Canakinumab (Ilaris) muckle wells

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  alicat2011 | Children and Pregnancy, Medication, Treatment & Care, Muckle-Wells, Canukinumab | published about 1 year ago | Originally written in English Canakinumab (Ilaris) muckle wells

  Hi there.
  My name is Alison and I would love to here from who is on Canakinumab (Ilaris) I'm on it, and next month will be my 5th dose. Also, if you have Muckle Wells, how has is your life improved?

  I really suffered with the rash, sore eyes and joint pains which had a major effect on my childhood and now touch wood thanks to Canakinumab (Ilaris) it is kept at bay

  My 3 year old child also suffers and I am thinking about once she turns 4, starting her on treatment that way she will have a better childhood hopefully.

  Reply to me with your thoughts...
  27 replies to the topic — see all replies

  • 
    Moss published about 1 year ago | Originally written in English

    Hi Alison

    I am glad you found the website, its has been very helpful for me.

    I have been on Canakinumab since March 2010 and have had 5 doses. After the 3rd dose, mine was increased to 300mil. I can say that the medicine has had a very good effect on me. Whilst not Muckle Wells, I also had the three symptoms you mentioned and all have improved as have many of my other symptoms. The most noticeable improvement was after the increase in dosage.

    I am sorry to hear your daughter suffers. The one area I am fortunate is my caps is not hereditary and daughters have not been affected. However, if you speak to the Doctors they will give you the right advice and Canakinumab may offer great hope.

    If I can offer any help, please contact me.

    Regards

    Alan
  • 
    karend published about 1 year ago | Originally written in English

    Dear Alison,

    Thanks for your post! I am sorry that I missed it a few months ago, but was offline for a bit after a surgery (I am on the mend now!) My son has NOMID, a form of CAPS and has been on the Ilaris since Aug. of 2008. He was in a clinical trial, but has been getting it through our insurance for over a year. He is currently on an 8 mg/kg every 6 weeks dose to cover his symptoms, which is 4 times the normal starting dosage for patients, and he needs it every 6 weeks to keep him controlled. He did not start at that high of a dose, he moved up to that over time, but the drug works very well for him, as long as he is getting the right dose. Before he was on Ilaris, he was on the anakinra as a daily injection.

    Where do you live? I am in the US, and also run The NOMID Alliance for patients with CAPS and other autoinflammatory diseases www.nomidalliance.net and know of many patients and children that are doing well on the Ilaris. I think that your daughter will be very happy to have this medication, even though it is a shot every few months. I would advise you to get all her pre-school vaccinations done before going onto this medication, especially if she needs a second dose of the MMR (Measles Mumps Rubella) or Varicella (chicken pox) vaccines. Once she is on any of these Il-1 modifying drugs (anakinra, Ilaris, arcalyst) they have advisories about live attenuated vaccines.

    She can have the other vaccines, and inactivated flu vaccines, and they are still determining things with the MMR and Varicella vaccines. My son started anakinra when he was 4.5 years old and it changed his life! He has to get a titer to test his immunity with the MMR and Varicella since he has not been able to have the second doses, but has been fine so far. I would not hesitate to start medications for this reason, but would just say get the shots that you can first, so it will not be an issue!

    You can contact me personally if you want to talk more about this, or anything.

    Best wishes,

    Karen Durrant
  • 
    rkerry1979 published about 1 year ago | Originally written in English

    Dear Alison,

    My seven year old daughter has just been diagnosed with muckle wells, and started on Canakinumab in December 2010, she has been brilliant since starting on it. I am trying to find more information about muckle wells so I can give it to her teachers and GP, if you can help me I would be grateful.

    Many thanks

    Rachel Tansley
  • 
    karend published about 1 year ago | Originally written in English

    Dear Rachel,

    If you send me a private email to site_manager@nomidalliance.net I can send you some of our CAPS medical guidebooks, and CAPS info cards for you to give out to teachers, doctors or anyone! The cards are the size of business cards, and have all the info about CAPS (in a nutshell) on them.

    My name is Karen, and I run The NOMID Alliance. I also have a child with CAPS that is 11.5 years old. I hope that we can help you with more information. The CAPS guidebook is also on our website nomidalliance.net at this link: www.nomidalliance.net/downloads/finalCAPSb...

    I hope this helps! Sorry I did not see your message sooner. I am not sure how to best follow the forums on this great site.

    Karen
  • 
    Jennie39939 published 5 months ago | Originally written in English

    Alison,
    Hello, I'm new here, just joined today. My name's Jennifer. I was born with MWS but wasn't properly diagnosed until I was 20 years old. I'm now 26. When I was diagnosed, they put me on Kineret first. around 2007/2008 I think, my doctor asked me to participate in a study for a new drug - Illaris. I've been on Illaris ever since then. At first I got the injection every 8 weeks, but I suffered a side-effect. I would sometimes get vertigo really bad within a few hours to a couple days after the injection. The vertigo symptoms (dizziness, nausea, vomiting) would last for a few hours and was so bad that I couldn't get out of bed. To fix it, my doctor suggested i get the dose every 7 weeks instead of 8. I don't know how taking it more often helps, but it does. I still sometimes suffer from vertigo, but it's much less often. I'd say about once or twice a year now. Other then that, I love it. All my MWS symptoms are gone now. I haven't had rash/hives, swollen joins, chills, or fevers since I started taking Illaris. I still get redness in my eyes once in while and I still sometimes feel "run down" or tired, but it's a much more normal amount than it used to be. Like you, my childhood was extremely hard before I was diagnosed. I support your decision to try to get your daughter on Illaris and I hope it works for her. She might not like have get injections, but it's far better than trying to go to school with a flair up. Those were hard times! Good luck to you.

    Jennifer
  • 
    karend published 5 months ago | Originally written in English

    Dear Jennifer,

    Thanks for joining this group, and sharing your story! My son is on Ilaris for NOMID, a form of CAPS and was in the Ilaris study for MWS and NOMID in 2008. He was on anakinra for 4.5 years before going on the Ilaris. Before going onto medications, he had frequent bouts of headaches, nausea and vomiting (often waking un early in the morning having this go on) along with fever, join pains, extreme thirst and the CAPS rash. The doctors found that he had elevated brain fluid from CAPS inflammation that would increase during flares. He also had papilledema which is pressure on the optic nerves in the back of the eyes due to the brain pressure being chronically elevated. This is commonly seen in patient with NOMID or severe MWS. Once he went onto medication and had his inflammation under control the headaches, brain pressure, papilledema and the terrible bouts of vomiting with the flares for hours went away, Whenever he needed to increase his dose of anakinra, the headaches and such would return.

    Once he went onto the Ilaris is 2008, he was put onto the lowest dose at first, and had a return of very bad symptoms of the headaches, nausea and vomiting, and even had some episodes of a loss of consciousness due to the the increased brain inflammation symptoms, but the doctors first wanted to call it "vertigo" which it was not that! This was completely related to elevated inflammation from CAPS, and went along with elevated CRP and other labs too, and other symptoms like the headaches, joint pains, etc. They raised his dose and it went away, but then came back around 6 months later, and a few other times. It was hard sometimes to convince the doctor for the study to consider going up on the dose when we would see signs of inflammation coming on a few weeks before the dose was due again, and they made him wait until he was having bad symptoms and elevated labs to go up on the dose. He ended the study in January 2010, and was on a 6 mg.kg every * weeks dose. A few months later, he had a return of very bad headaches, loss of consciousness with vomiting, nausea etc that was coming on within the last 2 weeks of needing his next dose. At that time he was getting the medication on our insurance, and the doctors did a spinal tap that proved he did have elevated brain pressure during these incidents, and it was not vertigo.

    They raised his dose to 8 mg/kg every 6 weeks ,and he has been very stable on that dose for over a year, with only occasional joint pains coming on sometimes before his next dose is due, and only one time with headache (no nausea, vomiting or loss of consciousness.)

    I have heard of some others having this symptom, and they had also been confirmed to be having elevated brain pressure too that was causing it, and it improved at higher doses.

    Some people may be having vertigo instead of elevated brain pressue, and I am not trying to diagnose anyone, but that is generally caused by disruptions in the inner ear. I am just letting you know about this other possibility to consider, and to have you discuss it with your doctor.

    You can read about this go to page two for more info on vertigo. www.webmd.com/brain/tc/dizziness-lighthead...

    My son does not have vertigo symptoms. When he is having issues, he often will awaken in bed from sleep early, and when he sits up he starts to vomit, and when he lays down it is lessened, but that is often associated as a symptom of elevated intercranial pressure, and not vertigo. Here is a list of common symptoms of chronic elevated intercranial pressure, and you will note that early morning emesis (vomiting) is on that list too!

    Chronic increased ICP symptoms:

    Chronic, progressive headache
    Seizures
    Early morning emesis
    Change in school performance
    Altered mental status
    Cranial neuropathy (eg, sixth cranial nerve palsy)
    Focal neurologic deficits
    Papilledema
    Visual changes

    www.pediatriccareonline.org/pco/ub/view/Po...

    Let me know if you need anything, as I also run The NOMID Alliance (www.nomidalliance.org) and we are here to help people with CAPS, and other autoinflammatory diseases.

    Have a great day!

    Karen Durrant
  • 
    Jennie39939 published 5 months ago | Originally written in English

    Karen, thanks so much for letting me know about the ICP! I had no idea that elevated brain pressure was even a part of this. I will read through all the forms and ask my doctor about it at my next visit or sooner if I think I might have ICP. I do have a hearing loss do to inflammation damaging my inner ears (from the MWS) so vertigo is also a likely possibility, but I've never experienced vertigo until after I started Illaris. I did sometimes vomit during fevers and flair ups before I had any medication - but I never felt dizzy before - just sick like I had a cold or the flue. I'll take a look at the links and read through everything and talk to my doctor. Thanks so much.

    Jennifer
  • 
    JacquiAdkins published 5 months ago | Originally written in English

    I was recently diagnosed with MWS and received my first injection of Ilaris today. :) I will report back with any life changes. I too am affected daily with the rash, joint & eye pain, and fatigue. I am really looking forward to feeling results!
  • 
    AnnaKolev published 4 months ago | Originally written in English

    I'am writting from Russia
    My daugher is affected daily with the rash from her born. We went from doctor to doctor 2 years and no result. that september we were in Moscow gene labolatory and they wrote MWS. She is on very extremly severe diete eating nothing as we all that time thought it ia allergy. We use antigistamin drops every day. Please advice us what to do as our doctors have not much experience in all that.
    Thanks
    Anna
  • 
    paul published 4 months ago | Originally written in French

    Annokolev, Hello from France

    welcome on the CAPS forum, I notice with pleasure that this forum is truly international.

    for your daughter: how old is she? it is important for the treatment.

    If indeed the genetic test indicates MWS, there's not use not have your daughter diet because I was myself on diet (I am going to be 70) for several years !! it made no difference at all.

    you can tell your doctors to look at the www.orpha.net/ website
    it is a site for rare disease in 6 languages including English

    on this site you will even find information on

    1 -muckle and wells syndrome established by Professor Gilles Grateau who I consulted in his hospital in Paris.

    2 - associations of patients, but only those available in Europe, including ours www.amws-cinca.eu.

    unfortunately our site is only in French.

    but on this Eurordis site, you will find the address of the American association of Karen Durrand who is active on this forum. It is en English

    www.nomidalliance.org

    so you can look at my posts on this site CAPS and you will notice that I am well treated for 5 years now, with KINERET (commercial name) which is ANAKINRA (international generic name)

    it is a subcateneous shot that works very well because after 48 hours symptoms disappear.

    does your daughter have hearing trouble? if yes, kineret can help with that, I have seen young children not needing hearing aids any longer.

    i don't know if this drug is available in your country, I will find out and let you know.

    I would be happy to hear from you.

    with this forum you won't be alone anymore

    With best wishes

    Paul Rivière president amws-cinca

    Notice: This text content has been translated automatically by a third-party service.
  • 
    AnnaKolev published 4 months ago | Originally written in English

    Hi Paul THanks a lot for your answer. My gaughet is 2 yeras old. Only red spots every day disturb. She hear good we also checked her on ther computer.She is on severe diet neally eat nothing. Is it necessary in that deasease to keep diete? not to eat sweets milk fruts? I know that one medicine can be used from 4 age children and Anakinra from 12 years old? is it true? Unfortunatelly all that drugs are not used in Russia
  • 
    paul published 4 months ago | Originally written in French

    Anna,

    so your little girl is two years old and her name is Gaughet?

    the red spots that you mention must be hives.

    Go to our website and search the MWS'symptoms; you will see 3 pictures of hives,one of a child. you can connect directly from this site, see the home page

    There is ABSOLUTELY NO need for your little girl to stop taking milk, fruits, candies and other sweets.

    There is no specified diet for the MWS (except for adults who can gain weight)

    I wrote to the pharmaceutical company that makes the drug; I'm waiting for an answer.

    regarding anakinra, children under 12 use this medicine with success; they began at four years of age.

    There is in Europe and in the United States another drug called canakinumab (ilaris); it is currently available to children under 4.

    There is a treatment protocol in progress for children under 4. I know two little children-a boy and a girl-who successfully receive this treatment.

    I am going to ask the company that makes the drug, which is only manufactured in a laboratory in France for the entire world.

    I'll keep you informed.

    Best wishes and please, kiss your little girl for me.

    Paul

    Notice: This text content has been translated automatically by a third-party service.
  • 
    karend published 4 months ago | Originally written in English

    Dear Anna,

    Thank you for your message to my email, and on the rarediseasecommunities.org page. My name is Karen Durrant and I am the president of The NOMID Alliance (nomidalliance.org)

    I am aware that there are two other children with CAPS that are in Russia that also travel to the United States to the National Institiutes of Health (NIH) in Bethesda, Maryland to get care and also the anakinra medications for their children. I am going to contact the doctor to learn more about how the families are able to do this, and also will contact the drug company that makes anakinra, and the ilaris for you.

    Anakinra is being used in children in the US, and elsewhere even when the patients are infants in research centers, and Ilaris is approved for patients that are 4 years old. There are some Ilaris studies in Europe for children that are going on for children that are 2 years old. ( 1 month of age to 5 years of age) Here is the link to this study, and I am going to see if they will allow you to travel to those clinics in Europe to be in the drug trial of Ilaris. www.clinicaltrials.gov/ct2/show/NCT0130286... The study is being done in Belgium, France, Germany, Spain and the United Kingdom (England), I think that this would be the best opportunity to help your child, with the least amount of travel.

    As for a speical diet, Paul is correct that there is no diet that can improve the Muckle-Wells. Some patients have been misdiagnosed as having allergies, and have been put onto strict elimination diets to see if that helped, before they found the genetic cause for Muckle-Wells, and CAPS. Some children have food allergies, or other allergies that also have CAPS, but those allergies are not from CAPS. My son is allergic to cows milk, but so is his brother that does not have CAPS, and myself. Unless your child has a documented food allergy, you should not be having to restrict their diet. In fact, children with CAPS that are not on medications are at risk for not having enough protein and nutrients while their body is growing, because of the inflammation from CAPS using up so much of their calories and energy. Some children can even be anemic (low iron in the blood, and low counts of red blood cells) due to CAPS inflammation.

    There is a rare disease organization in Russia, and you may also want to contact them for some suggestions on how you can get help outside Russia. The drug company that makes anakinra has a program to help some patients, and I will ask them if this is possible for your family in Russia. It is complicated if the drugs are not approved in Russia, but if you can get them prescribed by a doctor caring for your child outside the country, or into the clinical trials for Ilaris, then it is possible. There are other families that are doing this from Russia. I am going to try to get you connected with them too.

    I am also going to email you this information, and other things to your email that you sent, but I wanted to get this message to everyone, in case others are in need too.

    Best wishes,

    Karen Durrant
  • 
    karend published 4 months ago | Originally written in English

    Same Message but in Russian)

    Дорогая Анна,

    Спасибо за ваше сообщение на мою электронную почту, а на rarediseasecommunities.org странице. Меня зовут Карен Даррант, и я президент Альянса NOMID (nomidalliance.org)

    Я знаю, что Есть два других детей с CAPS, которые находятся в России, а также поездки в Соединенные Штаты в Национальный Institiutes здравоохранения (NIH) в Бетесде, штат Мэриленд, чтобы получить помощь, а также анакинра лекарства для своих детей. Я собираюсь обратиться к врачу, чтобы узнать больше о том, как семья в состоянии сделать это, а также свяжется с наркотиками, что делает компанию анакинра и ilaris для вас.

    Анакинра используется у детей в США, и в других местах, даже если больные младенцы в научно-исследовательских центров, а Ilaris, предназначен для пациентов, которые 4 года. Есть некоторые Ilaris исследований в Европе для детей, которые идут для детей, которые 2-х лет. (1 месяц в возрасте до 5 лет) Вот ссылка на это исследование, и я собираюсь посмотреть, если они позволят вам путешествовать в эти клиники в Европе, чтобы быть в суде над наркотиками Ilaris. Исследование проводится в Бельгии, Франции, Германии, Испании и Соединенном Королевстве (Англия), я думаю, что это будет лучшая возможность, чтобы помочь вашему ребенку, с наименьшим количеством поездок. www.clinicaltrials.gov/ct2/show/NCT0130286...

    Что же касается Speical диеты, Пол правильно, что не существует диеты, которые могут улучшить Макл-Уэллс. Некоторые пациенты были неправильно диагностируется как наличие аллергии, и не будут поставлены на строгую диету ликвидации, чтобы убедиться, что помогли, прежде чем они нашли генетическую причину Макл-Уэллс, и шапки. У некоторых детей пищевая аллергия, аллергия или другие, которые также имеют CAPS, но те, аллергии не из CAPS. Мой сын страдает аллергией на коровье молоко, но так же его брат, который не имеет CAPS, и я. Если у вашего ребенка есть документально пищевой аллергии, вы не должны быть того, чтобы ограничивать свой рацион. На самом деле, дети с CAPS, которых нет на лекарства находятся в опасности, что не достаточно белка и питательных веществ, в то время как их тела растет, из-за воспаления от CAPS использованием так много их калориями и энергией. У некоторых детей может быть даже анемией (низкое содержание железа в крови, и малым количеством эритроцитов в крови) из-за воспаления CAPS.

    Существует редкое заболевание организации в России, и вы также можете связаться с ними по несколько предложений о том, как вы можете получить помощь за пределами России. Фармацевтическая компания, которая делает анакинра имеет программу, чтобы помочь некоторым пациентам, и я буду просить их, если это возможно для вашей семьи в России. Это сложно, если препараты не будут утверждены в России, но если вы можете получить их по назначению врача уходу за ребенком за пределами страны, или в клинических испытаниях для Ilaris, то это возможно. Есть и другие семьи, которые делают это из России. Я постараюсь, чтобы вы связаны с ними.

    Я также собираюсь послать по электронной почте вам эту информацию, и другие вещи к вашей электронной почте, что вы прислали, но я хотел, чтобы это сообщение всем, в случае, другие нуждаются в слишком.

    С наилучшими пожеланиями,

    Карен Даррант
  • 
    karend published 4 months ago | Originally written in French

    Cher Anna,

    Merci pour votre message à mon adresse courriel, et sur la page rarediseasecommunities.org. Mon nom est Karen Durrant et je suis le président de l'Alliance NOMID (nomidalliance.org)

    Je suis conscient qu'il ya deux autres enfants avec des bouchons qui sont en Russie, qui a également voyager aux États-Unis à l'Institiutes national de la santé (NIH) à Bethesda, Maryland pour obtenir des soins et aussi les médicaments anakinra pour leurs enfants. Je vais contacter le médecin pour en apprendre davantage sur la façon dont les familles sont capables de faire cela, et aussi prendra contact avec la société pharmaceutique qui fait l'anakinra, et l'Ilaris pour vous.

    L'anakinra est utilisé chez les enfants aux États-Unis, et ailleurs, même lorsque les patients sont des nourrissons dans les centres de recherche, et Ilaris est approuvé pour les patients qui sont de 4 ans. Il existe quelques études Ilaris en Europe pour les enfants qui sont en cours pour les enfants qui sont de 2 ans. (1 mois d'âge de 5 ans) Voici le lien à cette étude, et je vais voir si elles vous permettront de voyager dans ces cliniques en Europe pour être dans l'essai de drogue d'Ilaris. L'étude est menée en Belgique, France, Allemagne, Espagne et Royaume-Uni (Angleterre), je pense que ce serait la meilleure occasion d'aider votre enfant, avec le moins de déplacements. www.clinicaltrials.gov/ct2/show/NCT0130286...

    Comme pour une alimentation spéciale, à Paul est exact que il n'ya pas de régime alimentaire qui peut améliorer le syndrome de Muckle-Wells. Certains patients ont été diagnostiqués à tort comme ayant des allergies, et ont été mis sur les régimes d'élimination stricte pour voir si cela a aidé, avant de trouver la cause génétique de Muckle-Wells, et CAPS. Certains enfants ont des allergies alimentaires, ou autres allergies qui ont également des CAPS, mais ces allergies ne sont pas du CAPS. Mon fils est allergique au lait de vache, mais c'est aussi son frère qui n'a pas de CAPS, et moi-même. Sauf si votre enfant a une allergie alimentaire documentée, vous ne devriez pas avoir à restreindre leur alimentation. En fait, les enfants atteints de CAPS qui ne sont pas des médicaments sont à risque de ne pas avoir suffisamment de protéines et de nutriments tandis que leur corps est en croissance, en raison de l'inflammation du CAPS en utilisant tellement de leurs calories et d'énergie. Certains enfants peuvent même être anémiques (faible teneur en fer dans le sang, et faible numération des globules rouges) due à une inflammation de CAPS.

    Il ya une organisation maladie rare en Russie, et vous pouvez également les contacter pour des suggestions sur comment vous pouvez obtenir de l'aide en dehors de Russie. La société pharmaceutique qui fait l'anakinra a un programme pour aider certains patients, et je vais leur demander si cela est possible pour votre famille en Russie. C'est compliqué si les médicaments ne sont pas approuvés en Russie, mais si vous pouvez les obtenir prescrits par un médecin s'occuper de votre enfant en dehors du pays, ou dans les essais cliniques pour Ilaris, il est alors possible. Il ya d'autres familles qui font ce de la Russie. Je vais essayer de vous connecté avec eux.

    Je vais aussi à l'email que vous cette information, et d'autres choses à votre e-mail que vous avez envoyé, mais je voulais faire passer ce message à tout le monde, au cas où d'autres sont dans le besoin aussi.

    Meilleurs voeux,

    Karen Durrant
  • 
    Jennie39939 published 4 months ago | Originally written in English

    Anna - It is possible for your daughter to have a food allergy, but this is not related to MWS. I have MWS and can eat anything I want. MWS is a genetic disorder, not an allergy. When I was a child, one doctor thought I was allergic to sunlight because of the rash, but they were wrong.

    No matter what your daughter eats or does, she will still get the symptoms of MWS until she gets proper medication. And I agree with Karen, your daughter should probably be eating more, not less. I was servilely anemic and underweight before I got medication.

    Jennifer
  • 
    karend published 4 months ago | Originally written in English

    Dear Jennifer and Anna,

    I agree with you Jennifer on the food issues. My son that has NOMID also had food allergies, but they are not from CAPS. It did complicate getting a diagnosis, but the food allergies came on after he was a year old, and the CAPS was there from birth. He was super anemic, and underweight before going onto the right medications, and had even stopped growing for over a year and a half before he went onto anakinra in 2004, and had no growth as a baby from 12-18 months after having a serious inflammation around his heart (pericardial effusion, near tamponade-from NOMID too.) Good nutrition is so important, and some children with these diseases have a real challenge with growing and being in the right weight. Some of the more severely affected ones have even needed special formula and extra nutrition to try to help them to grow, but nothing much helped until they went onto the right medications for CAPS.

    Anna, I have contacted some doctors, and emailed you some information. I am working on things so that you can get some way of getting medication for your child soon. The doctors in the US are working on it too.

    Thanks Jennifer for your insights! I really am so glad you are involved in this forum, and others.

    Karen
  • 
    AnnaKolev published 4 months ago | Originally written in English

    HI AL!!!! I can't express my joy that I got answers from you. I will wait information from doctors and medicine. In Moscow clinic we were told that 2 families went to USA by special programme for treatment but that doctоrs don't know the result unfortunatelly. About diet I understood - allergic tests show that we have only on cow milk. I've got question about antigistamin drops - our doctor said to use it every day - is it necessary untill we find wright medicine ?
  • 
    AnnaKolev published 4 months ago | Originally written in English

    Her name is Valeria she was born 18/01/2010
  • 
    karend published 4 months ago | Originally written in English

    Dear Anna,

    Paul from AMWS-CINCA is going to contact the drug companies in the EU for you. I am confident that he will find some very helpful options for your child, and we hope that she will soon be able to get the medications and care that she needs!

    I have gotten some information from the doctors at the NIH, and will email you with that next. It would help if you could email Paul and I privately with your child's doctor's name, contact information and also some information about Valeria, and your contact information. I am going to email you with this request, and will also have Paul's email on it so you can reply with this private information. It will help with the process of finding programs that can help your child, and the doctors that are available to help, either in the EU or the US can contact your doctor to advise them on how to best care for your child.

    For the Ilaris study the Novartis Investigative clinical sites in Germany, Belgium, and Spain are recruiting patients for the study with patients with CAPS that are young children, your child's age or younger. France, The United Kingdom are not recruiting yet, according to this clinical trial website, but often this information is not updated as often as it should. They may be taking patients in those countries now.

    For you, the closest country for the drug trial is Germany, and the Novartis Investigative site in Dresden Germany which is not far from Berlin.

    www.clinicaltrials.gov/ct2/show/NCT0130286...
    Please refer to this study by its ClinicalTrials.gov identifier: NCT01302860

    Contacts
    Contact: Novartis Pharmaceuticals 41 61 324 1111

    Air Berlin has very inexpensive flights from Moscow to Berlin, and they have fights to Dresden, but I do not think that they fly there directly from Moscow. You may find other airlines that are more reasonable.

    Thank you,

    Karen Durrant

    I have some information from the doctor at the NIH.
  • 
    AnnaKolev published 4 months ago | Originally written in English

    Hi Karend
    Thanks I wrote a letter to your mail. I will wait info. Of course to go to Europe for us is better.
  • 
    AnnaKolev published 4 months ago | Originally written in English

    But to tell the truth I am really afraid. It was written in past research that Canakinumab can be used only for children over 4 years old.
  • 
    karend published 4 months ago | Originally written in English

    Dear Anna,

    It is your choice to decide on having your child in this clinical trial or not. You would have to travel to Germany or another country for the clinical trial, but the drug and medical exams should be covered by the clinical trial. I do not know how the Ilaris drug trial is working in Europe, so you should call to find out if there is coverage for travel, hotel or other things in the study. Some drug trials in the United States in the past did offer to help the patients with travel costs related to the clinical trial, if they lived quite far from the study site. I am not sure if that is a part of the European studies.

    41 61 324 1111 is the number to call for Novartis clinical trials. This number is in Switzerland, and they will be able to answer any questions that you may have about the Ilaris clinical trial for your child. They would be the best people to contact with your concerns. I would suggest that you ask about clinical trials in Germany that may be open that are closest to you, such as the one in Dresden, and if they are taking patients from other countries, or only patients from the countries where the drug trials are being done (UK, Germany, Spain, Belgium, France.)

    Once you get to talk to them, and learn more about the study, then perhaps you will be able to decide if this is the right thing for your child, and your family. No one should do a clinical trial if they are not comfortable with the process, so you need to call them and learn about the program.

    I suggested this clinical trial, as it may be the easiest way for your child to get medications right now for CAPS, and the medications are usually provided to the patients by the study, and the patients do not have to buy the medications if they are in the clinical trials (at least that is how it works in the US.)

    There are no listed concerns that I am aware of in the research on Ilaris to suggest that Ilaris would not be safe for patients under 4 years of age, but the drug was only approved for patients 4 and older because the studies that were done were only on children 4 and older. The drug company could not seek approval for 2 years olds or younger until the research was done with younger children. Often drug companies do the research with older children to make sure the drug is safe, and not showing any concerns with growth or other issues that could affect little children before they want to try using it in younger children.

    They are currently doing testing on patients around the world with Ilaris in children under 4 years of age, and in Japan they have been doing tests for a few years, and even have been using it on infants! I am in touch with the Japanese patient organization, and they have been very happy with Ilaris. There are patients in other study sites in Canada, the UK and Europe that have been using this medication for young children, and also patients that are older for awhile too.

    For more information on Novartis in Russian, go to: www.novartis.ru/ru/homepage.shtml

    Another option would be to see if your doctor could prescribe anakinra for your child. That drug is made by Swedish Orphan Biovitrum (SOBI) and this is their contact in Russia. I am not aware that the drug is available yet in Russia, but I would contact them to find out if that is possible.
    Russia
    OOO Swedish Orphan Biovitrum
    E-mail mail.ru@sobi.com
    Preschistenka str. 40/2, bldn. 1
    119034, Moscow, Russia
    Tel +7 (495) 7881130
    Fax +7 (495) 787381

    I also have some information about some programs that may be available from SOBI if there are challenges with access to this medication, or the cost, but you should let me know if this is something that you want for your child, and have a doctor prescribe it, or ask for it for your child first. I cannot contact the drug company for your child unless you or your doctor specifically request this medication ,and want help to get it for your child in Russia.

    It may be easier to do the Ilaris clinical trial, since they usually provide the medication through the study, and that is why I suggested it as the first option. From what I have heard, it has been very hard for patients in Russia to get anakinra prescribed, or available in Russia. I am hoping that this will change soon, and that if that is your choice that we can help you with that process.

    Anakinra is currently being prescribed for CAPS patients of all ages, even infants, but it is being used as an "off-label" prescription for CAPS, There is extensive research on anakinra for CAPS over the past 10 years, and it has been effective, but at this time the drug company has not established official approval or licensing for anakinra specifically for CAPS patients. The drug has approval for use in patients with Rheumatoid Arthritis, and has been used for young patients for years, often in "off-label" prescriptive use, usually through clinical research trials for young children with arthritis, and also CAPS.

    My middle son has been on Ilaris for 3.5 years ,and is doing very well with no problems. He is very happy with it and started on it when he was 8 years old. He was in an Ilaris clinical trial in the US for NOMID and MWS patients. Before that, he was on anakinra from 4.5 years of age. He was in a clinical trial for the anakinra too. We have found that the clinical trials have been well run, and very helpful. Also, it was at first the only way that our son would be able to have access to these medications, and we wanted him on treatment as soon as possible, as he was very affected with his symptoms. We were concerned that he would get worse without the medication, as he has NOMID and had nearly died at birth ,and at 13 months of age due to inflammation in his body from NOMID, including inflammation around his heart at 13 months that made us more worried about what could happen if he went on longer without medication.

    There are always concerns about starting any medication for a medical condition, and every drug has benefits and side effects that need to be considered. For us, we felt that due to the severity of our son's CAPS symptoms, in addition to some being life-threatening, that we felt that taking the risk to try new drugs, and participate in a clinical trial when these drugs were not yet approved for CAPS (Such as Ilaris) or a new treatment for CAPS, such as anakinra, that was a fairly new drug in 2004 were worth the risk. For others that may have had less severe symptoms, or were not at the same level of risk to their heath chose to wait until more research or approval was granted for some of these drugs. Either way is fine, and you should do what feels best for you and your child.

    Best wishes,

    Karen Durrant
  • 
    AnnaKolev published 4 months ago | Originally written in English

    Hi all, Thanks a lot.
    We plan to go to Europe for diagnosis and treatment. We need to do best clinics in Europe in which the disease is treated. Would you be so kind to let me know. No matter study programme or usual treatment by anakinra. I thing it's better to go there first to try how it will go and after to find way how to bring that medicine in Russia
  • 
    karend published about 1 month ago | Originally written in English

    Dear Anna,

    Have you had any success with getting your daughter tested for CAPS, or have you been able to get her the medication? There is another family that has just joined this group that is from Russia, and it would be nice to find out if you have had any success with getting treatment for your daughter.

    Thanks,

    Karen Durrant
  • 
    AnnaKolev published about 1 month ago | Originally written in English

    Dear Karen
    I know - she has already called me. We have sent uor blood tests to NIH USA and after 1-2 weeks the result will be ready. I hope that they will advice me therapy ( if anakinra with which dose it;s better to start with maybe ones a week or I know that in USA be tablet therapy ( asatioprin sometimes ) can be used. By the way we are preparing all document to ministry of health to get permissin to supply anakinra from abroad for our children. I also have seen about study programmes for Ilaris - but they send me to Moscow office - where on information. How much Ilaris cost? Just to understand if our government will manage to buy it for us ? Anna
  • 
    karend published about 1 month ago | Originally written in English

    Dear Anna,

    I am very glad that the doctors at the NIH are helping you! This is good news. I had contacted them, but had not heard what had been done. It is also very good that you are working on trying to get permission from the government to have anakinra for your children. If you need help with this in any way, please let me know. You can email me directly at: karen.nomidalliance.org@gmail.com too. I have some forms that we can use to try to apply for the medication coverage, and I can try to help contact the drug company, but we will need to wait until your genetic testing is back. Your work with the government to get approval for this drug to be brought into Russia will be very helpful for your child, and many others too. Thank you!

    I am very glad that you are also talking with the other family from Russia on this group! I am hopeful that you have also been put in touch with the other 2 families in Russia that are getting help for their children that have CAPS too.

    As for the cost of Ilaris, it is very expensive, but in countries where the drug is approved, the insurance covers most of the cost, and there are patient assistance programs to also lower the cost to patients in the US. Anakinra is not as expensive, but it still costs a lot of money. In America, a month supply of anakinra based on 1 syringe a day of medication may cost around $20,000 a year, but Ilaris averages around $100,000 a year at the standard dosing. For some patients, they require higher doses of these medications. Here is an article about these drugs,a nd cost fro m2011: findarticles.com/p/articles/mi_hb4393/is_1...

    Anakinra works out to be around $1600 for 28 syringes (4 weeks supply). For a young child, this is more than enough medication to cover their needs, even if they need a higher dose, but as they get older, they will need a higher dose..

    One 150 mg vial of Ilaris costs around $16,000 but lasts for around 8 weeks for most patients, so it would cost around $8,000 a month, if you were getting Ilaris dosing every 8 weeks, or every 2 months (6 doses a year). Some patients need a much higher dosing of that medication, so the cost would be higher.

    So Ilaris costs about 8 times MORE money than the anakinra per year. Anakinra is a daily injection. Both drugs work very well for patients, so I am hoping that your government will approve one of them to help your child.

    Ilaris is approved by the European Medicines Agency for CAPS, but at this time anakinra is prescribed for CAPS as an off label prescription, which means that the drug is not officially approved at this time for CAPS by the medical agencies, as the drug was originally designed and approved for Rheumatoid Arthritis.

    I hope this helps!

    Karen