A Hematologist Answers Questions About Treating Idiopathic Thrombocytopen

What are some of the conventional ITP treatments?

There are several types of effective treatments for ITP to raise platelet counts and reduce the risk of serious bleeding.

Steroids. Steroids are often used as a first-line treatment. They suppress the immune system, which can interrupt autoimmune platelet destruction.

Intravenous immunoglobulin (IVIG). IVIG interferes with antibody-coated platelet binding to the receptors on the cells that destroy them. IVIG can be very effective, but responses are usually short-lived.

Anti-CD20 monoclonal antibodies (mAbs). These destroy B cells, the cells of the immune system that make the antiplatelet antibodies.

Thrombopoietin receptor agonists (TPO-RA). These mimic the action of the natural growth factor thrombopoietin and stimulate the bone marrow to overproduce platelets.

SYK inhibitor. This drug interferes with a key functional pathway in macrophages, the cells that are the primary site of platelet destruction.

Splenectomy. This surgery to remove the spleen eliminates the primary anatomical site of platelet destruction. It can lead to long-term remission in certain people.

How will I know if my treatment is working? Will it require testing?

The goal of ITP treatment is to reduce the risk of serious and fatal bleeding by keeping platelet counts in a safe range. The lower the platelet count, the greater the risk of bleeding. However, other factors can influence your bleeding risk, such as your age, activity level, and other medications you're taking.

A complete blood count (CBC) test is used to detect increased platelet counts and determine responses to treatment.

Are there side effects of treating ITP? Risks?

As with any chronic disease, there are risks, side effects, and benefits of treating ITP. For example, suppressing the immune system can work well to treat autoimmune diseases. But this also increases your risk of getting certain infections.

Since there are many effective ITP treatments available, discuss all your options with your doctor. Also, you always have the choice to switch to a different kind of therapy if you're experiencing intolerable side effects from your current treatment.

How can I manage the side effects of treatment?

The most important tool for managing the side effects of treatment is communicating with your doctor. For example, if I know one of my patients is experiencing crippling headaches with IVIG or severe weight gain and mood swings from steroids, my treatment recommendations will change. I'll seek other more tolerable treatment options.

Side effects of certain treatments often respond to supportive care medications. Also, doses may be adjusted based on side effects.

How often will I have to go to the doctor for testing? How important is ongoing testing?

An ongoing relationship with an experienced hematologist is critical for anyone with ITP. The frequency of testing will vary depending on if you're actively bleeding or your platelets are extremely low.

Once a new treatment is started, testing may be done daily or weekly. If platelets are in a safe range because of remission (e.g., after steroids or splenectomy) or due to active treatment (e.g., TPO-RAs or SYK inhibitors), testing can be done monthly or every few months.

Can ITP get better on its own?

For adults with ITP, having a spontaneous remission without treatment is rare (about 9 percent according to one report). It's more common to achieve durable remission after effective treatment.

Some treatments are given for a defined duration in hopes of achieving a prolonged treatment-free period, each with varying response rates. This includes steroids, IVIG, mAbs, and splenectomy. Other treatments are continuously administered to maintain platelets in a safe range. This includes TPO-RAs, SYK inhibitors, and chronic immunosuppressants.

What happens if I stop taking treatment?

Stopping treatment can cause a sudden drop in your platelet count. It can also lead to a high risk of serious or fatal bleeding. How fast and how low platelets can drop after stopping treatment varies among people with ITP.

There is little risk in stopping therapy if your platelet count is in a safe range. Many high-dose steroids need to be tapered slowly over time to avoid adrenal crisis and allow the body to adjust.

Of course, it's important to communicate frequently with your doctor about your concerns and needs.

Will my ITP treatment change over time? Will I be on treatment for the rest of my life?

Since adult ITP is generally a chronic disease, people living with the condition will often cycle through many different types of treatment throughout their lifetime.

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Dr. Ivy Altomare is an associate professor of medicine at Duke University Medical Center. She has clinical expertise in a wide variety of hematological and oncological conditions and diagnoses and has been conducting clinical and health services research in the field of ITP for over a decade. She is the honored recipient of both the Junior Faculty and Senior Faculty Teaching awards at Duke University and has a special interest in medical education for both patients and physicians.