Cytoreductive treatment in patients withCALR-mutated essential thrombocythaemia: a study comparing indications and efficacy among genotypes from the Spanish Registry of Essential Thrombocythaemia

被引：12

作者：

Alvarez-Larran, Alberto ^{[1
]}

Angona, Anna ^{[2
]}

Andrade-Campos, Marcio ^{[3
]}

Soledad Noya, M. ^{[4
]}

Teresa Gomez-Casares, M. ^{[5
]}

Cuevas, Beatriz ^{[6
]}

Caballero, Gonzalo ^{[7
]}

Garcia-Hernandez, Carmen ^{[8
]}

Garcia-Gutierrez, Valentin ^{[9
]}

Palomino, Alicia ^{[1
]}

Ferrer-Marin, Francisca ^{[10
]}

Isabel Mata-Vazquez, M. ^{[11
]}

Moreto, Ana ^{[12
]}

Magro, Elena ^{[13
]}

Murillo, Ilda ^{[14
]}

Alonso-Dominguez, Juan Manuel ^{[15
]}

Maria Guerra, Jose ^{[16
]}

Guerrero, Lucia ^{[17
]}

Maria Raya, Jose ^{[18
]}

Perez-Encinas, Manuel ^{[19
]}

Carreno-Tarragona, Gonzalo ^{[20
]}

Fox, Laura ^{[21
]}

Pastor-Galan, Irene ^{[22
]}

Bellosillo, Beatriz ^{[3
]}

Carlos Hernandez-Boluda, Juan ^{[22
]}

机构：

[1] IDIBAPS, Hosp Clin, Barcelona, Spain

[2] Hosp Josep Trueta, ICO Girona, Girona, Spain

[3] Hosp Mar, Inst Hosp Mar Invest Med, Barcelona, Spain

[4] Hosp Univ, La Coruna, Spain

[5] Hosp Univ Gran Canaria Doctor Negrin, Las Palmas Gran Canaria, Spain

[6] Hosp Univ Burgos, Burgos, Spain

[7] Hosp Univ Miguel Servet, Zaragoza, Spain

[8] Hosp Gen Univ, Alicante, Spain

[9] IRYCIS, Hosp Ramon & Cajal, Madrid, Spain

[10] UCAM, Hosp Morales Messeguer, CIBERER U765, IMIB Arrixaca, Murcia, Spain

[11] Hosp Costa Sol, Marbella, Spain

[12] Hosp Univ Cruces, Bilbao, Spain

[13] Hosp Principe Asturias, Alcala De Henares, Spain

[14] Hosp Gen San Jorge, Huesca, Spain

[15] IIS FJD, Hosp Univ Fdn Jimenez Diaz, Madrid, Spain

[16] Hosp Son Llatzer, Palma De Mallorca, Spain

[17] Complejo Asistencial Palencia, Palencia, Spain

[18] Hosp Univ Canarias, Santa Cruz De Tenerife, Spain

[19] Hosp Clin Univ, Santiago De Compostela, Spain

[20] Hosp Univ 12 Octubre, Madrid, Spain

[21] VHIO, Hosp Vall dHebron, Barcelona, Spain

[22] Hosp Clin Univ, INCLIVA, Valencia, Spain

来源：

BRITISH JOURNAL OF HAEMATOLOGY | 2021年 / 192卷 / 06期

关键词：

essential thrombocythaemia; therapy; haematological response; calreticulin mutation; genotype; myeloproliferative neoplasms; CALR; HYDROXYUREA; ANAGRELIDE; MUTATIONS; JAK2; RESISTANCE/INTOLERANCE; CALRETICULIN; DEFINITION; PHENOTYPE; CRITERIA;

D O I：

10.1111/bjh.16988

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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 wereCALR-mutated. In low risk patients, time from diagnosis to cytoreduction was shorter inCALR-positive than in the other genotypes (2 center dot 8, 3 center dot 2, 7 center dot 4 and 12 center dot 5 years forCALR,MPL,JAK2V617F and TN, respectively,P < 0 center dot 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% inCALRandJAK2V617F genotypes, respectively. Median time to CR was 192 days forJAK2V617F, 343 for TN, 433 forMPL, and 705 forCALRgenotypes (P < 0 center dot 0001). Duration of CR was shorter inCALR-mutated ET than in the remaining patients (P = 0 center dot 003). InCALR-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% forJAK2V617F,CALR,MPLand TN, respectively;P < 0 center dot 0001). In conclusion, conventional cytoreductive agents are less effective inCALR-mutated ET, highlighting the need for new treatment modalities and redefinition of haematologic targets for patients with this genotype.

引用

页码：988 / 996

页数：9

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