Phan Phuc Huu, Hoang Canh Ngoc, Nguyen Ha Thu T, Cao Tung Viet, Le Chi Quynh, Tran Dien Minh
Pediatric Intensive Care Unit, Vietnam National Children's Hospital, Ha Noi, Viet Nam
Pediatric Intensive Care Unit, Vietnam National Children's Hospital, Ha Noi, Viet Nam.
BMJ Paediatr Open. 2025 Mar 5;9(1):e003148. doi: 10.1136/bmjpo-2024-003148.
As a first-line therapeutic option for multisystem inflammatory syndrome in children (MIS-C) with surging demand, intravenous immunoglobulin (IVIG) is associated with escalating costs and supply shortages, particularly in low-income and middle-income countries. This study compares the effectiveness of methylprednisolone alone versus IVIG combined with methylprednisolone for managing MIS-C.
We conducted a retrospective cohort study from January 2022 to June 2023 at Vietnam National Children's Hospital. We used propensity score matching to compare the short-term outcomes based on immunomodulatory therapy with methylprednisolone alone or IVIG plus methylprednisolone.
We included 391 patients, comprising 255 boys and 136 girls, who fulfilled the MIS-C case definition of the US Centers for Disease Control and Prevention. Most patients (80.8%) received intravenous methylprednisolone monotherapy, and 19.2% were administered IVIG in addition to methylprednisolone. In general, the laboratory values indicative of hyperinflammatory and hyperthrombotic states displayed significant early response within 2-3 days after initial treatment, including white cell count (SE=1.77, p<0.001), NEU (SE=0.76, p=0.03), C reactive protein (SE=-46.51, p<0.001), PLT (SE=38.05, p=0.002), fibrinogen (SE=-0.37, p=0.002), d-dimer (SE=-849.8, p=0.02)); while subsequent improvement in cardiac markers was also observed, with pro-B-type natriuretic peptide (SE=-165.2, p<0.001) on day 5 and troponin I (SE=-0.05, p=0.004) on day 7. After propensity score weighting, there were 41 patients in each treatment group. Notably, there were no significant differences in the incidence of cardiac events between treatment groups regarding left ventricular dysfunction and coronary artery dilation or aneurysms (10.3% vs 20.7%, p=0.074 and 63.4% vs 56.1%, p=0.653, respectively). While the median paediatric intensive care unit length of stay (LOS) and hospital LOS were slightly lengthier in the IVIG and methylprednisolone group compared with those of the methylprednisolone group, these differences were not statistically significant ((5 vs 4, p=0.782) and (9 vs 7, p=0.725), respectively).
Initial treatment with methylprednisolone monotherapy appears not inferior in effectiveness to adjunctive IVIG plus methylprednisolone in MIS-C. Further investigations in randomised controlled trials deserve to be undergone to clarify if IVIG-sparing glucocorticoids are a viable option for achieving favourable outcomes in MIS-C, particularly in resource-limited settings with barriers approaching IVIG therapy.
作为需求激增的儿童多系统炎症综合征（MIS-C）的一线治疗选择，静脉注射免疫球蛋白（IVIG）导致成本不断上升且供应短缺，尤其是在低收入和中等收入国家。本研究比较了单独使用甲泼尼龙与IVIG联合甲泼尼龙治疗MIS-C的有效性。
我们于2022年1月至2023年6月在越南国家儿童医院进行了一项回顾性队列研究。我们使用倾向评分匹配法，比较基于单独使用甲泼尼龙或IVIG加甲泼尼龙的免疫调节治疗的短期结局。
我们纳入了391例患者，其中包括255名男孩和136名女孩，他们符合美国疾病控制与预防中心的MIS-C病例定义。大多数患者（80.8%）接受了静脉注射甲泼尼龙单一疗法，19.2%的患者除甲泼尼龙外还接受了IVIG治疗。总体而言，指示高炎症和高血栓形成状态的实验室指标在初始治疗后2 - 3天内显示出显著的早期反应，包括白细胞计数（标准误=1.77，p<0.001）、中性粒细胞（标准误=0.76，p=0.03）、C反应蛋白（标准误=-46.51，p<0.001）、血小板（标准误=38.05，p=0.002）、纤维蛋白原（标准误=-0.37，p=0.002）、D-二聚体（标准误=-849.8，p=0.02）；同时还观察到心脏标志物随后有所改善，第5天的脑钠肽前体（标准误=-165.2，p<0.001）和第7天的肌钙蛋白I（标准误=-0.05，p=0.004）。经过倾向评分加权后，每个治疗组有41例患者。值得注意的是，治疗组之间在左心室功能障碍、冠状动脉扩张或动脉瘤方面的心脏事件发生率没有显著差异（分别为10.3%对20.7%，p=0.074和63.4%对56.1%，p=0.653）。虽然与甲泼尼龙组相比，IVIG和甲泼尼龙组的儿科重症监护病房住院时间中位数（LOS）和医院住院时间稍长，但这些差异无统计学意义（分别为（5天对4天，p=0.782）和（9天对7天，p=0.725））。
在MIS-C中，初始使用甲泼尼龙单一疗法的有效性似乎并不低于辅助使用IVIG加甲泼尼龙。值得在随机对照试验中进行进一步研究，以明确节省IVIG的糖皮质激素是否是在MIS-C中取得良好结局的可行选择，特别是在接近IVIG治疗存在障碍的资源有限环境中。
