Vermylen Christiane
Université Catholique de Louvain, Cliniques Universitaires Saint Luc, Avenue Hippocrate 10, B 1200, Brussels, Belgium.
Eur J Pediatr. 2008 Apr;167(4):377-81. doi: 10.1007/s00431-007-0604-y. Epub 2007 Sep 26.
Children with severe chronic hemolytic anemia or congenital erythroblastopenia are transfusion dependent. Long-term transfusion therapy prolongs life but results in a toxic accumulation of iron in the organs. The human body cannot actively eliminate excess iron. Therefore, the use of a chelating agent is required to promote excretion of iron. So far, iron chelation has been done by subcutaneous infusion of deferoxamine given over 10 h, 5-6 days per week. Compliance is poor and chelation often insufficient. Ferritin measurements and sometimes liver biopsies are used to evaluate the iron burden in the body. At the present time, new iron chelators that can be given orally are available. Furthermore, magnetic resonance imaging (MRI) assessment of tissue iron is a noninvasive and highly reproducible method, which is able to quantitate organ iron burden. In conclusion, iron overload can be measured more accurately with noninvasive methods such as MRI. Deferasirox is a once-daily oral therapy for treating transfusional iron overload, which improves patient compliance and quality of life.
患有严重慢性溶血性贫血或先天性红细胞生成减少症的儿童依赖输血治疗。长期输血疗法可延长生命，但会导致铁在器官中有毒性蓄积。人体无法主动清除过量的铁。因此，需要使用螯合剂来促进铁的排泄。到目前为止，铁螯合是通过每周5 - 6天、持续10小时皮下输注去铁胺来进行的。依从性差且螯合作用往往不足。血清铁蛋白测量以及有时进行肝脏活检用于评估体内铁负荷。目前，有可口服的新型铁螯合剂。此外，组织铁的磁共振成像（MRI）评估是一种无创且高度可重复的方法，能够定量器官铁负荷。总之，使用MRI等无创方法可以更准确地测量铁过载。地拉罗司是一种每日一次的口服疗法，用于治疗输血性铁过载，可提高患者依从性和生活质量。
