A Novel Anti-CD38 Monoclonal Antibody for Treating Immune Thrombocytopenia
This is a promising new agent for ITP, where the median time of 50K platelets is one week. This is a small study though, awaiting more data in the future.
We value your privacy
We use cookies to enhance your browsing experience, serve personalized ads or content, and analyze our traffic. By clicking "Accept All", you consent to our use of cookies.
We use cookies to help you navigate efficiently and perform certain functions. You will find detailed information about all cookies under each consent category below.
The cookies that are categorized as "Necessary" are stored on your browser as they are essential for enabling the basic functionalities of the site. ...
Necessary cookies are required to enable the basic features of this site, such as providing secure log-in or adjusting your consent preferences. These cookies do not store any personally identifiable data.
No cookies to display.
Functional cookies help perform certain functionalities like sharing the content of the website on social media platforms, collecting feedback, and other third-party features.
No cookies to display.
Analytical cookies are used to understand how visitors interact with the website. These cookies help provide information on metrics such as the number of visitors, bounce rate, traffic source, etc.
No cookies to display.
Performance cookies are used to understand and analyze the key performance indexes of the website which helps in delivering a better user experience for the visitors.
No cookies to display.
Advertisement cookies are used to provide visitors with customized advertisements based on the pages you visited previously and to analyze the effectiveness of the ad campaigns.
No cookies to display.
This great Italian study uses eltrombopag in patients with low/intermediate risk MDS. The intervention arm had significantly fewer minor bleeding events and better platelet counts that were durable. Those with higher baseline Hb’s seemed to respond better to therapy, particularly if the Hb was >8g/dL. AML evolution/blast progression was not more common in the treatment arm, which refutes some prior concerns.
In myelodysplastic syndromes (MDS), severe thrombocytopenia is associated with poor prognosis. This multicenter trial presents the second-part long-term efficacy and safety results of eltrombopag in patients with low-risk MDS and severe thrombocytopenia.
In this single-blind, randomized, placebo-controlled, phase-II trial of adult patients with International Prognostic Scoring System low- or intermediate-1-risk MDS, patients with a stable platelet (PLT) count (<30 × 103/mm3) received eltrombopag or placebo until disease progression. Primary end points were duration of PLT response (PLT-R; calculated from the time of PLT-R to date of loss of PLT-R, defined as bleeding/PLT count <30 × 103/mm3 or last date in observation) and long-term safety and tolerability. Secondary end points included incidence and severity of bleeding, PLT transfusions, quality of life, leukemia-free survival, progression-free survival, overall survival and pharmacokinetics.
From 2011 to 2021, of 325 patients screened, 169 patients were randomly assigned oral eltrombopag (N = 112) or placebo (N = 57) at a starting dose of 50 mg once daily to maximum of 300 mg. PLT-R, with 25-week follow-up (IQR, 14-68) occurred in 47/111 (42.3%) eltrombopag patients versus 6/54 (11.1%) in placebo (odds ratio, 5.9; 95% CI, 2.3 to 14.9; P < .001). In eltrombopag patients, 12/47 (25.5%) lost the PLT-R, with cumulative thrombocytopenia relapse-free survival at 60 months of 63.6% (95% CI, 46.0 to 81.2). Clinically significant bleeding (WHO bleeding score ≥ 2) occurred less frequently in the eltrombopag arm than in the placebo group (incidence rate ratio, 0.54; 95% CI, 0.38 to 0.75; P = .0002). Although no difference in the frequency of grade 1-2 adverse events (AEs) was observed, a higher proportion of eltrombopag patients experienced grade 3-4 AEs (χ2 = 9.5, P = .002). AML evolution and/or disease progression occurred in 17% (for both) of eltrombopag and placebo patients with no difference in survival times.
Eltrombopag was effective and relatively safe in low-risk MDS with severe thrombocytopenia. This trial is registered with ClinicalTrials.gov identifier: NCT02912208 and EU Clinical Trials Register: EudraCT No. 2010-022890-33.
This is a promising new agent for ITP, where the median time of 50K platelets is one week. This is a small study though, awaiting more data in the future.
Benign heme article of the month– A significant portion of patients with stable platelet counts can be gently tapered from TPO-agonists. Those who needed to be re-challenged had a good response. This is likely an underutilized strategy.
Thought provoking article regarding a non-antibody mediated mechanism in chronic ITP.
New criteria for response to therapy in high grade MDS, focuses on pt centered outcomes.
Interesting prospective study assessing outcomes in ITP in pregnant women while measuring neonatal ITP in the developing child, with a control of non-pregnant women for comparison. Pregnancy-ITP was associated with a 2.7x higher rate of recurrent disease. However, bleeding was similar in pregnant vs. non-pregnant women. NITP risk was associated with more severe ITP disease in the mother and the severity of the disease, as well as prior h/o ITP in the mother.
FCS Hematology Oncology Review creates a platform for our physician network to observe the most recent articles and studies available in the oncology and hematology world. By sharing these articles we are building our wealth of knowledge of new observations and treatments as they come available.