Vivarelli Marina, Colucci Manuela, Bonanni Alice, Verzani Martina, Serafinelli Jessica, Emma Francesco, Ghiggeri Gianmarco
Division of Nephrology and Dialysis, IRCCS-Bambino Gesù Children's Hospital, Rome, Italy.
Division of Nephrology, Dialysis, and Transplantation, IRCCS-Istituto Giannina Gaslini, Genoa, Italy.
Pediatr Nephrol. 2017 Jan;32(1):181-184. doi: 10.1007/s00467-016-3498-y. Epub 2016 Sep 29.
Rituximab, a chimeric anti-CD20 monoclonal antibody, is an effective treatment in steroid-dependent nephrotic syndrome (SDNS). However, some patients develop adverse reactions.
CASE-DIAGNOSIS/TREATMENT: Patient 1, a 14-year-old boy with SDNS since the age of 2, was treated with oral prednisone, cyclosporine A (CsA) and mycophenolate mofetil. A first infusion of rituximab at age 12 years was well tolerated, but this was followed by a prolonged relapse unresponsive to oral prednisone, mycophenolate mofetil and CsA. A second rituximab infusion was attempted, but treatment was interrupted due to severe dyspnea. Treatment with a humanized anti-CD20 monoclonal antibody, ofatumumab, was then attempted. The patient experienced a mild allergic reaction and maintained remission despite interruption of all treatment at >12 months of follow-up. Patient 2, a 3-year-old boy who presented at 18 months with nephrotic syndrome initially resistant to treatment with oral prednisone, was given with three intravenous boluses of methylprednisolone followed by CsA and achieved remission. Upon steroid discontinuation, the NS relapsed. Prednisone was restarted and treatment with a single dose of rituximab was never completed due to a severe allergic reaction. Ofatumumab infusion was uneventful, and he maintained remission during the follow-up period (>12 months) despite interruption of prednisone therapy. B cells reappeared at 7 months in both patients.
Ofatumumab may be a therapeutic option in severe forms of NS with allergy to rituximab.
利妥昔单抗是一种嵌合抗CD20单克隆抗体,是治疗激素依赖型肾病综合征(SDNS)的有效药物。然而,一些患者会出现不良反应。
病例诊断/治疗:患者1,一名自2岁起患SDNS的14岁男孩,接受了口服泼尼松、环孢素A(CsA)和霉酚酸酯治疗。12岁时首次输注利妥昔单抗耐受性良好,但随后出现对口服泼尼松、霉酚酸酯和CsA无反应的长期复发。尝试第二次输注利妥昔单抗,但因严重呼吸困难中断治疗。随后尝试使用人源化抗CD20单克隆抗体奥法木单抗治疗。患者出现轻度过敏反应,尽管在随访超过12个月时中断了所有治疗,但仍维持缓解状态。患者2,一名18个月时出现肾病综合征且最初对口服泼尼松治疗耐药的3岁男孩,接受了三次静脉推注甲泼尼龙,随后使用CsA并实现缓解。停用类固醇后,肾病综合征复发。重新开始使用泼尼松,由于严重过敏反应,单剂量利妥昔单抗治疗未完成。奥法木单抗输注顺利,尽管泼尼松治疗中断,但他在随访期间(超过12个月)维持缓解状态。两名患者在7个月时B细胞均再次出现。
对于对利妥昔单抗过敏的严重形式的肾病综合征,奥法木单抗可能是一种治疗选择。