Editor's Note: At the 32nd International Society on Thrombosis and Haemostasis (ISTH 2024) conference, the research team led by Dr. Xiaoyu Zhu and Dr. Aijie Huang from The First Affiliated Hospital of the University of Science and Technology of China (Anhui Provincial Hospital) garnered significant attention. Their study on the efficacy and safety of avatrombopag for treating thrombocytopenia following cord blood transplantation (UCBT) was selected for oral presentation (OC33.3), with Dr. Huang delivering the report. This study not only showcases the exceptional capabilities of Chinese research teams in hematology but also contributes valuable insights to the global hematology community. "Oncology Frontier - Hematology Frontier" had an exclusive interview with Dr. Aijie Huang to delve deeper into the research and discuss future plans in the field of UCBT.

Oncology Frontier – Hematology Frontier: Your study demonstrates that avatrombopag significantly promotes platelet engraftment and recovery after UCBT, especially when used early post-transplant. What recommendations do you have for determining the optimal timing to start avatrombopag treatment?

Dr. Aijie Huang: Our study is a retrospective analysis aimed at evaluating the efficacy and safety of avatrombopag in patients who underwent transplantation at our center between 2021 and 2023. We selected 130 patients who received cord blood transplantation and avatrombopag, 13 of whom were treated for secondary platelet recovery failure, while the remaining 117 were given the drug to promote platelet engraftment.

To minimize bias, we matched patients from a 2020 randomized controlled trial on recombinant human thrombopoietin (rhTPO) published in Blood Advances with our current study patients based on disease, weight, and age. We restricted avatrombopag administration to within 30 days post-transplant and for a continuous 7-day period, ultimately including 52 patients treated to promote platelet engraftment. Our clinical data analysis revealed that avatrombopag significantly promoted platelet engraftment.

To explore the optimal timing for drug administration post-transplant, we divided patients into early-use (≤+14 days) and late-use (>+14 days) groups based on the median avatrombopag administration time (+14 days). Results showed that patients who used avatrombopag early had higher platelet engraftment rates, whereas late users had lower engraftment rates and poorer survival outcomes. This finding suggests that early post-transplant avatrombopag use may be more beneficial for platelet engraftment.

Based on these retrospective results, we registered a prospective clinical study (NCT05823376) to more accurately determine the optimal timing for avatrombopag use. This study aims to explore the effects of avatrombopag in treating bone marrow failure diseases with cord blood transplantation, administering the drug from the first to the twenty-eighth day post-transplant. To date, we have enrolled eight patients, seven of whom successfully achieved platelet engraftment with a median engraftment time of +28 days, which is superior to the +31 days median engraftment time observed in our previous retrospective study’s early-use group. Therefore, current clinical results suggest that earlier avatrombopag use may be more advantageous for platelet engraftment.

Oncology Frontier – Hematology Frontier: The study indicates that patients treated with avatrombopag alone had better cumulative platelet engraftment rates than those treated with rhTPO or a combination of rhTPO and avatrombopag. Could you explain this finding? What are its clinical implications?

Dr. Aijie Huang: Our current results show that avatrombopag monotherapy is slightly superior to the combination of avatrombopag and rhTPO and rhTPO monotherapy in terms of cumulative engraftment rates. The combination of the two drugs and rhTPO monotherapy had similar cumulative engraftment rates. However, since this analysis is based on retrospective data and the sample size is relatively small after screening, we cannot directly conclude that avatrombopag monotherapy is more effective. In the future, we plan to increase the sample size to further analyze whether the combination therapy is comparable to or inferior to monotherapy with avatrombopag or rhTPO.

Dr. Xiaoyu Zhu

Chief Physician, MD, PhD supervisor, and postdoctoral mentor, Director of the Department of Hematology, First Affiliated Hospital of the University of Science and Technology of China (Anhui Provincial Hospital), Deputy Director of the Institute of Hematology and Cell Therapy, University of Science and Technology of China, and Deputy Director of the Anhui Provincial Key Laboratory of Blood Cell Research and Application. He is a candidate for Anhui Province Academic and Technical Leaders, a leading health innovation talent in the ninth batch of Anhui Province’s “Special Support Plan,” a distinguished health talent of the first Anhui Province, recipient of the Anhui Provincial Outstanding Youth Fund, and leader of the excellent scientific research and innovation team for “Cord Blood Transplantation in Malignant Hematologic Diseases” in Anhui Province. He has studied at the University of Maryland in the United States and the University of Toronto in Canada.

Academic Appointments: Vice Chairman of the Aplastic Anemia Working Group of the Chinese Hematology Association; Member of the Hematopoietic Stem Cell Application Group of the 11th Committee of the Hematology Branch of the Chinese Medical Association; Member of the Hematology Physician Branch of the Chinese Medical Doctor Association; Member of the Hematology Oncology Professional Committee of the Chinese Anti-Cancer Association; Member of the Youth Committee of the 9th and 10th Hematology Branch of the Chinese Medical Association; Vice Chairman of the 8th Committee of the Hematology Branch of the Anhui Medical Association; Chairman of the Hematology Quality Control Center of Anhui Province. Dr. Zhu has long been engaged in the diagnosis and treatment of malignant hematological diseases and hematopoietic stem cell transplantation, particularly in the field of cord blood transplantation. He is dedicated to researching the biological characteristics and clinical application of double-negative T (DNT) cells, hematopoietic stem cell in vitro expansion, and megakaryocyte differentiation. He has led four National Natural Science Foundation of China projects and four provincial and ministerial-level projects. In recent years, he has published over 20 SCI papers as a corresponding author (including co-corresponding) in journals such as Blood, Blood Advances, Bioactive Materials, and American Journal of Hematology.

Dr. Aijie Huang

PhD in Clinical Medicine (Hematology) from the First Affiliated Hospital of the University of Science and Technology of China (Anhui Provincial Hospital). Dr. Huang is engaged in both basic and clinical research on allogeneic hematopoietic stem cell transplantation and is a member of the excellent scientific research and innovation team for “Cord Blood Transplantation in Malignant Hematologic Diseases” in Anhui Province. Over the past three years, Dr. Huang has published four SCI papers as the first or co-first author.