基于循证的指南可改善噬血细胞性淋巴组织细胞增生症和巨噬细胞活化综合征患者的结局。
An Evidence-Based Guideline Improves Outcomes for Patients With Hemophagocytic Lymphohistiocytosis and Macrophage Activation Syndrome.
机构信息
M.L. Taylor, BS, M.T. Chandler, MD, C. Platt, MD, PhD, E.M. Cohen, MD, M. Day-Lewis, RN, MSN, CPNP, F. Dedeoglu, MD, E. Janssen, MD, PhD, P.Y. Lee, MD, PhD, J. Lo, MD, M.S. Lo, MD, PhD, E. Meidan, MD, J.E. Roberts, MD, M.B.F. Son, MD, R.P. Sundel, MD, J.C. Chang, MD, MSCE, O. Halyabar, MD, M.M. Hazen, MD, L.A. Henderson, MD, MMSc, Division of Immunology, Boston Children's Hospital, Boston, Massachusetts.
K.J. Hoyt, MSc, Division of Immunology, Boston Children's Hospital, Boston, Massachusetts, and Virginia Tech Carilion School of Medicine, Roanoke, Virginia.
出版信息
J Rheumatol. 2022 Sep;49(9):1042-1051. doi: 10.3899/jrheum.211219. Epub 2022 Jul 15.
OBJECTIVE
To compare clinical outcomes in children with hemophagocytic lymphohistiocytosis (HLH) and macrophage activation syndrome (MAS) who were managed before and after implementation of an evidence-based guideline (EBG).
METHODS
A management algorithm for MAS-HLH was developed at our institution based on literature review, expert opinion, and consensus building across multiple pediatric subspecialties. An electronic medical record search retrospectively identified hospitalized patients with MAS-HLH in the pre-EBG (October 15, 2015, to December 4, 2017) and post-EBG (January 1, 2018, to January 21, 2020) time periods. Predetermined outcome metrics were evaluated in the 2 cohorts.
RESULTS
After the EBG launch, 57 children were identified by house staff as potential patients with MAS-HLH, and rheumatology was consulted for management. Ultimately, 17 patients were diagnosed with MAS-HLH by the treating team. Of these, 59% met HLH 2004 criteria, and 94% met 2016 classification criteria for MAS complicating systemic juvenile idiopathic arthritis. There was a statistically significant reduction in mortality from 50% before implementation of the EBG to 6% in the post-EBG cohort ( = 0.02). There was a significant improvement in time to 50% reduction in C-reactive protein level in the post-EBG vs pre-EBG cohorts (log-rank < 0.01). There were trends toward faster time to MAS-HLH diagnosis, faster initiation of immunosuppressive therapy, shorter length of hospital stay, and more rapid normalization of MAS-HLH-related biomarkers in the patients post-EBG.
CONCLUSION
While the observed improvements may be partially attributed to advances in treatment of MAS-HLH that have accumulated over time, this analysis also suggests that a multidisciplinary treatment pathway for MAS-HLH contributed meaningfully to favorable patient outcomes.
目的
比较实施循证指南（EBG）前后患有噬血细胞性淋巴组织细胞增生症（HLH）和巨噬细胞活化综合征（MAS）的儿童的临床结局。
方法
我们机构根据文献回顾、专家意见和多个儿科亚专业的共识制定了 MAS-HLH 管理算法。电子病历检索回顾性地确定了 EBG 前（2015 年 10 月 15 日至 2017 年 12 月 4 日）和 EBG 后（2018 年 1 月 1 日至 2020 年 1 月 21 日）住院的 MAS-HLH 患儿。在这两个队列中评估了预定的结局指标。
结果
在 EBG 推出后，住院医师确定了 57 名潜在的 MAS-HLH 患儿，并咨询了风湿病学以进行治疗。最终，治疗团队诊断出 17 名患者患有 MAS-HLH。其中，59%符合 HLH 2004 标准，94%符合 2016 年全身幼年特发性关节炎合并 MAS 的分类标准。在实施 EBG 前死亡率为 50%，而在 EBG 后死亡率为 6%，差异具有统计学意义（=0.02）。与 EBG 前队列相比，在 EBG 后队列中 C 反应蛋白水平降低 50%的时间明显缩短（对数秩检验<0.01）。在 EBG 后，MAS-HLH 诊断时间、开始免疫抑制治疗时间、住院时间以及 MAS-HLH 相关生物标志物正常化速度更快，呈趋势性差异。
结论
尽管观察到的改善可能部分归因于随着时间的推移，对 MAS-HLH 的治疗有所进展，但这项分析还表明，MAS-HLH 的多学科治疗途径对患者结局有重要意义。
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