Myelofibrosis: best practices, controversies and 2019 update

ABSTRACT

Introduction: Recent advances in the prognostic scheme and treatment of primary and secondary myelofibrosis (MF) have resulted in an overwhelming amount of clinical information to assimilate. The authors believe a comprehensive review that summarizes the most recent published literature, could serve as guidelines for the practicing hematologist.

Areas covered: The authors provide a summary of landmark articles regarding epidemiology, symptoms, and pathogenesis of disease. The authors conducted a systematic literature review to answer questions regarding differences between primary myelofibrosis (PMF) and secondary myelofibrosis (SMF), appropriate use and selection of the current risk-stratification models, early versus late treatment of MF and current practices in allogeneic hematopoietic stem cell transplantation (allo-HCT) for MF. The authors conclude the article with their clinical opinion based on their experience and literature review. The purpose of this article is to identify current practices, address any variation, identify and investigate conflicting results and produce statements to guide decision-making.

Expert opinion: In this section, the authors advocate for and provide examples of a standardized way of incorporating future discoveries in the pathogenesis and risk-stratification models of MF. They also discuss the importance of using only one risk-stratification model for PMF and one for SMF and their reasoning for early instead of late treatment of MF.

KEYWORDS:

• Hematopoietic stem cell transplantation
• interferon
• myelofibrosis
• myeloproliferative neoplasm
• prognostic scores

Article highlights

• It is important to recognize the different clinical characteristics and outcomes in PMF and SMF since disease status is associated with prognosis.

• After the diagnosis of PMF or SMF is made, the appropriate risk stratification system should be selected amongst the multiple risk prognostication scores available.

• For PMF, the MIPSS70+ version 2.0 (or GIPSS) should be used at diagnosis if karyotype and mutational status information is available while for SMF, the MYSEC-PM should be used at diagnosis if CALR mutation information is available.

• For a select group of patients with a hypercellular marrow and low or intermediate-1 risk disease, early treatment with low-dose rINF should be considered to delay progression of disease and reverse marrow fibrosis.

• Pre-transplant JAK inhibitor therapy and/or splenectomy should be considered in selected patients prior to transplant.

• Match related donors and peripheral blood stem cells should be used when possible.

• Conditioning regimens for transplant in MF remain controversial.

Declaration of interest

RT Silver has acted on advisory boards for Abbvie and PharmaEssentia. U Gergis has received speaker bureau for Incyte. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or conflict with the subject matter or materials discussed in this manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Correction Statement

This article has been republished with minor changes. These changes do not impact the academic content of the article.

Additional information

Funding

This paper was not funded.