{"id":456,"date":"2026-09-23T07:32:47","date_gmt":"2026-09-23T07:32:47","guid":{"rendered":"https:\/\/noimfine.com\/sph06\/?p=456"},"modified":"2026-09-23T07:32:47","modified_gmt":"2026-09-23T07:32:47","slug":"fda-approves-cablivi-new-treatment-option-for-rare-blood-clotting-disorder-in-children-ages-12-years-and-older","status":"publish","type":"post","link":"https:\/\/noimfine.com\/sph06\/2026\/09\/23\/fda-approves-cablivi-new-treatment-option-for-rare-blood-clotting-disorder-in-children-ages-12-years-and-older\/","title":{"rendered":"FDA Approves Cablivi: New Treatment Option for Rare Blood Clotting Disorder in Children Ages 12 Years and Older"},"content":{"rendered":"
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Cablivi<\/a> (caplacizumab) is an injectable medicine used to treat a rare and serious blood condition called acquired thrombotic thrombocytopenic purpura (aTTP). The FDA has approved Cablivi to be used along with plasma exchange and immunosuppressive therapy for treating aTTP in children ages 12 years and older. The medicine was first approved in 2019 for adults with aTTP.<\/p>\n

aTTP is a rare, life-threatening autoimmune blood disorder caused by unusually large molecules of von Willebrand factor (vWF), a blood protein that plays a key role in clotting. Abnormal vWF causes widespread clot formation in small blood vessels, leading to impaired blood supply to vital organs such as the heart, brain, and kidneys. As platelets (blood cells involved in clotting) clump together, few remain available to help with normal clotting elsewhere in the body. Red blood cells may also break apart, leading to anemia. aTTP is a medical emergency that requires treatment right away, often in an intensive care unit.<\/p>\n

Cablivi is a specially designed lab-made antibody fragment that targets the vWF protein in your blood. By blocking the part of the vWF protein that attaches to platelets, Cablivi reduces abnormal clumping of the platelets, decreases formation of harmful clots, and prevents platelets from getting used up too quickly.<\/p>\n

Although aTTP is extremely rare in kids, affecting about 1 in 10 million annually, this approval matters because it\u2019s the first time Cablivi can be used in children, giving younger people a new option for managing this life-threatening condition.<\/p>\n<\/section>\n

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Why Was It Approved?<\/h2>\n

The FDA approval of Cablivi was based on a clinical study that showed that it was effective in restoring platelet counts and reducing the damage to red blood cells in children ages 2-18 years with aTTP for at least a month.<\/p>\n

The clinical study looked at medical records of 30 children ages 2 to 18 years with aTTP, and 29 patients started plasma exchange (lasting for six days) after beginning Cablivi therapy. The median duration of Cablivi treatment was 31 days, and the median study duration was 0.31 years. The results showed that 80% of these patients went into remission, meaning their platelet counts returned to normal and results of a blood test (lactic acid dehydrogenase levels) linked to red blood cell damage stayed near normal for at least 30 days.<\/p>\n<\/section>\n<\/div>\n

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The side effects seen in children were similar to those seen in adults. The most common ones were nosebleeds, gum bleeding, and a fast heartbeat. One child had serious bleeding in the urinary tract.<\/p>\n

Because Cablivi affects how your blood clots, it can raise your risk of bleeding. This is especially true if you\u2019re also taking blood thinners or other clot-busting medicines or have a bleeding condition. If serious bleeding happens, your health care provider may stop the treatment.<\/p>\n<\/section>\n

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What Do I Need to Know?<\/h2>\n

Cablivi is given as an injection. On the first day, you or your child will get one intravenous dose before plasma exchange, followed by another dose under the skin after completing the exchange. After that, you\u2019ll get a daily injection under the skin in the belly area during and after plasma exchange for at least 30 days. If signs of aTTP are still present, your health care provider may extend treatment for up to 28 more days. For children, a health care provider or an adult caregiver must give the injections.<\/p>\n

Before starting the treatment, tell your health care provider if you or your child has had a serious allergic reaction to Cablivi or any of its ingredients in the past. Also, avoid using it for seven days before any planned surgery or dental work. If you or your child is taking blood thinners, your health care provider will need to watch you closely for signs of bleeding. You should always let your health care provider know about all the medicines that you or your child takes, including over-the-counter products, herbal remedies, and vitamin supplements.<\/p>\n<\/section>\n

There is limited information on the use of Cablivi during pregnancy, but it may increase the risk of bleeding in both the mother and the baby. Pregnant women taking Cablivi should be closely monitored for signs of bleeding, and newborns should also be checked after birth. If you are pregnant, planning to become pregnant, or breastfeeding, talk with your health care provider about the potential risks and benefits before using this medication.<\/p>\n

If you or your child notices excessive bruising or unusual bleeding, contact your health care provider right away. Warning signs include prolonged bleeding from cuts, frequent nosebleeds, bleeding gums from brushing, heavy menstrual or vaginal bleeding, red or dark urine, black or bloody stools, pain, swelling, headaches, dizziness, or weakness.<\/p>\n<\/section>\n<\/div>\n<\/div>\n

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Cablivi (caplacizumab) is an injectable medicine used to treat a rare and serious blood condition called acquired thrombotic thrombocytopenic purpura (aTTP). The FDA has approved Cablivi to be used along with plasma exchange and immunosuppressive therapy for treating aTTP in… <\/p>\n","protected":false},"author":2,"featured_media":458,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[11],"tags":[],"class_list":["post-456","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-insurance"],"_links":{"self":[{"href":"https:\/\/noimfine.com\/sph06\/wp-json\/wp\/v2\/posts\/456","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/noimfine.com\/sph06\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/noimfine.com\/sph06\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/noimfine.com\/sph06\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/noimfine.com\/sph06\/wp-json\/wp\/v2\/comments?post=456"}],"version-history":[{"count":1,"href":"https:\/\/noimfine.com\/sph06\/wp-json\/wp\/v2\/posts\/456\/revisions"}],"predecessor-version":[{"id":459,"href":"https:\/\/noimfine.com\/sph06\/wp-json\/wp\/v2\/posts\/456\/revisions\/459"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/noimfine.com\/sph06\/wp-json\/wp\/v2\/media\/458"}],"wp:attachment":[{"href":"https:\/\/noimfine.com\/sph06\/wp-json\/wp\/v2\/media?parent=456"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/noimfine.com\/sph06\/wp-json\/wp\/v2\/categories?post=456"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/noimfine.com\/sph06\/wp-json\/wp\/v2\/tags?post=456"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}