Comparison of clinical outcomes between preemptive and maintenance therapy after allogeneic hematopoietic stem cell transplantation for acute myeloid leukemia

• Published in: International journal of hematology
• Main Authors: Konishi, Hiroaki, Tachibana, Takayoshi, Arai, Shota, Izumi, Akihiko, Takeda, Takaaki, Hirose, Natsuki, Nukui, Jun, Sato, Shuku, Suzuki, Taisei, Ishii, Ryuji, Yamazaki, Etsuko, Matsunawa, Manabu, Mukae, Junichi, Takahata, Atsushi, Tanaka, Masatsugu, Suzuki, Takahiro, Nakajima, Hideaki
• Format: Journal Article
• Language: English
• Published: Japan 23.05.2025
• Subjects: Allogeneic stem cell transplantation; Maintenance therapy; WT1; Acute myeloid leukemia; Preemptive therapy
• ISSN: 0925-5710; 1865-3774; 1865-3774
• DOI: 10.1007/s12185-025-04009-w

This study aimed to compare the efficacy and safety of preemptive and maintenance therapies as post-transplant therapy for acute myeloid leukemia (AML). Patients with AML who underwent allogeneic stem cell transplantation and received post-transplant therapy were eligible. Preemptive therapy was initiated if elevated Wilms' tumor-1 mRNA was detected in the peripheral blood. Twenty-nine patients received either preemptive (n = 12) or maintenance therapy (n = 17). The median age was 56 years (range, 18-70). The median time from transplantation to intervention was 77 days (range, 43-203) for maintenance and 346 days (range, 104-1027) for preemptive therapy. Maintenance therapy consisted of azacitidine (AZA) monotherapy in six patients, venetoclax (VEN) + AZA in five, and VEN + cytarabine (AraC) in six. Preemptive therapy consisted of AZA monotherapy in two patients, VEN + AZA in nine, and VEN + AraC in one. One-year overall survival from intervention was 92% for maintenance and 73% for preemptive therapy (P = 0.28), and 1-year event-free survival was 83% and 66%, respectively (P = 0.29). No serious adverse events or treatment-related mortality were observed in either group. Both therapies were safe and effective in preventing disease relapse. A prospective study comparing these two groups is warranted.