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APLASTIC ANEMIA | Hetrombopag Achieves Significant Hematological Responses in Newly Diagnosed Patients

A recent study published in the Annals of Hematology found that adult patients with newly diagnosed transfusion-dependent non-severe aplastic anemia (TD-NSAA) experienced significantly higher hematologic response rates when treated with a combination of cyclosporin A (CsA) and hetrombopag, compared to CsA alone.

In this study, newly diagnosed patients with transfusion-dependent non-severe aplastic anemia (TD-NSAA) were initially treated with either a combination of cyclosporin A (CsA) and hetrombopag (HETROM+CsA group) or CsA alone (CsA control group). CsA was administered orally at a dose of 3 to 5 mg/kg/day, divided into two daily doses and adjusted based on plasma concentration.

CsA treatment was tapered after achieving optimal hematologic response and continued for at least 12 months. Hetrombopag was started at a dose of 10 mg/day, with the potential to increase to 15 mg/day, and similarly tapered after reaching optimal response. The study matched patients in each group based on baseline age, sex, and complete blood cell count to ensure comparability.

Each treatment group consisted of 28 patients. The median age was 45 years in the HETROM+CsA group and 43 years in the CsA control group, with median follow-up times of 15 and 16 months, respectively.

After 3 months of therapy, the HETROM+CsA group demonstrated a significantly higher response rate (57.1%) compared to the CsA control group (17.9%). At the 6-month mark, the response rate remained significantly higher for the HETROM+CsA group (57.1%) compared to the CsA control group (32.1%). By the last follow-up, response rates were 92.9% for the HETROM+CsA group and 50.0% for the CsA control group.

In the HETROM+CsA group, the most common adverse events included hair and gingival hyperplasia (21.4%), increased alanine aminotransferase levels (21.4%), gastrointestinal disorders (17.9%), and headaches (10.7%). One patient (3.6%) experienced Grade 3 hematochezia and hematuria, necessitating a dose reduction.

“Our study found that combining CsA with hetrombopag resulted in significantly higher overall response rates at 3, 6, and 12 months for newly diagnosed TD-NSAA compared to CsA alone, with manageable adverse events and a lower relapse rate,” the authors reported.

Source: Annals of Hematology 

 

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