Common Questions Families Ask
About PWS and Growth Hormone
What Causes Growth Problems in Children with Prader-Willi Syndrome?
Researchers strongly suspect that the part of the brain called the
hypothalamus is the main source of the growth differences in PWS. A tiny part
of the central brain, the hypothalamus connects the body’s two key systems
for survival and maintenance—the nervous system and the endocrine system. In
addition to playing a key role in growth and sexual development, the
hypothalamus regulates appetite, metabolism, body temperature, mood, and other
functions that we know are affected in people with PWS. It is likely that one
or more of the genes that are missing (or not functioning) in people with PWS
supply essential instructions to this part of the brain.
To understand growth and growth hormone treatment in Prader-Willi syndrome,
it is helpful to have a basic understanding of how the hormone—or endocrine—system
normally works. The endocrine system is made up of all the glands that produce
and release hormones into the bloodstream. Just below the hypothalamus, and
directly attached to it, is the pituitary gland. Called the "master
gland" because it receives messages from the hypothalamus and relays them
to the other endocrine glands, the pituitary makes and releases many hormones.
Among these are growth hormone (GH) and the hormones for sexual development
and reproduction (LH and FSH). If the pituitary does not make or release
enough of these hormones, then the organs that depend on them cannot do their
job.
How can I find an endocrinologist to assess my child?
Ask your child’s pediatrician or internist for suggestions. If you have a
chapter of the Prader-Willi Syndrome Association in your state or region, ask
that group for suggestions also. Call several doctors and ask about having
your child with PWS assessed for GH treatment. Ask how much experience they’ve
had working with children with PWS. If you’re unable to find a doctor you
feel you can trust, call PWSA (USA) for assistance: 1-800-926-4797.
Can an insurance company require my child to be tested for growth hormone
deficiency before they will cover GH treatment?
Health insurance plans are allowed to set their own requirements for
coverage, but usually they will follow the U.S. Food and Drug Administration’s
approvals. It should not be necessary for a child with PWS to be tested for
growth hormone deficiency since the FDA’s decision regarding Prader-Willi
syndrome, effective June 20, 2000. The FDA approved Pharmacia Corporation’s
application to market and promote their existing GH product, Genotropin®, for
"long-term treatment of pediatric patients who have growth failure due to
Prader-Willi syndrome." In creating a specific "indication" for
children with PWS, the FDA recognized that GHD testing is not a reliable
determinant of whether a child with PWS needs GH treatment. Those with PWS
only need to show signs of growth failure and have a genetic diagnosis of PWS
to qualify for GH treatment under this special Orphan Drug Act approval. It is
possible that your insurance company is unaware of this change. A letter from
your doctor to the insurance company might help. For more information, see
PWSA’s announcement of the FDA ruling (reprinted in the Appendix) and the
prescribing literature on Pharmacia’s Web site (www.genotropin.com).
What happens if my child stops GH treatment?
People who stop taking growth hormone will not lose their height gains, but
they gradually lose the other physical benefits that GH produces—i.e.,
muscle development, fat reduction, increased energy level, etc.—and will
most likely gain weight. If your child is benefiting from GH therapy but needs
to stop because of a side effect, it may be possible for him or her to
continue treatment at a lower dosage level. Even a low dose can improve body
composition. There is generally no problem with stopping and restarting GH
treatment, but risks and benefits of treatment should be discussed with your
child’s doctor.
Aren’t there natural GH supplements my child could take instead of
getting shots?
Although many nonprescription supplements and pills are being promoted
today as growth enhancers or GH releasers, they are not oral forms of growth
hormone, nor are they effective for the needs of children with PWS. Since
these supplements are not regulated by the Food and Drug Administration, there
is also no way of knowing their actual content, effects, or safety.
Some of the GH manufacturers are trying to develop alternative ways to
deliver GH to the body, and we may eventually see forms of synthetic growth
hormone that can be taken orally or through the nostrils. If these products do
come to market, they will be prescription drugs regulated by the FDA.
THESE QUESTIONS AND MANY, MANY MORE ARE ANSWERED IN OUR 64 PAGE
BOOK TITLED:
Growth Hormone
and
Prader-Willi Syndrome
A Reference
for Families and Care Providers
by
Linda S. Keder
Table of Contents
Introduction
Prader-Willi Syndrome and Growth
Effects of Growth Hormone Treatment in Children with PWS
What Is Involved in Growth Hormone Treatment
Questions Families Ask
Appendix:
Overview of Prader-Willi Syndrome
Information Resources on Prader-Willi Syndrome
Information Resources on Growth Hormone Use and Products
Glossary of Terms
Recent GH Studies of Children with PWS
PWSA (USA) Announcement of FDA Approval, June 2000
International Consensus Statement on Use of Growth Hormone in PWS, July 2000
Bibliography on PWS and Growth Hormone
Last edited
02/09/2012
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