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NCT Number: NCT07636486

Luspatercept vs Epoetin in Treating Poor Erythroid Engraftment for Hematological Malignancies

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective therapy for hematological malignancies. Nonetheless, poor graft function remains a life-threatening complication after allo-HSCT. Poor erythroid engraftment is associated with increased bleeding events and shorter survival. Current treatment methods such as epoetin or repeated red-cell transfusions are not effective for poor erythroid engraftment, with limited and transient responses. Retrospective studies suggested that luspatercept showed efficacy in patients with anemia post-transplantation or poor erythroid engraftment. However, there are no studies comparing luspatercept versus epoetin for the treatment of poor erythroid engraftment. Therefore, we conducted a randomized controlled study to compared the effect of luspatercept versus epoetin in treating poor erythroid engraftment for hematological malignancies.

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Key information

Conditions

Luspatercept Epoetin Hematologic Diseases Hematologic Neoplasms Hematological Malignancies Hemic and Lymphatic Diseases Neoplasms Neoplasms by Site Poor Erythroid Engraftment

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

About this study

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is an effective therapy for hematological malignancies. Nonetheless, poor graft function remains a life-threatening complication after allo-HSCT, characterized by persistent cytopenias despite evidence of complete donor chimerism. Poor erythroid engraftment refers to hemoglobin <70g/L and inability to detach from red blood cell transfusion after 28 days of transplantation, which is associated with increased bleeding events and shorter survival. Current treatment methods such as epoetin or repeated red-cell transfusions are not effective for poor erythroid engraftment, with limited and transient responses. New treatment strategies are needed to enhance the response rate in patients with poor erythroid engraftment.

Luspatercept is a specific activin receptor fusion protein that reduces SMAD2 and SMAD3 signaling by binding specific transforming growth factor β (TGF-β) superfamily ligands, thereby allowing erythrocyte maturation through late-stage erythroblast differentiation. Retrospective studies suggested that luspatercept showed efficacy in patients with anemia post-transplantation or poor erythroid engraftment. To date, there have been no studies comparing luspatercept versus epoetin for the treatment of poor erythroid engraftment. Therefore, we conducted a randomized controlled study to compared the effect of luspatercept versus epoetin in treating poor erythroid engraftment for hematological malignancies.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 18-65 years
  • Hematologic malignancies
  • Poor erythroid engraftment after the first allo-HSCT
  • Complete remission post-transplantation
  • Eastern Cooperative Oncology Group performance status of 0-2
  • Epoetin-naive
  • Endogenous serum erythropoietin concentration <500 U/L

Exclusion criteria

  • Life expectancy shorter than 30 days post-transplantation
  • Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)
  • Patients with any conditions not suitable for the trial (investigators' decision)

Treatment and study plan

Luspatercept

Drug

Luspatercept is administered 1.0mg/kg subcutaneously every 3 weeks; If the hemoglobin level does not increase after two consecutive administrations, the dose will be adjusted to 1.3mg/kg. If the hemoglobin level returns to the normal range, Luspatercept will be given once before discontinuing the medication.

Epoetin

Drug

Epoetin is administered 15000 IU subcutaneously every 3 weeks for 24 weeks

Primary outcomes

  1. Erythroid response

    Time frame: 24 weeks

    Erythroid response is defined as a reduction in transfusion of ≥ 4 red blood cell units/8 weeks or a mean hemoglobin increase of ≥ 1.5 g/dL/8 weeks in the absence of transfusion

Secondary outcomes

  1. Overall survival

    Time frame: 1 year

    Will calculate time from random assignment until death from any cause.

  2. Disease-free survival

    Time frame: 1 year

    Will calculate time from random assignment until relapse or death from any cause

  3. Relapse

    Time frame: 1 year

    Will calculate time from random assignment until relapse

  4. Non-relapse mortality

    Time frame: 1 year

    Defined as death from any cause not subsequent to relapse

Study contacts

Contact information is provided by the study sponsor or research team.

Li Xuan

CONTACT

[email protected]

+86-020-61641613

Qifa Liu

CONTACT

[email protected]

+86-020-62787883

Sponsors and collaborators

Lead sponsor

Nanfang Hospital, Southern Medical University

Other

Registry information

Official study title

Luspatercept Versus Epoetin in Treating Poor Erythroid Engraftment for Hematological Malignancies

Important dates

Study start
2026
Primary completion
2028
Study completion
2030
First posted
Jun 9, 2026
Registry last updated
Jun 9, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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