title: Efficacy of single-agent lenalidomide in patients with JAK2 (V617F) mutated refractory anemia with ring sideroblasts and thrombocytosis.
abstract: 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.
authors.affiliation: Department of Hematology, University Medical Center Groningen, University of Groningen, Hanzeplein 1, Groningen, The Netherlands. g.huls@int.umcg.nl
authors.forename: Gerwin
authors.initials: G
authors.lastname: Huls
authors.forename: André B
authors.initials: AB
authors.lastname: Mulder
authors.forename: Stefano
authors.initials: S
authors.lastname: Rosati
authors.forename: Arjan A
authors.initials: AA
authors.lastname: van de Loosdrecht
authors.forename: Edo
authors.initials: E
authors.lastname: Vellenga
authors.forename: Joost T M
authors.initials: JT
authors.lastname: de Wolf
mesh_terms: D000369:Aged, 80 and over; D045930:Anabolic Agents / Q000627:therapeutic use; D000753:Anemia, Refractory / Q000188:drug therapy* / Q000235:genetics; D000756:Anemia, Sideroblastic / Q000188:drug therapy* / Q000235:genetics; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D004921:Erythropoietin / Q000627:therapeutic use; D006801:Humans; D006976:Hypertension, Pulmonary / Q000150:complications; D053614:Janus Kinase 2 / Q000235:genetics*; D000077269:Lenalidomide; D008297:Male; D008875:Middle Aged; D009154:Mutation; D011655:Pulmonary Embolism / Q000150:complications; D011736:Pyridoxine / Q000627:therapeutic use; D020133:Reverse Transcriptase Polymerase Chain Reaction; D013792:Thalidomide / Q000031:analogs & derivatives* / Q000627:therapeutic use; D013922:Thrombocytosis / Q000188:drug therapy* / Q000235:genetics; D014803:Vitamin B Complex / Q000627:therapeutic use
chemical_list: D045930:Anabolic Agents; D000970:Antineoplastic Agents; D004921:Erythropoietin; D014803:Vitamin B Complex; D013792:Thalidomide; D053614:Janus Kinase 2; D000077269:Lenalidomide; D011736:Pyridoxine