MYELOPROLIFERATIVE NEOPLASM SYMPTOM BURDEN AND QUALITY OF LIFE: RESULTS FROM THE PILOT MOSAICC STUDY

Introduction: The myeloproliferative neoplasms (MPNs) including polycythaemia vera (PV), essential thrombocythaemia (ET) and primary myelofibrosis (PMF) are rare diseases which contribute to significant morbidity. Symptom management is a prime objective in MPN treatment but current symptom assessment tools have not been validated compared to the general population. Materials and Methods: The MPN-Symptom Assessment Form (MPN-SAF) is a reliable and validated clinical tool used to assess MPN symptom burden. The MPN-SAF was administered to MPN patients (n=106) and, for the first time, General Practice and non-blood relative/family controls (n=124) as part of a UK pilot case-control study. Mean symptom scores were compared between cases and controls adjusted for potential confounding variables. Mean patient scores were then compared to data collected by the Mayo Clinic, USA on 1,446 MPN patients to determine the representativeness of the patient group. Results: MPN patients had significantly higher mean scores than controls for 25 of the 26 symptoms measured (p<0.05), Figure 1. Fatigue was the most common symptom in both groups (92.4% and 78.1%, respectively). Female MPN patients suffered worse symptom burden than male patients (p<0.001) and substantially worse burden than female controls (p<0.001). Compared to MPN-USA patients, MPN-UK patients reported similar symptom burden, Figure 1, but lower satiety when adjusted for potential confounders (p=0.046). Patients with PMF reported the worst symptom burden (88.3%); significantly higher than that reported by PV cases (p<0.001). Overall quality of life was impaired (i.e., score of at least 1 on a 0-10 scale) in 78.4% compared with 57.4% of controls (p<0.001). Conclusions: MPN patients experience significant morbidity when compared to the general population highlighting the need to manage symptoms effectively. The results further validate the use of the MPN-SAF as a discriminatory tool to assess MPN symptomatic disease burden.