期刊
BLOOD REVIEWS
卷 55, 期 -, 页码 -出版社
CHURCHILL LIVINGSTONE
DOI: 10.1016/j.blre.2022.100947
关键词
MPN; PV; ET; AML; JAK2; Leukaemia
类别
Accelerated and blast phase myeloproliferative neoplasms present challenges in terms of treatment, with allogeneic stem cell transplantation being the only curative approach. Predicting the transformation events from chronic phase disease is difficult due to the lack of consistent risk factors. Enhanced prognostic approaches are needed to improve monitoring and early intervention for better overall treatment outcomes.
Accelerated and blast phase myeloproliferative neoplasms present many challenges despite the advent of more novel and targeted therapeutic approaches. Outcome following transformation is frequently dismal, and the only curative approach remains allogeneic stem cell transplantation, applicable to a relatively small proportion of cases. Historically, potential risk factors for transformation from chronic phase disease have been based loosely on increasing age, long disease duration, use of sequential DNA-damaging agents, cytogenetic anomalies and advancing disease burden and limited genomic profiling which frequently reveals disparate signatures. Overall, the risk of these transformation events remains unpredictable, can occur in young individuals even without detectable high risk genomic profiles. Enhanced prognostic approaches would optimise monitoring and early intervention with potential to improve overall outcomes. Within this review we will summarise advances on disease biology, prognostication and molecular annotation and how this may ultimately lead to more rationale, stratified and forward-thinking therapeutic approaches.
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