How I diagnose and treat patients in the pre-fibrotic phase of primary myelofibrosis (pre-PMF) - practical approaches of a German expert panel discussion in 2024

被引：0

作者：

Griesshammer, Martin ^{[1
]}

Al-Ali, Haifa Kathrin ^{[2
]}

Eckardt, Jan-Niklas ^{[3
,4
]}

Fiegl, Michael ^{[5
]}

Goethert, Joachim ^{[6
]}

Jentsch-Ullrich, Kathleen ^{[7
]}

Koschmieder, Steffen ^{[8
,9
]}

Kvasnicka, Hans Michael ^{[10
]}

Reiter, Andreas ^{[11
]}

Schmidt, Burkhard ^{[12
]}

Heidel, Florian H. ^{[13
,14
,15
,16
]}

机构：

[1] Univ Bochum, Univ Clin Hematol, Johannes Wesling Med Ctr Minden, Bochum, Germany

[2] Univ Hosp Halle, Krukenberg Canc Ctr, Halle, Germany

[3] Univ Hosp Dresden, Dept Internal Med 1, Dresden, Germany

[4] Tech Univ Dresden, Else Kroner Fresenius Ctr Digital Hlth, Dresden, Germany

[5] Hematol Oncol Germering, Germering, Germany

[6] Univ Hosp Essen, Dept Hematol & Stem Cell Transplantat, Essen, Germany

[7] Gemeinschaftspraxis Hamatol & Onkol, Magdeburg, Germany

[8] Rhein Westfal TH Aachen, Dept Hematol Oncol Hemostaseol & Stem Cell Transp, Fac Med, Aachen, Germany

[9] Aachen Bonn Cologne Dusseldorf CIO ABCD, Ctr Integrated Oncol, Dusseldorf, Germany

[10] Univ Witten Herdecke, Pathol, Dept Humanmed, Fak Gesundheit, Herdecke, Germany

[11] Univ Hosp Mannheim, Dept Hematol & Oncol, Mannheim, Germany

[12] Hamatol & Onkol Munchen Pasing MVZ GmbH, Munich, Germany

[13] Hannover Med Sch MHH, Hematol Hemostasis Oncol & Stem Cell Transplantat, Hannover, Germany

[14] Hannover Med Sch MHH, Cellular Therapy Ctr CTC, Hannover, Germany

[15] Leibniz Inst Aging, Fritz Lipmann Inst, Jena, Germany

[16] Hannover Med Sch, Hematol Hemostasis Oncol & Stem Cell Transplantat, Carl Neuberg Str 1, D-30625 Hannover, Germany

来源：

ANNALS OF HEMATOLOGY | 2025年 / 104卷 / 01期

关键词：

Pre-PMF; Prefibrotic myelofibrosis; MPN; Myeloproliferative neoplasm; Targeted therapy; HEALTH-ORGANIZATION CLASSIFICATION; RUXOLITINIB; PROGNOSIS; SURVIVAL; OUTCOMES; IMPACT;

D O I：

10.1007/s00277-025-06191-7

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

The prefibrotic phase of primary myelofibrosis (pre-PMF) represents a distinct subentity within the spectrum of myeloproliferative neoplasms (MPNs), recognized by the World Health Organization (WHO) and the International Consensus Classification (ICC). Pre-PMF is characterized by unique morphological, clinical, and molecular features, distinguishing it from essential thrombocythemia (ET) and overt myelofibrosis (overt-PMF). The diagnostic process for pre-PMF relies on bone marrow histology, identification of molecular mutations and exclusion of other myeloid neoplasms. Misclassification remains a significant challenge due to overlapping phenotypes and the heterogeneity of clinical presentations, which range from asymptomatic cases to severe cytopenias and a high thrombotic risk. Management strategies for pre-PMF focus on mitigating symptom burden, reducing thromboembolic events, and preventing disease progression. Low-risk patients often benefit from observational approaches or low-dose aspirin, while cytoreductive therapies, such as hydroxyurea or interferon-alpha, are utilized in symptomatic or high-risk cases. JAK inhibitors like ruxolitinib have shown promise in addressing splenomegaly and systemic symptoms, although their role in pre-PMF requires further investigation. Advances in artificial intelligence are enhancing diagnostic precision by refining bone marrow histopathological analysis, paving the way for more accurate disease classification and tailored therapeutic strategies. This position paper integrates insights from a German expert panel discussion, underscoring the need for interdisciplinary collaboration, adherence to updated WHO/ICC diagnostic criteria, and personalized treatment approaches. By addressing diagnostic challenges and therapeutic nuances, it seeks to improve outcomes and quality of life for patients navigating the complexities of pre-PMF.

引用

页码：295 / 306

页数：12