Observational Study: Romiplostim for Platelet Recovery in Haploidentical HSCT

Launched by HAIKOU AFFILIATED HOSPITAL OF CENTRAL SOUTH UNIVERSITY XIANGYA SCHOOL OF MEDICINE · May 25, 2025

Keywords

ClinConnect Summary

This observational study is looking at whether adding a drug called romiplostim (Nplate) after a haploidentical hematopoietic stem cell transplant helps children with severe thalassemia recover their platelets faster and with fewer transfusions. Researchers will track platelet recovery within 28 days after transplant, as well as how much platelet transfusion is needed. They will also monitor safety, results like bleeding or clots, and overall survival during that early post-transplant period.

Eligible participants are children aged 2 to 17 years with severe thalassemia who are undergoing haploidentical stem cell transplantation at Haikou People’s Hospital in China. Key criteria include specific diagnostic tests and blood work confirming severe thalassemia, consent to the transplant, and no major contraindications. The study plans to enroll about 30 participants and will collect follow-up information for about 28 days after transplant, with additional online surveys up to around 8 weeks. The study is currently enrolling by invitation, overseen by researchers at Haikou Affiliated Hospital, and individual data are not shared publicly at this time.

Gender

ALL

Eligibility criteria

• 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.