Observational Study: Hetrombopag for Platelet Recovery in Haploidentical HSCT

NCT07003269 | Enrolling By Invitation DMC FDA Drug

• 1 other identifier: SC20240561
• Study Type: observational
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

The objective of this observational study is to investigate the long-term effects of Hetrombopag in promoting platelet engraftment during haploidentical hematopoietic stem cell transplantation (HSCT) in children with thalassemia, with a specific focus on a 28-day time window post-transplantation. The core question to be addressed is: Is Hetrombopag safe and effective for platelet engraftment in children with thalassemia undergoing haploidentical HSCT within a 28-day post-transplant period? Subjects who received Hetrombopag as part of routine care for haploidentical HSCT in children with thalassemia will be required to complete a 28-day online survey on platelet engraftment outcomes.

Conditions

Thalassemia in Children

Keywords

Thalassemia; HSCT; Hetrombopag

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

  Required dosage of platelet suspension

  From enrollment to 28 days after transplantation

Secondary Outcomes (8)

• The rate of adverse drug reactions

  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

• Relapse-free survival rate

  From enrollment to 28 days after transplantation

Study Arms (1)

Experimental group

In allogeneic hematopoietic stem cell transplantation, hetrombopag is initiated at 3 µg/kg subcutaneously on day +6 post-transplant. The dose is increased by 2 µg/kg weekly up to a maximum of 10 µg/kg. Treatment is discontinued when platelet counts rise to 100×10⁹/L. If platelet counts remain ≤20×10⁹/L on day +20, hetrombopag is combined with eltrombopag 25 mg orally once daily. Fresh apheresis platelet suspensions (1 therapeutic dose, containing \>2.5×10¹¹ platelets) are administered when platelet counts are ≤20×10⁹/L or when counts are between 21-50×10⁹/L with active bleeding. If engraftment has not occurred by day +28 post-transplant, re-transplantation is required, and hetrombopag is considered ineffective.

Drug: Hetrombopag

Interventions

Hetrombopag DRUG

In allogeneic hematopoietic stem cell transplantation, hetrombopag is initiated at 3 µg/kg subcutaneously on day +6 post-transplant. The dose is increased by 2 µg/kg weekly up to a maximum of 10 µg/kg. Treatment is discontinued when platelet counts rise to 100×10⁹/L. If platelet counts remain ≤20×10⁹/L on day +20, hetrombopag is combined with eltrombopag 25 mg orally once daily. Fresh apheresis platelet suspensions (1 therapeutic dose, containing \>2.5×10¹¹ platelets) are administered when platelet counts are ≤20×10⁹/L or when counts are between 21-50×10⁹/L with active bleeding. If engraftment has not occurred by day +28 post-transplant, re-transplantation is required, and hetrombopag is considered ineffective.

Also known as: Hetrombopag Olamine

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

The Department of Hematology of Haikou People's Hospital accepts children with severe thalassemia aged 2 - 17 years old for haploidentical hematopoietic stem cell transplantation.

You may qualify if:

• 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.

You may not qualify if:

• 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.

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

hetrombopag

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
• 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 4, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)