Community Discussion Forum Parent support kuvan trial for 2 year old???

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    Avatar of Grace

    At my sons clinic appt yesterday that asked if I woud be interested in having him try kuvan…he just turned 2.. I have a holistic approach in most everything especiall when it comes to by boys. So i just  wanted to know what people know about if from a person perspective, i can read alot about what they want to tell me about it, but i had a hardtime finding personal views, my son has a good chance of not being a responder as he has CPKU, but i would just like more info if anyone has it. side effects, how much of a phe increase did u get ( I am aware it varies person to person) any help would be great. The age you started it and any good and/or bad experiences thanks

    Avatar of carrie

     We were asked to start our 4 month old on it- we declined. I'd heard too many stories about how it worked for a while then seemed to decrease in effectiveness and i thought it best we get used to the diet b/c it would be harder to go back after being on a more liberal diet. I also had fears about how the funding might work long term. 

    although now i wonder- is it time to raise the bar and start working on the other issues of pku- like improving executive .function? could kuvan help with that? i don't know- so many questions. 

    Avatar of Simon

    To be honest, I would not introduce drugs into my infant unless they where nesessary.  For years now many people are growing up just fine with PKU with a good diet in place.  I would just keep him eating green :) and let him make the choice later. 

    In the UK, we have not been told anything of this Kuvan drug.  And from what im hearing, there seems to be a lot of miss guided use of it.  Ive heard comments like take Kuvan and eat what you like.. fairly sure thats not correct… Its clearly states on their site (To be used with a low phe Diet)  and to me that seems pointless if you are on your diet then your phe will be low anyway.. Of course it may help when sick, as that raises your phe levels regardless of diet issues.

    To be honest i think its more of a hype, espicially as its the only drug the pharma companies have at the minute.  The way i see it is if it was great, then every PKU pateint would be given it.

    Avatar of Breanna


    Unfortunately not everyone responds to Kuvan, so giving this medication to every patient with PKU is not possible. If I remember correctly Kuvan only works for approximately 30-50% of individuals with PKU.

    As for people who use Kuvan being able to eat whatever they like, you are right that this statement is incorrect. Kuvan is suppose to be used with a low protein diet, not in place of it. However, some people are able to increase how much phe they can eat if their levels become too low as a result of Kuvan. This is entirely at the discretion of the patients doctor though.

    Other advantages to Kuvan that I have noticed since beginning Kuvan nearly four years ago include more energy, the ability to focus more, better temper control, and more stable phe levels.

    Hope this helps!

    Avatar of Ruth

    I am also from the UK – the reason PKU patients in the UK are not routinely offered a kuvan trial is because the NHS will not fund it at the moment. 

    I disagree with the statement that all patients are growing up just fine on the PKU diet.  Our almost 4 year old daughter has had digestive issues (she didn't do a poo for almost three weeks after starting the baby PKU formula; all other gastro problems were ruled out but we stayed in hospital for 5 days only for a gastroenterologist to conclude it was the formula) and growth delay (we have been seen by private and NHS endocrinologists and they can find no endocrine problems with her, but I have found numerous research papers about PKU patients having growth and bone age delays on the low protein diet) which we have only been able to put down to the synthetic low protein diet. 

    CLEARLY I am not saying she or anyone should not be on the diet – the benefits far outweigh the risks.  But I also think we in the UK should beware of becoming complacent about accepting the diet as our only option when there other other potentially useful therapies available.  These are already being used in the US and other European countries.  There is growing evidence that Kuvan therapy is effective FOR SOME  PEOPLE.   It is clear that the diet is not perfect; it is unpalatable, there are side effects (such as constipation) and as is evident by the numbers of adults not on diet, it is not always easy to stick to.  As the mother of a girl with PKU, I also worry about problems with pregnancy should she not be following a strict diet.

    Also, I am not saying Kuvan is an answer for everyone.  As Breamarie said, it does not work for every patient.  But, as she also says, it has brought other benefits to her such as more energy and focus. 

    I for one would like my daughter to have the advantage at least of doing a kuvan trial to see if she would respond.  If it meant she could have, say, 8 or 10 grams of protein a day rather than her current 5 it could make a big difference to her everyday life.  Add to that the potential for her to do better at school, have better mental health, and have better dietary adherance when she enters her teen and reproductive years, and I think it is worthwhile. 

    I think the more choice PKU patients have the better and I hope the UK government sees fit to start providing funding for Kuvan soon.



    My daughter has Hyper-Phe and It was a tough decision to put her through a trial of Kuvan. She was only three months old when we tried Kuvan and it worked wonders for her. We do NOT regret the Kuvan trial. Of course, she was very closely monitored every step of the way. She is now 9 1/2 months old and her Kuvan dosage has not had to be adjusted at all. She is doing tremendous! Personally, I would recommend trying Kuvan at a younger age, before they reach the stage where higher protein foods become a part of the picture. This has been a Godsend for our family and we are so thankful that she is responding so well. Her phe level is staying right on target where it is supposed to be, nice and level in spite of dietary changes that are currently happening. 

    She is hitting developmental and growth milestones even faster than her older sister did (and she is advanced for her age). We have experienced no developmental delays of any kind with her. I realize this is not the case with every Kuvan user. As a Mom, I want the best for my child, and for Hailey, that meant Kuvan. With Kuvan, my daughter is able to live a healthy, fairly normal life that she would not have had otherwise. Her restrictions are minimal and she is thriving. As to side effects, we have not seen any with our daughter but I understand they are minimal.

    Is it a risk, yes. It is worth it, YES!

    Avatar of Tanya

    We havent been offered a trial as of yet,as our dietian/doctors have decided that until funding is in place, they didnt want to tease him with it. (Increase levels of tolerance)

    Jake has been labelled Mild PKU (From the previous Hyperphe).

    As parents, we decided that we would simply work on and maintain healthy eating choices for Jake, despite the additional work. Placing the foundation for a healthy life if Kuvan never becomes available.

    Jake had a BH4 test when he was about 6 months old, and they were trying to establish why his levels were behaving in the odd pattern they were. As a result, they are confident he will respond well to the drug.

    Jakes development appears to be on track for most two year olds. I felt his speech was delayed…however, since hisbirthday, that has skyrocketted. He is in the 75 percential for growth/size and has been on that curve all his life. (With the exception of course of being 2lbs at birth, arriving 3 months early).

    A couple reasons we decided as we did 1) why introduce new foods, we may later have to take away 2) I feel he needs to be able to communicate side effects before introducing it.

    I think its a great option for those who respond. And I can completely state with confidence, we will re-look at it in the future. I think when he is older and harder for his diet to be achieved, we will need additional help..and Kuvan may be the answer!

    Hope this helps :) I dont think there is a wrong answer. You will know best what will work for you, your family and your child ;)

    Avatar of victoria

    I prefer to stay away from any new drug (kuvan is pretty new for PKU < than 10 years) for a person who is in brain development like 2 years old baby if they have another option like diet and is able to control the levels in normal range with it. I do not discard the future use of Kuvan for my son, when he get older and more difficult to stay in diet control (will be soon after school age). He will be 2 years old next month and ALWAYS kept his levels in excelllet range and his growth chart shows curve over 90 percentile in weight and lenght. His milestones are achive before his older brother (no PKU) and I know that your guys are thinking I'm so lucky and it is true, I'm, but the use of Kuvan it is very personal issue and justread about it as much as you can, made lot of questions to doctors and keep in mind that it is a clinical trial at this time.


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