FDA-approved for children 2 months and older with acute graft-versus-host disease (acute GvHD) who did not get better with steroids

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Understanding steroid-refractory acute GvHD in children

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A fresh start for familes

A stem cell transplant can be a fresh start for many families. For some, the days and weeks after can bring questions, especially if health problems come up. Learning about steroid-refractory acute GvHD can help. It can give you the tools to talk with your child's care team. And it can help you feel more ready for what comes next.

Why acute GvHD sometimes happens

After a bone marrow transplant, some children get sick with a condition called acute GvHD. This happens when donor immune cells get confused and attack parts of the child’s body. Acute GvHD may affect the gastrointestinal, or GI, tract (eg, stomach and intestines). It may also affect the liver and/or skin. Acute GvHD most often develops within the first 100 days after a bone marrow or stem cell transplant. However, sometimes it can appear later.

Not every child who has a transplant will get acute GvHD. It can affect 3 to 7 out of every 10 transplant patients.

  1. The transplant

    In a bone marrow transplant, sick blood-forming cells are replaced with healthy cells from a donor.

  2. The gift of new cells

    These new donor cells help the body make healthy blood again.

  3. The cells get confused

    Sometimes, these new cells get confused. They may think your child's own cells don't belong.

  4. The body's reaction

    This confusion may cause the new cells to attack parts of your child's body. This can lead to issues in the GI tract, liver, and/or skin.

After transplant

Donor cells may send attack signals that cause the immune system to go into overdrive.

Inflammation goes up

These attack signals may cause inflammation in the GI tract, liver, and/or skin.

acute GvHD most often shows up in the first 100 days after a bone marrow transplant.

Treating acute GvHD in children

Doctors usually treat acute GvHD with steroids. For many children, steroids help. But for about half, they don’t work well enough. When this happens, it's called steroid-refractory acute GvHD.

Watch: Dr Joanne Kurtzberg on treating steroid-refractory aGvHD

...Only half the children who get steroids respond.

Dr Joanne Kurtzberg

acute GvHD expert

Know the symptoms of steroid-refractory acute GvHD

You play an important role in your child’s care. That’s because you know your child best. Watching for symptoms can help you stay one step ahead of acute GvHD. If you notice changes, tell the care team right away. Working together can help your child get the support they need.

Possible symptoms

Steroid-refractory acute GvHD symptoms may affect certain parts of the body, including the GI tract, liver, and/or skin.

GI Tract

  • ​​Loose stools
  • Nausea
  • Belly pain
  • Poor appetite
  • Throwing up
  • Blood in the stool

Liver

Commonly has no symptoms you can see, but may include: 

  • Yellow skin or eyes

Symptoms of liver failure include: 

  • Bleeding
  • Confusion
  • Extra fluid in the belly

Skin

  • Redness or skin rash (anywhere on the body, including hands and feet)
  • Itchy rash that may look like a sunburn 
  • Severe rash with blisters or peeling skin 

Move beyond “What if?” to “What's next?”

If steroids failed to control your child's acute GvHD, you may wonder, “What if we need to move beyond steroids? What's our next step?” The good news is you are not out of options. RYONCIL was developed for moments like this, when more help is needed.

RYONCIL is a first-of-its-kind cell therapy. It has been shown to be safe and effective for children.

Download “Understanding What's Possible With RYONCIL”

RYONCIL Caregiver brochure

Get support at every step

Learning that your child has steroid-refractory acute GvHD can feel scary. But it’s important to know that you are not alone. Support is available to help your family move forward.

Get support resources

See why recovery is possible with RYONCIL

Learn more about the first and only FDA-approved treatment approved just for children with steroid-refractory acute GvHD.

About RYONCIL

Have questions? We have answers

Get answers to common questions about steroid-refractory acute GvHD and treatment with RYONCIL.

Explore Q&A
Indication and Important Safety Information for RYONCIL® (remestemcel-L-rknd)
What is Ryoncil?

RYONCIL is a prescription medicine used to treat a condition called steroid-refractory acute graft versus host disease (SR-aGvHD) in children 2 months and older.

Who should not receive Ryoncil?

Do not receive Ryoncil if you are allergic to DMSO or porcine and bovine proteins (e.g., pigs or cows).

Ryoncil is not recommended if you are pregnant. Talk to your doctor about the risks and benefits of breastfeeding during Ryoncil treatment.

What are the possible serious side effects?
  • Allergic reactions. Serious allergic reactions, including anaphylaxis, can happen during or after infusion. Tell your doctor or nurse right away if you experience signs of an allergic reaction, including fever, rash, hives, low blood sugar, or breathing problems.
  • Infections. Ryoncil is made from human cells using materials from humans and porcine and bovine sources (e.g., pigs, cows). Although all donors and materials are carefully screened and tested, these measures cannot completely eliminate potential risk of transmission of infectious disease.
  • Unusual tissue growth. There is a theoretical risk that Ryoncil could cause growth of tissue in other places, such as bone, cartilage, or fat.
What are the most common side effects?

The most common side effects include infections, fever, bleeding, swelling, stomach pain, high blood pressure, vomiting, irregular heartbeat, diarrhea, rash, joint pain, low blood pressure, cough, and trouble breathing.

What should I tell my doctor before receiving Ryoncil?

Tell your doctor about all your medical conditions, allergies, and if you are pregnant or breastfeeding.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1–800-FDA-1088.

For additional information, see full Prescribing Information.

Indication and Important Safety Information for RYONCIL® (remestemcel-L-rknd)
What is Ryoncil?

RYONCIL is a prescription medicine used to treat a condition called steroid-refractory acute graft versus host disease (SR-aGvHD) in children 2 months and older.

Who should not receive Ryoncil?

Do not receive Ryoncil if you are allergic to DMSO or porcine and bovine proteins (e.g., pigs or cows).

Ryoncil is not recommended if you are pregnant. Talk to your doctor about the risks and benefits of breastfeeding during Ryoncil treatment.

What are the possible serious side effects?
  • Allergic reactions. Serious allergic reactions, including anaphylaxis, can happen during or after infusion. Tell your doctor or nurse right away if you experience signs of an allergic reaction, including fever, rash, hives, low blood sugar, or breathing problems.
  • Infections. Ryoncil is made from human cells using materials from humans and porcine and bovine sources (e.g., pigs, cows). Although all donors and materials are carefully screened and tested, these measures cannot completely eliminate potential risk of transmission of infectious disease.
  • Unusual tissue growth. There is a theoretical risk that Ryoncil could cause growth of tissue in other places, such as bone, cartilage, or fat.
What are the most common side effects?

The most common side effects include infections, fever, bleeding, swelling, stomach pain, high blood pressure, vomiting, irregular heartbeat, diarrhea, rash, joint pain, low blood pressure, cough, and trouble breathing.

What should I tell my doctor before receiving Ryoncil?

Tell your doctor about all your medical conditions, allergies, and if you are pregnant or breastfeeding.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1–800-FDA-1088.

For additional information, see full Prescribing Information.