Transplantation for congenital bone marrow failure syndromes.

Bone Marrow Research Pub Date : 2011-01-01 Epub Date: 2010-11-29 DOI:10.1155/2011/849387
Kenji Morimoto, Theodore B Moore, Gary Schiller, Kathleen M Sakamoto
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引用次数: 2

Abstract

Congenital bone marrow failure syndromes (BMFSs) are relatively rare disorders characterized by aberrant development in one or more hematopoietic lineages. Genetic alterations have now been identified in most of these disorders although the exact role of the molecular defects has yet to be elucidated. Most of these diseases are successfully managed with supportive care, however, treatment refractoriness and disease progression-often involving malignant transformation-may necessitate curative treatment with hematopoietic stem cell transplantation. Due to the underlying molecular defects, the outcome of transplantation for BMFS may be dramatically different than those associated with transplantation for more common diseases, including leukemia. Given recent improvements in survival and molecular diagnosis of bone marrow failure syndrome patients presenting at adult ages without physical stigmata, it is important for both pediatric and adult hematologists to be aware of the possible diagnosis of BMF syndromes and the unique approaches required in treating such patients.

移植治疗先天性骨髓衰竭综合征。
先天性骨髓衰竭综合征(BMFSs)是一种相对罕见的疾病,其特征是一个或多个造血谱系的异常发育。尽管分子缺陷的确切作用尚未阐明,但在大多数这些疾病中已经确定了遗传改变。这些疾病大多可以通过支持性治疗成功治疗,然而,治疗难治性和疾病进展(通常涉及恶性转化)可能需要通过造血干细胞移植进行根治性治疗。由于潜在的分子缺陷,BMFS移植的结果可能与更常见疾病(包括白血病)的移植相关的结果显著不同。鉴于近年来无体污的成年期骨髓衰竭综合征患者生存率和分子诊断的改善,儿科和成人血液学家都必须了解BMF综合征的可能诊断以及治疗此类患者所需的独特方法。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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