Choice of Therapy for Patients With Waldenström Macroglobulinemia
Mené sur 414 patients atteints d'une macroglobulinémie de Waldenström ou d'un lymphome présentant des caractéristiques proches, cet essai international (101 centres hospitaliers, 5 pays) compare, du point de vue du taux de réponse globale, la fludarabine et le chlorambucil
Waldenstro¨mmacroglobulinemia(WM)is a distinct B-cell lymphoproliferative disorder characterized by the presence of lymphoplasmacytic cells in the bone marrow (BM), along with demonstration of an immunoglobulin (Ig)Mmonoclonal protein in the serum. In 1944, Jan Gosta Waldenstrom first reported this syndrome by describing two patients with lymphadenopathy, oronasal bleeding, anemia, thrombocytopenia, and infiltration of theBMby lymphoid cells. Since then, many advances have occurred in the biology and therapy of this unique entity. Most recently, whole genome sequencing of lymphoplasmacytic cells was performed from 30 patients with WM, showing a recurring sequence variant in chromosome 3p22.2 that results in a single nucleotide change in the myeloid differentiation primary response (MYD88) gene with a predicted nonsynonymous change at amino acid position 265 from leucine to proline (L265P). This MYD88 L265P mutation occurred in 90% of the WM samples and was not observed in normal germline cells or in most cases of multiple myeloma...
Journal of Clinical Oncology , éditorial en libre accès, 2012