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This was a retrospective non-interventional cohort study with secondary use of data from the Medical Data Vision (MDV) hospital-based database to evaluate the effectiveness of early drug intervention in preventing transfusion compared to watchful observation among adult transfusion-independent aplastic anemia (AA) patients in Japan.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Early drug intervention group | Patients who had at least one prescription record associated with any of the drug treatments of interest (cyclosporine A, eltrombopag, romiplostin, danazol, or methenolone) during each period of interest before the first transfusion record during the follow-up period. | ||
| Watchful observation group | Patients with no prescription record associated with any of the drug treatments of interest (cyclosporine A, eltrombopag, romiplostin, danazol, or methenolone) during the follow-up period and before the first transfusion record, if any. |
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| Measure | Description | Time Frame |
|---|---|---|
| Probability of Having Blood Transfusion in the Matched Effectiveness Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Crude Hazard Ratios of Having Blood Transfusion After 6 Months of Follow-up in the Matched Effectiveness Population | Baseline, 6 months | |
| Adjusted Hazard Ratios of Having Blood Transfusion After 6 Months of Follow-up in the Matched Effectiveness Population | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Sex | Baseline | |
| Age | Baseline | |
| Weight | Baseline |
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Inclusion Criteria:
Exclusion Criteria:
Safety Population - Additional Exclusion Criterion:
- Having a diagnosis of hepatotoxicity, kidney dysfunction, hypertension, or diabetes mellitus any time before the index date.
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This was a retrospective, noninterventional cohort study.
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis | Tokyo | 105-6333 | Japan |
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| ID | Term |
|---|---|
| D000741 | Anemia, Aplastic |
| ID | Term |
|---|---|
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000080983 | Bone Marrow Failure Disorders |
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| Body Mass Index (BMI) | Baseline |
| Number of Patients With Aplastic Anemia, per Severity Level | Baseline |
| Number of Patients With Immune Thrombocytopenia (ITP) Diagnosis Before Index Date | Baseline |
| Number of Patients With Myelodysplastic Syndrome (MDS) Diagnosis Before Index Date | Baseline |
| Number of Patients With Pre-index Comorbidities | Baseline |
| Number of Patients With Pre-index Use of Chloramphenicol | Baseline |
| Platelet Count | Baseline |
| Neutrophil Count | Baseline |
| Hemoglobin Level | Baseline |
| Number of Patients Per Treatment Therapy in the Matched Effectiveness Population | Baseline |
| Number of Patients With Aplastic Anemia in the Matched Effectiveness Population, per Severity Level | Baseline |
| Number of Patients in the Matched Effectiveness Population Who Discontinued | Baseline, at 6 and 9 months, and at 1 and 2 years |
| Probability of Having Blood Transfusion in Early Drug Intervention and Watchful Observation Matched Patients From the Effectiveness Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having Stem-Cell Transplantation (SCT) in Early Drug Intervention and Watchful Observation Matched Patients From the Effectiveness Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having Anti-Thymocyte Globulin (ATG) in Early Drug Intervention and Watchful Observation Matched Patients From the Effectiveness Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having Blood Transfusion at Different Timepoints by Drug Categories in the Matched Effectiveness Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having SCT at Different Timepoints by Drug Categories in the Matched Effectiveness Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having ATG at Different Timepoints by Drug Categories in the Matched Effectiveness Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having a Bleeding Event in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having Cataract in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having Diabetes in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having Hepatotoxicity in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having Hypertension in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having an Infection in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having Kidney Dysfunction in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having Acute Myeloid Leukemia (AML), Chronic Myelomonocytic Leukemia (CMML) or Another Leukemia in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having MDS in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having Paroxysmal Nocturnal Hemoglobinuria (PNH) in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having a Thromboembolic Event in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having Myelofibrosis in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having a Bleeding Event by Drug Categories in the Matched Safety Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having Cataract by Drug Categories in the Matched Safety Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having Diabetes by Drug Categories in the Matched Safety Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having Hepatotoxicity by Drug Categories in the Matched Safety Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having an Infection by Drug Categories in the Matched Safety Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having Kidney Dysfunction by Drug Categories in the Matched Safety Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having AML, CMML or Another Leukemia by Drug Categories in the Matched Safety Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having MDS by Drug Categories in the Matched Safety Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having PNH by Drug Categories in the Matched Safety Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having a Thromboembolic Event by Drug Categories in the Matched Safety Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| Probability of Having Myelofibrosis by Drug Categories in the Matched Safety Population | The Kaplan-Meier survival analysis technique was used to estimate probability. | Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years |
| D001855 | Bone Marrow Diseases |