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MPN symptom burden: A real-world cohort study from Quebec

By Sheetal Bhurke

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May 28, 2025

Learning objective: After reading this article, learners will be able to cite a new clinical development in myeloproliferative neoplasms.


 

Patients with myeloproliferative neoplasms (MPN) experience a significant symptom burden, impacting their QoL considerably. Addressing MPN symptom burden is important for individualizing treatments, and data on MPN symptom burden across subtypes and patient subpopulations are currently limited. 

Poullet et al. published a multicenter, retrospective, observational cohort study in the Blood Cancer Journal characterizing the symptom burden in patients with MPN.1 The cohort included 784 patients from the Quebec MPN research group. Eligible patients were aged >18 years with a diagnosis of PV (n = 285), ET (n = 422), or MF (n = 77) based on the WHO or IC criteria and had completed at least one MPN-SAF TSS between April 2013 and August 2022.   

 

Key learnings1

MF vs PV and ET cohorts showed higher mean and maximum MPN-SAF TSS scores (p = 0.0006 and p = 0.002, respectively). 

Subitem scores for fatigue, early satiety, inactivity, pruritus, bone pain, fever, and weight loss were higher in the MF cohort (p < 0.0001 to p = 0.04). Abdominal pain (p = 0.05) and having greater difficulty concentrating (p = 0.004) were also higher in the MF vs PV and ET cohorts. 

Mean and maximum subitem scores were comparable in PV and ET cohorts except for the mean pruritus score (1.3 vs 0.5, p < 0.0001).

In the PV cohort, patients with high vs low symptom burden were younger (p = 0.04), predominantly female (p = 0.003), had higher CRP levels (p = 0.04), and initiated treatment later (p = 0.01).

Patients in the ET cohort with high mean symptom burden were predominantly female (p = 0.0001), initiated treatment later (p = 0.07), and had lower median platelet counts (p = 0.08). 

In the MF cohort, high mean symptom burden was associated with elevated LDH levels (p = 0.02), absence of antiplatelet therapy (p = 0.04), high WBC levels (p = 0.06), and lower median platelet counts (p = 0.05). Of note, thrombocytosis was associated with lower symptom burden (p = 0.006).

A higher mean MPN-SAF TSS was associated with worse OS (p = 0.002) and increased risk of fibrotic transformation (p = 0.03) in the PV cohort.

These findings provide a comprehensive account of MPN symptom burden across subtypes in a real-world setting. Several important confirmatory and novel findings were disclosed, with implications for treatment individualization. 

Abbreviations: CRP, C-reactive protein; ET, essential thrombocythemia; IC, International Consensus; LDH, lactate dehydrogenase; MF, myelofibrosis; MPN, myeloproliferative neoplasms; MPN-SAF TSS, Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score; OS, overall survival; PRO, patient-reported outcome; PV, polycythemia vera; QoL, quality of life; WBC, white blood cell; WHO, World Health Organization.

References

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