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Myelofibrosis

GtoPdb Disease Summaries

This section gives an overview of the disease, and where available shows the following:

  • Synonyms: Shows known synonyms for the disease.
  • Description: Gives a basic description/definition of the disease.
  • Database Link: External links to the same disease at the Disease Ontology, OMIM or Orphanet sites.
  • Immunopharmacology comments: General comments about the target's role in immunopharmacology, provided by GtoImmuPdb curators.
  • Associated with: Counts are displayed for the total targets the disease is associated with in GtoPdb. The counts of targets and ligands of immunological relevance associated to the disease are also shown.

More information can be found in the help pages.

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Disease ID:655
Name:Myelofibrosis
Associated with:3 targets
1 immuno-relevant target
2 immuno-relevant ligands
Synonyms
Myelofibrosis with myeloid metaplasia
Database Links
Disease Ontology: DOID:4971
OMIM: 254450
Orphanet: ORPHA824

Targets

GtoPdb Disease Summaries - Targets

Click on the target name to link to its detailed view page

Where available, information is display on the role of the target in the disease; drugs which target the disease and their therapeutic use and side-effects.

If there is mutation data curated in GtoPdb this is indicated, with a link back to the appropriate section on the target detailed view page

Immuno ligand interactions - If available, a table of immuno-relevant ligands is shown. These ligands have been curated as having an association to the disease and possess interaction data with the target in GtoPdb. The approval status of the ligand is shown, along with curator comments and an indication of whether the target is considered the primary target of the ligand.

More information can be found in the help pages.

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Key to terms and symbols
NK1 receptor
References:  2,5-6
Thrombopoietin receptor
Janus kinase 2
Comments:  Somatic mutations in the JAK2 gene have been identified in many cases of myelofibrosis.
Ligand interactions: 
Ligand Comments
ruxolitinib
Approved drug for myelofibrosis, in particular for primary myelofibrosis, post-polycythemia vera (post-PV) myelofibrosis and post-essential thrombocyt ...
itacitinib
Phase 2 clinical candidate for myelofibrosis (see NCT03144687).

Ligands

GtoPdb Disease Summaries - Ligands

Click ligand name to view ligand summary page

  • Approved: If the ligand is an approved drug this is indicated, along with approval bodies.
  • Immuno: Immuno icon indicates the ligand is immuno-relevant

Click the arrow in the final column to expand comments

  • Immuno Disease Comments: Curatorial comments specifically added as part of GtoImmuPdb. They give more information on the association between the ligand and disease in the context of immunopharmacology.
  • Clinical Use: General clinical comments relating to the ligand and may not necessarily be specific to the disease in question. With hyperlink to more details on the ligand summary pages.
  • Bioactivty Comments: Curatorial comments specifically about the compounds biological activity - with hyperlink to more details on the ligand summary pages.

More information can be found in the help pages.

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Key to terms and symbols Click ligand name to view ligand summary Click column headers to sort
Ligand References Clinical and Disease comments
ruxolitinib
Immuno Disease Comments: Approved drug for myelofibrosis, in particular for primary myelofibrosis, post-polycythemia vera (post-PV) myelofibrosis and post-essential thrombocythemia (post-ET) myelofibrosis.
Clinical Use: Used to treat intermediate or high-risk myelofibrosis including including primary myelofibrosis, post-polycythemia vera (post-PV) myelofibrosis and post-essential thrombocythemia (post-ET) myelofibrosis.
In 2014 ruxolitinib was approved for use as a treatment for polycythemia vera, a chronic type of bone marrow disease.
In May 2019, the FDA approved ruxolitinib as a treatment for steroid-refractory acute graft-versus-host disease (GvHD) in patients ≥12 years old.

Interesting results from small trials in patients with alopecia areata suggest that by killing the immune cells responsible for hair loss in this disease, ruxolitinib can promote hair re-growth [4,7]. | View clinical data
itacitinib
Immuno Disease Comments: Phase 2 clinical candidate for myelofibrosis (see NCT03144687).
Clinical Use: INCB039110 is being assessed in Phase 2 clinical trials as a potential treatment for indications such as rheumatoid arthritis (RA), post essential thrombocythemia myelofibrosis, chronic plaque psoriasis and non-small cell lung cancer (NSCLC). Phase 3 trials in chronic graft-versus-host disease are also underway. Click here to link to ClinicalTrials.gov's complete list of INCB039110 studies. | View clinical data

References

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1. Baxter EJ, Scott LM, Campbell PJ, East C, Fourouclas N, Swanton S, Vassiliou GS, Bench AJ, Boyd EM, Curtin N et al.. (2005) Acquired mutation of the tyrosine kinase JAK2 in human myeloproliferative disorders. Lancet, 365 (9464): 1054-61. [PMID:15781101]

2. Chang VT, Yook C, Rameshwar P. (2013) Synergism between fibronectin and transforming growth factor-β1 in the production of substance P in monocytes of patients with myelofibrosis. Leuk Lymphoma, 54 (3): 631-8. [PMID:22906243]

3. Kralovics R, Passamonti F, Buser AS, Teo SS, Tiedt R, Passweg JR, Tichelli A, Cazzola M, Skoda RC. (2005) A gain-of-function mutation of JAK2 in myeloproliferative disorders. N Engl J Med, 352 (17): 1779-90. [PMID:15858187]

4. Mackay-Wiggan J, Jabbari A, Nguyen N, Cerise JE, Clark C, Ulerio G, Furniss M, Vaughan R, Christiano AM, Clynes R. (2016) Oral ruxolitinib induces hair regrowth in patients with moderate-to-severe alopecia areata. JCI Insight, 1 (15): e89790. [PMID:27699253]

5. Rameshwar P, Joshi DD, Yadav P, Qian J, Gascon P, Chang VT, Anjaria D, Harrison JS, Song X. (2001) Mimicry between neurokinin-1 and fibronectin may explain the transport and stability of increased substance P immunoreactivity in patients with bone marrow fibrosis. Blood, 97 (10): 3025-31. [PMID:11342427]

6. Rameshwar P, Oh HS, Yook C, Gascon P, Chang VT. (2003) Substance p-fibronectin-cytokine interactions in myeloproliferative disorders with bone marrow fibrosis. Acta Haematol, 109 (1): 1-10. [PMID:12486316]

7. Xing L, Dai Z, Jabbari A, Cerise JE, Higgins CA, Gong W, de Jong A, Harel S, DeStefano GM, Rothman L et al.. (2014) Alopecia areata is driven by cytotoxic T lymphocytes and is reversed by JAK inhibition. Nat Med, 20 (9): 1043-9. [PMID:25129481]