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Phase II study of roquinimex in myelodysplastic syndrome
C S Rosenfeld1, Z R Zeigler, R K Shadduck
1Texas Oncology, P.A., Dallas 75230, U.S.A.
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.
Insights
Roquinimex showed occasional benefit for myelodysplastic syndromes (MDS) patients, with transient improvements in blood counts. One patient achieved a durable complete remission, but side effects were common.
Area of Science:
- Immunology
- Hematology
- Clinical Pharmacology
Background:
- Myelodysplastic syndromes (MDS) are a group of clonal hematopoietic stem cell disorders.
- Roquinimex (Linomide) is an orally active immunomodulatory agent with demonstrated in vitro and clinical activity.
- Investigating novel therapeutic agents for MDS is crucial due to limited treatment options.
Purpose of the Study:
- To evaluate the efficacy and safety of roquinimex in patients with myelodysplastic syndromes.
- To assess the impact of roquinimex on hematologic and immunologic parameters in MDS patients.
Main Methods:
- A Phase II clinical trial was conducted with 17 patients diagnosed with myelodysplastic syndromes.
- Patients received oral roquinimex twice weekly for 12 weeks.
- Clinical, hematologic, and immunologic parameters were monitored frequently.
Main Results:
- An increase in CD8+ and CD56+/CD3- cells was observed within 3 weeks.
- Transient improvements in neutrophil and platelet counts were noted in some patients.
- One patient with RAEB achieved a complete remission lasting over 14 months post-therapy.
- Adverse events, including arthralgia and myalgia, occurred in over 25% of patients, leading to treatment withdrawal in some.
Conclusions:
- Roquinimex demonstrated occasional, transient hematologic benefits in myelodysplastic syndromes patients.
- The immunomodulatory effects, including increased CD8+ and CD56+ cells, warrant further investigation.
- Adverse events necessitate careful monitoring and patient selection for roquinimex therapy.