The American Society of Hematology (ASH) released new guidelines on the management of myelofibrosis which recommend expanded molecular testing, early transplant referral, appropriate use of JAK inhibitors, and aspirin therapy in select patients. The guidelines were published by Stein et al in Blood Advances.
Myelofibrosis is a rare type of myeloproliferative neoplasm, with estimates that up to 6 per 100,000 people are living with the disease. Individuals with myelofibrosis are at risk of developing acute myeloid leukemia and other serious, disease-related complications, including enlargement of the spleen and development of blood clots. Myelofibrosis can often be managed over time with medications, including Janus kinase (JAK) inhibitors, which can help reduce spleen enlargement and ease other symptoms of the disease. Currently, the only potentially curative treatment is allogeneic stem cell transplant.
ASH’s newly published evidence-based guidelines help address decision-making gaps in the care of patients with myelofibrosis. Because myelofibrosis is rare and progressive, approaches to treatment can vary substantially among clinicians.
The guidelines consist of 10 recommendations aimed at improving outcomes for individuals living with myelofibrosis, from testing to transplant management. They were developed through a rigorous review process by a multidisciplinary expert panel that included myelofibrosis and transplant specialists, methodology experts, and a patient and caregiver with lived experience.
The recommendations include guidance on:
- Extended molecular testing at diagnosis to help identify high-risk mutations and to guide care
- Early transplant consultation for higher-risk individuals receiving and responding to JAK inhibitors
- Strategies for managing anemia related to myelofibrosis or JAK inhibitor therapy
- Use of JAK inhibitors for lower-risk individuals with disease-related symptoms and avoidance of JAK inhibitors in higher-risk individuals without disease-related symptoms
- Low-dose aspirin for patients with the JAK2 V617F mutation.
“These recommendations address questions that routinely come up for clinicians and patients managing myelofibrosis, and our hope is that they will enable hematologists to more confidently deliver better care to their patients,” said guideline panel Chair Brady Stein, MD, Professor of Medicine at Northwestern University’s Feinberg School of Medicine. “That said, important gaps in evidence remain, and we hope the questions we asked can help set a future research agenda to further improve care for patients with this disease.”
The guidance identifies several areas in which more research is needed to continue improving outcomes for patients, including enhancing prediction of which patients will benefit from specific treatments, establishment of better approaches to myelofibrosis-related anemia, and clarification of timing for transplant.
Additional guideline resources, including snapshots, visual summaries, and teaching slides, can be accessed at hematology.org/myelofibrosis-guidelines.
DISCLOSURE: For full disclosures of the guidelines’ authors, visit ashpublications.org/bloodadvances.
