Department of Rheumatology and Immunology, Shenzhen Children's Hospital, 7019 Yitian Road, Shenzhen, China.
Pediatr Rheumatol Online J. 2024 Sep 27;22(1):87. doi: 10.1186/s12969-024-01023-w.
Cryopyrin-associated periodic syndrome (CAPS) is characterized by excessive IL-1β release resulting in systemic and organ inflammation. As an anti-IL-1 agent, canakinumab has been approved with all CAPS phenotypes in USA and European countries. However, the use of canakinumab in CAPS in Chinese patients was rarely reported. In this study, we aimed to assess the effectiveness and safety of canakinumab in Chinese patients with CAPS.
Patients with CAPS treated with canakinumab were included. Clinical data were collected retrospectively from medical records. Treatment response was evaluated by CAPS disease activity score, C-reactive protein (CRP), and/or serum amyloid A (SAA) levels. Data was analyzed at canakinumab initiation, at months 1, 3, 6, 9, and 12, or the last follow-up.
A total of 10 CAPS patients were included. 40% of patients were males, the median age at disease onset was 2.5 (2.5, 6) days and the median duration of follow-up while on canakinumab was 22.5 (8.5, 27.5) months. 80% (8/10) of CAPS patients presented with moderate-severe disease activity before the canakinumab treatment. 30% (3/10) of patients required canakinumab dose increase to control disease activity. After treatments, 60% (6/10) of CAPS patients achieved complete remission without relapse and the rest showed minimal disease activity. Clinical symptoms such as fever and rash were improved significantly in most patients (80%). Although abnormal imaging in brain MRI remained in over half of those patients, neurological manifestations were all relieved. 60% (6/10) of patients received prednisone before starting canakinumab therapy and five of them discontinued prednisone later. The most common adverse event was infection (40%). No serious adverse events occurred during the treatment of canakinumab.
Canakinumab may be effective and tolerable for Chinese CAPS patients, helping to reduce the dosage of corticosteroids. However, additional trials on large samples are required to further evaluate its efficacy and safety in China.
细胞浆型嗜中性粒细胞胞浆抗体相关周期性综合征（CAPS）的特征为过度释放白细胞介素-1β（IL-1β），从而导致全身和器官炎症。卡那单抗作为一种抗 IL-1 制剂，已在美国和欧洲国家获得批准，可用于所有 CAPS 表型。然而，卡那单抗在中国 CAPS 患者中的应用鲜有报道。本研究旨在评估卡那单抗在中国 CAPS 患者中的疗效和安全性。
纳入接受卡那单抗治疗的 CAPS 患者。从病历中回顾性收集临床数据。通过 CAPS 疾病活动评分、C 反应蛋白（CRP）和/或血清淀粉样蛋白 A（SAA）水平评估治疗反应。在开始使用卡那单抗时、第 1、3、6、9 和 12 个月或最后一次随访时进行数据分析。
共纳入 10 例 CAPS 患者。40%的患者为男性，发病中位年龄为 2.5（2.5，6）天，卡那单抗治疗中位随访时间为 22.5（8.5，27.5）个月。80%（8/10）的 CAPS 患者在接受卡那单抗治疗前表现为中重度疾病活动。30%（3/10）的患者需要增加卡那单抗剂量以控制疾病活动。治疗后，60%（6/10）的 CAPS 患者达到完全缓解且无复发，其余患者疾病活动度轻微。大多数患者（80%）的发热和皮疹等临床症状显著改善。尽管半数以上患者的脑 MRI 异常仍存在，但神经表现均得到缓解。60%（6/10）的患者在开始卡那单抗治疗前接受了泼尼松治疗，其中 5 例后来停用了泼尼松。最常见的不良反应是感染（40%）。在卡那单抗治疗期间未发生严重不良事件。
卡那单抗可能对中国 CAPS 患者有效且耐受，有助于减少皮质类固醇的剂量。然而，需要更大样本量的试验进一步评估其在中国的疗效和安全性。
