ORPHA:99921
Chronic graft versus host disease
Query health: suspect — Source fetch failed for trials.
Publications
11,683
Trials
—
Interventional, condition-specific
Researchers
1,501
Distinct authors in sample
Gene link
—
Readiness
3/6
Stages with a signal
Cross-references
Joined from Mondo / Orphanet. MeSH labels may enter searches; UMLS / OMIM / NCIT are stored for reference.
- MONDO:0020547
- UMLS:C0867389
- NCIT:C4981
Additional Mondo synonyms (2)
GVHD, chronic · graft versus host disease, chronic
Research stages
Trial readiness signals
Where this condition sits on an open-data research pipeline — not how close a treatment is, and not medical advice. Empty stages often mean “not in these databases under this Mondo ID,” not “impossible.”
3/6 stages with a signal
Research-stage checklist from open sources (GenCC, literature, Monarch when enriched, ClinicalTrials.gov). Not a prognosis or care recommendation.
- Gene identifiedNot found
No GenCC disease–gene assertion in this build
- LiteraturePresent
11,683 matched papers (6,628 in last 10 years) Source
- Phenotype characterisedPresent
53 HPO annotations (e.g. Poor wound healing; Alopecia; Onycholysis) Source
- Animal modelNot found
No Alliance genotype “model of” associations via Monarch for these Mondo IDs
- Orphan designationPresent
3 FDA designations (2 FDA orphan-indication approvals) — e.g. axatilimab Source
- Interventional trialNot checked
Trial fetch failed or incomplete
Biology
Genes and phenotypes
Gene–disease validity from GenCC, plus phenotypes and animal models joined from Monarch Initiative via Mondo ID — not a clinical diagnosis aid.
Do we know what causes it?
Not yet — the cause hasn't been pinned down in GenCC.
No strong gene–disease assertion joined for this Orphanet entity.
Phenotypes (Monarch / HPO)
53
Associated phenotypes · MONDO:0020547
- Poor wound healing
- Alopecia
- Onycholysis
- Elevated circulating hepatic transaminase concentration
- Nail dystrophy
Showing 5 of 53 — open Monarch for the full list.
Animal models (Monarch / Alliance)
None returned for this Mondo ID. Empty here is not proof that no model organism work exists under another name or gene.
Monarch fetch 2026-07-29
Therapies
Designations, candidates, and chemicals
FDA OOPD and EMA orphan designations, Open Targets clinical candidates, and CTD chemical associations via MyDisease.info. These never change the interventional-trial headline.
Orphan designation (FDA · EMA)
3
Designations · 2 with FDA orphan-indication approval
- FDA axatilimabChronic Graft Versus Host Disease · 2021-03-29 · Not FDA Approved for Orphan Indication
- FDA entospletinibChronic graft-versus-host disease · 2017-08-10 · Not FDA Approved for Orphan Indication
- FDA ibrutinib (Imbruvica)Chronic Graft Versus Host Disease · 2016-06-23
Sources: FDA OOPD · EMA orphan designations
Open Targets candidates
60
Drugs / clinical candidates · MONDO_0020547
- AXATILIMAB·phase 3
- BASILIXIMAB·phase 3
- BETAMETHASONE·phase 3
- BETAMETHASONE SODIUM PHOSPHATE·phase 3
- BUDESONIDE·phase 3
- DEXAMETHASONE·phase 3
- EVEROLIMUS·phase 3
- HYDROCORTISONE·phase 3
- IBRUTINIB·phase 3
- IGURATIMOD·phase 3
- IMATINIB·phase 3
- INFLIXIMAB·phase 3
- METHOTREXATE·phase 3
- METHYLPREDNISOLONE·phase 3
- MYCOPHENOLATE MOFETIL·phase 3
CTD chemicals (MyDisease.info)
No CTD chemical associations returned for this Mondo ID.
Literature
Is anyone studying this?
11,683
11,683 papers — among the better-studied rare conditions, though still a fraction of common-disease literature (breast cancer: over 700,000). Median papers in the last 10 years for a rare disease in this dataset (publications denominator n=3967) is 59.
11,683 papers since the earliest indexed year in this search — median last-10-year count for a rare disease in this dataset is 59 (publications denominator n=3967).
6,628 in the last 10 years · low confidence
Phrase hits: 11,683 · MeSH hits: 0
Who's working on it?
1,501
Distinct author names in 200 sampled papers — named people below.
Who's working on it?
People publishing on this condition (sampled Europe PMC records). Affiliation is the most recent found in that sample.
- 01
- 02Lee SJ9 papers · 2026
Clinical Research Division, Fred Hutchinson Cancer Center and Department of Medicine, University of Washington, Seattle, Washington.
Papers in Europe PMC - 03Pavletic SZ9 papers · 2026
Immune Deficiency Cellular Therapy Program, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland.
Papers in Europe PMC - 04El Jurdi N7 papers · 2026
Immune Deficiency Cellular Therapy Program, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Bethesda, Maryland.
Papers in Europe PMC - 05Cowen EW6 papers · 2026
Dermatology Branch, National Institute of Arthritis and Musculoskeletal and Skin Diseases, National Institutes of Health, Bethesda, Maryland.
Papers in Europe PMC - 06Cutler C5 papers · 2026
Division of Transplantation and Cellular Therapy, Dana-Farber Cancer Institute, Boston, MA.
Papers in Europe PMC - 07Hamilton BK5 papers · 2026
Blood and Marrow Transplant Program, Taussig Cancer Institute, Cleveland Clinic, Cleveland, Ohio. Electronic address: hamiltb2@ccf.org.
Papers in Europe PMC - 08Jędrzejczak WW5 papers · 2026
Department of Hematology, Transplantation and Internal Medicine, Medical University of Warsaw, ul. Banacha 1A, Warsaw, 02-097, Poland.
Papers in Europe PMC - 09Kitko CL5 papers · 2026
Department of Pediatrics, Vanderbilt University Medical Center, Nashville, TN.
Papers in Europe PMC - 10Mays JW5 papers · 2026
National Institute of Dental and Craniofacial Research, National Institutes of Health, Bethesda, Maryland, United States.
Papers in Europe PMC
Clinical research
Is a treatment being tested?
—
interventional trials for this specific condition
We could not load trial data for this condition right now.
Data as of 11 September 2026
low confidence
Recruiting interventional trials
From the matched ClinicalTrials.gov set
Trial data could not be loaded for this build. This is not the same as finding zero interventional trials.
Other registries (secondary)
Broader net from EU CTIS, ISRCTN, and ICTRP when available — deduped against ClinicalTrials.gov IDs already counted above. Dual-model LLM relevance gates what we keep. These rows are not added to the interventional headline.
raw 141 · after dedupe 137 · already on CT.gov 0 · kept 0 · parent 0 · uncertain 137 · dropped 0 · fetched 2026-07-30
Source notes: ictrp: Error: ICTRP public search unavailable (WHO portal is SPA-only; SOAP needs partnership). Tried: https://apps.who.int/tri
No secondary-registry studies passed dual-model relevance for this condition name (after dedupe).
Uncertain / not reviewed (137)
- ctis·2025-524468-40-00·Authorised, recruiting·A multiCenter phase I study of aUtologous CD19 CAR T cElls for therapy refractory chronic GvHD (CURE-cGvHD)
skipped — LLM skipped (--skip-llm)
- ctis·2025-521940-39-00·Authorised, ongoing·Phase 2 Trial to determine the efficacy of belumosudil for patients with steroid-refractory overlap of syndrome Graft-versus-Host-Disease (SR-osGVHD) - BEAT-GVHD
skipped — LLM skipped (--skip-llm)
- ctis·2025-522680-14-00·Authorised, ongoing·Study of Q702 in Subjects with Relapsed or Refractory Chronic Graft-versus-Host Disease (cGVHD)
skipped — LLM skipped (--skip-llm)
- ctis·2025-521849-25-00·Authorised, ongoing·A Phase 2, Randomized, Open-Label Study Of Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft Versus Host Disease After at Least 2 Prior Lines of Systemic Therapy (AGAVE-256)
skipped — LLM skipped (--skip-llm)
- ctis·2024-511508-18-00·Authorised, recruiting·An open-label, Phase 1/2, multicenter study of belumosudil in children aged 1 to <18 years requiring systemic treatment for active moderate-to-severe chronic graft versus host disease (cGVHD).
skipped — LLM skipped (--skip-llm)
- ctis·2024-519801-36-00·Cancelled·An open label, balanced, randomized, two-treatment, two-period, two-sequence, single-dose, crossover, bioequivalence study comparing Ruxolitinib Tablets, 20 mg, manufactured by Sun Pharmaceutical Industries Limited, India with JAKAVI (ruxolitinib) tablets 20 mg, (each Jakavi 20 mg tablet contains 26.40 mg of ruxolitinib phosphate equivalent to 20 mg of ruxolitinib). Imported by: Novartis Biociências SA Av. Prof. Vicente Rao, 90 - São Paulo - SP CNPJ: 56.994.502/0001-30. Manufactured by: Novartis Pharma Stein AG, Stein – Switzerland, in healthy adult, human subjects under fasting condition.
skipped — LLM skipped (--skip-llm)
- ctis·2024-518973-32-00·Authorised, recruiting·A Phase 3, Randomized, Open-Label Study of Axatilimab Versus Best Available Therapy in Participants With Chronic Graft-Versus-Host Disease After at Least 2 Prior Lines of Systemic Therapy
skipped — LLM skipped (--skip-llm)
- ctis·2023-510292-65-00·Authorised, ongoing·A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Axatilimab (INCA034176) and Corticosteroids as Initial Treatment for Chronic Graft-Versus Host Disease (AXemplify 357)
skipped — LLM skipped (--skip-llm)
- ctis·2024-514484-25-00·Authorised, ongoing·PHASE II STUDY OF TOTAL MARROW AND LYMPHOID IRRADIATION (TMLI) ADMINISTERED IN COMBINATION WITH A MYELOABLATIVE REGIMEN BASED ON CYCLOPHOSPHAMIDE AND ETOPOSIDE AS A CONDITIONING FOR ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION (AHSCT) IN PATIENTS WITH HIGH-RISK MYELODYSPLASTIC SYNDROME OR ACUTE MYELOID LEUKEMIA
skipped — LLM skipped (--skip-llm)
- ctis·2024-517477-24-00·Expired·Assessment of Tolerance and Effectiveness of Total Body Irradiation and Cladribine Before Allogeneic Hematopoietic Cell Transplantation in Patients With Acute Myeloid Leukemia and Myelodysplastic Syndromes
skipped — LLM skipped (--skip-llm)
- ctis·2024-514483-11-00·Authorised, ongoing·PHASE II STUDY OF TOTAL MARROW AND LYMPHOID IRRADIATION (TMLI) ADMINISTERED IN COMBINATION WITH A REDUCED-INTENSITY REGIMEN BASED ON FLUDARABINE AND MELPHALAN AS CONDITIONING FOR ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION (AHSCT) IN PATIENTS WITH HIGH-RISK MYELODYSPLASTIC SYNDROME OR ACUTE MYELOID LEUKEMIA.
skipped — LLM skipped (--skip-llm)
- ctis·2023-507754-33-00·Authorised, ongoing·Phase 2 Trial to determine the safety and efficacy of simultaneous treatment with ruxolitinib and extracorporeal photopheresis for patients with steroid-refractory chronic Graft-versus-Host-Disease (SR-cGvHD) - RUX-ECP
skipped — LLM skipped (--skip-llm)
- ctis·2024-515283-31-00·Expired·An open label, multi-center, Phase IV rollover protocol for patients who have completed a prior global Novartis or Incyte sponsored ruxolitinib (INC424) study or a study of ruxolitinib in combination with either panobinostat (LBH589) or siremadlin (HDM201) or rineterkib (LTT462), and are judged by the investigator to benefit from continued treatment
skipped — LLM skipped (--skip-llm)
- ctis·2022-502168-19-00·Expired·INCA34176-254: A Phase 2, Open-Label, Randomized, Multicenter Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host Disease
skipped — LLM skipped (--skip-llm)
- ctis·2024-510590-14-00·Cancelled·Allogeneic stem cell transplantation vs. conventional therapy as salvage therapy for relapsed / progressive patients with multiple myeloma after a first-line therapy
skipped — LLM skipped (--skip-llm)
- ctis·2024-514351-14-00·Authorised, ongoing·ErythroSIM : Randomized prospective trial evaluating the efficacy of the antiCD38 monoclonal antibody isatuximab in the treatment of PCRA by major ABO mismatch after allogeneic hematopoietic stem cell transplantation
skipped — LLM skipped (--skip-llm)
- ctis·2024-512978-99-00·Expired·AGAVE-201, A Phase 2, Open-label, Randomized, Multicenter Study to Evaluate the Efficacy, Safety and Tolerability of Axatilimab at 3 Different Doses in Patients with Recurrent or Refractory Active Chronic Graft Versus Host Disease who have Received at least 2 Lines of Systemic Therapy.
skipped — LLM skipped (--skip-llm)
- ctis·2023-508420-36-00·Authorised, ongoing·Phase I clinical trial on the use of fresh, allogeneic, second-generation CD19-CAR T cells for treatment of children and young adult with relapsed/refractory B-cell Acute Lymphoblastic Leukemia
skipped — LLM skipped (--skip-llm)
- ctis·2023-507330-24-00·Cancelled·Phase 1/2 Dose Finding, Safety and Efficacy Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)
skipped — LLM skipped (--skip-llm)
- ctis·2023-505737-26-00·Authorised, ongoing·A Randomised, Open label, Multicentre, Phase 3 Trial of First line Treatment with Mesenchymal Stromal Cells MC0518 Versus Best Available Therapy in Adult and Adolescent Subjects with Steroid refractory Acute Graft versus host Disease After Allogeneic Haematopoietic Stem Cell Transplantation (IDUNN Trial)
skipped — LLM skipped (--skip-llm)
- ctis·2023-505394-32-00·Expired·A randomized, double-blind, multicenter, Phase 3 study to evaluate efficacy and safety of belumosudil in combination with corticosteroids versus placebo in combination with corticosteroids in participants at least 12 years of age with newly diagnosed chronic graft versus host disease (cGVHD)
skipped — LLM skipped (--skip-llm)
- ctis·2023-503952-28-00·Expired·A Randomised, Open label, Controlled, Multicentre, Phase 2 Trial of First line Treatment with Mesenchymal Stromal Cells MC0518 Versus Best Available Therapy in Paediatric Participants with Steroid refractory Acute Graft versus host Disease After Allogeneic Stem Cell Transplantation (BALDER Trial)
skipped — LLM skipped (--skip-llm)
- ctis·2022-501661-47-00·Cancelled·INCB 39110-801 A Phase 2, Open-Label, Multicenter, Rollover Study to Provide Continued Treatment for Participants Previously Enrolled in Studies of Itacitinib (INCB039110)
skipped — LLM skipped (--skip-llm)
- ctis·2022-502019-12-00·Authorised, ongoing·FLUCLORIC - Randomized multicentric Phase III study comparing the efficacy of two reduced intensity conditioning regimens (clofarabine/busulfan versus fludarabine/busulfan) in adults with acute myeloid leukemia and eligible to allogeneic stem cell transplantation: a SFGM-TC study
skipped — LLM skipped (--skip-llm)
- ctis·2026-526368-18-00·Authorised·A Randomized Controlled Study Evaluating Short-Term Dual Antiplatelet Therapy with Low-Dose Ticagrelor (60 mg) Followed by Monotherapy versus Standard-Duration Dual Antiplatelet Therapy with Clopidogrel in Patients with Chronic Coronary Syndrome Undergoing Percutaneous Coronary Intervention: the STELAR trial
skipped — LLM skipped (--skip-llm)
Where to find support
Condition-specific patient organisations, when Orphanet lists them, are on the disease’s Orphanet page. We also link umbrella groups that support undiagnosed and ultra-rare families.
Orphanet entry for Chronic graft versus host disease — check Associations / patient organisations on that page.
India — NPRD
Last verified 2026-07-26This ORPHAcode is not on our curated NPRD list (direct or Mondo-parent match). That does not decide clinical eligibility; families in India should ask a notified Centre of Excellence about current coverage.
Hand-curated for this project. ORPHAcode mappings are best-effort and may be incomplete or imprecise for umbrella categories. Parent (Mondo) matches mean the policy lists a broader category — confirm eligibility with a Centre of Excellence. Financial entitlements summarised from public policy statements and may change. This is not official government guidance.
How we counted this
Europe PMC query (preferred label + any corrected label + Orphanet and Mondo exact synonyms, stoplisted; unioned with resolved MeSH labels when available). UMLS / OMIM / NCIT cross-references are stored on the overview but are not added to the query string.
"Chronic graft versus host disease" OR "GVHD, chronic" OR "graft versus host disease, chronic"
ClinicalTrials.gov query (quoted phrases + MeSH via query.cond, plus recall-expansion terms when used):
(empty)
Query health: suspect — strategies attempted: phrase; with hits: phrase
Source errors: trials: skipped ClinicalTrials.gov (--skip-trials; typically page-ceiling unmeasurable)
Confidence reasoning
- Preferred label is multi-word and distinctive
- No synonyms dropped by stoplist
- No label/synonym collisions with other diseases in this corpus
- Publication count (11683) is extremely high with unknown/missing prevalence — treat as possible over-matching, not proven research intensity
Ingested 2026-07-27T21:49:13.768Z