Lane P A
Colorado Sickle Cell Treatment and Research Center, University of Colorado Health Sciences Center, Denver 80262.
Curr Opin Pediatr. 1993 Feb;5(1):74-9. doi: 10.1097/00008480-199302000-00012.
Neonatal screening for hemoglobinopathies, coupled with comprehensive medical care that includes parental education, prophylactic penicillin, and immunizations, has markedly reduced mortality from sickle hemoglobinopathies during infancy and early childhood. However, despite an increased knowledge of pathophysiology, current therapy does little to prevent acute, noninfectious complications such as anemic crises, pain events, or strokes; nor does it reliably prevent or delay the development of chronic organ damage. Recent publications have increased our understanding of factors that contribute to the clinical heterogeneity of sickle hemoglobinopathies, improved modestly our ability to identify and treat acute complications, and provided real hope for the development of potentially curative therapies. Prospective therapeutic trials of hydroxyurea and of bone marrow transplantation have recently begun.
对血红蛋白病进行新生儿筛查，再加上包括对父母进行教育、预防性使用青霉素和免疫接种在内的全面医疗护理，已显著降低了婴儿期和幼儿期镰状血红蛋白病的死亡率。然而，尽管对病理生理学的认识有所增加，但目前的治疗方法在预防急性非感染性并发症（如贫血危象、疼痛发作或中风）方面效果甚微；也不能可靠地预防或延缓慢性器官损伤的发生。最近的出版物增进了我们对导致镰状血红蛋白病临床异质性因素的理解，在一定程度上提高了我们识别和治疗急性并发症的能力，并为开发可能治愈性的疗法带来了真正的希望。羟基脲和骨髓移植的前瞻性治疗试验最近已经开始。
