RT Generic
T1 Novel Therapies to Address Unmet Needs in ITP.
A1 Mingot-Castellano, Maria Eva
A1 Bastida, Jose Maria
A1 Caballero-Navarro, Gonzalo
A1 Entrena Ureña, Laura
A1 Gonzalez-Lopez, Tomas Jose
A1 Gonzalez-Porras, Jose Ramon
A1 Butta, Nora
A1 Canaro, Mariana
A1 Jimenez-Barcenas, Reyes
A1 Gomez-Del-Castillo-Solano, Maria Del Carmen
A1 Sanchez-Gonzalez, Blanca
A1 Pascual-Izquierdo, Cristina
K1 autoantibodies
K1 immune thrombocytopenia
K1 platelets
K1 targeted therapies
K1 thrombopoietin
AB Primary immune thrombocytopenia (ITP) is an autoimmune disorder that causes low platelet counts and subsequent bleeding risk. Although current corticosteroid-based ITP therapies are able to improve platelet counts, up to 70% of subjects with an ITP diagnosis do not achieve a sustained clinical response in the absence of treatment, thus requiring a second-line therapy option as well as additional care to prevent bleeding. Less than 40% of patients treated with thrombopoietin analogs, 60% of those treated with splenectomy, and 20% or fewer of those treated with rituximab or fostamatinib reach sustained remission in the absence of treatment. Therefore, optimizing therapeutic options for ITP management is mandatory. The pathophysiology of ITP is complex and involves several mechanisms that are apparently unrelated. These include the clearance of autoantibody-coated platelets by splenic macrophages or by the complement system, hepatic desialylated platelet destruction, and the inhibition of platelet production from megakaryocytes. The number of pathways involved may challenge treatment, but, at the same time, offer the possibility of unveiling a variety of new targets as the knowledge of the involved mechanisms progresses. The aim of this work, after revising the limitations of the current treatments, is to perform a thorough review of the mechanisms of action, pharmacokinetics/pharmacodynamics, efficacy, safety, and development stage of the novel ITP therapies under investigation. Hopefully, several of the options included herein may allow us to personalize ITP management according to the needs of each patient in the near future.
PB MDPI AG
SN 1424-8247
YR 2022
FD 2022-06-23
LK http://hdl.handle.net/10668/21535
UL http://hdl.handle.net/10668/21535
LA en
NO Mingot-Castellano ME, Bastida JM, Caballero-Navarro G, Entrena Ureña L, González-López TJ, González-Porras JR, et al. Novel Therapies to Address Unmet Needs in ITP. Pharmaceuticals (Basel). 2022 Jun 23;15(7):779.
DS RISalud
RD Aug 6, 2025