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

NCT06991894 | Completed

• 1 other identifier: CETB115EJP04
• Study Type: observational
• Target: 2,517
• Locations: 1 country
• Sites: 1

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

Keywords

Eltrombopag; Effectiveness; Safety; Treatment patterns

Outcome Measures

Primary Outcomes (6)

• Probability of Achieving Hematologic Response by AA Treatment Category

  The Kaplan-Meier analysis technique was used to estimate probability. Hematologic response was defined as the first occurrence of an 8-week period without any transfusion procedure. Treatment categories included: * CSA alone * CSA + ETB * ETB alone * ATG + CSA * ATG + CSA + ETB

  Month 6, Years 1, 2, and up to approximately 3 years

• Probability of Achieving Hematologic Response by ETB Dose Density

  The Kaplan-Meier analysis technique was used to estimate probability. Hematologic response was defined as the first occurrence of an 8-week period without any transfusion procedure. ETB dose density was defined as the dosage of ETB divided by the duration to maximum dose: * Optimal dose-density: 62.5 milligrams (mg) or more mg/8 weeks, * Suboptimal dose-density: 25.1-62.4 mg/8 weeks, * Minimum dose: 25 mg or less/8weeks.

  Month 6, Years 1, 2, and up to approximately 3 years

• Time to Achieve First Hematologic Response by AA Treatment Category

  Time to hematologic response was defined as the number of days from the index date + 28 days to the first hematologic response. The index date was defined as the date of the first prescription of the following AA treatments: ATG, CSA, ETB or ROM. Event-free patients were censored at the earliest record of any of the following: death, end of the database registration, or end of the study. Treatment categories included: * CSA alone * CSA + ETB * ETB alone * ATG + CSA * ATG + CSA + ETB

  Up to approximately 3 years

• Time to Achieve First Hematologic Response by ETB Dose Density

  Time to hematologic response was defined as the number of days from the index date + 28 days to the first hematologic response. The index date was defined as the date of the first prescription of the following AA treatments: ATG, CSA, ETB or ROM. Event-free patients were censored at the earliest record of any of the following: death, end of the database registration, end of the study. ETB dose density was defined as the dosage of ETB divided by the duration to maximum dose: * Optimal dose-density: 62.5 mg or more mg/8 weeks, * Suboptimal dose-density: 25.1-62.4 mg/8 weeks, * Minimum dose: 25 mg or less/8weeks.

  Up to approximately 3 years

• Percentage of Patients who Achieved Hematologic Response by Treatment Category

  Hematologic response was defined as the first occurrence of an 8-week period without any transfusion procedure. Treatment categories included: * CSA alone * CSA + ETB * ETB alone * ATG + CSA * ATG + CSA + ETB

  Months 3 and 6

• Cox Proportional Hazard Ratio for the Association Between Hematologic Response and Patient Characteristics

  Patient characteristics included age, body mass index (BMI), treatment category (CSA+ETB vs CSA and ETB vs CSA), gender, comorbidity, medications received (antibiotics, anti-inflammatory drugs, anticonvulsants, antipsychotics), and bone marrow conditioning received before bone marrow transplant (BMT).

  Up to approximately 3 years

Secondary Outcomes (20)

• Number of Patients per Demographic Category

  Baseline

• Hemoglobin Level

  Baseline

• Platelets Count

  Baseline

• Neutrophil Level

  Baseline

• Number of Blood Transfusions

  Up to approximately 7 months

Study Arms (2)

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.

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.

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 not qualify if:

• 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.
• 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.
• 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.
• 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.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (1)

Novartis

East Hanover, New Jersey, 07936, United States

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: May 16, 2025
• First Posted: May 28, 2025
• Study Start: July 14, 2021
• Primary Completion: May 17, 2024
• Study Completion: May 17, 2024
• Last Updated: May 28, 2025

Record last verified: 2025-05

Locations

US (1)