Efficacy of single-agent lenalidomide in patients with JAK2 (V617F) mutated refractory anemia with ring sideroblasts and thrombocytosis

• Published in: Blood Vol. 116; no. 2; pp. 180 - 182
• Main Authors: Huls, Gerwin, Mulder, André B., Rosati, Stefano, van de Loosdrecht, Arjan A., Vellenga, Edo, de Wolf, Joost T.M.
• Format: Journal Article
• Language: English
• Published: Washington, DC Elsevier Inc 15.07.2010; Americain Society of Hematology
• Subjects: Aged, 80 and over; Anabolic Agents - therapeutic use; Anemia, Refractory - drug therapy; Anemia, Refractory - genetics; Anemia, Sideroblastic - drug therapy; Anemia, Sideroblastic - genetics; Antineoplastic Agents - therapeutic use; Biological and medical sciences; Erythropoietin - therapeutic use; Hematologic and hematopoietic diseases; Humans; Hypertension, Pulmonary - complications; Janus Kinase 2 - genetics; Lenalidomide; Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis; Male; Medical sciences; Middle Aged; Mutation; Platelet diseases and coagulopathies; Pulmonary Embolism - complications; Pyridoxine - therapeutic use; Reverse Transcriptase Polymerase Chain Reaction; Thalidomide - analogs & derivatives; Thalidomide - therapeutic use; Thrombocytosis - drug therapy; Thrombocytosis - genetics; Vitamin B Complex - therapeutic use; Antineoplastic agent; Human; Refractory anemia with ringed sideroblasts; Hematology; Lenalidomide; Enzyme; JAK2 protein tyrosine kinase; Transferases; Treatment efficiency; Malignant hemopathy; Myelodysplastic syndrome; Platelet; Thrombocytosis; Mutation; Protein-tyrosine kinase; Cancer
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood-2010-01-263087

Patients with refractory anemia with ring sideroblasts and thrombocytosis (RARS-T) are difficult to treat because the cytoreductive treatment might be beneficial for the thrombocytosis component but harmful for the RARS component. As lenalidomide has shown to be efficacious in both myelodysplastic syndromes and myeloproliferative neoplasms, we have treated 2 RARS-T patients, who were transfusion dependent, with lenalidomide. We report the results of lenalidomide treatment in these patients and show that lenalidomide has clinical activity in this rare disorder. Both patients became transfusion independent, and 1 of the patients attained indeed a complete molecular remission.