title: Lenalidomide in patients with red blood cell transfusion-dependent myelodysplastic syndrome and del(5q): a single-centre "real-world" experience.
abstract: "Real life" data are needed to complement published trials on the efficacy of lenalidomide in patients with myelodysplastic syndrome (MDS) and del(5q) and on the risk of inducing acute myeloid leukemia (AML) progression. Here, we present results of lenalidomide treatment in a consecutive, population-based series of 21 red blood cell (RBC) transfusion-dependent elderly patients with multiple comorbidities. Of 18 evaluable patients (median follow-up: 22 months), 17 achieved an erythroid hematologic response (HI-E) and 16 an RBC transfusion independence. Cytogenetic response (CyR) rate was 80%, median overall survival was 48 months (range 3-164), and 5-year leukemia-free survival was 84%. Three patients progressed to AML; one, with baseline TP53 mutation, achieved HI-E, partial CyR, and did not progress to AML. Eighteen patients experienced hematological adverse events. Overall, lenalidomide was very effective and well tolerated even in unselected elderly patients with multiple comorbidities and did not appear to increase the risk of AML.
authors: a Department of Hematology , A.O. Spedali Civili , Brescia , Italy.
authors: Elisa
authors: E
authors: Cerqui
authors: a Department of Hematology , A.O. Spedali Civili , Brescia , Italy.
authors: Annamaria
authors: A
authors: Pelizzari
authors: a Department of Hematology , A.O. Spedali Civili , Brescia , Italy.
authors: Francesca
authors: F
authors: Schieppati
authors: a Department of Hematology , A.O. Spedali Civili , Brescia , Italy.
authors: Erika
authors: E
authors: Borlenghi
authors: a Department of Hematology , A.O. Spedali Civili , Brescia , Italy.
authors: Chiara
authors: C
authors: Pagani
authors: b Cytogenetics and Genetics Laboratory, Department of Genetic and Molecular Medicine , University of Brescia , Brescia , Italy.
authors: Daniela
authors: D
authors: Bellotti
authors: a Department of Hematology , A.O. Spedali Civili , Brescia , Italy.
authors: Cinzia
authors: C
authors: Lamorgese
authors: c Pathology Section, Department of Molecular and Translational Medicine , University of Brescia , Brescia , Italy.
authors: Leonardo
authors: L
authors: Boiocchi
authors: d Centro Ricerca Emato-oncologica AIL (CREA), Diagnostics Department, A.O. Spedali Civili , Brescia , Italy.
authors: Alessandra
authors: A
authors: Sottini
authors: d Centro Ricerca Emato-oncologica AIL (CREA), Diagnostics Department, A.O. Spedali Civili , Brescia , Italy.
authors: Luisa
authors: L
authors: Imberti
authors: a Department of Hematology , A.O. Spedali Civili , Brescia , Italy.
authors: Giuseppe
authors: G
authors: Rossi
mesh_terms: D000368:Aged; D000369:Aged, 80 and over; D000970:Antineoplastic Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D001853:Bone Marrow / Q000473:pathology; D002872:Chromosome Deletion; D002895:Chromosomes, Human, Pair 5; D015897:Comorbidity; D018450:Disease Progression; D017707:Erythrocyte Transfusion; D005260:Female; D005500:Follow-Up Studies; D015870:Gene Expression; D006801:Humans; D000077269:Lenalidomide; D015470:Leukemia, Myeloid, Acute / Q000175:diagnosis / Q000209:etiology; D008297:Male; D008875:Middle Aged; D009154:Mutation; D009190:Myelodysplastic Syndromes / Q000235:genetics* / Q000401:mortality / Q000473:pathology / Q000628:therapy*; D011379:Prognosis; D013792:Thalidomide / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D016896:Treatment Outcome; D016159:Tumor Suppressor Protein p53 / Q000235:genetics / Q000378:metabolism
chemical_list: D000970:Antineoplastic Agents; D016159:Tumor Suppressor Protein p53; D013792:Thalidomide; D000077269:Lenalidomide
keywords: Cytogenetic response; del(5q) MDS; elderly; erythroid response; lenalidomide; transfusion independence