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Editorial

Takayasu Arteritis
It Is Time to Work Together
Joshua A. Beckman, MD

I n the United States, the Orphan Drug Act of 1983 defines a


disease as rare if it affects <200 000 people. In the United
States, there are >6800 rare diseases that affect up to 30 mil-
Japan, registration is necessary for financial coverage, sug-
gesting that the vast majority of new patients with the disease
were identified. The variation in diagnostic criteria by nation
lion people. With an estimated incidence of 2 to 3 cases per is important here for interpretation of the data. In Japan, age
million people in the United States,1 Takayasu arteritis (TA) is not a diagnostic criteria for TA, and in this report, a sig-
easily meets this definition. Thus, it suffers from the chal- nificant portion, ≈20%, are >60 years of age. Several impor-
lenges inherent to its limited patient base. tant insights are identified, including the interaction between
time to diagnosis and hypertension, the frequency of head and
Articles see p 1693 and p 1701
neck complaints at presentation, and the confirmation of aortic
TA is a large-vessel vasculitis originally described by a aneurysmal disease in 25% of male patients but only 11% of
Downloaded from http://circ.ahajournals.org/ by guest on February 5, 2017

Japanese ophthalmologist that meets the Orphan Drug Act def- women. To understand the value of this report, it must be made
inition in the United State but may be more prevalent in Japan; clear that the group of newly diagnosed patients in this report
autopsy series show evidence of TA in 1 of 3000 people.2 The may be 10-fold larger than in any other similar publication of
disease is one of vascular inflammation with destruction of new patient presentations. These data will further refine our
elastic fibers, vascular smooth muscle, and vessel wall matrix understanding of the presentation of TA, increase awareness
components. Arterial stenosis remains the most common vas- of low-frequency sequelae present in Japanese patients, and
cular presentation; however, aortic aneurysmal disease has provide a pathway for others to follow in data collection.
been reported in as many as one quarter of patients. The varia- The report by Mekinian and colleagues4 demonstrates the
tion by nationality in incidence, clinical characteristics, and hard work necessary to advance disease therapeutics. In this
female/male ratio suggests a genetic component to the disease, retrospective study, the investigators determined the outcome
but no reliable marker has been identified. The most common of TA patients treated with tumor necrosis factor-α antagonists
time of diagnosis is the second and third decades of life. In the or tocilizumab, an interleukin-6 receptor antagonist. Of the 49
United States, the age of the patient is important, for it is one patients reported, 88% were inadequately controlled by stan-
method of distinguishing TA from giant-cell arteritis, which is dard therapy. The response to these agents significantly lowered
commonly diagnosed in the eighth decade of life but has many markers of inflammation, reduced daily prednisone require-
signs and symptoms overlapping those of TA. Patients who ments, tended to reduce vascular complications, and improved
present with symptoms of inflammation or arterial occlusive relapse-free survival to 90% at 3 years. These data affirm the
disease in the fifth and early sixth decades of life present a use of the tumor necrosis factor-α and interleukin-6 receptor
diagnostic challenge in determining the diagnosis. antagonists in TA. Unfortunately, because of the lack of pro-
Despite the yeoman’s work of many in the field, the lim- spective data collection and disposition of treating physicians,
ited patient numbers and modest efforts to accrue larger num- these data carry the burdens of a retrospective trial and cannot
bers of patients across centers have slowed our understanding confirm the value of these agents. Still, the data are reassuring.
of the disease. As with many other rare diseases, the lack of The care of patients with TA and other large-vessel vas-
animal models of disease, lack of large patient numbers in any culitides would benefit from 2 interventions. The first is
one center, and limited clinical development pathway render the development of an ongoing registry to aggregate cases
investigation more difficult and interpretation of small data in the United States. Two diseases provide strong models
sets challenging. It is with this background that the 2 publica- for this: aortic dissection and fibromuscular dysplasia. The
tions in this issue of Circulation are so welcome.
International Registry of Acute Aortic Dissections, organized
Watanabe and colleagues3 have analyzed 1372 newly reg-
in 1996, now has participating centers in >11 nations and has
istered patients with TA collected over 10 years in Japan. In
enrolled nearly 6000 patients with aortic dissection.5 In addi-
tion to significantly improving the understanding of disease
The opinions expressed in this article are not necessarily those of the presentation and outcome, this registry has now embarked on
editors or of the American Heart Association. clinical research to improve disease management with a com-
From Vanderbilt University, Nashville, TN.
Correspondence to Joshua A. Beckman, MD, Vanderbilt University, mitted group of investigators already used to working together.
1215 21st Ave S, Medical Center East, 5th Floor, South Tower, Nashville, A registry for fibromuscular dysplasia was organized in 2007
TN 37232–8802. E-mail joshua.a.beckman@vanderbilt.edu with the first online database patient entry in 2009. Three years
(Circulation. 2015;132:1685-1686.
DOI: 10.1161/CIRCULATIONAHA.115.018895.) later, the group published findings on 447 patients and has
© 2015 American Heart Association, Inc. provided insights into this disease that previously were undis-
Circulation is available at http://circ.ahajournals.org covered.6 Neither of these 2 registries was flush with resources
DOI: 10.1161/CIRCULATIONAHA.115.018895 or funds; what they had were clinician scientists interested in
1685
1686  Circulation  November 3, 2015

understanding disease and improving patient care. In addition, References


they had access to supportive expertise in distributed data col- 1. Creager MA. Takayasu arteritis. Rev Cardiovasc Med. 2001;2:211–214.
lection (Michigan Clinical Outcomes Research and Reporting 2. Hotchi M. Pathological studies on Takayasu arteritis. Heart Vessels Suppl.
1992;7:11–17.
Program for both). TA needs a similar registry. 3. Watanabe Y, Miyata T, Tanemoto K. Current clinical features of new
The second improvement in care is the creation of clini- patients with Takayasu arteritis observed from cross-country research
cal teams to care for these patients. As a vascular medicine in Japan: age and sex specificity. Circulation. 2015;132:1701–1709.
specialist, I have been fortunate to work with a cadre of col- doi: 10.1161/CIRCULATIONAHA.114.012547.
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Kim ES, Mace P, Matsumoto AH, McBane RD, Kline-Rogers E, White
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Disclosures
None. Key Words: Editorials ◼ aneurysm ◼ inflammation ◼ Takayasu arteritis
Takayasu Arteritis: It Is Time to Work Together
Joshua A. Beckman

Circulation. 2015;132:1685-1686; originally published online September 9, 2015;


doi: 10.1161/CIRCULATIONAHA.115.018895
Downloaded from http://circ.ahajournals.org/ by guest on February 5, 2017

Circulation is published by the American Heart Association, 7272 Greenville Avenue, Dallas, TX 75231
Copyright © 2015 American Heart Association, Inc. All rights reserved.
Print ISSN: 0009-7322. Online ISSN: 1524-4539

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