Pb1866 successful management of a child for paroxysmal nocturnal hemoglobinuria with long-term eculizumab therapy

Background: PNH arises from a genetic mutation of hematopoietic stem cells which leads to the acquired nonmalignant clonal expansion of cells lacking glycosyl phosphatidylinositol‐anchored proteins (GPI‐APs). Lack of GPI‐APs translates into PNH's most significant clinical features: bone marrow failure, intravascular hemolysis and thrombosis. PNH rarely occurs in children and has been reported to have a distinct clinical presentation compared to the adult population. Most of the pediatric patients in the litearture have arisen in a previous setting of bone marrow failure. There are few pediatric case reports presented with reccurrent hemolytic episodes. Aims: There is a paucity of published data on outcomes in children with PNH treated with eculizumab. Therefore, we find it significant to share our experience regarding the use of eculizumab for a pediatric patient, who was diagnosed as PNH at 5.5 years of age and have been managed successful by eculizumab for more than 9 years at Kanuni Sultan Süleyman Education and Research Hospital. Methods: The case is retrospectively evaluated. Results: The patient was diagnosed in March 2008 with immunphenotyping and verified with FLAER method. She had been given blood transfusions, hyperhydration therapy, low‐dose oral prednisolone continuously initially and then during hemolytic attacks. During follow‐up, attacks had become more frequent and recurred at 2‐3 weeks intervals. With the informed consent of the family, eculizumab therapy was commenced in December 2011. She was 9 year old at this time.The dose of eculizumab was set according to the weight of the child as 600 mg/week for 4 week, then 900 mg/every other week parenterally. After introduction of eculizumab, both the frequency and the severity of the attacks had declined significantly. She has been under eculizumab therapy for more than 9 years without any considerable advers effect. Reoccurence of hemolytic attack was observed, when the patient had infections or the treatment was interrupted due to problems in obtining drugs. Summary/Conclusion: Depending on our experience, eculizumab may have a substantial role in controlling hemolytic episodes and preventing thrombotic complications and improving quality of life in children similiar to adults.