WT1-guided pre-emptive therapy after allogeneic hematopoietic stem cell transplantation in patients with acute myeloid leukemia

• Published in: International journal of hematology Vol. 120; no. 3; pp. 337 - 346
• Main Authors: Arai, Shota, Tachibana, Takayoshi, Izumi, Akihiko, Takeda, Takaaki, Tamai, Yotaro, Sato, Shuku, Hashimoto, Chizuko, Fujimaki, Katsumichi, Ishii, Ryuji, Kabasawa, Noriyuki, Hirasawa, Akira, Inoue, Yasuyuki, Tanaka, Masatsugu, Suzuki, Takahiro, Nakajima, Hideaki
• Format: Journal Article
• Language: English
• Published: Singapore Springer Nature Singapore 01.09.2024; Springer Nature B.V
• Subjects: Acute myeloid leukemia; Adolescent; Adult; Aged; Allografts; Chemotherapy; Cytarabine; Female; Hematology; Hematopoietic Stem Cell Transplantation; Hematopoietic stem cells; Humans; Leukemia; Leukemia, Myeloid, Acute - therapy; Male; Medicine; Medicine & Public Health; Middle Aged; mRNA; Neoplasm, Residual; Oncology; Original Article; Retrospective Studies; Stem cell transplantation; Stem cells; Survival; Transplantation; Transplantation, Homologous; WT1 Proteins - genetics; Young Adult; Pre-emptive therapy; Acute myeloblastic leukemia; Disease relapse; WT1
• ISSN: 0925-5710; 1865-3774; 1865-3774
• DOI: 10.1007/s12185-024-03795-z

Measurable residual disease (MRD)-guided pre-emptive therapies are now widely used to prevent post-transplant hematological relapse in patients with acute myeloid leukemia (AML). This single-center retrospective study aimed to clarify the significance of pre-emptive treatment based on Wilms’ tumor gene-1 mRNA (WT1) monitoring for MRD in patients with AML who underwent allogeneic hematopoietic stem cell transplantation (allo-HSCT). Patients with AML who received chemotherapy for hematological relapse or WT1 increase after allo-HSCT were eligible for inclusion. From January 2017 to June 2022, 30 patients with a median age of 57 (16–70) years were included and stratified into two groups: 10 with WT1 increase and 20 with hematological relapse. The median times from HCT to WT1 increase or hematological relapse were 309 days (range: 48–985) or 242 days (range: 67–1116), respectively. Less intensive chemotherapy using azacitidine or cytarabine was selected for all patients with WT1 increase and 12 (60%) with hematological relapse. The 1-year overall survival and event-free survival rates for WT1 increase and hematological relapse were 70% vs. 44% ( P  = 0.024) and 70% vs. 29% ( P  = 0.029), respectively. These real-world data suggest that WT1-guided pre-emptive therapy may be superior to therapy after hematological relapse in patients with AML who have undergone allo-HSCT.