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

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT06669221

Enrollment

1603

Registered

2024-11-01

Start date

2022-05-05

Completion date

2023-11-03

Last updated

2024-11-01

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Aplastic Anemia (AA)

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.

Interventions

None listed

Study design

Observational model

COHORT

Time perspective

RETROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

15 Years to 90 Years

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame	Description
Probability of Having Blood Transfusion in the Matched Effectiveness Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
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	—

Secondary

Measure	Time frame	Description
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	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having Stem-Cell Transplantation (SCT) in Early Drug Intervention and Watchful Observation Matched Patients From the Effectiveness Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having Anti-Thymocyte Globulin (ATG) in Early Drug Intervention and Watchful Observation Matched Patients From the Effectiveness Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having Blood Transfusion at Different Timepoints by Drug Categories in the Matched Effectiveness Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having SCT at Different Timepoints by Drug Categories in the Matched Effectiveness Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having ATG at Different Timepoints by Drug Categories in the Matched Effectiveness Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having a Bleeding Event in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having Cataract in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having Diabetes in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having Hepatotoxicity in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having Hypertension in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having an Infection in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having Kidney Dysfunction in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
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	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having MDS in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having Paroxysmal Nocturnal Hemoglobinuria (PNH) in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having a Thromboembolic Event in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having Myelofibrosis in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having a Bleeding Event by Drug Categories in the Matched Safety Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having Cataract by Drug Categories in the Matched Safety Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having Diabetes by Drug Categories in the Matched Safety Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having Hepatotoxicity by Drug Categories in the Matched Safety Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having an Infection by Drug Categories in the Matched Safety Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having Kidney Dysfunction by Drug Categories in the Matched Safety Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having AML, CMML or Another Leukemia by Drug Categories in the Matched Safety Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having MDS by Drug Categories in the Matched Safety Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Sex	Baseline	—
Probability of Having a Thromboembolic Event by Drug Categories in the Matched Safety Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having Myelofibrosis by Drug Categories in the Matched Safety Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Probability of Having PNH by Drug Categories in the Matched Safety Population	Baseline, at 6 and 9 months, and at 1, 2, 5, and 10 years	The Kaplan-Meier survival analysis technique was used to estimate probability.
Age	Baseline	—
Weight	Baseline	—

Countries

Japan

Outcome results