Authors: Peter N. Malleson MBBS,
MRCPUK, FRCPC1,
Affiliations:
1Peter
Malleson: Professor, Department of Pediatrics, Division of Rheumatology,
2
3Michaela
Sailer-Hoeck, Pediatric Rheumatologist, Clinical Department of Pediatrics,
Clinical Division of General Pediatrics, Medical University of Innsbruck,
Austria
Correspondence to: Peter Malleson
Email address: malleson@interchange.ubc.ca
Introduction
Since the
introduction of the indirect immunofluorescence (IF) test for antinuclear antibodies
(ANA) by Friou in 1957 (1), ordering tests for ANA have become
almost a knee-jerk response to the question “could this patient have a
rheumatic disease?” What is the evidence
that ordering such tests is of any value, and what should be done with a
positive test?
The ANA test in health and disease
Ideally a
test should always be positive in those with a disease, and always negative in
those without a disease. Of course this situation rarely if ever occurs, but to
be useful a test generally needs to have a sensitivity and specificity of at
least 90%. That is at least 9 out of 10 individuals with the disease will have
a positive test and 9 out of 10 individuals without the disease will have a
negative test. Unfortunately the ANA test, whether performed by IF or by an
enzyme linked immunosorbent method, fails to demonstrate these test characteristics.
Part
of the problem is that the test is being used indiscriminately as part of “the
rheumatologic work-up” beloved of family physicians and residents. No test can
sensibly be expected to be accurate in the diagnoses of diseases as different
as juvenile idiopathic arthritis (JIA), rheumatoid arthritis, systemic lupus
erythematosus, scleroderma, and the vasculitides. Yet, in practice, this is
what often seems to be asked of the ANA test. However, even if the test is used
more sensibly to address specific questions such as: “does this child with a
rash and fever have lupus?” or “does this child with a swollen knee have JIA?”,
or “is this child with JIA going to
develop uveitis?”, we would argue that
the ANA test is simply not accurate enough to answer these questions.
A number
of studies have looked at the frequency of positive ANA tests in “healthy”
individuals. A study by Arroyave et al. in 1988 (2)
screened sera from 241 “normal” children, testing for only IgG ANA, using both mouse kidney and human epithelial
cells (HEp-2 cells). The study found a maximum positivity rate of only 2.0% at
the lowest dilutions. However, data from adult studies have found much higher
rates. In an adult study from 15 international laboratories using HEp-2 cells
as substrate (3), ANA positive tests occurred in
31.7% of a putatively normal population at a serum dilution of 1:40, and even
at a dilution of 1:320, 3.3% of the sera were positive. Interestingly the ANA
frequency did not differ significantly across the age range of 20-60 years. The
rate of ANA positivity among blood donors in
It is not
clear why there is such a low frequency of ANA positivity found in the only
childhood study of which we are aware, compared to the much higher frequency
found in most adult studies (of which only two of many are referenced here).
However, the frequency of ANA among clinic populations is more pertinent to our
discussion than the situation in the normal population as ANA tests are usually
performed on children or adults with musculoskeletal or rheumatologic symptoms
or signs.
In 1997 we
evaluated the results of all the ANA tests performed at British Columbia
Children’s Hospital between 1991 and 1995 (6). We found that the ANA test was
positive at a titer of
For lupus,
Mixed Connective Tissue Disease (MCTD) or overlap syndrome the ANA had a very
high sensitivity of 98%, but a very low positive predictive rate of only 10%.
Positive and negative predictive values are affected by the prevalence of the
disease being tested. Therefore one might expect somewhat better predictive
values from a pediatric rheumatology clinic than from a wide population of
unwell children. We concluded from this study that although a negative ANA test
made the diagnosis of lupus or MCTD extremely unlikely, a positive test at even
moderately high titers of 1:160 has little or no diagnostic value.
ANA immunofluorescent staining
As our
laboratory also provided information on the patterns of immunofluorescent
staining, we have also been able to evaluate what further use this information
might provide (previously unpublished data). Of 1369 individual patients sera tested,
445 were ANA positive in children with a known diagnosis; 135 children having a
rheumatic disease (JRA, lupus, MCTD, or juvenile dermatomyositis (JDM)) and 310
without a rheumatic disease.
Homogeneous, mitotic staining
patterns were seen much more commonly in children with a rheumatic disease than
those without (p=0.001). Interestingly, a nucleolar pattern of staining was
seen more commonly in children without a rheumatic disease (p=0.03). This lack
of association of the nucleolar pattern in children with scleroderma
specifically, and rheumatic disease in general, has been commented on
previously (7, 8).
No combination of ANA titer, or staining
pattern was specific for any particular rheumatic disease. The test
combinations with the best positive predictive values for a rheumatic disease
were: i. a titer ≥1:640 with mitotic positive staining or ii. a titer ≥1:640
with a homogenous and mitotic staining pattern; these tests had positive
predictive values of 77% and 72% respectively, but these results were only
slightly higher than the positive predictive value of 69% obtained with a titer
of ≥1:640 alone, ignoring the pattern of staining.
Although
the addition of patterns somewhat increases the specificity and positive
predictive value of the ANA test for a rheumatic disease, it does so at the
expense of both the sensitivity and negative predictive value of the test using
titers alone. The pattern of staining also does not appear to be helpful in
distinguishing between rheumatic diseases. For example, although high titer
homogenous, mitotic positive staining was the most common combination seen in
children with lupus, it was actually found in only 27.3% of ANA positive lupus
patients. This combination was also
found in 12.5% of ANA positive JRA and 15.4% of ANA positive JDM patients. This
lack of specificity of the ANA immunofluorescent pattern has been recognized
previously, both for adults and children (7, 9, 10).
A study by
Parker (10) evaluated the usefulness of
combining ANA titer and pattern; although they felt that knowledge of both
titer and pattern was helpful, they did not calculate specific test
characteristics, and in fact no combination was restricted to any single
rheumatic disease. Our assessment of these data is that the addition of
information about patterns of immunofluorescence does not appear to
significantly improve the utility of the ANA test.
Referrals for positive ANA titers
As part of
a study exploring what precipitated a referral to a pediatric rheumatologist,
McGhee et al. (11) found that children referred, at
least in part, because of a positive ANA test were no more likely to have a
chronic inflammatory disease than children with a negative test.
In another
study from a pediatric rheumatology clinic (12), only 55% of all of the children
with a positive ANA test had an inflammatory rheumatic disease. Positive
antibodies to dsDNA or to extractable nuclear antigens (Sm, RNP) indicating
lupus or MCTD were strongly correlated with an ANA titer ≥1:640. The authors recommended therefore, that these
more specific tests be performed only if the child had a positive ANA test at
high titer.
All these
studies demonstrate that a positive ANA test is found frequently in a pediatric
hospital population, and even in high titer has only a poor ability to
determine whether a child has an inflammatory rheumatic disease.
It could
be argued that the finding of a positive ANA test indicates that the child has
an occult disease that will become manifest later. Is there any evidence to
support or refute this?
There is
some evidence that ANA may sometimes precede the development of lupus by
several years. Using
Some
studies have evaluated the outcome in patients with fibromyalgia (a
musculoskeletal pain condition not thought to be an autoimmune disease) who
were also ANA positive, and have not found that the occurrence of a positive
ANA influences outcome. Al-Allaf et al. (15) found the ANA positivity rate
(titers not given, a positive result was simply defined as “plus”) in their
adult patients with fibromyalgia was 8.8% (almost identical to the 8.9% ANA
positivity rate in their control patients with osteoarthritis). The 12
individuals who had fibromyalgia and were ANA positive were matched for age and
sex with 12 ANA negative patients. Over a 2-4 year follow-up period one patient
in the ANA positive group fulfilled criteria for lupus, and one in the ANA
negative group fulfilled criteria for Sjögren’s syndrome. The authors concluded
that the ANA test (at least in low titer) was not a good predictor of future
connective tissue disease. In a study of 59 pediatric patients with
fibromyalgia (16), 17 (28.8%) were ANA positive (mean
titer 1:160). Fifty patients were followed for a mean of 18.3 months and during
that time no patient developed a connective tissue disease.
We would
conclude from these findings that only rarely is the presence of ANA positivity
the harbinger of undeclared lupus or another connective tissue disease.
It should
also be remembered that ANA are associated not only with the classical
autoimmune diseases, but also with infection (17), malignancy (18), and drugs (19). Environmental toxins may also
predispose to ANA production. Although hopefully not relevant in pediatrics,
there is evidence of an increased frequency of ANA in individuals with silicone
breast implants (20), and at least two studies have
suggested that rural populations have a higher frequency of ANA positivity than
urban populations, perhaps due to toxin exposure (21,22). Therefore the finding of a
positive ANA test should not blind the physician to the possibility of a
non-autoimmune diagnosis.
A
situation where a positive ANA test may be of some value is in children
diagnosed with idiopathic thrombocytopenic purpura (ITP). In a study of 87
children with ITP, it was found that 36% of those with a positive ANA (titer ≥1:40)
developed further “autoimmune symptoms”, five children developing lupus,
compared to none of those who were ANA negative (p<0.001) (23).
There is
little doubt that in children with JIA the ANA test is more frequently positive
in those with uveitis than in children without uveitis. The
So what should
be done with a positive ANA test? Our answer would be exemplified by the answer
a local inhabitant gave when asked directions from place A to B by a foreign
tourist: “I wouldn’t be leaving from here!”.
In other words, it would be best if the ANA test had not been done in
the first place!
We would
suggest that a positive ANA test can safely be ignored unless there are other
suggestive clinical signs, and simple laboratory tests (such as a raised ESR or
cytopenias) that point towards a diagnosis of lupus or similar connective
tissue disease. Given the high false positive rate of ANA tests, a positive
test cannot be used as confirmatory evidence that the child with a swollen
joint has JIA, rather than some other serious condition such as septic arthritis,
leukemia, or hemophilia. Similarly, symptoms such as fatigue and aches and
pains in a child, should not be ascribed to lupus simply because of a positive
ANA test. It is much more likely that she has an idiopathic pain syndrome such
as fibromyalgia. A negative ANA test is
more useful than a positive one, as it does, for all practical purposes,
exclude the diagnosis of lupus in a child.
What is
needed is a cost-effectiveness study to evaluate whether the screening ANA test
should be replaced by testing initially for anti-dsDNA and anti-ENA (anti-Sm,
RNP, SSA and SSB) antibodies. Until that study is done, we would recommend that
non-rheumatologists only do an ANA test if there is a fairly high probability
(perhaps a 10+% chance) that a child’s symptoms could be due to lupus or Mixed
Connective Tissue Disease. If the test is positive at a titer of ≥1:160
then it would be appropriate to order antibodies to dsDNA and ENA, with lower
titers being ignored. We would strongly
recommend that the ANA test is not ordered indiscriminately as part of “a
rheumatologic work-up”.
The
question why the ANA test is so frequently positive in populations without an
overt autoimmune disease is a fascinating one. It suggests that the breaking of
immunological tolerance is really quite common, but that this tolerance
breakdown only relatively rarely leads to disease.
However, from a practical, clinical
point of view, the ANA test has such a high false-positivity rate that a
positive test is of little, if any, clinical utility.
Acknowledgements:
We would like to acknowledge the
help of Louis Wadsworth MBBS, FRCPC, Director, Hematopathology Program
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