Gene/Protein Disease Symptom Drug Enzyme Compound
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Query: UMLS:C0026986 (myelodysplastic syndrome)
 14,926 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

The myelodysplastic syndromes (MDS) are a group of clonal hematopoietic disorders characterized by bone marrow failure and a risk of progression to acute myelogenous leukemia (AML). A precise diagnosis is critical, because there is overlap between the clinical and laboratory findings of MDS and other malignant and nonmalignant hematologic disorders. Several prognostic scoring systems (IPSS, WPSS, LR-PSS, and IPSS-R) assess a patient's risk of progression to AML and overall survival. Many patients are elderly, so age and comorbidities are an important consideration. Patients with lower-risk disease are treated with growth factors (erythropoietin stimulating agents and/or G-CSF) and immunomodulatory agents (antithymocyte globulin and/or lenalidomide). Patients with higher-risk disease have a higher risk of progression to AML and are treated with hypomethylating agents (azacitidine or decitabine) and allogeneic stem cell transplantation if appropriate. Recent laboratory studies have increased our understanding of the pathophysiology of this disease. Mutations in genes effecting ribosomes, splicing of RNA and epigenetics have been discovered. It is likely that these breakthroughs will lead to newer classes of targeted therapies against this disease.
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PMID:Myelodysplasia: new approaches. 2343 97

Allogeneic hematopoietic stem cell transplantation is the only potentially curative therapeutic option for many malignant and nonmalignant hematologic disorders. Despite this, several factors unfavorably affect the outcome of this procedure and in particular chronic graft-versus-host disease (cGVHD) remains the principal cause of morbidity after allogeneic transplantation. Here we present our experience regarding a patient affected by extensive chronic GVHD (cGVHD) treated only with extracorporeal photopheresis procedure (ECP) as first line treatment. The patient, presenting an high risk myelodysplastic syndrome (MDS), underwent an allogeneic peripheral stem cells transplantation. About 2 months after transplantation she experienced a hematological and clinical relapse of MDS. After reinduction therapy with azacitidine she obtained a second complete remission. Because of the risk of relapse related to a strong immunosuppressant therapy and the previous infectious complication, we decided to start a treatment with ECP alone for cGVHD. After six procedure the patient obtained a complete resolution of all signs and symptoms of the cGVHD. This experience may support the possibility to use only an immunomodulant treatment like ECP for the cGVHD, reducing the risk of complications of prolonged immunosuppressant treatment.
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PMID:Efficacy of photopheresis extracorporeal procedure as single treatment for severe chronic GVHD: a case report. 2349 65