Antiretroviral Activity of 5-azacytidine During Treatment of a HTLV-1 Positive Myelodysplastic Syndrome With Autoimmune Manifestations

Virol J. 2012 Jan 3;9:1. doi: 10.1186/1743-422X-9-1.


Myelodysplastic syndromes (MDS) are often accompanied by autoimmune phenomena. The underlying mechanisms for these associations remain uncertain, although T cell activation seems to be important. Human T-lymphotropic virus (HTLV-1) has been detected in patients with myelodysplastic syndromes, mostly in regions of the world which are endemic for the virus, and where association of HTLV-1 with rheumatological manifestation is not rare. We present here the case of a 58 year old man who presented with cytopenias, leukocytoclastic vasculitis of the skin and glomerulopathy, and was diagnosed as MDS (refractory anemia with excess blasts - RAEB 1). The patient also tested positive for HTLV-1 by PCR. After 8 monthly cycles of 5-azacytidine he achieved a complete hematologic remission. Following treatment, a second PCR for HTLV-1 was carried out and found to be negative. This is the first report in the literature of a HTLV-1-positive MDS with severe autoimmune manifestations, which was treated with the hypomethylating factor 5-azacitidine, achieving cytogenetic remission with concomitant resolution of the autoimmune manifestations, as well as HTLV-1-PCR negativity. HTLV-1-PCR negativity may be due to either immune mediated clearance of the virus, or a potential antiretroviral effect of 5-azacytidine. 5-azacytidine is known for its antiretroviral effects, although there is no proof of its activity against HTLV-1 infection in vivo.

Publication types

  • Case Reports

MeSH terms

  • Anti-Retroviral Agents / administration & dosage*
  • Antimetabolites, Antineoplastic / administration & dosage*
  • Autoimmune Diseases / drug therapy*
  • Azacitidine / administration & dosage*
  • HTLV-I Infections / drug therapy*
  • Human T-lymphotropic virus 1 / drug effects*
  • Human T-lymphotropic virus 1 / genetics
  • Human T-lymphotropic virus 1 / isolation & purification
  • Humans
  • Male
  • Middle Aged
  • Myelodysplastic Syndromes / drug therapy*
  • Polymerase Chain Reaction
  • Treatment Outcome


  • Anti-Retroviral Agents
  • Antimetabolites, Antineoplastic
  • Azacitidine