Comparing Precision Therapy to Standard Treatment for Acute Myeloid Leukemia and Myelodysplastic Syndrome in Older Adults

What this Trial Is About

Researchers are investigating whether precision therapy guided by a Clinical Tumour Board is more cost-effective and beneficial compared to standard treatment for elderly patients with acute myeloid leukemia AML and high-risk myelodysplastic syndrome MDS. This phase II, randomized clinical trial focuses on patients aged 60 and older who are not eligible for intensive chemotherapy or stem cell transplant. Precision therapy is tailored based on genetic changes found in the tumor using next generation sequencing NGS. Participants first receive two 28-day cycles of standard treatment, which for AML includes azacitidine plus venetoclax if available, or azacitidine alone for MDS patients. During this initial treatment, bone marrow samples are analyzed by the Tumour Board to determine suitable targeted therapies for the precision therapy group. After initial treatment, patients without disease progression are randomized in a 21 ratio to either continue standard therapy or receive precision therapy combined with standard treatment. Throughout the 4 to 5 years of follow-up, patients undergo regular assessments including bone marrow evaluations, genomic profiling, and monitoring of treatment response, adverse events, quality of life, and healthcare costs. The study measures cost-effectiveness as the primary outcome, alongside survival, toxicity, transfusion dependence, and progression. Safety and efficacy are carefully tracked to understand the impact of personalized precision therapy in this elderly population.

Precision Therapy Versus Standard Therapy in AML and MDS in Elderly