Relapsed and Refractory Acute Myeloid Leukemia (R/R AML) is an aggressive form of leukemia that resists treatment or returns after remission. Its complexity, therapy resistance, and lack of standardized care make it difficult to treat. Learn more about the challenges, current therapies, and future prospects.
Relapsed/Refractory Acute Myeloid Leukemia (R/R AML) refers to a form of AML where the disease either returns after successful initial treatment (relapsed) or does not respond to standard therapies (refractory). It is one of the most challenging haematological cancers to treat, with a 5-year overall survival (rate below 20% in adults and 30–35% in children).
Relapsed and Refractory Acute Myeloid Leukemia (R/R AML) poses significant challenges due to its high molecular and genetic variability, limited therapeutic options, and the lack of a universally accepted standard of care. Therapy resistance and relapse are the primary causes of treatment failure and mortality. Additionally, many therapeutic strategies are influenced by factors like treatment hospital experience, physician preferences, and socio-economic barriers, leading to heterogeneous outcomes.
There is no universal standard of care for Relapsed and Refractory Acute Myeloid Leukemia (R/R AML). Treatments typically include high doses of chemotherapeutics drugs and novel targeted therapies. For elderly or unfit patients, a combination of hypomethylating agents with venetoclax has shown promising Complete Remission rate. Multimodal strategies, including immunotherapy and small molecule inhibitors, aim to achieve deep remissions and serve as a bridge to hematopoietic stem cell transplantation (HSCT), which is currently the only curative strategy.
Standardizing therapies for Relapsed and Refractory Acute Myeloid Leukemia (R/R AML) are challenging due to the disease’s molecular complexity, lack of phase 3 trial data, and heterogeneity in treatment practices across centers and countries. Many novel drugs are initially tested for front-line therapy, leaving limited evidence for their effectiveness in R/R settings. Additionally, differences in reimbursement policies further complicate treatment accessibility.