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

• Published in: Therapeutic advances in hematology 2022-03, Vol.13, p.20406207221084487-20406207221084487
• Main Authors: Mead, Adam J., Butt, Nauman M., Nagi, Waseem, Whiteway, Alastair, Kirkpatrick, Suriya, Rinaldi, Ciro, Roughley, Catherine, Ackroyd, Sam, Ewing, Joanne, Neelakantan, Pratap, Garg, Mamta, Tucker, David, Murphy, John, Patel, Hitesh, Bains, Rozinder, Chiu, Gavin, Hickey, Joe, Harrison, Claire, Somervaille, Tim C. P.
• Format: Article
• Language: English
• Subjects: Blood cancer; Inhibitor drugs; Medical records; Original Research; Patients; Realism; Targeted cancer therapy
• ISSN: 2040-6207; 2040-6215
• DOI: 10.1177/20406207221084487

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. Plain Language Summary Background: Myelofibrosis is a rare blood cancer associated with symptoms that can seriously affect a patient’s daily life, such as enlarged spleen and decreased white and red blood cells. Although several treatments are available for patients with myelofibrosis, it is not clear which ones clinicians use most frequently. Methods: We aimed to review which treatments are usually given to patients with myelofibrosis in the UK, by collecting information from the medical records of 200 patients with myelofibrosis treated in different centres across the UK. Results: The results showed that the symptoms patients experienced were not always written down in the medical records. Similarly, clinical scores based on patient characteristics (which clinicians use to try to predict if a patient will respond to treatment well or not) were also missing from the medical records. Clinicians also rarely asked patients to complete questionnair