Real-world Outcomes of Aplastic Anemia Patients Treated With Eltrombopag: A Medical Claims Database Study

NCT ID: NCT06991894

Last Updated: 2025-05-28

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 2517 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2021-07-14
• Study Completion Date: 2024-05-17

Brief Summary

The main goal of this study was to investigate the effectiveness and safety of eltrombopag (ETB) when compared to other treatments in Japanese aplastic anemia (AA) patients using data from the Medical Data Vision (MDV) hospital-based database.

Conditions

Anemia, Aplastic

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: RETROSPECTIVE

Study Groups

Anatomical Therapeutic Chemical (ATG)-free Cohort

Japanese patients with AA from the MDV database who received treatment with ciclosporin A (CSA) alone, ETB alone, or a combination of CSA and ETB.

No interventions assigned to this group

ATG-Inclusive Cohort

Japanese patients with AA from the MDV database who received one of the following treatment combinations: CSA and ATG or CSA and ETB and ATG.

No interventions assigned to this group

Eligibility Criteria

Inclusion Criteria

• Patients with:

• At least one confirmed diagnosis of AA registered during the baseline period, or
• At least one confirmed diagnosis of idiopathic thrombocytopenic purpura (International Classification of Diseases, 10th Revision [ICD10] code: D69.3) registered before the index date and at least one confirmed diagnosis of AA during the follow-up period.
• Had at least 6 months of continuous enrolment prior to the index date.

Confirmed diagnosis was defined as having ≥1 inpatient or ≥2 outpatient claims with a relevant ICD-10 code and without any doubtful flag.

Continuous enrolment (being continuously followed in the database) was defined as having at least one claim every semester.

• Had at least one confirmed diagnosis of AA registered before the index date,
• Had at least one procedure for any type of transfusion such as red blood cell transfusion, platelet transfusion, or granulocyte transfusion registered during the baseline period or within 4 weeks after the index date,
• Had at least 6 months of continuous enrolment prior to the index date,
• Had at least a 6-month follow-up period.

Exclusion Criteria

• Not receiving ATG, CSA, ETB or romiplostim (ROM) during the selection period,
• Had a diagnosis of acute myeloid leukemia (AML), chronic myelomonocytic leukemia (CMML), myelofibrosis, other hematological malignancies, or cataract before the index date.

• Not receiving ATG, CSA, ETB or ROM during the selection period,
• Had at least one prescription of ATG, CSA, ETB or ROM before the index date,
• Had a diagnosis of AML, CMML or other hematological malignancies before the index date.

• Minimum Eligible Age: 15 Years
• Maximum Eligible Age: 90 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Novartis Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

Novartis

East Hanover, New Jersey, United States

Countries

United States

Other Identifiers

CETB115EJP04

Identifier Type: -

Identifier Source: org_study_id