THOMAS CHALLMAN

02/01/2016
C o m p l e m e n t a r y - A l t e r n a t i ve M e d i c i n e
a n d A u t i s m : W h a t ’s t h e E v i d e n c e ?
“Every complex problem has a solution that
is simple, direct, plausible, and wrong.”
Thomas Challman, MD
Medical Director
Geisinger Autism & Developmental Medicine Institute
H.L. Mencken
“ The less the evidence on which an opinion
is based, the firmer the conviction with
which it will be maintained.”
• “We have perhaps the
greatest find in the
whole earth at this time.
It is located on Native
American land in
western America. It is
loaded with minerals,
amino acids, vitamins
and DNA material.”
Leon Eisenberg
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Geisinger Autism & Developmental
Medicine Institute
A Real Case
Two brothers with autism
Saw Doctor “X” who recommended the following:
– Transdermal DMPS
(dimercaptopropanesulfonate) drops, to remove
‘toxic heavy metals’ ($160/ounce)
– A product called ‘Advanced Cell Life’
– Avoiding all future vaccines
“… anti-tumor, anti-viral, anti-bacterial and antiparasitical. They both repair the DNA, Detox and
rebuild the liver…
Also contains latent solar energy hidden deep within
its complex molecular structure …
High quality Fulvic acid does an amazing job at
repairing the DNA and re-connecting the DNA to all
cells.”
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02/01/2016
Management of Children with
Autism Spectrum Disorders
Educational and Behavioral Interventions
– Preschool and School Programs
– Specific Strategies
• Intensive behavior therapy - ABA
• Structured teaching
• Speech and language therapy
NCCAM classification of Complementary and
Alternative Medicine (CAM)
Mind-body medicine
Manipulative and body-based practices
Energy medicine
Biologically-based practices
Alternative medical systems
– Augmentative and alternative communication
• Occupational therapy, physical therapy
• Social skills instruction
Management of Children with
Autism Spectrum Disorders
Medication Management
– Challenging behaviors
– Seizures
– Gastrointestinal Problems
– Sleep Disturbance
NCCAM classification of Complementary and
Alternative Medicine (CAM)
Mind-body medicine
Manipulative and body-based practices
Energy medicine
Biologically-based practices
Alternative medical systems
The use of therapies considered CAM is common among
adults and children
NHIS (2002): 62% of adults used CAM in the previous
year
A majority of patients in these surveys did not discuss
their use of these interventions with their regular
medical provider
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02/01/2016
Prevalence of CAM Use
In pediatric populations, there is considerable variation in
estimates of CAM use
Selected pediatric outpatient and inpatient populations:
11-53%
CAM use in autism (Levy, et al. 2003):
284 children recently diagnosed with an autism spectrum
disorder
• 30% were using some form of CAM
• Vitamins, GI medications, melatonin
• Gluten-free/casein-free diet, secretin
• Cod liver oil, anti-infectives, chelation
• Non-biological therapies
Prevalence of CAM Use
CAM use is common among children with chronic health
conditions:
• Cancer, arthritis, inflammatory bowel disease, asthma
• ADHD, cerebral palsy, spina bifida, Down syndrome
• Autism
CAM use in autism (Levy, et al. 2003):
284 children recently diagnosed with an autism spectrum
disorder
• 16.9% were using a “biological” therapy
• 9% were using a potentially harmful therapy
Prevalence of CAM Use
CAM use in autism (Green, et al. 2006):
•
•
•
•
•
•
Internet survey of 552 caregivers
Alternative diets: 26.8%
Vitamin supplements: 42.6%
Detoxification (including chelation): 8.9%
Other alternative therapies: 25.9%
Mean number of interventions per child (standard +
nonstandard): 7
Prevalence of CAM Use
CAM use in autism (Hanson, et al. 2007):
•
•
•
•
•
Survey of 112 families
Biologically based practices: 54%
Mind-body interventions: 30%
Manipulation/body-based practices: 25%
Energy therapies: 8%
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02/01/2016
Prevalence of CAM Use
CAM use in a large pediatric autism sample (Perrin, et al.
2012):
3173 individuals with ASD
• Any CAM use: 28%
• Special diets: 17%
• Other CAM: 20%
• Children with GI symptoms, seizures, or behavior problems
had higher CAM use
Pediatrics, November 2012,Vol 130, Suppl 2
Some of what is going on - from one website:
Transdermal Secretin
Oral Secretin
Vitamin B-12
Intramuscular or Intravenous Magnesium
Gluten and Casein Free Diets
Pancreatic Enzymes
Super Nu Thera
Omega-3 Fatty Acids
Prevalence of CAM Use
Akins, et al (2014):
•
•
•
•
Survey of 578 children with ASD
39% used some form of CAM
8.6% used an invasive, disproven or unsafe CAM therapy
Higher parental education was associated with higher rates
of CAM use
J Dev Behav Pediatr, 2014, 35:1-10
Aloe Vera
Flower of Sulphur
Efalex Oil or DHA Oil
Hyperbaric Oxygen Treatments
Fibroblast Growth Factor 2
Live Cell and Stem Cell Therapy
Anti-fungal Treatment
Antibiotic Therapy
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02/01/2016
Why Do People Use CAM?
Naltrexone
IV Immunoglobulin Therapy
Detoxification for Heavy Metals
EEG Bio-feedback
Somatic therapies
Craniosacral Therapy
Traditional Chinese medicine
Immunotherapy
Why Do People Use CAM?
Adults who use CAM are generally not dissatisfied with
conventional medicine
• CAM therapies may be more congruent with their specific
values and life philosophies (Astin, 1998; Eisenberg, et al.,
2001)
Pediatrics:
• Higher parental education
• Higher rates of parental CAM use
Birdee GS, et al.: Factors Associated With Pediatric Use
of Complementary and Alternative Medicine
(Pediatrics Vol. 125 No. 2 February 1, 2010, pp. 249 256)
• Pediatric CAM users were more likely to take prescription
medications, have a parent who used CAM, and have chronic
conditions (anxiety or stress, musculoskeletal conditions,
dermatologic conditions, or sinusitis)
Why Do People Use CAM?
Hall SE, Riccio CA: Complementary and alternative
treatment use for autism spectrum disorders
(Complementary Therapies in Clinical Practice, 2012,
18:159-163)
• Four factors emerged as influencing decisions to use CAM:
severity, child acceptance of the treatment, marital status, and
educational level
• Research support for the therapy did not emerge as
contributing to variance for CAM use
Why Do People Use CAM?
In developmental disorders, non-standard interventions
are often pursued based unsupported theories
regarding the cause(s) of the disorder
• Mercury  chelation
• “Leaky gut”  GF/CF diet
• Yeast “overgrowth”  antifungals
“It is a capital mistake to theorize before one has
data. Insensibly one begins to twist facts to suit
theories, instead of theories to suit facts.”
Sherlock Holmes, in Arthur Conan Doyle’s
A Scandal in Bohemia
“Cottingley Fairies”
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02/01/2016
What Does the Evidence Show?
Research into CAM
Effectiveness of CAM in children
1999: creation of the National Center for
Complementary and Alternative Medicine (NCCAM)
The evidence-base for complementary medicine in children: a
critical overview of systematic reviews
Total NIH expenditure on research into CAM therapies
since 1999: >$3 billion
Hunt K, Ernst E. Arch Dis Child 2011;96:769–776.
Source: http://nccam.nih.gov/
National Center for Complementary and
Alternative Medicine:
Stated mission: investigate complementary and
alternative practices using the methods of rigorous
science, train CAM researchers, and disseminate
authoritative information regarding CAM to the
public and professionals
What Does the Evidence Show?
“Study results are mixed on whether echinacea
effectively treats colds or flu. For example, two
NCCAM-funded studies did not find a benefit
from echinacea….
Overview of 17 systematic reviews
– Acupuncture, chiropractic, herbal medicine, homeopathy,
hypnotherapy, massage and yoga
– Results were unconvincing for most conditions
– Acupuncture may be effective for postoperative nausea and
vomiting
– Hypnotherapy may be effective in reducing procedure-related
pain
However, other studies have shown that
echinacea may be beneficial in treating upper
respiratory infections.”
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02/01/2016
CAM therapies in autism
Summarized in “Complementary and Alternative Medicine
Treatments for Children with Autism Spectrum Disorders”,
Levy SE and Hyman SL. Child Adolesc Psychiatric Clin N
Am, 2015, 24:117-143
Levy SE and Hyman SL. Child Adolesc Psychiatric Clin N Am, 2015, 24:117-143
Levy SE and Hyman SL. Child Adolesc Psychiatric Clin N Am, 2015, 24:117-143
Levy SE and Hyman SL. Child Adolesc Psychiatric Clin N Am, 2015, 24:117-143
Levy SE and Hyman SL. Child Adolesc Psychiatric Clin N Am, 2015, 24:117-143
CAM therapies in autism
Therapies with evidence ratings of >=B
Efficacious
Melatonin
Vitamin C
Transcranial magnetic
stimulation
Music therapy
Non-efficacious
Auditory integration
Acupuncture
Hyperbaric oxygen therapy
Gluten-free, casein-free diet
Secretin
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02/01/2016
Melatonin
Wright B, et al. Melatonin versus placebo in children
with autism spectrum conditions and severe sleep
problems not amenable to behaviour management
strategies: a randomised controlled crossover trial.
Journal of Autism and Developmental Disorders. Feb
2011 v41 i2 p175(10).
Twenty-two children with autism spectrum disorders
with severe dysomnias
Double blind, randomized, controlled crossover trial
Levy SE and Hyman SL. Child Adolesc Psychiatric Clin N Am, 2015, 24:117-143
Levy SE and Hyman SL. Child Adolesc Psychiatric Clin N Am, 2015, 24:117-143
Melatonin
Several studies have shown that average night sleep
duration was longer on melatonin, and sleep-onset
latency was shorter (by about 30 minutes)
Melatonin
Melatonin significantly improved sleep latency (by an
average of 47 min) and total sleep (by an average of 52
min) compared to placebo, but not number of night
wakenings
The side effect profile was low and not significantly
different between the two arms
Levy SE and Hyman SL. Child Adolesc Psychiatric Clin N Am, 2015, 24:117-143
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02/01/2016
Melatonin
Appleton RE, et al. The use of melatonin in children with
neurodevelopmental disorders and impaired sleep: a
randomised, double-blind, placebo-controlled, parallel
study. Health Technology Assessment (Winchester,
England). 16(40):i-239, 2012.
Randomised, double-blind, placebo-controlled, parallel
study.
Starting dose was 0.5 mg; the dose could be escalated to
12 mg
Melatonin
146 children were randomized; 110 contributed data for
the primary outcome
Children treated with melatonin slept, on average, 23
minutes longer than those in the placebo group
Melatonin reduced sleep onset latency by a mean of 45
minutes
Melatonin
Wirojanan J, et al. The efficacy of melatonin for sleep
problems in children with autism, Fragile X syndrome,
or autism and Fragile X syndrome. Journal of Clinical
Sleep Medicine. 5(2):145-50, 2009 Apr 15.
4-week, randomized, DBPC, crossover design
Melatonin dose: 3 mg
Mean night sleep duration was longer on melatonin than
placebo by 21 minutes (p = .02)
Mean sleep-onset latency was shorter by 28 minutes (p =
.0001)
Omega-3 Fatty Acids
“…there is currently insufficient scientific evidence to
determine if omega-3 fatty acids are safe or effective for
ASD.”
Bent S, et al. Journal of Autism and Developmental Disorders. August 2009 v39 i8
p1145(10)
Omega-3 Fatty Acids
“To date there is no high quality evidence that omega-3
fatty acids supplementation is effective for improving
core and associated symptoms of ASD.”
James S, Montgomery P, Williams K. Omega-3 fatty acids supplementation for
autism spectrum disorders (ASD). Cochrane Database of Systematic Reviews.
(11):CD007992, 2011.
Amminger, et al. (Biological Psychiatry, 2007)
“We observed an advantage of omega-3 fatty acids
compared with placebo for hyperactivity and
stereotypy…. The results of this study provide
preliminary evidence that omega-3 fatty acids may be an
effective treatment for children with autism.”
Or does it ?
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02/01/2016
Table 1. Mean Scores on the Aberrant Behavior Checklist at
Baseline and 6 Weeks
Fish Oil
The study referenced:
“A Pilot Randomized Controlled Trial of Omega-3 Fatty
Acids for Autism Spectrum Disorder” Bent, et al, J
Autism Dev Disord, 2011, 41:545-554
Placebo
Baseline 6 wks
Baseline 6 wks
Stereotypy
14.4
13.0
7.8
8.8
Hyperactivity
33.3
29.3
24.6
27.6
Table 1. Mean Scores on the Aberrant Behavior Checklist at
Baseline and 6 Weeks
Fish Oil
Placebo
Baseline 6 wks
Baseline 6 wks
Stereotypy
14.4
13.0
7.8
8.8
Hyperactivity
33.3
29.3
24.6
27.6
• Double-blind, placebo-controlled trial
• No statistically significant benefit was
observed in the treatment group
Omega-3 Fatty Acids
“Internet-based, Randomized, Controlled Trial of Omega3 Fatty Acids for Hyperactivity in Autism”, Bent et al,
JAACAP, 2014, 53:658-666
• No statistically-significant improvement in
hyperactivity in the treatment group
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02/01/2016
Omega-3 Fatty Acids
Vitamin B-12
“Dietary Docosahexaenoic Acid Supplementation in
Children with Autism”, Voigt et al, JPGN, 2014, 58:715-722
• Dietary DHA supplementation of 200 mg/day did not
improve the core symptoms of autism
No convincing evidence of efficacy in the treatment of
ASD
Special diets
Highly Implausible Therapies
Gluten-free, casein-free diet
Several small published controlled studies
“Energy” Therapies
Therapeutic touch
Accupuncture
Bertoglio, et al (J Altern Complement Med, 2010,
16:555-560)
• 12 week, DBPC trial of methyl B-12
• No significant behavioral differences were found
between the B-12 and placebo groups
“Energy” healing
Homeopathy
Remember:
There is no alternative physics.
“…evidence for efficacy of these diets is poor.”
Millward C, Ferriter M, Calver SJ, Connell-Jones GG. Gluten- and casein-free
diets for autistic spectrum disorder. Cochrane Database of Systematic Reviews
2008, Issue 2
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02/01/2016
Placebo/Expectancy Effects
Placebo effect: “a genuine psychological or physiological
effect, which is attributable to receiving a substance or
undergoing a procedure, but which is not due to the
inherent powers of that substance or procedure”
(Stewart-Williams & Podd, 2004)
Placebo/Expectancy Effects
In studies of the medical treatment of depression,
response rate to placebo is as high as 50%
Placebo/Expectancy Effects
Placebo/expectancy effects have major implications for
the interpretation of improvement observed in the use
of nonstandard therapies in children with
developmental disorders
Highlights the necessity of well-designed clinical trials to
test specific interventions
Does Evidence Matter?
It should – but people often don’t care about evidence.
Why?
Sandler, et al. (1999): Trial of secretin in autism
• Nearly 70% percent of the parents of the children
in secretin study wanted to continue secretin
treatment for their children, even after they knew
the study showed that the secretin was ineffective
Bronislaw Malinowski: Magic, Science, and Religion:
and other essays (1954)
• Anthropological study of Trobriand
Islanders
Risperidone in ADHD (Armenteros, et al. JAACAP, 2007)
– 77% response rate in the placebo group
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02/01/2016
Environmental conditions that result in
increased danger or uncertainty lead to
an increase in magical thinking
Attention should therefore be directed toward
interventions that reduce caregiver anxiety
and uncertainty
Appropriate psychosocial supports
Education about what is truly known regarding
the etiology, potential outcomes, and best
treatments
In developmental disorders, causes can be
obscure and outcomes uncertain
Caregiver stress is often high
This contributes to an environment in which
caregivers are susceptible to the development
of idiosyncratic beliefs concerning etiologies
and treatments
Counseling Families about Nonstandard
Treatments
AAP (2001, reaffirmed 2010):
– Seek information and share it with families
– Evaluate the scientific merits of specific
therapeutic approaches
– Identify risks or potential harmful effects
– Educate families to evaluate information about
treatment options
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02/01/2016
What’s the harm?
Tolerance for risk is contingent upon the
effectiveness of a therapy for a particular
condition, and the severity of that condition
Studies of CAM therapies rarely measure or
report adverse effects
Adverse effects should be viewed broadly
Children seen by naturopathy and chiropractic
practitioners:
– are significantly less likely to receive the full
complement of recommended vaccines
– are significantly more likely to be diagnosed
with a vaccine-preventable disease
1.
Downey L, et al. Matern Child Health J 2010;14:922–30.
6.
Twelve questions that can help identify
questionable therapies (adapted from Nickel,
1996, and Lilienfeld, Lynn, and Lohr, 2003):
2.
3.
4.
5.
Is the treatment based on a theory that is overly
simplistic?
Is the treatment based on proposed forces or
principles that are inconsistent with accumulated
knowledge from other scientific disciplines?
Has the treatment changed little over a very long
period of time?
Is it possible to test the treatment claim?
Have well-designed studies of the treatment been
published in the peer-reviewed medical literature?
Do proponents of the treatment “cherry pick” data
that supports the value of the treatment, while ignoring
contradictory evidence?
7.
Do proponents of the treatment assume a treatment is
effective until there is sufficient evidence to the
contrary?
8. Do proponents claim that a particular treatment
cannot be studied in isolation, but only in combination
with a package of other interventions or practices?
9. Is the treatment promoted as being “natural” or free of
adverse effects?
10. Is the treatment promoted primarily through the use of
anecdotes?
11. Is scientific-sounding, but nonsensical, terminology used
to promote the treatment?
12. Is the treatment promoted for widely diverse
conditions?
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02/01/2016
Changes You May Wish to Make in Practice
1. Ask parents about CAM use
2. Learn what types of non-standard therapies are
most commonly used in your area
3. Talk to your families about the scientific
plausibility of, and evidence for/against, specific
types of CAM
4. Promote critical thinking skills among caregivers,
therapists, trainees
5. Help give the families of your patients the tools
they need to be able to evaluate evidence
“It is largely up to physicians to steer parents
away from these risky, ineffective, untested
practices. It is the physician’s responsibility to
encourage families to seek safe effective,
evidence-based interventions.”
Alison Singer and Ramita Ravi
AMA Journal of Ethics
April, 2015,Vol 17 No.4: 375-380
Additional References
“Uncritically accepting every proffered notion,
idea, and hypothesis is tantamount to knowing
nothing…. Some ideas really are better than
others.”
Carl Sagan
Challman TD, Myers SM (2011). Complementary and
alternative medicine in developmental and behavioral
pediatrics. In: Voigt RG, Macias MM, Myers SM, eds.
Developmental and Behavioral Pediatrics. Elk Grove
Village, IL: American Academy of Pediatrics.
Lilienfeld, S. O., Lynn, S. J., & Lohr, J. M. (Eds.). Science
and Pseudoscience in Clinical Psychology. Guilford
Press: (2003)
Additional References
Association for Science in Autism Treatment:
www.asatonline.com
My e-mail: [email protected]
Autism Science Foundation:
www.autismsciencefoundation.org
AAP:
www.aap.org/healthtopics/autism.cfm
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