Luspatercept vs Epoetin in Treating Poor Erythroid Engraftment for Hematological Malignancies
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Luspatercept, Epoetin.
- Who it may be relevant to
- Registry conditions: Luspatercept, Epoetin, Poor Erythroid Engraftment, Hematological Malignancies. Basic parameters: 18 years — 65 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Luspatercept Versus Epoetin in Treating Poor Erythroid Engraftment for Hematological Malignancies
Overview
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.
Detailed description
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.
Interventions
- Drug Luspatercept
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. - Drug Epoetin
Epoetin is administered 15000 IU subcutaneously every 3 weeks for 24 weeks
Primary outcome measures
- Erythroid response [Time frame: 24 weeks]
Secondary outcome measures (4)
- Overall survival [Time frame: 1 year]
- Disease-free survival [Time frame: 1 year]
- Relapse [Time frame: 1 year]
- Non-relapse mortality [Time frame: 1 year]
Eligibility criteria
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)
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 1 center
- Department of Hematology, Nanfang Hospital, Southern Medical University — Guangzhou
Publications
- 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 mye PMID 39038479
- 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
- Markham A. Luspatercept: First Approval. Drugs. 2020 Jan;80(1):85-90. doi: 10.1007/s40265-019-01251-5. PMID 31939073
- 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.
- 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
Identifiers
NCT: NCT07636486 · NFEC-2026-015