Current and future therapies for myelofibrosis

• Published in: Blood reviews Vol. 42; p. 100715
• Main Authors: Asher, Samir, McLornan, Donal P., Harrison, Claire N.
• Format: Journal Article
• Language: English
• Published: England Elsevier Ltd 01.07.2020
• Subjects: Clinical Trials as Topic; Disease Management; Disease Progression; Humans; Myelofibrosis; Myeloproliferative neoplasms; Primary Myelofibrosis - diagnosis; Primary Myelofibrosis - pathology; Primary Myelofibrosis - therapy; Prognosis; Ruxolitinib; Transplant; Treatment; Ruxolitinib; Myelofibrosis; Treatment; Myeloproliferative neoplasms; Transplant
• ISSN: 0268-960X; 1532-1681
• DOI: 10.1016/j.blre.2020.100715

Myelofibrosis is classified as a ‘Philadelphia-chromosome negative’ clonal myeloproliferative disorder. The heterogeneity of this condition and patient population and array of often challenging clinical manifestations can frequently make therapeutic decisions challenging. Despite many advances in therapy with targeted and combination approaches, following an enhanced understanding of underlying disease pathogenesis, cure only remains achievable with allogeneic stem cell transplant. This option is often limited to a small group of younger transplant-eligible patients with more advanced disease who have both a suitable donor and no or few co-morbidities. In this article, we will discuss up-to-date disease prognostication, common clinical challenges associated with myelofibrosis and both standard and novel therapeutic approaches. Increasingly complex prognostic modelling utilises patient-specific, haematological and genomic parameters to improve the accuracy of risk assessment and predict disease progression. We will also focus on difficult clinical scenarios such as disease-associated anaemia, thrombocytopenia and extremes of age. Future and evolving therapies within this field are highly anticipated and novel JAK inhibitor and non-JAK inhibitor-based therapy will also be discussed, including the new challenge of how to switch from one JAK inhibitor therapy to another.