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Please note: these responses are personal opinions and offered for your assistance. MAGIC does not offer Foundation authorized medical advice. Always consult your child's medical team for advice prior to making any medical decisions. See site disclaimer.
1. Question: The video on IGFD is very interesting. As the parent of a ghd child, I have a question. I know that growth hormone is crucial for cellular regeneration even the heart muscle etc. throughout my daughters entire lifetime. But I am confused- this deficiency via the growth hormone affects bone and tissue growth. So does IGF-1 also impact adults? Is this something we need to watch as well? Thank you.
Answer: Thank you for your very intersting question. In providing the information below, I remind you that your health care professional knows your daughter best and will surely offer the best treatment advice.
To the best of my current knowledge, administration of synthetic IGF has not been shown to demonstrate long term health benefits, except in patients who have a gene deletion disorder like Laron dwarfism. IGF 1 however, does have an importnat role to play in the bone metabolism in children with low physiological IGF secretion. At this time I have seen no convincing data that IGF should be used for anything but IGF deficiency in children, however, longer term collection of data my provide other options. GH remains the treatment of choice for patients who continue to be GH deficient as an adult.
2. Question: My daughter is IGF-1 deficit @370. She is in the Saizen growth hormone and is being changed to Increlex because of the real deficit that she has. However I have read about the secondary effects and I am not to sure it is worth it the risk. Can you please tell me what you think and if you have any patients that are on this medicine and what are their feelings about it, and their results?
Answer: Thank you for your question regarding IGF-1. The response given below is a personal opinion. Your doctor is your primary source of information, the information given here is not a medical opinion but is based on current information that is available to The MAGIC Foundation.
Its hard to answer your question without knowing the original diagnosis that your daughter was given. The key in the question is;
"Is your daughter doing well on growth hormone and is she growing"? If she is not doing well, Increlex is an alternative that may work. There is a great webcast at magicfoundation.org which explains IGF Deficiency in detail. You can also access the manufacturers web site to look at the clinical trial results.
As to the safety and side effects, I am sure your doctor has walked through all of the information with you. As a patient advocacy group we have not heard of any adverse experiences with Increlex. I have spoken with only a couple of patients on Increlex and they are happy with the results.
There are a couple of things that you should keep in mind if you are changing product:
1. The FDA would not license a product if they consider the risks out weigh the benefits, the fact that IGF treatments were granted licenses suggests that they are safe when used as prescribed. I have no reason to believe that there are any specific concerns with Increlex. This should be discussed with your Dr. who will advise you about any concerns you should be aware of.
2. If your daughter is not growing well on growth hormone an IGF product may be your best choice.
3. The injection schedule and procedure will change - We can send you the 'Know to Grow' brochure from the manufacturers of Increlex if you do not have one from your doctor.
3. Question: My son has been receiving growth hormone replacement therapy for nearly 8 months. To date wehave seen no increase in his growth velocity. If anything, his growth
rate has decelerated in recent months. His growth hormone levels are normal. His IGF-1 levels were very low but have improved somewhat since the initiation of his treatment, his
bone age is delayed and his height is off the bottom of the growth charts. He had his dosage increased and was started on
Synthroid after four months of growth hormone treatment, but there has been no improvement in his growth pattern. I'm wondering if there's any point
in continuing with this apparently unsuccessful treatment. Do you have any recommendations or suggestions for us?
Answer:
It is unusual to see no acceleration in growth rate after 8 months of therapy on GH therapy, especially after the addition of Thyroxin. Was your son hypothyroid from the beginning? There are a couple of possible reasons;
1. Poor absorbtion of GH can lead to the scenario you describe. A couple of questions, are you having any problems with the injections? - i.e. are you getting any leak back from the skin, you can check this by placing a piece of gauze on the site right after the injection, if you see a halo of clear fluid around the spot of blood, this is GH leaking out. If you are seeing leak back you could try holding the needle in place a little longer - the nurse at your pediatric endocrinologists office could show you this technique.
2. The cause of the short stature is not related to GH. If all is going well with the injections and you are sure the GH is being absorbed every day, then your Dr may order another blood test and consider changing you to a IGF product rather than a growth hormone. It has recently been established that IGF 1 is an alternative to GH treatment when the primary cause of the problem is considered to be IGF Deficiency.
4. Question: What is growth hormone (GH) resistance?
Answer: Growth hormone resistance is said to occur when the pituitary gland makes plenty of growth hormone ( so you would see high GH during stimulation tests i.e. over 15), but the body is unable to bind to the growth hormone receptor. You would expect to see low IGF levels in GH resistance as only GH in its bound form raises IGF 1.
Recent advances suggest that administering IGF 1 may correct his disordeer. IGF 1 is now available commercially.
5. Question: We just started our son on Increlex a week ago. When we started, I was under the impression that the drug testing period was over but as I have read up on Increlex on the Tercica website a few phrases have surprised and worried me. It said that the use of Increlex in the Primary IGFD population is investigational, that they are currently trying to determine the safety and efficacy of Increlex. It also said that children may qualify for inclusion in these "studies" and receive the medication, etc. for free. We have received the "starter" free but were told that the rest of the cost would be pending our insurance and household income. I'm confused on what this program really is. We were never told that we were part of a study, only that there was now a new and effective new drug approved by the FDA. If that is the case, why are they still studying it for safety? And is it so new that there is no record yet of long term effects? Are you familiar with any of this and what do you think? I know I've asked a lot of questions and I really appreciate your time and expertise in the matter. Thank you.
Answer:
1. Increlex is approved for use in SEVERE primary IGFD, its use to the best of my knowledge has been proven in this condition in studies, the FDA would not grant a license to market a drug without extensive drug trials.I suspect that your son is not in a trial as he has severe primary IGFD. I believe there are studies ongoing for other indications where IGF could be a primary player in the treatment of the condition, such as primary IGFD ( a less severe form than is seen in severe IGFD), idiopathic short stature or a genetic disorder
.
It is also common practice once a drug has been approved for the company to perform dose ranging and frequency studies.
Please see the statement below from the manufacturers of
Increlex™ (mecasermin [rDNA origin] injection).
Tercica, Inc. is currently conducting clinical trials throughout the US to determine the safety and efficacy of Increlex in children with growth failure due to Primary IGFD, which is a less serious form of severe Primary IGFD. The use of Increlex in the Primary IGFD population is investigational, but it is FDA-approved for use in the severe Primary IGFD population.
For full prescribing information on Increlex, visit www.increlex.com.
2. As to particpation in clinical trials, to participate in a clinical trial you must first complete a informed consent form for you or your child. A informed consent form details your rights as to the particpiation in the study. You should always read the informed consent carefully so that you are aware of your rights.
3. As to side effects, you are correct, long term side effects (dependant on how you define long term) have not yet been studied, as this is a new drug on the market. However, as this is a similar protein to growth hormone i would not expect the long term effects to be any different. The most important side effect related to IGF 1 is low blood sugar (hypoglycemia), which i am sure your Dr discussed with you before starting the treatment.
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