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

NCT06669221 | Completed

• 1 other identifier: CETB115EJP05
• Study Type: observational
• Target: 1,603
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Aplastic Anemia (AA)

Outcome Measures

Primary Outcomes (3)

• Probability of Having Blood Transfusion in the Matched Effectiveness Population

  The Kaplan-Meier survival analysis technique was used to estimate probability.

  Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years

• Crude Hazard Ratios of Having Blood Transfusion After 6 Months of Follow-up in the Matched Effectiveness Population

  Baseline, 6 months

• Adjusted Hazard Ratios of Having Blood Transfusion After 6 Months of Follow-up in the Matched Effectiveness Population

  6 months

Secondary Outcomes (44)

• Sex

  Baseline

• Age

  Baseline

• Weight

  Baseline

• Body Mass Index (BMI)

  Baseline

• Number of Patients With Aplastic Anemia, per Severity Level

  Baseline

Study Arms (2)

Early drug intervention group

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

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.

Eligibility Criteria

• Age: 15 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

This was a retrospective, noninterventional cohort study.

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (1)

Novartis

Tokyo, 105-6333, Japan

Location

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 30, 2024
• First Posted: November 1, 2024
• Study Start: May 5, 2022
• Primary Completion: November 3, 2023
• Study Completion: November 3, 2023
• Last Updated: November 1, 2024

Record last verified: 2024-10

Locations

JP (1)