Real-World Practice Patterns and Outcomes of Lower-Risk Myelodysplastic Syndrome Patients in Japan
Active, not recruiting
- Conditions
- Lower-risk Myelodysplastic Syndromes
- Interventions
- Procedure: Red blood cell transfusion
- Registration Number
- NCT06298643
- Lead Sponsor
- Bristol-Myers Squibb
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 300
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 & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Non-transfusion dependent Red blood cell transfusion Participants that received 0 units or \<2 units of red blood cell transfusion during the 8 weeks after initial myelodysplastic syndrome diagnosis date. Transfusion dependent Red blood cell transfusion Participants that received ≥2 units of red blood cell transfusion during the 8 weeks after initial myelodysplastic syndrome diagnosis date.
- Primary Outcome Measures
Name Time Method Participant age 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 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 Participant weight At date of diagnosis Participant height At date of diagnosis Treatment prescribed by line of therapy From date of diagnosis, up to 5-years Time between date of treatment initiation to treatment discontinuation Date of treatment initiation, up to 5-years 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 Participant sex At date of diagnosis Participant Eastern Cooperative Oncology Group (ECOG) score From 30 days prior to diagnosis up to 7 days post to 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 Time between date of diagnosis to date of first red-blood cell transfusion Date of diagnosis, up to 5-years Number of red-blood cell transfusions received by participants Up to 56 days post diagnosis Time between date of diagnosis and treatment From date of diagnosis to treatment initiation Duration of treatment by line of therapy From date of diagnosis, up to 5-years
- Secondary Outcome Measures
Name Time Method Overall survival (OS) of participants 1, 2, 3 and 5-years post treatment initiation 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 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 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 Number of hospital admissions Date of treatment initiation, up to 5-years Length of hospitalization Date of treatment initiation, up to 5-years 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 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 Number of participants diagnosed with Acute Myeloid Leukemia 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 Reason for hospitalization 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 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 Number of emergency department visits Date of treatment initiation, up to 5-years Medical costs 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
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 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 outpatient visits per month Date of treatment initiation, up to 5-years Supplemental medication use: Iron chelation use Date of treatment initiation, up to 5-years Mean hemoglobin change of participants during first-line of 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 Acute Myeloid Leukemia diagnosis Date of treatment initiation, up to 5-years Number of red-blood cell transfusion days per participant 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
Trial Locations
- Locations (1)
Mebix, Inc
🇯🇵Tokyo, Minato-ku, Japan