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Stiripentol
Posted by annmaxwell on 19 Feb 2009
Dear All,
The advice below is based on my experience of managing Muir since we introduced stiripentol two years ago.
I am hearing and reading disturbing misinformation about stiripentol. Firstly, it has been referred to by some as the drug of last resort for the treatment of dravet syndrome. My understanding is that whilst dravet syndrome exists on a sliding scale of severity, in its most severe form stiripentol can be extremely effective. I believe stiripentol is being referred to as a drug of last resort within the NHS simply because it is expensive, not because it is not effective. There is also a level of fear which comes with a lack of understanding about the drug.
Equally, it should not be dangerous, if prescribed correctly. The incidence of children going into a comma on stiripentol is because it is being prescribed inappropriately. Stiripentol is a very powerful drug that changes the metabolism, enhancing the effectiveness of other drugs. Stiripentol should always be introduced gradually as epilim and clobazam are reduced. If the combinations are wrong then the drugs become toxic which is very dangerous.
All of the above is based on my experience of these drugs for Muir (aged 12) and lengthy discussion with an emminent professor of pharmacology. Muir's epilim was originally reduced by 60% and is now down by 90% of the original prescription before stiripentol, his clobazam hasn't changed but he was always on the bare minimum and I have reduced his stiripentol to 50% of that originally prescribed.
I tried removing clobazam but his seizures returned. As yet I have not tried removing epilim even though he is down to just 100mg twice daily. I will report back on the result if I ever do.
We now have 100% seizure control and a very stable child who is responsive, happy and developing in a way that just wasn't possible until two years ago. If only stiripentol in combination with epilim and clobazam had been the drug of choice 10 years ago he would be a different child from the one we are managing now.
Stiripentol is new and it is not properly understood by the many of the medical profession both in terms of the benefit and equally the dangers. The benefits are huge in terms of seizure control for dravet syndrome children but potentially fatal if not prescribed in the right combination and in the right quantities.
Based on our experience it should definitely not be ruled out and you the parent are the best judge as to how your child is responding, although I would always recommend that you discuss matters relating to medication with your child's consultant first. That said, if you think your consultant is being unreasonable (and some are!) direct them to Sameer Zuberi at Yorkhill in Glasgow who is an authority on stiripentol for dravet syndrome children.
Good luck and best wishes,
Ann
9 Comments
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Kate Watts replied on 8 Jun 2009 at 11:24
It's terrible to hear that some UK doctors/PCTs are refusing stiripentol. It's been the best drug we've tried for Laura. She's been on it for 7 years now and is curently on it with a low dose (5mg) of clobazam and a low dose (50mg) of Topamax. She had to come off valproate due to blood problems but still has good seizure control with this combination.
I know the price has gone up dramatically (800%) in recent years but if it cuts PICU admissions and prevents brain damage (like Laura has got) then it is well worth it.
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justin james replied on 21 Jul 2009 at 16:49
hi kate and ann
cole is due to start it soon and we are just playing the waiting game. most dravets kids have to go into hospital when they have why is that? i just wish they would hurry up grrrrrrrrrrrrrrrrrrrrrrrr
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ann replied on 22 Jul 2009 at 09:29
Justin,
Its probably quite prudent to admit a child going on to stiripentol into hospital. Though I have never heard of it before. Stiripentol should always be introduced gradually as the other drugs are reduced. The only thing I would say is that this process should be long and slow so how long do they intend to keep Cole in hospital for?
In my experience with Muir he was good to begin with. The difficult time came weeks later when the amount of stiripentol was too high and the other drugs had not been reduced enough. Muir lost his appetite for both food and drink and became listless. In the end I sought permission to reduce the amount of Muir's stiripentol to what I had recognised as the optimal amount which was half of what they prescribed.
Later I reduced his other drugs even more - he is on sodium valporate (epilim) and clobazam. The outcome was fantastic. Stiripentol is great as long as your consultant understands how powerful it is. It will dramatically increase the effectiveness of the other drugs Cole is on - up to 100 times!!! That is why we the parents notice a difference immediately. If it is not introduced gradually and the other drugs are not reduced a toxic reaction occurs which can result in death.
It sounds as though your consultant is being cautious. That is either a good sign or a bad sign, depending on how much experience he/she has of stiripentol. You are his father. You will know instinctively when it is going right and when it is going wrong. Follow your instincts! If the combination is right he will most likely improve before your very eyes. If he fades the combinations are wrong.
Good Luck! But have faith. This drug combination, in the right combination really works!
Best wishes,
Ann Maxwell
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Catherine Park replied on 5 Aug 2009 at 16:00
Ann,
We have a 3 year old son called Louis who was confirmed in Oct 08 to have a mutation in the SCN1A gene. He is currently taking Epilim 5ml BD (each 5ml contains 200mg) and Clobazam 8ml BD (each 5ml contains 5mg) and we have been told we can put him on Siripentol as the current medication is not effective. As yet we have not done this as we are trying acupuncture and chinese herbal medicine before we try the three drug combination. As after a meeting with a Dr at Great Ormond Street Hospital he told us that putting him on three drugs may give him undesirable side effects even to the point that we might be better to "learn to live" with his condition without drugs at all.
We started the acupuncture in April 09 and pretty much all his day fits, absences and drops have stopped. We had been told that these were the ones that the drugs definately would not be able to stop. We are still tackling the night ones. We had said to ourselves we would give him 6mths before we put him on the Stiripentol but we have just had a few pretty bad nights over the last couple of weeks with Louis and are now wondering if we shouldn't start the Stiripentol sooner. After reading your information we are concerned that the quantities that the Dr from GOSH has suggested may be incorrect. He has suggested that we reduce the Epilim to 4ml BD and leave the clobazam the same 8ml BD. And start the Stiripentol at 75mg BD and increase this over 4 weeks to 150mg BD. From what you said "Muir's epilim was originally reduced by 60% and is now down by 90%" we are concerned about the doses recommended for Louis. Can you comment? We have an appointment with his local paediatrician, who has not used Stiripentol before, at the end of August and would like to be more knowledgeable about Stiripentol before then.
Thank you in advance.
Catherine
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Ann Maxwell replied on 17 Aug 2009 at 09:46
Hi Catherine,
Please forgive the delay in responding to your post on our forum. I have been on holiday with my family.
I am aware that some people have had success with alternative medicine for their children and I would never rule it out. One father who remains in touch with us has had a fantastic result with cranial osteopathy. This is something we tried with Muir too. Like, you I made a commitment of a year. Although it did not stop his seizures I was always amazed that he sat still through the treatment and seemed to enjoy it.
No one has ever mentioned acupuncture to me before. I am intrigued to hear that you have had some relief from seizures during the period of treatment with acupunture. As you know, it is very difficult for me or any other parent to comment on treatment for our children other than anecdotally. I am not a qualified doctor and therefore I am not allowed to give you advice as such.
I do understand why you might seriously be considering drugs now. I resisted for a very long time, particularly a cocktail of drugs. With hindsight I can say that this decision was wrong for Muir. If only we had introduced him to his current cocktail years ago he may well be less damaged. That said, the drug combination he now benefits from was not understood until quite recently.
Muir is 12 years old. He is on 200mg tablets of sodium valporate, 5mg tablets of clobazam and 500mg powder of stiripentol. Before stiripentol he was on 1000mg tablets of sodium valporate. He has only ever been on 5mg of clobazam which is considered very low. The amount of his prescription is determined by his body mass. If your son is only 3 years old his body mass is substantially less than Muir's and Muir is a tall boy for his age.
I find your precsription difficult to understand because Muir's is in tablet form and precribed in mg where your son's is in mls. You also have not detailed the current level of sodium valporate before the introduction of stiripentol. If your consultant suggests that you would be better off just living with the condition rather than introducing this combination of drugs that advice does imply that he/she doesn't understand the drugs but I have to be careful what I say. After all I am not the doctor!
My advice to you if you are lacking confidence in the proposed prescription would be to seek a second opinion. Why don't you ask to see Professor Helen Cross who is also based at Great Ormond Street? She totally understands Dravet Syndrome and stiripentol. She is hard to see but if you are insistant I am sure she will agree to an appointment. Mention me if you like and she will probably see you and I will get the blame - which is fine by me!
Good luck and best wishes,
Ann
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debbie hawkins replied on 25 Aug 2009 at 12:02
HI, we have just been turned down for stiripentol for our son George, We live on the Isle of Wight...Our doctor said he will try to appeal. So frustrating as his neuro in London said she wants him started on it and would be able to prescribe it for him if we lived back there !! Any ideas on getting it would be greatful.
Thanks Debbie x
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Ann Maxwell replied on 5 Mar 2010 at 10:44
Hi Debbie,
Well done !
So pleased that George is now receiving a prescription of stiripentol.
Let us know how he gets on .
Best wishes,
Ann
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Melanie replied on 8 Jun 2010 at 14:55
Hi Anne
Since we posted last year, we started Stiripentol in Feb this year. We started on 125mg twice a day, along with 5ml of Epilim and 2ml of Clobazam, and in 2 weeks, it went to 250ml, but the side effects were so awful (wobbly and falling over much much more than before) we went back down to 125mg twice a day again and have gradually gone back up to nearly 250mg twice a day. (Although it has vertually stopped his drops and myclonic jerks, he still has prolonged sz's lasting an hour or so when he has a fever- will this improve?)
We mix the packet of 250mg powder with 10ml of water and draw up the required amount into a syringe, now on 9ml twice a day, the other 2 meds are the same amount as before going on Stiripentol, they have never changed!! The problem we have now is that now he is on a high amount, he just cannot keep it down and is sick 90% of the time after taking it. We have tried it on an empty stomach, after food of 2 hours before and no dairy, but he is still being sick afterwards with the reaction in his tummy. We are so worried we don't know what to do. He is a bad eater anyway, so we cannot put it in his food.
Please can anyone give us some ideas on how to administer it without him being sick straight away, we end up giving it half a ml throughout the day and night, but even on this amount, he can still be sick!
Please help.
Thanks
Melanie - Mum to George - 2 1/2 years old
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ann maxwell replied on 23 Jun 2010 at 11:08
Melanie,
On this one I am not qualified to give you advice but something is definitely not right. You should speak with your consultant and if necessary get a second opinion regarding George's prescription.
Muir is nearly 14 years old and for the last 3 years he has been on 14mls of stiripentol twice daily, 20mg of sodium valporate twice daily and 5mg of clobazam twice daily. The latter two are in tablet form hence mg not mls. You need a dr to convert this for you to compare.
Remember every patient is different but the sodium valporate and the clobazam are usually reduced in combination with stiripentol and 9mls sounds a lot for George who is only 21/2.
I really can't give you advice but logically, compared to Muir, George's dose of stiripentol is very high.
Please discuss this with your consultant asap.
Ann