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A retrospective real-world study of the current treatment pathways for myelofibrosis in the United Kingdom: the REALISM UK study
Mead, A. J. Butt, N. M. Nagi, W. Whiteway, A. Kirkpatrick, S. Rinaldi, C. Roughley, C. Ackroyd, S. Ewing, J. Neelakantan, P.
Mead, A. J.
Butt, N. M.
Nagi, W.
Whiteway, A.
Kirkpatrick, S.
Rinaldi, C.
Roughley, C.
Ackroyd, S.
Ewing, J.
Neelakantan, P.
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Abstract
Background: Myelofibrosis (MF) is a blood cancer associated with splenomegaly, blood count abnormalities, reduced life expectancy and high prevalence of disease-associated symptoms. Current treatment options for MF are diverse, with limited data on management strategies in real-world practice in the United Kingdom. Methods: The REALISM UK study was a multi-center, retrospective, non-interventional study, which documented the early management of patients with MF. The primary endpoint was the time from diagnosis to active treatment. Discussion: Two hundred patients were included (63% [n = 126/200] with primary MF; 37% [n = 74/200] with secondary MF). Symptoms and prognostic scores at diagnosis were poorly documented, with infrequent use of patient reported outcome measures. ‘Watch and wait’ was the first management strategy for 53.5% (n = 107/200) of patients, while the most commonly used active treatments were hydroxycarbamide and ruxolitinib. Only 5% of patients proceeded to allogeneic transplant. The median (IQR) time to first active treatment was 46 days (0–350); patients with higher risk disease were prescribed active treatment sooner. Conclusion: These results provide insight into real-world clinical practice for patients with MF in the United Kingdom. Despite the known high prevalence of disease-associated symptoms in MF, symptoms were poorly documented. Most patients were initially observed or received hydroxycarbamide, and ruxolitinib was used as first-line management strategy in only a minority of patients.
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2022
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Mead AJ, Butt NM, Nagi W, Whiteway A, Kirkpatrick S, Rinaldi C, et al. A retrospective real-world study of the current treatment pathways for myelofibrosis in the United Kingdom: the REALISM UK study. Vol. 13, Therapeutic Advances in Hematology. SAGE Publications; 2022.