Idiopathic thrombocytopenic purpura: current concepts in pathophysiology and management.
Idiopathic thrombocytopenic purpura (ITP) is characterized by a low platelet count, which is the result of both increased platelet destruction and insufficient platelet production. Although the development of autoantibodies against platelet glycoproteins remains central in the pathophysiology of ITP, several abnormalities involving the cellular mechanisms of immune modulation have been identified. Conventional treatments for ITP aim at reducing platelet destruction, either by immunosuppression or splenectomy. Two new thrombopoietic agents, AMG 531 and eltrombopag, have been used in clinical trials to stimulate platelet production in ITP patients not responsive to standard treatments. These new molecules bear no structural resemblance to thrombopoietin, but still bind and activate the thrombopoietin receptor. This review will focus on the pathophysiology and treatment of ITP in adults, highlighting recent advances in both fields.
Stasi
Roberto
R
Department of Medical Sciences, Ospedale, Regina Apostolorum Hospital, Albano Laziale, Italy. roberto.stasi@uniroma2.it
Evangelista
Maria Laura
ML
Stipa
Elisa
E
Buccisano
Francesco
F
Venditti
Adriano
A
Amadori
Sergio
S
D000818:Animals; D001323:Autoantibodies / Q000097:blood; D001402:B-Lymphocytes / Q000187:drug effects; D001565:Benzoates / Q000627:therapeutic use; D001792:Blood Platelets / Q000187:drug effects* / Q000276:immunology; D002352:Carrier Proteins / Q000627:therapeutic use; D002450:Cell Communication / Q000187:drug effects; D005938:Glucocorticoids / Q000627:therapeutic use; D006401:Hematologic Agents / Q000494:pharmacology / Q000627:therapeutic use*; D006801:Humans; D006834:Hydrazines / Q000627:therapeutic use; D007108:Immune Tolerance / Q000187:drug effects; D016756:Immunoglobulins, Intravenous / Q000627:therapeutic use; D007155:Immunologic Factors / Q000494:pharmacology / Q000627:therapeutic use*; D010976:Platelet Count; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000097:blood / Q000276:immunology / Q000503:physiopathology* / Q000628:therapy*; D011720:Pyrazoles / Q000627:therapeutic use; D011961:Receptors, Fc / Q000627:therapeutic use; D011993:Recombinant Fusion Proteins; D013156:Splenectomy; D013601:T-Lymphocytes / Q000187:drug effects; D034061:Thrombopoiesis / Q000187:drug effects; D013926:Thrombopoietin; D017211:Treatment Failure
D001323:Autoantibodies; D001565:Benzoates; D002352:Carrier Proteins; D005938:Glucocorticoids; D006401:Hematologic Agents; D006834:Hydrazines; D016756:Immunoglobulins, Intravenous; D007155:Immunologic Factors; D011720:Pyrazoles; D011961:Receptors, Fc; D011993:Recombinant Fusion Proteins; D013926:Thrombopoietin; C488777:romiplostim; C520809:eltrombopag