BACKGROUND: Refractory anemia with ring sideroblasts associated with marked thrombocytosis was proposed as a provisional entity in the 2001 World Health Organization classification of myeloid neoplasms and also in the 2008 version, but its existence as a single entity is contested. We wish to define the clinical features of this rare myelodysplastic/myeloproliferative neoplasm and to compare its clinical outcome with that of refractory anemia with ring sideroblasts and essential thrombocythemia. DESIGN AND METHODS: We conducted a collaborative retrospective study across Europe. Our database included 200 patients diagnosed with refractory anemia with ring sideroblasts and marked thrombocytosis. For each of these patients, each patient diagnosed with refractory anemia with ring sideroblasts was matched for age and sex. At the same time, a cohort of 454 patients with essential thrombocythemia was used to compare outcomes of the two diseases. RESULTS: In patients with refractory anemia with ring sideroblasts and marked thrombocytosis, depending on the Janus Kinase 2 V617F mutational status (positive or negative) or platelet threshold (over or below 600 × 10(9)/L), no difference in survival was noted. However, these patients had shorter overall survival and leukemia-free survival with a lower risk of thrombotic complications than did patients with essential thrombocythemia (P<0.001) but better survival (P<0.001) and a higher risk of thrombosis (P=0.039) than patients with refractory anemia with ring sideroblasts. CONCLUSIONS: The clinical course of refractory anemia with ring sideroblasts and marked thrombocytosis is better than that of refractory anemia with ring sideroblasts and worse than that of essential thrombocythemia. The higher risk of thrombotic events in this disorder suggests that anti-platelet therapy might be considered in this subset of patients. From a clinical point of view, it appears to be important to consider refractory anemia with ring sideroblasts and marked thrombocytosis as a distinct entity.
BACKGROUND: Refractory anemia with ring sideroblasts associated with marked thrombocytosis was proposed as a provisional entity in the 2001 World Health Organization classification of myeloid neoplasms and also in the 2008 version, but its existence as a single entity is contested. We wish to define the clinical features of this rare myelodysplastic/myeloproliferative neoplasm and to compare its clinical outcome with that of refractory anemia with ring sideroblasts and essential thrombocythemia. DESIGN AND METHODS: We conducted a collaborative retrospective study across Europe. Our database included 200 patients diagnosed with refractory anemia with ring sideroblasts and marked thrombocytosis. For each of these patients, each patient diagnosed with refractory anemia with ring sideroblasts was matched for age and sex. At the same time, a cohort of 454 patients with essential thrombocythemia was used to compare outcomes of the two diseases. RESULTS: In patients with refractory anemia with ring sideroblasts and marked thrombocytosis, depending on the Janus Kinase 2 V617F mutational status (positive or negative) or platelet threshold (over or below 600 × 10(9)/L), no difference in survival was noted. However, these patients had shorter overall survival and leukemia-free survival with a lower risk of thrombotic complications than did patients with essential thrombocythemia (P<0.001) but better survival (P<0.001) and a higher risk of thrombosis (P=0.039) than patients with refractory anemia with ring sideroblasts. CONCLUSIONS: The clinical course of refractory anemia with ring sideroblasts and marked thrombocytosis is better than that of refractory anemia with ring sideroblasts and worse than that of essential thrombocythemia. The higher risk of thrombotic events in this disorder suggests that anti-platelet therapy might be considered in this subset of patients. From a clinical point of view, it appears to be important to consider refractory anemia with ring sideroblasts and marked thrombocytosis as a distinct entity.
Authors: Peter J Campbell; Linda M Scott; Georgina Buck; Keith Wheatley; Clare L East; Joanne T Marsden; Audrey Duffy; Elaine M Boyd; Anthony J Bench; Mike A Scott; George S Vassiliou; Donald W Milligan; Steve R Smith; Wendy N Erber; David Bareford; Bridget S Wilkins; John T Reilly; Claire N Harrison; Anthony R Green Journal: Lancet Date: 2005-12-03 Impact factor: 79.321
Authors: Gerwin Huls; André B Mulder; Stefano Rosati; Arjan A van de Loosdrecht; Edo Vellenga; Joost T M de Wolf Journal: Blood Date: 2010-03-01 Impact factor: 22.113
Authors: Luca Malcovati; Matteo G Della Porta; Daniela Pietra; Emanuela Boveri; Andrea Pellagatti; Anna Gallì; Erica Travaglino; Angela Brisci; Elisa Rumi; Francesco Passamonti; Rosangela Invernizzi; Laura Cremonesi; Jacqueline Boultwood; James S Wainscoat; Eva Hellström-Lindberg; Mario Cazzola Journal: Blood Date: 2009-08-19 Impact factor: 22.113
Authors: J Broséus; E Lippert; A S Harutyunyan; S Jeromin; E Zipperer; L Florensa; J D Milosevic; T Haferlach; U Germing; E Luño; S Schnittger; R Kralovics; F Girodon Journal: Leukemia Date: 2014-01-30 Impact factor: 11.528
Authors: M M Patnaik; T L Lasho; C M Finke; C A Hanson; R L King; R P Ketterling; N Gangat; A Tefferi Journal: Leukemia Date: 2016-08-01 Impact factor: 11.528
Authors: Laura Palomo; Manja Meggendorfer; Stephan Hutter; Sven Twardziok; Vera Ademà; Irene Fuhrmann; Francisco Fuster-Tormo; Blanca Xicoy; Lurdes Zamora; Pamela Acha; Cassandra M Kerr; Wolfgang Kern; Jaroslaw P Maciejewski; Francesc Solé; Claudia Haferlach; Torsten Haferlach Journal: Blood Date: 2020-10-15 Impact factor: 22.113