A Study of the Safety and Effectiveness of Eltrombopag in Combination With Immunosuppressive Therapy (IST) in Korean Pediatric Patients With Treatment Naïve Severe Aplastic Anemia

Retrospective Chart Review of Safety and Effectiveness of Eltrombopag in Combination With Immunosuppressive Therapy (IST) in Korean Pediatric Patients With Treatment Naïve Severe Aplastic Anemia

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT07415083

Enrollment

Registered

2026-02-17

Start date

2023-06-21

Completion date

2025-02-25

Last updated

2026-02-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Aplastic Anemia

Keywords

Severe Aplastic Anemia, Eltrombopag, Retrospective chart review, Safety

Brief summary

The aim of this study was to investigate the safety, tolerability, and effectiveness of eltrombopag in combination with immunosuppressive therapy (IST) in real world practice in pediatric patients with severe aplastic anemia (sAA) in Korea. The study used data over a 6-month period extracted from patients' medical charts (electronic medical records) starting from the initiation of eltrombopag treatment.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

RETROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

2 Years to 17 Years

Healthy volunteers

Inclusion criteria

1. Male or female pediatric patients aged ≥2 years and \<18 years at the time of initiating eltrombopag and IST treatment. 2. Subjects diagnosed with sAA and treated with eltrombopag in combination with IST as first-line therapy, based on the physician's decision in accordance with locally approved prescribing information at the time of data collection. 3. Subjects whose analytical index date (start date of eltrombopag administration) was at least 6 months prior to the data extraction date.

Exclusion criteria

None.

Design outcomes

Primary

Measure	Time frame	Description
Incidence of Adverse Events (AEs) and Serious Adverse Events (SAEs)	Up to 6 months	Incidence of all AEs/adverse drug reactions (ADRs), SAEs/serious adverse drug reactions (SADRs), and unexpected AEs/ADRs.

Secondary

Measure	Time frame	Description
Overall Response Rate (ORR)	6 months	ORR includes complete response (CR) and partial response (PR). CR was defined as meeting all three of the following criteria: * Absolute neutrophil count (ANC) \> 1.0 × 10\^9/L * Platelet count \> 100 × 10\^9/L * Hemoglobin \> 100 g/L PR was defined as hematologic improvement that did not meet the criteria for CR, but also did not meet the criteria for sAA.

Countries

United States

Contacts

STUDY_DIRECTORNovartis Pharmaceuticals

Novartis Pharmaceuticals

Outcome results