Dickerhoff R, Kremens B
Johanniter Kinderklinik, St. Augustin.
Klin Padiatr. 1994 Jul-Aug;206(4):296-8. doi: 10.1055/s-2008-1046617.
Sickle cell disease is a hemoglobin disorder which is characterized by live-threatening organ damage, both acute and chronic. It can be cured by bone marrow transplantation (BMT), mortality and severe morbidity being 10-15%. Severity of clinical manifestations in sickle cell disease varies considerably from patient to patient. As there are as yet no reliable criteria to predict a patient's individual course there is a dilemma when it comes to selecting patients for BMT. The presently existing protocols for BMT in sickle cell patients, Belgian, American and a British, are selecting patients who already had severe clinical problems without, however, showing signs of irreversible organ damage. As the number of sickle cell patients in Germany is very small (ca. 250) we plan to join one of the existing BMT studies.
镰状细胞病是一种血红蛋白紊乱疾病,其特征是存在危及生命的急慢性器官损伤。它可通过骨髓移植(BMT)治愈,死亡率和严重发病率为10 - 15%。镰状细胞病临床表现的严重程度在患者之间差异很大。由于目前尚无可靠标准来预测患者的个体病程,因此在选择进行骨髓移植的患者时存在两难困境。目前比利时、美国和英国针对镰状细胞病患者的骨髓移植方案,选择的是那些已经出现严重临床问题但尚未表现出不可逆器官损伤迹象的患者。由于德国镰状细胞病患者数量非常少(约250例),我们计划加入现有的一项骨髓移植研究。