Childhood Takayasu arteritis: disease course and response to therapy

Abstract

Background: Takayasu arteritis (TAK) is a large vessel vasculitis that rarely affects children. Data on childhood TAK are scarce. The aim of this study was to analyze the presenting features, course and outcome of children with TAK, compare efficacy of treatment regimens and identify high-risk factors for adverse outcome. Methods: A single-center cohort study of consecutive children fulfilling the EULAR/PRINTO/PReS criteria for childhood TAK between 1986 and 2015 was performed. Clinical phenotypes, laboratory markers, imaging features, disease course and treatment were documented. Disease activity was assessed using the Pediatric Vasculitis Disease Activity Score at each visit. Outcome: disease flare defined as new symptoms and/or increased inflammatory markers necessitating therapy escalation and/or new angiographic lesions, or death. Analysis: logistic regression tested relevant variables for flare. Kaplan-Meier analyses compared treatment regimens. Results: Twenty-seven children were included; 74% were female, median age at diagnosis was 12.4 years. Twenty-two (81%) children presented with active disease at diagnosis. Treatment regimens included corticosteroids alone (15%), corticosteroids plus methotrexate (37%), cyclophosphamide (19%), or a biologic agent (11%). Adverse outcomes were documented in 14/27 (52%) children: two (7%) died within 6 months of diagnosis, and 13 (48%) experienced disease flares. The 2-year flare-free survival was 80% with biologic treatments compared to 43% in non-biologic therapies (p = 0.03); at last follow-up, biologic therapies resulted in significantly higher rates of inactive disease (p = 0.02). No additional outcome predictor was identified.

Childhood‐onset Takayasu arteritis: A 15‐year experience from a tertiary referral center

“…In our cohort, there was no patient in the last 15 years, who died as a result of Takayasu arteritis and related complications. The mortality rate was between 22.55%‐35% in the surveys before 2000, which decreased below 15% in reports that have been published during the past decade . In a South African study, the deaths were as a result of uncontrolled systemic hypertension and complications related to surgery .…”

“…About one‐quarter of the subjects seem unresponsive to the conventional therapies . Biologic therapies, predominantly anti‐tumor necrosis factor alpha and anti‐interleukin‐6 agents, may have beneficial effects in these particular subjects; however, there has been no randomized therapeutic trial in Takayasu arteritis . Tocilizumab, which has been used in six out of 17 subjects, was the only preferred biologic agent in our clinic.…”

The importance of FDG PET/CT in the diagnostic process of the middle aortic syndrome in a 15-year-old boy patient with suspected systemic vasculitis and final diagnosis of Williams–Beuren syndrome

“…These processes lead to progressive limb or organ dysfunction secondary to ischemia. There is a strong female predominance, with the high prevalence in the 3rd and 4th decades of life, although the beginning of the disease may develop earlier, including in the childhood years [2][3][4][5][6]. The diagnosis is based on clinical signs and typical angiographic abnormalities in the aorta and its major branches after excluding other causes.…”

“…Childhood Takayasu arteritis is the most common large vessel vasculitis in children. The diagnosis is difficult since disease rarely affects children and some patients develop uncommon manifestations and associated diseases that may contribute to the delayed diagnosis [2][3][4][5][6].…”

Reply

“…However, in real life TAK is often a relapsing–remitting disease in many patients, and failures of all available biologic agents are not infrequently seen, resulting in the need to revisit “older” medications. In our recently published single‐center experience , cyclophosphamide was effective in controlling inflammation; however, methotrexate did not seem to control disease activity efficiently in childhood‐onset TAK. Overall, biologic therapies (anti‐TNF, anti–interleukin‐6) resulted in higher rates of inactive disease.…”