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Completed

NCT Number: NCT07003256

Observational Study: Romiplostim for Platelet Recovery in Haploidentical HSCT

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.

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Key information

Conditions

Thalassemia in Children Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Thalassemia

Age range

2 year–17 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Haikou Affiliated Hospital of Central South University Xiangya School of Medicine

Haikou, Hainan, 570208, China

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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.

Other names: Nplate

Primary outcomes

  1. Platelet recovery time

    Time frame: From enrollment to 28 days after transplantation

    The time at nodes such as platelet >20×10^9/L, 50×10^9/L and 100×10^9/L

  2. Platelet transfusion volume

    Time frame: From enrollment to 28 days after transplantation

    The required dosage of platelet suspension

Secondary outcomes

  1. Adverse drug reaction rate

    Time frame: From enrollment to 28 days after transplantation

    The rate of adverse drug reactions occurring during the follow-up period

  2. Bleeding incidence rate

    Time frame: From enrollment to 28 days after transplantation

    The incidence rate of bleeding that occurred during the follow-up period

  3. Thrombosis incidence rate

    Time frame: From enrollment to 28 days after transplantation

    The incidence rate of thrombosis occurring during the follow-up period

  4. Survival rate

    Time frame: From enrollment to 28 days after transplantation

    The survival rate of patients after transplantation medication

  5. Recurrence - free survival rate

    Time frame: From enrollment to 28 days after transplantation

    The survival rate of patients without recurrence of the disease after using the drug

  6. Transplant - related mortality

    Time frame: From enrollment to 28 days after transplantation

  7. Major transplant-related complications

    Time frame: From enrollment to 28 days after transplantation

  8. Cause of death

    Time frame: From enrollment to 28 days after transplantation

Sponsors and collaborators

Lead sponsor

Haikou Affiliated Hospital of Central South University Xiangya School of Medicine

Other

Registry information

Official study title

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

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
Jun 4, 2025
Registry last updated
Jul 28, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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