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This is an interesting article that highlights the controversy surrounding the continued use of brace treatment for scoliosis. I don't believe that the future of scoliosis treatment will include the continued use of bracing for scoliosis.
Professional Opinion Concerning the Effectiveness
of Bracing Relative to Observation in Adolescent
Idiopathic Scoliosis
Lori A. Dolan, PhD,* Melanie J. Donnelly, MD,Þ Kevin F. Spratt, PhD,Þ and Stuart L. Weinstein, MD*
Objective: To determine if community equipoise exists concerning
the effectiveness of bracing in adolescent idiopathic scoliosis.
Background Data: Bracing is the standard of care for adolescent
idiopathic scoliosis despite the lack of strong reasearch evidence
concerning its effectiveness. Thus, some researchers support the idea
of a randomized trial, whereas others think that randomization in the
face of a standard of care would be unethical.
Methods: A random of Scoliosis Research Society and Pediatric
Orthopaedic Society of North America members were asked to
consider 12 clinical profiles and to give their opinion concerning the
radiographic outcomes after observation and bracing.
Results: An expert panel was created from the respondents. They
expressed a wide array of opinions concerning the percentage of
patients within each scenario who would benefit from bracing.
Agreement was noted concerning the risk due to bracing for postmenarchal
patients only.
Conclusions: This study found a high degree of variability in
opinion among clinicians concerning the effectiveness of bracing,
suggesting that a randomized trial of bracing would be ethical.
Key Words: adolescent idiopathic scoliosis, bracing, concensus,
effectiveness, standard of care
(J Pediatr Orthop 2007;27:270Y276)
Bracing was adopted as the standard of care for nonoperative
treatment of adolescent idiopathic scoliosis
(AIS) long before the application of the current standards of
scientific evidence. It is questionable whether a new
technology would enjoy such widespread use if it was based
on a literature with limitations similar to those noted in
bracing: of the multiple published studies of bracing
effectiveness, the overwhelming majority are level IV case
series, with only a few level III case-control or retrospective
cohort studies, and only 1 level II prospective cohort study.
Another important limitation of the literature is the paucity
of evidence concerning the effect of bracing on surgical rates
despite the suggestion that the progression to surgery
indicates the ultimate failure of bracing treatment.1 To our
knowledge, only 2 studies2,3 have quantified (relative to
observation) the risk reduction (RR) in surgical rates due
to bracing.
The science of bracing has been hampered, ironically,
by the publication of uncontrolled studies to support bracing
as the standard of care. Consequently, researchers have
hesitated to conduct a randomized trial, stating that it would
be unethical to deny treatment (not brace) when bracing is
considered an effective therapy that has Bstood the test of
time[4 even when that test has been less than rigorous. To the
proponents of bracing, this may not be bothersome; however,
to those who are unconvinced by the evidence, following the
standard of care and prescribing a brace can itself be an
ethical battle.
More than 50 pediatric orthopaedic surgeons volunteered
to participate in a recent randomized trial proposed to
compare bracing with the observation on AIS.5 However,
several of those approached to participate declined on ethical
grounds. Concerned about this objection, we decided to test
the validity of the conclusion that a randomized study comparing
bracing with observation is unethical using the criteria
of clinical equipoise.6 Clinical equipoise has been defined as
Bthe state of honest, professional disagreement in the
community of expert practitioners as to the preferred treatment
6[. Other similar definitions of equipoise include the
state of uncertainty on the part of the pertinent community,
the opinion that no one arm of the trial is known to offer
greater harm or benefit,7 and the lack of consensus within the
expert community about the comparative merits of the
treatments being tested. Random assignment of treatments,
under the condition of equipoise, is not then a default on the
obligation to give the most appropriate treatment because this
is unknown.8
Judgments concerning the presence or absence of equipoise
can come from 3 sources of information: (1) informal
information from the opinions of local clinicians; (2)
semiformal information from evidence of different practices
across physicians or localities or from differing opinions in
the literature; and (3) formal information derived from the
specific measurement of expert opinion.8 In the literature,
only 2 published natural history studies report a rate of
surgery: Bunnell,9 in 1986, reported an overall surgery rate of
16% in curves diagnosed as having an angle of between 16
and 96 degrees, whereas Goldberg et al4 reported a 28%
surgery rate in curves with angle ranging from 10 to greater
than 60 degrees. Table 1 summarizes the results of bracing
outcome studies. Several uncontrolled retrospective case
series of braced patients have been published; those reporting
surgical rates demonstrate widely varying outcomes ranging
from 7% to 43%.10Y22 Two studies have simultaneously compared
untreated and braced curves.2,3 Fernandez-Feliberti et al2
reported a 26% surgery rate in the braced cases compared with
38% in the observed cases. Miller et al,3 in their case-control
study of small curves, found a 2% surgical rate in untreated
curves compared with 5% in the braced group. These
variations in outcomes are likely caused by different inclusion
criteria, including Cobb angle and sex. The literature on
surgical rates, then, is extremely variable and does not support
the superiority of bracing over observation with any certainty.
This variability provides some evidence of equipoise;
however, the evidence for a medical intervention does not
always equal the degree to which clinicians endorse the
intervention or agree on its outcomes.
We therefore sought another source of information, a
formal survey of expert opinion concerning the effect of
bracing relative to observation on cases of AIS. Sufficient
variability and lack of consensus in these estimates would
provide additional evidence of community equipoise and
would therefore support the ethics of randomization in a trial
of bracing on cases of AIS.
METHODS
Expert Panel
With institutional review board approval, we used the
membership rosters of the Scoliosis Research Society (SRS)
and the Pediatric Orthopaedic Society of North America as
the sampling frame for this study. Most members of both
societies are practicing physicians, although both include
small numbers of nurses, scientists, and other allied health
professionals involved in the care of children with orthopaedic
conditions. Both societies have official publications that
regularly publish research concerning the natural history and
treatment outcomes of AIS and include such articles and
posters at their national meetings. A sample of 423 members
was randomly selected. The responses were anonymous and
no attempt was made to follow up the nonresponders. All
responses were returned within 3 months after they were
mailed.
The members were also asked to supply information
concerning the following professional characteristics: number
of years in practice, specialty, whether they completed a
fellowship in that specialty, percentage of practice devoted to
AIS, and a self-rating of their familiarity with the literature
concerning bracing and AIS on a scale ranging from 1 to 3.
Surveys
The surveys were designed to gather the opinion of the
respondents concerning the radiographic outcomes of bracing
and the observation at the endpoint of skeletal maturity.
Skeletal maturity was chosen as the endpoint because the risk
of continued progression drops significantly after this point is
reached.23 The 45-degree-angle outcome was chosen as a
proxy for surgical indication, as in the studies by Little et al17
and Upadhyay et al.20
Each member received instructions and examples on
how to complete the surveys. The members were asked to
imagine patients between the ages of 10 and 15 years with
differing clinical profiles who present to their practice for
initial evaluation of AIS. The profiles included combinations
of 3 curve types (thoracic, thoracolumbar/lumbar, and double
major), the presence or absence of menarche, and the size of
the Cobb angle (25Y34 degrees or 35Y45 degrees).
The survey was structured as 6 decision trees, each
presenting (1) a treatment (bracing or observation); (2)
branches for the clinical profiles; and (3) branches for 2
radiographic outcomes (e45 or >45 degrees). Examples of
the decision trees are given in Figures 1A and B. The
members were also asked to estimate the percentage of
patients from their practice presenting with each clinical
profile. These estimates of practice mix were not used in the
analysis but were elicited to help the respondents concentrate
on each separate profile. Then, they were asked to
estimate the percentage of patients in whom they would
expect to achieve a curve with an angle less than 45 degrees
(success) of 45 degrees or greater (failure) at skeletal
maturity after both an observation (natural history) and a full
course of bracing. The members were instructed to use their
knowledge of the AIS literature and their experience to make
these estimates.
Statistical Analysis
Descriptive statistics were calculated for the (1) percentage
of patients defined as successes after bracing and after
observation, (2) the RR due to bracing (the percentage of
failure under observation minus the percentage of failure after
bracing), and (3) the number of profiles where the respondents
agreed on the RR. Agreement was defined similar to the
previous work of Wright et al24 and Dunn et al.25 Agreement
was present if greater than 80% of the respondents_ RR
estimates were within a given range (low, 0%Y39% RR;
moderate, 40%Y69% RR; and high, 70%Y100% RR). The
influence of curve type and profile on the outcomes was quantified
using analysis of variance and W2 tests of association.
RESULTS
Sample
A total of 423 surveys were mailed and 92 responses
were received. Of these, 10 were from clinicians who declined
to participate, and 4 responses were deemed invalid
because of bracing failure rates that were uniformly higher
than observation failure rates. Therefore, usable data was
obtained from 78 respondents (19%). Considering the low
response rate and, therefore, the questionable generalizability
across all clinicians, we decided instead to use an expert panel
approach similar to that used by Latthe et al26 and Lilford27 in
their studies of clinical equipoise. From the 78 respondents,
we chose an expert panel of those who reportedly devote
more than 25% of their practice to AIS and who also consider
themselves very familiar with the AIS and bracing literature.
Of these 29 experts, 20 (69%) listed pediatric orthopaedics as
their subspecialty; 3 (10%), spine; and 4, pediatric spine. On
average, the panel had spent 22.55 years in their specialty
(range, 6Y45 years) and 24 (83%) had completed a fellowship
in that specialty. The average percentage of practice devoted
to AIS was 49% (range, 28%Y100%).
Outcome Estimates
Table 2 summarizes the success estimates (percentage
of curves progressing to a Cobb angle less than 45 degrees)
FIGURE 1. Examples of survey. A, Estimates of observation outcomes. B, Estimates of bracing outcomes.
Dolan et al J Pediatr Orthop & Volume 27, Number 3, April/May 2007
272 * 2007 Lippincott Williams & Wilkins
Copyr ight © Lippincott Williams & Wilkins. Unauthorized reproduction of this article is prohibited.
for each of the profiles and the treatments (bracing or
observation). Although the average estimates indicate that the
group felt that bracing demonstrates an advantage over
observation in the risk of surgery, the estimates for both
bracing and observation varied widely. For example, the
panel estimated that anywhere from 20% to 80% of small
thoracic curves in immature patients would succeed without
treatment. For the same group of curves, the success rates
after bracing ranged from 49% to 90%. The type of curve
alone had no significant effect on the estimates, but the
estimates were higher or lower depending on the profile
(a statistical interaction of curve size and menarcheal
status; P G 0.0001). This interaction is illustrated by the
estimates for thoracic curves. The average success estimate
for small thoracic curves in premenarcheal patients without
treatment was 44.87% compared with 77.22% for small
curves in postmenarcheal patients (difference of approximately
22%), whereas the average success estimate for large
thoracic curves was 20.39% in premenarcheal patients and
55.85% in postmenarcheal patients (difference of approximately
35%). Figure 2 summarizes these relationships.
Table 3 summarizes the RR estimates for each clinical
profile. The RR estimates were obtained by subtracting the
bracing failure rates from the observation failure rates. Like
the raw success rate estimates, there was wide variation in the
RR estimates. The minimum RR was 0% for all profiles, and
the maximum ranged from 55% (small curves in postmenarcheal
patients with thoracolumbar/lumbar curves) to 75%
(small thoracic curves in postmenarcheal patients and large
thoracic curves in premenarcheal patients). Another way to
demonstrate the variability between the raters is to look at the
median estimates. For example, the median RR for postmenarcheal
patients with large thoracic curves was 30%;
therefore, one half of the panel thought that the RR due to
bracing was 0% to 30%, whereas the other half thought that it
was 30% to 75%.
The RR estimates did not significantly differ across
curve types, but they were significantly different between
profiles (P G 0.0001). This is expected considering the
interaction observed for the raw success estimates. The
interaction suggests that on average, the panel thought that
the impact of bracing was dependent on both the curve size
and the menarcheal status of the patient. Consistently,
however, the RR estimates for premenarcheal patients were
greater than those for the postmenarcheal patients. Thus, the
panel responses suggest that bracing has a greater positive
impact for premenarcheal patients than for postmenarcheal
patients. Boxplots summarizing the RR estimates are
provided in Figure 3.
Agreement
We divided the range of RR estimates into 3 intervals:
small effect (0%Y39% fewer failures with bracing), medium
effect (40%Y69% fewer failures with bracing), and large
effect (70%Y100% fewer failures with bracing). Clinical
agreement was present if more than 80% of the experts_
estimates were within 1 of the 3 intervals. We evaluated
whether there was clinical agreement on the outcomes of each
of the 12 profiles.
According to this definition, there was clinical agreement
on only 4 of the 12 profiles, all of which proposed a
small RR due to bracing in postmenarcheal patients. These
agreements are highlighted in Table 4. More than 80% of
the experts indicated that bracing would have a small
effect on postmenarcheal patients with thoracic curves (for
both small and large curves), postmenarcheal patients with
small thoracolumbar/lumbar curves, and postmenarcheal
patients with small double major curves. The respondents
were very close to agreement (77% and 79%) that bracing
would have only a small effect on postmenarcheal patients
with either large thoracolumbar/lumber curves or double
major curves.
DISCUSSION
This study used the experts_ opinion of radiographic
outcomes to estimate the surgical rates after observation and
bracing for cases of AIS. These rates varied widely within the
panel for both treatments, as did the consequent RR due to
bracing. The reported RR ratios indicate a wide spectrum of
opinion, from substantial benefit from brace use to no benefit
at all. Agreement, defined as greater than 80% endorsement,
existed in about one third of the profiles. These data
demonstrate significant uncertainty within this expert group
concerning the outcomes of observation and bracing.
Consequently, we think that there is evidence of community
equipoise for most clinical profiles contained in this survey
and that the equipoise requirement for an ethical randomized
trial has been met.
The method of expert group input has been widely used
in other health research applications, including technology
assessment, education and training, priorities and information,
and development of clinical practice.28 The design used
here allowed a panel of geographically dispersed experts to be
surveyed efficiently and confidentially.29 All clinicians
reviewed exactly the same material, with no uncertainty
concerning Cobb angle measurement or other evaluations that
might occur in actual practice. In addition, because these
estimates reflect the initial clinical judgments in the absence
of knowledge of the estimates of other clinicians, they
provide an indication of the extent of interclinician variation
that might occur in actual practice. Some might argue that the
contrived situation of this research design does not reflect
clinical practice. However, would the outcomes be any less
variable if it was a parent, rather than a researcher, asking BOf
children like mine, how many will need surgery without
treatment? How many will need surgery after treatment?[ It
seems very unlikely that the results of this exercise overestimate
the extent of interclinician variation in predicting the
effectiveness of bracing in this population.
Several recent articles have reported on the clinical
agreement concerning the indications and the outcomes of
medical treatment for other purposes in addition to clinical
trial planning.24Y26,30,31 Two of these papers involve
orthopaedics and each defined agreement similarly to this
paper. Wright et al24 demonstrated disagreement similar to
that shown in this article in their survey concerning the
indications and the outcomes of total knee replacement.
For example, their respondents indicated that anywhere
from 1% to 95% of patients would require a revision
within 10 years of their primary replacement. Dunn et al25
also found a significant variation in decision making and a
lack of clinical agreement concerning the indications for
rotator cuff surgery.
To our knowledge, there have been only 3 published
reports measuring community equipoise to specifically assess
the ethics and the feasibility of conducting randomized
clinical trials. Young et al32 mailed surveys to all members of
a vascular surgery professional organization and asked the
members to rate several common clinical scenarios describing
2 alternative treatments for the same condition. The
respondents showed great variability in their responses, and
each treatment was endorsed to some degree in all of the
scenarios. There were only 1 in 6 scenarios where more than
70% of the respondents agreed that the same treatment was
preferable. The authors conclude that this variation indicates
equipoise within the membership and, therefore, that
randomized clinical trials would be ethically justified.
Lilford27 surveyed the expectations of a 10-member expert
panel concerning the probable relative risk of morbidity
resulting from immediate or delayed delivery in scenarios
involving at-risk fetuses. For each scenario the average result
was no relative RR, but the range in estimates for each
scenarios was large. In 1 scenario, the estimates ranged from
a 75% decrease to a 25% increase in the risk to a fetus
delivered early. Lambert et al33 investigated the perceptions
of the parents and the members of the Pediatric Ophthalmology
and Strabismus Society concerning the treatment
for infants with congenital cataracts. On a scale ranging from
1 to 10, with 1 strongly favoring an intraocular implant and
10 strongly favoring a contact lens, the median score of the
respondents was 7.5. This range of opinions also manifested
itself as a support for randomized controlled trials because
61% of the respondents indicated that they would be willing
to randomize children to one of these 2 treatments.
These studies indicate that clinical disagreement is a
reality across specialties and interventions. Three possible
explanations for clinical disagreement demonstrated by
studies such as these include the limitations of available
knowledge, the controversy within the research literature, and
the inadequate dissemination or adoption of available information.
24 A recent article by the SRS Bracing Committee34
addresses these issues by calling for completeness and
uniformity in the subjects, endpoints, and outcomes of
bracing studies to maximize the likelihood of developing a
coherent, accepted body of knowledge concerning this
disease. If future articles adopt this approach, disagreements
may diminish.
It has been suggested that progression to surgery
indicates the ultimate failure of bracing treatment.1 The key
question of any future study of bracing, randomized or not,
must be BHow many patients avoided surgery because of
bracing treatment?[ This study found a high degree of
variability in opinion among experts concerning the effectiveness
of bracing. Yet, bracing is the standard of care for
AIS, and all 362 respondents to a recent survey from the
SRS35 indicated that they advocate its use. This implies a
major disconnect between opinions of effectiveness and
endorsement of bracing by the community. Patients considering
their treatment options need to be aware of this
disconnect; instead of considering bracing as the only option,
they should take this variation into account along with
their personal goals and tolerance for risk. In addition, these
results indicate that a randomized trial of bracing would not
only be ethical but also necessary.
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