Raza A et al. (2008) Phase 2 study of lenalidomide in transfusion-dependent, low-risk, and intermediate-1 risk myelodysplastic syndromes with karyotypes other than deletion 5q. Blood 111: 86–93

Lenalidomide is approved in the US for the treatment of transfusion-dependent anemia in patients who have low-risk or intermediate-1-risk myelodysplastic syndromes (MDS) with a chromosome 5q interstitial deletion. A multicenter phase II study has now shown that lenalidomide is effective in treating patients who have MDS with normal karyotypes or cytogenetic abnormalities other than 5q deletion.

The study included 214 patients, predominantly with low-risk or intermediate-1-risk MDS without deletion 5q, who had transfusion-dependent anemia. The patients initially received lenalidomide either at 10 mg daily for 21 days of a 28-day cycle or at 10 mg daily (n = 114 and n = 100, respectively). Sustained improvement for at least eight consecutive weeks was seen in 93 patients; 56 patients achieved transfusion independence together with a ≥10 g/l (1 g/dl) peak rise in hemoglobin level (median rise in hemoglobin at maximal improvement 32 g/l [3.2 g/dl]), and 37 patients had a ≥50% reduction in transfusions. The median time to transfusion independence was 4.8 weeks (range 1–39 weeks), and transfusion independence lasted for a median of 41 weeks (range 8–136 weeks).

On the basis of these results, the authors conclude that patients with low-risk or intermediate-1-risk MDS who lack the deletion 5q anomaly and have a poor response to erythropoiesis-stimulating agents might benefit from lenalidomide treatment. They recommend, however, that alternative treatments are considered if a patient does not respond to lenalidomide within 4 months.