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Blood, 23 April 2009, Vol. 113, No. 17, pp. 3947-3952.
Prepublished online as a Blood First Edition Paper on November 5, 2008; DOI 10.1182/blood-2008-08-175778.


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CLINICAL TRIALS AND OBSERVATIONS

Efficacy and safety of lenalidomide in intermediate-2 or high-risk myelodysplastic syndromes with 5q deletion: results of a phase 2 study

Lionel Adès1,2,11, Simone Boehrer1,2,11, Thomas Prebet3,11, Odile Beyne-Rauzy4,11, Laurence Legros5,11, Christophe Ravoet6,11, François Dreyfus7,11, Aspasia Stamatoullas8,11, Marie Pierre Chaury9,11, Jacques Delaunay10,11, Guy Laurent4,11, Norbert Vey3,11, Sara Burcheri1,11, Rose-Marie Mbida1,11, Natacha Hoarau1,11, Claude Gardin1,11, and Pierre Fenaux1,2,11

1 Hôpital Avicenne, Université Paris, Paris, France; 2 Inserm U848, Institut Gustave Roussy, Villejuif, France; 3 Institut Paoli Calmettes, Marseille, France; 4 Centre Hospitalo-Universitaire de Toulouse, Toulouse, France; 5 Centre Hospitalo-Universitaire Nice, Nice, France; 6 Hôpital de Jolimont, la Louviere, Belgium; 7 Hôpital Cochin, Université Paris, Paris, France; 8 Centre Hospitalo-Universitaire de Rouen, Rouen, France; 9 Centre Hospitalo-Universitaire de Limoges, Limoges, France; 10 Centre Hospitalo-Universitaire de Nantes, Nantes, France; and 11 Groupe Francophone des Myelodysplasies

Higher-risk MDS with del5q carry a poor prognosis. In this phase 2 trial, 47 patients with higher-risk MDS received lenalidomide 10 mg/day. International Prognostic Scoring System was high in 60%, intermediate-2 risk in 40%. del 5q was isolated, with one additional and more than one additional abnormality in 19%, 23%, and 58% patients, respectively. Thirteen (27%) patients achieved hematologic response, including 7 hematologic complete remission (CR) (with complete [4] or partial [3] cytogenetic response), 2 marrow CR and 4 hematologic improvement erythroid, and 12 became red blood cell (RBC) transfusion independent, for a median duration of 6.5 months. Median CR duration was 11.5 months. Six of 9 (67%) patients with isolated del 5q achieved CR, versus 1 of 11 and none of 27 patients with one or more than one additional abnormality, respectively (P < .001). Seven of 20 (35%) with initial platelets more than 100 000/mm3 obtained CR, compared with none of the 27 with lower platelet counts less than 100 000/mm3 (P = .001). Our data support a potential role of lenalidomide in higher-risk MDS with isolated del 5q. This trial was registered at www.clinicaltrials.gov as NCT00424229 [ClinicalTrials.gov] .


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REVelation (del: 5q)
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Blood 2009 113: 3888-3889. [Full Text] [PDF]



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