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This randomized trial aims at validating the efficacy and safety of low-dose decitabine for PGF post allo-HSCT.
Poor graft function (PGF), defined by the presence of multilineage cytopenias in the presence of 100% donor chimerism, is a serious complication of allogeneic stem cell transplant (allo-HSCT). Emerging evidence demonstrates that the inadequate stem cells infusion, bone marrow microenvironment and immune dysregulation play a crucial role in maintaining and regulating hematopoiesis. Current therapies remain debatable, including selected CD34+ cells infusion, mesenchymal stromal cells infusion, prophylactic N-acetyl cysteine administration, etc. Thereafter, the investigators conduct a randomized trial aiming at validating the efficacy and safety of low-dose decitabine in PGF post allo-HSCT patients.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Arm A | Experimental | Decitabine 6 mg/m2 daily subcutaneously for consecutive 3 days (day 1 to day 3) |
|
| Arm B | Active Comparator | The hematologic growth factors (granulocyte-colony stimulating factor, thrombopoietin receptor agonists, recombinant human erythropoietin) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Decitabine | Drug | Decitabine 6 mg/m2 daily subcutaneously for consecutive 3 days (day 1 to day 3) |
|
| Measure | Description | Time Frame |
|---|---|---|
| The treatment response | The rate of hematological response of after HSCT | day +28 |
| Survival | The rate of overall survival | 1 year |
| Measure | Description | Time Frame |
|---|---|---|
| Bone marrow recovery | Number of participants with granulopoiesis, erythropoiesis and megakaryopoiesis recovery of bone marrow after HSCT | day +28 |
| Relapse and GVHD | The rate of relapse and GVHD after HSCT |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Yaqiong Tang, Doctor | Contact | 18896588075 | tangyaqiong@suda.edu.cn |
| Name | Affiliation | Role |
|---|---|---|
| Yue Han, Professor | The First Affiliated Hospital of Soochow University | Principal Investigator |
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| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 27088376 | Background | Alchalby H, Yunus DR, Zabelina T, Ayuk F, Kroger N. Incidence and risk factors of poor graft function after allogeneic stem cell transplantation for myelofibrosis. Bone Marrow Transplant. 2016 Sep;51(9):1223-7. doi: 10.1038/bmt.2016.98. Epub 2016 Apr 18. | |
| 16818281 | Background | Larocca A, Piaggio G, Podesta M, Pitto A, Bruno B, Di Grazia C, Gualandi F, Occhini D, Raiola AM, Dominietto A, Bregante S, Lamparelli T, Tedone E, Oneto R, Frassoni F, Van Lint MT, Pogliani E, Bacigalupo A. Boost of CD34+-selected peripheral blood cells without further conditioning in patients with poor graft function following allogeneic stem cell transplantation. Haematologica. 2006 Jul;91(7):935-40. |
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| Type | Date | Date Unknown |
|---|---|---|
| Release | Apr 26, 2026 | |
| Reset | May 18, 2026 | |
| Release | May 25, 2026 | |
| Unrelease | Jun 1, 2026 | |
| Release | Jun 2, 2026 | |
| Unrelease | Jun 20, 2026 |
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| Release Date | Unrelease Date | Unrelease Date Unknown | Reset Date | MCP Release Number |
|---|---|---|---|---|
| Apr 26, 2026 | May 18, 2026 | |||
| May 25, 2026 | Jun 1, 2026 |
| ID | Term |
|---|---|
| D000077209 | Decitabine |
| D016179 | Granulocyte Colony-Stimulating Factor |
| C520809 | eltrombopag |
| C533238 | avatrombopag |
| ID | Term |
|---|---|
| D001374 | Azacitidine |
| D001372 | Aza Compounds |
| D009930 | Organic Chemicals |
| D003562 | Cytidine |
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| Granulocyte Colony-Stimulating Factor | Drug | 5ug/kg/d when absolute neutrophil count ≤ 1.5 × 109/L |
|
|
| Thrombopoietin Receptor Agonist | Drug | Eltrombopag initial dose: 25 mg orally once a day, may increase to up to 75 mg/day, when platelet count ≤ 30 × 109/L; Avatrombopag initial dose: 20 mg orally once a day, may increase to up to 60 mg/day, when platelet count ≤ 30 × 109/L. |
|
|
| Recombinant human erythropoietin | Drug | 10000 U/day when hemoglobin ≤ 85 g/L |
|
|
| 3-month |
| Event free survival | The rate of event free survival after HSCT | 1 year |
| 34492685 | Background | Prabahran A, Koldej R, Chee L, Ritchie D. Clinical features, pathophysiology, and therapy of poor graft function post-allogeneic stem cell transplantation. Blood Adv. 2022 Mar 22;6(6):1947-1959. doi: 10.1182/bloodadvances.2021004537. |
| 28323004 | Background | Ghobadi A, Fiala MA, Ramsingh G, Gao F, Abboud CN, Stockerl-Goldstein K, Uy GL, Grossman BJ, Westervelt P, DiPersio JF. Fresh or Cryopreserved CD34+-Selected Mobilized Peripheral Blood Stem and Progenitor Cells for the Treatment of Poor Graft Function after Allogeneic Hematopoietic Cell Transplantation. Biol Blood Marrow Transplant. 2017 Jul;23(7):1072-1077. doi: 10.1016/j.bbmt.2017.03.019. Epub 2017 Mar 18. |
| 21483003 | Background | Lubbert M, Suciu S, Baila L, Ruter BH, Platzbecker U, Giagounidis A, Selleslag D, Labar B, Germing U, Salih HR, Beeldens F, Muus P, Pfluger KH, Coens C, Hagemeijer A, Eckart Schaefer H, Ganser A, Aul C, de Witte T, Wijermans PW. Low-dose decitabine versus best supportive care in elderly patients with intermediate- or high-risk myelodysplastic syndrome (MDS) ineligible for intensive chemotherapy: final results of the randomized phase III study of the European Organisation for Research and Treatment of Cancer Leukemia Group and the German MDS Study Group. J Clin Oncol. 2011 May 20;29(15):1987-96. doi: 10.1200/JCO.2010.30.9245. Epub 2011 Apr 11. |
| 33679776 | Background | Han P, Yu T, Hou Y, Zhao Y, Liu Y, Sun Y, Wang H, Xu P, Li G, Sun T, Hu X, Liu X, Li L, Peng J, Zhou H, Hou M. Low-Dose Decitabine Inhibits Cytotoxic T Lymphocytes-Mediated Platelet Destruction via Modulating PD-1 Methylation in Immune Thrombocytopenia. Front Immunol. 2021 Feb 17;12:630693. doi: 10.3389/fimmu.2021.630693. eCollection 2021. |
| 33876188 | Background | Han P, Hou Y, Zhao Y, Liu Y, Yu T, Sun Y, Wang H, Xu P, Li G, Sun T, Hu X, Liu X, Li L, Peng J, Zhou H, Hou M. Low-dose decitabine modulates T-cell homeostasis and restores immune tolerance in immune thrombocytopenia. Blood. 2021 Aug 26;138(8):674-688. doi: 10.1182/blood.2020008477. |
| 33921194 | Background | Zambuzi FA, Cardoso-Silva PM, Castro RC, Fontanari C, Emery FDS, Frantz FG. Decitabine Promotes Modulation in Phenotype and Function of Monocytes and Macrophages That Drive Immune Response Regulation. Cells. 2021 Apr 12;10(4):868. doi: 10.3390/cells10040868. |
| 23328088 | Background | Kopp LM, Ray A, Denman CJ, Senyukov VS, Somanchi SS, Zhu S, Lee DA. Decitabine has a biphasic effect on natural killer cell viability, phenotype, and function under proliferative conditions. Mol Immunol. 2013 Jul;54(3-4):296-301. doi: 10.1016/j.molimm.2012.12.012. Epub 2013 Jan 16. |
| 33646303 | Background | Tang Y, Chen J, Liu Q, Chu T, Pan T, Liang J, He XF, Chen F, Yang T, Ma X, Wu X, Hu S, Cao X, Hu X, Hu J, Liu Y, Qi J, Shen Y, Ruan C, Han Y, Wu D. Low-dose decitabine for refractory prolonged isolated thrombocytopenia after HCT: a randomized multicenter trial. Blood Adv. 2021 Mar 9;5(5):1250-1258. doi: 10.1182/bloodadvances.2020002790. |
| 26181032 | Background | Han Y, Tang Y, Chen J, Liang J, Ye C, Ruan C, Wu D. Low-Dose Decitabine for Patients With Thrombocytopenia Following Allogeneic Hematopoietic Stem Cell Transplantation: A Pilot Therapeutic Study. JAMA Oncol. 2015 May;1(2):249-51. doi: 10.1001/jamaoncol.2014.316. No abstract available. |
| 12010787 | Background | DeSimone J, Koshy M, Dorn L, Lavelle D, Bressler L, Molokie R, Talischy N. Maintenance of elevated fetal hemoglobin levels by decitabine during dose interval treatment of sickle cell anemia. Blood. 2002 Jun 1;99(11):3905-8. doi: 10.1182/blood.v99.11.3905. |
| Jun 2, 2026 | Jun 20, 2026 |
| Jun 22, 2026 |
| D011741 |
| Pyrimidine Nucleosides |
| D011743 | Pyrimidines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D009705 | Nucleosides |
| D009706 | Nucleic Acids, Nucleotides, and Nucleosides |
| D012263 | Ribonucleosides |
| D003115 | Colony-Stimulating Factors |
| D006023 | Glycoproteins |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
| D016298 | Hematopoietic Cell Growth Factors |
| D016207 | Cytokines |
| D036341 | Intercellular Signaling Peptides and Proteins |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011506 | Proteins |
| D001685 | Biological Factors |