{"id":76218,"date":"2026-04-07T13:32:20","date_gmt":"2026-04-07T11:32:20","guid":{"rendered":"https:\/\/www.cmtc.nl\/pedia\/treatment\/medication\/huidige-en-opkomende-medicatie-voor-de-behandeling-van-vasculaire-malformaties\/"},"modified":"2026-04-08T12:40:01","modified_gmt":"2026-04-08T10:40:01","slug":"current-and-emerging-pharmacotherapies-for-the-treatment-of-vascular-malformations","status":"publish","type":"page","link":"https:\/\/www.cmtc.nl\/en\/pedia\/treatment\/medication\/current-and-emerging-pharmacotherapies-for-the-treatment-of-vascular-malformations\/","title":{"rendered":"Current and emerging pharmacotherapies for the treatment of vascular malformations"},"content":{"rendered":"<p><strong>This is a summary of the article written by Emmanuel Seront, An Van Damme, Julien Coulie, Laurence M Boon &amp; Miikka Vikkula.<\/strong><\/p>\n<p><strong>This article explains:<\/strong><\/p>\n<ol>\n<li data-section-id=\"12b35q0\" data-start=\"0\" data-end=\"69\">What vascular malformations are (abnormalities in blood vessels).<\/li>\n<li data-section-id=\"ppvem4\" data-start=\"70\" data-end=\"109\">Why they occur (genetic mutations).<\/li>\n<li data-section-id=\"1rurdoj\" data-start=\"110\" data-end=\"166\">Why traditional treatments are often not sufficient.<\/li>\n<li data-section-id=\"g582un\" data-start=\"167\" data-end=\"234\" data-is-last-node=\"\">And especially: new medications that target the underlying cause.<\/li>\n<\/ol>\n<p>&nbsp;<\/p>\n<h2>What are vascular malformations?<\/h2>\n<p>These are congenital abnormalities of blood vessels. The vessels are structured differently than normal.<\/p>\n<p><strong>Important:<\/strong><\/p>\n<ul>\n<li>They are usually present at birth.<\/li>\n<li>They often grow slowly over time.<\/li>\n<\/ul>\n<p>These can cause symptoms such as:<\/p>\n<ul>\n<li>Pain.<\/li>\n<li>Swelling.<\/li>\n<li>Bleeding.<\/li>\n<li>Reduced function.<\/li>\n<\/ul>\n<p><strong><br \/>\nTypes<\/strong><\/p>\n<ul>\n<li>Slow-flow: venous, lymphatic, capillary.<\/li>\n<li>Fast-flow: arteriovenous.<\/li>\n<\/ul>\n<p>&nbsp;<\/p>\n<h2>Why they are difficult to treat?<\/h2>\n<p data-start=\"0\" data-end=\"25\">Traditional treatments:<\/p>\n<ul data-start=\"27\" data-end=\"133\">\n<li data-section-id=\"1hlhlyt\" data-start=\"27\" data-end=\"38\">Surgery.<\/li>\n<li data-section-id=\"1q59pou\" data-start=\"39\" data-end=\"74\">Embolization (blocking vessels).<\/li>\n<li data-section-id=\"fizo9i\" data-start=\"75\" data-end=\"133\">Sclerotherapy (injecting a substance into the vessels).<\/li>\n<\/ul>\n<p data-start=\"135\" data-end=\"145\">Problem:<\/p>\n<ul>\n<li>Often not fully effective.<\/li>\n<li>Malformation can recur.<\/li>\n<li>Difficult to treat large or complex areas.<\/li>\n<\/ul>\n<p>&nbsp;<\/p>\n<h2>The real cause: Errors in cells<\/h2>\n<p data-start=\"0\" data-end=\"90\">The key discovery: these conditions are caused by genetic changes in blood vessel cells.<\/p>\n<p data-start=\"92\" data-end=\"171\" data-is-last-node=\"\" data-is-only-node=\"\">These errors cause the cells to grow excessively and receive incorrect signals.<\/p>\n<p data-start=\"0\" data-end=\"26\">Key \u201csignaling pathways\u201d<\/p>\n<p data-start=\"28\" data-end=\"77\">You can think of them as the cell\u2019s gas pedals:<\/p>\n<p data-start=\"79\" data-end=\"123\">[Signal] \u2192 [Growth pathway] \u2192 [Cell grows]<\/p>\n<p data-start=\"125\" data-end=\"141\">Main pathways:<\/p>\n<ol data-start=\"142\" data-end=\"186\">\n<li data-section-id=\"1jgmmg1\" data-start=\"142\" data-end=\"164\">PI3K \u2192 AKT \u2192 mTOR.<\/li>\n<li data-section-id=\"p51udu\" data-start=\"165\" data-end=\"186\">RAS \u2192 MAPK \u2192 ERK.<\/li>\n<\/ol>\n<p data-start=\"188\" data-end=\"253\">If these \u201con-switches\u201d get stuck \u2192 the vessel grows abnormally.<\/p>\n<p data-start=\"255\" data-end=\"272\">Simple diagram:<\/p>\n<p data-start=\"274\" data-end=\"327\"><strong data-start=\"274\" data-end=\"285\">Normal:<\/strong><br data-start=\"285\" data-end=\"288\" \/>Signal \u2192 ON \u2192 OFF \u2192 controlled growth.<\/p>\n<p data-start=\"329\" data-end=\"386\" data-is-last-node=\"\" data-is-only-node=\"\"><strong data-start=\"329\" data-end=\"349\">In malformation:<\/strong><br data-start=\"349\" data-end=\"352\" \/>Signal \u2192 ON \u2192 ON \u2192 ON \u2192 overgrowth.<\/p>\n<p>&nbsp;<\/p>\n<h2>New treatments (key part of the article)<\/h2>\n<p>Instead of relying solely on surgery, doctors are now trying to inhibit the cause within the cells using medications.<\/p>\n<h3>Sirolimus (key medication)<\/h3>\n<p>Works on: mTOR-pad.<\/p>\n<p><strong>Effect:<\/strong><\/p>\n<ul>\n<li>Inhibits the growth of abnormal vessels.<\/li>\n<li>Reduces symptoms.<\/li>\n<li>Improves quality of life.<\/li>\n<\/ul>\n<p>It is often used for venous malformations and lymphatic malformations.<\/p>\n<p><strong>PI3K-Inhibitors<\/strong><\/p>\n<ul>\n<li>Even more targeted than sirolimus.<\/li>\n<li>Especially for specific genetic mutations (such as PIK3CA).<\/li>\n<\/ul>\n<p>Advantage: potentially more effective.<br data-start=\"38\" data-end=\"41\" \/>Disadvantage: still limited long-term data.<\/p>\n<p><strong>MEK-Inhibitors<\/strong><\/p>\n<ul>\n<li data-start=\"0\" data-end=\"43\">They act on the other pathway (RAS\/MAPK).<\/li>\n<li data-start=\"45\" data-end=\"86\">Used for fast-flow malformations (AVM).<\/li>\n<li data-start=\"88\" data-end=\"157\">Anti-angiogenesis drugs inhibit the formation of new blood vessels.<\/li>\n<li data-start=\"159\" data-end=\"206\">Examples include Bevacizumab and Thalidomide.<\/li>\n<li data-start=\"208\" data-end=\"262\">Used, among others, for hereditary bleeding disorders.<\/li>\n<\/ul>\n<p><strong>Overview treatments<\/strong><\/p>\n<ul>\n<li>Genetic problem \u2192 Targeted therapy.<\/li>\n<li>PI3K mutation \u2192 sirolimus \/ PI3K inhibitor.<\/li>\n<li>RAS mutation \u2192 MEK inhibitor.<\/li>\n<li>Excessive vessel growth \u2192 anti-angiogenesis.<\/li>\n<\/ul>\n<p>&nbsp;<\/p>\n<h2>Combination of treatments<\/h2>\n<p><strong>Key insight<\/strong><\/p>\n<div class=\"flex flex-col text-sm pb-25\">\n<section class=\"text-token-text-primary w-full focus:outline-none [--shadow-height:45px] has-data-writing-block:pointer-events-none has-data-writing-block:-mt-(--shadow-height) has-data-writing-block:pt-(--shadow-height) [&amp;:has([data-writing-block])&gt;*]:pointer-events-auto scroll-mt-[calc(var(--header-height)+min(200px,max(70px,20svh)))]\" dir=\"auto\" data-turn-id=\"request-WEB:5ab8bf69-a499-4802-8351-eb6557e3db18-23\" data-testid=\"conversation-turn-48\" data-scroll-anchor=\"true\" data-turn=\"assistant\">\n<div class=\"text-base my-auto mx-auto pb-10 [--thread-content-margin:var(--thread-content-margin-xs,calc(var(--spacing)*4))] @w-sm\/main:[--thread-content-margin:var(--thread-content-margin-sm,calc(var(--spacing)*6))] @w-lg\/main:[--thread-content-margin:var(--thread-content-margin-lg,calc(var(--spacing)*16))] px-(--thread-content-margin)\">\n<div class=\"[--thread-content-max-width:40rem] @w-lg\/main:[--thread-content-max-width:48rem] mx-auto max-w-(--thread-content-max-width) flex-1 group\/turn-messages focus-visible:outline-hidden relative flex w-full min-w-0 flex-col agent-turn\">\n<div class=\"flex max-w-full flex-col gap-4 grow\">\n<div class=\"min-h-8 text-message relative flex w-full flex-col items-end gap-2 text-start break-words whitespace-normal outline-none keyboard-focused:focus-ring [.text-message+&amp;]:mt-1\" dir=\"auto\" tabindex=\"0\" data-message-author-role=\"assistant\" data-message-id=\"df4c8831-41d5-4bc0-8be1-9d4f8ba9241e\" data-message-model-slug=\"gpt-5-mini\" data-turn-start-message=\"true\">\n<div class=\"flex w-full flex-col gap-1 empty:hidden\">\n<div class=\"markdown prose dark:prose-invert w-full wrap-break-word light markdown-new-styling\">\n<p data-start=\"0\" data-end=\"116\" data-is-last-node=\"\" data-is-only-node=\"\">The new approach is NOT just medication or surgery alone, but:<br data-start=\"62\" data-end=\"65\" \/>Medication + surgery + embolization = best results.<\/p>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<div class=\"z-0 flex min-h-[46px] justify-start\"><\/div>\n<div class=\"mt-3 w-full empty:hidden\">\n<div class=\"text-center\"><\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/section>\n<\/div>\n<p>&nbsp;<\/p>\n<h2>Future: \u201cPrecision medicine\u201d<\/h2>\n<p data-start=\"0\" data-end=\"107\">Doctors increasingly start by identifying the genetic problem and then choosing a personalized treatment:<\/p>\n<ol>\n<li data-start=\"109\" data-end=\"203\">Step 1: DNA analysis.<\/li>\n<li data-start=\"109\" data-end=\"203\">Step 2: select the appropriate medication.<\/li>\n<li data-start=\"109\" data-end=\"203\">Step 3: evaluate and adjust.<\/li>\n<\/ol>\n<p>&nbsp;<\/p>\n<h2>Challenges<\/h2>\n<div class=\"flex flex-col text-sm pb-25\">\n<section class=\"text-token-text-primary w-full focus:outline-none [--shadow-height:45px] has-data-writing-block:pointer-events-none has-data-writing-block:-mt-(--shadow-height) has-data-writing-block:pt-(--shadow-height) [&amp;:has([data-writing-block])&gt;*]:pointer-events-auto scroll-mt-[calc(var(--header-height)+min(200px,max(70px,20svh)))]\" dir=\"auto\" data-turn-id=\"request-WEB:5ab8bf69-a499-4802-8351-eb6557e3db18-26\" data-testid=\"conversation-turn-54\" data-scroll-anchor=\"true\" data-turn=\"assistant\">\n<div class=\"text-base my-auto mx-auto pb-10 [--thread-content-margin:var(--thread-content-margin-xs,calc(var(--spacing)*4))] @w-sm\/main:[--thread-content-margin:var(--thread-content-margin-sm,calc(var(--spacing)*6))] @w-lg\/main:[--thread-content-margin:var(--thread-content-margin-lg,calc(var(--spacing)*16))] px-(--thread-content-margin)\">\n<div class=\"[--thread-content-max-width:40rem] @w-lg\/main:[--thread-content-max-width:48rem] mx-auto max-w-(--thread-content-max-width) flex-1 group\/turn-messages focus-visible:outline-hidden relative flex w-full min-w-0 flex-col agent-turn\">\n<div class=\"flex max-w-full flex-col gap-4 grow\">\n<div class=\"min-h-8 text-message relative flex w-full flex-col items-end gap-2 text-start break-words whitespace-normal outline-none keyboard-focused:focus-ring [.text-message+&amp;]:mt-1\" dir=\"auto\" tabindex=\"0\" data-message-author-role=\"assistant\" data-message-id=\"cea77f74-6f98-48bc-9b89-933eff2f9f6e\" data-message-model-slug=\"gpt-5-mini\" data-turn-start-message=\"true\">\n<div class=\"flex w-full flex-col gap-1 empty:hidden\">\n<div class=\"markdown prose dark:prose-invert w-full wrap-break-word light markdown-new-styling\">\n<p data-start=\"0\" data-end=\"29\">There are still challenges:<\/p>\n<ul data-start=\"31\" data-end=\"230\" data-is-last-node=\"\" data-is-only-node=\"\">\n<li data-section-id=\"12bsu2p\" data-start=\"31\" data-end=\"79\">Genetic testing is not available everywhere.<\/li>\n<li data-section-id=\"mnrupb\" data-start=\"80\" data-end=\"141\">Medications can be expensive or not covered by insurance.<\/li>\n<li data-section-id=\"1lcp129\" data-start=\"142\" data-end=\"199\">Side effects (e.g., skin issues, metabolic problems).<\/li>\n<li data-section-id=\"1s4pr8p\" data-start=\"200\" data-end=\"230\" data-is-last-node=\"\">Limited long-term knowledge.<\/li>\n<\/ul>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<div class=\"z-0 flex min-h-[46px] justify-start\"><strong>Key message of this article<\/strong><\/div>\n<\/div>\n<\/div>\n<\/section>\n<\/div>\n<p data-start=\"0\" data-end=\"37\">Before: focus on treating symptoms.<\/p>\n<p data-start=\"39\" data-end=\"93\">Now: focus on treating the cause (within the cells).<\/p>\n<p data-start=\"95\" data-end=\"168\" data-is-last-node=\"\" data-is-only-node=\"\">Goal: not always cure, but disease control with improved quality of life.<\/p>\n<p>&nbsp;<\/p>\n<h2>Summary<\/h2>\n<div class=\"flex flex-col text-sm pb-25\">\n<section class=\"text-token-text-primary w-full focus:outline-none [--shadow-height:45px] has-data-writing-block:pointer-events-none has-data-writing-block:-mt-(--shadow-height) has-data-writing-block:pt-(--shadow-height) [&amp;:has([data-writing-block])&gt;*]:pointer-events-auto scroll-mt-[calc(var(--header-height)+min(200px,max(70px,20svh)))]\" dir=\"auto\" data-turn-id=\"request-WEB:5ab8bf69-a499-4802-8351-eb6557e3db18-28\" data-testid=\"conversation-turn-58\" data-scroll-anchor=\"true\" data-turn=\"assistant\">\n<div class=\"text-base my-auto mx-auto pb-10 [--thread-content-margin:var(--thread-content-margin-xs,calc(var(--spacing)*4))] @w-sm\/main:[--thread-content-margin:var(--thread-content-margin-sm,calc(var(--spacing)*6))] @w-lg\/main:[--thread-content-margin:var(--thread-content-margin-lg,calc(var(--spacing)*16))] px-(--thread-content-margin)\">\n<div class=\"[--thread-content-max-width:40rem] @w-lg\/main:[--thread-content-max-width:48rem] mx-auto max-w-(--thread-content-max-width) flex-1 group\/turn-messages focus-visible:outline-hidden relative flex w-full min-w-0 flex-col agent-turn\">\n<div class=\"flex max-w-full flex-col gap-4 grow\">\n<div class=\"min-h-8 text-message relative flex w-full flex-col items-end gap-2 text-start break-words whitespace-normal outline-none keyboard-focused:focus-ring [.text-message+&amp;]:mt-1\" dir=\"auto\" tabindex=\"0\" data-message-author-role=\"assistant\" data-message-id=\"342c2d88-d899-44dc-ba2c-77305ecceec4\" data-message-model-slug=\"gpt-5-mini\" data-turn-start-message=\"true\">\n<div class=\"flex w-full flex-col gap-1 empty:hidden\">\n<div class=\"markdown prose dark:prose-invert w-full wrap-break-word light markdown-new-styling\">\n<ol>\n<li data-start=\"0\" data-end=\"241\">Vascular malformations = congenital abnormalities of blood vessels.<\/li>\n<li data-start=\"0\" data-end=\"241\">Cause = genetic errors in cells.<\/li>\n<li data-start=\"0\" data-end=\"241\">Old treatments often do not work completely.<\/li>\n<li data-start=\"0\" data-end=\"241\">New therapies target molecular processes.<\/li>\n<li data-start=\"0\" data-end=\"241\">Future = personalized treatment based on DNA.<\/li>\n<\/ol>\n<\/div>\n<\/div>\n<\/div>\n<\/div>\n<div class=\"z-0 flex min-h-[46px] justify-start\"><\/div>\n<div class=\"mt-3 w-full empty:hidden\">\n<div class=\"text-center\"><em>Article citation: Emmanuel Seront, An Van Damme, Julien Coulie, Laurence M Boon &amp; Miikka Vikkula (17 Mar 2026): Current and emerging pharmacotherapies for treating vascular malformations, Expert Review of Clinical Pharmacology, DOI: 10.1080\/17512433.2026.2641807<\/em><\/div>\n<\/div>\n<\/div>\n<\/div>\n<\/section>\n<\/div>\n<p><em>Link to full article: <a href=\"https:\/\/doi.org\/10.1080\/17512433.2026.2641807\" target=\"_blank\" rel=\"noopener\">https:\/\/doi.org\/10.1080\/17512433.2026.2641807<\/a><\/em><\/p>\n","protected":false},"excerpt":{"rendered":"<p>This is a summary of the article written by Emmanuel Seront, An Van Damme, Julien Coulie, Laurence M Boon &amp; Miikka Vikkula. This article explains: What vascular malformations are (abnormalities in blood vessels). Why they occur (genetic mutations). Why traditional treatments are often not sufficient. And especially: new medications that target the underlying cause. &nbsp; [&hellip;]<\/p>\n","protected":false},"author":2,"featured_media":2266,"parent":69494,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"aw-modules\/awcf_create_page_templates_with_elementor_content\/page-templates\/page-aw-page-template-67351.php","meta":{"_acf_changed":false,"footnotes":""},"class_list":["post-76218","page","type-page","status-publish","has-post-thumbnail","hentry"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.2 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Current and emerging pharmacotherapies for the treatment of vascular malformations - CMTC-OVM<\/title>\n<meta name=\"description\" content=\"Ontwikkelingen op medicatie gebied vasculaire malformaties.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.cmtc.nl\/en\/pedia\/treatment\/medication\/current-and-emerging-pharmacotherapies-for-the-treatment-of-vascular-malformations\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Current and emerging pharmacotherapies for the treatment of vascular malformations - 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