The mpn Hub website uses a third-party service provided by Google that dynamically translates web content. Translations are machine generated, so may not be an exact or complete translation, and the mpn Hub cannot guarantee the accuracy of translated content. The mpn and its employees will not be liable for any direct, indirect, or consequential damages (even if foreseeable) resulting from use of the Google Translate feature. For further support with Google Translate, visit Google Translate Help.
The MPN Hub is an independent medical education platform, sponsored by AOP Health and GSK, and supported through an educational grant from Bristol Myers Squibb. The funders are allowed no direct influence on our content. The levels of sponsorship listed are reflective of the amount of funding given. View funders.
Now you can support HCPs in making informed decisions for their patients
Your contribution helps us continuously deliver expertly curated content to HCPs worldwide. You will also have the opportunity to make a content suggestion for consideration and receive updates on the impact contributions are making to our content.
Find out moreCreate an account and access these new features:
Bookmark content to read later
Select your specific areas of interest
View mpn content recommended for you
Understanding of real-world management patterns for myelofibrosis (MF) is elusive due to the diversity of treatment options. Considering the burden of MF-related symptoms, such as splenomegaly and anemia, it is important to understand whether current treatment initiation is time efficient; for example, if patients who are undergoing the ‘watch-and-wait’ approach may benefit from treatment instead, and whether optimal care is being given for the correct patients according to recognized international prognostic scoring systems.
The REALISM UK multicenter, noninterventional retrospective study investigated the early management strategies for MF in the UK, and was recently published by Mead et al. in Therapeutic Advances in Hematology.1 We summarize the key findings below.
The study design, including primary and secondary endpoints, is depicted in Figure 1. A total of 15 National Health Service (NHS) hospitals participated in the data collection.
Figure 1. Study design*
MF, myelofibrosis.
*Adapted from Mead et al.1
†Patient characteristics included demographics, diagnosis method, distribution of MF types, mutational status, MF symptoms, spleen size, IPSS prognostic score, and percentage of patients with recorded IPSS scores at diagnosis.
Patient characteristics are shown in Table 1.
Table 1. Patient characteristics*
ET, essential thrombocytopenia; IPSS, International Prognostic Scoring System; JAK2, Janus kinase 2; MF, myelofibrosis; PV, polycythemia vera. |
|
Characteristic |
N = 200 |
---|---|
Median age, years (range) |
69.7 (20.3–91.8) |
Female/male, % |
40.5/59.5 |
MF diagnosis, % |
|
Primary |
63.0 |
Secondary (post-ET) |
16.5 |
Secondary (post-PV) |
20.5 |
Positive for JAK2V617F mutation, % |
81.0 |
IPSS risk category, % |
|
Low-0 |
7.5 |
Intermediate-1 |
29.0 |
Intermediate-2 |
29.5 |
High ≥3 |
19.5 |
Unavailable |
14.5 |
Table 2. Median time to active treatment from diagnosis by IPSS risk score*
IPSS, International Prognostic Scoring System. |
|
IPSS risk score |
Median time to treatment, days (range) |
---|---|
Overall (n = 171) |
46 (0–342) |
Low-0 (n = 15) |
153 (0–667) |
Intermediate-1 (n = 58) |
89.5 (0–473) |
Intermediate-2 (n = 59) |
0 (0–251) |
High ≥3 (n = 39) |
0 (0–216) |
Figure 2. First-line treatment by IPSS risk score*
IPSS, International Prognostic Scoring System.
*Adapted from Mead et al.1
Figure 3. First-line treatment strategies by year*
*Figure from Mead et al.1
The shortest median duration of treatment (interquartile range [IQR]) was observed for ruxolitinib (541.5 days [313.5–998.8]), then hydroxycarbamide (608.0 days [407.5–988.5]), and finally, ‘watch-and-wait’ (619.0 days [392.0–973.0]).
Figure 4. Causes of death*
MF, myelofibrosis.
*Adapted from Mead et al1
Retrospective, real-world data from the REALISM UK study demonstrate a lack of substantial recording of patient-reported symptoms and diagnostic criteria which are important for deciding treatment. Although in decline in recent years, almost half of patients were reported to have ‘watch-and-wait’ treatment, which was sometimes given for patients classified as high risk. These data demonstrate a need for earlier treatment intervention in the UK. Hydroxycarbamide remains a common approach, though less effective than ruxolitinib, indicating that patients with a high symptom burden were treated with options that may not improve disease symptoms. The discontinuation rates with hydroxycarbamide and ruxolitinib were high, highlighting the need for more understanding of reasons behind this, and a guidance on when to discontinue treatment.
In this Novartis sponsored real world observational note review we focussed upon the pathway from diagnosis to treatment for myelofibrosis in the UK. We found that both symptoms and prognostic scores at diagnosis were poorly documented. Time to first treatment was shorter for patients with higher risk disease and only 5% of patients underwent stem cell transplant. Other important insights included that 35% of patients required blood transfusion.
References
Please indicate your level of agreement with the following statements:
The content was clear and easy to understand
The content addressed the learning objectives
The content was relevant to my practice
I will change my clinical practice as a result of this content