Did you know that Elaprase is expected to have a positive effect on the height of MPS II boys?
As parents, we read the literature and expect that our boys’ growth will slow starting before age 5 and that they are not normally expected to exceed 4 to 5 feet in height. Because Case has always been a tall kid and has continued to stay above the 97th percentile in height and weight, I had wondered whether Elaprase would have an affect on height. This pondering was supported by the fact that a boy we know of from the original clinical trial of Elaprase (so, he’s been on it for 10 years or more) is taller than 5 feet.
If you read the article below, you can see that those on Elaprase for 3 years were able to increase their height, and in boys starting younger than 10, they were able to maintain height stably within the 3-97th percentiles. So, it appears that our boys may be able to grow taller and with a lesser degree of joint contractures than those that came before them. This is one piece of great news! Height challenges just add an additional facet to the already challenging Hunter Syndrome puzzle.
However, let’s be practical… assisting, caring for, and protecting a growing child with cognitive deficits is more difficult the larger and heavier the child. And, as I’ve learned of late, a taller child can reach ever more dangerous things like door slide locks and other things that seemed previously safe from their prying and fast little hands.
So, while this is a great news, I am interested in two additional pieces of information: (1) Will the height growth continue as the children age or will there be a point that they still fall off the charts and still have height challenges. I hope the authors of this study will write a follow-on in three years more time. The Hunter Outcome Survey that produced the data continues until 2022, so hopefully this will happen. (2) Will the cognitive treatments like the IT trial and possibly the Zacharon/Pfizer model successfully improve or stabilize the cognition of our boys so that the growth is positive without new challenges.
I have included the text of the article below or you can download it in PDF form here.
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