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

February 16, 2026 updated by: Novartis Pharmaceuticals

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

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.

Study Overview

Status

Completed

Conditions

Study Type

Observational

Enrollment (Actual)

13

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • New Jersey
      • East Hanover, New Jersey, United States, 07963
        • Novartis

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Pediatric patients with sAA who initiated eltrombopag and IST treatment in Korea.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Eltrombopag and IST Treatment Cohort
Pediatric patients with sAA who received first-line eltrombopag in combination with IST.

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Overall Response Rate (ORR)
Time Frame: 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.
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis Pharmaceuticals

Investigators

  • Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 21, 2023

Primary Completion (Actual)

February 25, 2025

Study Completion (Actual)

February 25, 2025

Study Registration Dates

First Submitted

February 11, 2026

First Submitted That Met QC Criteria

February 11, 2026

First Posted (Actual)

February 17, 2026

Study Record Updates

Last Update Posted (Actual)

February 19, 2026

Last Update Submitted That Met QC Criteria

February 16, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CETB115GKR02

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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