Bone marrow failure syndromes constitute a group of inherited and acquired disorders manifested as cytopenia of one on more blood cell lines. Recently, the molecular characterization of many of these disorders has led to a better understanding of the biology and pathophysiology of the diseases and the identification of genes that may serve as molecular targets for novel agents. Several aspects of these syndromes are still obscure and new entities are characterized and added with time to the list, while current therapies such as hypomethylating agents, immunosuppression, and complement inhibitors form the base for the incorporation of emerging agents in the management of these disorders.
Through this Research Topic we aim to present several biological aspects of bone marrow failure, by either presenting new unpublished data or reviewing the available information for special topics of interest, such as myelodysplastic syndrome (MDS), aplastic anemia, paroxysmal nocturnal hemoglobinuria (PNH), hemophagocytosis and others, while at the same time we focus on recent advances in the molecular changes that drive the pathophysiology and guide the efforts for the identification of novel targeted therapies in an ever-evolving field. Integrating molecular and emerging treatment data, we aim to contribute to a better understanding of the pathophysiology and management of bone marrow failure syndromes.
We would like contributors to address themes such as:
(1) the emerging targeted therapies for MDS
(2) the role of hypomethylating agents in hypoplastic MDS
(3) the biology of the microenvironment of bone marrow in MDS and the potential for molecular targeting
(4) the differential diagnosis of aplastic anemia and hypoplastic MDS
(5) the role of molecular techniques in the differential diagnosis of acquired sideroblastic anemias
(6) paroxysmal nocturnal hemoglobinuria and thrombosis: a national multicenter retrospective study (original research article)
(7) Primary or Familial Hemophagocytic Lymphohistiocytosis
(8) Secondary Hemophagocytic Lymphohistiocytosis
(9) the pathophysiology of acquired neutropenia
(10) GATA2 deficiency and other inherited bone marrow failure syndromes
(11) the VEXAS syndrome
Bone marrow failure syndromes constitute a group of inherited and acquired disorders manifested as cytopenia of one on more blood cell lines. Recently, the molecular characterization of many of these disorders has led to a better understanding of the biology and pathophysiology of the diseases and the identification of genes that may serve as molecular targets for novel agents. Several aspects of these syndromes are still obscure and new entities are characterized and added with time to the list, while current therapies such as hypomethylating agents, immunosuppression, and complement inhibitors form the base for the incorporation of emerging agents in the management of these disorders.
Through this Research Topic we aim to present several biological aspects of bone marrow failure, by either presenting new unpublished data or reviewing the available information for special topics of interest, such as myelodysplastic syndrome (MDS), aplastic anemia, paroxysmal nocturnal hemoglobinuria (PNH), hemophagocytosis and others, while at the same time we focus on recent advances in the molecular changes that drive the pathophysiology and guide the efforts for the identification of novel targeted therapies in an ever-evolving field. Integrating molecular and emerging treatment data, we aim to contribute to a better understanding of the pathophysiology and management of bone marrow failure syndromes.
We would like contributors to address themes such as:
(1) the emerging targeted therapies for MDS
(2) the role of hypomethylating agents in hypoplastic MDS
(3) the biology of the microenvironment of bone marrow in MDS and the potential for molecular targeting
(4) the differential diagnosis of aplastic anemia and hypoplastic MDS
(5) the role of molecular techniques in the differential diagnosis of acquired sideroblastic anemias
(6) paroxysmal nocturnal hemoglobinuria and thrombosis: a national multicenter retrospective study (original research article)
(7) Primary or Familial Hemophagocytic Lymphohistiocytosis
(8) Secondary Hemophagocytic Lymphohistiocytosis
(9) the pathophysiology of acquired neutropenia
(10) GATA2 deficiency and other inherited bone marrow failure syndromes
(11) the VEXAS syndrome