Observational Study: Romiplostim for Platelet Recovery in Haploidentical HSCT

NCT07003256 | Enrolling By Invitation DMC FDA Drug

• 1 other identifier: JVKY2024-0101015002
• Study Type: observational
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Thalassemia in Children

Keywords

Thalassemia; Romiplostim; HSCT

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (8)

• Adverse drug reaction rate

  From enrollment to 28 days after transplantation

• Bleeding incidence rate

  From enrollment to 28 days after transplantation

• Thrombosis incidence rate

  From enrollment to 28 days after transplantation

• Survival rate

  From enrollment to 28 days after transplantation

• Recurrence - free survival rate

  From enrollment to 28 days after transplantation

Study Arms (1)

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

Interventions

Romiplostim DRUG

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.

Also known as: Nplate

experimental group

Eligibility Criteria

• Age: 2 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Haikou Affiliated Hospital of Central South University Xiangya School of Medicine: lead

Study Sites (1)

Haikou Affiliated Hospital of Central South University Xiangya School of Medicine

Haikou, Hainan, 570208, China

Location

MeSH Terms

Conditions

Thalassemia

Interventions

romiplostim

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Xiaoyang Yang, MD

  Department of Hematology, Haikou People's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: PROSPECTIVE
• Target Duration: 28 Days
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 15, 2025
• First Posted: June 4, 2025
• Study Start: September 1, 2024
• Primary Completion: April 30, 2026
• Study Completion: April 30, 2026
• Last Updated: June 6, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)