Dear Robin,

       Have you spoken to anyone at the FDA personally?  

	I faced a similar problem when I was in Australia.  There is a
carer's allowance (basically a tax credit) for children who require extra
care--seizures, MR, motor problems, diabetes, renal failure, etc--anything
that meant the parents spent more time taking care of a special-needs child
than a "normal" one.  Our PKU families applied for this coverage, but were
turned down initially because their children DIDN'T have seizures, MR, etc.
Our calculation was that PKU diet needs could add 4 hours work to the day.
The cost of the formula was covered.  Our appeal was based on the fact that
the four hours work was what kept the children from having seizures, being
retarded, etc, and so it should be covered.  (If the children became
retarded, of course, the amount of work required would be much greater.)  Our
appeal was successful.
	
	I've not read the articles yet, but I'm not surprised your patient
doesn't have short stature yet. And if he's properly cared for, he should
never get seriously obese.  Since the benefits of GH extend beyond linear
growth, and there is good published data, it only makes sense to optimize
outcome by starting earlier rather than later.


Steve

	(On the negative side, I referred a 3-month-old with Down syndrome to
the local developmental center in North Carolina, and heard he was turned
down because he didn't have developmental delay yet.  We said "Just wait"--he
got in a few months later. I wish he'd been enrolled from the beginning.)

Stephen G. Kahler, MD
Professor, Division of Medical Genetics 
Department of Pediatrics 
University of Arkansas for Medical Sciences
Email:[log in to unmask]


Office Phone 501-364-2966
Fax  501-364-1564
Pager 501-395-7865 

Mail:Division of Clinical Genetics 
Slot 512-22
Arkansas Children's Hospital
One Children's Way
Little Rock, AR  72202-3591
 
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-----Original Message-----
From: Communication for the Heartland Regional Genetics Group
[mailto:[log in to unmask]] On Behalf Of Troxell, Robin M.
Sent: Tuesday, April 07, 2009 11:11 AM
To: [log in to unmask]
Subject: GH in PWS coverage

I am trying to obtain coverage for GH for a one year old with PWS. He is not
failure to thrive because he is 100% G-tube fed due to oral hypotonia.  He is
also on a vent for about 22 hrs a day.  My initial try was denied because he
is neither obese nor minus 2 to 3 SD on the growth curve.  They state the FDA
only approves GH for kids with PWS who are growth deficient.  I included the
following references:
 

Carrel AL, Myers SE, Whitman BY, Allen DB.  Benefits of long-term GH therapy
in Prader-Willi syndrome: a 4-year study. Journal of Clinical Endocrinology
and Metabolism. 2002;87(4):1581-1585.

 

Haqq AM, Stadler DD, Jackson RH, Rosenfeld RG, Purnell JQ, LaFranchi SH.
Effects of growth hormone on pulmonary function, sleep quality, behavior,
cognition, growth velocity, body composition, and resting energy expenditure
in Prader-Willi syndrome.  Journal of Clinical Endocrinology and Metabolism.
2003;88(5):2206-2212

 

Myers SE, Carrel AL, Whitman BY, Allen DB. Sustained benefit after 2 years of
growth hormone on body composition, fat utilization, physical strength and
agility, and growth in Prader-Willi syndrome.  Journal of Pediatrics.
2000;137(1):42-49.

 

There is one appeal left - any other thoughts?

Thanks,

Robin

************************************
Robin M. Troxell, MS, CGC
Genetic Counselor
University of Missouri Department of Child Health
Mercy St. John's Hospital (Springfield, MO)
(p) 417-820-9839
(f) 417-820-3720
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miracle. The other is as though everything is." -- Albert Einstein

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