Myelodysplastic Syndromes
http://www.100md.com
《新英格兰医药杂志》
The myelodysplastic syndromes encompass a heterogeneous group of clonal hematopoietic cell disorders that are characterized by ineffective and dysplastic hematopoiesis, which causes cytopenias and functional defects that frequently involve all three blood-cell lineages. There is also an increased risk of transformation to acute myeloid leukemia. The incidence of the myelodysplastic syndromes increases with age and currently appears to be the most common hematologic malignant condition in elderly persons.
In these disorders, there is a wide spectrum of the morphologic features of dysplasia; there are cytopenias, chromosomal abnormalities, and clinical manifestations, and the prognoses also vary. A variable degree of anemia, typically macrocytic and hyporegenerative, is the most frequent abnormality. In about one third of patients, anemia is the only abnormality in the blood, whereas in the remaining two thirds, neutropenia and thrombocytopenia are also observed. The symptoms and the major causes of death result directly from cytopenias and functional abnormalities of blood cells. Some patients may remain free of symptoms for many years and are incidentally found to have anemia, neutropenia, or thrombocytopenia during a routine checkup, but most patients have clinically significant morbidity and mortality even without progression to acute myeloid leukemia. Although rarely life-threatening, the symptoms of chronic anemia are the most common causes of impaired quality of life among patients with myelodysplastic syndromes. However, other complications due to marrow failure such as infection and hemorrhage, which also produce morbidity, are the leading causes of death. Progression to acute myeloid leukemia occurs in approximately one third of patients, with a higher rate of transformation occurring in patients with secondary myelodysplastic syndrome.
For a number of reasons, including the apparent increase in the incidence of the myelodysplastic syndromes, this group of disorders has recently attracted considerable interest, and substantial progress has been made in the pathophysiology, diagnosis, prognosis, development of new treatments, and hematopoietic stem-cell transplantation. This book provides, in a practice-oriented fashion, a comprehensive review of all the important topics related to the myelodysplastic syndromes. The roster of 10 authors includes renowned researchers and clinicians, and their experience is reflected in their contributions to the book. The text consists of 13 chapters. The first chapter addresses the clinical presentation, with a detailed and well-structured description of symptoms, physical examination, and laboratory findings in the blood and bone marrow; the differential diagnosis of a broad spectrum of conditions presenting similar findings is discussed in detail in another chapter. A third chapter covers causes and epidemiology and provides a complete analysis of the most recent epidemiologic studies and the factors that are potentially involved in causing the myelodysplastic syndromes. The fourth chapter summarizes the biologic mechanisms implicated in the pathophysiology of the myelodysplastic syndromes, including genomic instability, epigenetic changes, abnormal apoptosis machinery, immune dysregulation, abnormal signal-transduction pathways, and the role of the bone marrow microenvironment. Two superb chapters address exhaustively but schematically all issues related to the diagnosis, classification, and staging of the myelodysplastic syndromes, including cytomorphology and cytochemistry, multiparameter flow cytometry, cytogenetics, and molecular genetics. A series of chapters dealing with therapy are preceded by a chapter that includes a critical analysis of recently published material on the response criteria of the International Working Group and the guidelines for the management of the myelodysplastic syndromes.
Apart from chemotherapy, which for some reason is not covered in the book, the role of supportive care, hematopoietic growth factors, biology-based treatment, and hematopoietic stem-cell transplantation are all exhaustively reviewed. Finally, there is also a chapter that presents a timely overview of the peculiarities of the myelodysplastic syndromes in children, and the book ends with a chapter on the emerging problem of second malignant diseases after chemotherapy and hematopoietic stem-cell transplantation. All of these chapters are very informative and contain carefully compiled lists of references. The contributors have fully achieved their stated goal of providing students, physicians, and other health care professionals with comprehensive, up-to-date information on the myelodysplastic syndromes.
Miguel A. Sanz, M.D., Ph.D.
Hospital Universitario La Fe
46009 Valencia, Spain
msanz@uv.es((Hematologic Malignancies)
In these disorders, there is a wide spectrum of the morphologic features of dysplasia; there are cytopenias, chromosomal abnormalities, and clinical manifestations, and the prognoses also vary. A variable degree of anemia, typically macrocytic and hyporegenerative, is the most frequent abnormality. In about one third of patients, anemia is the only abnormality in the blood, whereas in the remaining two thirds, neutropenia and thrombocytopenia are also observed. The symptoms and the major causes of death result directly from cytopenias and functional abnormalities of blood cells. Some patients may remain free of symptoms for many years and are incidentally found to have anemia, neutropenia, or thrombocytopenia during a routine checkup, but most patients have clinically significant morbidity and mortality even without progression to acute myeloid leukemia. Although rarely life-threatening, the symptoms of chronic anemia are the most common causes of impaired quality of life among patients with myelodysplastic syndromes. However, other complications due to marrow failure such as infection and hemorrhage, which also produce morbidity, are the leading causes of death. Progression to acute myeloid leukemia occurs in approximately one third of patients, with a higher rate of transformation occurring in patients with secondary myelodysplastic syndrome.
For a number of reasons, including the apparent increase in the incidence of the myelodysplastic syndromes, this group of disorders has recently attracted considerable interest, and substantial progress has been made in the pathophysiology, diagnosis, prognosis, development of new treatments, and hematopoietic stem-cell transplantation. This book provides, in a practice-oriented fashion, a comprehensive review of all the important topics related to the myelodysplastic syndromes. The roster of 10 authors includes renowned researchers and clinicians, and their experience is reflected in their contributions to the book. The text consists of 13 chapters. The first chapter addresses the clinical presentation, with a detailed and well-structured description of symptoms, physical examination, and laboratory findings in the blood and bone marrow; the differential diagnosis of a broad spectrum of conditions presenting similar findings is discussed in detail in another chapter. A third chapter covers causes and epidemiology and provides a complete analysis of the most recent epidemiologic studies and the factors that are potentially involved in causing the myelodysplastic syndromes. The fourth chapter summarizes the biologic mechanisms implicated in the pathophysiology of the myelodysplastic syndromes, including genomic instability, epigenetic changes, abnormal apoptosis machinery, immune dysregulation, abnormal signal-transduction pathways, and the role of the bone marrow microenvironment. Two superb chapters address exhaustively but schematically all issues related to the diagnosis, classification, and staging of the myelodysplastic syndromes, including cytomorphology and cytochemistry, multiparameter flow cytometry, cytogenetics, and molecular genetics. A series of chapters dealing with therapy are preceded by a chapter that includes a critical analysis of recently published material on the response criteria of the International Working Group and the guidelines for the management of the myelodysplastic syndromes.
Apart from chemotherapy, which for some reason is not covered in the book, the role of supportive care, hematopoietic growth factors, biology-based treatment, and hematopoietic stem-cell transplantation are all exhaustively reviewed. Finally, there is also a chapter that presents a timely overview of the peculiarities of the myelodysplastic syndromes in children, and the book ends with a chapter on the emerging problem of second malignant diseases after chemotherapy and hematopoietic stem-cell transplantation. All of these chapters are very informative and contain carefully compiled lists of references. The contributors have fully achieved their stated goal of providing students, physicians, and other health care professionals with comprehensive, up-to-date information on the myelodysplastic syndromes.
Miguel A. Sanz, M.D., Ph.D.
Hospital Universitario La Fe
46009 Valencia, Spain
msanz@uv.es((Hematologic Malignancies)