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Autoimmune thrombocytopenic purpura

Disease ID:1211
Name:Autoimmune thrombocytopenic purpura
Associated with:0 target
5 immuno-relevant ligands
idiopathic thrombocytopenic purpura | Immune thrombocytopenia | Immune thrombocytopenic purpura | ITP | primary immune thrombocytopenia | primary thrombocytopenic purpura
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


No target related data available for Autoimmune thrombocytopenic purpura


Key to terms and symbols Click ligand name to view ligand summary Click column headers to sort
Ligand References Clinical and Disease comments
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
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
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
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
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


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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]