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

Novartis

Status: Completed

Conditions: Anemia, Aplastic

Study type: Observational

Funder types: Industry

Identifiers

NCT06991894

CETB115EJP04

Details and patient eligibility

About

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.

Enrollment: 2,517 patients

Sex: All

Ages: 15 to 90 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion and exclusion criteria for the safety population:

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.

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.

Inclusion and exclusion criteria for the effectiveness population:

Inclusion criteria:

• 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 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.

Trial design

2,517 participants in 2 patient groups

Anatomical Therapeutic Chemical (ATG)-free Cohort

Description:

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.

ATG-Inclusive Cohort

Description:

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.