title: Phase II study of roquinimex in myelodysplastic syndrome.
abstract: A Phase II clinical trial was undertaken using roquinimex (Linomide) in patients with myelodysplastic syndromes (MDS). Roquinimex is an orally active drug with immunostimulating activities demonstrated in vitro and clinically. Seventeen patients with MDS were enrolled in the study. Eligibility was limited to cytopenic patients with <20% marrow blasts. The drug was given orally twice weekly for 12 weeks with frequent monitoring of clinical, hematologic, and immunologic parameters. An increase in CD8+ and CD56+/CD3- cells was detected by 3 weeks. There was, however, no augmentation of natural killer or lymphokine-activated killer cell activity; progenitor cells were unchanged. Four patients had improvement in neutrophil counts, and two patients had improvement in platelet counts. Despite this improvement, the responses were transient or not maintained after discontinuation of therapy. One patient with RAEB, who was red cell transfusion dependent, experienced a complete remission that has persisted 14 months after completion of therapy. Adverse events developed in >25% of patients and included arthralgia, fever, headache, and myalgia. These side effects led to early withdrawal of therapy in five patients. These findings suggest that roquinimex may be of occasional benefit to patients with myelodysplastic syndromes.
authors.affiliation: Texas Oncology, P.A., Dallas 75230, U.S.A.
authors.forename: C S
authors.initials: CS
authors.lastname: Rosenfeld
authors.forename: Z R
authors.initials: ZR
authors.lastname: Zeigler
authors.forename: R K
authors.initials: RK
authors.lastname: Shadduck
authors.forename: B
authors.initials: B
authors.lastname: Nilsson
mesh_terms: D000276:Adjuvants, Immunologic / Q000627:therapeutic use* / Q000633:toxicity; D000368:Aged; D005260:Female; D006410:Hematopoiesis / Q000187:drug effects*; D006801:Humans; D006912:Hydroxyquinolines / Q000627:therapeutic use* / Q000633:toxicity; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000188:drug therapy*; D011446:Prospective Studies
chemical_list: D000276:Adjuvants, Immunologic; D006912:Hydroxyquinolines; C045282:roquinimex