Lower-risk Myelodysplastic SyndromesNCT06298643
Real-World Practice Patterns and Outcomes of Lower-Risk Myelodysplastic Syndrome Patients in Japan
An observational study of Lower-risk Myelodysplastic Syndromes, run by Bristol-Myers Squibb at 1 site in Japan.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Lower-risk Myelodysplastic Syndromes
- What is being tested?
- Red blood cell transfusion
- Type of treatment
- Surgery or procedure
- How many people
- 177 participants
- Where
- 1 siteJapan
- Study dates
- 22 Dec 2023to 9 Jul 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Adult, Older adult
Study population: Adult participants with myelodysplastic syndrome in Japan
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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
- Being tested
- Red blood cell transfusion
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Red blood cell transfusionProcedure or surgery
≥2 units of red blood cell transfusion
Red blood cell transfusionProcedure or surgery
0 units or <2 units of red blood cell transfusion
| Study group | Role | What they receive |
|---|---|---|
| Transfusion dependent | Participants that received ≥2 units of red blood cell transfusion during the 8 weeks after initial myelodysplastic syndrome diagnosis date. Receives: 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. Receives: Procedure: Red blood cell transfusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Participant age
Measured: At date of diagnosis
Participant sex
Measured: At date of diagnosis
Participant weight
Measured: At date of diagnosis
Participant height
Measured: At date of diagnosis
Participant comorbidities calculated using the Charlson Comorbidity Index (CCI) method
Measured: From 30 days prior to diagnosis up to 7 days post to diagnosis
Participant Eastern Cooperative Oncology Group (ECOG) score
Measured: 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
Measured: Date of diagnosis, 6-months and 12 months post-diagnosis
Percentage of ring sideroblasts present in participants
Measured: 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
Measured: From 30 days prior to diagnosis up to 7 days post to diagnosis
Histopathology results
Measured: From 30 days prior to diagnosis up to 7 days post to diagnosis
Number of red-blood cell transfusions received by participants
Measured: Up to 56 days post diagnosis
Treatment prescribed by line of therapy
Measured: From date of diagnosis, up to 5-years
Duration of treatment by line of therapy
Measured: From date of diagnosis, up to 5-years
Time between date of diagnosis and treatment
Measured: From date of diagnosis to treatment initiation
Time between date of treatment initiation to treatment discontinuation
Measured: Date of treatment initiation, up to 5-years
Time between date of diagnosis to date of first red-blood cell transfusion
Measured: 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
Measured: From week 1-24 and week 1-48 post date of treatment initiation
Mean hemoglobin change of participants during first-line of therapy
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: From week 1-24 post date of treatment initiation
Modified Hematologic Improvement - Erythroid (mHI-E) of transfusion dependant participants receiving first line therapy
Measured: From week 1-24 post date of treatment initiation
Hematologic Improvement - Erythroid (HI-E) response of non-transfusion dependant participants receiving first line therapy
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: From date of treatment initiation to date of treatment discontinuation, up 5-years
Number of participants diagnosed with Acute Myeloid Leukemia
Measured: Date of treatment initiation, up to 5-years
Time to Acute Myeloid Leukemia diagnosis
Measured: Date of treatment initiation, up to 5-years
Overall survival (OS) of participants
Measured: 1, 2, 3 and 5-years post treatment initiation
Number of red-blood cell transfusion days per participant
Measured: Date of treatment initiation, up to 5-years
Number of red-blood cell units used per transfusion
Measured: Date of treatment initiation, up to 5-years
Number of outpatient visits per month
Measured: Date of treatment initiation, up to 5-years
Number of hospital admissions
Measured: Date of treatment initiation, up to 5-years
Length of hospitalization
Measured: Date of treatment initiation, up to 5-years
Reason for hospitalization
Measured: Date of treatment initiation, up to 5-years
Number of emergency department visits
Measured: Date of treatment initiation, up to 5-years
Number of Intensive care unit admissions (ICU)
Measured: Date of treatment initiation, up to 5-years
Number of outpatient/inpatient medication claims
Measured: Date of treatment initiation, up to 5-years
Supplemental medication use: Iron chelation use
Measured: Date of treatment initiation, up to 5-years
Supplemental medication use: Granulocyte-colony stimulating factors (G-CSF)
Measured: Date of treatment initiation, up to 5-years
Number of hematology tests ordered
Measured: 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
Measured: Date of treatment initiation, up to 5-years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2024
- Study start22 Dec 2023
- Primary completion9 Jul 2025
- Study completion9 Jul 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lower-risk Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by Bristol-Myers SquibbThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 177 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bristol-Myers Squibb
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06298643
- Sponsor's protocol ID
- CA056-1094
- Status last verified
- Nov 2025
- First submitted
- 1 Mar 2024
- Last updated
- 4 Dec 2025
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Real-World Practice Patterns and Outcomes of Lower-Risk Myelodysplastic Syndrome Patients in Japan
- ClinicalTrials.gov ID
- NCT06298643
- Acronym / sponsor ID
- CA056-1094
- Conditions
- Lower-risk Myelodysplastic Syndromes
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 177 participants (actual)
- Lead sponsor
- Bristol-Myers SquibbIndustry (pharma, biotech, devices)
- Study start
- 22 Dec 2023 (actual)
- Primary completion
- 9 Jul 2025 (actual)
- Study completion
- 9 Jul 2025 (actual)
- First posted
- 7 Mar 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Transfusion dependent | – | Procedure: Red blood cell transfusionParticipants that received ≥2 units of red blood cell transfusion during the 8 weeks after initial myelodysplastic syndrome diagnosis date. |
| Non-transfusion dependent | – | Procedure: Red blood cell transfusionParticipants that received 0 units or <2 units of red blood cell transfusion during the 8 weeks after initial myelodysplastic syndrome diagnosis date. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
- Study population
- Adult participants with myelodysplastic syndrome in Japan
Key inclusion criteria (6 listed)
- 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:
- 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
Key exclusion criteria (3 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Red blood cell transfusion≥2 units of red blood cell transfusion | Procedure or surgery | Transfusion dependent |
| Red blood cell transfusion0 units or <2 units of red blood cell transfusion | Procedure or surgery | Non-transfusion dependent |
Primary outcome
Participant age
Time frame: At date of diagnosis
Participant sex
Time frame: At date of diagnosis
Participant weight
Time frame: At date of diagnosis
Participant height
Time frame: At date of diagnosis
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
Participant Eastern Cooperative Oncology Group (ECOG) score
Time frame: 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
Time frame: Date of diagnosis, 6-months and 12 months post-diagnosis
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
Percentage of bone marrow blasts present in participants
Time frame: From 30 days prior to diagnosis up to 7 days post to diagnosis
Histopathology results
Time frame: From 30 days prior to diagnosis up to 7 days post to diagnosis
Number of red-blood cell transfusions received by participants
Time frame: Up to 56 days post diagnosis
Treatment prescribed by line of therapy
Time frame: From date of diagnosis, up to 5-years
Duration of treatment by line of therapy
Time frame: From date of diagnosis, up to 5-years
Time between date of diagnosis and treatment
Time frame: From date of diagnosis to treatment initiation
Time between date of treatment initiation to treatment discontinuation
Time frame: Date of treatment initiation, up to 5-years
Time between date of diagnosis to date of first red-blood cell transfusion
Time frame: Date of diagnosis, up to 5-years
28 secondary outcomes
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
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
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
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
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
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
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
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
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
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
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
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
Number of participants diagnosed with Acute Myeloid Leukemia
Time frame: Date of treatment initiation, up to 5-years
Time to Acute Myeloid Leukemia diagnosis
Time frame: Date of treatment initiation, up to 5-years
Overall survival (OS) of participants
Time frame: 1, 2, 3 and 5-years post treatment initiation
Number of red-blood cell transfusion days per participant
Time frame: Date of treatment initiation, up to 5-years
Number of red-blood cell units used per transfusion
Time frame: Date of treatment initiation, up to 5-years
Number of outpatient visits per month
Time frame: Date of treatment initiation, up to 5-years
Number of hospital admissions
Time frame: Date of treatment initiation, up to 5-years
Length of hospitalization
Time frame: Date of treatment initiation, up to 5-years
Reason for hospitalization
Time frame: Date of treatment initiation, up to 5-years
Number of emergency department visits
Time frame: Date of treatment initiation, up to 5-years
Number of Intensive care unit admissions (ICU)
Time frame: Date of treatment initiation, up to 5-years
Number of outpatient/inpatient medication claims
Time frame: Date of treatment initiation, up to 5-years
Supplemental medication use: Iron chelation use
Time frame: Date of treatment initiation, up to 5-years
Supplemental medication use: Granulocyte-colony stimulating factors (G-CSF)
Time frame: Date of treatment initiation, up to 5-years
Number of hematology tests ordered
Time frame: Date of treatment initiation, up to 5-years
Medical costs
Time frame: Date of treatment initiation, up to 5-years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Dec 2025.
- BMS Clinical Trial InformationLink provided by the sponsor.
- FDA Safety Alerts and RecallsLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Lower-risk Myelodysplastic Syndromes, run by Bristol-Myers Squibb at 1 site in Japan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Participant age (time frame: At date of diagnosis). The study lists 16 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Dec 2025.