Lower-risk Myelodysplastic Syndromes
Conditions
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.
Interventions
PROCEDURERed blood cell transfusion
≥2 units of red blood cell transfusion
Sponsors
Bristol-Myers Squibb
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
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
Design outcomes
Primary
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 | — |
| Supplemental medication use: Iron chelation use | 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 |
| 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 | — |
| Number of outpatient/inpatient medication claims | Date of treatment initiation, up to 5-years | — |
| 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 | — |
Countries
Japan
Outcome results
None listed