Pilot study of low-dose interleukin-11 in patients with bone marrow failure

Purpose: Interleukin-11 (IL-11) is a thrombopoietic cytokine that attenuates postchemotherapy thrombocytopenia at doses of 50 μg/kg/d subcutaneously. Very little is known about the activity of IL-11 in patients with bone marrow failure states. Patients and Methods: Our preliminary experience with IL-11 at doses of 50 μg/kg/d suggested that patients with bone marrow failure developed significant peripheral and pulmonary edema after the prolonged dosing necessary for treating these conditions. We, therefore, initiated a study of low-dose IL-11 (starting dose, 10 μg/kg/d). Results: Sixteen patients were assessable for response. Six patients had diploid cytogenetics; the others had a variety of chromosomal abnormalities. Six (38%) of 16 patients showed a platelet response to IL-11, and two had a multilineage response (to IL-11 alone, n = 1; to IL-11 plus G-CSF and erythropoietin, n = 1). The median increase in peak platelet counts was 95 x 10⁹/L above baseline in the responders (range, increase of 55 x 10⁹/L to 130 x 10⁹/L above baseline). Responders included five of 11 patients with myelodysplasia and one of four patients with aplastic anemia. Response durations were 12, 13, 14+, 25, 30, and 30+ weeks. Side effects of IL-11 were mild (peripheral edema, n = 7; conjunctival injection, n = 7; myalgia, n = 1; all grade 1). Seven patients had no side effects. Conclusion: Our pilot study suggests that administration of low-dose IL-11 (10 μg/kg/d) can raise platelet counts without significant toxicity in selected thrombocytopenic patients with bone marrow failure.