What Myelofibrosis Treatment Types Are Available?
Dr. Naveen Pemmaraju outlines available myelofibrosis therapies, such as JAK inhibitors, and discusses the role of clinical trials and emerging treatments for managing the disease.
Dr. Naveen Pemmaraju is Director of the Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Program and Professor in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Pemmaraju.
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Transcript:
Katherine Banwell:
So, once all testing is complete and the patient has an accurate diagnosis, they’ll work with their doctor then on a treatment approach. You’ve touched on this a little bit, but
What are the types of treatment available for people with myelofibrosis?
Dr. Naveen Pemmaraju:
Yeah, thanks, Katherine. We’ll keep it general and standard of care. As you mentioned at the top, I’ll reiterate, that none of these are intended to be specific instructions for specific folks. However, in general, for the category of patients with myelofibrosis, in general, there’s not many treatments, unfortunately. As of 2024, we have only four standard JAK inhibitors. So, that’s this pathway we’re talking about, JAK-STAT. Interestingly, you don’t have to be JAK2V617F mutated. These are for the whole pathway.
So, all patients with myelofibrosis, are intermediate to high risk. The first one, Katherine, is ruxolitinib (Jakafi), which has been around for more than a decade, and first in class JAK inhibitor. The second drug is fedratinib (Inrebic). The third is pacritinib (Vonjo), approved only in 2020 for those patients with less than platelets of 50. Then the myelofibrosis drug, momelotinib (Ojjaara), just approved not even a year ago, in September of 2023 for myelofibrosis with anemia. So, those four are considered as called JAK inhibitors.
They are really the only targeted therapy class of drugs specifically approved in the MF space. Outside of that, there’s older and other drugs that me and others have used, if you will, so-called off-label or historical use, hydroxyurea (Hydrea), interferon products such as pegylated interferon. Hypomethylators such as azacitidine (Vidaza) and decitabine (Dacogen), particularly in more advanced cases. Some of those drugs are borrowed from MDS and AML and have been around for decades.
Then of course, finally, clinical trials. We really recommend folks, if they have the ability and feasibility, clinical trials, even in the first diagnosis setting. So, untreated, first therapy. These clinical trials, Katherine, are based on three factors. One is JAK inhibitor plus another agent. So, that’s kind of like a combination trial. Two is add-on agents. So, you’re already on the JAK inhibitor for a while, maybe it’s starting to not work. Then you add in a third agent.
Then three is a completely novel agent beyond the JAK-STAT pathway. Then maybe we can even add a fourth one now as this is evolving in real-time, which is anemia-targeting drugs. Many of our patients have either transfusion-dependent or bad anemia. Some of the drugs that are being developed are specifically aimed at them.
