How should I approach the management of a patient with myelofibrosis who is experiencing significant symptoms but is not a candidate for stem cell tra

In managing a patient with myelofibrosis who is symptomatic but not eligible for stem cell transplantation, the approach should focus on symptom control, improving quality of life, and managing complications such as anemia and splenomegaly. Symptom-directed therapy includes the use of JAK inhibitors like ruxolitinib, which have demonstrated efficacy in reducing splenomegaly and alleviating constitutional symptoms such as fatigue, night sweats, and weight loss NICE NG35.

Anemia management is critical, as anemia is a common and debilitating feature in myelofibrosis. Treatment options include erythropoiesis-stimulating agents, androgens, or transfusions depending on severity and patient tolerance Passamonti et al. 2022. Emerging therapies targeting anemia are under investigation but not yet standard of care NICE NG35.

Splenomegaly can be managed medically with JAK inhibitors; if refractory and causing significant symptoms, other options such as splenectomy or radiotherapy may be considered cautiously, weighing risks and benefits NICE NG35.

Supportive care measures including management of infections, thrombosis risk, and symptom palliation are essential components of care NICE NG35.

While allogeneic stem cell transplantation remains the only curative option, it is not suitable for all patients due to age, comorbidities, or donor availability. For these patients, the focus remains on symptom control and quality of life improvement through the above strategies Kröger et al. 2024Kröger et al. 2025.