TY  - JOUR
A1  - Robin, Marie
A1  - de Wreede, Liesbeth C.
A1  - Wolschke, Christine
A1  - Schetelig, Johannes
A1  - Eikema, Diderik-Jan
A1  - Van Lint, Maria Teresa
A1  - Knelange, Nina Simone
A1  - Beelen, Dietrich
A1  - Brecht, Arne
A1  - Niederwieser, Dietger
A1  - Vitek, Antonin
A1  - Bethge, Wolfgang
A1  - Arnold, Renate
A1  - Finke, Jürgen
A1  - Volin, Liisa
A1  - Yakoub-Agha, Ibrahim
A1  - Nagler, Arnon
A1  - Poiré, Xavier
A1  - Einsele, Hermann
A1  - Chevallier, Patrice
A1  - Holler, Ernst
A1  - Ljungman, Per
A1  - Robinson, Stephen
A1  - Radujkovic, Alekxandar
A1  - McLornan, Donal
A1  - Chalandon, Yves
A1  - Kröger, Nicolaus
T1  - Long-term outcome after allogeneic hematopoietic cell transplantation for myelofibrosis
JF  - Haematologica
N2  - Allogeneic hematopoietic stem cell transplant remains the only curative treatment for myelofibrosis. Most post-transplantation events Aoccur during the first two years and hence we aimed to analyze the outcome of 2-year disease-free survivors. A total of 1055 patients with myelofibrosis transplanted between 1995 and 2014 and registered in the registry of the European Society for Blood and Marrow Transplantation were included. Survival was compared to the matched general population to determine excess mortality and the risk factors that are associated. In the 2-year survivors, disease-free survival was 64% (60-68%) and overall survival was 74% (71-78%) at ten years; results were better in younger individuals and in women. Excess mortality was 14% (8-21%) in patients aged <45 years and 33% (13-53%) in patients aged >= 65 years. The main cause of death was relapse of the primary disease. Graft-versus-host disease (GvHD) before two years decreased the risk of relapse. Multivariable analysis of excess mortality showed that age, male sex recipient, secondary myelofibrosis and no GvHD disease prior to the 2-year landmark increased the risk of excess mortality. This is the largest study to date analyzing long-term outcome in patients with myelofibrosis undergoing transplant. Overall it shows a good survival in patients alive and in remission at two years. However, the occurrence of late complications, including late relapses, infectious complications and secondary malignancies, highlights the importance of screening and monitoring of long-term survivors.
KW  - Prognostic scoring system
KW  - Societe Francaise
KW  - Gruppo-italiano
KW  - European group
KW  - Late mortality
KW  - Midollo-Osseo
KW  - LATE DEATHS
KW  - Survival
KW  - Blood
KW  - Ruxolitinib
Y1  - 2019
U6  - http://nbn-resolving.de/urn/resolver.pl?urn:nbn:de:bvb:20-opus-226386
VL  - 104
IS  - 9
ER  - 
TY  - JOUR
A1  - Maucher, Marius
A1  - Srour, Micha
A1  - Danhof, Sophia
A1  - Einsele, Hermann
A1  - Hudecek, Michael
A1  - Yakoub-Agha, Ibrahim
T1  - Current limitations and perspectives of chimeric antigen receptor-T-cells in acute myeloid leukemia
JF  - Cancers
N2  - Adoptive transfer of gene-engineered chimeric antigen receptor (CAR)-T-cells has emerged as a powerful immunotherapy for combating hematologic cancers. Several target antigens that are prevalently expressed on AML cells have undergone evaluation in preclinical CAR-T-cell testing. Attributes of an ‘ideal’ target antigen for CAR-T-cell therapy in AML include high-level expression on leukemic blasts and leukemic stem cells (LSCs), and absence on healthy tissues, normal hematopoietic stem and progenitor cells (HSPCs). In contrast to other blood cancer types, where CAR-T therapies are being similarly studied, only a rather small number of AML patients has received CAR-T-cell treatment in clinical trials, resulting in limited clinical experience for this therapeutic approach in AML. For curative AML treatment, abrogation of bulk blasts and LSCs is mandatory with the need for hematopoietic recovery after CAR-T administration. Herein, we provide a critical review of the current pipeline of candidate target antigens and corresponding CAR-T-cell products in AML, assess challenges for clinical translation and implementation in routine clinical practice, as well as perspectives for overcoming them.
KW  - AML
KW  - CAR-T-cell
KW  - hematology
KW  - gene therapy
KW  - adoptive cell therapy
Y1  - 2021
U6  - http://nbn-resolving.de/urn/resolver.pl?urn:nbn:de:bvb:20-opus-252180
SN  - 2072-6694
VL  - 13
IS  - 24
ER  -