Myelofibrosis is a rare hematologic malignancy associated with abnormal blood counts, splenomegaly, symptoms of chronic inflammation, and scarring of the bone marrow. Prognosis varies, although in most cases is poor. Without hematopoietic stem cell transplant, cure is not possible.
Characteristics of the disease, goals of treatment, and new treatment strategies were discussed in an oral presentation at the 2023 ONA Summit Live Virtual Meeting.
Treatment is focused on alleviation of symptoms, management of cytopenias, and prevention of complications. Current care is focused on inhibition of altered signaling of the JAK/STAT pathway. Ruxolitinib, fedratinib, and pacritinib are FDA-approved for intermediate- and high-risk myelofibrosis. Ruxolitinib was the first in class, JAK 1 and 2 inhibitor, approved in 2011 based on spleen volume reduction and improvement in myelofibrosis-related symptoms.
Fedratinib was approved in 2019 and pacritinib in 2022. Both fedratinib and pacritinib have the potential for spleen volume reduction, and symptom improvement. Pacritinib provides a treatment option for patients with thrombocytopenia.
A new drug application for momelotinib, a JAK1/JAK2/ACVR1/ALK2 inhibitor, was recently accepted by the US FDA. In the MOMENTUM clinical trial (ClinicalTrials.gov Identifier: NCT04173494), momelotinib met all primary and secondary endpoints. Spleen volume response, improvement in symptoms, and transfusion independence were achieved with this new agent.
Nurses and other allied healthcare professionals can benefit from understanding myelofibrosis, potential treatments, adverse side effects, and recommendations for monitoring to educate patients and engage them in discussion for shared decision-making.
Sara Tinsley-Vance is a nurse practitioner and quality of life researcher in the Malignant Hematology Program at Moffitt Cancer Center in Tampa, Florida
Tinsley-Vance SM. Myelofibrosis: therapeutic options for a complex rare disease. Oral presentation at: 2023 ONA Summit Live Virtual Meeting; March 17-19, 2023.