Ajmi Houda, Mabrouk Sameh, Hassayoun Saida, Regaieg Haifa, Tfifha Minyar, Jalel Chemli, Skouri Hadef, Zouari Noura, Abroug Saoussan
Pediatrics Department, Sahloul Teaching Hospital, 4054, Sousse, Tunisia.
Hematology Department, Farhat Hached Teaching Hospital, 4031, Sousse, Tunisia.
J Med Case Rep. 2017 Nov 14;11(1):321. doi: 10.1186/s13256-017-1449-2.
Autoimmune hemolytic anemia is rare in children. First-line therapies for this disease consist of corticosteroids and intravenously administered immunoglobulin that are effective in most patients. However, a small proportion of cases (5 to 10%) is refractory to these therapies and may represent a medical emergency, especially when hemolysis is due to warm immunoglobulin M. Recently, reports of the use of rituximab in adult autoimmune diseases have shown promising results. In children, there are few studies on the use of rituximab in the treatment for autoimmune hemolytic anemia, especially on its long-term efficacy and adverse effects.
Here, we report the case of a 10-year-old Tunisian girl with refractory acute autoimmune hemolytic anemia caused by warm-reactive immunoglobulin A, immunoglobulin G, immunoglobulin M, and C3d autoantibodies. First-line treatments using corticosteroids and intravenously administered immunoglobulin were ineffective in controlling her severe disease. On the other hand, she was successfully treated with rituximab. In fact, her hemolytic anemia improved rapidly and no adverse effects were observed.
The case that we report in this paper shows that rituximab could be an alternative therapeutic option in severe acute autoimmune hemolytic anemia with profound hemolysis refractory to conventional treatment. Moreover, it may preclude the use of plasmapheresis in such an urgent situation with a sustained remission.
自身免疫性溶血性贫血在儿童中较为罕见。该疾病的一线治疗方法包括皮质类固醇和静脉注射免疫球蛋白，大多数患者使用这些方法有效。然而，一小部分病例（5%至10%）对这些治疗方法无效，可能构成医疗紧急情况，尤其是当溶血由温反应性免疫球蛋白M引起时。最近，有关利妥昔单抗用于成人自身免疫性疾病的报道显示出了有前景的结果。在儿童中，关于利妥昔单抗用于治疗自身免疫性溶血性贫血的研究很少，尤其是其长期疗效和不良反应方面。
在此，我们报告一例10岁突尼斯女孩的病例，她患有由温反应性免疫球蛋白A、免疫球蛋白G、免疫球蛋白M和C3d自身抗体引起的难治性急性自身免疫性溶血性贫血。使用皮质类固醇和静脉注射免疫球蛋白的一线治疗未能有效控制她的严重病情。另一方面，她接受利妥昔单抗治疗后获得成功。事实上，她的溶血性贫血迅速改善，且未观察到不良反应。
我们在本文中报告的病例表明，对于传统治疗难治且溶血严重的严重急性自身免疫性溶血性贫血，利妥昔单抗可能是一种替代治疗选择。此外，在这种紧急情况下，它可能避免使用血浆置换并实现持续缓解。
