MWS and Canakinumab

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  Yufan | Treatment & Care | published about 1 month ago | Originally written in English MWS and Canakinumab

  My daughter just be diagnosed as MWS 2 days ago. She has serious inflammation on the day she was born. We have been to different doctors and specialists since she is a baby. Now she is 8, has pseudo tumor cerebri, hearing loss, and all other symptoms of MWS. She will begin her treatment 2 weeks later. Her doctor said she is the first case here at the Children hospital. It kind of makes me nervous about the treatment.

  I am still kind of lost here about the disease information and how is the cost of the treatment. Will insurance cover this treatment? I read a article about the cost of Canakinum is 100,000 a year???
  Also any long term effect on this drug?
  I have read some posts. Really helpful. Thanks for those who share their experience. It's let me feel we are not the only one here.
  7 replies to the topic — see all replies

  • 
    karend published about 1 month ago | Originally written in English

    Dear Yufan,

    Thank you for sharing your concerns. I am so sorry that your daughter has been suffering for so long, and I hope that medications will help her soon, You are correct that Ilaris (Canakinumab) is very expensive, but it does work very well for this disease. I am not sure what country you live in, but insurance will cover this medication, and there are programs from the drug company to help with the cost, and co-payments.

    My son is 12 years old, and has been on Ilaris for NOMID, a form of CAPS since 2008. He was on anakinra for 4 years before that, and both drugs work well. There is more risk for infection for any of these medications, but for many that have CAPS, they are doing very well on these medications. The injections are not very painful, and there are minimal side effects, other than the infection risk. There is a lot about Ilaris on the drug company website at: www.ilaris.com/index.jsp and you are welcome to contact me about anything, My name is Karen, and I am the President of The NOMID Alliance, (www.nomidalliance.org an organization that is dedicated to helping patients with CAPS and other autoinflammatory diseases. We are located in the United States, but help anyone in need. Also, there is SWMS-CINCA www.amws-cinca.eu that is located in France that can help you too, and the Canadian CAPS Network.

    Please feel free to contact me with any questions, and you can email me too. We are here to help you! It can be very overwhelming when you are new to this diagnosis, but there is a lot of hope that with the right medications, your child will feel better soon.

    Best wishes,

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

    Thanks Karen, I have read some of your posts and they are very helpful.
    We live in Ohio, so we go to the children hospital at columbus ohio. Could you send me some link or information about the programs from drug company? Or just the site I can search with. I will also go to the ilaris.com to take a look.
    The doctor and nurse are also helpful here, Right now I am doing some research on this disease, and put all of my questions together before our first treatment.
    Thank you for you replay, it does make me feel a lot of better.

    Yufan :)
  • 
    karend published about 1 month ago | Originally written in English

    Dear Yufan,

    Thanks for your message, and I am sorry for the delayed response. My youngest child got sick yesterday, and I was not online. There are a lot of patients in Ohio, and some very good doctors there too. We have a lot of information about CAPS on our website www.nomidalliance.org/learn_intro.php and there is an area about CAPS treatments at this link that may be helpful, www.nomidalliance.org/capstreat.php and links to the drug company sites. We also have a downloadable CAPS guidebook that you can read, or print to take with you or share with the doctor, or others at this link: www.nomidalliance.org/capsguide.php that was written with some of the leading experts on CAPS in the US, and the world.

    Here is the link to the Ilaris website patient assistance program that can help with the insurance and co-pays for medication www.ilaris.com/info/supportProgram/patient...

    Please feel free to contact me anytime to talk about your questions, or anything at 415-831-8782. If you are interested, we are planning some patient picnics for the summer, and will be having one in Ohio for anyone with CAPS, or other autoinflammatory diseases to be able to meet each other in person. There are a lot of people with CAPS in Ohio, of all ages. It is helpful to get to talk to others, and see children and adults with this disease ,and how they are doing now.

    I am sorry to hear that your child was not diagnosed earlier, and has suffered with hearing loss, and other affects of this disease. There are a number of other children in Ohio that have been seen in Columbus, Cincinnatti, Cleveland Clinic, etc. I have heard a lot of good things about
    Dr Sharon M. Bout-Tabaku, MD
    700 Children's Dr
    Columbus, OH 43205 [ map ]
    PH: (614) 722-5525
    FX: (614) 722-3194

    and she has seen others with CAPS too.

    Please give me a call, or email me anytime at karen.nomidalliance.org @gmail.com as we are here to help you, and there is great hope that your child will feel better with these medications. Her hearing loss may not improve, but the medication may help to keep it from getting worse, and other things can improve too. The National Institutes of Health (NIH) in Bethesda, MD is doing a lot of research on CAPS, and can also help to advise your doctors, or you can bring your child there for care too. The Cleveland Clinic is doing a lot for autoinflammatory diseases too, with Dr Spaulding.

    Best wishes, and please contact me. There are many to support you and your child, and we are here to help you in any way that we can. Also, if you have challenges with the medication coverage with insurance, please give me a call and we can help you to navigate that challenge, and direct you to programs that can help.

    Karen Durrant
  • 
    Yufan published 30 days ago | Originally written in English

    Thank you thank you so much for all these information. After reading more about this disease, I feel much better now. At least it has treatments.
    Thanks a million, you are a great help.
  • 
    karend published 29 days ago | Originally written in English

    You are very welcome! Please do not hesitate to contact me for anything. We are here to help.

    Karen Durrant
  • 
    paul published 9 days ago | Originally written in French

    Bonjour Yufan,

    hélas, je constate que mon message, posté voici une quinzaine de jours ne figure pas sur ce forum. dommage !

    Votre fille a-t-elle commencé son traitement ?
    ne vous inquiétez pas pour le traitement, il a fait ses preuves aussi bien sur des adultes que sur des enfants.
    Encore en ce moment un suivi de petits malades (moins de deux ans) de notre association est très positif pour le canakinumab.

    dans notre famille nous sommes dix à être traités, soit avec l'anakinra (kineret) ou le canakinumab (ilaris) et tous nous supportons bien les piqûres sous cutanées sans effets secondaires.

    pour mes deux petits enfants pratiquement de l'âge de votre fille, traités avec le kineret, la piqûre journalière, un peu difficile au début se passe très bien maintenant.

    pour éviter la douleur, il existe des patchs anti douleur.

    un des petits refuse maintenant le patch, c'est en effet une contrainte car il faut penser à l'apposer une heure avant la piqûre.

    en ce qui concerne la perte d'audition, un des petits était appareillé pour aller à l'école. Avec un an de traitement, il a abandonné ses appareils auditifs. Donc il faut garder espoir.

    Pour votre médecin qui voit le premier cas dans son hôpital, il faut savoir que la communauté médicale connait bien maintenant les CAPS. Lors d'une conférence internationale à Amsterdam, dont une représentante de Nomidalliance était présente avec moi et mon épouse, nous avons constaté un échange très important entre associations de malades et médecins du monde entier. Des communications sont régulièrement mises à jour sur pubmed (en anglais)

    j'ai fais le même constat lors d'une réunion organisée à Barcelone (Espagne) par Denis Costello qui gère les communautés au sein d'Eurordis.
    Lors de la visite dans les locaux de leur hôpital, les médecins espagnols nous ont montrés qu'ils sont en communication permanente avec leurs collègues français mais aussi avec d'autres spécialistes mondiaux.

    coût du canakinumab (ilaris) très élevé : en France 12.000 euros par piqûre toutes les 8 semaines. Très bien pris en charge par notre système de santé publique.

    hélas ce n'est pas le cas en Suisse, Allemagne, Belgique, Russie et aussi chez vous aux USA.
    Ce sont hélas des informations transmises par des familles qui ont contacté notre association AMWS-CINCA (www.amws-cinca.eu)

    Vous avez la chance de pouvoir compter sur Karen que je connais bien puisque nous nous sommes rencontrés en France au mois de juillet dernier -voir le compte rendu sur cette communauté dans la rubrique COMPRENDRE sous le titre Cinquième anniversaire.
    ce fut une rencontre très enrichissante.

    à vous lire

    bien cordialement Paul Président AMWS-CINCA (France)
  • 
    Yufan published 8 days ago | Originally written in English

    Dear Paul:

    Thanks for your message. The most fear I had when I got the news was that I could not see my daughter grow up. After reading your message I feel much better. I can see she still can have a normal happy life in the future.
    Thank you very much !!!