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.

Study Overview

• Status: Completed
• Conditions: Lower-risk Myelodysplastic Syndromes
• Intervention / Treatment: Procedure: Red blood cell transfusion; Procedure: Red blood cell transfusion

• Study Type: Observational
• Enrollment (Actual): 177

Contacts and Locations

Study Locations

• Japan
  • Minato-ku
    • Tokyo, Minato-ku, Japan, 107-0052
      • Mebix, Inc

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: N/A

• Sampling Method: Non-Probability Sample

Study Population

Adult participants with myelodysplastic syndrome in Japan

Description

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

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
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; ≥2 units of red blood cell transfusion; Procedure: Red blood cell transfusion; 0 units or <2 units of 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; ≥2 units of red blood cell transfusion; Procedure: Red blood cell transfusion; 0 units or <2 units of red blood cell transfusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
Hematologic Improvement - Erythroid (HI-E) response of transfusion dependant participants receiving first line therapy		From week 1-24 post date of treatment initiation
Modified Hematologic Improvement - Erythroid (mHI-E) of transfusion dependant participants receiving first line therapy		From week 1-24 post date of treatment initiation
Hematologic Improvement - Erythroid (HI-E) response of non-transfusion dependant participants receiving first line therapy		From week 1-24 and week 1-48 post date of treatment initiation
Modified Hematologic Improvement - Erythroid (mHI-E) of non-transfusion dependant participants receiving first line therapy		From week 1-24 and week 1-48 post date of treatment initiation
Time to Hematologic Improvement - Erythroid (HI-E) or Modified hematologic improvement - Erythroid (mHI-E) during first-line therapy		From week 1-24 and week 1-48 post date of treatment initiation
Duration of Hematologic Improvement - Erythroid (HI-E) or Modified hematologic improvement - Erythroid (mHI-E) during first-line therapy		From date of treatment initiation to date of treatment discontinuation, up 5-years
Duration of red-blood call transfusion independence of ≥12 weeks for transfusion dependant participants receiving first line therapy		From date of treatment initiation to date of treatment discontinuation, up 5-years
Number of participants diagnosed with Acute Myeloid Leukemia		Date of treatment initiation, up to 5-years
Time to Acute Myeloid Leukemia diagnosis		Date of treatment initiation, up to 5-years
Overall survival (OS) of participants		1, 2, 3 and 5-years post treatment initiation
Number of red-blood cell transfusion days per participant		Date of treatment initiation, up to 5-years
Number of red-blood cell units used per transfusion		Date of treatment initiation, up to 5-years
Number of outpatient visits per month		Date of treatment initiation, up to 5-years
Number of hospital admissions		Date of treatment initiation, up to 5-years
Length of hospitalization		Date of treatment initiation, up to 5-years
Reason for hospitalization		Date of treatment initiation, up to 5-years
Number of emergency department visits		Date of treatment initiation, up to 5-years
Number of Intensive care unit admissions (ICU)		Date of treatment initiation, up to 5-years
Number of outpatient/inpatient medication claims		Date of treatment initiation, up to 5-years
Supplemental medication use: Iron chelation use		Date of treatment initiation, up to 5-years
Supplemental medication use: Granulocyte-colony stimulating factors (G-CSF)		Date of treatment initiation, up to 5-years
Number of hematology tests ordered		Date of treatment initiation, up to 5-years
Medical costs	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	Date of treatment initiation, up to 5-years

Collaborators and Investigators

• Sponsor: Bristol-Myers Squibb

Publications and helpful links

Helpful Links

• BMS Clinical Trial Information
• FDA Safety Alerts and Recalls

Study record dates

Study Major Dates

• Study Start (Actual): December 22, 2023
• Primary Completion (Actual): July 9, 2025
• Study Completion (Actual): July 9, 2025

Study Registration Dates

• First Submitted: March 1, 2024
• First Submitted That Met QC Criteria: March 1, 2024
• First Posted (Actual): March 7, 2024

Study Record Updates

• Last Update Posted (Estimated): December 4, 2025
• Last Update Submitted That Met QC Criteria: November 27, 2025
• Last Verified: November 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms; Disease; Bone Marrow Diseases; Hematologic Diseases; Precancerous Conditions; Syndrome; Myelodysplastic Syndromes; Preleukemia; Therapeutics; Biological Therapy; Blood Transfusion; Blood Component Transfusion; Erythrocyte Transfusion
• Other Study ID Numbers: CA056-1094

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No