Clinical trial NCT06669221: Effectiveness of Early Intervention in Transfusion Independent Aplastic Anemia: a Retrospective Medical Claims Database Study
Official title: Effectiveness of Early Intervention in Transfusion Independent Aplastic Anemia: a Retrospective Medical Claims Database Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Aplastic Anemia (AA)
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2024-11-01
Enrollment: 1603
Sex: ALL
Minimum age (years): 15
Maximum age (years): 90
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: This was a retrospective, noninterventional cohort study.

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

* Having at least one confirmed diagnosis of AA within the selection period (1 inpatient or 2 outpatient claims per the International Statistical Classification of Diseases, 10th Revision \[ICD-10\] code, without any suspicion flag).
* Being aged 15 to 90 years at the index date.
* Having at least 3 months of continuous enrolment prior to the index date.

Exclusion Criteria:

* Having a blood transfusion recorded any time before the index date.
* Having anti-thymocyte globulin (ATG) treatment recorded within 3 months after the index date.
* Having at least one prescription record for any of the drug treatments of interest any time before the index date.
* Having a diagnosis of acute myeloid leukemia (AML), chronic myelomonocytic leukemia (CMML) or other leukemia any time before the index date.
* Having an AA diagnosis (without suspicious flag) date earlier than the start of patient's observation in the database.
* Having less than 6 months of continuous follow-up.

Safety Population - Additional Exclusion Criterion:

- Having a diagnosis of hepatotoxicity, kidney dysfunction, hypertension, or diabetes mellitus any time before the index date.

Primary outcomes:
measure: Probability of Having Blood Transfusion in the Matched Effectiveness Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Crude Hazard Ratios of Having Blood Transfusion After 6 Months of Follow-up in the Matched Effectiveness Population
timeFrame: Baseline, 6 months
measure: Adjusted Hazard Ratios of Having Blood Transfusion After 6 Months of Follow-up in the Matched Effectiveness Population
timeFrame: 6 months
Secondary outcomes:
measure: Sex
timeFrame: Baseline
measure: Age
timeFrame: Baseline
measure: Weight
timeFrame: Baseline
measure: Body Mass Index (BMI)
timeFrame: Baseline
measure: Number of Patients With Aplastic Anemia, per Severity Level
timeFrame: Baseline
measure: Number of Patients With Immune Thrombocytopenia (ITP) Diagnosis Before Index Date
timeFrame: Baseline
measure: Number of Patients With Myelodysplastic Syndrome (MDS) Diagnosis Before Index Date
timeFrame: Baseline
measure: Number of Patients With Pre-index Comorbidities
timeFrame: Baseline
measure: Number of Patients With Pre-index Use of Chloramphenicol
timeFrame: Baseline
measure: Platelet Count
timeFrame: Baseline
measure: Neutrophil Count
timeFrame: Baseline
measure: Hemoglobin Level
timeFrame: Baseline
measure: Number of Patients Per Treatment Therapy in the Matched Effectiveness Population
timeFrame: Baseline
measure: Number of Patients With Aplastic Anemia in the Matched Effectiveness Population, per Severity Level
timeFrame: Baseline
measure: Number of Patients in the Matched Effectiveness Population Who Discontinued
timeFrame: Baseline, at 6 and 9 months, and at 1 and 2 years
measure: Probability of Having Blood Transfusion in Early Drug Intervention and Watchful Observation Matched Patients From the Effectiveness Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having Stem-Cell Transplantation (SCT) in Early Drug Intervention and Watchful Observation Matched Patients From the Effectiveness Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having Anti-Thymocyte Globulin (ATG) in Early Drug Intervention and Watchful Observation Matched Patients From the Effectiveness Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having Blood Transfusion at Different Timepoints by Drug Categories in the Matched Effectiveness Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having SCT at Different Timepoints by Drug Categories in the Matched Effectiveness Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having ATG at Different Timepoints by Drug Categories in the Matched Effectiveness Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having a Bleeding Event in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having Cataract in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having Diabetes in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having Hepatotoxicity in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having Hypertension in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having an Infection in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having Kidney Dysfunction in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: 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
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having MDS in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having Paroxysmal Nocturnal Hemoglobinuria (PNH) in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having a Thromboembolic Event in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having Myelofibrosis in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having a Bleeding Event by Drug Categories in the Matched Safety Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having Cataract by Drug Categories in the Matched Safety Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having Diabetes by Drug Categories in the Matched Safety Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having Hepatotoxicity by Drug Categories in the Matched Safety Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having an Infection by Drug Categories in the Matched Safety Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having Kidney Dysfunction by Drug Categories in the Matched Safety Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having AML, CMML or Another Leukemia by Drug Categories in the Matched Safety Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having MDS by Drug Categories in the Matched Safety Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having PNH by Drug Categories in the Matched Safety Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having a Thromboembolic Event by Drug Categories in the Matched Safety Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
measure: Probability of Having Myelofibrosis by Drug Categories in the Matched Safety Population
timeFrame: Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years
Other outcomes:
Locations:
city: Tokyo
country: Japan
facility: Novartis
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: