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Long‐term follow‐up of essential thrombocythemia patients treated with anagrelide: subgroup analysis according to JAK 2 / CALR / MPL mutational status
Author(s) -
Mela Osorio María J.,
Ferrari Luciana,
Goette Nora P.,
Gutierrez Marina I.,
Glembotsky Ana C.,
Maldonado Ana C.,
Lev Paola R.,
Alvarez Clarisa,
Korin Laura,
Marta Rosana F.,
Molinas Felisa C.,
Heller Paula G.
Publication year - 2016
Publication title -
european journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.904
H-Index - 84
eISSN - 1600-0609
pISSN - 0902-4441
DOI - 10.1111/ejh.12614
Subject(s) - anagrelide , essential thrombocythemia , medicine , adverse effect , myelofibrosis , gastroenterology , anemia , oncology , bone marrow , polycythemia vera
Background Anagrelide represents a treatment option for essential thrombocythemia, although its place in therapy remains controversial. Aim To assess the impact of mutational status in response rates and development of adverse events during long‐term use of anagrelide. Methods We retrospectively evaluated 67 patients with essential thrombocythemia treated with anagrelide during 68 (4–176) months. Results Mutational frequencies were 46.3%, 28.3%, and 1.5% for JAK 2 V617F, CALR and MPL mutations. Anagrelide yielded a high rate of hematologic responses, which were complete in 49.25% and partial in 46.25%, without differences among molecular subsets. The rate of thrombosis during treatment was one per 100 patient‐years, without excess bleeding. Anemia was the major adverse event, 30.3% at 5‐yr follow‐up, being more frequent in CALR + ( P < 0.05). Myelofibrotic transformation developed in 14.9% (12.9%, 21%, and 12.5% in JAK 2 V617F + , CALR + , and triple‐negative patients, respectively, P = NS ) and those treated >60 months were at higher risk, OR (95% CI ) 9.32 (1.1–78.5), P < 0.01, indicating the need for bone marrow monitoring during prolonged treatment. Conclusion Although CALR + patients were at higher risk of developing anemia, anagrelide proved effective among all molecular subsets, indicating that mutational status does not seem to represent a major determinant of choice of cytoreductive treatment among essential thrombocythemia therapies.