MDS (Myelodysplastic Syndrome)Phase 2PROMPTNCT07400341
Romiplostim Versus rhTPO for Platelet Engraftment After Transplant in MDS and AA
A Phase 2 study testing Romiplostim and Recombinant Human Thrombopoietin in people with MDS (Myelodysplastic Syndrome) and Aplastic Anemia (AA), run by The First Affiliated Hospital of Soochow University at 1 site in China.
Trial summary
This study is for adults aged 18-65 with myelodysplastic syndrome (MDS) or severe aplastic anemia (AA) who are scheduled to receive a donor stem cell transplant (allogeneic hematopoietic stem cell transplant). After the transplant, it is critical for the body to start making its own blood cells again. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- MDS (Myelodysplastic Syndrome), Aplastic Anemia (AA)
- What is being tested?
- Romiplostim and Recombinant Human Thrombopoietin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 66 participants
- Where
- 1 siteChina
- Study dates
- 31 Jan 2026to 31 Dec 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age 18-65 years (inclusive).
- Diagnosis of Myelodysplastic Syndrome (MDS) per WHO criteria, or Severe/Very Severe Aplastic Anemia (SAA/VSAA) per Camitta criteria, and deemed eligible for allogeneic hematopoietic stem cell transplantation (allo-HSCT).
- Planned to receive allo-HSCT from a matched sibling, haploidentical, or unrelated donor (including cord blood).
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
- Persistent platelet count <20×10⁹/L with platelet transfusion dependence between post-transplant days +4 and +10. Transfusion dependence is defined as platelet count not doubling within 24-48 hours after transfusion or ongoing need for prophylactic transfusion.
- Adequate cardiac, hepatic, and renal function as required for transplantation, per investigator assessment.
- Voluntary participation with written informed consent obtained prior to any study-specific procedures.
Who cannot join
- Active, uncontrolled bacterial, fungal, or viral infection at the time of enrollment.
- History of arterial thrombosis, or venous thromboembolism within the past 6 months (unless cured or stable for over 6 months).
- Active transplant-associated thrombotic microangiopathy (TA-TMA).
- Pre-transplant bone marrow biopsy showing fibrosis grade ≥ MF-2 (according to WHO criteria).
- Known hypersensitivity to Romiplostim, recombinant human thrombopoietin (rhTPO), or any of their excipients.
- Pregnant or lactating women.
- Women of childbearing potential or men with partners of childbearing potential who are unwilling to use highly effective contraception during the study period and for at least 3 months after the last dose of study drug.
- Any other condition that, in the opinion of the investigator, would make the patient unsuitable for participation in the study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Depei Wu Professor, Director of Hematology Department, MD contact
China1 site, 1 recruiting
- Recruiting
The First Affiliated Hospital of Soochow University, Department of Hematology
Suzhou, Jiangsu, 215006
Clinical Trial Coordinator, Hematology Department, +8651267780111, wuxiaojin@suda.edu.cn
About this study
Official title: A Randomized Phase II Study of Romiplostim vs. rhTPO for Platelet Engraftment After Allo-HSCT in Patients With MDS and Aplastic Anemia (PROMPT)
This study is for adults aged 18-65 with myelodysplastic syndrome (MDS) or severe aplastic anemia (AA) who are scheduled to receive a donor stem cell transplant (allogeneic hematopoietic stem cell transplant). After the transplant, it is critical for the body to start making its own blood cells again. A common and serious problem is a delay in the recovery of platelets (the cells that help stop bleeding), which increases the risk of bleeding, infection, and death.
This study aims to see if a new treatment can help platelets recover faster and more safely after transplant. We are comparing two drugs:
Read more
Romiplostim: A long-acting injection given just once a week. rhTPO (Recombinant Human Thrombopoietin): A standard injection given every day. Both drugs are designed to help the body make more platelets. The main question is whether the once-weekly romiplostim works as well or better than the daily rhTPO, and if it is safe.
About 66 patients will participate. By random chance (like flipping a coin), each participant will be assigned to receive either romiplostim or rhTPO. The treatment will start a few days after the transplant and continue until platelets recover to a safe level or for up to 8 weeks. Doctors will closely monitor all participants for 100 days to track platelet recovery, need for transfusions, side effects, and overall health.
Read the detailed description
This is a prospective, single-center, randomized, open-label, parallel-controlled Phase II clinical trial. The primary objective is to provide a preliminary efficacy estimate and evaluate the safety of romiplostim compared to recombinant human thrombopoietin (rhTPO) for promoting platelet engraftment following allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with myelodysplastic syndrome (MDS) or aplastic anemia (AA).
Study Design and Population:
The study will enroll approximately 74 patients to achieve 66 evaluable subjects, randomized in a 1:1 ratio to the romiplostim group or the rhTPO group. Randomization will be stratified by the underlying disease (MDS vs. AA). Eligible patients are aged 18-65 years, diagnosed with MDS or severe/very severe AA (SAA/VSAA), and are candidates for allo-HSCT from a matched sibling, haploidentical, or unrelated donor (including cord blood). Key inclusion criteria require a platelet count <20×10⁹/L with transfusion dependence between day +4 and day +10 post-transplant. Major exclusion criteria include uncontrolled active infection, significant history of thrombosis, active transplant-associated thrombotic microangiopathy (TA-TMA), pre-transplant bone marrow fibrosis ≥ MF-2 grade, and known allergy to the study drugs.
Interventions:
Experimental Group (n≈33): Romiplostim administered subcutaneously at a starting dose of 5.0 µg/kg once weekly, beginning on transplant day +4. Subsequent doses (from day +18) will be adjusted weekly based on platelet counts.
Active Control Group (n≈33): rhTPO administered subcutaneously at a fixed dose of 15000 U once daily, beginning on transplant day +4.
Treatment for both groups continues until the platelet count is ≥50×10⁹/L without transfusion support for 7 consecutive days, until day +60 post-transplant, or for a maximum of 8 weeks, whichever occurs first. All patients will receive standardized transplant supportive care.
Endpoints:
Primary Endpoints:
Platelet engraftment time, defined as the first of three consecutive days with a platelet count ≥20×10⁹/L without platelet transfusion in the preceding 7 days.
Incidence of ≥ Grade 3 adverse events (AEs) within 100 days post-transplant, with specific focus on graft-versus-host disease (GVHD) and thrombotic events.
Secondary Endpoints: Platelet engraftment rate at day 60 (≥50×10⁹/L), total platelet transfusion units from day 0 to 60, overall survival (OS), progression-free survival (PFS), and non-relapse mortality (NRM).
Exploratory Endpoints: Immune reconstitution (lymphocyte subsets), megakaryocyte-related biomarkers, and a pharmacoeconomic evaluation.
Oversight and Safety:
An Independent Data Safety Monitoring Committee (DSMB) will be established to periodically review accumulated safety data. Safety will be actively monitored through weekly clinical and laboratory assessments during the treatment period, with specific focus on thrombotic events, GVHD, and potential clonal evolution. The study will be conducted at the Department of Hematology, The First Affiliated Hospital of Soochow University, China, following approval by its institutional Ethics Committee.
- Being tested
- Romiplostim, Recombinant Human Thrombopoietin
- Keywords
- Romiplostim, Recombinant Human Thrombopoietin (rhTPO), Platelet Engraftment, Delayed Platelet Recovery
- Medical subject headings
- Anemia, Refractory, with Excess of Blasts, Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RomiplostimDrug
Also called TPO-RA
Romiplostim is a thrombopoietin receptor agonist (TPO-RA) that stimulates platelet production. It is a fusion protein (peptibody) that binds to and activates the TPO receptor, promoting megakaryocyte proliferation and differentiation. In this study, it is administered as a subcutaneous injection once weekly.
Recombinant Human ThrombopoietinDrug
Also called rhTPO
Recombinant human thrombopoietin is a cytokine that stimulates platelet production by binding to the TPO receptor on megakaryocytes. In this study, it is administered as a subcutaneous injection once daily.
| Study group | Role | What they receive |
|---|---|---|
| rhTPO (Recombinant Human Thrombopoietin) | Active comparator | Participants will receive subcutaneous rhTPO injections at a fixed dose of 15000 U once daily, beginning on transplant day +4. Treatment continues until the platelet count is ≥50×10⁹/L without transfusion for 7 days, until day +60 post-transplant, or for a maximum of 8 weeks, whichever occurs first. Receives: Drug: Recombinant Human Thrombopoietin |
| Romiplostim | Experimental | Participants will receive subcutaneous Romiplostim injections at a starting dose of 5.0 µg/kg once weekly, beginning on transplant day +4. The dose will be adjusted weekly based on platelet counts. Treatment continues until the platelet count is ≥50×10⁹/L without transfusion for 7 days, until day +60 post-transplant, or for a maximum of 8 weeks, whichever occurs first. Receives: Drug: Romiplostim |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of Platelet Engraftment by Day +60 (Platelets ≥50×10^9/L without transfusion for 7 consecutive days)
To preliminarily evaluate and compare the efficacy of romiplostim versus rhTPO in promoting platelet recovery after allo-HSCT. Engraftment is defined as achieving a platelet count ≥50×10^9/L without transfusion support for 7 consecutive days.
Measured: From the start of transplantation until Day 60 post-transplant (or until the engraftment criterion is met).
Incidence of ≥ Grade 3 Adverse Events within 100 Days Post-Transplant (including GVHD and thrombotic events)
To assess and compare the safety profile between romiplostim and rhTPO, focusing on severe adverse events. Events of special interest include acute/chronic GVHD (graded per NIH criteria) and thrombotic events (e.g., TA-TMA, deep vein thrombosis).
Measured: From the start of transplantation until Day 100 post-transplant.
Secondary outcomes (5)
60-Day Platelet Engraftment Rate
Proportion of participants achieving platelet engraftment (defined as platelet count ≥50×10⁹/L) by Day 60 post-transplant without platelet transfusion support.
Measured: At Day 60 post-transplant.
Platelet Transfusion Requirements (Days 0-60)
Total number of platelet transfusion units (including both prophylactic and therapeutic transfusions) administered from Day 0 (day of transplant) to Day 60 post-transplant.
Measured: From Day 0 (day of transplant) to Day 60 post-transplant.
Overall Survival (OS)
Time from the date of transplant until death from any cause. Participants alive at the end of follow-up will be censored.
Measured: From date of transplant until death from any cause, assessed up to 2 years.
Progression-Free Survival (PFS)
Time from the date of transplant until disease progression/relapse or death from any cause, whichever occurs first.
Measured: From date of transplant until disease progression/relapse or death from any cause, assessed up to 2 years.
Non-Relapse Mortality (NRM)
Cumulative incidence of death from causes other than disease relapse/progression.
Measured: From date of transplant until death from non-relapse causes, assessed up to 2 years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Feb 2026
- Study start31 Jan 2026
- Primary completion31 Jan 2028 (estimated)
- Study completion31 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (12)Papers the sponsor has linked to this study.
- Other trials for MDS (Myelodysplastic Syndrome)Studies of the same condition, open and under way.
- More trials run by The First Affiliated Hospital of Soochow UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 66 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07400341
- Sponsor's protocol ID
- SOOCHOW-WXJ-2025-1294
- Acronym
- PROMPT
- Status last verified
- Feb 2026
- First submitted
- 19 Jan 2026
- Last updated
- 15 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Individual de-identified participant data that underlie the reported results may be shared upon reasonable request after article publication, subject to a data use agreement.
Publications
Scordo M, Gilbert LJ, Hanley DM, Flynn JR, Devlin SM, Nguyen LK, Ruiz JD, Shah GL, Sauter CS, Chung DJ, Landau HJ, Lahoud OB, Lin RJ, Dahi PB, Perales MA, Giralt SA, Soff GA. Open-label pilot study of romiplostim for thrombocytopenia after autologous hematopoietic cell transplantation. Blood Adv. 2023 Apr 25;7(8):1536-1544. doi: 10.1182/bloodadvances.2022007838.
PubMed 36409612Xie C, Zhao H, Bao X, Fu H, Lou L. Pharmacological characterization of hetrombopag, a novel orally active human thrombopoietin receptor agonist. J Cell Mol Med. 2018 Nov;22(11):5367-5377. doi: 10.1111/jcmm.13809. Epub 2018 Aug 29.
PubMed 30156363Wang R, Cai J, Chen Z, Tian H, Cong D, Bai Y, Zhang W. Recombinant human thrombopoietin does not promote platelet engraftment in newly diagnosed multiple myeloma patients following autologous stem cell transplantation. Sci Rep. 2025 Feb 13;15(1):5393. doi: 10.1038/s41598-025-89535-7.
PubMed 39948233Wang H, Huang M, Zhao Y, Qi JQ, Chen C, Tang YQ, Qiu HY, Fu CC, Tang XW, Wu DP, Ruan CG, Han Y. Recombinant Human Thrombopoietin Promotes Platelet Engraftment and Improves Prognosis of Patients with Myelodysplastic Syndromes and Aplastic Anemia after Allogeneic Hematopoietic Stem Cell Transplantation. Biol Blood Marrow Transplant. 2017 Oct;23(10):1678-1684. doi: 10.1016/j.bbmt.2017.06.010. Epub 2017 Jun 19.
PubMed 28642072Han TT, Xu LP, Liu DH, Liu KY, Wang FR, Wang Y, Yan CH, Chen YH, Sun YQ, Ji Y, Wang JZ, Zhang XH, Huang XJ. Recombinant human thrombopoietin promotes platelet engraftment after haploidentical hematopoietic stem cell transplantation: a prospective randomized controlled trial. Ann Hematol. 2015 Jan;94(1):117-28. doi: 10.1007/s00277-014-2158-1. Epub 2014 Jul 30.
PubMed 25069650Blumberg N, Heal JM, Phillips GL, Phipps RP. Platelets--to transfuse or not to transfuse. Lancet. 2012 Oct 13;380(9850):1287-9. doi: 10.1016/S0140-6736(12)60983-0. Epub 2012 Aug 8. No abstract available.
PubMed 22877505Bolwell B, Pohlman B, Sobecks R, Andresen S, Brown S, Rybicki L, Wentling V, Kalaycio M. Prognostic importance of the platelet count 100 days post allogeneic bone marrow transplant. Bone Marrow Transplant. 2004 Feb;33(4):419-23. doi: 10.1038/sj.bmt.1704330.
PubMed 14688814Kuzmina Z, Eder S, Bohm A, Pernicka E, Vormittag L, Kalhs P, Petkov V, Stary G, Nepp J, Knobler R, Just U, Krenn K, Worel N, Greinix HT. Significantly worse survival of patients with NIH-defined chronic graft-versus-host disease and thrombocytopenia or progressive onset type: results of a prospective study. Leukemia. 2012 Apr;26(4):746-56. doi: 10.1038/leu.2011.257. Epub 2011 Sep 16.
PubMed 21926960Kanda Y, Chiba S, Hirai H, Sakamaki H, Iseki T, Kodera Y, Karasuno T, Okamoto S, Hirabayashi N, Iwato K, Maruta A, Fujimori Y, Furukawa T, Mineishi S, Matsuo K, Hamajima N, Imamura M. Allogeneic hematopoietic stem cell transplantation from family members other than HLA-identical siblings over the last decade (1991-2000). Blood. 2003 Aug 15;102(4):1541-7. doi: 10.1182/blood-2003-02-0430. Epub 2003 Apr 24.
PubMed 12714500Yamazaki R, Kuwana M, Mori T, Okazaki Y, Kawakami Y, Ikeda Y, Okamoto S. Prolonged thrombocytopenia after allogeneic hematopoietic stem cell transplantation: associations with impaired platelet production and increased platelet turnover. Bone Marrow Transplant. 2006 Sep;38(5):377-84. doi: 10.1038/sj.bmt.1705444.
PubMed 16915226Mohty M, Kuentz M, Michallet M, Bourhis JH, Milpied N, Sutton L, Jouet JP, Attal M, Bordigoni P, Cahn JY, Boiron JM, Blaise D; Societe Francaise de Greffe de Moelle et de Therapie Cellulaire (SFGM-TC). Chronic graft-versus-host disease after allogeneic blood stem cell transplantation: long-term results of a randomized study. Blood. 2002 Nov 1;100(9):3128-34. doi: 10.1182/blood.V100.9.3128.
PubMed 12384409Huang XJ. Current status of haploidentical stem cell transplantation for leukemia. J Hematol Oncol. 2008 Dec 31;1:27. doi: 10.1186/1756-8722-1-27.
PubMed 19117511
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Phase II Study of Romiplostim vs. rhTPO for Platelet Engraftment After Allo-HSCT in Patients With MDS and Aplastic Anemia (PROMPT)
- ClinicalTrials.gov ID
- NCT07400341
- Acronym / sponsor ID
- PROMPT / SOOCHOW-WXJ-2025-1294
- Keywords
- Romiplostim, Recombinant Human Thrombopoietin (rhTPO), Platelet Engraftment, Delayed Platelet Recovery
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 66 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital of Soochow UniversityUniversity, hospital or other
- Study start
- 31 Jan 2026 (actual)
- Primary completion
- 31 Jan 2028 (estimated)
- Study completion
- 31 Dec 2028 (estimated)
- First posted
- 10 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is for adults aged 18-65 with myelodysplastic syndrome (MDS) or severe aplastic anemia (AA) who are scheduled to receive a donor stem cell transplant (allogeneic hematopoietic stem cell transplant). After the transplant, it is critical for the body to start making its own blood cells again. A common and serious problem is a delay in the recovery of platelets (the cells that help stop bleeding), which increases the risk of bleeding, infection, and death.
This study aims to see if a new treatment can help platelets recover faster and more safely after transplant. We are comparing two drugs:
Romiplostim: A long-acting injection given just once a week. rhTPO (Recombinant Human Thrombopoietin): A standard injection given every day. Both drugs are designed to help the body make more platelets. The main question is whether the once-weekly romiplostim works as well or better than the daily rhTPO, and if it is safe.
About 66 patients will participate. By random chance (like flipping a coin), each participant will be assigned to receive either romiplostim or rhTPO. The treatment will start a few days after the transplant and continue until platelets recover to a safe level or for up to 8 weeks. Doctors will closely monitor all participants for 100 days to track platelet recovery, need for transfusions, side effects, and overall health.
Detailed description
This is a prospective, single-center, randomized, open-label, parallel-controlled Phase II clinical trial. The primary objective is to provide a preliminary efficacy estimate and evaluate the safety of romiplostim compared to recombinant human thrombopoietin (rhTPO) for promoting platelet engraftment following allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with myelodysplastic syndrome (MDS) or aplastic anemia (AA).
Study Design and Population:
The study will enroll approximately 74 patients to achieve 66 evaluable subjects, randomized in a 1:1 ratio to the romiplostim group or the rhTPO group. Randomization will be stratified by the underlying disease (MDS vs. AA). Eligible patients are aged 18-65 years, diagnosed with MDS or severe/very severe AA (SAA/VSAA), and are candidates for allo-HSCT from a matched sibling, haploidentical, or unrelated donor (including cord blood). Key inclusion criteria require a platelet count <20×10⁹/L with transfusion dependence between day +4 and day +10 post-transplant. Major exclusion criteria include uncontrolled active infection, significant history of thrombosis, active transplant-associated thrombotic microangiopathy (TA-TMA), pre-transplant bone marrow fibrosis ≥ MF-2 grade, and known allergy to the study drugs.
Interventions:
Experimental Group (n≈33): Romiplostim administered subcutaneously at a starting dose of 5.0 µg/kg once weekly, beginning on transplant day +4. Subsequent doses (from day +18) will be adjusted weekly based on platelet counts.
Active Control Group (n≈33): rhTPO administered subcutaneously at a fixed dose of 15000 U once daily, beginning on transplant day +4.
Treatment for both groups continues until the platelet count is ≥50×10⁹/L without transfusion support for 7 consecutive days, until day +60 post-transplant, or for a maximum of 8 weeks, whichever occurs first. All patients will receive standardized transplant supportive care.
Endpoints:
Primary Endpoints:
Platelet engraftment time, defined as the first of three consecutive days with a platelet count ≥20×10⁹/L without platelet transfusion in the preceding 7 days.
Incidence of ≥ Grade 3 adverse events (AEs) within 100 days post-transplant, with specific focus on graft-versus-host disease (GVHD) and thrombotic events.
Secondary Endpoints: Platelet engraftment rate at day 60 (≥50×10⁹/L), total platelet transfusion units from day 0 to 60, overall survival (OS), progression-free survival (PFS), and non-relapse mortality (NRM).
Exploratory Endpoints: Immune reconstitution (lymphocyte subsets), megakaryocyte-related biomarkers, and a pharmacoeconomic evaluation.
Oversight and Safety:
An Independent Data Safety Monitoring Committee (DSMB) will be established to periodically review accumulated safety data. Safety will be actively monitored through weekly clinical and laboratory assessments during the treatment period, with specific focus on thrombotic events, GVHD, and potential clonal evolution. The study will be conducted at the Department of Hematology, The First Affiliated Hospital of Soochow University, China, following approval by its institutional Ethics Committee.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| rhTPO (Recombinant Human Thrombopoietin) | Active comparator | Drug: Recombinant Human ThrombopoietinParticipants will receive subcutaneous rhTPO injections at a fixed dose of 15000 U once daily, beginning on transplant day +4. Treatment continues until the platelet count is ≥50×10⁹/L without transfusion for 7 days, until day +60 post-transplant, or for a maximum of 8 weeks, whichever occurs first. |
| Romiplostim | Experimental | Drug: RomiplostimParticipants will receive subcutaneous Romiplostim injections at a starting dose of 5.0 µg/kg once weekly, beginning on transplant day +4. The dose will be adjusted weekly based on platelet counts. Treatment continues until the platelet count is ≥50×10⁹/L without transfusion for 7 days, until day +60 post-transplant, or for a maximum of 8 weeks, whichever occurs first. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Age 18-65 years (inclusive).
- Diagnosis of Myelodysplastic Syndrome (MDS) per WHO criteria, or Severe/Very Severe Aplastic Anemia (SAA/VSAA) per Camitta criteria, and deemed eligible for allogeneic hematopoietic stem cell transplantation (allo-HSCT).
- Planned to receive allo-HSCT from a matched sibling, haploidentical, or unrelated donor (including cord blood).
- Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
- Persistent platelet count <20×10⁹/L with platelet transfusion dependence between post-transplant days +4 and +10. Transfusion dependence is defined as platelet count not doubling within 24-48 hours after transfusion or ongoing need for prophylactic transfusion.
Key exclusion criteria (8 listed)
- Active, uncontrolled bacterial, fungal, or viral infection at the time of enrollment.
- History of arterial thrombosis, or venous thromboembolism within the past 6 months (unless cured or stable for over 6 months).
- Active transplant-associated thrombotic microangiopathy (TA-TMA).
- Pre-transplant bone marrow biopsy showing fibrosis grade ≥ MF-2 (according to WHO criteria).
- Known hypersensitivity to Romiplostim, recombinant human thrombopoietin (rhTPO), or any of their excipients.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RomiplostimAlso: TPO-RARomiplostim is a thrombopoietin receptor agonist (TPO-RA) that stimulates platelet production. It is a fusion protein (peptibody) that binds to and activates the TPO receptor, promoting megakaryocyte proliferation and differentiation. In this study, it is administered as a subcutaneous injection once weekly. | Drug | Romiplostim |
| Recombinant Human ThrombopoietinAlso: rhTPORecombinant human thrombopoietin is a cytokine that stimulates platelet production by binding to the TPO receptor on megakaryocytes. In this study, it is administered as a subcutaneous injection once daily. | Drug | rhTPO (Recombinant Human Thrombopoietin) |
Primary outcome
Rate of Platelet Engraftment by Day +60 (Platelets ≥50×10^9/L without transfusion for 7 consecutive days)
Time frame: From the start of transplantation until Day 60 post-transplant (or until the engraftment criterion is met).
To preliminarily evaluate and compare the efficacy of romiplostim versus rhTPO in promoting platelet recovery after allo-HSCT. Engraftment is defined as achieving a platelet count ≥50×10^9/L without transfusion support for 7 consecutive days.
Incidence of ≥ Grade 3 Adverse Events within 100 Days Post-Transplant (including GVHD and thrombotic events)
Time frame: From the start of transplantation until Day 100 post-transplant.
To assess and compare the safety profile between romiplostim and rhTPO, focusing on severe adverse events. Events of special interest include acute/chronic GVHD (graded per NIH criteria) and thrombotic events (e.g., TA-TMA, deep vein thrombosis).
5 secondary outcomes
60-Day Platelet Engraftment Rate
Time frame: At Day 60 post-transplant.
Platelet Transfusion Requirements (Days 0-60)
Time frame: From Day 0 (day of transplant) to Day 60 post-transplant.
Overall Survival (OS)
Time frame: From date of transplant until death from any cause, assessed up to 2 years.
Progression-Free Survival (PFS)
Time frame: From date of transplant until disease progression/relapse or death from any cause, assessed up to 2 years.
Non-Relapse Mortality (NRM)
Time frame: From date of transplant until death from non-relapse causes, assessed up to 2 years.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Apr 2026.
- Background: PubMed 36409612Scordo M, Gilbert LJ, Hanley DM, Flynn JR, Devlin SM, Nguyen LK, Ruiz JD, Shah GL, Sauter CS, Chung DJ, Landau HJ, Lahoud OB, Lin RJ, Dahi PB, Perales MA, Giralt SA, Soff GA. Open-label pilot study of romiplostim for thrombocytopenia after autologous hematopoietic cell transplantation. Blood Adv. 2023 Apr 25;7(8):1536-1544. doi: 10.1182/bloodadvances.2022007838.
- Background: PubMed 30156363Xie C, Zhao H, Bao X, Fu H, Lou L. Pharmacological characterization of hetrombopag, a novel orally active human thrombopoietin receptor agonist. J Cell Mol Med. 2018 Nov;22(11):5367-5377. doi: 10.1111/jcmm.13809. Epub 2018 Aug 29.
- Background: PubMed 39948233Wang R, Cai J, Chen Z, Tian H, Cong D, Bai Y, Zhang W. Recombinant human thrombopoietin does not promote platelet engraftment in newly diagnosed multiple myeloma patients following autologous stem cell transplantation. Sci Rep. 2025 Feb 13;15(1):5393. doi: 10.1038/s41598-025-89535-7.
- Background: PubMed 28642072Wang H, Huang M, Zhao Y, Qi JQ, Chen C, Tang YQ, Qiu HY, Fu CC, Tang XW, Wu DP, Ruan CG, Han Y. Recombinant Human Thrombopoietin Promotes Platelet Engraftment and Improves Prognosis of Patients with Myelodysplastic Syndromes and Aplastic Anemia after Allogeneic Hematopoietic Stem Cell Transplantation. Biol Blood Marrow Transplant. 2017 Oct;23(10):1678-1684. doi: 10.1016/j.bbmt.2017.06.010. Epub 2017 Jun 19.
- Background: PubMed 25069650Han TT, Xu LP, Liu DH, Liu KY, Wang FR, Wang Y, Yan CH, Chen YH, Sun YQ, Ji Y, Wang JZ, Zhang XH, Huang XJ. Recombinant human thrombopoietin promotes platelet engraftment after haploidentical hematopoietic stem cell transplantation: a prospective randomized controlled trial. Ann Hematol. 2015 Jan;94(1):117-28. doi: 10.1007/s00277-014-2158-1. Epub 2014 Jul 30.
- Background: PubMed 22877505Blumberg N, Heal JM, Phillips GL, Phipps RP. Platelets--to transfuse or not to transfuse. Lancet. 2012 Oct 13;380(9850):1287-9. doi: 10.1016/S0140-6736(12)60983-0. Epub 2012 Aug 8. No abstract available.
- Background: PubMed 14688814Bolwell B, Pohlman B, Sobecks R, Andresen S, Brown S, Rybicki L, Wentling V, Kalaycio M. Prognostic importance of the platelet count 100 days post allogeneic bone marrow transplant. Bone Marrow Transplant. 2004 Feb;33(4):419-23. doi: 10.1038/sj.bmt.1704330.
- Background: PubMed 21926960Kuzmina Z, Eder S, Bohm A, Pernicka E, Vormittag L, Kalhs P, Petkov V, Stary G, Nepp J, Knobler R, Just U, Krenn K, Worel N, Greinix HT. Significantly worse survival of patients with NIH-defined chronic graft-versus-host disease and thrombocytopenia or progressive onset type: results of a prospective study. Leukemia. 2012 Apr;26(4):746-56. doi: 10.1038/leu.2011.257. Epub 2011 Sep 16.
- Background: PubMed 12714500Kanda Y, Chiba S, Hirai H, Sakamaki H, Iseki T, Kodera Y, Karasuno T, Okamoto S, Hirabayashi N, Iwato K, Maruta A, Fujimori Y, Furukawa T, Mineishi S, Matsuo K, Hamajima N, Imamura M. Allogeneic hematopoietic stem cell transplantation from family members other than HLA-identical siblings over the last decade (1991-2000). Blood. 2003 Aug 15;102(4):1541-7. doi: 10.1182/blood-2003-02-0430. Epub 2003 Apr 24.
- Background: PubMed 16915226Yamazaki R, Kuwana M, Mori T, Okazaki Y, Kawakami Y, Ikeda Y, Okamoto S. Prolonged thrombocytopenia after allogeneic hematopoietic stem cell transplantation: associations with impaired platelet production and increased platelet turnover. Bone Marrow Transplant. 2006 Sep;38(5):377-84. doi: 10.1038/sj.bmt.1705444.
- Background: PubMed 12384409Mohty M, Kuentz M, Michallet M, Bourhis JH, Milpied N, Sutton L, Jouet JP, Attal M, Bordigoni P, Cahn JY, Boiron JM, Blaise D; Societe Francaise de Greffe de Moelle et de Therapie Cellulaire (SFGM-TC). Chronic graft-versus-host disease after allogeneic blood stem cell transplantation: long-term results of a randomized study. Blood. 2002 Nov 1;100(9):3128-34. doi: 10.1182/blood.V100.9.3128.
- Results publication: PubMed 19117511Huang XJ. Current status of haploidentical stem cell transplantation for leukemia. J Hematol Oncol. 2008 Dec 31;1:27. doi: 10.1186/1756-8722-1-27.
- Individual participant dataSharing planned: Yes. Individual de-identified participant data that underlie the reported results may be shared upon reasonable request after article publication, subject to a data use agreement.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Depei Wu Professor, Director of Hematology Department, MD contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Romiplostim and Recombinant Human Thrombopoietin in people with MDS (Myelodysplastic Syndrome) and Aplastic Anemia (AA), run by The First Affiliated Hospital of Soochow University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 15 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Depei Wu Professor, Director of Hematology Department, MD (+86-512-67972861, depeiwu@suda.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rate of Platelet Engraftment by Day +60 (Platelets ≥50×10^9/L without transfusion for 7 consecutive days) (time frame: From the start of transplantation until Day 60 post-transplant (or until the engraftment criterion is met).). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07400341, which also lists 12 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Apr 2026.