Prefibrotic myelofibrosis: treatment algorithm 2018

Area of Science:

• Hematology
• Oncology
• Internal Medicine

Background:

• Prefibrotic myelofibrosis (pre-PMF) is a distinct chronic myeloproliferative neoplasm under the 2016 WHO classification.
• Clinical presentation varies widely, from isolated thrombocytosis resembling essential thrombocythemia (ET) to high-risk myelofibrosis symptoms.
• Retrospective data indicate pre-PMF survival is intermediate between ET and overt myelofibrosis.

Purpose of the Study:

• To evaluate the prognostic value of the International Prognostic Scoring System (IPSS) in pre-PMF.
• To guide clinical management strategies based on patient risk stratification.
• To define pragmatic approaches for managing bleeding and thrombosis risks.

Main Methods:

• Retrospective analysis of pre-PMF patient data.
• Application of the International Prognostic Scoring System (IPSS) for survival prediction.
• Review of current treatment guidelines for myeloproliferative neoplasms.

Main Results:

• The IPSS can predict survival in pre-PMF but does not adequately differentiate between intermediate-1 and intermediate-2 risk groups.
• Management strategies should balance life expectancy with risks of bleeding and thrombosis.
• Low-risk patients may be observed; intermediate-risk patients require symptom-based treatment; high-risk patients need intensive management.

Conclusions:

• Risk stratification is crucial for managing pre-PMF patients.
• Tailored treatment approaches, including observation, symptom management, or intensive therapy, are necessary.
• Pragmatic interventions for bleeding and thrombosis include aspirin or anticoagulation, with hydroxyurea for cytoreduction when indicated.