Myelofibrosis Clinical Considerations

Management Approach and Challenges

The primary goals of therapy in primary myelofibrosis include prolongation of survival, and when possible, cure, which may be achieved by allogeneic stem-cell transplantation. Palliation of symptoms and improved quality of life also represent important management goals.1

Clinical challenges in patients with primary myelofibrosis1:
  • Shortened survival
  • Increased risk of leukemic transformation
  • Severe anemia, often creating the need for frequent red blood cell transfusions
  • Severe constitutional symptoms
  • Marked leukocytosis or thrombocytosis
  • Thrombohemorrhagic complications
  • Marked hepatosplenomegaly often accompanied by related symptoms
  • Nonhepatosplenic extramedullary hematopoiesis that could lead to complications
  • Recurrent gout

Patient Symptom Burden

The Myeloproliferative Neoplasm-Symptom Assessment Form Total Symptom Score (MPN-SAF TSS); is a 10-item instrument designed to monitor the most representative and clinically relevant symptoms among patients with MPNs. The tool records the patient’s assessment of the incidence and severity of the following symptoms2:

  • Fatigue
  • Early satiety
  • Abdominal discomfort
  • Inactivity
  • Concentration problems
  • Night sweats
  • Itching
  • Bone pain
  • Fever
  • Weight loss

The tool can be used to provide a fast, accurate assessment of symptoms associated with MPNs, track symptoms over time, and guide subsequent management decisions.2

Analysis of MPN Symptom Burden The MPN Landmark survey was a large-scale analysis of 813 patients with MPNs (myelofibrosis, n=207) and assessed perceptions of overall disease burden, quality of life (QOL), activities of daily living, and work productivity. In this survey, the 5 most common symptoms reported over the prior 12 months among patients with MF were3*†:
show male body profile with icons surrounding him that depict physical activity, social functioning, independence with daily tasks and global quality of life
*Survey sponsored by Incyte Corporation. Symptoms represent only those included in the MPN-SAF TSS and are not inclusive of all symptoms that were assessed in the MPN Landmark survey.

Effects on Quality of Life (QOL), Activities of Daily Living, and Work/Productivity

In the MPN Landmark survey3:

  • 81% of patients reported reduced QOL related to their condition
    • This was true even for patients with low prognostic risk scores and with lowest symptom severity
  • 53% (110/207) of patients reported interference with daily activities at some point
    • 21% (43/207) reported a great deal of interference§
  • 79% (163/207) indicated that myelofibrosis interfered with family or social life
  • 59% (70/119) reported reduced work hours at some point
A score of >1 on a scale of 1 (not at all) to 5 (a great deal) §A score of 5 on a scale of 1 (not at all) to 5 (a great deal)
References

1. Barbui T, Barosi G, Birgegard G, et al. J Clin Oncol. 2011;29(6):761-770. 2. Emanuel RM, Dueck AC, Geyer HL, et al. J Clin Oncol. 2012:30:4098-4104. 3. Mesa R, Miller CB, Thyne M, et al. BMC Cancer. 2016;16:167. doi: 10.1186/s12885-016-2208-2.

 
Resources
By: World Health Organization (WHO)
A diagnostic worksheet from the WHO for primary MF, PV, and ET.
By: International Working Group for Myeloproliferative Neoplasms Research and Treatment (IWG-MRT)
Diagnostic criteria from the IWG-MRT.