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Allogeneic Stem Cell Transplantation Prolongs Recurrence-Free Survival in NPM1-Mutant AML

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Key Points

  • Three-year recurrence-free survival was 71% in the donor group vs 47% in the no-donor group.
  • In patients with normal karyotype and no FLT3 internal tandem duplication, 3-year recurrence-free survival was 83% vs 53%.

In an analysis of the Study Alliance Leukemia (SAL) AML 2003 trial reported in the Journal of Clinical Oncology, Röllig et al found that allogeneic stem cell transplantation significantly prolonged recurrence-free survival in patients with NPM1-mutant acute myeloid leukemia (AML). NPM1 mutation is associated with more favorable prognosis in AML.

The current study involved 304 patients in the SAL-AML 2003 trial (N = 1,179, aged 18–60 years) who had NPM1-mutant disease with an intermediate-risk karyotype. Of these, 77 had an available HLA-identical sibling donor and were intended to receive allogeneic stem cell transplantation; 227 had no available matched family donor and received consolidation therapy or autologous stem cell transplantation.

Improved Recurrence-Free Survival

Three-year recurrence-free survival was 71% in the donor group vs 47% in the no-donor group (P = .005); 3-year overall survival was 70% vs 60% (P = .114). Among 148 patients with normal karyotype and no FLT3 internal tandem duplication, 3-year recurrence-free survival was 83% vs 53% (P = .004), and 3-year overall survival was 81% vs 75% (P = .300).

The investigators concluded: “Allogeneic [stem cell transplantation] led to a significantly prolonged [recurrence-free survival] in patients with NPM1[-mutant] AML. The absence of a statistically significant difference in [overall survival] is most likely a result of the fact that NPM1[-mutant] patients who experienced relapse responded well to salvage treatment. Allogeneic [stem cell transplantation] in first remission has potent antileukemic efficacy and is a valuable treatment option in patients with NPM1[-mutant] AML with a sibling donor.”

Christoph Röllig, MD, of Universitätsklinikum “Carl Gustav Carus,” Dresden, is the corresponding author for the Journal of Clinical Oncology article.

Christian Thiede reported an employment or leadership position and stock ownership with AgenDix GmbH.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.


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