Prognostic value of response to first-line hydroxyurea according to IPSET stratification in essential thrombocythemia

Hydroxyurea (HU) constitutes the first-line treatment in most patients with essential thrombocythemia (ET), but criteria for changing therapy are not clearly established. The prognostic value of complete hematological response (CHR) and resistance/intolerance to HU was assessed in 1080 patients from...

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Detalles Bibliográficos
Autores: Santaliestra, Marta, Garrote i Ordeig, Marta, Noya, María Soledad, Pérez Encinas, Manuel, Senín, Alicia, Pérez López, Raúl, Ferrer Marín, Francisca, Carreño Tarragona, Gonzalo, Caballero, Gonzalo, Magro, Elena, Vélez, Patricia, Cortés Vázquez, Miguel Ángel, Moretó, Ana, Angona, Anna, Pastor Galán, Irene, Guerra, José María, García Hernández, Carmen, Mata, María Isabel, Stuckey, Ruth, Gómez Casares, María Teresa, Fox, Laura, Cuevas, Beatriz, García Gutiérrez, Valentín, Triguero, Ana, Arellano Rodrigo, Eduardo, Hernández Boluda, Juan Carlos, Álvarez Larrán, Alberto
Tipo de recurso: artículo
Estado:Versión aceptada para publicación
Fecha de publicación:2024
País:España
Institución:Universidad de Barcelona
Repositorio:Dipòsit Digital de la UB
OAI Identifier:oai:diposit.ub.edu:2445/215666
Acceso en línea:https://hdl.handle.net/2445/215666
Access Level:acceso abierto
Palabra clave:Quimioteràpia
Medul·la òssia
Trastorns de les plaquetes sanguínies
Medicaments
Chemotherapy
Bone marrow
Blood platelet disorders
Drugs
Descripción
Sumario:Hydroxyurea (HU) constitutes the first-line treatment in most patients with essential thrombocythemia (ET), but criteria for changing therapy are not clearly established. The prognostic value of complete hematological response (CHR) and resistance/intolerance to HU was assessed in 1080 patients from the Spanish Registry of ET, classified according to revised IPSET-Thrombosis stratification (Very low- n = 61, Low- n = 83, Intermediate- n = 261, and High-risk n = 675). With a median therapy duration of 5 years, CHR was registered in 720 (67%) patients (1-year probability 51%) and resistance/intolerance in 219 (20%) patients (5-years probability 13%). After correction by other risk factors, High-risk patients achieving CHR showed a reduced risk of arterial thrombosis (HR: 0.35, 95%CI: 0.2-0.6, p = 0.001) and a trend towards lower risk of venous thrombosis (HR: 0.45, 95%CI: 0.2-1.02, p = 0.06) whereas no association was observed for intermediate- or low-risk patients. In comparison with non-responders, intermediate- and high-risk patients achieving CHR had longer survival and lower myelofibrosis incidence. Development of resistance/intolerance to HU, mainly cytopenia, was associated with higher probability of myelofibrosis but no effect on survival or thrombotic risk was demonstrated. In conclusion, CHR with HU is associated with better outcomes and might be an early indicator for selecting candidates to second-line clinical trials.

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