Startseite Kongressberichte 2020 Virtual Edition of the 25th Annual Congress of the European Hematology Association Oral Sessions ITP:

ITP: What's new in 2020

 
Francesca Basso-Valentina, Alessandro Donada, Manuel Lisetto, et al.
 
The authors of the abstract conclude: 
Using different cellular models we demonstrate here for the first time a crucial role of ANKRD26 as a new regulator of signaling for the entire class-I cytokine receptor family. Due to abnormal ANKRD26 expression levels detected in THC2 patients, we demonstrated that in the early stages of erythroid, megakaryocytic and granulocytic differentiation, the ANKRD26 is necessary to preserve a strong cytokine/receptor type-I signaling in order to ensure a robust proliferation. Conversely, this signal should be progressively silenced during differentiation, in order to shut-down signaling and achieve a correct final maturation for all the three hematopoietic myeloid lineages. 
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David Kuter, Ralph V. Boccia, Eun-Ju Lee, et al.
 
The authors of the abstract conclude: 
Rilzabrutinib was active at all dose levels, despite a short follow-up period, and with substantial improvements with longer treatment. Rilzabrutinib was well tolerated, whether given as a monotherapy or with allowed concomitant ITP therapy. The dose-escalation portion of the study showed rapid, durable clinical activity and tolerable safety in patients refractory to multiple treatments and who had no alternative therapeutic options. Enrollment is expanding at the optimal 400 mg BID rilzabrutinib starting dose to further characterize the magnitude and durability of clinical benefit. Results may be updated prior to presentation at the meeting.
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Shaoqiu Leng, Jun Peng.
 
The authors of the abstract conclude: 
This study demonstrated that GR-associated MDSCs impairments are involved in the pathogenesis of ITP. It might provide a new target to treat primary thrombocytopenia  associated with GR-dependent mitochondrial respiration and metabolism.
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Andrea Fan, Janet Anderl, Brian Tuch, et al.
 
The authors of the abstract conclude: 
KZR-616 is a novel therapy for autoimmune diseases including AIHA and ITP.  Biomarker data from SLE patients treated with KZR-616 suggest positive effects on erythropoiesis.  We are applying similar biomarker analyses to an ongoing phase II study of KZR-616 in AIHA and ITP, the MARINA trial, where clinical samples will be evaluated for pharmacodynamics, changes in gene expression profiles, and immune effector cell phenotypes. 
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Vincent Jachiet, Guillaume Moulis, Delphine Gobert, et al.
 
The authors of the abstract conclude: 
MDS/CMML-associated ITP have particular outcome with more severe bleeding and multirefractory profile than primary ITP, less progression toward acute myeloid leukemia than MDS/CMML without ITP, and similar response profile to primary ITP therapy.