Running For Research - PWSHuman growth hormone is effective not only in increasing height, but also in decreasing body fat, increasing muscle mass, improving weight distribution, increasing stamina, and increasing bone mineral density in PWS. In addition to these positive effects on growth and body composition, studies suggest positive effects on development and behavior.
Human Growth Hormone ( HGH) is the only medication approved by the FDA as an effective treatment for PWS. It was only in 2000 that approval was granted. For most families, getting a physician to write a prescription for GH isn’t difficult. Most pediatricians in the US, and particularly pediatric endocrinologists, are aware of the benefits of GH use. There does seem to be some dissent regarding the appropriate age to begin treatment but it is generally recognized that the earlier one begins, the greater the overall benefit.
Most of the difficulty surrounding GH lies with the insurance companies and their willingness to approve coverage benefits. Many are quick to deny the medication, even though it is indicated for use by the FDA, because of the tremendous expense involved. I can tell you from my 11 years of experience in this process and having used many insurance companies in that time, it is almost always a fight. The whole process is time consuming, frustrating, overwhelming for new parents and completely unnecessary. Having a physician experienced in getting GH approval can make all the difference. Luckily for Clem, she has the single most experienced PWS specialist in the world!
Clementine has been receiving daily GH injections since she was 10 weeks old. She gets her shot at night, before bed, because this is the time of day that GH is most greatly produced by the body. In Clem’s case, she produces almost no natural GH. She is on what is considered a very high dose for her size and weight but it’s vital to help her reach her growth potential as well as help with her body composition, metabolism, cognition and overall stamina.
GH does have some negative side effects, particularly if given in too large a dose for the individual, so we routinely test her to make sure her levels aren’t too high. Some of the possible side effects are things like the increased risk for cancerous tumors, the accelerated growth of facial features, hands and feet and the increased risk for heart disease and diabetes. While we take these risks very seriously, we have weighed the pros and cons and for us, there are more benefits than risks.
Growth hormone is known to decrease sensitivity to injected insulin. This is a particular concern for us because Clem also has T1D. Again, this just demonstrates the tight rope we walk every day between keeping Clem healthy and providing the best chance for her to reach her highest potential. Every option must be carefully considered and weighed but in the end, we go with what makes the most sense and just hope that we made the right decision.
We are so thankful that our circumstances allow us access to GH. So many people suffering with PWS are living in countries where the health system doesn’t recognize the benefits of early GH use ( or maybe at all) or don’t provide it because the cost is more than can be born. This makes me sad and angry for these families. It just illustrates the worldwide inequities of healthcare. That’s an issue for another time.
At the end of the day, the fact that GH is the only FDA approved treatment for PWS highlights how desperately we need continuing PWS research. More options must be made available for Clementine and all others within our community!
Please visit our fundraising page if you are interested in helping us raise money for the PWS Research Fund at the University of Florida. Every penny helps and every penny is so gratefully received!
