Rezurock for Chronic Graft Versus Host Disease (GvHD)

Medically Reviewed by Goldina I. Erowele, MBA, PharmD on September 27, 2024
Written by Alexandria McIntire
8 min read

People with severe symptoms of chronic graft versus host disease (GvHD) often need to use multiple treatments to manage their condition. In recent years, a new class of medicines known as kinase inhibitors have been approved for this condition. One of these medicines, called Rezurock, may be a helpful option for people who still have symptoms even after trying multiple other medicines. 

Graft versus host disease is a condition that can occur in people who have received a stem cell or bone marrow transplant from a donor, usually for the treatment of blood cancers or certain other disorders. As the name suggests, this condition occurs when the cells in the donated tissues (the “graft”) attack the healthy cells of the person who received the transplant (the “host”). 

GvHD can be both acute (short-term) and chronic (long-term). Acute GvHD usually appears within the first 100 days after the transplant. Chronic GvHD typically appears more than 100 days after the transplant and, in some cases, may not appear until many years later. About 40%-50% of people who receive a donated stem cell or bone marrow transplant will develop this condition, although certain people are at a higher risk than others.

Chronic GvHD is a long-term condition that can last for years. It affects many organs in the body, causing various symptoms that range from mild to severe. The most commonly affected organs are the skin, gastrointestinal (GI) tract, and liver. When these organs are affected, a person may experience a wide range of symptoms, including:

  • Dry skin or mouth
  • Itchy skin or skin rash
  • Changes in the color of the skin or nails
  • Brittle nails
  • Hair loss
  • Sores on the skin or in the mouth
  • Sensitivity to certain types of flavors
  • Difficulty eating
  • Trouble swallowing
  • Poor appetite and weight loss
  • Nausea and vomiting
  • Diarrhea
  • Stomach pain or swelling

Other organs, such as the lungs, muscles, joints, and eyes, may also be affected. When these organs are affected, people may experience a range of symptoms including:

  • Dry, itchy, or painful eyes
  • Blurred vision
  • Sensitivity to bright light
  • Difficulty breathing
  • Chronic cough
  • Muscle weakness or cramps
  • Stiff joints

The treatment plan for chronic GvHD will depend on which organs are affected and how severe the symptoms are. Milder symptoms that only affect one or two organs may be treated with targeted therapies, such as topical products that are applied to the skin or the eyes. More severe symptoms that affect multiple organs may require “systemic” treatments, which are treatments that affect the entire body. These treatments, which are typically taken by mouth or injected, work by slowing down the attacks from the graft tissue. 

Oral corticosteroids (such as prednisone) are a common systemic treatment for chronic GvHD. There are also many medicines, called immunosuppressants, that work by limiting the activity of the immune system throughout the body. Many of these medicines are used “off-label,” meaning that they have not been approved for this use. Examples include mycophenolate (Cellcept), sirolimus (Rapamune), tacrolimus (Prograf). 

In recent years, a new class of drugs called kinase inhibitors have been approved for the treatment of chronic GvHD. These medicines reduce symptoms by targeting specific functions used by the immune system to send signals. Rezurock (belumosudil) is one of these kinase inhibitors. It blocks a substance in the immune system called ROCK (rho-associated, coiled-coil containing protein kinase) from sending signals the way that it normally would. Other kinase inhibitors that target different signaling functions in the immune system have also been approved for chronic GVHD, such as ibrutinib (Imbruvica) and ruxolitinib (Jakafi).

Rezurock is approved for use in people with chronic GvHD who have not gotten enough benefit from at least two other systemic treatments. For most people, this means that they still have symptoms even after using oral corticosteroids and at least one immunosuppressant. Some people may have also tried another kinase inhibitor. If you have already tried at least two systemic treatments for chronic GvHD but still have difficult symptoms, your specialist may discuss a treatment like Rezurock with you.

Rezurock is a tablet that is taken by mouth once every day, around the same time each day. The tablet should be swallowed whole with a glass of fluid and should be taken with a meal. If you miss a dose of Rezurock, take it as soon as you remember, but only if it is still the same day as the missed dose. If it is already the next day, do not take the missed dose. Instead, take your next dose at the regularly scheduled time. Taking double or extra doses of this medicine can increase your risk of side effects. 

Some people may need to take Rezurock two times every day. If your health care provider tells you to take this medicine two times every day, then each dose should be taken about 12 hours apart, with a meal and a glass of water. 

Your health care provider will monitor your blood to see if Rezurock is causing certain side effects. If you do experience side effects, your health care provider may tell you to stop taking the medicine for a few days or to stop taking the medicine completely.

One study was done to see if Rezurock was safe and effective for treating chronic GvHD. The people in this study were at least 12 years old, with an average age of 56. A little more than half (57%) of the people in this study were male.

Everyone in the study had chronic GvHD that affected multiple organs. On average, the people in the study had symptoms in four organs and had been diagnosed with chronic GvHD about two years before the study started. Here is some other important information about the people in this study:

  • Everyone was still experiencing symptoms even though they were already taking an oral corticosteroid. 
  • Everyone had already tried at least two treatments for their GVHD, and most (72%) had tried at least three treatments. 
  • About one-third (34%) of people had already tried ibrutinib and about one-third (29%) had already tried ruxolitinib. 

Everyone in this study took Rezurock every day, along with their oral corticosteroids. Most people continued taking oral immunosuppressants during the study but were told to stop taking any other kinase inhibitors. Once someone had been in the study for at least two weeks, they were allowed to start reducing their oral corticosteroid dose if their symptoms made this possible.

This study measured effectiveness by counting the number of people who had either a partial or complete “response” to the medicine. A complete response to the medicine meant that a person no longer had any symptoms of GvHD. A partial response to the medicine meant that a person had some of their symptoms get better and none of their symptoms get worse. The study also tracked each person’s daily corticosteroid dose to see if it changed during the study.

Number of people who responded to treatment. About three-quarters (74%-77%) of people responded to treatment with Rezurock. Almost all of these responses were considered a partial response; only a small number were a complete response. Of the people who had already tried ibrutinib, about three-quarters (74%) responded to treatment. Of the people who had already tried ruxolitinib, about two-thirds (66%) responded to treatment. 

About two-thirds (65%) of the people in the study were able to reduce their daily dose of corticosteroids while taking Rezurock. Some people (about one-fifth, or 21%) were able to stop taking corticosteroids completely. 

These findings show that Rezurock may be helpful in some people who still have symptoms after using other systemic medicines, including other kinase inhibitors.

If Rezurock is working, you may notice an improvement in your symptoms within the first 1-2 months of treatment. But it may take up to 6 months for some people to notice their symptoms improve. Over time, this may mean that you have fewer symptoms overall or that fewer organs are affected by your GvHD. If you take oral corticosteroids and your symptoms are improving, your specialist may work with you to lower your daily corticosteroid dose.

Do not stop taking Rezurock or any other medicines for GvHD without talking to your specialist first, even if your symptoms have gotten better. Your symptoms may get worse again if you stop or change any of your medicines. Let your specialist know if Rezurock does not seem to be working after about 4-6 months of treatment. 

Rezurock may cause changes in liver function. Before starting Rezurock, let your health care provider know if you have a history of liver problems. Once you have started the medicine, your health care provider will monitor your liver function through blood tests. Make sure that you keep all of your appointments for blood work. 

These are not all of the possible side effects. Talk with your health care provider if you are having symptoms that bother you. If you experience anything that you think may be caused by Rezurock, you can also report side effects to the FDA at 800-FDA-1088.

Rezurock may cause changes in fertility for both males and females. It may also cause harm to an unborn child if used during pregnancy. Anyone who is able to get pregnant or get someone else pregnant must use an effective form of birth control to prevent pregnancy while taking Rezurock. 

Let your specialist know if you are trying to have a child or would like to have a child in the future. They can discuss whether Rezurock is the right option for you. 

Let your health care provider know if you are taking a proton pump inhibitor (PPI) such as esomeprazole (Nexium), lansoprazole (Prevacid), omeprazole (Prilosec), pantoprazole (Protonix), or rabeprazole (Aciphex). These medicines, which are often used for acid reflux, may reduce the levels of Rezurock in your body.

Some medicines can alter the blood levels of Rezurock, reducing its effects. Rezurock may also alter the blood levels of other medicines. In certain cases, these effects are so significant that these medicines should not be taken together. Make sure your pharmacist or health care provider is aware of all the prescription or over-the-counter (OTC) medicines, vitamins/minerals, herbal products, or other supplements you take or have recently taken.

Rezurock is a type of medicine called a “specialty” medicine. This means that you may need to get it from a specialty pharmacy and that it may require prior authorization from your insurance company. 

There is a savings coupon available from the manufacturer that may allow you to pay as little as $0 for your prescription. Whether you are eligible depends on whether you have prescription insurance and what type of insurance you have. You can find out more at https://www.rezurock.com/kadmon-assist.