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Real-World data reveals Luspatercept's impact on MDS patients

NCT ID NCT06851065

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study looked back at medical records of 418 patients with lower-risk myelodysplastic syndromes (MDS) who had not previously used erythropoiesis-stimulating agents (ESAs). Researchers examined how luspatercept was used in everyday practice, including dosing, treatment changes, and outcomes like blood transfusion needs and disease progression. The goal was to understand the real-world effectiveness of luspatercept in this patient group.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Luspatercept
What this could lead to
If results are positive, this could help doctors understand how well luspatercept works in everyday practice for MDS patients.
What could go wrong
This is a retrospective chart review, not a controlled trial, so it cannot prove cause and effect. Results may not apply to all patients.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

418 people

The number who actually took part.

Started

Aug 2024

Finished

Mar 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

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

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

myelodysplastic syndrome Myelodysplastic Syndromes

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • RTI Health Solutions

    Raleigh, North Carolina, 27709-2194, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.