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TOPLINE:

Eltrombopag combined with immunosuppressive therapy in pediatric patients with severe aplastic anemia achieved a 54.9% overall response rate at 26 weeks, with a 71.4% response in relapsed/refractory patients and 48.6% in treatment-naive patients. Among transfusion-dependent patients, 66.7% and 76.7% achieved red blood cell and platelet transfusion independence, respectively.

METHODOLOGY:

• A phase 2, open-label, noncontrolled, interpatient dose-escalation study evaluated eltrombopag combined with immunosuppressive therapy in pediatric patients aged 1 to < 18 years with relapsed/refractory or treatment-naive severe aplastic anemia.
• Initial eltrombopag dosing started at 25 mg/d for patients aged 1 to < 6 years and at 50 mg/d for patients aged 6 to < 18 years, with allowed dose modifications up to 150 mg/d to achieve target platelet counts of 50 × 10⁹/L to 200 × 10⁹/L.
• Treatment protocol included eltrombopag administration for 26 weeks with possible extension if clinically beneficial, alongside cyclosporine A, with or without horse antithymocyte globulin.
• A total of 51 patients were enrolled and treated, comprising 14 patients with relapsed/refractory severe aplastic anemia and 37 treatment-naive patients.

TAKEAWAY:

• Overall response rate at 26 weeks reached 54.9% across both cohorts, with 71.4% in the relapsed/refractory cohort and 48.6% in the treatment-naive cohort, with most responders maintaining responses after discontinuing eltrombopag.
• Among baseline transfusion-dependent patients, 66.7% achieved red blood cell transfusion independence and 76.7% achieved platelet transfusion independence, with rates of 70% and 80% for the relapsed/refractory cohort and 65.6% and 75.8% for the treatment-naive cohort, respectively.
• The most common treatment-related adverse events included increased bilirubin (43.1%), increased alanine aminotransferase (37.3%), and increased aspartate aminotransferase (33.3%).
• Researchers noted that eltrombopag with immunosuppressive therapy showed a trend toward a favorable overall response rate in the relapsed/refractory cohort, with no new safety signals identified.

IN PRACTICE:

“Recent trials show that adding eltrombopag to standard IST [immunosuppressive therapy] improves the hematologic response in untreated adult patients with SAA [severe aplastic anemia], without impacting safety,” the authors of the study wrote.

SOURCE:

This study was led by Akiko Shimamura, MD, PhD, Division of Hematology/Oncology, Boston Children’s Hospital, Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, Harvard Medical School in Boston. It was published online in Blood Advances.

LIMITATIONS:

According to the authors, the single-arm phase 2 study design without a control arm inherently limited the research. The small sample size restricted statistical analysis of treatment efficacy. The relapsed/refractory cohort included only 14 patients, with unknown prior treatments, and some patients did not receive horse antithymocyte globulin, warranting cautious interpretation. The absence of data on prior treatments for the relapsed/refractory cohort made it difficult to account for potential influences of previous therapies on eltrombopag outcomes. The high patient attrition rate posed a significant limitation, and comparing response rates with previously published studies was challenging due to variations in study designs, efficacy assessment criteria, and sample sizes.

DISCLOSURES:

This study was funded by Novartis Pharma AG in Basel, Switzerland. Carolyn Bennett, PhD, disclosed having ties with Novartis and Sobi. Sujith Samarasinghe, MD, reported having ties with Pfizer. Relationships with Novartis were reported by Jason E. Farrar, MD, Jennifer A. Rothman, MD, Taizo A. Nakano, MD, Shimamura, Winfred Wang, MD, and Adrianna Vliachos, MD. Tze Fang Wong, PhD, Qinxia Wang, PhD, and Patrick Urban, PhD, reported being employees of Novartis. Brigitte Strahm, MD, disclosed having ties with Novartis and Pfizer. David A. Williams, MD, reported having relationships with Beam Therapeutics, Skyline Therapeutics, Tessera Therapeutics, Verve Therapeutics, Vertex Pharmaceuticals, and ExCellThera.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.