Patients with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndromes (MDS) do not respond well to standard chemotherapy and have a poor outcome if they harbor TP53 mutations. Surprisingly, this patient population was found to be exceptionally sensitive to treatment with the chemotherapeutic drug Decitabine, with a response rate of 100% (21 of 21 patients) compared to only 41% (32 of 78) in patients with wild-type TP53. Although no complete mutation clearance was observed and responses were therefore not long lasting, these results nevertheless represent a first encouraging step towards being able to provide improved therapeutic options for this high-risk patient subgroup.
Welch JS., et al., N Engl J Med., 375:2023-2036, 2016.DOI: 10.1056/NEJMoa1605949
2. Laurent-Puig., et al., ESMO 2016 abstract 4590:
Annals of Oncology 27 (Supplement 6): vi149–vi206, 2016. DOI: 10.1093/annonc/mdw370.8
3. The New England Journal of Medicine: