Clinical trial NCT06991894: Real-world Outcomes of Aplastic Anemia Patients Treated With Eltrombopag: A Medical Claims Database Study
Official title: Real-world Outcomes of Aplastic Anemia Patients Treated With Eltrombopag: A Retrospective Medical Claims Database Study (Eltrombopag for Aplastic Anemia)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Anemia, Aplastic
Keywords: Eltrombopag
Effectiveness
Safety
Treatment patterns
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2025-05-28
Enrollment: 2517
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: The main goal of this study was to investigate the effectiveness and safety of eltrombopag (ETB) when compared to other treatments in Japanese aplastic anemia (AA) patients using data from the Medical Data Vision (MDV) hospital-based database.


Eligibility criteria: Inclusion and exclusion criteria for the safety population:

Inclusion criteria:

* Patients with:

  * At least one confirmed diagnosis of AA registered during the baseline period, or
  * At least one confirmed diagnosis of idiopathic thrombocytopenic purpura (International Classification of Diseases, 10th Revision \[ICD10\] code: D69.3) registered before the index date and at least one confirmed diagnosis of AA during the follow-up period.
* Had at least 6 months of continuous enrolment prior to the index date.

Confirmed diagnosis was defined as having ≥1 inpatient or ≥2 outpatient claims with a relevant ICD-10 code and without any doubtful flag.

Continuous enrolment (being continuously followed in the database) was defined as having at least one claim every semester.

Exclusion Criteria:

* Not receiving ATG, CSA, ETB or romiplostim (ROM) during the selection period,
* Had a diagnosis of acute myeloid leukemia (AML), chronic myelomonocytic leukemia (CMML), myelofibrosis, other hematological malignancies, or cataract before the index date.

Inclusion and exclusion criteria for the effectiveness population:

Inclusion criteria:

* Had at least one confirmed diagnosis of AA registered before the index date,
* Had at least one procedure for any type of transfusion such as red blood cell transfusion, platelet transfusion, or granulocyte transfusion registered during the baseline period or within 4 weeks after the index date,
* Had at least 6 months of continuous enrolment prior to the index date,
* Had at least a 6-month follow-up period.

Exclusion criteria:

* Not receiving ATG, CSA, ETB or ROM during the selection period,
* Had at least one prescription of ATG, CSA, ETB or ROM before the index date,
* Had a diagnosis of AML, CMML or other hematological malignancies before the index date.

Primary outcomes:
measure: Probability of Achieving Hematologic Response by AA Treatment Category
timeFrame: Month 6, Years 1, 2, and up to approximately 3 years
measure: Probability of Achieving Hematologic Response by ETB Dose Density
timeFrame: Month 6, Years 1, 2, and up to approximately 3 years
measure: Time to Achieve First Hematologic Response by AA Treatment Category
timeFrame: Up to approximately 3 years
measure: Time to Achieve First Hematologic Response by ETB Dose Density
timeFrame: Up to approximately 3 years
measure: Percentage of Patients who Achieved Hematologic Response by Treatment Category
timeFrame: Months 3 and 6
measure: Cox Proportional Hazard Ratio for the Association Between Hematologic Response and Patient Characteristics
timeFrame: Up to approximately 3 years
Secondary outcomes:
measure: Number of Patients per Demographic Category
timeFrame: Baseline
measure: Hemoglobin Level
timeFrame: Baseline
measure: Platelets Count
timeFrame: Baseline
measure: Neutrophil Level
timeFrame: Baseline
measure: Number of Blood Transfusions
timeFrame: Up to approximately 7 months
measure: Total Units of Blood Transfusions
timeFrame: Up to approximately 7 months
measure: Total Amount of Blood and Plasma Transfused
timeFrame: Up to approximately 7 months
measure: Percentage of Patients With at Least One Transfusion of 400 Milliliters (mL) or More of red Blood Cells
timeFrame: Up to approximately 8 weeks
measure: Percentage of Patients who Achieved Complete Response (CR)
timeFrame: Month 6
measure: Percentage of Patients who Achieved Partial Response (PR)
timeFrame: Month 6
measure: Percentage of Patients who Achieved Overall Response
timeFrame: Month 6
measure: Probability of Achieving an Effectiveness Event by Treatment Category
timeFrame: Month 6, Years 1, 2, and up to approximately 3 years
measure: Probability of Achieving an Effectiveness Event by ETB Dose Density
timeFrame: Month 6, Years 1, 2, and up to approximately 3 years
measure: Time to Achieve an Effectiveness Event by Treatment Category
timeFrame: Up to approximately 3 years
measure: Time to Achieve an Effectiveness Event by ETB Dose Density
timeFrame: Up to approximately 3 years
measure: Number of Patients who Received First Line Treatment by Type of Treatment Received
timeFrame: Up to approximately 3 years
measure: Number of Patients who Received Second Line Treatment by Type of Treatment Received
timeFrame: Up to approximately 3 years
measure: Number of Patients who Received Third Line Treatment by Type of Treatment Received
timeFrame: Up to approximately 3 years
measure: Probability of Having a Safety Event by Treatment Category
timeFrame: Month 6, and Years 1, 2, and 5
measure: Time to Having a Safety Event by Treatment Category
timeFrame: Up to approximately 9 years
Other outcomes:
Locations:
city: East Hanover
country: United States
facility: Novartis
state: New Jersey
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: