Effectiveness of Early Intervention in Transfusion Independent Aplastic Anemia: a Retrospective Medical Claims Database Study

Novartis

Status: Completed

Conditions: Aplastic Anemia (AA)

Study type: Observational

Funder types: Industry

Identifiers

NCT06669221

CETB115EJP05

Details and patient eligibility

About

This was a retrospective non-interventional cohort study with secondary use of data from the Medical Data Vision (MDV) hospital-based database to evaluate the effectiveness of early drug intervention in preventing transfusion compared to watchful observation among adult transfusion-independent aplastic anemia (AA) patients in Japan.

Enrollment: 1,603 patients

Sex: All

Ages: 15 to 90 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Having at least one confirmed diagnosis of AA within the selection period (1 inpatient or 2 outpatient claims per the International Statistical Classification of Diseases, 10th Revision [ICD-10] code, without any suspicion flag).
• Being aged 15 to 90 years at the index date.
• Having at least 3 months of continuous enrolment prior to the index date.

Exclusion criteria

• Having a blood transfusion recorded any time before the index date.
• Having anti-thymocyte globulin (ATG) treatment recorded within 3 months after the index date.
• Having at least one prescription record for any of the drug treatments of interest any time before the index date.
• Having a diagnosis of acute myeloid leukemia (AML), chronic myelomonocytic leukemia (CMML) or other leukemia any time before the index date.
• Having an AA diagnosis (without suspicious flag) date earlier than the start of patient's observation in the database.
• Having less than 6 months of continuous follow-up.

Safety Population - Additional Exclusion Criterion:

• Having a diagnosis of hepatotoxicity, kidney dysfunction, hypertension, or diabetes mellitus any time before the index date.

Trial design

1,603 participants in 2 patient groups

Early drug intervention group

Description:

Patients who had at least one prescription record associated with any of the drug treatments of interest (cyclosporine A, eltrombopag, romiplostin, danazol, or methenolone) during each period of interest before the first transfusion record during the follow-up period.

Watchful observation group

Description:

Patients with no prescription record associated with any of the drug treatments of interest (cyclosporine A, eltrombopag, romiplostin, danazol, or methenolone) during the follow-up period and before the first transfusion record, if any.