Autoimmune thrombocytopenic purpura

Disease ID:1211
Name:Autoimmune thrombocytopenic purpura
Associated with:0 targets
0 immuno-relevant targets
4 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


Ligand Approved Immuno References Clinical comments
Clinical Use: Rozanolixizumab (as research code UCB7665) has progressed to Phase 2 clinical trial in patients with myasthenia gravis (NCT03052751) and primary immune thrombocytopenia (NCT02718716).
Immuno Disease Comments: Phase 2 clinical candidate for primary immune thrombocytopenia- see NCT02718716
prednisolone  [  ( ]
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).
Immuno Disease Comments: Glucocorticoid drug used to treat many inflammatory condtions including ATP.
deflazacort  [  ( ]
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 to treat Duchenne muscular dystrophy [1].
Immuno Disease Comments: Approved corticosteroid that can be prescribed for ATP.
fostamatinib  [  ( ]
Clinical Use: Fostamatinib (as the disodium hexahydrate salt) received FDA approval for the treatment of chronic immune thrombocytopenia (ITP) [4] 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 [2] (NCT01197534 & NCT01197755), Phase 2 for autoimmune thrombocytopenia [3] and lymphoma (NCT00446095) and a range of solid tumours (NCT00923481).
Immuno Disease Comments: Approved drug for ITP. FDA approval granted in April 2018.


Show »

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. 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]

3. Newland A, Lee EJ, McDonald V, Bussel JB. (2018) Fostamatinib for persistent/chronic adult immune thrombocytopenia. Immunotherapy, 10 (1): 9-25. [PMID:28967793]

4. 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]