Clinical trial NCT07003269: Observational Study: Hetrombopag for Platelet Recovery in Haploidentical HSCT
Official title: An Observational Study on the Promotion of Platelet Recovery by Hetrombopag During Haploidentical Hematopoietic Stem Cell Transplantation
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Thalassemia in Children
Keywords: Thalassemia
HSCT
Hetrombopag
Lead sponsor: Haikou Affiliated Hospital of Central South University Xiangya School of Medicine
Sponsor class: OTHER
First posted: 2025-06-04
Enrollment: 30
Sex: ALL
Minimum age (years): 2
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: CASE_CONTROL
Time perspective: PROSPECTIVE
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.

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.

Detailed description: Allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains a critical and often the sole curative modality for diverse hematological malignancies and disorders. Post-transplant thrombocytopenia (platelet count \<20×10⁹/L) significantly compromises long-term patient survival, with an incidence of 5-20% in allo-HSCT recipients, thereby escalating treatment morbidity and costs. Current research on platelet engraftment promotion in pediatric thalassemia patients undergoing allo-HSCT is limited. Repeated platelet transfusions are associated with substantial complications, including transfusion reactions, platelet alloimmunization, and transfusion-transmitted viral infections. Eltrombopag, a thrombopoietin receptor agonist (TPO-RA), carries a black-box warning for hepatotoxicity, with a real-world incidence of 11.8%. Transplant recipients frequently experience diarrhea, which impairs the absorption of oral thrombopoietic agents, while daily subcutaneous injections exacerbate pediatric patient discomfort and reduce treatment adherence. In contrast, long-acting TPO-RAs administered once post-transplant have demonstrated favorable tolerability and promising efficacy in thalassemia transplant pediatric populations. To date, clinical data on Hetrombopag use for platelet recovery in haploidentical HSCT for pediatric thalassemia are lacking. Therefore, this observational study aims to evaluate the efficacy and safety of Hetrombopag in facilitating platelet engraftment during haploidentical HSCT in children with thalassemia.

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.

Primary outcomes:
measure: Platelet recovery time
timeFrame: From enrollment to 28 days after transplantation
measure: Platelet transfusion volume
timeFrame: From enrollment to 28 days after transplantation
Secondary outcomes:
measure: The rate of adverse drug reactions
timeFrame: From enrollment to 28 days after transplantation
measure: Bleeding incidence rate
timeFrame: From enrollment to 28 days after transplantation
measure: Thrombosis incidence rate
timeFrame: From enrollment to 28 days after transplantation
measure: Survival rate
timeFrame: From enrollment to 28 days after transplantation
measure: Relapse-free survival rate
timeFrame: From enrollment to 28 days after transplantation
measure: Transplant-related mortality rate
timeFrame: From enrollment to 28 days after transplantation
measure: Major transplant-related complications
timeFrame: From enrollment to 28 days after transplantation
measure: Cause of death
timeFrame: From enrollment to 28 days after transplantation
Other outcomes:
Locations:
city: Haikou
country: China
facility: Haikou Affiliated Hospital of Central South University Xiangya School of Medicine
state: Hainan
Study officials:
affiliation: Department of Hematology, Haikou People's Hospital
name: Xiaoyang Yang, MD
role: PRINCIPAL_INVESTIGATOR
References: