The 62nd American Society of Hematology (ASH) Annual Meeting and Exposition will be held virtually this year from December 5 through 8. The goal of the meeting is to provide insight into hematology research and other important topics in this sphere. Recently, precision therapy company Blueprint Medicines Corporation (“Blueprint”) announced that the company would present a number of oral and poster presentations during the meeting. The presentations will relate to AYVAKIT (avapritinib) and its impact on patients with systemic mastocytosis. Learn more about the ASH Annual Meeting, and the accepted abstracts, here.
AYVAKIT Presentations
Currently, AYVAKIT is indicated for use in adult patients with gastrointestinal stromal tumors (GIST) with specific PDGFRA exon 18 mutations. This kinase inhibitor targets D816V mutant KIT, which specifically spurs SM progression. Although AYVAKIT received Breakthrough Therapy designation for the treatment of multiple SM subtypes, it is not yet approved for use in this patient population in the United States or Europe.
During the ASH Annual Meeting, Blueprint will share the following presentations:
- Abstract #345: In this oral presentation, researchers will discuss data from the Phase 1 EXPLORER trial. Data will show how patients with SM, who received AYVAKIT, experienced better overall survival (OS) rates.
- Abstract #1248: This poster presentation will discuss data from the Phase 2 PIONEER trial, which evaluated AYVAKIT in patients with indolent SM. In this trial, researchers compared results from AYVAKIT as compared to a placebo.
- Abstract #1638: During this poster presentation, Blueprint researchers will discuss the TouchStone Survey. The survey allowed patients with SM to self-report their symptoms and outcomes during routine care.
- Abstract #2988: Ultimately, this presentation builds upon that in #1248. Researchers continue to discuss their insights into the Phase 2 PIONEER trial. During this poster session, researchers will discuss the safety, efficacy, and tolerability of AYVAKIT.
- Abstract #3004: Within this poster presentation, researchers discuss some of their detection methods within the PIONEER trial. For example, researchers used a High Sensitivity Droplet Digital PCR Assay to identify specific mutations in blood samples sourced from patients. During the trial, researchers also compared this identification and detection method with that of next generation sequencing (NGS) to discover the most effective option.
- Abstract #3403: In the final poster presentations, researchers continue sharing TouchStone results. This presentation delves deeper into how patients view burden of care and healthcare providers.
Systemic Mastocytosis (SM)
KIT gene mutations typically cause systemic mastocytosis (SM), a disorder characterized by excess mast cells. Normally, mast cells play a role in disease protection and wound healing. However, excess mass cells can overwhelm the body and cause detrimental physical effects. Symptoms, which are often triggered by something such as alcohol or temperature changes, include:
- Abdominal pain
- Nausea
- Diarrhea
- Pruritis (itchy skin)
- Lightheadedness or dizziness
- Flushed face
- Loss of consciousness
- Anemia
- Changes in mood
- Depression
- Difficulty concentrating
Learn more about systemic mastocytosis.
