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

NCT06298643 | Completed FDA Drug

• 1 other identifier: CA056-1094
• Study Type: observational
• Target: 177
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Lower-risk Myelodysplastic Syndromes

Outcome Measures

Primary Outcomes (16)

• Participant age

  At date of diagnosis

• Participant sex

  At date of diagnosis

• Participant weight

  At date of diagnosis

• Participant height

  At date of diagnosis

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

  From 30 days prior to diagnosis up to 7 days post to diagnosis

• Participant Eastern Cooperative Oncology Group (ECOG) score

  From 30 days prior to diagnosis up to 7 days post to diagnosis

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

  Date of diagnosis, 6-months and 12 months post-diagnosis

• Percentage of ring sideroblasts present in participants

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

• Percentage of bone marrow blasts present in participants

  From 30 days prior to diagnosis up to 7 days post to diagnosis

• Histopathology results

  From 30 days prior to diagnosis up to 7 days post to diagnosis

• Number of red-blood cell transfusions received by participants

  Up to 56 days post diagnosis

• Treatment prescribed by line of therapy

  From date of diagnosis, up to 5-years

• Duration of treatment by line of therapy

  From date of diagnosis, up to 5-years

• Time between date of diagnosis and treatment

  From date of diagnosis to treatment initiation

• Time between date of treatment initiation to treatment discontinuation

  Date of treatment initiation, up to 5-years

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

  Date of diagnosis, up to 5-years

Secondary Outcomes (28)

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

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

• Mean hemoglobin change of participants during first-line of therapy

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

• 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

  Week 1-24 post treatment initiation

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

  Week 24, 48 and 72 post date of initiation of treatment

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

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

Study Arms (2)

Transfusion dependent

Participants that received ≥2 units of red blood cell transfusion during the 8 weeks after initial myelodysplastic syndrome diagnosis date.

Procedure: Red blood cell transfusion

Non-transfusion dependent

Participants that received 0 units or \<2 units of red blood cell transfusion during the 8 weeks after initial myelodysplastic syndrome diagnosis date.

Procedure: Red blood cell transfusion

Interventions

Red blood cell transfusion PROCEDURE

≥2 units of red blood cell transfusion

Transfusion dependent

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Adult participants with myelodysplastic syndrome in Japan

You may qualify if:

• 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

You may not qualify if:

• Participants \<18 years of age at index date
• Participants who have a look back period \<30 days from initial myelodysplastic syndrome diagnosis date

Sponsors & Collaborators

• Bristol-Myers Squibb: lead

Study Sites (1)

Mebix, Inc

Tokyo, Minato-ku, 107-0052, Japan

Location

Related Links

• BMS Clinical Trial Information
• FDA Safety Alerts and Recalls

MeSH Terms

Interventions

Erythrocyte Transfusion

Intervention Hierarchy (Ancestors)

Blood Component Transfusion; Blood Transfusion; Biological Therapy; Therapeutics

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 1, 2024
• First Posted: March 7, 2024
• Study Start: December 22, 2023
• Primary Completion: July 9, 2025
• Study Completion: July 9, 2025
• Last Updated: December 4, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

JP (1)