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Article
Open AccessFinding a balance in reduced toxicity hematopoietic stem cell transplantation for thalassemia: role of infused CD3+ cell count and immunosuppression
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 all...
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Article
Open AccessTreosulfan vs busulfan conditioning for allogeneic bmt in children with nonmalignant disease: a randomized phase 2 trial
Optimal conditioning prior to allogeneic hematopoietic stem cell transplantation for children with non-malignant diseases is subject of ongoing research. This prospective, randomized, phase 2 trial compared sa...
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Article
Open AccessIncidence of subsequent malignancies after total body irradiation-based allogeneic HSCT in children with ALL – long-term follow-up from the prospective ALL-SCT 2003 trial
Total body irradiation (TBI)-based conditioning is associated with superior leukemia-free survival in children with ALL undergoing HSCT. However, the risk for subsequent malignant neoplasms (SMN) remains a sig...
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Article
Open AccessResults of a multicenter phase I/II trial of TCRαβ and CD19-depleted haploidentical hematopoietic stem cell transplantation for adult and pediatric patients
Hematopoietic stem cell transplantation (HSCT) from haploidentical donors is a viable option for patients lacking HLA-matched donors. Here we report the results of a prospective multicenter phase I/II trial of...
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Article
Open AccessNBAS Variants Are Associated with Quantitative and Qualitative NK and B Cell Deficiency
Biallelic pathogenic NBAS variants manifest as a multisystem disorder with heterogeneous clinical phenotypes such as recurrent acute liver failure, growth retardation, and susceptibility to infections. This study...
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Article
Open AccessTreosulfan–fludarabine–thiotepa-based conditioning treatment before allogeneic hematopoietic stem cell transplantation for pediatric patients with hematological malignancies
Treosulfan-based conditioning prior to allogeneic transplantation has been shown to have myeloablative, immunosuppressive, and antineoplastic effects associated with reduced non-relapse mortality (NRM) in adul...
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Article
Correction: Long-term outcome after allogeneic hematopoietic stem cell transplantation for Shwachman–Diamond syndrome: a retrospective analysis and a review of the literature by the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation (SAAWP-EBMT)
An amendment to this paper has been published and can be accessed via a link at the top of the paper.
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Article
Long-term outcome after allogeneic hematopoietic stem cell transplantation for Shwachman–Diamond syndrome: a retrospective analysis and a review of the literature by the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation (SAAWP-EBMT)
Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative procedure in patients with Shwachman–Diamond syndrome (SDS) with bone marrow abnormalities. The results of 74 patients with SDS (6 acute ...
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Article
Correction: Hematopoietic stem cell transplantation for children with acute myeloid leukemia—results of the AML SCT-BFM 2007 trial
An amendment to this paper has been published and can be accessed via a link at the top of the paper.
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Article
Hematopoietic stem cell transplantation for children with acute myeloid leukemia—results of the AML SCT-BFM 2007 trial
AML SCT-BFM 2007 was the first hematopoietic stem cell transplantation (HCT) trial in Germany to comply with the European Clinical Trials Directive, and aimed to standardize pediatric HCT for acute myeloid leu...
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Article
Favorable immune recovery and low rate of GvHD in children transplanted with partially T cell-depleted PBSC grafts
Transplantation of peripheral blood stem cells (PBSC) from matched unrelated donors (MUD) is still associated with a significant risk for graft vs. host disease (GvHD), especially in pediatric patients receivi...
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Article
Open AccessEffective treatment of steroid and therapy-refractory acute graft-versus-host disease with a novel mesenchymal stromal cell product (MSC-FFM)
The inability to generate mesenchymal stromal cells (MSCs) of consistent potency likely is responsible for inconsistent clinical outcomes of patients with aGvHD receiving MSC products. We developed a novel MSC...
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Article
Selective induction of apoptosis in leukemic B-lymphoid cells by a CD19-specific TRAIL fusion protein
Although the treatment outcome of lymphoid malignancies has improved in recent years by the introduction of transplantation and antibody-based therapeutics, relapse remains a major problem. Therefore, new ther...
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Article
Targeting leukemic fusion proteins with small interfering RNAs: recent advances and therapeutic potentials
RNA interference has become an indispensable research tool to study gene functions in a wide variety of organisms. Because of their high efficacy and specificity, RNA interference-based approaches may also tra...
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Article
Biased distribution of chromosomal breakpoints involving the MLL gene in infants versus children and adults with t(4;11) ALL
Derivative chromosomes of 40 patients diagnosed with t(4;11) acute lymphoblastic leukemia (ALL) were analysed on the genomic DNA level. Chromosomal breakpoints were identified in most cases within the known br...
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Article
A DNA damage repair mechanism is involved in the origin of chromosomal translocations t(4;11) in primary leukemic cells
Some chromosomal translocations involved in the origin of leukemias and lymphomas are due to malfunctions of the recombinatorial machinery of immunoglobulin and T-cell receptor-genes. This mechanism has also b...
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Article
Fine structure of translocation breakpoints in leukemic blasts with chromosomal translocation t(4;11): the DNA damage-repair model of translocation
Chromosomal translocations t(4;11) are regularly associated with a specific type of acute leukemias and probably initiate the development of this disease. It has been proposed by others, that these translocati...
