Mutations and prognosis in myelodysplastic syndromes: Karyotype ‐adjusted analysis of targeted sequencing in 300 consecutive cases and development of a genetic risk model
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Conclusion: Targeted drug delivery assisted by genomic and proteomic data provides an adaptable model system that can be extended to effectively treat other cancers and diseases.
Allogeneic stem cell transplantation (alloSCT) is a potentially curative therapeutic option for patients with myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML). Disease relapse occurs in 35-45% of patients following alloSCT, and is the most frequent cause of treatment failure and mortality [1-4]. Moreover, relapse after alloSCT is associated with poor prognosis despite salvage chemotherapy, donor lymphocyte infusions, and/or second transplants .
British Journal of Haematology, EarlyView.
Conditions: Myelodysplastic Syndromes; Hematopoietic Cell Transplant Intervention: Behavioral: ePRO survey Sponsors: Center for International Blood and Marrow Transplant Research; Medical College of Wisconsin Not yet recruiting
Authors: Shammo JM, Komrokji RS Abstract INTRODUCTION: Patients with myelodysplastic syndromes (MDS) are at increased risk of iron overload due to ineffective erythropoiesis and chronic transfusion therapy. The clinical consequences of iron overload include cardiac and/or hepatic failure, endocrinopathies, and infection risk. Areas covered: Iron chelation therapy (ICT) can help remove excess iron and ultimately reduce the clinical consequences of iron overload. The authors reviewed recent (last five years) English-language articles from PubMed on the topic of iron overload-related complications and the use of ICT (...
Monosomy 7 (–7) and del(7q) are high-risk cytogenetic abnormalities common in myeloid malignancies. We previously reported that CUX1, a homeodomain-containing transcription factor encoded on 7q22, is frequently inactivated in myeloid neoplasms, and CUX1 myeloid tumor suppressor activity is conserved from humans to Drosophila. CUX1-inactivating mutations are recurrent in clonal hematopoiesis of indeterminate potential as well as myeloid malignancies, in which they independently carry a poor prognosis. To determine the role for CUX1 in hematopoiesis, we generated 2 short hairpin RNA-based mouse models with ~54% (Cux1mi...
tti A PMID: 29903759 [PubMed - as supplied by publisher]
Abstract Accumulating clinical evidence implicates a suppressive effect on hematopoiesis in myelodysplastic syndrome patients with iron overload. However, how iron overload influences hematopoiesis in myelodysplastic syndrome remains unknown. Here, the RUNX1S291fs-transduced bone marrow mononuclear cells were yielded and transplanted into lethally irradiated recipient mice together with radioprotective bone marrow cells to generate myelodysplastic syndrome mice. Eight weeks post transplantation, the recipient mice were administered intraperitoneal injection of 0.2 ml iron dextran at a concentration of 25mg/ml once...
We report the results of a prospective trial of lirilumab in patients with myelodysplastic syndrome(MDS). A total of 10 patients included. Higher-risk patients received lirilumab plus azacitidine, lower-risk received single agent lirilumab. Two patients achieved CR and 5 achieved marrow CR. Although the small sample size precludes definitive conclusions, the results of this study indicate the efficacy and safety of lirilumab in MDS.