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    A retrospective real-world study of the current treatment pathways for myelofibrosis in the United Kingdom: the REALISM UK study

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    Authors
    Mead, A. J.
    Butt, N. M.
    Nagi, W.
    Whiteway, A.
    Kirkpatrick, S.
    Rinaldi, C.
    Roughley, C.
    Ackroyd, S.
    Ewing, J.
    Neelakantan, P.
    Garg, M.
    Tucker, D.
    Murphy, J.
    Patel, H.
    Bains, R.
    Chiu, G.
    Hickey, J.
    Harrison, C.
    Somervaille, Tim C P
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    Issue Date
    2022
    
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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.
    Citation
    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.
    Journal
    Ther Adv Hematol
    URI
    http://hdl.handle.net/10541/625156
    DOI
    10.1177/20406207221084487
    PubMed ID
    35371428
    Additional Links
    https://dx.doi.org/10.1177/20406207221084487
    Type
    Article
    Language
    en
    ae974a485f413a2113503eed53cd6c53
    10.1177/20406207221084487
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