rhTPO + CsA vs CsA in the Treatment of LR-MDS With Thrombocytopenia

rhTPO + CsA Versus CsA Alone in the Treatment of Lower-risk MDS With Thrombocytopenia: a Single-center Randomized Controlled Trial

This study aims to compare the efficacy and safety of recombinant human thrombopoietin (rhTPO) + cyclosporine (CsA) and CsA monotherapy in the treatment of lower-risk myelodysplastic neoplasms with thrombocytopenia.

Study Overview

• Status: Not yet recruiting
• Conditions: Lower-risk Myelodysplastic Syndromes
• Intervention / Treatment: Drug: Cyclosporine (CsA); Drug: rhTPO

• Study Type: Interventional
• Enrollment (Estimated): 78
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Bing Han, Doctor; Phone Number: +86 13601059938; Email: hanbing_li@sina.com.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Age ≥ 18 years old;
2. Confirmed diagnosis of lower-risk MDS;
3. Platelet ≤ 30×109/L, or < 50×109/L and accompanied by bleeding events;
4. Baseline liver and kidney functions <2 ULN;
5. Signed the informed consent form;
6. ECOG 0-2;

Exclusion Criteria:

1. History of leukemia or stem cell transplantation, treatment-related MDS or malignant tumors;
2. With splenomegaly or myelofibrosis;
3. Infections or bleeding that cannot be controlled by standard treatment;
4. Any concurrent malignant tumors within the past 5 years, or basal cell carcinoma of the skin at the local site;
5. History of thromboembolic events, heart attack or stroke and current use of anticoagulants;
6. Previously received cyclosporine (CsA) or ATG within 6 months;
7. Previously used IL-11, rhTPO or TPO-RA;
8. Pregnant or lactating women;
9. Participated in other clinical trials within 3 months.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: CsA; CsA 3-5mg/kg/d	Drug: Cyclosporine (CsA); CsA 3-5mg/kg/d
Experimental: rhTPO+CsA; rhTPO 300U/kg/d, CsA 3-5mg/kg/d	Drug: Cyclosporine (CsA); CsA 3-5mg/kg/d; Drug: rhTPO; rhTPO 300U/kg/d, subcutaneous injection, once a month for 14 consecutive days

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
HI-P	hematological response - platelet	1-month, 2-month, 3-month, 6-month
ORR	ORR=CRR+PRR+HI	1-month, 2-month, 3-month, 6-month

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of bleeding incidents and severity assessed by the World Health Organization Bleeding Scale	minimum value = 0, maximum value = 4; higher scores indicate worse outcome	3-month, 6-month
Platelet transfusion independence	according to the number and interval of platelet transfusions	3-month, 6-month
Time to CR/OR	Time from baseline to first response	through study period, an average of 6 months
Duration of HI-P	Time to patients achieving HI-P to losing response	through study period, an average of 6-month
Quality of life score measured by the Short Form-36 Health Survey	minimum value = 0, maximum value = 100; higher scores indicate better quality of life	3-month
Clonal evolution	The incidence of elevation of blast cells in bone marrow and peripheral blood within one year, and the incidence of progressing to higher-risk MDS or AML within one year	12-month
OS	Overall survival (OS), defined as the time from randomization/treatment start to death from any cause.	12-month

Collaborators and Investigators

• Sponsor: Peking Union Medical College Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): June 1, 2026
• Primary Completion (Estimated): December 1, 2028
• Study Completion (Estimated): December 1, 2029

Study Registration Dates

• First Submitted: June 7, 2026
• First Submitted That Met QC Criteria: June 11, 2026
• First Posted (Actual): June 17, 2026

Study Record Updates

• Last Update Posted (Actual): June 17, 2026
• Last Update Submitted That Met QC Criteria: June 11, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Peptides; Amino Acids, Peptides, and Proteins; Polycyclic Compounds; Macrocyclic Compounds; Peptides, Cyclic; Cyclosporins; Cyclosporine
• Other Study ID Numbers: K10660

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No