Clinical trial NCT06851065: A Study to Evaluate Luspatercept Treatment Patterns and Outcomes in Erythropoiesis-Stimulating Agents-Naïve Patients With Lower-Risk Myelodysplastic Syndromes in the United States
Official title: Luspatercept Treatment Patterns and Outcomes Among ESA-Naïve Patients With Lower-Risk MDS - A Retrospective Medical Record Review in the United States
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Myelodysplastic Syndromes
Keywords: Lower-risk myelodysplastic syndromes (LR-MDS)
Lead sponsor: Bristol-Myers Squibb
Sponsor class: INDUSTRY
First posted: 2025-02-28
Enrollment: 400
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study population will include adults in the United States with a clinician-confirmed diagnosis of primary or secondary lower-risk myelodysplastic syndromes (LR-MDS) who initiated first-line luspatercept treatment on or after LR-MDS diagnosis date between 28 August 2023 to 31 July 2024

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


Eligibility criteria: 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

Primary outcomes:
measure: Participant baseline demographics
timeFrame: Baseline
measure: Participant baseline clinical characteristics
timeFrame: Baseline
measure: Time from Lower Risk- myelodysplastic syndromes diagnosis to index treatment initiation
timeFrame: Baseline
measure: Rationale for therapy selection
timeFrame: Baseline
measure: Duration of index treatment
timeFrame: Up to 15 months
measure: Treatment dose at treatment initiation and discontinuation
timeFrame: Up to 6 months
measure: Treatment dose/dosing schedule changes, and treatment interruptions
timeFrame: Up to 12 months
measure: Other supportive care therapies prescribed while on index treatment
timeFrame: Up to 15 months
measure: Treatments prescribed post index treatment
timeFrame: Up to 15 months
measure: Treatments for anemia management received after discontinuing the index treatment
timeFrame: Up to 15 months
measure: Receipt of stem cell transplant at any time post index treatment
timeFrame: Up to 15 months
measure: Participant red-blood cell (RBC) transfusion burden post index treatment
timeFrame: At 3-months, and up to 6 months
measure: Hematologic improvement-erythroid (HI-E) response post index treatment
timeFrame: At 3-months, and up to 6 months
measure: Progression to acute myeloid leukemia post index treatment
timeFrame: Up to 15 months
measure: Progression to high-risk myelodysplastic syndromes per the International Prognostic Scoring System (IPSS) or its revised version (IPSS-R) criteria
timeFrame: Up to 15 months
measure: Participant adverse events during and post index treatment
timeFrame: Up to 15 months
measure: Overall survival (OS)
timeFrame: At 3-, 6-, 12-, and up to 15-months
measure: Healthcare resource utilization (HCRU) during index treatment
timeFrame: Up to 15 months
Secondary outcomes:
Other outcomes:
Locations:
city: Raleigh
country: United States
facility: RTI Health Solutions
state: North Carolina
Study officials:
affiliation: Bristol-Myers Squibb
name: Bristol Myers Squibb
role: STUDY_DIRECTOR
References: