My son Nicolas is growth hormone defecient per Dr. Wong's team.  Can anyone give me their insight of growth hormones?

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My son had an argenine test in Cinci.  They gave him argenine through an IV which ran through quickly.  The whole test lasted about 2-1/2 hours and they withdrew blood every half hour.  This test showed that he is growth deficient.  He hasn't grown in over 18 months and he is 8.  I was told from the doctor that the Humotrope will not miraculously make him as tall as he should have been if he was without steroids, but it will give him more height.  I also was told that about 90% of DMD boys seen at Dr. Wong's clinic are on growth hormones.  Also, Dr. Rutter at Cinci has already started the IGF-1 study.  It lasts 6 months.  The only reason we chose not to be in that study is because exon skipping is supposed to start in September and to be in that trial, Nicolas cannot be in any other study for a 3 month period (it's either 3 or 6 months).

 

Just like with everything there are side effects and some of them are serious.  One side effect that Dr. Rutter's nurse mentioned is a displacement of the hip joint.  She said she saw this 2 times before, but stated this is the only side effect she has seen with Humotrope.  The other she mentioned and Dr. Rutter mentioned is contractions in DMD boys.  They have to be aggressively stretched.  Of course we will keep an eye on any and all side effects.  But even aspirin has side effects.  We knew of the side effects of deflazacort, including slowing down or stopping growth for a period of time, but we chose to put him on steroids.  I'm hoping nothing horrible happens to him, these boys go through too much as it is.

Michelle,

 

What deletion does you son have, 51?  If so, where/what about the exon skipping trials?  My son has exon deletion 51-55.  Would the exon skipping trial you mentioned help him?  We're thinking about the IGF-1 at this time...

 

Thanks!

Darcy

 



Michelle Gonzales said:

My son had an argenine test in Cinci.  They gave him argenine through an IV which ran through quickly.  The whole test lasted about 2-1/2 hours and they withdrew blood every half hour.  This test showed that he is growth deficient.  He hasn't grown in over 18 months and he is 8.  I was told from the doctor that the Humotrope will not miraculously make him as tall as he should have been if he was without steroids, but it will give him more height.  I also was told that about 90% of DMD boys seen at Dr. Wong's clinic are on growth hormones.  Also, Dr. Rutter at Cinci has already started the IGF-1 study.  It lasts 6 months.  The only reason we chose not to be in that study is because exon skipping is supposed to start in September and to be in that trial, Nicolas cannot be in any other study for a 3 month period (it's either 3 or 6 months).

 

Just like with everything there are side effects and some of them are serious.  One side effect that Dr. Rutter's nurse mentioned is a displacement of the hip joint.  She said she saw this 2 times before, but stated this is the only side effect she has seen with Humotrope.  The other she mentioned and Dr. Rutter mentioned is contractions in DMD boys.  They have to be aggressively stretched.  Of course we will keep an eye on any and all side effects.  But even aspirin has side effects.  We knew of the side effects of deflazacort, including slowing down or stopping growth for a period of time, but we chose to put him on steroids.  I'm hoping nothing horrible happens to him, these boys go through too much as it is.

Nicolas has exon deletions 45 through 50 so the next rung is exon 51, which is what Nicolas needs to skip over his exons to get to 51.  So hard to explain but there are so many discussions on this board to explain exon skipping.  Unfortunately, it doesn't seem like your son will be eligible.  I think your son would have to be on a trial for exon skipping 56.  The exon skipping trials we are considering is of course Dr. Wong's GSK study - since he sees Dr. Wong, and also Dr. Mendel's trial because I signed him up for both and for us, it's a first come first serve basis.  We need to start something soon, and the FDA just keeps pushing things out with all their red tape.  It's frustrating.  If you are wanting to start IGF-1 study, I would recommend that you talk to the research team at Cincinnati, or Dr. Rutter's office.  Her nurse's name is Bonnie and is wonderful.

I think Char mentioned another serious side effect her son had while on GH here:

http://community.parentprojectmd.org/forum/topics/1187424:Topic:107745

Michelle Gonzales said:

My son had an argenine test in Cinci.  They gave him argenine through an IV which ran through quickly.  The whole test lasted about 2-1/2 hours and they withdrew blood every half hour.  This test showed that he is growth deficient.  He hasn't grown in over 18 months and he is 8.  I was told from the doctor that the Humotrope will not miraculously make him as tall as he should have been if he was without steroids, but it will give him more height.  I also was told that about 90% of DMD boys seen at Dr. Wong's clinic are on growth hormones.  Also, Dr. Rutter at Cinci has already started the IGF-1 study.  It lasts 6 months.  The only reason we chose not to be in that study is because exon skipping is supposed to start in September and to be in that trial, Nicolas cannot be in any other study for a 3 month period (it's either 3 or 6 months).

 

Just like with everything there are side effects and some of them are serious.  One side effect that Dr. Rutter's nurse mentioned is a displacement of the hip joint.  She said she saw this 2 times before, but stated this is the only side effect she has seen with Humotrope.  The other she mentioned and Dr. Rutter mentioned is contractions in DMD boys.  They have to be aggressively stretched.  Of course we will keep an eye on any and all side effects.  But even aspirin has side effects.  We knew of the side effects of deflazacort, including slowing down or stopping growth for a period of time, but we chose to put him on steroids.  I'm hoping nothing horrible happens to him, these boys go through too much as it is.

We did not start him on both medications at the same time.  Connor had at least 7 months between.  It may seem close enough but we had already seen improvements in existing muscle function with the steroid prior to starting the GH.  We've seen a bigger improvement since starting the GH. 

Ofelia Marin said:
Right. GH helps the boys grow. The only thing that I do not know, since there is no data supporting it, is if they help improve strength in any way. In other words, if your son started steroids and GH around the same time, at age 5.5 the strength improvement might be due to steroid and age alone. We cannot say if GH would contribute to that at all until a clinical trial is conducted. From what I heard Dr. Wong's team has several boys on GH (older ages) but the results obtained so far are mixed, one cannot say that GH does more than helping the boys grow at this point...which of course is important, being too short cannot be good for their self confidence... Then you run into the other thing doctors say when recommending steroids, "short is good in DMD"...either way it's hard to know what's best to do, a personal decision as everything else in DMD.

Liisa Underwood said:

@ Ofelia:  I agree that it is not a proven DMD therapy and is not for every kid.  Connor was already deficient when we went to get a baseline.  There will never be an answer to the question:  Was he deficient becuase of the steriods or not? However, I do like the fact it has improved the functionality he does have.  It has given him much more confidence in himself, especially as he is finally coming to a point where he understands that his body won't do what he wants it to do all the time.  He also understands that his body's ability to do things is different from his friends.

 

In any case, I am very happy we decided to it with him.  He's a real trooper for taking the shots.  And, we are closely monitoring him with the doctor.

Good to know, thanks Liisa!

 

It's also good to hear that the IGF-1 trial started! I'm looking forward to seeing some data...all our boys might need to be on GH at some point in the future, it would be great if they prove effective in improving strength in addition to helping them grow. When I asked Dr. Wong 2 months ago, she just said that their results are mixed, and that they recommend them to the boys deficient to help with growth (no mention about strength improvement). I think there was a statement posted here about GH therapy from Cincinnati Childrens last year?



Liisa Underwood said:

We did not start him on both medications at the same time.  Connor had at least 7 months between.  It may seem close enough but we had already seen improvements in existing muscle function with the steroid prior to starting the GH.  We've seen a bigger improvement since starting the GH. 

Ofelia Marin said:
Right. GH helps the boys grow. The only thing that I do not know, since there is no data supporting it, is if they help improve strength in any way. In other words, if your son started steroids and GH around the same time, at age 5.5 the strength improvement might be due to steroid and age alone. We cannot say if GH would contribute to that at all until a clinical trial is conducted. From what I heard Dr. Wong's team has several boys on GH (older ages) but the results obtained so far are mixed, one cannot say that GH does more than helping the boys grow at this point...which of course is important, being too short cannot be good for their self confidence... Then you run into the other thing doctors say when recommending steroids, "short is good in DMD"...either way it's hard to know what's best to do, a personal decision as everything else in DMD.

Liisa Underwood said:

@ Ofelia:  I agree that it is not a proven DMD therapy and is not for every kid.  Connor was already deficient when we went to get a baseline.  There will never be an answer to the question:  Was he deficient becuase of the steriods or not? However, I do like the fact it has improved the functionality he does have.  It has given him much more confidence in himself, especially as he is finally coming to a point where he understands that his body won't do what he wants it to do all the time.  He also understands that his body's ability to do things is different from his friends.

 

In any case, I am very happy we decided to it with him.  He's a real trooper for taking the shots.  And, we are closely monitoring him with the doctor.

My son is 9 and has been on GH for almost a year.  He has done great on it. I don't think he has increased in strength, but he has been very stable and has a consistent broad jump of 21 inches.  Although he is obviously unable to function as well on the playground as the "regular" kids-- which is very frustrating for him, he is doing so well otherwise that his diagnosis has been changed by CHOP to "intermediate."  We'll see Dr.  Wong again next week, so we'll see if she concurs.  One thing that was remarkable, was that his CK levels dropped initially from just over 26,000 to just over 8,000.  Three months ago they had risen back to just over 11,000, and will be retested this month.  I attributed that initial drop to the GH, but I guess we don't have any real way of knowing.  In any case, I consider his deficiency a blessing that allows us to give him everything we possibly can right now that even might be helping.
Thanks!  Yes, considering the IGF trail.  In touch.

Michelle Gonzales said:
Nicolas has exon deletions 45 through 50 so the next rung is exon 51, which is what Nicolas needs to skip over his exons to get to 51.  So hard to explain but there are so many discussions on this board to explain exon skipping.  Unfortunately, it doesn't seem like your son will be eligible.  I think your son would have to be on a trial for exon skipping 56.  The exon skipping trials we are considering is of course Dr. Wong's GSK study - since he sees Dr. Wong, and also Dr. Mendel's trial because I signed him up for both and for us, it's a first come first serve basis.  We need to start something soon, and the FDA just keeps pushing things out with all their red tape.  It's frustrating.  If you are wanting to start IGF-1 study, I would recommend that you talk to the research team at Cincinnati, or Dr. Rutter's office.  Her nurse's name is Bonnie and is wonderful.
Please let us know how it goes Darcy! My son is age 3 and not deficient at this point, but very interested for the future.

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