From the Division of Immunology (M.B.F.S.), and the Departments of Cardiology (K.F., J.W.N.) and Anesthesiology, Critical Care, and Pain Medicine (C.C.Y., M.M.N., A.G.R.), Boston Children's Hospital, the Division of Pediatric Critical Care Medicine, MassGeneral Hospital for Children (P.H.Y.), and the Departments of Anesthesia (A.G.R.) and Pediatrics (M.B.F.S., K.F., P.H.Y., J.W.N., A.G.R.), Harvard Medical School - all in Boston; the COVID-19 Response Team, Centers for Disease Control and Prevention (N.M., L.R.F., C.E.R., M.M.P.), and the Division of Critical Care Medicine, Department of Pediatrics, Emory University School of Medicine, Children's Healthcare of Atlanta (K.M.T.) - both in Atlanta; the Commissioned Corps of the U.S. Public Health Service, Rockville (L.R.F., M.M.P.), and the Department of Anesthesiology and Critical Care Medicine, Division of Pediatric Anesthesiology and Critical Care Medicine, Johns Hopkins School of Medicine, Baltimore (B.J.R.) - both in Maryland; the Section of Critical Care Medicine, Department of Pediatrics, Baylor College of Medicine, Houston (L.L.L.); the Department of Pediatrics, Division of Critical Care Medicine, University of Texas Southwestern, Children's Medical Center of Dallas, Dallas (M.M.); the Pediatric Critical Care Division, Maria Fareri Children's Hospital at Westchester Medical Center and New York Medical College, Valhalla (A.R.S.), the Division of Pediatric Infectious Diseases, Department of Pediatrics, New York University Grossman School of Medicine, New York (V.L.S.), and the Division of Pediatric Critical Care, Department of Pediatrics, State University of New York Downstate Health Sciences University (S.D.), and Pediatric Critical Care, New York City Health and Hospitals, Kings County Hospital (M.A.K.), Brooklyn - all in New York; the Department of Pediatrics, Division of Pediatric Critical Care Medicine, Central Michigan University, Detroit (S.M. Heidemann); the Division of Critical Care, Department of Anesthesiology and Critical Care, University of Pennsylvania Perelman School of Medicine, Philadelphia (J.C.F.); the Division of Pediatric Critical Care Medicine, Department of Pediatrics, University of Alabama at Birmingham, Birmingham (M.K.); the Department of Pediatrics, Division of Critical Care, Yale University School of Medicine, New Haven (J.S.G.), and the Division of Critical Care, Connecticut Children's, Hartford (C.L.C.) - both in Connecticut; the Division of Pediatric Critical Care, M Health Fairview University of Minnesota Masonic Children's Hospital, Minneapolis (J.R.H.); the Department of Pediatrics, Department of Microbiology, Division of Infectious Diseases, University of Mississippi Medical Center, Jackson (C.V.H.); the Division of Pediatric Infectious Diseases, Department of Pediatrics, Children's Mercy Kansas City, Kansas City, MO (J.E.S.); the Department of Pediatrics, Joseph M. Sanzari Children's Hospital at Hackensack University Medical Center, Hackensack (K.N.C.), and the Department of Pediatrics, Division of Pediatric Critical Care, Bristol-Myers Squibb Children's Hospital at Robert Wood Johnson Medical School, Rutger's University, New Brunswick (S.M. Horwitz) - both in New Jersey; the Division of Critical Care Medicine, Department of Pediatrics, Nationwide Children's Hospital, Columbus, OH (M.W.H.); the Department of Pediatrics, Division of Pediatric Critical Care Medicine, University of Washington, Seattle (L.S.S.); the Department of Pediatrics, University of North Carolina Children's Hospital, Chapel Hill (S.P.S.); the Section of Pediatric Critical Care, Department of Pediatrics, Arkansas Children's Hospital, Little Rock (K.I.); the Department of Pediatrics, Division of Cardiology, Louisiana State University Health Sciences Center and Children's Hospital of New Orleans, New Orleans (T.T.B.); the Department of Pediatrics, Section of Critical Care Medicine, University of Colorado School of Medicine and Children's Hospital Colorado, Aurora (A.B.M.); the Division of Pediatric Critical Care, Miller Children's and Women's Hospital of Long Beach, Long Beach (C.J.B.), and the Division of Critical Care Medicine, University of California San Francisco Benioff Children's Hospital Oakland, Oakland (N.Z.C.) - both in California; the Division of Pediatric Critical Care Medicine, Department of Pediatrics, Indiana University School of Medicine, Riley Hospital for Children, Indianapolis (C.M.R.); the Division of Pediatric Critical Care Medicine, Department of Pediatrics, University of Miami Miller School of Medicine, Miami (G.E.M.); the Division of Pediatric Critical Care Medicine, Medical University of South Carolina, Charleston (E.H.M.); the Department of Pediatrics, University of Louisville and Norton Children's Hospital, Louisville, KY (V.L.M.); the Division of Pediatric Infectious Diseases, Department of Pediatrics, Vanderbilt University Medical Center, Nashville (N.B.H.); and the Division of Critical Care Medicine, Department of Pediatrics, Northwestern University Feinberg School of Medicine, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago (B.M.C.).
N Engl J Med. 2021 Jul 1;385(1):23-34. doi: 10.1056/NEJMoa2102605. Epub 2021 Jun 16.
BACKGROUND: The assessment of real-world effectiveness of immunomodulatory medications for multisystem inflammatory syndrome in children (MIS-C) may guide therapy. METHODS: We analyzed surveillance data on inpatients younger than 21 years of age who had MIS-C and were admitted to 1 of 58 U.S. hospitals between March 15 and October 31, 2020. The effectiveness of initial immunomodulatory therapy (day 0, indicating the first day any such therapy for MIS-C was given) with intravenous immune globulin (IVIG) plus glucocorticoids, as compared with IVIG alone, was evaluated with propensity-score matching and inverse probability weighting, with adjustment for baseline MIS-C severity and demographic characteristics. The primary outcome was cardiovascular dysfunction (a composite of left ventricular dysfunction or shock resulting in the use of vasopressors) on or after day 2. Secondary outcomes included the components of the primary outcome, the receipt of adjunctive treatment (glucocorticoids in patients not already receiving glucocorticoids on day 0, a biologic, or a second dose of IVIG) on or after day 1, and persistent or recurrent fever on or after day 2. RESULTS: A total of 518 patients with MIS-C (median age, 8.7 years) received at least one immunomodulatory therapy; 75% had been previously healthy, and 9 died. In the propensity-score-matched analysis, initial treatment with IVIG plus glucocorticoids (103 patients) was associated with a lower risk of cardiovascular dysfunction on or after day 2 than IVIG alone (103 patients) (17% vs. 31%; risk ratio, 0.56; 95% confidence interval [CI], 0.34 to 0.94). The risks of the components of the composite outcome were also lower among those who received IVIG plus glucocorticoids: left ventricular dysfunction occurred in 8% and 17% of the patients, respectively (risk ratio, 0.46; 95% CI, 0.19 to 1.15), and shock resulting in vasopressor use in 13% and 24% (risk ratio, 0.54; 95% CI, 0.29 to 1.00). The use of adjunctive therapy was lower among patients who received IVIG plus glucocorticoids than among those who received IVIG alone (34% vs. 70%; risk ratio, 0.49; 95% CI, 0.36 to 0.65), but the risk of fever was unaffected (31% and 40%, respectively; risk ratio, 0.78; 95% CI, 0.53 to 1.13). The inverse-probability-weighted analysis confirmed the results of the propensity-score-matched analysis. CONCLUSIONS: Among children and adolescents with MIS-C, initial treatment with IVIG plus glucocorticoids was associated with a lower risk of new or persistent cardiovascular dysfunction than IVIG alone. (Funded by the Centers for Disease Control and Prevention.).
背景：评估免疫调节药物在儿童多系统炎症综合征（MIS-C）中的实际疗效，可能有助于指导治疗。
方法：我们分析了 2020 年 3 月 15 日至 10 月 31 日期间，年龄在 21 岁以下且因 MIS-C 入住美国 58 家医院之一的住院患者的监测数据。采用倾向性评分匹配和逆概率加权法，比较静脉注射免疫球蛋白（IVIG）加糖皮质激素与 IVIG 单药治疗的初始免疫调节治疗（第 0 天，即首次使用任何治疗 MIS-C 的免疫调节药物的日期）效果，调整基线 MIS-C 严重程度和人口统计学特征。主要结局为第 2 天或之后出现心血管功能障碍（左心室功能障碍或休克导致使用血管加压药的复合结局）。次要结局包括主要结局的组成部分、第 1 天或之后接受辅助治疗（第 0 天未接受糖皮质激素的患者给予糖皮质激素、生物制剂或第二剂 IVIG）、第 2 天或之后持续或复发热。
结果：518 例 MIS-C 患儿（中位年龄 8.7 岁）至少接受了一种免疫调节治疗；75%患儿既往健康，9 例死亡。在倾向性评分匹配分析中，与 IVIG 单药治疗（103 例）相比，IVIG 加糖皮质激素（103 例）初始治疗后第 2 天或之后发生心血管功能障碍的风险较低（17% vs. 31%；风险比，0.56；95%置信区间[CI]，0.34 至 0.94）。接受 IVIG 加糖皮质激素治疗的患儿，复合结局组成部分的风险也较低：分别有 8%和 17%的患儿出现左心室功能障碍（风险比，0.46；95%CI，0.19 至 1.15），分别有 13%和 24%的患儿出现休克导致使用血管加压药（风险比，0.54；95%CI，0.29 至 1.00）。与接受 IVIG 单药治疗的患儿相比，接受 IVIG 加糖皮质激素治疗的患儿使用辅助治疗的比例较低（34% vs. 70%；风险比，0.49；95%CI，0.36 至 0.65），但发热风险无差异（分别为 31%和 40%；风险比，0.78；95%CI，0.53 至 1.13）。逆概率加权分析结果与倾向性评分匹配分析结果一致。
结论：在 MIS-C 患儿中，与 IVIG 单药治疗相比，IVIG 加糖皮质激素治疗初始治疗后新发或持续心血管功能障碍的风险较低。（由美国疾病控制与预防中心资助）。
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