Pediatric Stem Cell Transplantation
http://www.100md.com
《新英格兰医药杂志》
Several outstanding books describe the science and clinical practice of hematopoietic stem-cell transplantation (HSCT), but information in these works generally is based on data collected from studies in adult patients. HSCT plays an important role in the treatment of life-threatening disorders, which in children differ, in many respects, from their adult counterparts. Disorders diagnosed mainly in young patients are uncommon and receive little attention in books with a broad focus. Children also differ from adults in their developmental biology, which contributes to the differences in risk and benefit from HSCT. For these reasons, a book devoted to the procedure in children is a valuable addition to the field.
The strength of this book lies in the description of the role of HSCT in the treatment of disorders diagnosed in childhood. In particular, recognized experts provide detailed discussions of such transplantation for the treatment of nonmalignant disorders. There is an excellent review of the various metabolic diseases, which clearly outlines the indications for the procedure and the distinct complications that may be encountered in this patient population. The discussion of inherited syndromes of bone marrow failure provides a thorough overview of the pathophysiology of each disorder and a rational approach to its treatment. This and several other in-depth reviews provide an extremely useful reference for transplantation physicians with sporadic exposure to these relatively uncommon nonmalignant disorders.
More frequently, transplantation physicians treat children with advanced hematologic cancers. The indications for HSCT in pediatric patients differ from those for adults with similar disorders. The role of transplantation in the treatment of acute lymphoblastic leukemia, the most common pediatric cancer and the most common pediatric disorder treated by HSCT, is reviewed thoroughly, with an even-handed discussion of controversial areas such as the degree of benefit of the procedure in a first remission or with alternative donors. The role of transplantation for the treatment of acute myeloid leukemia is discussed less thoroughly. The more uncommon myeloproliferative disorders exemplify the challenge for pediatric hematologists in that these disorders differ in biologic and prognostic characteristics, yet the information in most textbooks focuses on HSCT in adults. The concise discussion of these disorders highlights the studies performed in the pediatric population.
The book is well worth the investment just for the chapter describing the late effects after HSCT. Clearly, this is an area in which primary care providers and specialists need current information to help guide the follow-up care of surviving patients. The extremely thorough review presents detailed information about common and less common late effects, such as endocrine, pulmonary, cardiac, and skeletal disorders. Not only will this review help providers monitor surviving patients, it will also provide information that is useful to hematologists and families when they assess the risks of HSCT.
The basic scientific principles and pathophysiology of allogeneic HSCT are not unique to the pediatric population. Except for the account by Robert A. Good of his distinguished achievements in the field, the discussions of the immunology and pathophysiology are cursory and best left to more encompassing textbooks.
Ann E. Woolfrey, M.D.
Fred Hutchinson Cancer Research Center
Seattle, WA 98109(Paulette Mehta. 486 pp., )
The strength of this book lies in the description of the role of HSCT in the treatment of disorders diagnosed in childhood. In particular, recognized experts provide detailed discussions of such transplantation for the treatment of nonmalignant disorders. There is an excellent review of the various metabolic diseases, which clearly outlines the indications for the procedure and the distinct complications that may be encountered in this patient population. The discussion of inherited syndromes of bone marrow failure provides a thorough overview of the pathophysiology of each disorder and a rational approach to its treatment. This and several other in-depth reviews provide an extremely useful reference for transplantation physicians with sporadic exposure to these relatively uncommon nonmalignant disorders.
More frequently, transplantation physicians treat children with advanced hematologic cancers. The indications for HSCT in pediatric patients differ from those for adults with similar disorders. The role of transplantation in the treatment of acute lymphoblastic leukemia, the most common pediatric cancer and the most common pediatric disorder treated by HSCT, is reviewed thoroughly, with an even-handed discussion of controversial areas such as the degree of benefit of the procedure in a first remission or with alternative donors. The role of transplantation for the treatment of acute myeloid leukemia is discussed less thoroughly. The more uncommon myeloproliferative disorders exemplify the challenge for pediatric hematologists in that these disorders differ in biologic and prognostic characteristics, yet the information in most textbooks focuses on HSCT in adults. The concise discussion of these disorders highlights the studies performed in the pediatric population.
The book is well worth the investment just for the chapter describing the late effects after HSCT. Clearly, this is an area in which primary care providers and specialists need current information to help guide the follow-up care of surviving patients. The extremely thorough review presents detailed information about common and less common late effects, such as endocrine, pulmonary, cardiac, and skeletal disorders. Not only will this review help providers monitor surviving patients, it will also provide information that is useful to hematologists and families when they assess the risks of HSCT.
The basic scientific principles and pathophysiology of allogeneic HSCT are not unique to the pediatric population. Except for the account by Robert A. Good of his distinguished achievements in the field, the discussions of the immunology and pathophysiology are cursory and best left to more encompassing textbooks.
Ann E. Woolfrey, M.D.
Fred Hutchinson Cancer Research Center
Seattle, WA 98109(Paulette Mehta. 486 pp., )