Navigating GI Management in LDS
35:31-39:28
Q:
I will say that there are a couple of themes coming up. One is about medication management and EoE, another's on like EoE. So I'm going to just try and sort through these and go through them in an order that'll help flow nicely. There are a couple of questions about biologics which you did mention, and people asking, you know, if they are at a place where they are being prescribed biologics, they've exhausted other options. Do you have a sense of what works well for patients with LDS and Crohn's? And then if you could also explain a bit more about the process for biologic medications, and if you have a name of a medication that people might want to bring up, that would be great.
EoE（好酸球性食道炎）やIBD（炎症性腸疾患）で使われる生物学的製剤に関して、様々な質問をいただいております。「LDS患者でクローン病持ち。生物学的製剤以外の選択肢がなく、現在その治療を受けている。効果が期待できる生物学的製剤はあるか」「生物学的製剤が効くしくみを教えてほしい」「具体的な薬の名前を知りたい」といった内容です。回答をお願いします。
A:
So right now we do not have a separate treatment algorithm for people with Loeys-Dietz syndrome. One of the big issues with treatment of inflammatory bowel disease, so Crohn's and ulcerative colitis, whether or not you have Loeys-Dietz syndrome, is we currently do not have a good way to predict which medicine is going to work for which person. We just don't have it. Lots of people are working very hard to do that, but we don't have it.
現状、LDS患者さん向けの治療法があるわけではありません。IBD治療における大きな問題の一つは、LDS患者さんであるか否かに関わらず、どの薬がどの患者さんに効くかを予測する方法がないということです。この問題に取り組んでいる人々は大勢いるのですが、成果は出ていません。
Biologic medications come in different classes. So what do we mean when we say a biologic? When we say a biologic, we mean it's an antibody. White blood cells, when they get excited, release little signaling molecules, and those signaling molecules excite the other white blood cells around them. These different signaling molecules have different names: TNF alpha, IL-17, IL-23. The names aren't terribly important, but what these antibodies that you get either by a shot or by an infusion where they put an IV in and they drip the medicine in that way is this antibody go into your body and bind up all of the signaling molecules to prevent the white blood cells from exciting each other.
生物学的製剤には様々な分類がありますが、抗体だと考えてください。白血球が興奮状態になると、シグナル伝達分子を放出します。これらの分子が、周囲の白血球を興奮させます。シグナル伝達分子には様々な種類があり、TNF-α、IL-17、IL-23などの名前がついていますが、名前はそれほど重要ではありません。生物学的製剤（抗体）は、注射や点滴によって投与され、体内でシグナル伝達分子と結合し、白血球がお互いに刺激し合うことを防ぐことで効果を発揮します。
If you get a TNF alpha inhibitor and for you, your disease is primarily driven by the white blood cells sending out too much TNF alpha, it's going to work great for you. If that's not what's driving your disease, that's not going to help. There are many biologics now working on many different pathways. If you've tried, you know, if you've had to switch once or maybe twice, that's actually not terribly uncommon.
質問者さんのご病気が、白血球によるTNF-αの過剰放出に起因するものであれば、TNF-α阻害薬の効果が期待できます。しかし、そうでない場合には、その薬では効果は見込めません。異なる経路で効果を発揮する生物学的製剤は数多く知られていますので、そういった別の作用経路をもつ薬に変更する方法もあります。実際、薬を複数回変更するケースは珍しいことではありません。
If you have really severe disease and nothing is working, I'm happy to talk with your provider about other medicines we've tried in Loeys-Dietz syndrome that seem to work and, based on the pathway, we think are a good idea to try. But for right now, in terms of sort of what medicines your doctor is picking, I don't have any evidence to say that they should pick this over the other. There are lots of really good choices out there.
病気が重度で、どの治療法も効果がないということであれば、ぜひ主治医の先生と直接お話させていただきたいと思います。私たちがLDS患者さんに実際に使って効果がありそうな薬や病気を引き起こしている経路に基づいて効果が期待できる薬についてお話したいと思います。ですが、主治医の先生がお使いになられている薬について、代わりとなる薬は多く存在するのですが、良い悪いを申し上げられるようなエビデンスがないというというのが実情です。
The biologic for EoE - and I'm seeing other questions about this - just want to talk about biologics. The biologic for EoE, that's dupilumab. It blocks another signaling molecule that works with - that's been approved for EoE. So that's an IL-4. There's - I see something about an IL-5. The IL-4, the dupilumab, is the only medication, biologic, that's been shown to work for eosinophilic esophagitis.
EoEの治療薬についての質問もありますので、回答します。デュピルマブという生物学的製剤です。EoEの治療薬として認可されています。この薬はIL-4のシグナル伝達を阻害します。IL-5を阻害する薬もありますが、現状、EoEに対して効果が示されている生物学的製剤はデュピルマブのみです。
The Marfan Foundation did not participate in the translation of these materials and does not in any way endorse them. If you are interested in this topic, please refer to our website, Marfan.org, for materials approved by our Professional Advisory Board.
The Marfan Foundation は、当翻訳には関与しておらず、翻訳内容に関してはいかなる承認も行っておりません。このトピックに興味をお持ちの方は、Marfan.org にアクセスし、当協会の専門家から成る諮問委員会が承認した内容をご参照ください。
