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Cytoreductive treatment in patients with CALR-mutated essential thrombocythaemia: a study comparing indications and efficacy among genotypes from the Spanish Registry of Essential Thrombocythaemia.
- Source :
-
British journal of haematology [Br J Haematol] 2021 Mar; Vol. 192 (6), pp. 988-996. Date of Electronic Publication: 2020 Aug 03. - Publication Year :
- 2021
-
Abstract
- The present study assessed the criteria for initiating cytoreduction and response to conventional therapies in 1446 patients with essential thrombocythemia (ET), 267 (17%) of which were CALR-mutated. In low risk patients, time from diagnosis to cytoreduction was shorter in CALR-positive than in the other genotypes (2·8, 3·2, 7·4 and 12·5 years for CALR, MPL, JAK2V617F and TN, respectively, P < 0·0001). A total of 1104 (76%) patients received cytoreductive treatment with hydroxycarbamide (HC) (n = 977), anagrelide (n = 113), or others (n = 14). The estimated cumulative rates of complete haematological response (CR) at 12 months were 40 % and 67% in CALR and JAK2V617F genotypes, respectively. Median time to CR was 192 days for JAK2V617F, 343 for TN, 433 for MPL, and 705 for CALR genotypes (P < 0·0001). Duration of CR was shorter in CALR-mutated ET than in the remaining patients (P = 0·003). In CALR-positive patients, HC and anagrelide had similar efficacy in terms of response rates and duration. CALR-mutated patients developed resistance/intolerance to HC more frequently (5%, 23%, 27% and 15% for JAK2V617F, CALR, MPL and TN, respectively; P < 0·0001). In conclusion, conventional cytoreductive agents are less effective in CALR-mutated ET, highlighting the need for new treatment modalities and redefinition of haematologic targets for patients with this genotype.<br /> (© 2020 British Society for Haematology and John Wiley & Sons Ltd.)
- Subjects :
- Adolescent
Adult
Aged
Aged, 80 and over
Amino Acid Substitution
Child
Female
Follow-Up Studies
Humans
Janus Kinase 2 genetics
Male
Middle Aged
Spain
Calreticulin genetics
Genotype
Hydroxyurea administration & dosage
Mutation, Missense
Quinazolines administration & dosage
Registries
Thrombocythemia, Essential drug therapy
Thrombocythemia, Essential genetics
Subjects
Details
- Language :
- English
- ISSN :
- 1365-2141
- Volume :
- 192
- Issue :
- 6
- Database :
- MEDLINE
- Journal :
- British journal of haematology
- Publication Type :
- Academic Journal
- Accession number :
- 32745264
- Full Text :
- https://doi.org/10.1111/bjh.16988