David J. Kuter, MD, DPhil, discusses the latest results regarding immune thrombocytopenia (ITP) presented at ASH 2024.

 


 

ITP is an autoimmune bleeding disorder characterized by too few platelets in the blood. Symptoms may include bruising, nosebleed or bleeding in the mouth, bleeding into the skin, and abnormally heavy menstruation. Rarely, ITP may become a chronic ailment in adults and reappear, even after remission.

Dr. Kuter provided an overview of clinically relevant data about ITP presented at the ASH 2024 meeting.

LUNA 3 Clinical Trial

LUNA 3 is phase 3, placebo-controlled, parallel-group, multicenter study evaluating the safety and efficacy of rilzabrutinib in patients with ITP. Rilzabrutinib is an investigational, oral, reversible, covalent BTK inhibitor. BTK plays an important role in inflammatory pathways and immune-mediated disease processes. The primary endpoint of the study was durable platelet response.

Platelet response was achieved in 65%  of rilzabrutinib patients versus 33% in placebo patients. The primary endpoint was also met with 23% of rilzabrutinib patients, with zero patients in the placebo group meeting the endpoint. Additionally, the primary endpoint was met in 29% of rilzabrutinib-randomized patients in the double-blind and open-label periods. 

Statistically significant improvements were also observed in all secondary endpoints with rilzabrutinib over placebo. These include median time to first platelet count, reduction in the need for rescue therapy,  and improvements in physical fatigue, bleeding, and quality of life. Favorable safety profiles and tolerability were also illustrated with rilzabrutinib.

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Clinical Burden of Illness Study

The aim of this study was to evaluate the clinical burden of disease among chronic ITP patients who are treated with advanced therapies versus the non-ITP population. Clinical events including bleeding, infections, and TEs, were measured in the follow up of 1140 patients with ITP and compared with 5657 non-ITP participants.

At baseline, the ITP subgroup had higher prevalence of solid tumors, infections, and TEs than the non-ITP subgroup. They also had higher rates of mental health issues, cardiovascular risk factors, and oral steroid use.

During average follow-up of about two years, the ITP subgroup experienced more bleed-related hospitalization (15.2% vs 4.5% non-ITP) and TEs (20.3% vs 9.1% non-ITP). Oral steroid use remained higher in the ITP subgroup as well (41% vs 14% non-ITP). Additionally the ITP cohort had higher incidences of developing new malignancies, cognitive impairment, autoimmune conditions, and infections. Death was also more prevalent at 21% for the ITP subgroup and 10% for the non-ITP subgroup.

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To learn more about ITP and other rare hematologic conditions, visit https://checkrare.com/diseases/hematologic-disorders/