Real-world Outcomes of Aplastic Anemia Patients Treated With Eltrombopag: A Medical Claims Database Study
- Conditions
- Anemia, Aplastic
- Registration Number
- NCT06991894
- Lead Sponsor
- Novartis Pharmaceuticals
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 2517
Not provided
- 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.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Probability of Achieving Hematologic Response by AA Treatment Category 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 + ETBProbability of Achieving Hematologic Response by ETB Dose Density 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.Time to Achieve First Hematologic Response by AA Treatment Category 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 + ETBTime to Achieve First Hematologic Response by ETB Dose Density 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.Percentage of Patients who Achieved Hematologic Response by Treatment Category 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 + ETBCox Proportional Hazard Ratio for the Association Between Hematologic Response and Patient Characteristics 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 Outcome Measures
Name Time Method Number of Patients per Demographic Category Baseline Demographics included:
* Age category
* Gender
* BMI
* Most frequent comorbidities
* Most frequent medications receivedNeutrophil Level Baseline Number of Blood Transfusions Up to approximately 7 months Percentage of Patients With at Least One Transfusion of 400 Milliliters (mL) or More of red Blood Cells Up to approximately 8 weeks Percentage of Patients who Achieved Complete Response (CR) Month 6 CR was defined as hemoglobin \>100 grams per liter (g/L) and neutrophils \>1.0x10\^9/L and platelets \>100x10\^9/L.
Number of Patients who Received First Line Treatment by Type of Treatment Received Up to approximately 3 years Total Units of Blood Transfusions Up to approximately 7 months Total Amount of Blood and Plasma Transfused Up to approximately 7 months Platelets Count Baseline Percentage of Patients who Achieved Partial Response (PR) 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.
Percentage of Patients who Achieved Overall Response 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.
Probability of Achieving an Effectiveness Event by Treatment Category 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 + ETBProbability of Achieving an Effectiveness Event by ETB Dose Density 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.Time to Achieve an Effectiveness Event by Treatment Category 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 + ETBTime to Achieve an Effectiveness Event by ETB Dose Density 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.Number of Patients who Received Second Line Treatment by Type of Treatment Received Up to approximately 3 years Probability of Having a Safety Event by Treatment Category 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 + ETBTime to Having a Safety Event by Treatment Category 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 + ETBNumber of Patients who Received Third Line Treatment by Type of Treatment Received Up to approximately 3 years Hemoglobin Level Baseline
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Trial Locations
- Locations (1)
Novartis
🇺🇸East Hanover, New Jersey, United States
Novartis🇺🇸East Hanover, New Jersey, United States