Vol 13, No 1 (2022)
Case report
Published online: 2022-03-31

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Midostaurin in the treatment of a patient with acute myeloid leukaemia with FLT3-TKD mutation and NPM1 mutation

Aleksander Salomon-Perzyński1, Urszula Walczak1, Kinga Kos-Zakrzewska1, Ewa Lech-Marańda1, Bożena Katarzyna Budziszewska1
Hematology in Clinical Practice 2022;13(1):37-40.


Acute myeloid leukaemia (AML) is aggressive cancer with a diverse clinical course which mainly results from the heterogeneous and complex molecular landscape of the disease. For some genetic alterations, in particular mutations in the FMS-like tyrosine kinase 3 (FLT3) gene, the prognosis of patients with newly diagnosed AML depends on the coexistence of other genetic disorders. A thorough understanding of the interactions between mutations is key to improving risk stratification in newly diagnosed AML and personalizing therapy. The addition of midostaurin, an FLT3 tyrosine kinase inhibitor, to standard chemotherapy improved outcomes in the unfavourable prognostic group of AML patients. This research describes a therapeutic strategy in a patient with newly diagnosed AML with FLT3-tyrosine kinase domain (TKD) mutation and wild-type NPM1 who has received a standard remission induction and consolidation chemotherapy in combination with midostaurin, followed by maintenance therapy with an FLT3 inhibitor and haploidentical allogeneic hematopoietic stem cell transplantation.

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  1. Herold T, Rothenberg-Thurley M, Grunwald VV, et al. Validation and refinement of the revised 2017 European LeukemiaNet genetic risk stratification of acute myeloid leukemia. Leukemia. 2020; 34(12): 3161–3172.
  2. Döhner H, Estey E, Grimwade D, et al. Diagnosis and management of AML in adults: 2017 ELN recommendations from an international expert panel. Blood. 2017; 129(4): 424–447.
  3. Lewandowski K. Znaczenie prognostyczne zaburzeń molekularnych u chorych na ostrą białaczkę szpikową z prawidłowym kariotypem. Hematologia. 2012; 3(3): 231–242.
  4. Daver N, Schlenk RF, Russell NH, et al. Targeting FLT3 mutations in AML: review of current knowledge and evidence. Leukemia. 2019; 33(2): 299–312.
  5. Boddu P, Kantarjian H, Borthakur G, et al. Co-occurrence of -TKD and mutations defines a highly favorable prognostic AML group. Blood Adv. 2017; 1(19): 1546–1550.
  6. Voso MT, Larson RA, Jones D, et al. Midostaurin in patients with acute myeloid leukemia and FLT3-TKD mutations: a subanalysis from the RATIFY trial. Blood Adv. 2020; 4(19): 4945–4954.
  7. Bacher U, Haferlach C, Kern W, et al. Prognostic relevance of FLT3-TKD mutations in AML: the combination matters--an analysis of 3082 patients. Blood. 2008; 111(5): 2527–2537.
  8. Bullinger L. Novel insights into genomic classification and prognosis in acute myeloid leukemia based on a pan-European public-private partnership, the Harmony Alliance. EHA Library. https://library.ehaweb.org/eha/2020/eha25th/294950 (May 24, 2022).
  9. Tallman MS, Wang ES, Altman JK, et al. OCN. Acute Myeloid Leukemia, Version 3.2019, NCCN Clinical Practice Guidelines in Oncology. J Natl Compr Canc Netw. 2019; 17(6): 721–749.
  10. Heuser M, Ofran Y, Boissel N, et al. ESMO Guidelines Committee. Electronic address: clinicalguidelines@esmo.org. Acute myeloid leukaemia in adult patients: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2020; 31(6): 697–712.
  11. Stone RM, Mandrekar SJ, Sanford BL, et al. Midostaurin plus chemotherapy for acute myeloid leukemia with a FLT3 mutation. N Engl J Med. 2017; 377(5): 454–464.
  12. Patkowska E, Szczepaniak A, Barańska M, et al. Primary and secondary central nervous system involvement in acute myeloid leukemia. Journal of Leukemia. 2019; 07(02).
  13. Alakel N, Stölzel F, Mohr B, et al. Symptomatic central nervous system involvement in adult patients with acute myeloid leukemia. Cancer Manag Res. 2017; 9: 97–102.
  14. Bazarbachi A, Bug G, Baron F, et al. Clinical practice recommendation on hematopoietic stem cell transplantation for acute myeloid leukemia patients with -internal tandem duplication: a position statement from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation. Haematologica. 2020; 105(6): 1507–1516.

Hematology in Clinical Practice