Clinical trial NCT06298643: Real-World Practice Patterns and Outcomes of Lower-Risk Myelodysplastic Syndrome Patients in Japan
Official title: Real-World Practice Patterns and Outcomes of Lower-Risk Myelodysplastic Syndrome Patients in Japan
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Lower-risk Myelodysplastic Syndromes
Lead sponsor: Bristol-Myers Squibb
Sponsor class: INDUSTRY
First posted: 2024-03-07
Enrollment: 300
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adult participants with myelodysplastic syndrome in Japan

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.


Eligibility criteria: 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

Primary outcomes:
measure: Participant age
timeFrame: At date of diagnosis
measure: Participant sex
timeFrame: At date of diagnosis
measure: Participant weight
timeFrame: At date of diagnosis
measure: Participant height
timeFrame: At date of diagnosis
measure: Participant comorbidities calculated using the Charlson Comorbidity Index (CCI) method
timeFrame: From 30 days prior to diagnosis up to 7 days post to diagnosis
measure: Participant Eastern Cooperative Oncology Group (ECOG) score
timeFrame: From 30 days prior to diagnosis up to 7 days post to diagnosis
measure: Participant International Prognostic Scoring System (IPSS) or Revised International Prognostic Scoring System (IPSS-R) risk category
timeFrame: Date of diagnosis, 6-months and 12 months post-diagnosis
measure: Percentage of ring sideroblasts present in participants
timeFrame: Date of bone marrow test; from 30 days prior to diagnosis up to 7 days post to diagnosis
measure: Percentage of bone marrow blasts present in participants
timeFrame: From 30 days prior to diagnosis up to 7 days post to diagnosis
measure: Histopathology results
timeFrame: From 30 days prior to diagnosis up to 7 days post to diagnosis
measure: Number of red-blood cell transfusions received by participants
timeFrame: Up to 56 days post diagnosis
measure: Treatment prescribed by line of therapy
timeFrame: From date of diagnosis, up to 5-years
measure: Duration of treatment by line of therapy
timeFrame: From date of diagnosis, up to 5-years
measure: Time between date of diagnosis and treatment
timeFrame: From date of diagnosis to treatment initiation
measure: Time between date of treatment initiation to treatment discontinuation
timeFrame: Date of treatment initiation, up to 5-years
measure: Time between date of diagnosis to date of first red-blood cell transfusion
timeFrame: Date of diagnosis, up to 5-years
Secondary outcomes:
measure: Number of participants that achieved red-blood cell transfusion independence during first-line of therapy treatment
timeFrame: From week 1-24 and week 1-48 post date of treatment initiation
measure: Mean hemoglobin change of participants during first-line of therapy
timeFrame: From week 1-24 and week 1-48 post date of treatment initiation
measure: 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
timeFrame: Week 1-24 post treatment initiation
measure: Number of non-transfusion dependant participants achieving red-blood cell transfusion independence during first-line therapy for ≥24, 48 and 72 weeks
timeFrame: Week 24, 48 and 72 post date of initiation of treatment
measure: Time to red-blood call transfusion independence of ≥12 weeks for transfusion dependant participants receiving first line therapy
timeFrame: From week 1-24 and week 1-48 post date of treatment initiation
measure: Hematologic Improvement - Erythroid (HI-E) response of transfusion dependant participants receiving first line therapy
timeFrame: From week 1-24 post date of treatment initiation
measure: Modified Hematologic Improvement - Erythroid (mHI-E) of transfusion dependant participants receiving first line therapy
timeFrame: From week 1-24 post date of treatment initiation
measure: Hematologic Improvement - Erythroid (HI-E) response of non-transfusion dependant participants receiving first line therapy
timeFrame: From week 1-24 and week 1-48 post date of treatment initiation
measure: Modified Hematologic Improvement - Erythroid (mHI-E) of non-transfusion dependant participants receiving first line therapy
timeFrame: From week 1-24 and week 1-48 post date of treatment initiation
measure: Time to Hematologic Improvement - Erythroid (HI-E) or Modified hematologic improvement - Erythroid (mHI-E) during first-line therapy
timeFrame: From week 1-24 and week 1-48 post date of treatment initiation
measure: Duration of Hematologic Improvement - Erythroid (HI-E) or Modified hematologic improvement - Erythroid (mHI-E) during first-line therapy
timeFrame: From date of treatment initiation to date of treatment discontinuation, up 5-years
measure: Duration of red-blood call transfusion independence of ≥12 weeks for transfusion dependant participants receiving first line therapy
timeFrame: From date of treatment initiation to date of treatment discontinuation, up 5-years
measure: Number of participants diagnosed with Acute Myeloid Leukemia
timeFrame: Date of treatment initiation, up to 5-years
measure: Time to Acute Myeloid Leukemia diagnosis
timeFrame: Date of treatment initiation, up to 5-years
measure: Overall survival (OS) of participants
timeFrame: 1, 2, 3 and 5-years post treatment initiation
measure: Number of red-blood cell transfusion days per participant
timeFrame: Date of treatment initiation, up to 5-years
measure: Number of red-blood cell units used per transfusion
timeFrame: Date of treatment initiation, up to 5-years
measure: Number of outpatient visits per month
timeFrame: Date of treatment initiation, up to 5-years
measure: Number of hospital admissions
timeFrame: Date of treatment initiation, up to 5-years
measure: Length of hospitalization
timeFrame: Date of treatment initiation, up to 5-years
measure: Reason for hospitalization
timeFrame: Date of treatment initiation, up to 5-years
measure: Number of emergency department visits
timeFrame: Date of treatment initiation, up to 5-years
measure: Number of Intensive care unit admissions (ICU)
timeFrame: Date of treatment initiation, up to 5-years
measure: Number of outpatient/inpatient medication claims
timeFrame: Date of treatment initiation, up to 5-years
measure: Supplemental medication use: Iron chelation use
timeFrame: Date of treatment initiation, up to 5-years
measure: Supplemental medication use: Granulocyte-colony stimulating factors (G-CSF)
timeFrame: Date of treatment initiation, up to 5-years
measure: Number of hematology tests ordered
timeFrame: Date of treatment initiation, up to 5-years
measure: Medical costs
timeFrame: Date of treatment initiation, up to 5-years
Other outcomes:
Locations:
city: Tokyo
country: Japan
facility: Mebix, Inc
state: Minato-ku
Study officials:
References: