Observational Study: Hetrombopag 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 is an observational study being done at Haikou Affiliated Hospital in China to see if Hetrombopag, a medicine that helps the body make platelets, can improve platelet recovery after a haploidentical hematopoietic stem cell transplant in children with severe thalassemia. About 30 kids aged 2 to 17 who are receiving this type of transplant as part of routine care may participate. To be eligible, a child must have confirmed severe thalassemia, be in the 2–17 age range, and be approved for a haploidentical transplant. Children with a fully matched donor (instead of haploidentical), very high liver enzyme levels, active hepatitis B, an active infection, very high donor antibodies, or other transplant contraindications would not be eligible.

Participants will be followed for about 28 days after the transplant. The main things researchers will look at are how quickly platelets recover (reaching certain platelet levels) and how many platelet transfusions are needed. They will also monitor safety (any drug side effects), bleeding, blood clots, overall survival, and whether the transplant works long term. Kids receiving Hetrombopag will complete a 28-day online survey about their platelet recovery. The study is still enrolling by invitation and is not a randomized trial; the dosing and treatment plan follow routine care, starting Hetrombopag around day +6 after transplant and adjusting the dose as needed. Final study completion is expected in 2026.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Diagnosed with severe thalassemia via thalassemia gene testing, transfusion history, and complete blood count (CBC).
• • Pediatric patients aged 2-17 years.
• • Consented to haploidentical transplantation and evaluated by the transplant team as having no transplant contraindications.
• Exclusion Criteria:
• • Presence of a fully matched donor with refusal of haploidentical transplantation.
• • Donor or recipient with transaminase levels \>2× the upper limit of normal (ULN).
• • Positive hepatitis B DNA test result.
• • Active infection at the time of enrollment.
• • Donor-specific antibodies (DSA) \>5,000 and unable to decrease below 3,000 after antibody therapy.
• • Presence of transplant contraindications as assessed by the transplant team.