An observational study in Aplastic Anemia (AA), sponsored by Novartis Pharmaceuticals. Completed at 1 site in Japan. Open to participants aged 15 Years to 90 Years. Per ClinicalTrials.gov, last updated 2024-11-01.
Sponsored by Novartis Pharmaceuticals (part of Novartis) · Observational
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.
1,733 studies on the registry are indexed under Anemia; 246 are open to participants now.
This study's enrollment of 1,603 is above the median of 200 across 326 observational studies indexed under Anemia.
Browse Anemia studies →Novartis Pharmaceuticals is the lead sponsor of 2,673 studies on the registry; 228 are open to participants now.
Of its 576 completed or terminated interventional studies of FDA-regulated products, 431 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
This was a retrospective, noninterventional cohort study.
Exclusion Criteria:
Safety Population - Additional Exclusion Criterion:
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.
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.
Probability of Having Blood Transfusion in the Matched Effectiveness Population
The Kaplan-Meier survival analysis technique was used to estimate probability.
Time frame: 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
Time frame: Baseline, 6 months
Adjusted Hazard Ratios of Having Blood Transfusion After 6 Months of Follow-up in the Matched Effectiveness Population
Time frame: 6 months
Sex
Time frame: Baseline
Age
Time frame: Baseline
Weight
Time frame: Baseline
Body Mass Index (BMI)
Time frame: Baseline
Number of Patients With Aplastic Anemia, per Severity Level
Time frame: Baseline
Number of Patients With Immune Thrombocytopenia (ITP) Diagnosis Before Index Date
Time frame: Baseline
Number of Patients With Myelodysplastic Syndrome (MDS) Diagnosis Before Index Date
Time frame: Baseline
Number of Patients With Pre-index Comorbidities
Time frame: Baseline
Number of Patients With Pre-index Use of Chloramphenicol
Time frame: Baseline
Platelet Count
Time frame: Baseline
Neutrophil Count
Time frame: Baseline
Hemoglobin Level
Time frame: Baseline
Number of Patients Per Treatment Therapy in the Matched Effectiveness Population
Time frame: Baseline
Number of Patients With Aplastic Anemia in the Matched Effectiveness Population, per Severity Level
Time frame: Baseline
Number of Patients in the Matched Effectiveness Population Who Discontinued
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
This study is completed, as verified in Oct 2024. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.