Última actualización:
January 10, 2022
Años publicados: 1991, 1992, 1993, 1997, 1998, 1999, 2000, 2001, 2002, 2009
NORD gratefully acknowledges Catharina Schuetz, MD, MSc, Ulm University, Department of Pediatrics (Immunology, BMT), Germany, for assistance in the preparation of this report.
Graft versus Host Disease (GVHD) is a rare disorder that can strike persons whose immune system is deficient or suppressed and who have received a bone marrow transplant or a nonirradiated blood transfusion. Symptoms may include skin rash, intestinal problems and liver dysfunction.
GVHD occurs most frequently after allogeneic bone marrow transplant and initially leads to dermatitis (a skin rash), gastrointestinal problems and liver dysfunction. In its chronic form involvement of mucosa (mouth and eyes) resembling sicca syndrome, lungs (resembling bronchiolitis obliterans) and the muskuloskeletal system (resembling myositis) is observed. GVHD affects about 60% of all bone marrow transplant but usually is limited and mild.
GVHD can be acute (sudden) or chronic (long lasting). Acute GVHD occurs in the first 100 days (at earliest 2 to 3 weeks) following bone marrow transplantation. The first symptoms are usually mild skin rash, liver dysfunction and intestinal problems. In some cases the patients may suddenly show very severe skin problems, diarrhea, nausea, abdominal pain and liver failure.
Chronic GVHD is used for GVHD lasting beyond 100 days and usually persists long after a bone marrow transplant. The signs and symptoms are similar to those of the acute GVHD, but in addition to the skin, intestinal and liver problems, chronic GVHD may also involve mucosa, lungs and the musculoskeletal system. Long term consequences may be scleroderma-like skin changes and bronchiolitis obliterans.
GVHD is caused by donor T cells recognizing foreign antigens (histocompatibility or human leucocyte antigens) on the recipient’s cells and reacting to them. Prior to allogeneic bone marrow transplants recipients usually undergo myeloablative treatment with radiation or chemotherapy to destroy their own diseased bone marrow and weaken their immune system. When receiving the bone marrow or stem cell transplant, immunocompetent donor lymphocytes recognize foreign minor locus histocompatibility antigens on the recipient’s cells resulting in GVHD.
GVHD affects males and females of all ages who have been immunosuppressed before being given a bone marrow transplant or a nonirradiated blood transfusion containing allogeneic lymphocytes. The risk of GVHD usually increases with the recipient’s age and with the degree of HLA differences between donor and recipient unless fully T-cell depleted.
Treatment
Treatment of GVHD usually consists of immunosuppressive drugs including glucocorticoid (steroid) drugs and a combination of cyclosporine (Sandimmune) and methotrexate. Instead of cyclosporine other calcineurin inhibitors (tacrolimus) or an mTOR inhibitor (sirolimus) may be chosen. In some cases, where GVHD is resistant to the above treatments antithymocyte globulin (ATG) may be used. Prevention of GVHD consists of prophylactic treatment prior to bone marrow transplant, mostly using cyclosporine and T-cell depletion of the graft. Blood may be treated by radiation before being given to the recipient in order to suppress the donor’s lymphocytes. These prophylactic measures often keep GVHD from developing.
In 2017, The U.S. Food and Drug Administration (FDA) approval Imbruvica (ibrutinib) for the treatment of adult patients with chronic graft versus host disease (cGVHD) after failure of one or more treatments. Imbruvica is manufactured by Pharmacyclics LLC.
In 2021, Orencia (abatacept) was approved for the prevention of acute graft versus host disease (aGVHD). This is the first FDA drug approval for aGVHD prevention.
Information on current clinical trials is posted on the Internet at www.clinicaltrials.gov. All studies receiving U.S. government funding, and some supported by private industry, are posted on this government website.
For information about clinical trials being conducted at the National Institutes of Health (NIH) in Bethesda, MD, contact the NIH Patient Recruitment Office:
Tollfree: (800) 411-1222
TTY: (866) 411-1010
Email: prpl@cc.nih.gov
Some current clinical trials also are posted on the following page on the NORD website:
https://rarediseases.org/living-with-a-rare-disease/find-clinical-trials/
For information about clinical trials sponsored by private sources, contact:
www.centerwatch.com
For information about clinical trials conducted in Europe, contact:
https://www.clinicaltrialsregister.eu/
TEXTBOOKS
Bennett JC, Plum F., eds. Cecil Textbook of Medicine. 20th ed. Philadelphia, PA: W.B. Saunders Co; 1996: 975.
JOURNAL ARTICLES
Jacobsohn DA, Novel therapeutics for the treatment of graft-versus-host disease. Expert Opin Investig Drugs. 2002;11:1271-80.
Carpenter PA, et al., A humanized non-FcR-binding anti-CD3 antibody, visilizumab, for treatment of steroid-refractory acute graft-versus-host disease. Blood. 2002;99:2712-9.
Rivkina AM, Stump LS, Infliximab in graft-versus-host disease. Am J Health Syst Pharm. 2002;59:1271-5.
Deeg HJ, et al., Treatment of steroid-refractory acute graft-versus-host disease with antibody-CD147 monoclonal antibody ABX-CBL. Blood. 2001;98:2052-8.
Greinix HT, et al., Extracorporeal photochemotherapy in the treatment of severe steroid-refractory acute graft-versus-host disease: a pilot study. Blood. 2000;96:2426-31.
McDonald GB, et al., Oral beclomethasone dipropionate for treatment of intestinal graft-versus-host disease: a radonmized, controlled trial. Gastroenterology. 1998;115:28-35. Comment In: Gastroenterology. 1998;115:220-2.
Zic JA, et al., The North American experience with photopheresis. Ther Apher. 1999;3:50-62.
Bonnie C, et al., HSV-TV gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia. Science. 1997;276:1719-24.
Chao NJ, et al., Cyclosporine, methotrexate, and prednisone compared with cyclosporine and prednisone for prophylaxis of acute graft-versus-host disease. N Engl J Med. 1993;329:1225-30.
Decoste AD, et al., Transfusion-associated graft-vs-host disease in patients with malignancies. Report of two cases and review of the literature. Arch Dermatol. 1990;126:1324-29.
Martin PJ, et al., A retrospective analysis of therapy for acute graft-vs-host disease: initial treatment. Blood. 1990;76:1464-72.
Jones B, et al., Gastrointestinal inflammation after bone marrow transplantation: graft- vs-host disease or opportunistic infection? AJR Am J Roentgenol. 1988;150:277-81.
NORD y la Fundación MedicAlert se han asociado en un nuevo programa para brindar protección a pacientes con enfermedades raras en situaciones de emergencia.
Aprende más https://rarediseases.org/patient-assistance-programs/medicalert-assistance-program/Asegurarse de que los pacientes y los cuidadores estén equipados con las herramientas que necesitan para vivir su mejor vida mientras manejan su condición rara es una parte vital de la misión de NORD.
Aprende más https://rarediseases.org/patient-assistance-programs/rare-disease-educational-support/Este programa de asistencia, primero en su tipo, está diseñado para los cuidadores de un niño o adulto diagnosticado con un trastorno raro.
Aprende más https://rarediseases.org/patient-assistance-programs/caregiver-respite/