阵发性睡眠性血红蛋白尿症合并缺铁性贫血患者补铁治疗疗效及安全性评价

OBJECTIVE: To evaluate the efficacy and safety of iron supplement in patients who have paroxysmal nocturnal hemoglobinuria (PNH) with iron deficiency. METHODS: We performed analyses on the clinical data of 48 patients who accepted oral and/or intravenous iron treatment. Forty-eight consecutive PNH patients with iron deficiency who visited our hospital between November 2011 and August 2018 were enrolled in the study. RESULTS: Total 30 patients received oral iron; 18 patients received intravenous iron supplements, including 6 who did not respond to oral iron. The median PNH clone size was 90.2% (38.5%–99.9%) in the granulocytes and 69.7% (27.6%–98.1%) in the red blood cells. The response rate was 56% (20/36) in patients who received oral iron, and the hemoglobin concentration increased 21 (10–52) g/L compared to that at baseline. Sixteen out of eighteen (89%) patients responded to intravenous iron; 6 patients who did not respond to oral iron received intravenous iron, and the hemoglobin level of 5 patients increased. Patients exhibited increased LDH levels and deepen urine after iron supplementation; however, no severe adverse events, such as thrombosis and iron-related adverse effects, were noted. CONCLUSION: Iron treatment is safe and effective in increasing the hemoglobin level in PNH patients with iron deficiency; those who did not respond to oral iron could benefit from intravenous iron supplement.