Oshiro Hiroko, Goi Kumiko, Akahane Koshi, Inukai Takeshi, Sugita Kanji
Department of Pediatrics, School of Medicine, University of Yamanashi, Yamanashi, Japan.
Pediatr Int. 2015 Apr;57(2):e27-9. doi: 10.1111/ped.12522.
A 9-year-old boy with paroxysmal nocturnal hemoglobinuria/aplastic anemia syndrome (PNH/AA) developed hemolytic crisis after receiving immunosuppressive therapy. Eculizumab dramatically relieved the signs and symptoms and then he safely underwent unrelated bone marrow transplantation, suggesting the feasibility and effectiveness of eculizumab before stem cell transplantation in children with PNH/AA in hemolytic crisis.
一名患有阵发性夜间血红蛋白尿/再生障碍性贫血综合征(PNH/AA)的9岁男孩在接受免疫抑制治疗后发生了溶血危象。依库珠单抗显著缓解了体征和症状,随后他安全地接受了非亲缘骨髓移植,这表明依库珠单抗在患有溶血危象的PNH/AA儿童进行干细胞移植前应用具有可行性和有效性。