Anemia, AplasticNCT06991894
Real-world Outcomes of Aplastic Anemia Patients Treated With Eltrombopag: A Medical Claims Database Study
An observational study of Anemia, Aplastic, run by Novartis Pharmaceuticals at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 15 to 90 years
- Conditions the study is for
- Anemia, Aplastic
- How many people
- 2,517 participants
- Where
- 1 siteUnited States
- Study dates
- 14 Jul 2021to 17 May 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 15 to 90 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: This was a retrospective, noninterventional cohort study.
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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:
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Real-world Outcomes of Aplastic Anemia Patients Treated With Eltrombopag: A Retrospective Medical Claims Database Study (Eltrombopag for Aplastic Anemia)
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
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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.
Measured: 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
Measured: 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.
Measured: 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
Measured: 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).
Measured: Up to approximately 3 years
Secondary outcomes (20)
Number of Patients per Demographic Category
Demographics included:
- Age category
- Gender
- BMI
- Most frequent comorbidities
- Most frequent medications received
Measured: Baseline
Hemoglobin Level
Measured: Baseline
Platelets Count
Measured: Baseline
Neutrophil Level
Measured: Baseline
Number of Blood Transfusions
Measured: Up to approximately 7 months
Total Units of Blood Transfusions
Measured: Up to approximately 7 months
Total Amount of Blood and Plasma Transfused
Measured: Up to approximately 7 months
Percentage of Patients With at Least One Transfusion of 400 Milliliters (mL) or More of red Blood Cells
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: 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.
Measured: 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
Measured: 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.
Measured: Up to approximately 3 years
Number of Patients who Received First Line Treatment by Type of Treatment Received
Measured: Up to approximately 3 years
Number of Patients who Received Second Line Treatment by Type of Treatment Received
Measured: Up to approximately 3 years
Number of Patients who Received Third Line Treatment by Type of Treatment Received
Measured: 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
Measured: 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
Measured: Up to approximately 9 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 May 2025
- Study start14 Jul 2021
- Primary completion17 May 2024
- Study completion17 May 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Anemia, AplasticStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 2,517 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06991894
- Sponsor's protocol ID
- CETB115EJP04
- Status last verified
- May 2025
- First submitted
- 16 May 2025
- Last updated
- 28 May 2025
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Real-world Outcomes of Aplastic Anemia Patients Treated With Eltrombopag: A Retrospective Medical Claims Database Study (Eltrombopag for Aplastic Anemia)
- ClinicalTrials.gov ID
- NCT06991894
- Acronym / sponsor ID
- CETB115EJP04
- Conditions
- Anemia, Aplastic
- Keywords
- Eltrombopag, Effectiveness, Safety, Treatment patterns
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 2,517 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 14 Jul 2021 (actual)
- Primary completion
- 17 May 2024 (actual)
- Study completion
- 17 May 2024 (actual)
- First posted
- 28 May 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- 15 to 90 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- This was a retrospective, noninterventional cohort study.
Key inclusion criteria (10 listed)
- Patients with:
- 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.
- Had at least one confirmed diagnosis of AA registered before the index date,
Key exclusion criteria (6 listed)
- 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.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
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.
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
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.
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
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).
20 secondary outcomes
Number of Patients per Demographic Category
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)
Time frame: Month 6
Percentage of Patients who Achieved Partial Response (PR)
Time frame: Month 6
Percentage of Patients who Achieved Overall Response
Time frame: Month 6
Probability of Achieving an Effectiveness Event by Treatment Category
Time frame: Month 6, Years 1, 2, and up to approximately 3 years
Probability of Achieving an Effectiveness Event by ETB Dose Density
Time frame: Month 6, Years 1, 2, and up to approximately 3 years
Time to Achieve an Effectiveness Event by Treatment Category
Time frame: Up to approximately 3 years
Time to Achieve an Effectiveness Event by ETB Dose Density
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
Time frame: Month 6, and Years 1, 2, and 5
Time to Having a Safety Event by Treatment Category
Time frame: Up to approximately 9 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 May 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Anemia, Aplastic, run by Novartis Pharmaceuticals at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Probability of Achieving Hematologic Response by AA Treatment Category (time frame: Month 6, Years 1, 2, and up to approximately 3 years). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 May 2025.