Myelodysplastic Syndromes
Conditions
Keywords
Lower-risk myelodysplastic syndromes (LR-MDS)
Brief summary
The purpose of this study is to understand real-world effectiveness of luspatercept treatment among erythropoiesis-stimulating agents -naïve patients with lower-risk- myelodysplastic syndromes in the United States
Interventions
DRUGLuspatercept
As per product lable
DRUGErythropoiesis-stimulating agents
As per product label
Sponsors
Bristol-Myers Squibb
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Had a documented diagnosis of primary or secondary myelodysplastic syndromes (MDS) * MDS diagnosis confirmed through bone marrow testing on (or 30 days prior to) MDS diagnosis date or within 1 year of MDS diagnosis date * Had a documented determination of Lower Risk (LR)-MDS as measured by International Prognostic Scoring System (IPSS) or its revised version (IPSS-R) at or before index treatment (i.e., first-line luspatercept or first-line erythropoiesis-stimulating agents (ESA)) initiation * IPSS risk level: low, intermediate-1 (level-1 risk) * IPSS-R risk level: very low, low, intermediate * Received luspatercept as the first-line treatment for anemia any time from 28 August 2023 to 31 July 2024 (Cohort 1) * Receipt of combination therapy with ESAs and/or granulocyte colony-stimulating factors (G-CSFs) will be allowed OR * Received ESA as the first-line treatment for anemia any time from 28 August 2023 to 31 July 2024 (Cohort 2) * Was aged 18 years or older at the time of initial diagnosis of MDS * Known vital status (i.e., living, or deceased) at the time of record abstraction. * Records for patients who are dead or alive will be eligible * Complete medical record covering relevant past medical history, diagnosis of LR-MDS, treatment, laboratory assessments, red-blood cell (RBC) transfusions, and regular monitoring for LR-MDS, including any transfer record from other physicians/facilities (if applicable) is available to the abstracting physician for data abstraction
Exclusion criteria
* Had a history of acute myeloid leukemia (AML) prior to MDS diagnosis * Received previous treatment with hypomethylating agents, disease-modifying agents (including lenalidomide), other immunosuppressants/immunomodulatory agents, or other MDS-directed chemotherapy * Received stem cell transplant prior to index treatment initiation * Participated in a clinical trial for the treatment of MDS before or while on index treatment (i.e., clinical trial participation after first-line luspatercept or ESA treatment discontinuation will be allowed) * Had evidence of other malignant neoplasms in the 12 months prior to diagnosis of MDS, except basal or squamous cell carcinoma of the skin, carcinoma in situ of the cervix, carcinoma in situ of the breast, or incidental histologic finding of prostate cancer (stage T1a or T1b) * Patients for whom this information is not available (i.e., "unknown") will be included in the study * For Cohort 1 (i.e., first-line luspatercept treatment), receipt of combination therapy with hypomethylating agents, lenalidomide, other immunosuppressants/ immunomodulatory agents, or other MDS-directed chemotherapy * For Cohort 2 (i.e., first-line ESA treatment), receipt of combination therapy with hypomethylating agents, lenalidomide, luspatercept, other immunosuppressants/ immunomodulatory agents, or other MDS-directed chemotherapy
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Participant adverse events during and post index treatment | Up to 15 months |
| Overall survival (OS) | At 3-, 6-, 12-, and up to 15-months |
| Healthcare resource utilization (HCRU) during index treatment | Up to 15 months |
| Participant baseline demographics | Baseline |
| Participant baseline clinical characteristics | Baseline |
| Time from Lower Risk- myelodysplastic syndromes diagnosis to index treatment initiation | Baseline |
| Rationale for therapy selection | Baseline |
| Duration of index treatment | Up to 15 months |
| Treatment dose at treatment initiation and discontinuation | Up to 6 months |
| Treatment dose/dosing schedule changes, and treatment interruptions | Up to 12 months |
| Other supportive care therapies prescribed while on index treatment | Up to 15 months |
| Treatments prescribed post index treatment | Up to 15 months |
| Treatments for anemia management received after discontinuing the index treatment | Up to 15 months |
| Receipt of stem cell transplant at any time post index treatment | Up to 15 months |
| Participant red-blood cell (RBC) transfusion burden post index treatment | At 3-months, and up to 6 months |
| Hematologic improvement-erythroid (HI-E) response post index treatment | At 3-months, and up to 6 months |
| Progression to acute myeloid leukemia post index treatment | Up to 15 months |
| Progression to high-risk myelodysplastic syndromes per the International Prognostic Scoring System (IPSS) or its revised version (IPSS-R) criteria | Up to 15 months |
Countries
United States
Contacts
STUDY_DIRECTORBristol Myers Squibb
Bristol-Myers Squibb
Outcome results
None listed