Ignite Creation Date:
2025-12-25 @ 1:41 AM
Ignite Modification Date:
2025-12-26 @ 1:17 AM
Study NCT ID:
NCT06991894
Status:
COMPLETED
Last Update Posted:
2025-05-28
First Post:
2025-05-16
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Real-world Outcomes of Aplastic Anemia Patients Treated With Eltrombopag: A Medical Claims Database Study
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000741', 'term': 'Anemia, Aplastic'}], 'ancestors': [{'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D000080983', 'term': 'Bone Marrow Failure Disorders'}, {'id': 'D001855', 'term': 'Bone Marrow Diseases'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'RETROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 2517}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2021-07-14', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-05', 'completionDateStruct': {'date': '2024-05-17', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-05-16', 'studyFirstSubmitDate': '2025-05-16', 'studyFirstSubmitQcDate': '2025-05-16', 'lastUpdatePostDateStruct': {'date': '2025-05-28', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2025-05-28', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2024-05-17', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Probability of Achieving Hematologic Response by AA Treatment Category', 'timeFrame': 'Month 6, Years 1, 2, and up to approximately 3 years', 'description': '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:\n\n* CSA alone\n* CSA + ETB\n* ETB alone\n* ATG + CSA\n* ATG + CSA + ETB'}, {'measure': 'Probability of Achieving Hematologic Response by ETB Dose Density', 'timeFrame': 'Month 6, Years 1, 2, and up to approximately 3 years', 'description': '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:\n\n* Optimal dose-density: 62.5 milligrams (mg) or more mg/8 weeks,\n* Suboptimal dose-density: 25.1-62.4 mg/8 weeks,\n* Minimum dose: 25 mg or less/8weeks.'}, {'measure': 'Time to Achieve First Hematologic Response by AA Treatment Category', 'timeFrame': 'Up to approximately 3 years', 'description': '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:\n\n* CSA alone\n* CSA + ETB\n* ETB alone\n* ATG + CSA\n* ATG + CSA + ETB'}, {'measure': 'Time to Achieve First Hematologic Response by ETB Dose Density', 'timeFrame': 'Up to approximately 3 years', 'description': '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:\n\n* Optimal dose-density: 62.5 mg or more mg/8 weeks,\n* Suboptimal dose-density: 25.1-62.4 mg/8 weeks,\n* Minimum dose: 25 mg or less/8weeks.'}, {'measure': 'Percentage of Patients who Achieved Hematologic Response by Treatment Category', 'timeFrame': 'Months 3 and 6', 'description': 'Hematologic response was defined as the first occurrence of an 8-week period without any transfusion procedure. Treatment categories included:\n\n* CSA alone\n* CSA + ETB\n* ETB alone\n* ATG + CSA\n* ATG + CSA + ETB'}, {'measure': 'Cox Proportional Hazard Ratio for the Association Between Hematologic Response and Patient Characteristics', 'timeFrame': 'Up to approximately 3 years', 'description': '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).'}], 'secondaryOutcomes': [{'measure': 'Number of Patients per Demographic Category', 'timeFrame': 'Baseline', 'description': 'Demographics included:\n\n* Age category\n* Gender\n* BMI\n* Most frequent comorbidities\n* Most frequent medications received'}, {'measure': 'Hemoglobin Level', 'timeFrame': 'Baseline'}, {'measure': 'Platelets Count', 'timeFrame': 'Baseline'}, {'measure': 'Neutrophil Level', 'timeFrame': 'Baseline'}, {'measure': 'Number of Blood Transfusions', 'timeFrame': 'Up to approximately 7 months'}, {'measure': 'Total Units of Blood Transfusions', 'timeFrame': 'Up to approximately 7 months'}, {'measure': 'Total Amount of Blood and Plasma Transfused', 'timeFrame': 'Up to approximately 7 months'}, {'measure': 'Percentage of Patients With at Least One Transfusion of 400 Milliliters (mL) or More of red Blood Cells', 'timeFrame': 'Up to approximately 8 weeks'}, {'measure': 'Percentage of Patients who Achieved Complete Response (CR)', 'timeFrame': 'Month 6', 'description': 'CR was defined as hemoglobin \\>100 grams per liter (g/L) and neutrophils \\>1.0x10\\^9/L and platelets \\>100x10\\^9/L.'}, {'measure': 'Percentage of Patients who Achieved Partial Response (PR)', 'timeFrame': 'Month 6', 'description': '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.'}, {'measure': 'Percentage of Patients who Achieved Overall Response', 'timeFrame': 'Month 6', 'description': '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.'}, {'measure': 'Probability of Achieving an Effectiveness Event by Treatment Category', 'timeFrame': 'Month 6, Years 1, 2, and up to approximately 3 years', 'description': '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:\n\n* CSA alone\n* CSA + ETB\n* ETB alone\n* ATG + CSA\n* ATG + CSA + ETB'}, {'measure': 'Probability of Achieving an Effectiveness Event by ETB Dose Density', 'timeFrame': 'Month 6, Years 1, 2, and up to approximately 3 years', 'description': '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:\n\n* Optimal dose-density: 62.5 mg or more mg/8 weeks,\n* Suboptimal dose-density: 25.1-62.4 mg/8 weeks,\n* Minimum dose: 25 mg or less/8weeks.'}, {'measure': 'Time to Achieve an Effectiveness Event by Treatment Category', 'timeFrame': 'Up to approximately 3 years', 'description': '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.\n\nEffectiveness events included death, treatment discontinuation, relapse, BMT, bleeding, bleeding requiring transfusion, bleeding requiring a treatment, and infection. Treatment categories included:\n\n* CSA alone\n* CSA + ETB\n* ETB alone\n* ATG + CSA\n* ATG + CSA + ETB'}, {'measure': 'Time to Achieve an Effectiveness Event by ETB Dose Density', 'timeFrame': 'Up to approximately 3 years', 'description': '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.\n\nEffectiveness 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:\n\n* Optimal dose-density: 62.5 mg or more mg/8 weeks,\n* Suboptimal dose-density: 25.1-62.4 mg/8 weeks,\n* Minimum dose: 25 mg or less/8weeks.'}, {'measure': 'Number of Patients who Received First Line Treatment by Type of Treatment Received', 'timeFrame': 'Up to approximately 3 years'}, {'measure': 'Number of Patients who Received Second Line Treatment by Type of Treatment Received', 'timeFrame': 'Up to approximately 3 years'}, {'measure': 'Number of Patients who Received Third Line Treatment by Type of Treatment Received', 'timeFrame': 'Up to approximately 3 years'}, {'measure': 'Probability of Having a Safety Event by Treatment Category', 'timeFrame': 'Month 6, and Years 1, 2, and 5', 'description': '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).\n\nTreatment categories included:\n\n* CSA alone\n* CSA + ETB\n* ETB alone\n* ATG + CSA\n* ATG + CSA + ETB'}, {'measure': 'Time to Having a Safety Event by Treatment Category', 'timeFrame': 'Up to approximately 9 years', 'description': '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.\n\nSafety events included diabetes, hepatotoxicity event, hypertension event, thromboembolic event, transformation to MDS, AML, CMML or other leukemia, and transformation to PNH.\n\nTreatment categories included:\n\n* CSA alone\n* CSA + ETB\n* ETB alone\n* ATG + CSA\n* ATG + CSA + ETB'}]}, 'conditionsModule': {'keywords': ['Eltrombopag', 'Effectiveness', 'Safety', 'Treatment patterns'], 'conditions': ['Anemia, Aplastic']}, 'descriptionModule': {'briefSummary': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '90 Years', 'minimumAge': '15 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'This was a retrospective, noninterventional cohort study.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion and exclusion criteria for the safety population:\n\nInclusion criteria:\n\n* Patients with:\n\n * At least one confirmed diagnosis of AA registered during the baseline period, or\n * 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.\n* Had at least 6 months of continuous enrolment prior to the index date.\n\nConfirmed diagnosis was defined as having ≥1 inpatient or ≥2 outpatient claims with a relevant ICD-10 code and without any doubtful flag.\n\nContinuous enrolment (being continuously followed in the database) was defined as having at least one claim every semester.\n\nExclusion Criteria:\n\n* Not receiving ATG, CSA, ETB or romiplostim (ROM) during the selection period,\n* Had a diagnosis of acute myeloid leukemia (AML), chronic myelomonocytic leukemia (CMML), myelofibrosis, other hematological malignancies, or cataract before the index date.\n\nInclusion and exclusion criteria for the effectiveness population:\n\nInclusion criteria:\n\n* Had at least one confirmed diagnosis of AA registered before the index date,\n* 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,\n* Had at least 6 months of continuous enrolment prior to the index date,\n* Had at least a 6-month follow-up period.\n\nExclusion criteria:\n\n* Not receiving ATG, CSA, ETB or ROM during the selection period,\n* Had at least one prescription of ATG, CSA, ETB or ROM before the index date,\n* Had a diagnosis of AML, CMML or other hematological malignancies before the index date.'}, 'identificationModule': {'nctId': 'NCT06991894', 'briefTitle': 'Real-world Outcomes of Aplastic Anemia Patients Treated With Eltrombopag: A Medical Claims Database Study', 'organization': {'class': 'INDUSTRY', 'fullName': 'Novartis'}, 'officialTitle': 'Real-world Outcomes of Aplastic Anemia Patients Treated With Eltrombopag: A Retrospective Medical Claims Database Study (Eltrombopag for Aplastic Anemia)', 'orgStudyIdInfo': {'id': 'CETB115EJP04'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Anatomical Therapeutic Chemical (ATG)-free Cohort', 'description': '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.'}, {'label': 'ATG-Inclusive Cohort', 'description': '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.'}]}, 'contactsLocationsModule': {'locations': [{'zip': '07936', 'city': 'East Hanover', 'state': 'New Jersey', 'country': 'United States', 'facility': 'Novartis', 'geoPoint': {'lat': 40.8201, 'lon': -74.36487}}], 'overallOfficials': [{'name': 'Novartis Pharmaceuticals', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Novartis Pharmaceuticals'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Novartis Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}