Observational Study: Romiplostim for Platelet Recovery in Haploidentical HSCT

An Observational Study on the Promotion of Platelet Recovery by Romiplostim During Haploidentical Hematopoietic Stem Cell Transplantation

The objective of this observational study is to explore the long-term effects of roprastine given to promote platelet implantation in hematopoietic stem cell hemicongruent transplantation in children with thalassemia. The main questions it aimed to answer is:

Is roprastine safe and effective for platelet implantation in children with thalassemia hemicongruent transplantation? Participants who have already received roprastine as part of routine medical care for hematopoietic stem cell hemicongruent transplantation in children with thalassemia will answer online survey questions about the effects of their platelet implantation within 8 weeks.

Study Overview

• Status: Enrolling by invitation
• Conditions: Thalassemia in Children
• Intervention / Treatment: Drug: Romiplostim

Detailed Description

Allogeneic hematopoietic stem cell transplantation is an important, if not the only, means of curing many diseases of the blood system. Thrombocytopenia after transplantation seriously affects the long-term survival rate of patients. allo - The incidence of thrombocytopenia in HSCT patients is 5-20% (< 20 x 10 ^ 9/L), increasing the risk and cost of treatment. There are currently few studies on the promotion of platelet growth in children with thalassemia. Repeated infusion of platelet suspension can lead to many adverse consequences, including blood transfusion reactions, platelet homeoimmune responses, and transfusion-associated virus infections. There is a black box warning of liver toxicity in eltropopa, and the incidence of real-world liver toxicity is 11.8%. Transplant patients are prone to diarrhea, which affects the absorption of oral platelet-raising drugs. Daily subcutaneous injection increases children's pain and poor tolerance. However, the long-acting platelet-raising drugs once after transplantation are well tolerated in children with thalassemia transplantation, and the efficacy is worth looking forward to. Up to now, there is a lack of relevant clinical data on the application of roprastine to promote platelet recovery in children with thalassemia hemiphase transplantation. Therefore, this study aimed to explore the observational study of roprastine to promote platelet implantation in children with thalassemia hemiphase transplantation, and to explore the efficacy and safety of the drug.

• Study Type: Observational
• Enrollment (Estimated): 30

Contacts and Locations

Study Locations

• China
  • Hainan
    • Haikou, Hainan, China, 570208
      • Haikou Affiliated Hospital of Central South University Xiangya School of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Children aged 2 - 17 years old who were diagnosed with severe thalassemia at Haikou People's Hospital and received haploidentical hematopoietic stem cell transplantation.

Description

Inclusion Criteria:

• After undergoing Mediterranean gene testing, reviewing the history of blood transfusions, and conducting a blood routine examination, the patient was diagnosed with severe thalassemia.
• Children aged 2 - 17 years old.
• Agree to the haploidentical transplantation, and the team has evaluated that there are no transplantation contraindications.

Exclusion Criteria:

• There is a fully matched donor, and transplantation with a half - matched donor is not agreed.
• For donors and recipients, the transaminase is more than twice the normal value.
• Donor specific antibody Greater than 5000, and after antibody treatment, it should not be lower than 3000.
• Positive for hepatitis B DNA.
• There is an active infection.
• After evaluation by the transplantation team, there are contraindications for transplantation.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

experimental group; After transplantation + 6 days, the subcutaneous injection of roprastine 3ug/kg was started, and the number of platelets in the machine-taken platelet suspension was increased by 2 µg/kg per week, and the maximum dose was 10 μg/kg. Discontinued until platelets rose to 100 × 109/L. If platelets ≤ 20 X 109/L were used for + 20 days, roprastine combined with atropopa 25mg was given orally once a day. Patients were given 1 therapeutic dose of fresh machine-taken platelet suspension when platelets were ≤ 20 X 109/L, or when there was active bleeding at 21~ 50 × 109/L. The number of platelets in the machine-taken platelet suspension per therapeutic dose was > 2.5 × 1011. If not implanted at + 28 days after transplantation, re-transplantation is required, and roprastine is considered ineffective.

Drug: Romiplostim; After transplantation + 6 days, the subcutaneous injection of roprastine 3ug/kg was started, and the number of platelets in the machine-taken platelet suspension was increased by 2 µg/kg per week, and the maximum dose was 10 μg/kg. Discontinued until platelets rose to 100 × 109/L. If platelets ≤ 20 X 109/L were used for + 20 days, roprastine combined with atropopa 25mg was given orally once a day. Patients were given 1 therapeutic dose of fresh machine-taken platelet suspension when platelets were ≤ 20 X 109/L, or when there was active bleeding at 21~ 50 × 109/L. The number of platelets in the machine-taken platelet suspension per therapeutic dose was > 2.5 × 1011. If not implanted at + 28 days after transplantation, re-transplantation is required, and roprastine is considered ineffective.; Other Names: Nplate

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Platelet recovery time	The time at nodes such as platelet >20×10^9/L, 50×10^9/L and 100×10^9/L	From enrollment to 28 days after transplantation
Platelet transfusion volume	The required dosage of platelet suspension	From enrollment to 28 days after transplantation

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Major transplant-related complications		From enrollment to 28 days after transplantation
Cause of death		From enrollment to 28 days after transplantation
Adverse drug reaction rate	The rate of adverse drug reactions occurring during the follow-up period	From enrollment to 28 days after transplantation
Bleeding incidence rate	The incidence rate of bleeding that occurred during the follow-up period	From enrollment to 28 days after transplantation
Thrombosis incidence rate	The incidence rate of thrombosis occurring during the follow-up period	From enrollment to 28 days after transplantation
Survival rate	The survival rate of patients after transplantation medication	From enrollment to 28 days after transplantation
Recurrence - free survival rate	The survival rate of patients without recurrence of the disease after using the drug	From enrollment to 28 days after transplantation
Transplant - related mortality		From enrollment to 28 days after transplantation

Collaborators and Investigators

• Sponsor: Haikou Affiliated Hospital of Central South University Xiangya School of Medicine
• Investigators: Principal Investigator: Xiaoyang Yang, MD, Department of Hematology, Haikou People's Hospital

Study record dates

Study Major Dates

• Study Start (Actual): September 1, 2024
• Primary Completion (Estimated): April 30, 2026
• Study Completion (Estimated): April 30, 2026

Study Registration Dates

• First Submitted: May 15, 2025
• First Submitted That Met QC Criteria: May 25, 2025
• First Posted (Actual): June 4, 2025

Study Record Updates

• Last Update Posted (Actual): June 6, 2025
• Last Update Submitted That Met QC Criteria: June 3, 2025
• Last Verified: June 1, 2025

More Information

Terms related to this study

• Keywords: Thalassemia; HSCT; Romiplostim
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemoglobinopathies; Thalassemia
• Other Study ID Numbers: JVKY2024-0101015002

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: The IPD encompasses highly sensitive and confidential data that was collected through significant investment of our resources, both in terms of time and funding. This data is integral to our ongoing research initiatives, which are at a crucial stage of development. Premature sharing could disrupt our research timelines and strategic plans. Additionally, we have not yet established comprehensive safeguards to ensure that the data will be used appropriately by external researchers. Without proper protocols in place, there is a risk of misuse or misinterpretation of the data, which could lead to inaccurate research outcomes and potential reputational damage to our institution. For these reasons, we have decided not to share the IPD at this time.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No