Outcomes and prognostic factors of hematopoietic stem cell transplantation in adult patients with acute myeloid leukemia and central nervous system involvement.
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OBJECTIVE: Acute myeloid leukemia (AML) with central nervous system (CNS) involvement is rare and associated with a poor prognosis, but allogeneic hematopoietic stem cell transplantation (HSCT) offers the potential for a cure. However, comprehensive data on clinical characteristics, prognosis, and post-transplant CNS complications remain limited. METHODS: We conducted a retrospective analysis of 64 CNS+AML patients who underwent allogeneic HSCT at Toranomon Hospital and Toranomon Hospital Kajigaya. RESULTS: CNS involvement was limited to the cerebrospinal fluid in 64%, while 36% had intracranial mass lesions. All patients received intrathecal chemotherapy, and 40.6% underwent cranial irradiation. Refined Disease Risk Index (rDRI) was low-intermediate in 25% and high-very high in 75%, and 56.2% had active CNS disease at transplantation. The 2-year DFS rate was 34.8%, and the 2-year cumulative incidence of relapse (CIR) was 35.2%. High or very high rDRI and poor performance status were associated with inferior outcomes in the multivariate analysis. The CIR of CNS disease was 12.7%, with a trend toward higher rates (18.8%) observed in patients with high/very high rDRI. Post-transplant CNS complications included two cases of leukoencephalopathy within 1 month and two cases of radiation-induced brain necrosis approximately 19 months after transplantation. CONCLUSIONS: Outcomes after HSCT for CNS+ AML are primarily determined by rDRI and performance status, underscoring the importance of systemic disease control.