Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Case Reports
. 2025 Mar 11;7(1):40-46.
doi: 10.46989/001c.131722. eCollection 2025.

Therapeutics of acute myeloid leukemia with central nervous system involvement

Edwin U Suárez  1 , Tamara Castaño-Bonilla  1 , Rocio Salgado  1 , Laura Solán  1 , Alberto Lázaro-García  1 , Juan M Alonso-Domínguez  1
Affiliations
Case Reports

Therapeutics of acute myeloid leukemia with central nervous system involvement

Edwin U Suárez et al. Clin Hematol Int. .

Abstract

FLT3-mutated acute myeloid leukemia (AML) with central nervous system (CNS) involvement poses therapeutic challenges. We describe two cases and performed a systematic review evaluating the efficacy of therapeutic strategies in CNS involvement for both FLT3-mutated and wild-type (WT) AML. A MEDLINE, EMBASE, and Cochrane literature search identified relevant studies. Although CNS involvement in AML is associated with poor prognosis, routine CNS prophylaxis is not standard. Due to the uncertainty regarding the effect of intermediate doses of cytarabine on CNS involvement, we support a diagnostic lumbar puncture (LP) after achieving complete remission in patients with risk factors for CNS infiltration. Consolidation management should be modified depending on the result of the LP. The impact of total body irradiation (TBI) as a conditioning regimen in allogeneic stem cell transplantation on CNS AML outcomes remains ambiguous. Routine craniospinal irradiation is not recommended due to its associated higher morbidity rates, while cranial radiotherapy is preferred, particularly when combined with TBI. Fortunately, currently we can employ a FLT3 inhibitor with CNS penetrance in FLT3-mutated (either gilteritinib or sorafenib) or FLT3-WT (sorafenib) AML patients.

Keywords: FLT3; acute myeloid leukemia; extramedullary manifestation of AML; refractory central nervous system leukemia; sorafenib.

PubMed Disclaimer

Conflict of interest statement

No competing interests were disclosed.

References

    1. Myeloid sarcoma, chloroma, or extramedullary acute myeloid leukemia tumor: A tale of misnomers, controversy and the unresolved. Shallis R. M., Gale R. P., Lazarus H. M., Roberts K. B., Xu M. L.., et al. May;2021 Blood Rev. 47:100773. doi: 10.1016/j.blre.2020.100773. - DOI - PubMed
    1. CNS involvement in AML at diagnosis is rare and does not affect response or survival: data from 11 ECOG-ACRIN trials. Ganzel C., Lee J. W., Fernandez H. F., Paietta E. M., Luger S. M.., et al. Nov 23;2021 Blood Adv. 5(22):4560–4568. doi: 10.1182/bloodadvances.2021004999. - DOI - PMC - PubMed
    1. Neurologic complications of acute myeloid leukemia. Diagnostic approach and therapeutic modalities. Siegal T., Benouaich-Amiel A., Bairey O. May;2022 Blood Rev. 53:100910. doi: 10.1016/j.blre.2021.100910. - DOI - PubMed
    1. Factors associated with risk of central nervous system relapse in patients with non-core binding factor acute myeloid leukemia. Jabbour E., Guastad Daver N., Short N. J., Huang X., Chen H. C.., et al. Sep;2017 Am J Hematol. 92(9):924–928. doi: 10.1002/ajh.24799. - DOI - PMC - PubMed
    1. Central Nervous System Involvement in Adults with Acute Leukemia: Diagnosis, Prevention, and Management. Paul S., Short N. J. Apr;2022 Curr Oncol Rep. 24(4):427–436. doi: 10.1007/s11912-022-01220-4. - DOI - PubMed

Publication types

LinkOut - more resources