A healthy platelet count is crucial for proper blood clotting and preventing excessive bleeding. When a person's platelet count drops too low, a condition known as thrombocytopenia, a healthcare provider may prescribe medications to address the issue. The treatment approach depends heavily on the underlying cause, which could be an autoimmune disorder like immune thrombocytopenia (ITP), a side effect of another illness or medication, or a problem with bone marrow production.
Thrombopoietin Receptor Agonists (TPO-RAs)
Thrombopoietin (TPO) is a hormone that naturally stimulates the bone marrow to produce platelets. TPO-RAs are a newer class of medications that mimic this hormone, directly binding to and activating the TPO receptor on precursor cells to increase platelet production. They are typically used for chronic ITP when other treatments have failed and are also approved for patients with chronic liver disease who require a procedure and for severe aplastic anemia.
Common TPO-RAs include:
- Romiplostim (Nplate): An injectable 'peptibody' administered subcutaneously, usually once per week. It binds to the TPO receptor in the same location as endogenous TPO.
- Eltrombopag (Promacta, Alvaiz): An oral tablet or suspension taken daily. Its absorption is significantly reduced by multivalent cations like calcium and iron, so it must be taken at least two hours before or four hours after meals, supplements, or antacids containing these minerals.
- Avatrombopag (Doptelet): An oral tablet approved for use in adults with ITP and those with chronic liver disease requiring a procedure. Unlike eltrombopag, it can be taken with food.
- Lusutrombopag (Mulpleta): An oral tablet specifically for patients with chronic liver disease needing a procedure.
Corticosteroids
As a frontline treatment for ITP, corticosteroids such as prednisone and dexamethasone suppress the immune system, which helps prevent the destruction of platelets. While highly effective at raising platelet counts relatively quickly, they are typically prescribed for a short duration due to the risk of significant side effects with long-term use. Side effects can include high blood sugar, high blood pressure, mood changes, insomnia, digestive problems, and an increased risk of infection.
Immunoglobulins
Immunoglobulins, or concentrated antibodies collected from donors, can be used to treat ITP and other causes of thrombocytopenia, especially in emergency situations or before surgery.
- Intravenous Immunoglobulin (IVIG): Administered as an infusion, IVIG is thought to work by blocking the immune system's destruction of platelets. Its effect is generally temporary, lasting for a few weeks.
- Anti-D Immunoglobulin: Used specifically for some people with Rh-positive blood who have immune thrombocytopenia. The exact mechanism is not fully understood, but it is believed to stop the spleen from destroying platelets.
Other Immunosuppressants
For patients with ITP who do not respond to corticosteroids or other initial therapies, more potent immunosuppressants may be necessary to control the immune system's attack on platelets. Examples include azathioprine, cyclophosphamide, and cyclosporine. Rituximab, a type of antibody treatment, can also be used to suppress the immune system response that destroys platelets. Danazol, an attenuated androgen, is another option that may provide long-term remission in some cases of chronic ITP. These medications carry a higher risk of side effects, particularly increasing the patient's susceptibility to infections.
Comparison of Key Medications that Can Raise Platelet Count
Feature | TPO-RAs | Corticosteroids | Immunoglobulins | Danazol |
---|---|---|---|---|
Mechanism | Stimulates bone marrow to produce more platelets. | Suppresses immune system and inflammation. | Blocks immune system from destroying platelets. | Reduces immune-mediated destruction of platelets. |
Route | Oral (Eltrombopag, Avatrombopag) or Subcutaneous Injection (Romiplostim). | Oral (Prednisone, Dexamethasone). | Intravenous (IVIG, Anti-D Immunoglobulin). | Oral. |
Use Case | Chronic ITP, liver disease, aplastic anemia (often second-line). | Initial treatment for ITP, typically short-term. | Emergency treatment, rapid boost before surgery. | Chronic ITP (second-line), particularly for long-term management. |
Speed of Effect | Can take 1-2 weeks or longer. | Can show effect within 5-10 days. | Within days, but effect is temporary. | Can take months for full effect. |
Primary Side Effects | Blood clots, liver issues, cataracts. | High blood sugar, high blood pressure, osteoporosis, mood changes. | Headaches, chills, fever, nausea (infusion reaction). | Liver damage, hyperglycemia, virilization (in women). |
Dietary Restrictions | Eltrombopag requires spacing from polyvalent cations (calcium, iron). | No specific dietary restrictions listed. | None specified. | None specified. |
Considerations for Prescribing and Monitoring
Choosing the right medication to increase platelet count is a complex decision made by a hematologist or other healthcare provider. It involves weighing the patient's specific condition, potential risks, side effects, and treatment goals. Regular blood monitoring is essential to track platelet levels and adjust dosages. For example, platelet levels should be checked regularly during TPO-RA therapy to prevent overshooting the target range, which could increase the risk of blood clots. Patients should also be aware of specific monitoring needs, such as liver function tests for those on eltrombopag.
Conclusion
Therapy for low platelet count has evolved, with newer options like TPO-RAs offering targeted approaches to increase platelet production, while older, well-established treatments like corticosteroids and immunoglobulins remain vital in certain clinical situations. For many patients with chronic or relapsed ITP, TPO-RAs like romiplostim and eltrombopag have proven to be effective and durable treatment options. It is critical to work closely with a healthcare team to determine the most appropriate course of action, which may involve a combination of therapies, regular monitoring, and lifestyle adjustments to manage the condition safely and effectively.
For more detailed information on specific conditions like immune thrombocytopenia, consult the National Heart, Lung, and Blood Institute: Immune Thrombocytopenia (ITP) - NHLBI.