What is your MPN patient not telling you? Patient and physician perceptions on the burden of myeloproliferative neoplasms (MPNs).

“The clinical presentation is associated with a substantial disease burden and this often leads to a reduced quality of life for many patients.”

Professor Mary Frances McMullin

 

In the first webinar of this series, Professor Mary Frances McMullin, Queen’s University Belfast, gives an overview of MPNs, focusing on the symptom burden for patients and its implications for disease management.

Read a summary and watch short clips of the webinar below, or watch the webinar in full ›

MPNs are a group of disorders that affect multipotent progenitor cells1

WHO classifies MPN based on the presence of the BCR-ABL1 fusion gene on the Philadelphia chromosome.2 The three BCR-ABL1 negative disorders are myelofibrosis (MF), polycythaemia vera (PV) and essential thrombocythaemia (ET), with MF being further subclassified as primary, post-PV or post-ET MF.

  MF PV ET
Characteristics Bone marrow fibrosis and abnorma cell haematopoiesis3 Elevated red blood cells4 Elevated platelets and enlarged megakaryocytes5
Incidence 0.47 per 100,0006 0.84 per 100,0006 1.03 per 100,0006
Survival 4–7 years (median)3 13.5–24 years (overall)7 10–15 years (median)5

MPNs are associated with a risk of transformation8–10

Both PV and ET may transform into MF.8 All three of the disorders can transform into acute myeloid leukaemia (AML), which responds poorly to therapy and demonstrates low survival rates.8 31% of deaths associated with MF are due to transformation to AML.3

  Risk of transformation to AML over 10 years
MF 10–20%9
PV 2.3–14.4%10
ET 0.7–3.0%10

Disease-related symptoms can have a substantial impact on the quality of life for patients with an MPN, impairing activity levels and causing significant morbidity.12

Top 5 symptoms for each disorder by prevalence (%)11

Myelofibrosis (MF) Polycythaemia vera (PV) Essential thrombocythaemia (ET)
Fatigue (98.9%) Fatigue (91.7%) Fatigue (90.3%)
Quality of life (94.7%) Quality of life (85.5%) Quality of life (76.8%)
Inactivity (76.5%) Insomnia (68.1%) Numbness (58.8%)
Early satiety (75.5%) Numbness (66.2%) Insomnia (58.0%)
Insomnia (73.9%) Itching (65.0%) Sad mood (57.3%)

From the MPN-symptom assessment form (MPN-SAF), coadministered with the Brief Fatigue Inventory.

In MF, both splenomegaly and anaemia further add to the already sizeable disease burden.13,14

Here, Professor Mary Frances McMullin describes fatigue in MF and the complications of progressive splenomegaly

 

Prescribing information and adverse event reporting

ET, essential thrombocythaemia; MF, myelofibrosis; PV, polycythaemia vera.

The international MPN Landmark study was carried out in 2016, to understand:12

  • how patients with MF, PV or ET are affected by their disease
  • how physicians view the MPN patient experience
  • gaps between patient and physician perceptions of:
    • symptom burden
    • treatment goals
    • disease management

In this clip, Professor Mary Frances McMullin describes the international MPN Landmark survey, including study design and objectives

 

Prescribing information and adverse event reporting

ET, essential thrombocythaemia; MF, myelofibrosis; PV, polycythaemia vera.

Patients with MPNs experience a significant symptom burden12

The results of the Landmark survey showed that 90% of patients had experienced MPN-related symptoms in the past 12 months, with fatigue or tiredness being the most commonly reported.12 MPN symptoms were reported to be responsible for impaired quality of life in 83% of patients with MF, 72% of patients with PV and 74% of patients with ET.12

Many patients did not recognise symptoms such as numbness, dizziness and abdominal discomfort as being related to their MPN.15 As such, it is likely that patients may not mention these symptoms to their physician unless explicitly asked.

Here, Professor Mary Frances McMullin explains which symptoms patients with MF, PV or ET would most like resolved

 

Prescribing information and adverse event reporting

Patient and physician perception on treatment goals may differ12

When asked their three most important treatment goals, “reduction of symptoms, particularly in MF” was commonly selected by both patient (70%) and physicians (80%).12 However, delaying progression of condition was shown to be more important in all three conditions for patient compared with physician (MF: 58% vs 43%; PV: 57% vs 28%; ET: 66% vs 37%).12 Only 27% of physicians completely agreed with their patients on treatment goals; 66% agreed somewhat.12

ET, essential thrombocythaemia; MF, myelofibrosis; PV, polycythaemia vera. 

The Landmark survey showed that patients with MPNs experience:12

  • severe disease burden
  • reduced quality of life
  • impaired productivity
  • emotional hardship

The results of the survey implied that management of MPNs could be optimised for greater emphasis on treatment of symptoms and better patient–physician communication.12 Treatment of the disease and the symptoms may improve the disease burden and quality of life in a durable way.12

In this clip, Professor Mary Frances McMullin summarises the results of the MPN Landmark survey and explains the implications for disease management

 

Prescribing information and adverse event reporting

MPN webinar 2: managing MF in real-world practice ›  Watch all webinars in full ›  

References

  1. Harrison C, Vannucchi A M. Ruxolitinib: a potent and selective Janus kinase 1 and 2 inhibitor in patients with myelofibrosis. An update for clinicians. Ther Adv Hematol 2012;3:341–354.
  2. Vardiman J W, Thiele J et al. The 2008 revision of the World Health Organization (WHO) classification of myeloid neoplasms and acute leukemia: rationale and important changes. Blood 2009;114(5):937–951.
  3. Cervantes F, Dupriez B et al. New prognostic scoring system for primary myelofibrosis based on a study of the International Working Group for Myelofibrosis Research and Treatment. Blood 2009;113(13):2895–2901.
  4. Polycythemia vera (PV). MPN Research Foundation. Available at: www.mpnresearchfoundation.org. Accessed May 2020.
  5. PathologyOutlines.com. Chronic myeloid neoplasms: essential thrombocythemia. Available at: www.pathologyoutlines.com. Accessed May 2020.
  6. Titmarsh G J, Duncombe A S et al. How common are myeloproliferative neoplasms? A systematic review and meta-analysis. Am J Hematol 2014;89(6):581–587.
  7. Tefferi A, Barbui T. Polycythemia Vera and Essential Thrombocythemia: 2017 Update on Diagnosis, Risk-Stratification, and Management. Am J Hematol 2017;92(1):94–108.
  8. Mesa R, Jamieson C et al. Myeloproliferative neoplasms, version 2.2017, NCCN Clinical Practice Guidelines in Oncology. J Natl Compr Canc Netw 2016;14(12):1572–1611.
  9. Yogarajah M, Tefferi A. Leukemic transformation in myeloproliferative neoplasms: a literature review on risk, characteristics, and outcome. Mayo Clin Proc 2017;92(7):1118–1128.
  10. Cerquozzi S, Tefferi A. Blast transformation and fibrotic progression in polycythemia vera and essential thrombocythemia: a literature review of incidence and risk factor. Blood Cancer J 2015;5:e366.
  11. Scherber R, Dueck A C et al. The Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF): international prospective validation and reliability trial in 402 patients. Blood 2011;118(2):401–408.
  12. Harrison C N, Koschmieder S et al. The impact of myeloproliferative neoplasms (MPNs) on patient quality of life and productivity: results from the international MPN Landmark survey. Ann Hematol 2017;96(10):1653–1665.
  13. Mesa R A. How I treat symptomatic splenomegaly in patients with myelofibrosis. Blood 2009;113(2):5394–5400.
  14. Mughal T I, Vaddi K et al. Myelofibrosis-associated complications: pathogenesis, clinical manifestations, and effects on outcomes. Int J Gen Med 2014;7:89–101.
  15. Mesa R A, Miller C B et al. Differences in treatment goals and perception of symptom burden between patients with myeloproliferative neoplasms (MPNs) and hematologists/oncologists in the United States: findings from the MPN Landmark survey. Cancer 2017;123:449–458.
UK | January 2021 | 100477
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