Pankit Vachhani, MD, reviews recent changes to the NCCN Guidelines, highlighting the addition of luspatercept for the treatment of primary myelofibrosis.
Ruben Mesa, MD, FACP: Pankit, you’re a member of the panel that’s on the NCCN [National Comprehensive Cancer Network]. They recently had some updates to the guidelines in treating myelofibrosis. Why don’t you share what a hematologist needs to know about some of the changes, or the adjustments that you made to the guidelines?
Pankit Vachhani, MD: Absolutely. I will mention 2 of those. One is related to the point about ruxolitinib and fedratinib, which is that the NCCN Guidelines have made it more clear in terms of its language. If you had an inadequate response or you lost response to 1, then you could use the other, in cases of high-risk myelofibrosis. The biggest change that there has been compared with the NCCN Guidelines from 2020 is the addition of the luspatercept to the guidelines. Luspatercept, as you may know, was approved a couple of years ago for the treatment of anemia of patients with beta-thalassemia, as well as those with myelodysplastic syndromes, ringed sideroblasts, myelodysplastic syndromes, myeloproliferative neoplasms, and thrombocytosis.
Luspatercept is given as a skin injection once every 3 weeks. It was studied in a phase 2 trial that was previously reported, a trial that examined patients who were on ruxolitinib or who were not on ruxolitinib, who were transfusion dependent or not. Efficacy was seen in these different cohorts; it was somewhere in the range of 15% to 30%. It’s now added as a suggestion to use in those patients with myelofibrosis [MF] related–anemia and for patients with an underlying erythropoietin level of more than 500 IU/L. That’s an area where erythropoietin-stimulating agents are less likely to work. Of note is the phase 3 clinical trial looking at luspatercept vs placebo in patients with MF-related anemia while on JAK inhibitors and transfusion dependent; the phase 3 independent study is ongoing. That’s a big addition to the guidelines.
Ruben Mesa, MD, FACP: In the guidelines, it has been gratifying to see. The guidelines provide some framework for a practice that had been quite heterogeneous, particularly in the United States, where some utilized the last review article they read or the last board review they heard or an outdated chapter of the Ronald Hoffman textbook [Hematology: Basic Principles and Practice] to guide how they were treating folks. It has helped to bring it into greater alignment. With more therapies in development, its needs have been so important. According to those guidelines, you start people on the therapy. Then you monitor the patients for response and assess signs and symptoms of disease progression.
Transcript Edited for Clarity