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ORPHA:39812

Graft versus host disease

medium confidenceDisorder

Also known as: GvHD

Publications

90,744

99.5th percentile

Trials

794

Interventional, condition-specific

Researchers

1,512

Distinct authors in sample

Gene link

—

Readiness

4/6

Stages with a signal

Clinical definition (Orphanet)

A rare immune disease characterized by donor-derived immune cells, primarily T cells, recognizing the recipient's tissues as foreign and mounting an immune response against them. It is a significant complication following allogeneic hematopoietic cell transplantation (alloHCT). Graft versus host disease (GVHD) is classified into acute (aGVHD) and chronic (cGVHD) forms.

How rare: 1-9 / 100 000 — about one to nine people per hundred thousand.

Orphanet entry

Cross-references

Joined from Mondo / Orphanet. MeSH labels may enter searches; UMLS / OMIM / NCIT are stored for reference.

Additional Mondo synonyms (13)

GVH · GVHD · disease, graft-versus-host · disease, graft-vs-host · diseases, graft-versus-host · diseases, graft-vs-host · graft versus host disease · graft vs host disease · graft vs. host disease · graft-versus-host disease · graft-versus-host-disease · graft-vs-host disease · runt disease

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.”

4/6 stages with a signal

An interventional trial matched this condition name on ClinicalTrials.gov — see trials below.

  1. Gene identifiedNot found

    No GenCC disease–gene assertion in this build

  2. LiteraturePresent

    90,744 matched papers (48,044 in last 10 years) Source

  3. Phenotype characterisedPresent

    94 HPO annotations (e.g. Inflammatory abnormality of the skin; Oral ulcer; Skin erosion) Source

  4. Animal modelNot found

    No Alliance genotype “model of” associations via Monarch for these Mondo IDs

  5. Orphan designationPresent

    15 FDA · 5 EMA designations (15 FDA orphan-indication approvals) — e.g. arsenic trioxide Source

  6. Interventional trialPresent

    794 matched on ClinicalTrials.gov (130 recruiting in sample)

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)

94

Associated phenotypes · MONDO:0013730

  • Inflammatory abnormality of the skin
  • Oral ulcer
  • Skin erosion
  • Chronic hepatitis
  • Irritability

Showing 5 of 94 — 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)

30

Designations · 15 with FDA orphan-indication approval

  • FDA arsenic trioxideGraft-Versus-Host Disease · 2018-07-30 · Not FDA Approved for Orphan Indication
  • FDA synthetic preImplantation factorGRAFT VERSUS HOST DISEASE · 2018-05-08 · Not FDA Approved for Orphan Indication
  • FDA abataceptGRAFT VERSUS HOST DISEASE · 2017-12-26 · Not FDA Approved for Orphan Indication
  • FDA alpha-1 proteinase inhibitor (human)GRAFT VERSUS HOST DISEASE · 2017-07-31 · Not FDA Approved for Orphan Indication
  • FDA itacitinibGRAFT VERSUS HOST DISEASE · 2017-04-17 · Not FDA Approved for Orphan Indication
  • FDA VedolizumabGRAFT VERSUS HOST DISEASE · 2017-03-21 · Not FDA Approved for Orphan Indication
  • FDA echinomycinGraft-Versus-Host Disease · 2017-01-05 · Not FDA Approved for Orphan Indication
  • FDA cannabidiolGRAFT VERSUS HOST DISEASE · 2016-11-29 · Not FDA Approved for Orphan Indication

Sources: FDA OOPD · EMA orphan designations

Open Targets candidates

134

Drugs / clinical candidates · MONDO_0013730

CTD chemicals (MyDisease.info)

21 associated chemicals · 73 pathways. Therapeutic evidence is listed first when present — not a treatment recommendation.

  • ((1-ethylpyrrolidin-2-yl) methyl)-4-hydroxy-7-methoxy-quinoline-3-carboxamide · therapeutic
  • Azathioprine · therapeutic
  • Cyclophosphamide · therapeutic
  • Cyclosporine · therapeutic
  • Cyclosporins · therapeutic
  • Everolimus · therapeutic
  • Ketotifen · therapeutic
  • Methotrexate · therapeutic
  • Methylprednisolone · therapeutic
  • Mycophenolic Acid · therapeutic
  • Prednisolone · therapeutic
  • Prednisone · therapeutic

Pathways: Pentose and glucuronate interconversions; Ascorbate and aldarate metabolism; Steroid hormone biosynthesis; One carbon pool by folate; Retinol metabolism; Porphyrin and chlorophyll metabolism; Metabolism of xenobiotics by cytochrome P450; Drug metabolism - cytochrome P450

MyDisease.info · MONDO:0013730

Literature

Is anyone studying this?

90,744

90,744 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.

90,744 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).

48,044 in the last 10 years · medium confidence · 99.5th percentile (publications denominator)

Phrase hits: 90,744 · MeSH hits: 0

Open Europe PMC search

Who's working on it?

1,512

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.

  1. 01
    Wang X12 papers · 2026

    Department of Hematology, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, Hubei, China; Immunotherapy Research Center for Hematologic Diseases of Hubei Province, Wuhan, Hubei, China.

    Papers in Europe PMC
  2. 02
    Wang L7 papers · 2026

    Department of Hematology, Shanghai Ruijin Hospital, Shanghai Jiaotong University School of Medicine, Shanghai, China.

    Papers in Europe PMC
  3. 03
    Zhang Y7 papers · 2026

    Department of Hematology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.

    Papers in Europe PMC
  4. 04
    Lee SJ5 papers · 2026

    Clinical Research Division, Fred Hutchinson Cancer Center, Seattle, Washington.

    Papers in Europe PMC
  5. 05
    Chen Y4 papers · 2026

    Department of Hematology, Xiangya Hospital, Central South University, Changsha, Hunan, China.

    Papers in Europe PMC
  6. 06
    Cutler C4 papers · 2026

    Division of Stem Cell Transplantation and Cellular Therapy, Dana-Farber Cancer Institute, Boston, Massachusetts.

    Papers in Europe PMC
  7. 07
    Holler E4 papers · 2026

    Department of Internal Medicine III, University Hospital Regensburg, Regensburg, Germany.

    Papers in Europe PMC
  8. 08
    Li N4 papers · 2026

    State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin, China; Tianjin Institutes of Health Science, Tianjin, China.

    Papers in Europe PMC
  9. 09
    Li X4 papers · 2026

    Department of Hematology, The Third Xiangya Hospital, Central South University, Changsha, Hunan, China.

    Papers in Europe PMC
  10. 10
    Malard F4 papers · 2026

    Service d'Hématologie Clinique et Thérapie Cellulaire, Hôpital Saint-Antoine, Sorbonne Université, INSERM UMRs 938, Paris, France. florent.malard@inserm.fr.

    Papers in Europe PMC

Clinical research

Is a treatment being tested?

794

interventional trials for this specific condition

794 interventional trials matched this specific condition name; 130 currently recruiting in our sample.

Data as of 11 September 2026

794 interventional trials — more than 77.2% of diseases in the trials denominator have none at all (5501 of 7126; this disease is at the 99.9th percentile).

medium confidence · 99.9th percentile (trials denominator)

Recruiting interventional trials

From the matched ClinicalTrials.gov set

794 interventional trials matched after quoted-phrase search and title/condition post-filter.

Observational and natural-history studies

127 observational studies match this condition. These do not test a treatment and are not counted in the interventional-trial headline, but they are genuine research: natural-history work often defines the endpoints needed for a future rare-disease trial, and families may be able to enroll.

Recruiting or not-yet-recruiting

Open the complete matched search on ClinicalTrials.gov

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 161 · after dedupe 158 · already on CT.gov 0 · kept 0 · parent 0 · uncertain 158 · dropped 0 · fetched 2026-07-29

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 (158)

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 Graft versus host disease — check Associations / patient organisations on that page.

India — NPRD

Last verified 2026-07-26

This 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.

"Graft versus host disease" OR "disease, graft-versus-host" OR "disease, graft-vs-host" OR "diseases, graft-versus-host" OR "diseases, graft-vs-host" OR "graft vs host disease" OR "graft vs. host disease" OR "graft-versus-host disease" OR "graft-versus-host-disease" OR "graft-vs-host disease" OR "runt disease"

Run this search on Europe PMC

ClinicalTrials.gov query (quoted phrases + MeSH via query.cond, plus recall-expansion terms when used):

"Graft versus host disease" OR "disease, graft-versus-host" OR "disease, graft-vs-host" OR "diseases, graft-versus-host" OR "diseases, graft-vs-host" OR "graft vs host disease" OR "graft vs. host disease" OR "graft-versus-host disease" OR "graft-versus-host-disease" OR "graft-vs-host disease" OR "runt disease"

Interventional trials matched via: phrase (mesh = registered under a MeSH descriptor no name phrase would catch; recall-expansion = gene / selected parent terms used only for trials).

Study-type breakdown: 794 interventional · 127 observational · 11 expanded access. Only interventional studies enter the trial headline.

Query health: ok — strategies attempted: phrase; with hits: phrase

Run this search on ClinicalTrials.gov

Synonyms dropped by stoplist: GvHD; GVH

Confidence reasoning

  • Preferred label is multi-word and distinctive
  • 2 synonym(s) dropped by stoplist (may under-count)
  • No label/synonym collisions with other diseases in this corpus

Ingested 2026-07-27T00:03:18.686Z