recommended dosage, and potential for efficacy of blinatumomab in children with relapsed/refractory precursor acute lymphoblastic leukemia (BCP-ALL). Methods This open-label study enrolled < 18 years old BCP-ALL phase I dosage-escalation part II part, using 6-week treatment cycles. Primary end points were maximum-tolerated dosage (phase I)...
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...
In HLA-nonidentical bone marrow transplantation, we studied the characteristics of donor NK cells, recipient leukemia and cytokine environment that predict antileukemia effects allogeneic cells. We found risk relapse in pediatric patients with hematologic malignancies was best predicted by a model taking into consideration presence inhibitory killer cell Ig-like receptors (KIRs) on donor's cells absence corresponding KIR ligand recipient's HLA repertoire (a receptor-ligand model). The...
A record number of 40 829 hematopoietic stem cell transplantation (HSCT) in 36 469 patients (15 765 allogeneic (43%), 20 704 autologous (57%)) were reported by 656 centers 47 countries to the 2014 survey. Trends include: continued growth transplant activity, more so Eastern European than west; a increase use haploidentical family donors (by 25%) and slower for unrelated donor HSCT. The cord blood as source has decreased again 2014. Main indications HSCT leukemias: 11 853 (33%; 96%...
Minimal residual disease (MRD) before allogeneic stem-cell transplantation was shown to predict outcome in children with relapsed acute lymphoblastic leukemia (ALL) retrospective analysis. To verify this, the Acute Lymphoblastic Leukemia Relapse Berlin-Frankfurt-Münster (ALL-REZ BFM) Study Group conducted a prospective trial.Between March 1999 and July 2005, 91 ALL treated according ALL-REZ BFM 96 or 2002 protocols receiving >or= second remission were enrolled. MRD quantification performed...
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...
Information on incidence, and factors associated with mortality is a prerequisite to improve outcome after hematopoietic stem cell transplantation (HSCT). Therefore, 55′668 deaths in 114′491 patients HSCT (83.7% allogeneic) for leukemia were investigated landmark analysis causes of death at day 30 (very early), 100 (early), 1 year (intermediate) 5 years (late). Mortality from all decreased cohort (1980–2001) 2 (2002–2015) post-transplant phases autologous HSCT. After allogeneic HSCT,...
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...
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...
Purpose Although hematopoietic stem-cell transplantation is widely performed in children with high-risk acute lymphoblastic leukemia (ALL), the influence of donor types poorly understood. Thus, outcomes were compared prospective multinational Berlin-Frankfurt-Muenster (BFM) study group trial: ALL-SCT-BFM 2003 (Allogeneic Stem Cell Transplantation Children and Adolescents Acute Lymphoblastic Leukemia). Patients Methods After conditioning total-body irradiation etoposide, 411 ALL received...
Clinical trials frequently include multiple end points that mature at different times. The initial report, typically based on the primary point, may be published when key planned co-primary or secondary analyses are not yet available. Trial Updates provide an opportunity to disseminate additional results from studies, in JCO elsewhere, for which point has already been reported. In analysis of global phase II ELIANA trial (ClinicalTrials.gov identifier: NCT02435849), tisagenlecleucel provided...
A record number of 39 209 HSCT in 34 809 patients (14 950 allogeneic (43%) and 19 859 autologous (57%)) were reported by 658 centers 48 countries to the 2013 survey. Trends include: more growth than HSCT, increasing use sibling unrelated donors a pronounced increase haploidentical family when compared with cord blood for those without matched related or donor. Main indications leukemias, 11 190 (32%; 96% allogeneic); lymphoid neoplasias, 958 (57%; 11% solid tumors, 1543 (4%; 4% nonmalignant...