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Autoimmune thrombocytopenic purpura

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:1211
Name:Autoimmune thrombocytopenic purpura
Associated with:0 target
5 immuno-relevant ligands
Synonyms
idiopathic thrombocytopenic purpura | Immune thrombocytopenia | Immune thrombocytopenic purpura | ITP | primary immune thrombocytopenia | primary thrombocytopenic purpura
Description
Autoimmune thrombocytopenic purpura (a.k.a. primary immune thrombocytopenia) is an acquired autoimmune disorder of children and adults. The condition is caused by destruction of platelets by an autoantibody to platelet glycoproteins. It is characterised by a low platelet count, and clinical signs include petechiae, purpura, bruising, and overt bleeding.
Database Links
Disease Ontology: DOID:8924
OMIM: 188030
Orphanet: ORPHA3002

Targets

GtoPdb Disease Summaries - Targets

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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

No target related data available for Autoimmune thrombocytopenic purpura

Ligands

GtoPdb Disease Summaries - Ligands

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  • 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
rozanolixizumab
Immuno Disease Comments: Phase 2 clinical candidate for primary immune thrombocytopenia- see NCT02718716
Clinical Use: Rozanolixizumab (as research code UCB7665) was progressed into clinical trial in patients with myasthenia gravis and primary immune thrombocytopenia. EMA orphan designation as a treatment for myasthenia gravis has been in place since 2019.
First FDA approval was granted in June 2023, with rozanolixizumab authorised to treat patients with generalized myasthenia gravis who are positive for anti-acetylcholine receptor or anti-muscle-specific tyrosine kinase antibodies [2]. | View clinical data
prednisolone
Immuno Disease Comments: Glucocorticoid drug used to treat many inflammatory condtions including ATP.
Clinical Use: This drug used as an antiinflammatory or immunosuppressive agent and is indicated for the treatment of various inflammatory pathologies, including acute asthma, suppression of inflammatory and allergic disorders, ulcerative colitis, Crohn's disease, idiopathic thrombocytopenic purpura, rheumatoid arthritis, polymyalgia rheumatica, systemic lupus erythematosus and chronic obstructive pulmonary disease (COPD). | View clinical data
deflazacort
Immuno Disease Comments: Approved corticosteroid that can be prescribed for ATP.
Clinical Use: Deflazacort can be prescribed for many inflammatory conditions including asthma, rheumatoid arthritis, Crohn's disease, juvenile chronic arthritis, idiopathic thrombocytopenic purpura, polymyalgia rheumatica, systemic lupus erythematosus and ulcerative colitis. More recently approved by the FDA as a treatment for Duchenne muscular dystrophy [1]. | View clinical data
Bioactivity Comments: In vitro binding to rat kidney, thymus and liver glucocorticoid receptors is reported in [3]. | View biological activity
fostamatinib
Immuno Disease Comments: Approved drug for ITP. FDA approval granted in April 2018.
Clinical Use: Fostamatinib (as the disodium hexahydrate salt) received FDA approval for the treatment of chronic immune thrombocytopenia (ITP) [5] in April 2018, in particular for the treatment of patients with an inadequete response to previous treatment.
Fostamatinib has completed Phase 3 clinical trials for rheumatoid arthritis [4] (NCT01197534 & NCT01197755), and Phase 2 for a range of solid tumours (NCT00923481). | View clinical data
Bioactivity Comments: As fostamatinib is a prodrug it is unlikely to have intrinsic inhibitory activity. Bioactivity will be associated with its active form, tamatinib. | View biological activity
romiplostim
Immuno Disease Comments: Approved drug for autoimmune thrombocytopenic purpura (a.k.a. idiopathic thrombocytopenic purpura)
Clinical Use: Romiplostim is used to increase the platelet count in patients suffering from chronic idiopathic thrombocytopenic purpura (ITP), a disease characterised by autoimmune destruction of platelets. As of December 2018 the FDA permits use of romiplostim in pediatric ITP patients ≥1 year of age. | View clinical data

References

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1. Griggs RC, Miller JP, Greenberg CR, Fehlings DL, Pestronk A, Mendell JR, Moxley 3rd RT, King W, Kissel JT, Cwik V et al.. (2016) Efficacy and safety of deflazacort vs prednisone and placebo for Duchenne muscular dystrophy. Neurology, 87 (20): 2123-2131. [PMID:27566742]

2. Hoy SM. (2023) Rozanolixizumab: First Approval. Drugs, 83 (14): 1341-1347. [PMID:37656420]

3. Luzzani F, Glässer A. (1981) Differential binding in vitro to glucocorticoid receptors of deflazacort and prednisolone. Eur J Pharmacol, 76 (4): 427-30. [PMID:7327211]

4. Maringwa J, Kågedal M, Hamrén UW, Martin P, Cox E, Hamrén B. (2015) Pharmacokinetic-pharmacodynamic modeling of fostamatinib efficacy on ACR20 to support dose selection in patients with rheumatoid arthritis (RA). J Clin Pharmacol, 55 (3): 328-35. [PMID:25280085]

5. Niscola P, Scaramucci L, Giovannini M. (2018) Spleen tyrosine kinase inhibition: a new promising approach to chronic and refractory immune thrombocytopenia. Immunotherapy, 10 (1): 5-7. [PMID:29192558]