Rare Hematology News

Spotlight On

Graft versus host disease

Chronic graft versus host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted donor cells attack the transplant recipient's body.



Age of Onset





Autosomal dominant


Autosomal recessive




X-linked dominant


X-linked recessive


Rare View

A condition that occurs when donated stem cells or bone marrow (the graft) see the healthy tissues in the patient’s body (the host) as foreign and attack them. It can also occur after an organ transplant. Graft-versus-host disease can cause damage to the host’s tissues and organs, especially the skin, liver, intestines, eyes, mouth, hair, nails, joints, muscles, lungs, kidneys, and genitals. The signs and symptoms may be severe and life threatening. Graft-versus-host disease can occur within the first few months after transplant (acute) or much later (chronic).

5 Facts you should know



Chronic graft-versus-host disease (GVHD) is a dangerous complication of allogeneic stem cell transplantation.



Symptoms may include skin rash, mouth sores, dry eyes, liver inflammation, development of scar tissue in the skin and joints, and damage to the lungs.



Mild forms of chronic GVHD are often manageable with local or low-dose systemic immunosuppression and do not affect long-term survival.



More severe forms of chronic GVHD require intensive medical management and adversely affect survival.



Prednisone is the standard first-line therapy for chronic GVHD.

Graft versus host disease is also known as...

Graft versus host disease is also known as:

  • Chronic GVHD

What’s your rare IQ?

In the USA, what percentage of allogeneic transplant patients develop chronic GVHD?

Common Signs & Symptoms

Pain in the abdomen

Skin rashes, thickness, or yellow skin and eyes

Gastrointestinal: diarrhea or nausea

Mouth: dryness or ulcers

Also common: abnormality of taste, dry eyes, frequent infections, or weight loss

Current treatments

The medication(s) listed below have been approved by the Food and Drug Administration (FDA) as orphan products for treatment of this condition. Learn more orphan products.


Manufactured by Pharmacyclics, LLC
FDA-approved indication: Treatment of adult patients with chronic graft versus host disease (cGVHD).
National Library of Medicine Drug Information Portal
Medline Plus Health Information

Top Clinical Trials

TitleDescriptionPhasesStatusInterventionsLocationsMore Information
A Study of Axatilimab at 3 Different Doses in Patients With Chronic Graft Versus Host Disease (cGVHD) AGAVE-201 is a Phase 2 study to evaluate the efficacy, safety, and tolerability of axatilimab at 3 different dose levels in patients with recurrent or refractory active chronic graft versus host disease (cGVHD) who have received at least 2 prior lines of systemic therapy due to progression of disease, intolerability or toxicity.Phase 2RecruitingDrug: axatilimabIndiana University Health Melvin and Bren Simon Cancer Center, Indianapolis, Indiana, United StatesMore Information
Itacitinib (INCB039110) and Extracorporeal Photopheresis (ECP) for First-Line Treatment in Chronic GVHD An open-label, Phase II trial designed to assess the recommended phase 2 dose (RP2D) of itacitinib in combination ECP and efficacy of the combination after 24 weeks of therapy. The trial will consist of two parts: Part One will assess the RP2D. For dose-finding purposes, the DLT evaluation period will be defined as the time from the first dose of itacitinib lead-in (7-day lead-in) to the last day of cycle one combination therapy (Cycle one day 28). Part Two will further describe and characterize the safety and efficacy of the regimen. The RP2D will be determined by a 3+3 dose de-escalation design. Should dose level one be deemed intolerable, enrollment will proceed at dose level -1. The RP2D will be affirmed according to the rules of the 3+3 dose de-escalation scheme (Section 4.2). Once an RP2D has been confirmed, Part 2 will open as an expansion cohort.Phase 2RecruitingDrug: Itacitinib|Device: Extracorporeal Photopheresis (ECP)Huntsman Cancer Institute at University of Utah, Salt Lake City, Utah, United StatesMore Information
Front Line Ibrutinib for Newly Diagnosed Chronic Graft-Versus Host Disease To evaluate efficacy of ibrutinib as a first-line treatment for persons with newly diagnosed chronic GvHD by measuring the overall response rate (complete response [CR] + partial response [PR]) at 6 months, according to the 2014 NIH Consensus CriteriaPhase 2RecruitingDrug: IbrutinibNational Institutes of Health Clinical Center, Bethesda, Maryland, United States|Washington University, St. Louis, Saint Louis, Missouri, United StatesMore Information
Topical Ruxolitinib for Cutaneous Chronic Graft Versus Host Disease (cGVHD) The purpose of this study is to compare the safety and effects of ruxolitinib 1.5% cream with those of standard moisturizers in people with non-sclerotic chronic cutaneous GVHD.
Phase 2RecruitingDrug: Ruxolitinib 1.5% cream|Drug: Vehicle creamNational Institutes of Health Clinical Center, Bethesda, Maryland, United StatesMore Information
A Study of Itacitinib for the Treatment of Chronic Graft Versus Host Disease (cGVHD)This study is being done in patients who have been receiving corticosteroids or other immunosuppressive therapies for the treatment of cGVHD for at least 6 months. The purpose of this study is to find out if itacitinib in combination with corticosteroids or other immunosuppressive therapies is safe and effective in people with cGVHD.Phase 2RecruitingDrug: ItacitinibColorado Blood Cancer Institute, Denver, Colorado, United States|The Sarah Cannon Research Institute, Nashville, Tennessee, United States|South Austin Medical Center, Austin, Texas, United States|Texas Transplant Institute, San Antonio, Texas, United StatesMore Information
Ibrutinib and Rituxan for Chronic GVHD This is a phase II trial evaluating the safety and efficacy of the combination of Ibrutinib and Rituximab as primary treatment of chronic GVHD. We plan to enroll 35 patients on this study. Patients will be formally monitored monthly for 12 months to evaluate for outcome and safety endpoints. All other assessments will be done at the physician's discretion or institutional standards. All patients, responders and treatment failures, will be followed for a period of one year from the time of initiation of therapy. The primary endpoint will be the proportion of patients that are alive and off all systemic IST at 12 months following initiation of treatment.Phase 2RecruitingDrug: Rituximab|Drug: IbrutinibNorthside Hospital, Atlanta, Georgia, United StatesMore Information
Acalabrutinib for the Treatment of Chronic Graft Versus Host DiseaseThis phase II trial studies how well acalabrutinib works in treating patients with chronic graft versus host disease. Acalabrutinib may be an effective treatment for graft-versus-host disease caused by a stem cell transplant.Phase 2RecruitingDrug: Acalabrutinib|Other: Questionnaire AdministrationMoffitt Cancer Center, Tampa, Florida, United States|Roswell Park Comprehensive Cancer Center, Buffalo, New York, United States|Vanderbilt University Medical Center, Nashville, Tennessee, United States|Fred Hutch/University of Washington Cancer Consortium, Seattle, Washington, United StatesMore Information
GRAVITAS-309: Itacitinib and Corticosteroids as Initial Treatment for Chronic Graft-Versus-Host Disease The purpose of this study is to assess the efficacy and safety of itacitinib in combination with corticosteroids as first-line treatment for moderate or severe chronic graft-versus-host disease (cGVHD).
Phase 2|Phase 3RecruitingDrug: Itacitinib|Drug: Placebo|Drug: Methylprednisolone|Drug: PrednisoneUniversity of Arizona Cancer Center - Out Pt., Tucson, Arizona, United States|University of Arkansas For Medical Sciences - Winthrop P Rockefeller Cancer Institute, Little Rock, Arkansas, United States|City of Hope National Medical Center, Duarte, California, United States|University of California San Diego Medical Center, Moores Cancer Center, La Jolla, California, United States|University of Miami Sylvester Comprehensive Cancer Center, Miami, Florida, United States|Winship Cancer Institute of Emory University, Atlanta, Georgia, United States|Augusta University - Medical College of Georgia, Augusta, Georgia, United States|Rush University Medical Center, Chicago, Illinois, United States|Illinois Cancer Specialists, Chicago, Illinois, United States|Loyola University Medical Center, Maywood, Illinois, United States|Advocate Lutheran General Hospital - Oncology Specislists Sc, Park Ridge, Illinois, United States|Indiana University Melvin and Bren Simon Cancer Center, Indianapolis, Indiana, United States|University of Kansas Hospital Authority, Westwood, Kansas, United States|Tulane University, New Orleans, Louisiana, United States|University of Maryland - Greenebaum Cancer Center, Baltimore, Maryland, United States|Tufts Medical Center, Boston, Massachusetts, United States|Massachusetts General Hospital, Boston, Massachusetts, United States|University of Michigan, Ann Arbor, Michigan, United States|Henry Ford Health System, Detroit, Michigan, United States|Spectrum Health, Grand Rapids, Michigan, United States|University of Minnesota, Masonic Cancer Center, Minneapolis, Minnesota, United States|Hackensack University Medical Center, Hackensack, New Jersey, United States|Stony Brook University Medical Center, Stony Brook, New York, United States|Duke University Medical Center, Durham, North Carolina, United States|Oncology Hematology Care, Inc, Cincinnati, Ohio, United States|University Hospitals Cleveland Medical Center, Cleveland, Ohio, United States|University of Pennsylvania Abramson Cancer Center, Philadelphia, Pennsylvania, United States|Jefferson University Hospitals, Philadelphia, Pennsylvania, United States|Western Pennsylvania Hospital, Pittsburgh, Pennsylvania, United States|University of Pittsburgh, Pittsburgh, Pennsylvania, United States|Avera Cancer Institute, Sioux Falls, South Dakota, United States|Tri Star Bone Marrow Transplant, Nashville, Tennessee, United States|Vanderbilt-Ingram Cancer Center, Nashville, Tennessee, United States|St David'S South Austin Medical Center, Austin, Texas, United States|Texas Oncology - Medical City Dallas, Dallas, Texas, United States|Texas Oncology - Baylor Sammons Cancer Center, Dallas, Texas, United States|Fred Hutchinson Cancer Research Center, Seattle, Washington, United States|Ordensklinikum Linz Gmbh Elisabethinen, Linz, Austria|Zna Stuivenberg, Antwerpen, Belgium|Institut Jules Bordet, Brussels, Belgium|Universitair Ziekenhuis Antwerpen (Uza), Edegem, Belgium|Universitair Ziekenhuis Gent, Gent, Belgium|Universitaire Ziekenhuis Leuven - Gasthuisberg, Leuven, Belgium|CENTRE HOSPITALIER UNIVERSITAIRE DE LI�GE - SART TILMAN, Liege, Belgium|AZ DELTA, Roeselare, Belgium|University of Alberta, Edmonton, Alberta, Canada|University Health Network, Toronto, Ontario, Canada|Hospital Maisonneuve Rosemont, Montreal, Quebec, Canada|The Finsen Centre National Hospital, Copenhagen, Denmark|Turku University Hospital, Turku, Finland|Chu Amiens Picardie - Hopital Sud, Amiens, France|Centre Hospitalier D'Angers, Angers, France|Chu de Grenoble - Hopital Albert Michallon, Grenoble Cedex, France|Institut Paoli-Calmettes, Marseille, France|Centre Hospitalier Universitaire de Nantes (Chu de Nantes) - Hotel-Dieu, Nantes, France|Chu de Nice - Hospital L Archet, Nice, France|CHU DE RENNES - H�PITAL PONTCHAILLOU, Rennes, France|Centre Henri Becquerel, Rouen, France|Chru Hopitaux de Tours Hospital Bretonneau, Tours, France|Hopitaux de Brabois, Vandoeuvre Les Nancy, France|Charite Berlin, Berlin, Germany|Universitatsklinikum Bonn Aoer, Bonn, Germany|University Clinic Carl Gustav Carus Technical University Dresden, Dresden, Germany|Universitaetsklinikum Erlangen - Medizinische Klinik 5, Erlangen, Germany|Universitatsklinikum Essen, Essen, Germany|UNIVERSIT�TSKLINIKUM HALLE (SAALE), Halle, Germany|University Medical Centre Hamburg-Eppendorf Centre of Oncology, Hamburg, Germany|Universitaetsklinikum Jena, Jena, Germany|Universitatsklinikum Koln, Koeln, Germany|Selbststandige Abteilung Fur Hamatologie Und Internistische Onkologie, Leipzig, Germany|Universitatsmedizin Der Johannes Gutenberg-Universitat Mainz Iii, Mainz, Germany|University Hospital Mannheim, Mannheim, Germany|Universitatsklinikum Munster, Muenster, Germany|Iii Medizinische Klinik Und Poliklinik Klinikum Rechts Der Isar Technische Universitat Munchen, Munich, Germany|Universitaetsmedizin Rostock, Rostock, Germany|Universitaetsklinikum in Tubingen, T�BINGEN, Germany|University Hospital of West Attica - Attikon, Chaidari, Greece|General Hospital of Thessaloniki G. Papanikolaou, Thessaloniki, Greece|Rambam Medical Center, Haifa, Israel|Hadassah Hebrew University Medical Center Ein Karem Hadassah, Jerusalem, Israel|Rabin Medical Center - Beilinson Hospital, Petach Tiqwa, Israel|Tel Aviv Sourasky Medical Center, Tel Aviv-yafo, Israel|CLINICA DI EMATOLOGIA, UNIVERSIT� POLITECNICA DELLE MARCHE, Ancona, Italy|Azienda Ospedaliera Papa Giovanni Xxiii, Bergamo, Italy|L AZIENDA OSPEDALIERO-UNIVERSITARIA DI BOLOGNA POLICLINICO S. ORSOLA � MALPIGHI, Bologna, Italy|Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia, Brescia, Italy|Azienda Policlinico Vittorio Emanuele, Catania, Italy|Istituto Di Ricovero E Cura A Carattere Scientifico (Irccs) Ospedale San Raffaele, Milano, Italy|Fondazione Irccs Ca Granda Ospedale Maggiore, Milan, Italy|A.O.U. Di Modena - Policlinico, Modena, Italy|A.O.U. Federico Ii, Napoli, Italy|Azienda Ospedaliera Ospedali Riuniti "Villa Sofia - Cervello", Palermo, Italy|Comitato Di Bioetica Della Fondazione Irccs Policlinico San Matteo, Pavia, Italy|Azienda Ospedaliera Bianchi-Melacrino-Morelli Ospedali Riuniti, Reggio Calabria, Italy|Universita Degli Studi Di Roma La Sapienza - Umberto I Policlinico Di Roma - Centro Di Ematologia, Roma, Italy|Policlinico Universitario Agostino Gemelli Universita Cattolica Del Sacro Cuore, Roma, Italy|Irrcs Instituto Clinico Humanitas, Rozzano, Italy|I.R.C.C.S. Casa Sollievo Della Sofferenza, San Giovanni Rotondo, Italy|Azienda Ospedaliero Universitaria Citta Della Salute E Della Scienza, Torino, Italy|Azienda Ospedaliero-Universitaria Santa Maria Della Misericordia, Udine, Italy|Centro Ricerche Cliniche Di Verona (Crc), Verona, Italy|Uniwersyteckie Centrum Kliniczne, Gdansk, Poland|Narodowy Instytut Onkologii Im. Marii Sklodowskiej-Curie Panstwowy Instytut Badawczy Oddzial W Gliwi, Gliwice, Poland|Szpital Kliniczny Przemienienia Panskiego, Poznan, Poland|Mtz Clinical Research Sp. Zo.O., Warszawa, Poland|Institut Catala D Oncologia, Badalona, Spain|Hospital de La Santa Creu I Sant Pau, Barcelona, Spain|Hospital Universitario Virgen de Las Nieves, Granada, Spain|Ico Institut Catala D Oncologia, L'hospitalet de Llobregat, Spain|Hospital General Universitario Gregorio Maranon, Madrid, Spain|Hospital Universitario Ramon Y Cajal, Madrid, Spain|Hospital Regional Universitario de Malaga, Malaga, Spain|Son Espases University Hospital, Palma, Spain|Clinica Universidad de Navarra (Cun), Pamplona, Spain|Hospital Clinico Universitario de Salamanca, Salamanca, Spain|Hospital Universitario Marques de Valdecilla, Santander, Spain|Hospital Clinico de Santiago de Compostela, Santiago de Compostela, Spain|Hospital Universitario Y Politcnico de La Fe, Valencia, Spain|Hospital Clinico Universitario de Valencia, Valencia, Spain|Karolinska University Hospital Huddinge, Huddinge, Sweden|Skane University Hospital Lund, Lund, Sweden|Universitatsspital Zurich, Zurich, Switzerland|Bristol Haematology & Oncology Centre, Bristol, United Kingdom|University Hospital of Wales, Cardiff, United Kingdom|Barts Health Nhs Trust - St Bartholomews Hospital, London, United Kingdom|King'S College Hospital (Nhs Foundation), London, United Kingdom|Imperial College Healthcare Nhs Trust - Hammersmith Hospital, London, United Kingdom|Nottingham University Hospitals Nhs Trust, Nottingham, United Kingdom|The Royal Marsden Nhs Foundation Trust - Sutton, Sutton, United Kingdom|St. George'S University Hospitals Nhs Foundation Trust, Tooting, United KingdomMore Information
Efficacy and Safety of KD025 in Subjects With cGVHD After At Least 2 Prior Lines of Systemic Therapy This is a Phase 2, randomized, multicenter study to evaluate the efficacy and safety of KD025 in subjects with Chronic Graft Versus Host Disease (cGVHD) after at least 2 prior lines of systemic therapyPhase 2RecruitingDrug: Belumosudil (KD025)The University of Arizona Cancer Center, Tucson, Arizona, United States|City of Hope National Medical Center, Duarte, California, United States|University of California, Los Angeles, Los Angeles, California, United States|University of California, San Francisco, San Francisco, California, United States|Stanford Cancer Center, Blood and Marrow Transplantation, Stanford, California, United States|Colorado Blood Cancer Institute, Denver, Colorado, United States|University of Miami - Sylvester Cancer Center, Miami, Florida, United States|H. Lee Moffitt Cancer Center & Research Institute, Tampa, Florida, United States|Emory University School of Medicine, Atlanta, Georgia, United States|Georgia Cancer Center at Augusta University, Augusta, Georgia, United States|Northwestern Memorial Hospital, Chicago, Illinois, United States|University of Illinois at Chicago, Chicago, Illinois, United States|University of Iowa, Iowa City, Iowa, United States|University of Kansas Cancer Center, Fairway, Kansas, United States|National Cancer Institute, Bethesda, Maryland, United States|Massachusetts General Hospital, Boston, Massachusetts, United States|Dana Farber Cancer Institute, Boston, Massachusetts, United States|University of Michigan, Ann Arbor, Michigan, United States|Barbara Ann Karmanos Cancer Institute, Detroit, Michigan, United States|University of Minnesota, Minneapolis, Minnesota, United States|Washington University School of Medicine, Division of Oncology, Saint Louis, Missouri, United States|University of Rochester, Rochester, New York, United States|Wake Forest Baptist Medical Center Wake Forest University School of Medicine, Winston-Salem, North Carolina, United States|Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States|The Ohio State University, Columbus, Ohio, United States|Oregon Health & Science University - Knight Clinical Research, Portland, Oregon, United States|University of Pittsburgh Medical Center Hillman Cancer Center, Pittsburgh, Pennsylvania, United States|Sarah Cannon Research Institute at Tennessee Oncology, Nashville, Tennessee, United States|Vanderbilt University Medical Center, Nashville, Tennessee, United States|St. David's South Austin Medical Center, Austin, Texas, United States|The University of Texas MD Anderson Cancer Center, Houston, Texas, United States|Methodist Physician Practices, PLLC, San Antonio, Texas, United States|Fred Hutch Cancer Research Center, Seattle, Washington, United States|University of Wisconsin -Carbone Cancer Center, Madison, Wisconsin, United States|Froedtert Hospital and the Medical College of Wisconsin, Milwaukee, Wisconsin, United StatesMore Information
Naive T Cell Depletion for Preventing Chronic Graft-versus-Host Disease in Children and Young Adults With Blood Cancers Undergoing Donor Stem Cell TransplantThis phase II trial studies how well naive T-cell depletion works in preventing chronic graft-versus-host disease in children and young adults with blood cancers undergoing donor stem cell transplant. Sometimes the transplanted white blood cells from a donor attack the body's normal tissues (called graft versus host disease). Removing a particular type of T cell (naive T cells) from the donor cells before the transplant may stop this from happening.Phase 2RecruitingRadiation: Total-Body Irradiation|Drug: Thiotepa|Drug: Fludarabine|Drug: Cyclophosphamide|Drug: Busulfan|Procedure: Allogeneic Bone Marrow Transplantation|Drug: Tacrolimus|Drug: Methotrexate|Procedure: Naive T Cell-Depleted Hematopoietic Stem Cell TransplantationChildren's Hospital of Los Angeles, Los Angeles, California, United States|Children's National Medical Center, Washington, District of Columbia, United States|Children's Healthcare of Atlanta, Atlanta, Georgia, United States|University of Iowa Stead Family Children's Hospital, Iowa City, Iowa, United States|Dana Farber / Boston Children's Hospital, Boston, Massachusetts, United States|UH Rainbow Babies and Children's Hospital (University Hospitals Cleveland Medical Center), Cleveland, Ohio, United States|Cleveland Clinic Foundation, Cleveland, Ohio, United States|Oregon Health and Science University, Portland, Oregon, United States|Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pennsylvania, United States|Fred Hutch/University of Washington Cancer Consortium, Seattle, Washington, United StatesMore Information
Obinutuzumab in cGVHD After Allogeneic Peripheral Blood Stem Cell Transplantation This research study is studying a drug called obinutuzumab as a means of preventing chronic Graft vs. Host Disease (cGVHD).
Phase 2RecruitingDrug: Obinutuzumab|Drug: PlaceboStanford University, Palo Alto, California, United States|Massachusetts General Hospital, Boston, Massachusetts, United States|Dana Farber Cancer Institute, Boston, Massachusetts, United States|Masonic Cancer Center, Minneapolis, Minnesota, United States|Huntsman Cancer Institute, Salt Lake City, Utah, United StatesMore Information

Top Treatments in Research

AgentClass/Mechanism of ActionDevelopment StatusCompanyCompany ContactClinical StudiesMore Information
axatilimab (SNDX-6352)Donor-derived, pro-inflammatory macrophages are dependent on CSF-1R signaling and have been shown in preclinical studies to be responsible for symptoms associated with chronic graft versus host disease (cGVHD). Syndax believes that CSF-1R blockade with axatilimab (SNDX-6352) may reduce the number of these pro-inflammatory macrophages and play a meaningful role in the treatment of chronic graft versus host disease.Phase 2Syndax PharmaceuticalsMichael L. Meyers, M.D., Ph.D.Chief Medical OfficerMore InfoMore Info
Ruxolitiniba first-in-class JAK1/JAK2 inhibitor approved by the U.S. FDA for treatment of polycythemia vera (PV) in adults who have had an inadequate response to or are intolerant of hydroxyurea, in adults with intermediate or high-risk myelofibrosis (MF), including primary MF, post-polycythemia vera MF and post-essential thrombocythemia MF and for treatment of steroid-refractory acute GVHD in adult and pediatric patients 12 years and older.
Phase 2/3Incyte CorporationSteven H. Stein, MD

Executive Vice President and Chief Medical Officer
More InfoMore Info
AcalabrutinibAcalabrutinib is a highly selective, irreversible, second generation BTK inhibitor, with approximately 1,000 patients treated to date in clinical studies across the entire development program.Phase 2AstraZenecaNot availableMore InfoMore Info
ObinutuzumabAn engineered monoclonal antibody designed to attach to CD20, a protein found only on B-cells. It attacks targeted cells both directly and together with the body's immune system. Gazyva was discovered by Roche Glycart AG, a wholly owned, independent research unit of Roche. In the United States, Gazyva is part of a collaboration between Genentech and BiogenPhase 2Roche-GenentechNot availableMore InfoMore Info