Luspatercept in Preventing Poor Erythroid Engraftment for Hematological Malignancies With Moderate to Severe Myelofibrosis

April 13, 2026 updated by: Nanfang Hospital, Southern Medical University

The Efficacy and Safety of Luspatercept in Preventing Poor Erythroid Engraftment After Allo-HSCT for Hematological Malignancies With Moderate to Severe Myelofibrosis: A Prospective, Multicenter, Randomized Controlled Study

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an important treatment for hematological malignancies. Poor erythroid engraftment after transplantation is a serious complication, especially in patients with moderate to severe myelofibrosis (MF). Currently, there is a lack of effective prevention strategies for poor erythroid engraftment after transplantation. Luspatercept, a novel TGF-β superfamily signaling pathway modulator, has shown potential in small-sample studies for the treatment and prevention of post-transplant anemia. Given the high proportion and poor prognosis of poor engraftment function in hematological malignancies with moderate to severe myelofibrosis after transplantation, we plan to conduct a prospective, multicenter, randomized controlled study to explore the efficacy and safety of luspatercept in preventing poor erythroid engraftment after allo-HSCT in hematological malignancies with moderate to severe myelofibrosis.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Detailed Description

Exploring appropriate prevention strategies for poor erythroid engraftment during transplantation is not only a significant scientific issue but also a major clinical problem. Luspatercept is a recombinant fusion protein that can target and regulate the signaling pathway of the transforming growth factor-β (TGF-β) superfamily. By binding to multiple TGF-β superfamily ligands, it weakens the Smad2/3 signaling pathway, thereby promoting the maturation and generation of red blood cells. In recent years, some small-sample studies have explored the efficacy and safety of luspatercept for preventive treatment in patients with hematological malignancies who received allo-HSCT, showing certain efficacy. However, these studies were retrospective and requires large-sample, prospective, randomized controlled studies for further verification. Currently, there are no prospective studies on preventive treatment for poor erythroid engraftment after allo-HSCT in patients with hematological malignancies.

The presence of fibrosis in the bone marrow before transplantation (MF), especially moderate/severe MF, is one of the main causes of post-transplant PGF. A small-sample clinical trial in our center previously showed that in patients with MDS/MPN and acute leukemia with MF grade 2/3, applying luspatercept at +7 and +21 days after transplantation could reduce the risk of early red cell transfusion after transplantation, and showed good safety. Based on the current research status and our previous studies, we plan to conduct a prospective, multicenter, randomized controlled study to explore the efficacy and safety of luspatercept in preventing poor erythroid engraftment after allo-HSCT in patients with hematological malignancies accompanied by moderate/severe MF.

Study Type

Interventional

Enrollment (Estimated)

196

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • China
    • Guangdong
      • Guangzhou, Guangdong, China, 510515
        • Nanfang Hospital, Southern Medical University
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Age 18-65 years old, gender not restricted;
  • ECOG score 0-2 points;
  • Hematological malignancies with moderate to severe myelofibrosis
  • Willing to undergo the first allo-HSCT with a suitable donor
  • In a CR state before transplantation.

Exclusion Criteria:

  • Has previously undergone allo-HSCT;
  • ECOG score is 3-5;
  • Expected lifespan after transplantation is less than 30 days;
  • Has severe cardiac dysfunction, severe arrhythmia or severe pulmonary dysfunction (obstructive and/or restrictive ventilation disorder);
  • Has severe liver dysfunction, with liver function indicators (aspartate aminotransferase (AST), alanine aminotransferase (ALT), total bilirubin (TBIL)) more than twice the upper limit of normal;
  • Has severe renal dysfunction, with creatinine (Cr) more than twice the upper limit of normal or 24-hour creatinine clearance rate (Ccr) lower than 30 ml/min;
  • Has severe active bleeding;
  • Patients judged by the investigator to be unsuitable for participating in this trial.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Luspatercept group
Luspatercept
Drug: Luspatercept
On the 7th day after allo-HSCT, the first dose of Luspatercept 1.0mg/kg was administered subcutaneously. If the peripheral blood HGB was < 70g/L on the 21st day after allo-HSCT, the second dose of Luspatercept 1.0mg/kg was given subcutaneously; if the peripheral blood HGB was ≥ 70g/L on the 21st day after allo-HSCT, no second dose of Luspatercept subcutaneous injection was given.
Other: Control group
Control
Other: Control
The patient will receive the best supportive treatment including blood transfusion.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Cumulative incidence of poor erythroid engraftment
Time Frame: 28 days
Poor erythroid engraftment after allo-HSCT is defined as HGB < 70g/L at 28 days post-transplantation and the inability to discontinue red blood cell transfusion in the case of complete donor engraftment.
28 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Red blood cell units
Time Frame: 28 days
The number of red blood cell units administered
28 days
Overall survival
Time Frame: 1 year
Will calculate time from random assignment until death from any cause.
1 year
Disease-free survival
Time Frame: 1 year
Will calculate time from random assignment until relapse or death from any cause
1 year
Cumulative rate of poor graft engraftment
Time Frame: 1 year
The definition of PGF is as follows: More than 28 days after transplantation, there is at least a continuous period of 3 days of two-line or three-line blood cell reduction (absolute neutrophil count < 0.5 × 109/L, platelet (PLT) < 20 × 109/L, hemoglobin (HGB) < 70 g/L), and blood transfusion support treatment is required; bone marrow examination indicates a low degree of bone marrow hyperplasia; complete donor engraftment; no active severe GVHD or hematological recurrence.
1 year
Relapse
Time Frame: 1 year
Will calculate time from random assignment until relapse
1 year
Non-relapse mortality
Time Frame: 1 year
Defined as death from any cause not subsequent to relapse
1 year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Nanfang Hospital, Southern Medical University

Investigators

  • Principal Investigator: Qifa Liu, Nanfang Hospital, Southern Medical University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

May 1, 2026

Primary Completion (Estimated)

January 1, 2030

Study Completion (Estimated)

December 31, 2030

Study Registration Dates

First Submitted

April 13, 2026

First Submitted That Met QC Criteria

April 13, 2026

First Posted (Actual)

April 20, 2026

Study Record Updates

Last Update Posted (Actual)

April 20, 2026

Last Update Submitted That Met QC Criteria

April 13, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NFEC-2026-016

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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