Clinical Research Directory

Romiplostim N01 for Platelet Recovery After Haploidentical HSCT

This is a prospective, randomized, controlled clinical study designed to evaluate the efficacy and safety of Romiplostim N01 in promoting platelet engraftment after haploidentical allogeneic hematopoietic stem cell transplantation (haplo-HSCT) in patients with hematologic malignancies. A total of 130 patients who undergo haplo-HSCT for acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), or other hematologic malignancies will be enrolled and randomized 1:1 into a treatment group and a control group. The treatment group will receive Romiplostim N01 subcutaneously once weekly at a starting dose of 5 µg/kg, with dose adjustments based on platelet counts (maximum 10 µg/kg), for up to 4 weeks or until platelet counts reach ≥100 × 10⁹/L. The control group will not receive rh-TPO or any thrombopoietin receptor agonist (TPO-RA) therapy. Supportive care including transfusions and growth factors (G-CSF, ESA) is allowed in both groups. The primary endpoint is the cumulative platelet engraftment rate by day +21 post-transplant, defined as sustained platelet counts \> 20 × 10⁹/L for at least 7 consecutive days without transfusion. Secondary endpoints include median time to platelet engraftment, median time to achieve platelet counts ≥ 50 × 10⁹/L and ≥ 100 × 10⁹/L, total platelet transfusion volume, erythroid and neutrophil responses within 4 weeks, and overall hematopoietic recovery. Safety endpoints include the incidence of adverse events, thromboembolic events, and treatment-related serious adverse events. The study aims to determine whether early administration of Romiplostim N01 can accelerate platelet recovery and reduce bleeding risk in patients undergoing haplo-HSCT, thereby improving post-transplant outcomes.

Hetrombopag in Promoting Platelet Engraftment After Auto-HSCT in Children With Neuroblastoma

The goal of this clinical trial is to learn if hetrombopag promotes platelet engraftment in neuroblastoma children undergoing autologous hematopoietic stem cell transplantation. The main question it aims to answer is: · Does drug hetrombopag promote platelet engraftment? Participants will take hetrombopag every day for 6 weeks or until platelet \> 100\*10\^9/L.