PHASE-II STUDY OF ROQUINIMEX IN MYELODYSPLASTIC SYNDROME

A Phase II clinical trial was undertaken using roquinimex (Linomide) i n patients with myelodysplastic syndromes (MDS). Roquinimex is an oral ly active drug with immunostimulating activities demonstrated in vitro and clinically. Seventeen patients with MDS were enrolled in the stud y. Eligibility was limited to cytopenic patients with <20% marrow blas ts. The drug was given orally twice weekly for 12 weeks with Frequent monitoring of clinical, hematologic, and immunologic parameters. An in crease in CD8(+) and CD56(+)/CD3(-) cells was detected by 3 weeks. The re was, however, no augmentation of natural killer or lymphokine-activ ated killer cell activity; progenitor cells were unchanged. Four patie nts had improvement in neutrophil counts, and two patients had improve ment in platelet counts. Despite this improvement, the responses were transient or not maintained after discontinuation of therapy. One pati ent with RAEB, who was red cell transfusion dependent, experienced a c omplete remission that has persisted 14 months after completion of the rapy. Adverse events developed in >25% of patients and included arthra lgia, fever, headache, and myalgia. These side effects led to early wi thdrawal of therapy in five patients. These findings suggest that roqu inimex may be of occasional benefit to patients with myelodysplastic s yndromes.