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Completed

NCT Number: NCT06991894

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

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.

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Key information

Conditions

Anemia, Aplastic Anemia Bone Marrow Diseases Bone Marrow Failure Disorders Hematologic Diseases Hemic and Lymphatic Diseases

Age range

15 year–90 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Novartis

East Hanover, New Jersey, 07936, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

  1. Probability of Achieving Hematologic Response by AA Treatment Category

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

    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

  2. Probability of Achieving Hematologic Response by ETB Dose Density

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

    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.

  3. Time to Achieve First Hematologic Response by AA Treatment Category

    Time frame: Up to approximately 3 years

    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

  4. Time to Achieve First Hematologic Response by ETB Dose Density

    Time frame: Up to approximately 3 years

    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.

  5. Percentage of Patients who Achieved Hematologic Response by Treatment Category

    Time frame: Months 3 and 6

    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

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

    Time frame: Up to approximately 3 years

    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).

Secondary outcomes

  1. Number of Patients per Demographic Category

    Time frame: Baseline

    Demographics included:

    • Age category
    • Gender
    • BMI
    • Most frequent comorbidities
    • Most frequent medications received

  2. Hemoglobin Level

    Time frame: Baseline

  3. Platelets Count

    Time frame: Baseline

  4. Neutrophil Level

    Time frame: Baseline

  5. Number of Blood Transfusions

    Time frame: Up to approximately 7 months

  6. Total Units of Blood Transfusions

    Time frame: Up to approximately 7 months

  7. Total Amount of Blood and Plasma Transfused

    Time frame: Up to approximately 7 months

  8. Percentage of Patients With at Least One Transfusion of 400 Milliliters (mL) or More of red Blood Cells

    Time frame: Up to approximately 8 weeks

  9. Percentage of Patients who Achieved Complete Response (CR)

    Time frame: Month 6

    CR was defined as hemoglobin >100 grams per liter (g/L) and neutrophils >1.0x10^9/L and platelets >100x10^9/L.

  10. Percentage of Patients who Achieved Partial Response (PR)

    Time frame: Month 6

    PR was defined as no longer meeting severe AA criteria, transfusion independence, hemoglobin >8 grams per deciliter (gr/dL) and neutrophils >0.5x10^9/L and platelets >20x10^9/L.

  11. Percentage of Patients who Achieved Overall Response

    Time frame: Month 6

    Overall response was defined as having CR or PR. CR was defined as hemoglobin >100 g/L and neutrophils >1.0x10^9/L and platelets >100x10^9/L. PR was defined as no longer meeting severe AA criteria, transfusion independence, hemoglobin >8 gr/dL and neutrophils >0.5x10^9/L and platelets >20x10^9/L.

  12. Probability of Achieving an Effectiveness Event by Treatment Category

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

    The Kaplan-Meier analysis technique was used to estimate probability. Effectiveness events included death, treatment discontinuation, relapse, BMT, bleeding, bleeding requiring transfusion, bleeding requiring a treatment, and infection. Treatment categories included:

    • CSA alone
    • CSA + ETB
    • ETB alone
    • ATG + CSA
    • ATG + CSA + ETB

  13. Probability of Achieving an Effectiveness Event by ETB Dose Density

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

    The Kaplan-Meier analysis technique was used to estimate probability. Effectiveness events included death, treatment discontinuation, relapse, BMT, bleeding, bleeding requiring transfusion, bleeding requiring a treatment, and infection. 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.

  14. Time to Achieve an Effectiveness Event by Treatment Category

    Time frame: Up to approximately 3 years

    Time to an effectiveness event was defined as the number of days from the index date + 28 days until the earliest record of an event of interest. 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 follow-up, or end of the study.

    Effectiveness events included death, treatment discontinuation, relapse, BMT, bleeding, bleeding requiring transfusion, bleeding requiring a treatment, and infection. Treatment categories included:

    • CSA alone
    • CSA + ETB
    • ETB alone
    • ATG + CSA
    • ATG + CSA + ETB

  15. Time to Achieve an Effectiveness Event by ETB Dose Density

    Time frame: Up to approximately 3 years

    Time to an effectiveness event was defined as the number of days from the index date + 28 days until the earliest record of an event of interest. 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 follow-up, or end of the study.

    Effectiveness events included death, treatment discontinuation, relapse, BMT, bleeding, bleeding requiring transfusion, bleeding requiring a treatment, and infection. 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.

  16. Number of Patients who Received First Line Treatment by Type of Treatment Received

    Time frame: Up to approximately 3 years

  17. Number of Patients who Received Second Line Treatment by Type of Treatment Received

    Time frame: Up to approximately 3 years

  18. Number of Patients who Received Third Line Treatment by Type of Treatment Received

    Time frame: Up to approximately 3 years

  19. Probability of Having a Safety Event by Treatment Category

    Time frame: Month 6, and Years 1, 2, and 5

    The Kaplan-Meier analysis technique was used to estimate probability. Safety events included diabetes, hepatotoxicity event, hypertension event, thromboembolic event, transformation to myelodysplastic syndrome (MDS), AML, CMML or other leukemia, and transformation to paroxysmal nocturnal hemoglobinuria (PNH).

    Treatment categories included:

    • CSA alone
    • CSA + ETB
    • ETB alone
    • ATG + CSA
    • ATG + CSA + ETB

  20. Time to Having a Safety Event by Treatment Category

    Time frame: Up to approximately 9 years

    Time to each safety event was defined as the number of days from the index date until the earliest record of the event of interest. 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 follow-up, or end of the study.

    Safety events included diabetes, hepatotoxicity event, hypertension event, thromboembolic event, transformation to MDS, AML, CMML or other leukemia, and transformation to PNH.

    Treatment categories included:

    • CSA alone
    • CSA + ETB
    • ETB alone
    • ATG + CSA
    • ATG + CSA + ETB

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Official study title

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

Important dates

Study start
2021
Primary completion
2024
Study completion
2024
First posted
May 28, 2025
Registry last updated
May 28, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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