Thrombocytopenia is a condition in which the platelet count is lower than normal. Platelets are tiny cell fragments that stick together to help your blood clot — when your counts are low, you’re at an increased risk of bleeding. Platelets are made by specialized cells known as megakaryocytes, which are found in the spongy tissue in your bones (bone marrow). Some health conditions and medications impact your bone marrow and decrease platelet production, or platelets may be destroyed faster than usual.1
If you’ve recently been diagnosed with thrombocytopenia, your doctor will work with you to develop a treatment plan to meet your specific needs. You may also need treatment to address an underlying cause of low platelet count, which may include infection or cancer.2
Thrombocytopenia treatments depend on the underlying mechanism of the condition. Treatments may focus on boosting platelet production in your bone marrow to help prevent bleeding, or decreasing their destruction, if that is the reason for a low platelet count. Another reason for having low platelet count is sequestration (pooling) of platelets in the spleen. The spleen is a spongy organ, which—if it enlarges for other reasons—can keep more platelets inside it. As a result, the machine that counts platelets in the blood produces a lower measurement of platelet counts. You may have surgery to remove your spleen to prevent it from trapping platelets.
Many people living with thrombocytopenia don’t know they have it because they don’t experience any symptoms. Depending on your case, symptoms, such as increased bleeding or spots on the skin, may appear suddenly or slowly over time. Surgery and blood transfusions can manage and, in some cases, cure the disease. It’s possible to live a normal, healthy life with somewhat low platelet count.
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Corticosteroids for Thrombocytopenia
Antibodies are one part of your immune system’s defense against viruses and bacteria. They bind to these foreign invaders, tagging them so that your immune system recognizes and destroys them. While antibodies are extremely helpful in clearing out infections, they can sometimes make mistakes and tag your body’s own tissues and cells for destruction.3
In some cases of thrombocytopenia, your immune system creates antibodies targeted at your platelets. As a result, your platelets are destroyed and counts drop, putting you at risk of bleeding.
Corticosteroids are often the first choice for treating thrombocytopenia. These medications work similarly to the natural anti-inflammatory hormone cortisol to help dampen your immune system and limit antibody production. Corticosteroids help boost platelet levels, typically within two to four weeks of starting treatment. Your doctor will likely prescribe you prednisone in pill form to take daily.4
While corticosteroids are an effective treatment, they come with some unwanted side effects, including:5
- Mood changes
- Increased appetite and weight gain
- Round face
- Increased risk of infections
- Vision problems
- Muscle weakness
Corticosteroids are only prescribed for a short amount of time to avoid health complications such as:
- High blood sugar levels, which may cause diabetes
- Bone problems, including osteoporosis
- Eye problems, such as glaucoma or cataracts
- Fluid retention, leading to swelling in the legs, feet, arms, or hands
- High blood pressure
Intravenous Immune Globulin (IVIG) for Thrombocytopenia
Immune globulin (IG) is a super-concentrated formulation of antibodies made from blood donations. Intravenous IG (IVIG) is used to stop your immune system from attacking your platelets. It’s often given alongside corticosteroids as an IV injection for first-line treatment of thrombocytopenia. You’ll likely only need one large dose of IVIG for your doctor to determine if the treatment is working, but then it may need to be continued on a regular basis.6
Side effects from IVIG are less common than other treatments, and they tend to be mild. Headaches are the most common side effect — others include:
- Flu-like symptoms, including aches and joint pain
- Nausea or vomiting
Rituximab (Rituxan®) for Thrombocytopenia
Rituximab belongs to a class of medications known as monoclonal antibodies. These are specially engineered antibodies designed to target and kill immune cells. Rituximab binds to B cells, which are the cells responsible for making platelet-targeted antibodies. As a result, your immune system makes fewer antibodies, preventing platelets from being destroyed. Rituximab is also approved for treating B cell-related cancers and other autoimmune conditions like rheumatoid arthritis.7
Rituximab is typically given as a weekly IV infusion for four weeks, and many people continue to have normal platelet levels for an extended period of time after treatment. Doctors use this as a second-line treatment if your platelet counts stop improving after trying corticosteroids or IVIG.8
Since rituximab dampens your immune system, you’ll be at an increased risk of infections. If you begin to notice any of these symptoms, call your doctor, as you may have an infection:9
- Joint or back pain
- Night sweats
- Mouth sores
- Feeling more anxious or worried than usual
Thrombopoietin Receptor Agonists (TPO-RAs) for Thrombocytopenia
TPO-RAs are a class of medication used to increase platelet count in thrombocytopenia. Like rituximab, these may be prescribed as a second-line therapy after corticosteroids or IVIG. TPO is a hormone that’s essential for regulating megakaryocyte function and, as a result, platelet production.10
In thrombocytopenia, TPO-RAs work by stimulating megakaryocytes, similar to how the TPO hormone does naturally. As a result, the cells begin making more platelets, raising your counts. These medications are taken as tablets once daily.
Examples of TPO-RAs that are approved by the U.S. Food and Drug Administration (FDA) include:
- Avatrombopag (Doptelet®)
- Eltrombopag (Promacta®)
- Lusutrombopag (Mulpleta®)
- Romiplostim (Nplate®)
- Fostamatinib (Tavalisse®)
In general, TPO-RAs cause mild side effects, such as:
- Increased risk of infection
- Abdominal pain
- Aches and pains
Other Therapies for Low Platelet Count
Other types of therapies in use to treat thrombocytopenia include inhibitors of certain enzymes, such as fostamatinib, which inhibits splenic tyrosine kinase (SYK), and rilzabrutinib, which inhibits bruton tyrosine kinase (BTK).11 Rilzabrutinib received a fast-track designation from the FDA but is not fully approved.
Platelet Transfusions for Thrombocytopenia
In some cases, platelet transfusions are performed to quickly increase your platelet levels. Specialized machines are available to filter platelets out of whole blood given by donors, which can then be used to treat thrombocytopenia.12
During a transfusion, you’ll have an IV with a thin, flexible tube (cannula) inserted into your vein. This is connected to a drip with the platelets, which are infused over the course of 15 to 30 minutes. You’ll be monitored during your transfusion for any signs of an allergic reaction. Let your nurse know right away if you:13
- Develop a fever
- Begin shivering
- Have an itchy skin rash
They will stop the transfusion right away and may give you medication to stop the allergic reaction.
Splenectomy for Thrombocytopenia
In some cases, thrombocytopenia develops when your platelets become trapped in your spleen. This organ is part of your lymphatic system, which filters your blood and stores white blood cells to help fight infections.
Splenomegaly (enlarged spleen) may be caused by one of several health conditions, such as blood cancers, blood clots, or liver problems. These cause swelling of the spleen, trapping platelets inside — as a result, your circulating platelet levels fall, leading to thrombocythemia.14
If you have splenomegaly, your doctor may suggest a splenectomy. During this procedure, your surgeon will remove the spleen with either minimally invasive laparoscopic surgery or open surgery. The larger your spleen is, the more likely you’ll need to have open surgery to remove it. While the spleen is an important part of your body’s function, it’s possible to live without one and still be healthy.
After a splenectomy, you may be at an increased risk of infections. Your doctor will recommend keeping up with your vaccination schedule to help prevent viral infections. If you begin to have a fever, chills, sore throat, or notice any sore spots in your body, call your doctor.15
The Future of Thrombocytopenia Treatments
As research continues on thrombocytopenia, doctors and researchers are finding new ways to treat it. Before these therapies can be used in hospitals and doctors’ offices, they must first go through clinical trials to ensure they’re safe and effective. Currently, there are several clinical trials recruiting participants with thrombocytopenia. These studies offer the opportunity to try new therapies to improve your condition and contribute to the advancement of science in a meaningful way.