Moleiro Ana Filipa, Vilares-Morgado Rodrigo, Alves Gonçalo Coutinho, Rodrigues Mariana, Falcão-Reis Fernando, Magalhães Augusto, Leal Vitor, Meira Jorge
Department of Ophthalmology, Centro Hospitalar e Universitário de São João, Porto, Portugal.
UnIC@RISE, Department of Surgery and Physiology, Faculty of Medicine of the University of Porto, Porto, Portugal.
Case Rep Ophthalmol. 2022 Nov 17;13(3):877-884. doi: 10.1159/000526546. eCollection 2022 Sep-Dec.
Thyroid eye disease (TED) characterizes by inflammation and remodeling of orbital tissues. Although the majority of pediatric TED is mild, some children present progressive and severe disease. The approach to severe disease in this group of patients, especially when noncortico-responsive, is challenging. The purpose of this work was to describe the effective use of tocilizumab as second-line therapy in steroid-resistant pediatric TED. A 13-year-old female with a history of Graves' disease presented with right eye proptosis for at least 8 months associated with mild pain on eye movements and ocular surface complaints. The ophthalmologic evaluation revealed Hertel exophthalmometry readings of 22 mm on the right eye (OD) and 19 mm on the left (OS). The remaining ophthalmic examination was unremarkable. Intravenous methylprednisolone pulses of 500 mg were initiated without any improvement after 4 treatments. Following multidisciplinary team discussion, therapy was switched to monthly tocilizumab injections at 4 mg/kg. Significant reduction of proptosis and resolution of pain and ocular surface complaints were noted immediately after the treatment switch. Exophthalmometry readings after the end of treatment that included 4 tocilizumab injections were 20 mm on OD and 19 mm on OS. No side effects were reported during the entire follow-up. Six months after treatment cessation, the patient remains stable, without any signs of orbitopathy relapse and no ophthalmologic complaints. This is the second case report showing the effectiveness of tocilizumab in pediatric TED and the first one showing its efficacy when steroids fail. Our results support the potential safety and efficacy of this immunosuppressor in children with TED.
甲状腺眼病（TED）的特征是眼眶组织的炎症和重塑。尽管大多数儿童TED病情较轻，但有些儿童会出现进展性和严重性疾病。对于这组患者中的严重疾病，尤其是对皮质类固醇无反应时，治疗方法具有挑战性。这项工作的目的是描述托珠单抗作为二线治疗在对类固醇耐药的儿童TED中的有效应用。一名13岁女性，有格雷夫斯病病史，出现右眼突出至少8个月，伴有眼球运动时轻度疼痛和眼表不适。眼科检查显示，右眼（OD）赫特尔眼球突出计读数为22毫米，左眼（OS）为19毫米。其余眼科检查无异常。开始静脉注射500毫克甲泼尼龙脉冲，4次治疗后无任何改善。经过多学科团队讨论，治疗改为每月注射4毫克/千克托珠单抗。治疗转换后立即注意到眼球突出明显减轻，疼痛和眼表不适症状消失。包括4次托珠单抗注射的治疗结束后，眼球突出计读数OD为20毫米，OS为19毫米。整个随访期间未报告任何副作用。停药6个月后，患者病情稳定，没有任何眼眶病复发迹象，也没有眼科不适症状。这是第二例显示托珠单抗在儿童TED中有效性的病例报告，也是第一例显示其在类固醇治疗失败时疗效的病例报告。我们的结果支持这种免疫抑制剂在儿童TED中的潜在安全性和有效性。
