RESUMEN
The bone marrow failure (BMF) syndromes are a group of rare disorders characterized by ineffective hematopoiesis resulting from deficiencies in the hematopoietic stem cell compartment. Although these diseases are typically acquired, some forms (e.g., Fanconi anemia, dyskeratosis congenita, Diamond Blackfan anemia, and Shwachman Diamond syndrome) are inherited. Patients with BMF syndromes can develop peripheral blood cytopenias and pancytopenia, and their disease can ultimately progress to acute myelogenous leukemia (AML). Research around the world is shedding light on the biology of the BMF syndromes, their clinical effects, and novel treatments. The Aplastic Anemia and MDS International Foundation (AAMDSIF) is an independent nonprofit organization whose mission is to help patients and family members cope with BMF syndromes. This report summarizes presentations on the latest scientific discoveries in BMF syndromes from the Sixth International Bone Marrow Failure Disease Scientific Symposium sponsored by AAMDSIF on March 22-23, 2018, in Rockville, Maryland.
Asunto(s)
Anemia Aplásica/terapia , Trastornos de Fallo de la Médula Ósea/terapia , Síndromes Mielodisplásicos/terapia , Anemia Aplásica/diagnóstico , Anemia Aplásica/genética , Trastornos de Fallo de la Médula Ósea/diagnóstico , Trastornos de Fallo de la Médula Ósea/genética , Congresos como Asunto , Humanos , Síndromes Mielodisplásicos/diagnóstico , Síndromes Mielodisplásicos/genética , PronósticoRESUMEN
Patients with acquired and inherited bone marrow failure syndromes (BMFS) have ineffective hematopoiesis due to impairments of the hematopoietic stem cell compartment. Common manifestations of BMFS include varying degrees of peripheral blood cytopenias and, sometimes, progression to acute myelogenous leukemia. Research efforts have been made all over the world to improve understanding of the pathogenesis of these diseases and their clinical implications. The Aplastic Anemia and MDS International Foundation (AAMDSIF) is an independent nonprofit organization whose mission is to help patients and family members cope with BMFS. Here, we summarize recent scientific discoveries in several BMFS that were presented at the fifth International Bone Marrow Failure Disease Scientific Symposium 2016 that AAMDSIF sponsored on March 17-18, 2016, in Rockville, Maryland.
Asunto(s)
Anemia Aplásica/patología , Enfermedades de la Médula Ósea/patología , Hemoglobinuria Paroxística/patología , Anemia Aplásica/terapia , Enfermedades de la Médula Ósea/terapia , Trastornos de Fallo de la Médula Ósea , Células Madre Hematopoyéticas , Hemoglobinuria Paroxística/terapia , Humanos , Síndromes Mielodisplásicos/patología , Síndromes Mielodisplásicos/terapiaRESUMEN
Bone marrow failure syndromes (BMFS) are characterized by a failure of the hematopoietic stem cells to produce adequate blood cells, resulting in either cytopenia (defect in one or more blood cell lineages) or pancytopenia (defect in all blood cell lineages). BMFS can be inherited or acquired. The pathogenesis of these diseases is very heterogeneous. Research efforts have been made all over the world to improve the basic knowledge of these diseases. The Aplastic Anemia and MDS International Foundation (AA&MDSIF) is an independent nonprofit organization whose mission is to help patients and family members cope with BMFS. Here, we summarize novel scientific discoveries in several BMFS that were presented at the 4th International Bone Marrow Failure Disease Scientific Symposium 2014 that AA&MDSIF sponsored on March 27-28, 2014, in Rockville, MD.
Asunto(s)
Anemia Aplásica , Hemoglobinuria Paroxística , Síndromes Mielodisplásicos , Anemia Aplásica/genética , Anemia Aplásica/metabolismo , Anemia Aplásica/patología , Anemia Aplásica/terapia , Enfermedades de la Médula Ósea , Trastornos de Fallo de la Médula Ósea , Congresos como Asunto , Hemoglobinuria Paroxística/metabolismo , Hemoglobinuria Paroxística/patología , Hemoglobinuria Paroxística/terapia , Humanos , Síndromes Mielodisplásicos/genética , Síndromes Mielodisplásicos/metabolismo , Síndromes Mielodisplásicos/patología , Síndromes Mielodisplásicos/terapiaRESUMEN
Aplastic anemia (AA), myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) are rare disorders of bone marrow failure. Once considered distinct entities, these three diseases are now believed to have overlapping pathophysiologies. The Aplastic Anemia and MDS International Foundation, a nonprofit organization that supports patients and families living with bone marrow failure disorders, sponsored a scientific symposium in Bethesda, MD, in March 2012. This report summarizes the symposium presentations by 30 of the world's leading AA, MDS, and PNH researchers on recent findings, current areas of controversy, and recommendations for basic and clinical research to advance the field.
Asunto(s)
Anemia Aplásica , Enfermedades de la Médula Ósea , Hemoglobinuria Paroxística , Síndromes Mielodisplásicos , Investigación Biomédica , Congresos como Asunto , Fundaciones , Humanos , Agencias Internacionales , Sociedades CientíficasRESUMEN
Immunotherapy has become an important part of hematopoietic stem cell (HSC) transplantation and cancer therapy. Regenerative and reparative properties of somatic cell-based therapies hold tremendous promise for repairing injured tissue, preventing and reversing damage to organs, and restoring balance to compromised immune systems. The principles and practices of the diverse aspects of immune therapy for cancer, HSC transplantation and regenerative medicine have many commonalities. This meeting report summarizes a workshop sponsored by the National Heart, Lung and Blood Institute (NHLBI) and Production Assistance for Cellular Therapies (PACT), held on 23-24 April 2009 at the National Institutes of Health (NIH, USA). A series of scientific sessions and speakers highlighted key aspects of the latest scientific, clinical and technologic developments in cell therapy, involving a unique set of cell products with a special emphasis on converging concepts in these fields.