Effectiveness of Early Intervention in Transfusion Independent Aplastic Anemia: a Retrospective M… (NCT06669221) | Clinical Trial Compass
CompletedNot Applicable
Effectiveness of Early Intervention in Transfusion Independent Aplastic Anemia: a Retrospective Medical Claims Database Study
Japan1,603 participantsStarted 2022-05-05
Plain-language 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.
Who can participate
Age range
15 Years – 90 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
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
2
Crude Hazard Ratios of Having Blood Transfusion After 6 Months of Follow-up in the Matched Effectiveness Population
Timeframe: Baseline, 6 months
3
Adjusted Hazard Ratios of Having Blood Transfusion After 6 Months of Follow-up in the Matched Effectiveness Population