Current therapeutic strategies in the management of primary myelofibrosis in young adults

Abstract

Background. Primary myelofibrosis (PMF) is a Ph-chromosome-negative myeloproliferative neoplasm. In young patients (<40 years), PMF is rare and presents with diverse clinical manifestations, but it is characterized by a longer life expectancy, which necessitates a personalized therapeutic approach. Objective(s). The aim of this paper is to analyze treatment strategies for PMF in young patients, considering the molecular features, established prognostic models, and available therapeutic options. Materials and methods. A literature review was conducted using the PubMed, Scopus, and Web of Science databases over the past 10 years. Modern therapeutic strategies used in the treatment of PMF were analyzed and systematized, with particular attention given to the molecular features of the disease and their impact on clinical course and therapeutic management. Results. The only curative treatment is allogeneic hematopoietic stem cell transplantation, with transplant indications determined based on prognostic scoring systems (DIPSS, MIPSS70+, GIPSS). In young patients, CALR mutations are more frequently encountered and are associated with a favorable prognosis; the presence of high-risk mutations requires a more aggressive therapeutic strategy. JAK inhibitors (ruxolitinib, fedratinib, momelotinib) are used to control symptoms and splenomegaly and also serve as bridging therapy before transplantation. Questions remain regarding optimal treatment duration, tolerability, and the long-term impact on survival. Conclusion(s). The treatment of primary myelofibrosis in young patients requires a personalized approach based on molecular profiling and prognostic stratification. Additional studies are needed to assess the long-term efficacy of these modern therapeutic strategies in this age group.