title: Early lenalidomide treatment for low and intermediate-1 International Prognostic Scoring System risk myelodysplastic syndromes with del(5q) before transfusion dependence.
abstract: Lenalidomide is approved for the treatment of transfusion-dependent (TD) del(5q) myelodysplastic syndromes (MDS). However, few data are available in patients with transfusion-independent (TI) del(5q) MDS. In the first, observational, part of this 2-part study, we assessed the impact of transfusion dependence on overall survival (OS) and non-leukemic death in untreated del(5q) MDS patients who were TD (n = 136), TI with hemoglobin (Hb) ≥10 mg/dL (n = 88), or TI with Hb <10 mg/dL (n = 96). In the second, interventional, part we assessed the quality-of-life (QoL) benefits and clinical efficacy of lenalidomide (10 mg/day) in 12 patients with TI del(5q) MDS and Hb <10 mg/dL. In the untreated population, OS was significantly longer in TI than in TD patients (TI [Hb ≥10 g/dL], 108 months; TI [Hb <10 g/dL], 77 months; TD, 44 months). Transfusion dependence also negatively impacted non-leukemic death rates. In the interventional part of the study, baseline Hb levels were found to correlate significantly with physical (R = 0.666, P = 0.035) and fatigue (R = 0.604, P = 0.049) QoL scores. Median physical QoL scores improved significantly after 12 weeks' treatment with lenalidomide (+12.5; P = 0.020). Evaluable TI patients experienced early increases in Hb levels, and all attained an erythroid response. Our findings suggest that TI patients with moderate anemia may benefit from early treatment with lenalidomide.
authors.affiliation: Hematology Unit, Bianchi-Melacrino-Morelli Hospital, Reggio Calabria, Italy.
authors.forename: Esther N
authors.initials: EN
authors.lastname: Oliva
authors.affiliation: Institute for Medical Information Science, Biometry and Epidemiology, Ludwig-Maximilians-University, Munich, Germany.
authors.forename: Michael
authors.initials: M
authors.lastname: Lauseker
authors.affiliation: Department of Clinical and Molecular Medicine, La Sapienza University, Rome, Italy.
authors.forename: Maria Antonietta
authors.initials: MA
authors.lastname: Aloe Spiriti
authors.affiliation: Marche Polytechnic, Ancona, Italy.
authors.forename: Antonella
authors.initials: A
authors.lastname: Poloni
authors.affiliation: IRCCS Ca' Granda Foundation, Maggiore General Hospital, University of Milan, Milan, Italy.
authors.forename: Agostino
authors.initials: A
authors.lastname: Cortelezzi
authors.affiliation: Vittorio Emanuele General Hospital, University of Catania, Catania, Italy.
authors.forename: Giuseppe A
authors.initials: GA
authors.lastname: Palumbo
authors.affiliation: Hematology Unit, San Martino Hospital, Genoa, Italy.
authors.forename: Enrico
authors.initials: E
authors.lastname: Balleari
authors.affiliation: Hematology Division, Casa Sollievo della Sofferenza Hospital, San Giovanni Rotondo, Italy.
authors.forename: Grazia
authors.initials: G
authors.lastname: Sanpaolo
authors.affiliation: San G Moscati Hospital, Avellino, Italy.
authors.forename: Antonio
authors.initials: A
authors.lastname: Volpe
authors.affiliation: University of Bari, Bari, Italy.
authors.forename: Alessandra
authors.initials: A
authors.lastname: Ricco
authors.affiliation: Hematology Unit, Bianchi-Melacrino-Morelli Hospital, Reggio Calabria, Italy.
authors.forename: Francesca
authors.initials: F
authors.lastname: Ronco
authors.affiliation: Hematology Unit, Bianchi-Melacrino-Morelli Hospital, Reggio Calabria, Italy.
authors.forename: Caterina
authors.initials: C
authors.lastname: Alati
authors.affiliation: Medical Genetic Unit, Bianchi-Melacrino-Morelli Hospital, Reggio Calabria, Italy.
authors.forename: Maria Grazia
authors.initials: MG
authors.lastname: D'Errigo
authors.affiliation: Hematology Unit, Bianchi-Melacrino-Morelli Hospital, Reggio Calabria, Italy.
authors.forename: Irene
authors.initials: I
authors.lastname: Santacaterina
authors.affiliation: Department of Hematology, Oncology and Clinical Immunology, Heinrich-Heine-University of Düsseldorf, Düsseldorf, Germany.
authors.forename: Andrea
authors.initials: A
authors.lastname: Kündgen
authors.affiliation: Department of Hematology, Oncology and Clinical Immunology, Heinrich-Heine-University of Düsseldorf, Düsseldorf, Germany.
authors.forename: Ulrich
authors.initials: U
authors.lastname: Germing
authors.affiliation: Department of Cellular Biotechnology and Hematology, La Sapienza University, Rome, Italy.
authors.forename: Roberto
authors.initials: R
authors.lastname: Latagliata
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D015415:Biomarkers; D002872:Chromosome Deletion; D002895:Chromosomes, Human, Pair 5; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007155:Immunologic Factors / Q000008:administration & dosage / Q000627:therapeutic use*; D000077269:Lenalidomide; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000188:drug therapy* / Q000235:genetics* / Q000401:mortality; D011379:Prognosis; D011788:Quality of Life; D013792:Thalidomide / Q000008:administration & dosage / Q000031:analogs & derivatives* / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D015415:Biomarkers; D007155:Immunologic Factors; D013792:Thalidomide; D000077269:Lenalidomide
keywords: 5q deletion syndrome; anemia; lenalidomide; myelodysplastic syndromes; quality of life