Study Examines Time to Treatment in UK Cases of Myelofibrosis

April 6, 2019

"Watch and wait” is typically used first for asymptomatic patients with myelofibrosis, according to a retrospective review of management strategies from the United Kingdom.

A retrospective review presented recently showed the diverse range of management strategies used for patients with myelofibrosis (MF), where “watch and wait” is typically used first for asymptomatic patients.

An interim analysis from a UK study was presented at the British Society for Haematology Annual Scientific Meeting. Real-world data on the management of patients with MF are limited, and the REALISM study aimed to document the early management pathways in routine clinical practice. The primary endpoint was the time from diagnosis to active treatment.

REALISM UK is a multi-center, non-interventional study ongoing in 15 UK secondary care centers. Eligible patients were older than 18 years at diagnosis, with diagnosis greater than 6 months and 5 or more years prior to data collection and with 1 or more follow-up visits. Data on patient demographics, clinical characteristics, MF management strategies and outcomes were collected from patients’ medical records; the observation period was from the date of MF diagnosis until data collection.

The study included 200 patients, with 64% having primary MF and 36% having secondary MF. The median age at MF diagnosis was 69.7 (interquartile range [IQR] 63.5—75.7) years; 63% (125/200) had a Janus kinase 2 (JAK2) gene mutation. The International Prognostic Scoring System category for 60% of the patients was an intermediate-2 or high at diagnosis.

Symptoms of disease at diagnosis included an enlarged spleen (47%), anemia (44%), and constitutional symptoms such as night sweats, unexplained fever or weight loss (30%).

The median time from diagnosis to active treatment was 0.0 (IQR 0.0—369.3) days (n = 200). During the study observation period “watch and wait” was the first management strategy for 47% (94/200) of patients (primary MF, 56% [71/127]; secondary MF, 32% [23/73]), with 69% (65/94) progressing to active treatment.

In the 94 patients who progressed to active treatment (including 29 with no active treatment at data collection), the median (IQR) time from diagnosis to active treatment (or data collection) was 409 days. Those scored low intermediate progressed at 280.5 days; those scored intermediate-1 were 284.5 days; those scored intermediate-2 were 461 days; and those scored high were 119 days.

Active treatment was started at diagnosis for 53% of patients. A total of 409 management strategies (defined as any clinical decision on patient management) were observed. Watch and wait was the most used strategy (26% [107/409]), followed by ruxolitinib (25% [102/409]), hydroxycarbamide (17% [68/409]) and allogenic stem cell transplantation (3% [11/409]).

The median treatment duration during the observation period was 398 (IQR 180.3—699.3) days for ruxolitinib and 336.5 (IQR 108.5–767.5) days for hydroxycarbamide.

Nearly one-fourth of patients died between diagnosis and data collection. The recorded causes of death were: MF-related (38%), other malignancies (9%), infection (9%), and heart failure (6%). The cause was unknown in 38% of cases.

Reference

Mead A, Butt N, Nagi W, et al. A retrospective real world evidence study to review the current treatment pathways for myelofibrosis from across the United Kingdom (The REALISM UK Study). Presented at the British Society for Haematology Annual Scientific Meeting; April 1-3, 2019; Glasgow, Scotland. Abstract BSH19-OR-008.