Effectiveness of Early Intervention in Transfusion Independent Aplastic Anemia: a Retrospective Medical Claims Database Study

Novartis Pharmaceuticals1,603 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

1,603

Conditions

Aplastic Anemia (AA)

Eligibility

Sex: ALLMin age: 15 YearsMax age: 90 Years

Medical Language ↔ Plain English

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.

Locations (1)

Novartis

Tokyo, Japan

Outcomes

Primary Outcomes

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

Secondary Outcomes

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

Data from ClinicalTrials.gov

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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