Understanding of Avatrombopag Maleate Tablets in the Real World: Clinical Choices under the Price Reduction of Original Drugs and the Rise of Generic Drugs

In recent years, the treatment strategies surrounding thrombocytopenia are undergoing significant changes, especially in the field of chronic liver disease-associated thrombocytopenia (CLD-TP) and immune thrombocytopenia (ITP). Oral thrombopoietin receptor agonists have gradually become a key recommendation in international guidelines. Avatrombopag maleate (Avatrombopag, trade name Doptelet, domestic trade name Su Kexin) is a new generation of non-peptide TPO receptor agonist. Its overseas real-world research and clinical application in China will be promoted simultaneously in 2026, making it no longer just a "drug in the instructions", but an important tool gradually integrated into the clinical decision-making path.

As the original drug of avatrombopag is launched in China and entered the Category B medical insurance catalog, and gradually replaces traditional platelet transfusion or some hormone regimens in real diagnosis and treatment scenarios, its clinical value, medication boundaries and patient benefit models are being redefined.

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The mechanism of action of avatrombopag: why it is called"non-competitive TPO receptor agonist"

Unlike early recombinant thrombopoietin or peptidesTPO‑RA, avatrombopag is a small molecule oral preparation, and its binding site with the thrombopoietin receptor (TPOR) does not compete with endogenous TPO. This feature allows it to theoretically produce an "additive effect" with TPO in the body, rather than replacing or antagonizing it.

At the molecular level, avatrombopag promotes the proliferation and maturation of megakaryocyte progenitor cells in the bone marrow by activating JAK-STAT and other downstream signaling pathways, thereby improving the efficiency of peripheral platelet production. This mechanism is considered to be closer to "physiological regulation" and also explains its relatively stable platelet elevation curve in chronic use.

Innovation in treatment strategies for thrombocytopenia associated with chronic liver disease

In patients with chronic liver disease, thrombocytopenia has long been regarded as an "irreversible complication", and traditional treatment methods mostly rely on platelet transfusion or delayed surgery. In recent years, the International Society of Liver Diseases has gradually included TPO receptor agonists in the perioperative management framework.

Avatrombopag is uniquely positioned in that it is not used to correct platelet counts long-term, but as a short-term intervention before elective invasive procedures. Multiple overseas real-world data suggest that using avatrombopag within a reasonable time window can significantly reduce the need for perioperative platelet transfusion and reduce transfusion-related adverse events.

This concept has also gradually influenced domestic clinical practice, making "planned plate elevation" an important management strategy for patients with chronic liver disease before undergoing invasive procedures.

Position changes in the treatment pathway for immune thrombocytopenia

In the field of ITP, the treatment goal has already shifted from "pursuing normal platelets" to "reducing the risk of bleeding and improving quality of life." Avatrombopag is considered in this context as one of the oral options suitable for long-term management.

Compared with traditional glucocorticoids or immunosuppressants, avatrombopag does not directly interfere with the immune system, but does so by promoting platelet production"Functional compensation". This mechanism gives it certain advantages in patients with hormone-dependent or recurrent ITP, and is especially suitable for long-term management people who want to reduce hormone-related adverse reactions.

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ChildrenNew international trends in ITP treatment

It is worth noting that avatrombopag has been approved overseas for use in children 1 year old and above with persistent or chronic ITP. Compared with injectable TPO‑RA, oral administration has obvious advantages in children’s compliance.

In recent years, the European and American pediatric hematology community has emphasized more the "minimum intervention principle", that is, avoiding over-treatment while controlling the risk of bleeding. The dose adjustability and oral characteristics of avatrombopag make it easier to integrate into this concept, and also provide an important reference for the future expansion of domestic indications for children.

Clinical Interpretation of Adverse Reactions: How to UnderstandThe meaning behind “incidence”

Judging from the published data, the spectrum of adverse reactions of avatrombopag varies among different groups of people. In patients with chronic liver disease, fever, abdominal discomfort and edema are the main symptoms; in ITP patients, symptoms related to headache, fatigue and mild bleeding are more common. Most of these adverse reactions are mild to moderate, and often clinically overlap with the underlying disease itself. International experts generally recommend that the assessment of adverse reactions be placed within an "overall risk management" framework rather than looking at a symptom in isolation.

Differential comparison of avatrombopag and otherTPO receptor agonists

Among similar drugs, avatrombopag is often compared with eltrombopag, romiplostim, etc. The main differences are in the mode of administration, effects of food, and risk of drug interactions. Avatrombopag is not significantly affected by a high-calcium diet and does not require complex dietary restrictions, which significantly improves the feasibility of long-term treatment for patients in the real world. For this reason, some overseas guidelines have listed it as one of the priority oralTPO‑RAs.

New challenges brought about by real-world research and changes in health insurance policies

As avatrombopag enters the domestic medical insurance system, its user population is expanding, but it also faces practical problems such as indication restrictions and review of reimbursement conditions. It is understood that the common specifications of avatrombopag sold in China are 20mg*15 tablets and 20mg*10 tablets. The price per box may range from 5,000 to 7,000 yuan, and the price may be reduced after medical insurance reimbursement.

In addition, the emergence of overseas generic drugs has also triggered discussions on drug accessibility and economic burden at the patient level. Its drug ingredients are basically the same as those of original drugs sold domestically and abroad. For example, the price of Laos pharmaceutical production specifications of 20mg*30 tablets per box may be more than 400 yuan (the price may fluctuate due to the exchange rate).

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From guidelines to practice: future application prospects of avatrombopag

Based on the current international research trends, it is foreseeable that the application scenarios of avatrombopag are expected to be further expanded, including potential areas such as chemotherapy-related thrombocytopenia. As more real-world data accumulate, its long-term safety and benefit patterns in different populations will become clearer.

For clinicians, understanding avatrombopag is not only "Will it be used?" but also a systemic issue of "when to use it, how long to use it, and how to stop it." For patients, it is also crucial to scientifically understand drug positioning and avoid blind pursuit of platelet values.

Avatrombopag is the world's first oral TPO-RA approved for the treatment ofCLD-related thrombocytopenia, and its clinical and economic value will be fully released in 2026. From cutting the price of original drugs to controllable quality of generic drugs, from cross-indication breakthroughs to personalized treatment, avatrombopag is leading the treatment of thrombocytopenia into a new era of "safe, effective and affordable".

References:

American Society of Hematology (ASH) ITP Guidelines

European Association for the Study of the Liver (EASL) Clinical Practice Guidelines

https://go.drugbank.com/drugs/DB11995

https://doptelet.com/home/

https://www.drugs.com/avatrombopag.html