Allogeneic hematopoietic cell transplantation in intermediate risk acute myeloid leukemia negative for FLT3-ITD, NPM1- or biallelic CEBPA mutations

作者：Heidrich K^*; Thiede C; Schaefer Eckart K; Schmitz N; Aulitzky W E; Kraemer A; Roesler W; Haenel M; Einsele H; Baldus C D; Trappe R U; Stoelzel F; Middeke J M; Roellig C; Taube F; Kramer M; Serve H; Berdel W E; Ehninger G; Bornhaeuser M; Schetelig J

来源：Annals of Oncology, 2017, 28(11): 2793-2798.

DOI：10.1093/annonc/mdx500

• hematopoietic stem cell transplantation
• acute myeloid leukemia
• postremission therapy
• CEBPA
• FLT3-ITD
• NPM1

The value of allogeneic hematopoietic cell transplantation (alloHCT) as postremission treatment is not well defined for patients with intermediate-risk acute myeloid leukemia (AML) without FLT3-ITD, biallelic CEBPA-, or NPM1 mutations (here referred to as NPM1(mut-neg)/CEBPA(dm-neg)/FLT3-ITDneg AML) in first complete remission (CR1).
We addressed this question using data from two prospective randomized controlled trials on intensive induction- and risk-stratified postremission therapy. The NPM1(mut-neg)/CEBPA(dm-neg)/FLT3-ITDneg AML subgroup comprised 497 patients, aged 18-60 years.
In donor versus no-donor analyses, patients with a matched related donor had a longer relapse-free survival (HR 0.5; 95% CI 0.3-0.9, P = 0.02) and a trend toward better overall survival (HR 0.6, 95% CI 0.3-1.1, P = 0.08) compared with patients who received postremission chemotherapy. Notably, only 58% of patients in the donor group were transplanted in CR1. We therefore complemented the donor versus no-donor analysis with multivariable Cox regression analyses, where alloHCT was tested as a time-dependent covariate: overall survival (HR 0.58, 95% CI 0.37-0.9, P = 0.02) and relapse-free survival (HR 0.51, 95% CI 0.34-0.76; P = 0.001) for patients who received alloHCT compared with chemotherapy in CR1 were significantly longer.
Outside clinical trials, alloHCT should be the preferred postremission treatment of patients with intermediate risk NPM1(mut-neg)/CEBPA(dm-neg)/FLT3-ITDneg AML in CR1.
NCT00180115, NCT00180102.

• 出版日期2017-11