ShanghaiJune 4, 2026 /PRNewswire/ — Chemotherapy-induced thrombocytopenia (CIT) is a common complication in anti-tumor therapy, often leading to chemotherapy delays, dose reductions, or even treatment interruptions, severely impacting patient prognosis. Globally, there remains a significant unmet need for convenient, safe, and effective oral CIT treatment options. A Chinese-originated oral thrombopoietin receptor agonist, Hetrombopag, is reshaping the treatment landscape in this field.
Recently, the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting was held in Chicago. Dr. Jodi V. Mones from Memorial Sloan Kettering Cancer Center presented a global Phase III, randomized, double-blind study on Hetrombopag for the treatment of CIT. This study showcases the scientific design and clinical value of this Chinese-originated oral thrombopoietin receptor agonist (TPO-RA) in an international population, marking global recognition of China’s innovation in cancer supportive care. As one of the lead investigators of this study, Dr. Jodi V. Mones gave an exclusive interview during the ASCO meeting, sharing deep insights into the unmet needs in CIT, the mechanistic advantages of Hetrombopag, and its potential to transform the global treatment landscape.
Dr. Jodi V. Mones from Memorial Sloan Kettering Cancer Center presented a global Phase III, randomized, double-blind study on Hetrombopag for the treatment of CIT at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting
CIT Management Faces Severe Challenges, Urgent Need for New Options
Dr. Mones noted that CIT is defined as a platelet count below 100×109/L in the context of myelosuppressive chemotherapy. Clinically, CIT patients may experience varying degrees of bleeding. Additionally, CIT can lead to chemotherapy dose reductions, cycle delays, and a decrease in relative dose intensity (RDI), thereby affecting the efficacy of anti-tumor therapy. However, current treatment options for CIT are very limited. Platelet transfusions face global supply shortages and risks such as alloimmunization and allergic reactions; recombinant human interleukin-11 (rhIL-11) may cause tachycardia and edema and is rarely used clinically. Although some TPO-RAs are available, most require weekly subcutaneous injections, necessitating frequent hospital visits for patients, with doses adjusted weekly based on platelet counts—extremely inconvenient for those living far from medical centers. Therefore, a more convenient oral option like Hetrombopag has the potential to become an ideal solution.
Hetrombopag: Mechanism Innovation Drives Chinese Data, Solidly Propelling Global Research
Hetrombopag is a novel oral thrombopoietin receptor agonist (TPO-RA) independently developed by Hengrui Medicine. It promotes megakaryocyte proliferation, differentiation, and platelet production by activating TPO-R-mediated STAT and MAPK signaling pathways. Explaining its unique mechanism, Dr. Mones stated that Hetrombopag binds to the transmembrane domain of the TPO receptor, without competing with endogenous TPO for binding sites. Therefore, it theoretically does not antagonize the physiological function of the patient’s own TPO, offering higher targeting and safety.
A Phase III study conducted in China laid a solid foundation for its global advancement. The study showed that among CIT patients receiving platinum-based chemotherapy, daily administration of Hetrombopag achieved a response rate of 75.7%, with no adverse events of special concern such as thrombosis or ≥Grade 2 bleeding observed, demonstrating good safety. Dr. Mones emphasized that these high-quality positive data directly prompted the initiation of the global Phase III study.
Dr. Mones introduced that the global Phase III study presented at ASCO aims to systematically evaluate the safety and efficacy of Hetrombopag. The study is divided into two parts: Part A enrolls 8-12 non-Asian patients to assess its pharmacokinetic profile in non-Asian populations; Part B randomly assigns patients in a 2:1 ratio to receive Hetrombopag or placebo, to comprehensively evaluate its efficacy and safety across multiple international populations.
CIT affects the normal course of cancer treatment, and an oral drug that helps patients maintain their original chemotherapy regimen would significantly benefit such patients. Dr. Mones believes Hetrombopag is particularly suitable for: patients requiring multiple, repeated chemotherapy cycles; patients living far from medical centers who find it difficult to frequently visit clinics for injections; and patients who wish to manage their platelet counts at home while maintaining a normal daily routine.
CSCO Guidelines Upgraded to 1A Recommendation, Chinese Solution Leads Globally
In China, the clinical value of Hetrombopag has been highly recognized by authoritative guidelines. In the latest edition of the “CSCO Guidelines on the Management of Thrombocytopenia Induced by Antitumor Therapy (2026 Version),” the recommendation level for Hetrombopag has been upgraded to 1A—the highest recommendation level in the current CTIT treatment field. Based on high-level evidence from the Hetrombopag Phase III study, the drug has become an important new standard for CTIT treatment. Notably, a new indication for Hetrombopag in chemotherapy-induced thrombocytopenia was accepted by China’s National Medical Products Administration in August 2025. Previously, the CIT indication had received Orphan Drug Designation from the U.S. FDA, injecting strong momentum into its internationalization process.
Cancer supportive care is as important as anti-tumor therapy. Effective management of CIT is a key prerequisite for ensuring the full dose and timely completion of chemotherapy cycles, directly impacting whether patients can maximize the benefits of anti-tumor treatment. The presentation of this global Phase III study at the ASCO meeting not only marks a significant step in the internationalization of Hengrui’s supportive care product pipeline but also serves as powerful evidence of Chinese-originated innovative solutions contributing Chinese wisdom to CIT patients worldwide.
In concluding the interview, Dr. Mones expressed hope that Hetrombopag will serve as a cornerstone for the comprehensive management of CIT, leading cancer supportive care from a reactive approach to a new phase of proactive prevention, offering safer, more convenient, and more accessible oral treatment options for chemotherapy patients globally.