The 2020 Pediatric Rheumatology Symposium, originally scheduled for April 29 – May 2, was postponed due to COVID-19; therefore, abstracts were not presented as scheduled.
Session Type: ACR Abstract Session
Session Time: 4:15PM-5:15PM
Background/Purpose: Rapid identification of hemophagocytic lymphohistiocytosis (HLH) and macrophage activation syndrome (MAS) coupled with a multidisciplinary approach to management is essential to improve patient outcomes. We describe our experience with a newly instituted Evidence Based Guideline (EBG) for HLH/MAS at Boston Children’s Hospital (BCH).
Methods: A diagnostic and therapeutic algorithm for HLH/MAS was developed at BCH based on literature review and expert opinion. The resulting EBG was activated at BCH in 1/2018 with the goal to review quality metrics after implementation. An electronic medical record (EMR) search algorithm (patients with an oncology and/or rheumatology note, fever ≥38.2C, and ferritin ≥500ng/mL) was developed to retrospectively identify all hospitalized patients in the EBG from 1/2018 to 4/2019. The selected medical records were then reviewed by an attending rheumatologist, and clinical data were entered into a REDCap database. Subjects with a previously known diagnosis of HLH/MAS before hospital admission were excluded.
Results: Twenty-seven patients (12 girls, 15 boys) with an average age of 8.8 years were identified by house staff as potential HLH/MAS patients and rheumatology was consulted for management per the EBG. After rheumatology consult, there was concern for HLH/MAS in 17 patients (63%), and a diagnostic evaluation was initiated. Ultimately, 10 patients were diagnosed with HLH/MAS by the treating team. Of these, 6 met HLH 2004 criteria, and 9 met the 2016 MAS classification criteria for systemic juvenile idiopathic arthritis (sJIA). The following underlying diagnoses were identified in the patients with HLH/MAS: sJIA (n=3), systemic lupus erythematosus (n=1), malignancy (n=1), familial HLH type 2 (n=1), STAT2 deficiency (n=1), and congenital disorder of glycosylation type 2 defect (n=1).
These 10 patients were treated for HLH/MAS according to EBG guidelines. The most common first line treatments were IVIG (n=4), glucocorticoids (n=3), and anakinra (n=3). One patient was treated per the HLH2004 protocol. For treated patients, the average hospital stay was 29.9 days with a mean time from admission to HLH/MAS directed therapy of 3.2 days. Management guided by the EBG led to a mean fever duration of 6.0 days. Ferritin levels decreased by 50% during admission in 6 subjects, and the mean time to this decrease was 5.3 days. Eight patients required higher level care, and mortality was reported in 1 patient during the hospital stay.
Conclusion: Implementation of EBG for HLH and MAS was associated with rapid initiation of HLH/MAS directed therapy (3.2 days after admission) and an overall survival rate of 90%, which compares favorably to historical reports. Further studies are needed to compare these outcomes to those from the pre-EBG implementation period to confirm improved quality of care.
To cite this abstract in AMA style:Hoyt K, Halyabar O, Han J, Case S, Chang M, Cohen E, Dedeoglu F, Gorman M, Hausmann J, Janssen E, Lee P, Lo J, Lo M, Meidan E, Nigrovic P, Roberts J, Son M, Sundel R, Degar B, Hazen M, Henderson L. Outcomes of an Evidence Based Guideline for the Treatment of Hemophagocytic Lymphohistiocytosis and Macrophage Activation Syndrome [abstract]. Arthritis Rheumatol. 2020; 72 (suppl 4). https://acrabstracts.org/abstract/outcomes-of-an-evidence-based-guideline-for-the-treatment-of-hemophagocytic-lymphohistiocytosis-and-macrophage-activation-syndrome/. Accessed September 30, 2022.
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ACR Meeting Abstracts - https://acrabstracts.org/abstract/outcomes-of-an-evidence-based-guideline-for-the-treatment-of-hemophagocytic-lymphohistiocytosis-and-macrophage-activation-syndrome/