High throughput screening aids clinical decision‐making in refractory acute myeloid leukaemia

Abstract

Abstract Background Despite advances in therapeutics for adverse‐risk acute myeloid leukaemia (AML), overall survival remains poor, especially in refractory disease. Comprehensive tumour profiling and pre‐clinical drug testing can identify effective personalised therapies. Case We describe a case of ETV6‐MECOM fusion‐positive refractory AML, where molecular analysis and in vitro high throughput drug screening identified a tolerable, novel targeted therapy and provided rationale for avoiding what could have been a toxic treatment regimen. Ruxolitinib combined with hydroxyurea led to disease control and enhanced quality‐of‐life in a patient unsuitable for intensified chemotherapy or allogeneic stem cell transplantation. Conclusion This case report demonstrates the feasibility and role of combination pre‐clinical high throughput screening to aid decision making in high‐risk leukaemia. It also demonstrates the role a JAK1/2 inhibitor can have in the palliative setting in select patients with AML.

Keywords

• acute myeloid leukaemia
• case report
• precision medicine
• pre‐clinical
• ruxolitinib