Pilot study of recombinant human soluble tumor necrosis factor receptor (TNFR:Fc) in patients with low risk myelodysplastic syndrome.

In low risk myelodysplastic syndrome (MDS), increased apoptosis of marrow cells is a reproducible finding. Cytokines may drive this apoptosis. Several studies have demonstrated elevated levels of tumor necrosis factor-alpha (TNF-alpha) in MDS. Soluble tumor necrosis factor receptor (TNFR:Fc) can eliminate biologically active TNF in vivo. This data provided the rationale for a clinical trial of TNFR:Fc in low risk MDS. Eligibility was limited to cytopenic MDS patients with < 10% marrow blasts. Secondary MDS was an exclusion. The study design was to administer 25mg TNFR:Fc twice a week for 10 weeks. Toxicity did not exceed grade 1. No responses were observed in the 10 treated patients and one had disease progression. At this dosing schedule, TNFR:Fc is unlikely to ameliorate cytopenias in low risk MDS.