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Completed

NCT Number: NCT06669221

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

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.

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Key information

Conditions

Aplastic Anemia (AA) Anemia Anemia, Aplastic Bone Marrow Diseases Bone Marrow Failure Disorders Hematologic Diseases Hemic and Lymphatic Diseases

Age range

15 year–90 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Novartis

Tokyo, 105-6333, Japan

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

  1. Probability of Having Blood Transfusion in the Matched Effectiveness Population

    Time frame: 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.

  2. Crude Hazard Ratios of Having Blood Transfusion After 6 Months of Follow-up in the Matched Effectiveness Population

    Time frame: Baseline, 6 months

  3. Adjusted Hazard Ratios of Having Blood Transfusion After 6 Months of Follow-up in the Matched Effectiveness Population

    Time frame: 6 months

Secondary outcomes

  1. Sex

    Time frame: Baseline

  2. Age

    Time frame: Baseline

  3. Weight

    Time frame: Baseline

  4. Body Mass Index (BMI)

    Time frame: Baseline

  5. Number of Patients With Aplastic Anemia, per Severity Level

    Time frame: Baseline

  6. Number of Patients With Immune Thrombocytopenia (ITP) Diagnosis Before Index Date

    Time frame: Baseline

  7. Number of Patients With Myelodysplastic Syndrome (MDS) Diagnosis Before Index Date

    Time frame: Baseline

  8. Number of Patients With Pre-index Comorbidities

    Time frame: Baseline

  9. Number of Patients With Pre-index Use of Chloramphenicol

    Time frame: Baseline

  10. Platelet Count

    Time frame: Baseline

  11. Neutrophil Count

    Time frame: Baseline

  12. Hemoglobin Level

    Time frame: Baseline

  13. Number of Patients Per Treatment Therapy in the Matched Effectiveness Population

    Time frame: Baseline

  14. Number of Patients With Aplastic Anemia in the Matched Effectiveness Population, per Severity Level

    Time frame: Baseline

  15. Number of Patients in the Matched Effectiveness Population Who Discontinued

    Time frame: Baseline, at 6 and 9 months, and at 1 and 2 years

  16. Probability of Having Blood Transfusion in Early Drug Intervention and Watchful Observation Matched Patients From the Effectiveness Population

    Time frame: 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.

  17. Probability of Having Stem-Cell Transplantation (SCT) in Early Drug Intervention and Watchful Observation Matched Patients From the Effectiveness Population

    Time frame: 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.

  18. Probability of Having Anti-Thymocyte Globulin (ATG) in Early Drug Intervention and Watchful Observation Matched Patients From the Effectiveness Population

    Time frame: 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.

  19. Probability of Having Blood Transfusion at Different Timepoints by Drug Categories in the Matched Effectiveness Population

    Time frame: 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.

  20. Probability of Having SCT at Different Timepoints by Drug Categories in the Matched Effectiveness Population

    Time frame: 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.

  21. Probability of Having ATG at Different Timepoints by Drug Categories in the Matched Effectiveness Population

    Time frame: 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.

  22. Probability of Having a Bleeding Event in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population

    Time frame: 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.

  23. Probability of Having Cataract in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population

    Time frame: 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.

  24. Probability of Having Diabetes in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population

    Time frame: 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.

  25. Probability of Having Hepatotoxicity in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population

    Time frame: 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.

  26. Probability of Having Hypertension in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population

    Time frame: 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.

  27. Probability of Having an Infection in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population

    Time frame: 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.

  28. Probability of Having Kidney Dysfunction in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population

    Time frame: 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.

  29. 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

    Time frame: 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.

  30. Probability of Having MDS in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population

    Time frame: 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.

  31. Probability of Having Paroxysmal Nocturnal Hemoglobinuria (PNH) in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population

    Time frame: 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.

  32. Probability of Having a Thromboembolic Event in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population

    Time frame: 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.

  33. Probability of Having Myelofibrosis in Early Drug Intervention and Watchful Observation Matched Patients From the Safety Population

    Time frame: 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.

  34. Probability of Having a Bleeding Event by Drug Categories in the Matched Safety Population

    Time frame: 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.

  35. Probability of Having Cataract by Drug Categories in the Matched Safety Population

    Time frame: 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.

  36. Probability of Having Diabetes by Drug Categories in the Matched Safety Population

    Time frame: 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.

  37. Probability of Having Hepatotoxicity by Drug Categories in the Matched Safety Population

    Time frame: 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.

  38. Probability of Having an Infection by Drug Categories in the Matched Safety Population

    Time frame: 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.

  39. Probability of Having Kidney Dysfunction by Drug Categories in the Matched Safety Population

    Time frame: 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.

  40. Probability of Having AML, CMML or Another Leukemia by Drug Categories in the Matched Safety Population

    Time frame: 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.

  41. Probability of Having MDS by Drug Categories in the Matched Safety Population

    Time frame: 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.

  42. Probability of Having PNH by Drug Categories in the Matched Safety Population

    Time frame: 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.

  43. Probability of Having a Thromboembolic Event by Drug Categories in the Matched Safety Population

    Time frame: 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.

  44. Probability of Having Myelofibrosis by Drug Categories in the Matched Safety Population

    Time frame: 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.

Sponsors and collaborators

Lead sponsor

Novartis Pharmaceuticals

Industry

Registry information

Important dates

Study start
2022
Primary completion
2023
Study completion
2023
First posted
Nov 1, 2024
Registry last updated
Nov 1, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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