ASH 2021 | Lenalidomide and eltrombopag in patients with low or intermediate-risk MDS with thrombocytopenia

There is currently no FDA-approved therapy for patients with myelodysplastic syndromes (MDS) with thrombocytopenia. Lenalidomide is approved in patients with MDS with chromosome 5q deletion (del(5q)) where it has been shown to induce disease remission and transfusion independence in over half of patients with del(5q), and in one-quarter of patients without del(5q). Nevertheless, its use is limited due to its megakaryopoietic effect. Pre-clinical studies have shown that eltrombopag can reverse this effect. Jesus D. Gonzalez-Lugo, MD, Albert Einstein College of Medicine, Montefiore Medical Center, Bronx, NY, discusses the design and findings of a Phase II study evaluating the safety and efficacy of eltrombopag and lenalidomide in low and intermediate-risk MDS (NCT01772420). Patients were divided into two arms based on their platelet counts. Arm A was treated with lenalidomide alone until their platelet count fell below 50,000, after which they were switched to eltrombopag until their platelets went above this number. Lenalidomide treatment was then resumed. If their platelet count fell below this number again, they received eltrombopag again and followed with lenalidomide plus eltrombopag. Arm B received eltrombopag alone until their platelet count increased above 50,000 and then received treatment as in Arm A. The study reported overall responses rates (ORR) of 32% in arm A and 39% in arm B. Interestingly, 29% of patients treated with eltrombopag achieved bilineage responses and thus continued eltrombopag monotherapy. 14% of patients treated with eltrombopag plus lenalidomide achieved a bilineage response. Overall, these results suggest this combination as a promising option for patients with MDS with thrombocytopenia, but it needs to be further investigated in a larger trial. This interview took place at the 63rd ASH Annual Meeting and Exposition congress in Atlanta, GA.