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

Novartis Pharmaceuticals2,517 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

2,517

Conditions

Anemia, Aplastic

Eligibility

Sex: ALLMin age: 15 YearsMax age: 90 Years

Medical Language ↔ Plain English

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.

Locations (1)

Novartis

East Hanover, New Jersey, United States

Outcomes

Primary Outcomes

Probability of Achieving Hematologic Response by AA Treatment Category

Time frame: 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.

Time frame: 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

Time frame: 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.

Data from ClinicalTrials.gov

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Secondary Outcomes

Number of Patients per Demographic Category

Demographics included: * Age category * Gender * BMI * Most frequent comorbidities * Most frequent medications received

Time frame: Baseline

Hemoglobin Level

Time frame: Baseline

Platelets Count

Time frame: Baseline

Neutrophil Level

Time frame: Baseline

Number of Blood Transfusions

Time frame: Up to approximately 7 months

Total Units of Blood Transfusions

Time frame: Up to approximately 7 months

Total Amount of Blood and Plasma Transfused

Time frame: Up to approximately 7 months

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

Percentage of Patients who Achieved Complete Response (CR)

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

Time frame: Month 6

Percentage of Patients who Achieved Partial Response (PR)

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.

Time frame: Month 6

Percentage of Patients who Achieved Overall Response

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.

Time frame: Month 6

Probability of Achieving an Effectiveness Event by Treatment Category

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

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

Probability of Achieving an Effectiveness Event by ETB Dose Density

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.

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

Time to Achieve an Effectiveness Event by Treatment Category

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

Time frame: Up to approximately 3 years

Time to Achieve an Effectiveness Event by ETB Dose Density

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.

Time frame: Up to approximately 3 years

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

Time frame: Up to approximately 3 years

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

Time frame: Up to approximately 3 years

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

Time frame: Up to approximately 3 years

Probability of Having a Safety Event by Treatment Category

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

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

Time to Having a Safety Event by Treatment Category

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

Time frame: Up to approximately 9 years