4.2.3 Treatment options – refractory disease

Resistance to therapy (refractory AML) is the major cause of treatment failure, rather than mortality due to infections and other treatment-related complications. Patients failing to respond to one or two cycles of induction treatment can be considered chemotherapy refractory and are at very high risk of ultimate treatment failure. In this circumstance other alternatives should be explored (non-chemotherapy options or clinical trials). While there are no standard salvage regimens for AML, intensive salvage chemotherapy can result in a second remission in approximately 55 per cent of patients aged 16–49, of which approximately two-thirds can then proceed to an allo-SCT (Döhner et al. 2017).

Patients offered an allo-SCT are carefully selected and must have an appropriately HLA-matched donor. It should be noted that patients with refractory disease who undergo an allo-SCT have limited chances of success and considerable morbidity from this procedure. For patients unsuited to this approach, palliative systemic treatment is often a reasonable option with limited toxic effects (Döhner et al. 2010).