Safety and efficacy of 5-azacytidine treatment in myelodysplastic syndrome patients with moderate and mild renal impairment

Leuk Res. 2013 Aug;37(8):889-93. doi: 10.1016/j.leukres.2013.05.005. Epub 2013 May 29.


Myelodysplastic syndrome (MDS) patients with renal impairment (RI) were not assessed in the approval trials of 5-azacytidine, thus the optimal use of 5-azacytidine in such patients is currently undefined. We retrospectively analyzed 42 IPSS intermediate-2 and high-risk patients with moderate, mild or no RI undergoing 5-azacytidine therapy in a non-trial setting. We demonstrate that patients in all three groups achieved comparable responses and had similar overall and event-free survival. Likewise, both treatment toxicity and dose adjustments were not significantly influenced by renal function status. A transient but reversible decline in glomerular filtration rate was observed in patients either with or without RI, without affecting the therapeutic schedule. Our results provide the first evidence that 5-azacytidine is effective and well-tolerated in patients with mild and moderate RI and, if confirmed by prospective randomized studies, advocate that such patients can be managed in an analogous fashion to patients with normal renal function.

MeSH terms

  • Aged
  • Aged, 80 and over
  • Antimetabolites, Antineoplastic / adverse effects
  • Antimetabolites, Antineoplastic / therapeutic use
  • Azacitidine / adverse effects
  • Azacitidine / therapeutic use*
  • Disease-Free Survival
  • Drug Administration Schedule
  • Female
  • Glomerular Filtration Rate / drug effects
  • Humans
  • Infections / chemically induced
  • Male
  • Middle Aged
  • Myelodysplastic Syndromes / complications
  • Myelodysplastic Syndromes / drug therapy*
  • Myelodysplastic Syndromes / pathology
  • Neutropenia / chemically induced
  • Remission Induction
  • Renal Insufficiency / complications
  • Renal Insufficiency / drug therapy*
  • Renal Insufficiency / pathology
  • Retrospective Studies
  • Thrombocytopenia / chemically induced
  • Treatment Outcome


  • Antimetabolites, Antineoplastic
  • Azacitidine