More treatment options are needed for patients with ITP1,2
Current treatment goals in ITP are to stop active bleeding and reduce the risk of further bleeding. Response rates for ITP treatments vary widely from 18% to 80% as measured by improved platelet counts. Some currently available treatments may also be associated with unfavorable side effect profiles and dosing regimens or offer a limited duration of response.
“I worry about my treatment failing at some point. I worry about the long-term health effects of the drugs that I’m taking.”
Tracey, US, patient with ITP
“Despite advances in treatment options over the years, some patients remain refractory to existing therapies and durable remission remains elusive.”
David Kuter, MD, Director of Clinical Hematology at Massachusetts General Hospital
Ongoing research aims to better address the multiple dimensions of ITP by targeting the underlying pathology of the disease.3,4
The following investigational agents aim to address the underlying pathology of ITP3,4:
Bruton’s tyrosine kinase (BTK) inhibitors,
which inhibit macrophage phagocytosis leading to a decrease in platelet destruction, prevent the production of autoantibodies, and may inhibit inflammation
%20blockers.svg 767w, /dam/jcr:eac53057-68a7-4ecb-ba19-215374a8bb0b/Neonatal%20Fc%20receptor%20(FcRn)%20blockers.svg 1200w)
Neonatal Fc receptor (FcRn) blockers,
which increase platelet antibody clearance, leading to a decrease in peripheral platelet destruction and immune response
Recombinant immunoglobulin multimers,
which decrease platelet destruction and increase antiplatelet antibody clearance
Complement inhibitors,
which decrease complement-dependent cytotoxicity
Plasma cell-depleting therapies,
which decrease antiplatelet antibodies
%20inhibitors.svg 767w, /dam/jcr:91c626b0-6e9c-437b-9e4e-4b50e45217ea/B%20cell%20activating%20factor%20(BAFF)%20inhibitors.svg 1200w)
B cell activating factor (BAFF) inhibitors,
which reduce B cell survival and consequently decrease antiplatelet antibodies
The clinical significance of these mechanisms is currently under investigation.
1. Cooper N, Ghanima W. Immune thrombocytopenia. N Engl J Med. 2019;381(10):945-955. 2. Bussel J, Arnold DM, Grossbard E, et al. Fostamatinib for the treatment of adult persistent and chronic immune thrombocytopenia: results of two phase 3, randomized, placebo-controlled trials. Am J Hematol. 2018;93(7):921-930. 3. Audia S, Bonnotte B. Emerging therapies in immune thrombocytopenia. J Clin Med. 2021;10(5):1004-1023. 4. Mingot-Castellano ME, Bastida JM, Caballero-Navarro G, et al. Novel therapies to address unmet needs in ITP. Pharmaceuticals (Basel). 2022;15(7):779.