What is GvHD prophylaxis?

Graft-versus-host disease (GvHD) prophylaxis refers to steps taken to prevent GvHD, a condition that can occur after a stem cell transplant. Before and after the transplant, patients are given treatments to reduce the number of donor immune cells and lower the risk of GvHD. Common preventive treatments include:

• Methotrexate: a chemotherapy drug given in low doses to slow the growth of immune cells
• Tacrolimus or cyclosporine: to reduce the immune system's activity
• Mycophenolate mofetil: to help control the immune system
• Cyclophosphamide: used when the donor is a half-matched family member

How are patients with GvHD treated?

The treatment for GvHD depends on the severity and the organs affected. Healthcare professionals aim to manage symptoms and protect the patient's health.

Mild GvHD: For cases affecting only the skin, steroid creams can be used. For mild chronic GvHD in specific organs, treatments like eye drops or topical creams may be recommended.

Severe GvHD: Patients with more extensive symptoms may need treatments that work throughout the entire body. Steroids are often the first step, but if they don't work (steroid-refractory GvHD), other therapies are considered.

What are the treatment options for patients with GvHD?

Common treatments

Corticosteroids

Corticosteroids are used to reduce inflammation and immune cell attacks. They can be applied as creams, taken as pills, or given intravenously. Two commonly used corticosteroids for patients with GvHD are prednisolone, usually taken as a pill, and methylprednisolone, which is usually administered intravenously.

Extracorporeal photopheresis

Extracorporeal photopheresis (ECP) is a light therapy where blood is treated and returned to the body to target immune cells. The patient's blood is drawn from the body and passed through a machine to separate out the white blood cells, which are then treated before being returned to the patient.

Cyclosporine and tacrolimus

Cyclosporine and tacrolimus are calcineurin inhibitors, which are used to prevent and treat GvHD by reducing immune activity. They can be administered as a pill, intravenously, or as an ointment, and are often used in combination with corticosteroids.

Mycophenolate mofetil

Mycophenolate mofetil is a pill used to suppress the immune system.

Advanced therapies

Ruxolitinib

Ruxolitinib helps when steroids don't work, especially for acute or chronic GvHD.

Ibrutinib

Ibrutinib stops certain cells from growing and is used for chronic GvHD after other treatments fail.

Belumosudil

Belumosudil targets tissue inflammation and scarring in chronic GvHD.

Axatilimab

Axatilimab is a newer treatment used to reduce inflammation in chronic GvHD.

Off-label treatments and clinical trials

Some treatments may not be specifically approved for GvHD but are used for other conditions. Healthcare professionals might recommend these off-label options if they believe they could help. Clinical trials are also an option for patients who haven't responded to other treatments. These trials test new therapies under strict guidelines.

References

1. Preventing GvHD. Cancer Research UK. https://www.cancerresearchuk.org/about-cancer/coping/physically/gvhd/preventing-gvhd. Accessed Dec 18, 2024.
2. Justiz Vaillant AA, Modi P, Mohammadi O. Graft-versus-host disease. In: StatPearls. StatPearls Publishing; 2024. http://www.ncbi.nlm.nih.gov/books/NBK538235/. Accessed Dec 18, 2024.
3. Graft-versus-host disease. Leukemia & Lymphoma Society. https://www.lls.org/treatment/types-of-treatment/stem-cell-transplantation/graft-versus-host-disease. Accessed Dec 18, 2024.
4. Treatment of GvHD. Cancer Research UK. https://www.cancerresearchuk.org/about-cancer/coping/physically/gvhd/treatment. Accessed Dec 18, 2024.
5. National Comprehensive Cancer Network. Graft-versus-host disease NCCN guidelines for patients. http://www.nccn.org/patients/guidelines/content/PDF/GVDH-patient-guideline.pdf. Accessed Dec 18, 2024.
6. U.S. Food and Drug Administration. FDA approves axatilimab-csfr for chronic graft-versus-host disease. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-axatilimab-csfr-chronic-graft-versus-host-disease. Accessed Dec 18, 2024.