Efficacy of Lenalidomide in Myelodysplastic Syndromes

• Published in: The New England journal of medicine Vol. 352; no. 6; pp. 549 - 557
• Main Authors: List, Alan, Kurtin, Sandy, Roe, Denise J, Buresh, Andrew, Mahadevan, Daruka, Fuchs, Deborah, Rimsza, Lisa, Heaton, Ruth, Knight, Robert, Zeldis, Jerome B
• Format: Journal Article
• Language: English
• Published: Boston, MA Massachusetts Medical Society 10-02-2005
• Subjects: Adult; Aged; Aged, 80 and over; Anemia, Refractory - drug therapy; Anemia, Refractory - etiology; Angiogenesis Inhibitors - adverse effects; Angiogenesis Inhibitors - pharmacology; Angiogenesis Inhibitors - therapeutic use; Biological and medical sciences; Bone Marrow - pathology; Cytokines; Female; Gene Deletion; General aspects; Hematologic and hematopoietic diseases; Hematopoiesis - drug effects; Humans; Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis; Male; Medical research; Medical sciences; Medical treatment; Middle Aged; Myelodysplastic syndromes; Myelodysplastic Syndromes - complications; Myelodysplastic Syndromes - drug therapy; Myelodysplastic Syndromes - genetics; Myelodysplastic Syndromes - pathology; Neutropenia - chemically induced; Thalidomide - adverse effects; Thalidomide - analogs & derivatives; Thalidomide - pharmacology; Thalidomide - therapeutic use; Thrombocytopenia - chemically induced; Medicine; Malignant hemopathy; Lenalidomide; Treatment efficiency; Myelodysplastic syndrome
• ISSN: 0028-4793; 1533-4406
• DOI: 10.1056/NEJMoa041668

In a study of 43 patients with low-risk myelodysplastic syndromes, lenalidomide, a thalidomide derivative, ameliorated anemia and allowed the discontinuation of transfusions in over half the patients.
In patients with low-risk myelodysplastic syndromes, a thalidomide derivative ameliorated anemia and allowed the discontinuation of transfusions in over half the patients.
Refractory anemia resulting from ineffective hematopoiesis is the principal therapeutic challenge for patients with myelodysplastic syndromes.
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Recombinant erythropoietin alone or in combination with myeloid growth factors ameliorates anemia in some patients but is generally ineffective in patients who require two or more red-cell transfusions per month; its use rarely induces cytogenetic remissions.
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Hematopoietic precursors in patients with myelodysplastic syndromes have an accelerated cell-cycle transition and impaired responsiveness to cytokine stimulation.
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Survival signals from the microenvironment are compromised, owing in part to the presence of angiogenic molecules, disruption of the medullary architecture, and excess production of inflammatory cytokines.
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