Doptelet (avatrombopag) is a prescription medicine for thrombocytopenia (low blood platelet counts). The FDA approved it in 2018 for adults with thrombocytopenia with long-term (chronic) liver disease to take before a medical or dental procedure, in 2019 for adults with chronic immune thrombocytopenia (ITP) when other medicines did not help enough, and in 2025 for children ages 1 year to under 6 years with ITP when other medicines did not help enough. Doptelet comes as a tablet and as granules (for younger children) that you take by mouth.
How Does Doptelet Work in Thrombocytopenia?
Thrombocytopenia is when your blood has too few platelets (also called thrombocytes). Platelets are blood cells made in the soft, spongy tissue inside your bones (bone marrow). Their job is to form clots that stop bleeding when you are injured. If your platelet count is low, it may take longer for your body to stop bleeding.
Your platelets can be low for many reasons. Your bone marrow might not make enough, your body might destroy them, or your spleen may store too many of them. A hormone in your body called thrombopoietin (TPO) signals the bone marrow to make platelets by attaching to special proteins called receptors. Doptelet also attaches to those receptors and works together with TPO to increase the number of platelets your body makes.
Is Doptelet Right for Me?
Your health care provider may examine you, review your medical history, and check the medicines you are currently taking and have taken in the past. They may also order blood tests to decide if Doptelet is right for you.
How Do I Take Doptelet?
Take this medicine with food.
For adults and children 6 years and older, Doptelet comes as a tablet you swallow.
For children who cannot swallow tablets, Doptelet also comes as Doptelet Sprinkle, which is a capsule that has granules inside of it. To take Doptelet Sprinkle, open the capsule and mix the granules inside the capsule with either a liquid or a soft food. Soft food can include applesauce, strawberry jelly, or plain yogurt, and liquids can include whole or skim milk, orange juice, unflavored electrolyte solution, or water. When taking the mixture, do not crush or chew the granules; they must be swallowed whole. The mixture must also be taken right away; do not save it to take later. You can also follow the "Instructions for Use" that comes with Doptelet Sprinkle to learn more. If your child is prescribed Doptelet Sprinkle, it is important that they do not swallow the capsule or the empty capsule shell that the medicine comes in.
If you are changed from the tablet to the oral granules or vice versa, your dose will be different. One 20 mg tablet is NOT the same as two 10 mg capsules. Always check with your health care provider to make sure that you are taking the right amount of Doptelet.
How Was Doptelet Studied for Thrombocytopenia?
Four studies were done to look at the benefits and risks of Doptelet. Two studies were in adults with thrombocytopenia from chronic liver disease. For people with immune thrombocytopenia, one study was in adults and one was in children.
What medicines did the people in the studies take?
People in these studies were randomly selected to take either Doptelet or a placebo. The placebo looked like Doptelet but did not contain any medicine. Neither the people in the study nor the doctors knew who took Doptelet or the placebo.
For the chronic liver disease studies, adults with platelets below 40,000 took Doptelet 60 mg or a placebo each day for five days. People with platelets between 40,000 and 50,000 took Doptelet 40 mg each day or a placebo for five days.
For children with ITP, those at least 6 years old took either Doptelet 20 mg tablet or a placebo daily. People who were younger than 6 years took the medicine or a placebo as a granule that was mixed into a soft food or liquid. If needed, the Doptelet dose was changed or stopped to keep the platelet count between 50,000 and 150,000.
Adults with ITP took either Doptelet 20 mg tablet or a placebo daily. If needed, the Doptelet dose was changed or stopped to keep the platelet count between 50,000 and 150,000.
Adults with chronic liver disease
Two studies were done (ADAPT-1 and ADAPT-2). The main question these studies wanted to answer was what percentage of people, after taking Doptelet, could have a procedure without needing to receive platelets (transfusion) or other urgent care to control bleeding during the procedure and for up to a week afterward.
Who was included in the studies?
- Adults with chronic liver disease and a platelet count below 50,000
- People scheduled for a procedure that could cause bleeding and would require a platelet transfusion at their starting platelet level
- Procedures included low-risk (like an endoscopy), moderate-risk (like a liver biopsy), or high-risk (like major surgeries or dental work)
Who was excluded from the studies?
- People with a history of blood clots, blood disorders, serious heart conditions, or advanced hepatocellular carcinoma
- People who had a platelet transfusion or taken certain medicines that affect blood clotting or that boost red blood cell production within a week before the studies
What were the study populations?
Across both studies, 277 people took Doptelet and 158 people took a placebo. People were placed in two groups based on their starting platelet count. One group had platelet counts below 40,000 (lower platelet count), and the other had platelet counts between 40,000 and 50,000 (higher platelet count). Their ages ranged from 19 to 86, with an average of 58. Most (about 65%) were male. About 60% were White, 33% Asian, 3% Black, and 3% other.
How long were the studies?
People took Doptelet or a placebo once daily for five days with procedures scheduled five days to eight days after the last dose. Researchers looked at the platelet count on the procedure day and for up to a week after the last dose.
What were the main benefits of Doptelet seen in the studies?
In both the lower and higher platelet count groups, more people who took Doptelet were able to have their procedures without a platelet transfusion or urgent care for bleeding, compared with those who took placebo.
The table below shows the percentages of people who did not need a platelet transfusion or urgent care for bleeding up to a week after having a procedure.
| Study group | ADAPT-1 Doptelet | ADAPT-1 Placebo | ADAPT-2 Doptelet | ADAPT-2 Placebo |
| Lower platelet count | 66% | 23% | 69% | 35% |
| Higher platelet count | 88% | 38% | 88% | 33% |
Adults with ITP
One study was done in adults with ITP. The main question this study wanted to answer was how long Doptelet could keep platelet counts at at least 50,000 over a six-month period. To find out, researchers measured how many weeks people maintained platelet levels of at least 50,000. The researchers also wanted to see if Doptelet could help people take fewer of their other ITP medicines.
Who was excluded from the study?
- People whose ITP that was caused by another condition (secondary ITP)
- People with serious blood clots or other disorders that affect platelets
- People with cancer or serious liver, stomach, blood, heart, or bone marrow conditions
- People who had taken other medicines for low platelets within four weeks before the study
What was the study population?
People with ITP for at least a year and had tried one or more ITP treatments took part in the study. A total of 32 people took Doptelet and 17 people took a placebo. Their average age was about 45 years, and most (67%) were female. About 94% were White, 4% Asian, and 2% Black.
How long was the study?
People took Doptelet or a placebo once daily for 26 weeks. Some people joined an extension study. During this time, any other ITP medicines they were using may have been slowly stopped by researchers. Researchers followed up with all people for a month after the last dose of Doptelet.
What were the main benefits of Doptelet seen in the study?
People taking Doptelet maintained their platelet counts at or above 50,000 for a much longer time without urgent care. They stayed at this level for an average of about 12 weeks, compared with zero weeks for people taking a placebo. About 1 out of 3 people taking Doptelet had other ITP medicines reduced versus none of the people on a placebo. But this difference was not considered strong enough to prove it was a result of taking Doptelet. Only a few people were taking other ITP medicines at the start of the study.
Children with ITP
One study was done in children ages 1 to 17 years with chronic ITP. The main question this study wanted to answer was how well Doptelet worked for those who had ITP for at least six months and who had not improved enough with other medicines. To learn this, researchers measured the percentage of people who reached platelet levels of at least 50,000 and maintained this level for at least six out of the last eight weeks of the study without rescue care.
What was the study population?
People with ITP that had lasted at least six months took part in the study. Almost 3 out of 4 people had tried at least one other medicine similar to Doptelet for raising platelets. Around 2 out of 3 people had tried three or more ITP treatments. Most people in the study (84%) were White, 5% Asian, and 7% Hispanic or Latino. Males made up 52% of those in the study.
A total of 54 people took Doptelet, and 21 people took a placebo. The average age was 9 years for those who took Doptelet (range 1 to 17) and 10 years for those who took a placebo (range 3 to 17).
Who was excluded from the study?
- Children born with low platelet counts, heart problems, or heart rhythm problems
- Children with a history of blood clots or blood or bone marrow conditions, like myelodysplastic syndrome
- Children who had ITP caused by another condition
How long was the study?
The study lasted 12 weeks. Some people joined a longer extension study.
What were the main benefits of Doptelet seen in the study?
None of the people who got the placebo and 28% of those who got Doptelet reached platelet levels of at least 50,000 and maintained this level for at least six out of the last eight weeks of the study without rescue treatment.
What Are the Possible Side Effects, and How Can I Prevent or Manage Them?
People in the studies reported the below problems most commonly.
Fever or viral infections. Watch for fever, tiredness, coughing, a runny nose, or a sore throat. Drink plenty of fluids, rest, and stay home if you are sick. For coughing, pain, or a fever, your pharmacist may suggest medicines that are right for your age.
Headache. Rest, fluids, or warm/cold compresses may help. Your pharmacist may suggest a pain reliever medicine that is safe for you.
Tiredness. Take frequent breaks, pace your activities, and rest when needed. Tiredness usually improves as platelet counts rise.
Bleeding gums, nosebleeds, bruising, or purple/red spots on the skin. Be gentle when brushing your teeth, and do not do activities that may cause cuts or bruises. Keep your nose moist with saline sprays, and gently press the nose if it starts bleeding. Tell your health care provider right away if you have any unusual bruising or bleeding.
If any of these side effects don't go away or get worse, contact your health care provider.
Doptelet and Doptelet Sprinkle can sometimes cause serious side effects, including blood clots. People with chronic liver conditions, chronic or persistent ITP, or certain blood clotting problems are more likely to get a clot. Call your doctor right away or get emergency help if you have any of the following.
- Swelling, pain, or tenderness in your legs
- Fast heartbeat
- Shortness of breath
- Stomach pain or tenderness
- Chest pain
These are not all the possible side effects of Doptelet. Contact your health care provider for medical advice about side effects if you are having symptoms that bother you. You can report side effects to the FDA at 800-FDA-1088 or www.fda.gov/medwatch. You can also report side effects to the drugmaker, Sobi Inc., at 866-773-5274.
How Do I Know if Doptelet Is Working?
If you take Doptelet to raise low platelet counts caused by a chronic liver condition before a medical or dental procedure, your health care provider will order a blood test before you start Doptelet, and again on the day of your procedure. This shows whether Doptelet worked to raise your platelet levels enough to help lower the risk of bleeding.
If you or a child in your care is taking Doptelet for low platelet counts caused by persistent or chronic ITP, your health care provider will order blood tests to check your platelet counts before and during your course of Doptelet, and for at least four weeks after stopping it.
Do not stop taking Doptelet or change your dose unless your health care provider tells you to do so. If you accidentally take too much, call your health care provider or Poison Control center at 800-222-1222, or go to the nearest emergency room right away.
How Do Other Medicines Interact With Doptelet?
Tell your health care provider and pharmacist about all the other prescription and nonprescription medicines, vitamins, supplements, and herbal products you take before starting Doptelet. Some medicines may change how Doptelet works because they interfere with how the medicine breaks down before it leaves your body.
Some medicines can cause Doptelet to stay in your body longer, which can cause more side effects. Other medicines can make Doptelet leave your body too quickly, which can make it less effective.
Call your health care provider if you notice any symptoms of low platelets, which may include heavy bleeding or bleeding that takes too long to stop, easy bruising, bleeding gums, nosebleeds, or small red or purple spots on your skin, or if you see blood when you go to the bathroom.
Talk to your health care provider before starting any other prescription or nonprescription medicines, vitamins, supplements, and herbal products while you are on Doptelet. They will decide if you need to stop taking a medicine while you are taking Doptelet or if you need to adjust the dose of these medicines. Your pharmacist can tell you which foods or medicines interact with Doptelet.
How Can I Get Doptelet?
You will need a prescription from a health care provider to get Doptelet, which is only available at specialty pharmacies. There is a copay assistance program offered by the drugmaker that may allow you to pay as little as $0 per prescription. If you have a chronic liver condition, visit www.doptelet.com/cld/ to learn more. If you have chronic ITP, visit www.doptelet.com/support-and-resources/financial-assistance/ to learn more. You can also contact the drugmaker at 833-368-2663.