2026 Korean expert recommendations for the use of thrombopoietin receptor agonists in severe aplastic anemia

초록

The treatment landscape for severe aplastic anemia (SAA) has undergone a significant paradigm shift with the integration of thrombopoietin receptor agonists (TPO-RAs) into standard immunosuppressive therapy (IST). However, clinical practice in Korea is shaped by a distinct regulatory and therapeutic environment characterized by the exclusive availability of rabbit antithymocyte globulin (rabbit ATG) and restrictive reimbursement policies governing TPO-RA use. This expert consensus statement provides a systematic framework and age-stratified treatment algorithms tailored to Korea-specific constraints. For treatment-na & iuml;ve patients with SAA who are not candidates for immediate hematopoietic stem cell transplantation, a combination of rabbit ATG-based IST and TPO-RA is recommended as the first-line strategy. While eltrombopag is supported by robust global data, the panel emphasized the clinical utility of romiplostim in the Korean setting, given its established evidence in rabbit ATG-based regimens and the limited approved dosing of eltrombopag in Korea relative to pivotal global trials. The consensus also provides practical guidance on TPO-RA initiation, retreatment, and switching in refractory or relapsed disease, while emphasizing careful evaluation of clonal evolution in patients with insufficient response or newly emerging hematologic abnormalities, regardless of the specific TPO-RA used. Ultimately, these recommendations aim to standardize SAA management in Korea, optimize patient outcomes, and support future policy refinements to better align domestic practices with evolving global evidence.

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