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Ataluren ‐driven Restoration of Shwachman‐Bodian‐Diamond Syndrome Protein Function in Shwachman‐Diamond Syndrome Bone Marrow Cells

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Source: American Journal of Hematology - Category: Hematology Authors: Tags: Research Article Source Type: research

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, Khandanpour C, no Abstract Differentiation of haematopoietic stem cells is regulated by a concert of different transcription factors. Disturbed transcription factors function can be the basis of (pre)malignancies such as myelodysplastic syndrome or acute myeloid leukaemia. Growth factor independence 1b is a repressing transcription factor regulating quiescence of hematopoietic stem cellss and differentiation of erythrocytes and platelets. Here, we show that low expression of Growth factor independence 1b in blast cells is associated with an inferior prognosis of myelodysplastic syndrome and acute myeloid leukaem...
Source: Haematologica - Category: Hematology Authors: Tags: Haematologica Source Type: research
AbstractIn contrast to the evidence regarding azacitidine (Aza), there is limited knowledge about the combination of decitabine (DAC) and donor lymphocyte infusions as salvage therapy for relapse after allogeneic stem cell transplantation (allo-SCT) so far. We retrospectively analyzed data of 36 patients with hematological (n = 35) or molecular relapse (n = 1) of acute myeloid leukemia (AML,n = 29) or myelodysplastic syndrome (MDS,n = 7) collected from 6 German transplant centers. Patients were treated with a median of 2 cycles DAC (range, 1 to 11). DAC was the first ...
Source: Annals of Hematology - Category: Hematology Source Type: research
Haplo/cord transplantation combines an umbilical cord blood (UCB) graft with CD34-selected haploidentical cells and results in rapid hematopoietic recovery followed by durable UCB engraftment. We compared outcomes of transplants in older patients with acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS) who received either HLA-matched unrelated donor cells (MUD) or haplo/cord grafts. Between 2007 and 2013, 109 adults ages 50 and older underwent similar reduced intensity conditioning (RIC) with fludarabine and melphalan and antibody-mediated T-cell depletion for AML (n=83) or high risk MDS (n=26) follow...
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research
Relapse remains the main obstacle for patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) after allogeneic stem cell transplantation and novel therapeutic strategies are urg... Author: VJHemOnc Added: 12/18/2017
Source: Oncology Tube - Category: Cancer & Oncology Source Type: podcasts
The outcome for patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS) after allogeneic stem cell transplantation remains poor. In this interview, Charles Craddock, CBE, FRCP (U... Author: VJHemOnc Added: 12/18/2017
Source: Oncology Tube - Category: Cancer & Oncology Source Type: podcasts
Conclusion Danazol as first-line therapy is an acceptable treatment option with low side effects for patients with MDS who cannot receive ASCT. Micro-Abstract At present, no standard therapy is available for most patients with myelodysplastic syndrome. In this retrospective study, we analyze data from 42 patients with myelodysplastic syndrome treated with low-dose danazol. More than half achieved a response. Results show that danazol remains an attractive option because of its low cost and good safety profile in centers with reduced access to novel therapeutic alternatives.
Source: Clinical Lymphoma Myeloma and Leukemia - Category: Cancer & Oncology Source Type: research
Conclusion Danazol as frontline therapy is an acceptable treatment option with low side effects for patients with MDS that cannot receive ASCT Teaser At present, no standard therapy is available for most patients with myelodysplastic syndromes. In this retrospective study, we analyse data from 42 patients with myelodysplastic syndrome treated with low-dose danazol. More than half achieved a response. Results show that danazol remains an attractive option due to its low cost and good safety profile in centers with reduced access to novel therapeutic alternatives.
Source: Clinical Lymphoma Myeloma and Leukemia - Category: Cancer & Oncology Source Type: research
Authors: Alter BP Abstract Patients with inherited bone marrow failure syndromes are usually identified when they develop hematologic complications such as severe bone marrow failure, myelodysplastic syndrome, or acute myeloid leukemia. They often have specific birth defects or other physical abnormalities that suggest a syndrome, and sequencing of specific genes or next-generation sequencing can determine or confirm the particular syndrome. The 4 most frequent syndromes are Fanconi anemia, dyskeratosis congenita, Diamond Blackfan anemia, and Shwachman Diamond syndrome. This review discusses the major complications...
Source: Hematology ASH Education Program - Category: Hematology Tags: Hematology Am Soc Hematol Educ Program Source Type: research
AbstractImmunocompromised patients are at high risk of invasive fungal infections (IFI), in particular those with haematological malignancies undergoing remission-induction chemotherapy for acute myeloid leukaemia (AML) or myelodysplastic syndrome (MDS) and recipients of allogeneic haematopoietic stem cell transplants (HSCT). Despite the development of new treatment options in the past decades, IFI remains a concern due to substantial morbidity and mortality in these patient populations. In addition, the increasing use of new immune modulating drugs in cancer therapy has opened an entirely new spectrum of at risk periods. ...
Source: Annals of Hematology - Category: Hematology Source Type: research
Abstract Disease overviewThe myelodysplastic syndromes (MDS) are a very heterogeneous group of myeloid disorders characterized by peripheral blood cytopenias and increased risk of transformation to acute myelogenous leukemia (AML). MDS occurs more frequently in older males and in individuals with prior exposure to cytotoxic therapy. DiagnosisDiagnosis of MDS is based on morphological evidence of dysplasia upon visual examination of a bone marrow aspirate and biopsy. Information obtained from additional studies such as karyotype, flow cytometry or molecular genetics is usually complementary and may help refine diagnosis. Ri...
Source: American Journal of Hematology - Category: Hematology Authors: Tags: ANNUAL CLINICAL UPDATES IN HEMATOLOGICAL MALIGNANCIES Source Type: research
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