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Completed

NCT Number: NCT06298643

Real-World Practice Patterns and Outcomes of Lower-Risk Myelodysplastic Syndrome Patients in Japan

The purpose of this study is to describe the treatment patterns, clinical outcomes, healthcare resource utilization (HCRU) and medical costs of lower-risk myelodysplastic syndromes patients in Japan.

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Key information

Conditions

Lower-risk Myelodysplastic Syndromes

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Mebix, Inc

Tokyo, Minato-ku, 107-0052, Japan

Who can participate

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

Inclusion criteria

  • Participants with at least one definitive myelodysplastic syndrome (MDS) diagnosis as per the 10th Revision of the International Statistical Classification of Diseases and Related Health Problems (MDS; ICD10: D46.X) between 01-May-2017 and 31-Jan-2022
  • Participants with confirmed low-risk MDS by International Prognostic Scoring System (IPSS) or Revised International Prognostic Scoring System (IPSS-R) during baseline as follows:
  • Record of low (0 points) or intermediate-1 (>0 to 1 point) MDS according to IPSS scoring; or
  • Record of very low (≤1.5), low (>1.5-3), or intermediate (>3-4.5) MDS according to IPSS-R scoring; or
  • Mention of very low, low, intermediate, intermediate-1, or lower risk MDS in the Electronic Medical Records (EMR)
  • Participants with a record of a confirmed bone marrow procedure (bone marrow aspiration code D404-00 and/or bone marrow biopsy code D404-02) during baseline period

Exclusion criteria

  • Participants not meeting the inclusion criteria
  • Participants <18 years of age at index date
  • Participants who have a look back period <30 days from initial myelodysplastic syndrome diagnosis date

Treatment and study plan

Red blood cell transfusion

Procedure

≥2 units of red blood cell transfusion

Primary outcomes

  1. Participant age

    Time frame: At date of diagnosis

  2. Participant sex

    Time frame: At date of diagnosis

  3. Participant weight

    Time frame: At date of diagnosis

  4. Participant height

    Time frame: At date of diagnosis

  5. Participant comorbidities calculated using the Charlson Comorbidity Index (CCI) method

    Time frame: From 30 days prior to diagnosis up to 7 days post to diagnosis

  6. Participant Eastern Cooperative Oncology Group (ECOG) score

    Time frame: From 30 days prior to diagnosis up to 7 days post to diagnosis

  7. Participant International Prognostic Scoring System (IPSS) or Revised International Prognostic Scoring System (IPSS-R) risk category

    Time frame: Date of diagnosis, 6-months and 12 months post-diagnosis

  8. Percentage of ring sideroblasts present in participants

    Time frame: Date of bone marrow test; from 30 days prior to diagnosis up to 7 days post to diagnosis

  9. Percentage of bone marrow blasts present in participants

    Time frame: From 30 days prior to diagnosis up to 7 days post to diagnosis

  10. Histopathology results

    Time frame: From 30 days prior to diagnosis up to 7 days post to diagnosis

  11. Number of red-blood cell transfusions received by participants

    Time frame: Up to 56 days post diagnosis

  12. Treatment prescribed by line of therapy

    Time frame: From date of diagnosis, up to 5-years

  13. Duration of treatment by line of therapy

    Time frame: From date of diagnosis, up to 5-years

  14. Time between date of diagnosis and treatment

    Time frame: From date of diagnosis to treatment initiation

  15. Time between date of treatment initiation to treatment discontinuation

    Time frame: Date of treatment initiation, up to 5-years

  16. Time between date of diagnosis to date of first red-blood cell transfusion

    Time frame: Date of diagnosis, up to 5-years

Secondary outcomes

  1. Number of participants that achieved red-blood cell transfusion independence during first-line of therapy treatment

    Time frame: From week 1-24 and week 1-48 post date of treatment initiation

  2. Mean hemoglobin change of participants during first-line of therapy

    Time frame: From week 1-24 and week 1-48 post date of treatment initiation

  3. Number of transfusion dependant participants achieving re-blood cell transfusion independence for ≥12 weeks with associated concurrent mean hemoglobin increase of ≥1.5 g/dL during first-line of therapy

    Time frame: Week 1-24 post treatment initiation

  4. Number of non-transfusion dependant participants achieving red-blood cell transfusion independence during first-line therapy for ≥24, 48 and 72 weeks

    Time frame: Week 24, 48 and 72 post date of initiation of treatment

  5. Time to red-blood call transfusion independence of ≥12 weeks for transfusion dependant participants receiving first line therapy

    Time frame: From week 1-24 and week 1-48 post date of treatment initiation

  6. Hematologic Improvement - Erythroid (HI-E) response of transfusion dependant participants receiving first line therapy

    Time frame: From week 1-24 post date of treatment initiation

  7. Modified Hematologic Improvement - Erythroid (mHI-E) of transfusion dependant participants receiving first line therapy

    Time frame: From week 1-24 post date of treatment initiation

  8. Hematologic Improvement - Erythroid (HI-E) response of non-transfusion dependant participants receiving first line therapy

    Time frame: From week 1-24 and week 1-48 post date of treatment initiation

  9. Modified Hematologic Improvement - Erythroid (mHI-E) of non-transfusion dependant participants receiving first line therapy

    Time frame: From week 1-24 and week 1-48 post date of treatment initiation

  10. Time to Hematologic Improvement - Erythroid (HI-E) or Modified hematologic improvement - Erythroid (mHI-E) during first-line therapy

    Time frame: From week 1-24 and week 1-48 post date of treatment initiation

  11. Duration of Hematologic Improvement - Erythroid (HI-E) or Modified hematologic improvement - Erythroid (mHI-E) during first-line therapy

    Time frame: From date of treatment initiation to date of treatment discontinuation, up 5-years

  12. Duration of red-blood call transfusion independence of ≥12 weeks for transfusion dependant participants receiving first line therapy

    Time frame: From date of treatment initiation to date of treatment discontinuation, up 5-years

  13. Number of participants diagnosed with Acute Myeloid Leukemia

    Time frame: Date of treatment initiation, up to 5-years

  14. Time to Acute Myeloid Leukemia diagnosis

    Time frame: Date of treatment initiation, up to 5-years

  15. Overall survival (OS) of participants

    Time frame: 1, 2, 3 and 5-years post treatment initiation

  16. Number of red-blood cell transfusion days per participant

    Time frame: Date of treatment initiation, up to 5-years

  17. Number of red-blood cell units used per transfusion

    Time frame: Date of treatment initiation, up to 5-years

  18. Number of outpatient visits per month

    Time frame: Date of treatment initiation, up to 5-years

  19. Number of hospital admissions

    Time frame: Date of treatment initiation, up to 5-years

  20. Length of hospitalization

    Time frame: Date of treatment initiation, up to 5-years

  21. Reason for hospitalization

    Time frame: Date of treatment initiation, up to 5-years

  22. Number of emergency department visits

    Time frame: Date of treatment initiation, up to 5-years

  23. Number of Intensive care unit admissions (ICU)

    Time frame: Date of treatment initiation, up to 5-years

  24. Number of outpatient/inpatient medication claims

    Time frame: Date of treatment initiation, up to 5-years

  25. Supplemental medication use: Iron chelation use

    Time frame: Date of treatment initiation, up to 5-years

  26. Supplemental medication use: Granulocyte-colony stimulating factors (G-CSF)

    Time frame: Date of treatment initiation, up to 5-years

  27. Number of hematology tests ordered

    Time frame: Date of treatment initiation, up to 5-years

  28. Medical costs

    Time frame: Date of treatment initiation, up to 5-years

    Including: cost of red-blood cell transfusion, cost of red-blood cell transfusion administration, cost of erythropoiesis stimulating agents (ESA) medication, cost of ESA administration, cost of iron chelation therapy medications, cost of iron chelation therapy administration, other costs and total costs

Sponsors and collaborators

Lead sponsor

Bristol-Myers Squibb

Industry

Registry information

Important dates

Study start
2023
Primary completion
2025
Study completion
2025
First posted
Mar 7, 2024
Registry last updated
Dec 4, 2025

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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