On December 6 (local time), a highly anticipated special session on the ASH Clinical Practice Guidelines on Myelofibrosis was successfully held during the American Society of Hematology (ASH) Annual Meeting. The session focused on core aspects of myelofibrosis (MF) management, including thrombosis risk management, iron chelation therapy, the use of JAK inhibitors, and strategies for hematopoietic stem cell transplantation. Oncology Frontier · Hematology Frontier invited Professor Duan Minghui from Peking Union Medical College Hospital to provide a timely interpretation of the guideline highlights and to share insights into their relevance for clinical practice in China.
Oncology Frontier · Hematology Frontier: In the ASH Clinical Practice Guidelines on Myelofibrosis, which recommendations do you consider the most important? In particular, how do you view the clinical value of next-generation sequencing (NGS), aspirin therapy, and iron chelation therapy?
Professor Duan Minghui:
At present, the full text of the ASH Myelofibrosis Clinical Practice Guidelines has not yet been officially released. The session held yesterday discussed the core content in a summarized format, focusing mainly on three key areas.
First, thrombosis risk management. Unlike previous perceptions, thrombosis prevention in patients with myelofibrosis has now been elevated to the same level of importance as in polycythemia vera and essential thrombocythemia. Risk assessment should comprehensively incorporate factors such as age, genetic profile, and prior history of thrombosis, and based on this evaluation, decisions should be made regarding the initiation of antiplatelet therapy, including aspirin.
Second, iron chelation therapy. In transfusion-dependent patients with myeloproliferative neoplasms, long-term iron overload should not be overlooked. The use of iron chelators has become an essential intervention to improve survival outcomes and preserve organ function.
Third, indications for JAK inhibitor therapy. The meeting reiterated that the use of JAK inhibitors should be primarily driven by symptom control rather than risk stratification alone. Therefore, regardless of whether patients are classified as low-, intermediate-, or high-risk, JAK inhibitors are recommended for those with MPN-related symptoms and splenomegaly. This recommendation is particularly instructive and highly relevant for clinical practice in China.
In addition, the application of next-generation sequencing (NGS) remains critically important in patients with myeloproliferative neoplasms (MPNs). The guidelines recommend the use of standardized gene panel testing whenever possible, so that MPN patients and clinicians worldwide can rely on unified interpretation standards. Systematic analysis of both driver mutations and non-driver genetic abnormalities enables prognostically relevant research and supports the development of targeted therapeutic strategies, ultimately facilitating more scientific and harmonized treatment planning.
Oncology Frontier · Hematology Frontier: Regarding the timing of transplantation and pre-transplant treatment strategies, what recommendations does the guideline provide? How can these be implemented in real-world clinical practice in China, and are there any special considerations?
Professor Duan Minghui:
For patients with myelofibrosis, hematopoietic stem cell transplantation should still be guided by individualized assessment. Risk stratification plays a central role in this decision-making process. Patients classified as intermediate- or high-risk should be more strongly considered for transplantation. Although JAK inhibitors and related therapies can improve symptoms, clinical parameters, and overall physical condition, treatment responses must be closely monitored. Once a decline in efficacy is observed, transplantation should be initiated in a timely manner. Therefore, for patients with myelofibrosis, hematopoietic stem cell transplantation remains an essential and unavoidable component of the overall treatment strategy.
Oncology Frontier · Hematology Frontier: Overall, what lessons do international guidelines offer for the management of myelofibrosis in China? Based on your team’s research, are there any distinctive experiences from China in diagnostic or management strategies that are worth sharing?
Professor Duan Minghui:
The challenges of myelofibrosis (MF) management in China are largely similar to those seen internationally. In the past, treatment was mainly constrained by limited access to JAK inhibitors, with ruxolitinib being the only available option for a long time. Since last year, the approval of gilteritinib and the reporting of multiple investigational JAK inhibitors—including domestically developed agents—at this ASH meeting have significantly expanded the therapeutic landscape, and more options are expected in the near future.
With a broader range of available drugs, clinicians can now offer more individualized treatment choices beyond ruxolitinib, tailored to patients’ specific symptoms and clinical needs. For example, gilteritinib may demonstrate advantages in spleen volume reduction and anemia improvement, although it has certain limitations in terms of non-hematologic toxicity, which may affect tolerability in some patients. Therefore, the diversification of therapeutic options not only broadens treatment strategies but also supports more personalized decision-making, ultimately improving overall patient outcomes.
However, more importantly, for patients with myelofibrosis, the efficacy of JAK inhibitor monotherapy remains limited and is insufficient to fully meet clinical needs. Therefore, both in China and globally, future treatment strategies should broaden their scope and actively explore combination regimens centered on JAK inhibitors. The therapeutic focus may gradually shift toward reducing the quantitative burden of driver gene mutations and suppressing clonal evolution driven by non-driver genes. Through such proactive treatment strategies, it may be possible to decrease patients’ reliance on hematopoietic stem cell transplantation, prolong survival, and ultimately achieve a meaningful improvement in overall treatment outcomes for patients with myelofibrosis.
Expert Profile
Professor Duan Minghui
Peking Union Medical College Hospital
Chief Physician, Department of Hematology, Peking Union Medical College Hospital
Vice Chair, First Youth Committee, Beijing Society of Hematology
Member, Hematopoietic Stem Cell Application Group, Chinese Society of Hematology
Vice Chair, MPN/MDS Branch, Chinese Medical Education Association
Member, Expert Committee of the China MDS and MPN Working Group, Hematologic Malignancies Committee, Chinese Anti-Cancer Association
Member, Expert Committee, Anti-Leukemia Alliance, Chinese Society of Clinical Oncology (CSCO)
Vice Chair, Hematology Expert Committee of patient assistance programs (including Glivec), China Charity Federation
Medical Science Communication Expert, Clinical Physician Science Popularization Program, Ministry of Health
Main focus areas include hematopoietic stem cell transplantation, lymphoma, leukemia, myeloproliferative neoplasms (MPN), and myelodysplastic syndromes (MDS)
Specializes in the diagnosis and treatment of complex and rare hematologic diseases
