Luspatercept vs Epoetin in Treating Poor Erythroid Engraftment for Hematological Malignancies

NCT07636486 | Not Yet Recruiting Phase 2 DMC

• 1 other identifier: NFEC-2026-015
• Study Type: interventional
• Target: 90
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Luspatercept; Epoetin; Hematological Malignancies; Poor Erythroid Engraftment

Outcome Measures

Primary Outcomes (1)

• Erythroid response

  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

  24 weeks

Secondary Outcomes (4)

• Overall survival

  1 year

• Disease-free survival

  1 year

• Relapse

  1 year

• Non-relapse mortality

  1 year

Study Arms (2)

Luspatercept group

EXPERIMENTAL

Luspatercept

Drug: Luspatercept

Epoetin group

ACTIVE COMPARATOR

Epoetin

Drug: Epoetin

Interventions

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.

Luspatercept group

Epoetin DRUG

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

Epoetin group

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• Nanfang Hospital, Southern Medical University: lead

Study Sites (1)

Department of Hematology, Nanfang Hospital, Southern Medical University

Guangzhou, Guangdong, 510515, China

Location

Related Publications (5)

• Della Porta MG, Garcia-Manero G, Santini V, Zeidan AM, Komrokji RS, Shortt J, Valcarcel D, Jonasova A, Dimicoli-Salazar S, Tiong IS, Lin CC, Li J, Zhang J, Pilot R, Kreitz S, Pozharskaya V, Keeperman KL, Rose S, Prebet T, Lai Y, Degulys A, Paolini S, Cluzeau T, Fenaux P, Platzbecker U. Luspatercept versus epoetin alfa in erythropoiesis-stimulating agent-naive, transfusion-dependent, lower-risk myelodysplastic syndromes (COMMANDS): primary analysis of a phase 3, open-label, randomised, controlled trial. Lancet Haematol. 2024 Sep;11(9):e646-e658. doi: 10.1016/S2352-3026(24)00203-5. Epub 2024 Jul 19.

  PMID: 39038479 BACKGROUND

• Tang C, Chen F, Kong D, Ma Q, Dai H, Yin J, Li Z, Chen J, Zhu X, Mao X, Wu D, Tang X. Successful treatment of secondary poor graft function post allogeneic hematopoietic stem cell transplantation with eltrombopag. J Hematol Oncol. 2018 Aug 16;11(1):103. doi: 10.1186/s13045-018-0649-6.

  PMID: 30115080 BACKGROUND

• Markham A. Luspatercept: First Approval. Drugs. 2020 Jan;80(1):85-90. doi: 10.1007/s40265-019-01251-5.

  PMID: 31939073 BACKGROUND

• Zhu L, Liu J, Liu H, et al. Clinical Study on the Treatment of Poor Erythroid Engraftment after Allogeneic Hematopoietic Stem Cell Transplantation with Luspatercept. Blood 2024; 144: 2159.

  BACKGROUND

• Xin X, Zhang W, Li Z, Gui R, Wang J, Ji L, Zhang Y, Fang B, Song Y, Zu Y, Zhou J. Luspatercept for the treatment of anemia in allo-HSCT for patients with hematological diseases. Blood Cancer J. 2025 Feb 5;15(1):12. doi: 10.1038/s41408-025-01218-8. No abstract available.

  PMID: 39910033 BACKGROUND

MeSH Terms

Conditions

Hematologic Neoplasms

Interventions

luspatercept; Erythropoietin

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Study Officials

• Qifa Liu

  Nanfang Hospital, Southern Medical University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Li Xuan

CONTACT

+86-020-61641613; 356135708@qq.com

Qifa Liu

CONTACT

+86-020-62787883; liuqifa628@163.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 4, 2026
• First Posted: June 9, 2026
• Study Start: June 15, 2026
• Primary Completion (Estimated): June 30, 2028
• Study Completion (Estimated): December 31, 2030
• Last Updated: June 9, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)