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<p>The clinical success of <i>FLT3</i> inhibitors has led to their steadily increasing use in the treatment of acute myeloid leukemia (AML), both in the relapsed/refractory setting and as post-transplant maintenance. Despite their expanding application, there is currently no guidance on the optimal duration of therapy or the feasibility of discontinuation. In the maintenance context, current practice is largely based on trial protocols with predefined treatment periods, yet relapses after cessation have been documented. Similarly, in the relapsed/refractory setting, the management of long-term responders to <i>FLT3</i>-directed monotherapy lacks evidence-based guidance.</p><p>We report two cases of <i>FLT3</i>-ITD AML patients with relapse after discontinuation of prolonged <i>FLT3</i> inhibitor therapy, despite sustained remission prior to withdrawal. As such scenarios remain insufficiently characterized in the literature, these case vignettes are presented to highlight the unresolved challenge of defining the appropriate duration of <i>FLT3</i> inhibitor therapy and to underscore the need for systematic evaluation to establish evidence-based strategies for safe discontinuation or extended administration.</p>

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Relapse following FLT3 inhibitor cessation in FLT3-ITD-positive AML: lessons from two clinical cases

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年2月4日

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  • Julia-Annabell Georgi,
  • Christoph Röllig,
  • Johannes Schetelig,
  • Christian Thiede,
  • Sascha Brückmann,
  • Martin Bornhäuser,
  • Jan Moritz Middeke

摘要

The clinical success of FLT3 inhibitors has led to their steadily increasing use in the treatment of acute myeloid leukemia (AML), both in the relapsed/refractory setting and as post-transplant maintenance. Despite their expanding application, there is currently no guidance on the optimal duration of therapy or the feasibility of discontinuation. In the maintenance context, current practice is largely based on trial protocols with predefined treatment periods, yet relapses after cessation have been documented. Similarly, in the relapsed/refractory setting, the management of long-term responders to FLT3-directed monotherapy lacks evidence-based guidance.

We report two cases of FLT3-ITD AML patients with relapse after discontinuation of prolonged FLT3 inhibitor therapy, despite sustained remission prior to withdrawal. As such scenarios remain insufficiently characterized in the literature, these case vignettes are presented to highlight the unresolved challenge of defining the appropriate duration of FLT3 inhibitor therapy and to underscore the need for systematic evaluation to establish evidence-based strategies for safe discontinuation or extended administration.