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Haploidentical Hematopoietic Stem Cell Transplantation for Myelodysplastic Syndrome

1 CIBMTR scoring system can predict long-term outcomes of MDS following haplo-HSCT.2 We refined this scoring system and proposed a new one for haplo-HSCT recipients.3 Our refined scoring system can predict short- and long-term outcomes after haplo-HSCT.
Source: Biology of Blood and Marrow Transplantation - Category: Hematology Authors: Source Type: research

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We present the clinical case of a 26 year-old male with dendritic ulcers in his cornea, marrow aplasia and idiopathic chronic lymphedema. He was diagnosed with GATA2 deficiency. GATA2 gene is critical for the genesis and function of hematopoietic stem cells. Its deficiency can cause myelodysplastic syndromes, congenital lymphedema and severe viral infections. Our patient presented these three manifestations, added to a deletion in 20q12 that confirmed the suspicion of GATA2 mutation. A bone marrow transplant was suggested as definitive treatment. The corneal herpetic epithelial lesion was analogous to a regular hepetic ker...
Source: Saudi Journal of Ophthalmology - Category: Opthalmology Source Type: research
Abstract Despite the curative potential of allogeneic stem cell transplantation (allo-SCT) in patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS), many patients will relapse. Until recently therapeutic options mainly consisted of palliative care, chemotherapy, donor lymphocyte infusions and second transplantation in selected cases. Still many patients either do not tolerate intensive therapies or do not achieve durable remissions and will finally succumb. Given this unmet medical need the hypomethylating agents (HMA), Azacitidine (Aza) and Decitabine (DAC) have been tested as salvage the...
Source: International Journal of Hematology - Category: Hematology Authors: Tags: Int J Hematol Source Type: research
Publication date: Available online 9 November 2017 Source:Seminars in Cancer Biology Author(s): Christian Flotho, Sebastian Sommer, Michael Lübbert Myelodysplastic syndrome (MDS) is a clonal bone marrow disorder, typically of older adults, which is characterized by ineffective hematopoiesis, peripheral blood cytopenias and risk of progression to acute myeloid leukemia. Juvenile myelomonocytic leukemia (JMML) is an aggressive myeloproliferative neoplasm occurring in young children. The common denominator of these malignant myeloid disorders is the limited benefit of conventional chemotherapy and a particular responsiv...
Source: Seminars in Cancer Biology - Category: Cancer & Oncology Source Type: research
M, Arnold R, De Witte T, Robin M, Kröger N Abstract No standard exists for the treatment of myelodysplastic syndrome relapsing after allogeneic stem cell transplantation. We performed a retrospective registry analysis of outcome and risk factors in 698 patients, treated with different strategies. Median overall survival from relapse was 4.7 months (4.1-5.3), 2-year survival was 17.7% (14.8-21.2%). Shorter remission after transplantation (p
Source: Haematologica - Category: Hematology Authors: Tags: Haematologica Source Type: research
Conclusions: This first-in-pediatric trial of epigenetic priming in patients with newly diagnosed AML demonstrates that decitabine pre-treatment followed by standard combination chemotherapy is well tolerated in children with newly diagnosed AML. Morphologic complete responses were similar in both treatment arms. MRD at Day 30 following induction therapy suggests a deeper remission in patients receiving decitabine. No differences were observed between treatment arms in hematologic toxicities although decitabine-treated patients were noted to have more gastrointestinal toxicities, anorexia and hypophosphatemia. Decitabine P...
Source: GEO: Gene Expression Omnibus - Category: Genetics & Stem Cells Tags: Methylation profiling by array Homo sapiens Source Type: research
Summary A disease risk index (DRI) has been defined for stratifying heterogeneous cohorts of patients undergoing allogeneic haematopoietic stem cell transplantation (HSCT). This index defines 4 distinct groups with different outcomes, dividing patients by disease type and status and considering cytogenetics for acute myeloid leukaemia and myelodysplastic syndromes (MDS). Recently, the DRI has been refined to include rare diseases and improve MDS stratification by blast percentage and response to prior therapy. Previous reports on DRI include only a small number of UCBT recipients. The current study aims to determine the ap...
Source: British Journal of Haematology - Category: Hematology Authors: Tags: Research Paper Source Type: research
We report a male patient diagnosed with indolent SM associated with MM. He did not require the therapy for his SM, but started the treatment against MM. He received the induction regiment consisting of bortezomibe, thalidomide and dexamethasone (VTD). After six cycles of VTD he achieved a very good partial response, but refused autologous stem cell transplantation as response consolidation and eventually died of myeloma progression a couple months later. Herein we discuss the likely pathophysiologic mechanisms underlying those two separate entities.
Source: Acta Haematologica Polonica - Category: Hematology Source Type: research
Bone Marrow Transplantation, Published online: 9 October 2017; doi:10.1038/bmt.2017.215
Source: Bone Marrow Transplantation - Category: Hematology Authors: Source Type: research
AbstractChronic myelomonocytic leukemia (CMML) is a myeloid malignancy which shares clinical and morphologic features of myelodysplastic syndromes (MDS) and myeloproliferative neoplasms (MPNs) and is classified by the WHO as an MDS/MPN. The defining feature of CMML is clonal hematopoiesis that results in peripheral monocytosis. The benefit of early treatment is currently unclear, and treatment may be held until the disease exhibits accelerated blast counts or the patient becomes symptomatic. Optimal treatments for CMML are not well defined. Conventional treatments include hydroxyurea, cytarabine, and hypomethylating agents...
Source: Current Hematologic Malignancy Reports - Category: Hematology Source Type: research
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Source: Transplant Infectious Disease - Category: Transplant Surgery Authors: Tags: Case Report Source Type: research
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