背景:原卟啉IX(PPIX)是血红素的最终前体,当铁插入时形成血红素。红细胞生成原卟啉病(EPP)的个体有PPIX的积累,导致光敏性和增加肝脏疾病的风险。许多人还患有缺铁和贫血。我们调查了EPP患者口服铁补充剂的结果。
方法:系统评价确定EPP患者口服铁补充剂的文献。随后,我们对缺铁EPP患者给予铁补充剂.主要结果是对PPIX水平的影响。次要结果是不良事件以及血红蛋白和铁参数的相对差异。
结果:系统评价发现13例病例报告和1例结果不确定的非对照临床试验。来自我们部门的10例EPP和铁缺乏症患者每天服用330mg富马酸亚铁,持续两个月。我们的5名患者在基线时出现贫血。补充2个月后,与基线相比,7例患者的PPIX水平升高,两个减少了,一个保持不变。铁的管理导致了铁蛋白的上升,在四名贫血患者中,血液血红蛋白也有所改善。在补充期间观察到血浆丙氨酸转氨酶浓度短暂升高。
结论:总体而言,EPP患者补充铁可以补充铁储备和升高的红细胞PPIX和血浆丙氨酸转氨酶。对于贫血患者,血红蛋白水平有一定程度的正常化.如果EPP患者需要铁治疗,光敏性监测,PPIX,血红蛋白,和血浆肝酶是可取的。
BACKGROUND: Protoporphyrin IX (PPIX) is the final precursor of heme, forming heme when iron is inserted. Individuals with erythropoietic protoporphyrias (EPP) have accumulation of PPIX, causing photosensitivity and increased liver disease risk. Many also have iron deficiency and anemia. We investigated outcomes of oral iron supplements in individuals with EPP.
METHODS: A systematic review identified literature on oral iron supplements in EPP patients. Subsequently, we administered iron supplements to EPP patients with iron deficiency. The primary outcome was impact on PPIX level. Secondary outcomes were adverse events and relative differences in hemoglobin and iron parameters.
RESULTS: The systematic review found 13 case reports and one uncontrolled clinical trial with uncertain results. From our department 10 patients with EPP and iron deficiency took daily dosages of 330 mg of ferrous fumarate for two months. Five of our patients had anemia at baseline. After 2 months of supplementation seven patients had increased PPIX level compared to baseline, two had decrease, one remained unchanged. The administration of iron led to a rise in ferritin, and in four of the anemic patients also to an improvement in blood hemoglobin. A small transiently elevation in plasma alanine transaminase concentration was observed during supplementation.
CONCLUSIONS: Overall, iron supplementation in EPP patients replenished iron stores and elevated erythrocyte PPIX and plasma alanine transaminase. For anemic patients, there was some degree of normalization of the hemoglobin level. If iron therapy is needed for EPP patients, monitoring of photosensitivity, PPIX, hemoglobin, and plasma liver enzymes is advisable.