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Channel: 2020 Pediatric Rheumatology Symposium Archives - ACR Meeting Abstracts
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Cyclophosphamide as Rescue Therapy for Kawasaki Disease with Peripheral Vascular Disease: A Single Center Experience

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Background/Purpose: Kawasaki Disease (KD) is the most common systemic vasculitis affecting children 5 years and younger. With coronary vessel involvement, KD is now the most common cause of acquired heart disease in developed countries. Focus of management is primarily treatment of systemic inflammation, decrease in coronary artery disease and prevention adverse cardiac outcome. As peripheral vessel disease is typically associated with more severe coronary vessel abnormalities, additional therapies are warranted. Rescue treatment includes glucocorticoids and biologic agents (TNF alpha and IL1 blockade). Other medications used include cyclophosphamide (CYC), an alkylating agent that exerts immunomodulatory effects via B and T cell depletion. The use of CYC in KD is typically reserved for those with refractory disease or those with ongoing disease activity despite treatment. Ongoing disease activity can be described as worsening in Z scores noted on echocardiogram as well as worsening or persistent elevations in inflammatory markers. There are no standard recommendations regarding the use of CYC and given its described side effect profile, discussions about its safety and efficacy in KD are warranted.

Methods: With our Institutional Review Board approval, a retrospective review of the electronic health records of patients with Kawasaki Disease with peripheral aneurysms from July 2011 to May 2019. Patients who were treated with cyclophosphamide were included.  Data included demographic, treatment, disease course, and outcome of both coronary and peripheral vessel disease. 

Results: At the time of the study, 7 patients were identified to have peripheral vascular disease. 71% were Hispanics, 57% males, and mean age of 20 months. All patients had significant coronary artery disease, with initial echo of 6 of the patients showing the worse coronary Z score with a mean of 5.73 (range 0.82- 16.31). One patient’s initial echo was excluded as it was done at an outside hospital. In addition to standard IVIG and aspirin, rescue therapy included 2nd IVIG, corticosteroids, anti-TNF, and anti-IL1. With persistent markers of systemic inflammation worsening coronary changes, 3 received IV CYC. Two were female, all Hispanic, ages 9, 15, and 88 months. Mean days of initial fever prior to diagnosis was 11.3 (Range 6-15). Mean Z score of most severely affected coronary artery noted on second echo was 8.78 (range: 1.69 to 15.02) and peripheral vessel involvement included iliac, subclavian, and axillary arteries. Mean cumulative dose of CYC given was 1454.33 mg/m2 (1065 mg). Improvement of the coronary changes was noted in all at most recent echo. No patients developed cytopenias, significant infection, hemorrhagic cystitis or hypogammaglobinemia during CYC therapy. 

Conclusion: The true incidence of extra coronary vascular disease in KD is not well known. As therapy of KD aims to decrease systemic inflammation and prevent coronary artery disease and cardiac outcome, the significance of peripheral vascular disease is not well defined in the consideration of further therapy.  We treated three children with KD with prominent peripheral vessel involvement with cyclophosphamide and noted its efficacy as additional safe immunomodulatory agent.

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