Unmet Needs for Treating Myelofibrosis Beyond Symptoms


Raajit Rampal, MD, PhD, discusses unmet needs for therapies to treat patients with myelofibrosis.

Raajit Rampal, MD, PhD, hematologic oncologist at Memorial Sloan Kettering Cancer Center, discusses unmet needs for therapies to treat patients with myelofibrosis.

Janus kinase (JAK) inhibitors are used to treat myelofibrosis symptoms, and new agents are in clinical trials to improve the efficacy of therapy. Rampal says that while these drugs are effective at reducing splenomegaly and controlling other symptoms of myelofibrosis, there is no clear disease-modifying therapy that does more to address the underlying cause of these symptoms. Therefore, a major unmet need is a disease-modifying intervention that will improve patients’ survival outcomes.

Other needs include drugs that can treat anemia and agents that produce a greater response since current agents have approximately 30% response rate in reducing spleen size and total symptom score. Patients with low platelet counts need better therapies since they have a poor prognosis, and some JAK inhibitors cannot be used to treat them.

Finally, patients with progressive myelofibrosis in the accelerated or blast phase have very few options and their expected overall survival is extremely short, meaning there is an urgent need for any more effective treatment for these patients.


0:08 | We don’t have, at this point, what I would call a disease-modifying therapy. All of the drugs that we have are good at controlling symptoms, shrinking spleen, and all of those things are important, but none of them I would at this point characterize as a disease-modifying intervention. Will any of the new drugs fulfill that? I don't know, [and] I think that remains to be seen. We need to see data to support that idea, [and] I think that's the biggest need.

We still need better drugs for anemia. Most of the drugs we have only have about a 30% or so response rate. For patients with low platelets, they have an especially poor prognosis, and we need to have better interventions for them. For people who have progressive disease, accelerated or blast phase disease, we have very few options for those patients. To me, that's a completely unmet need.

Related Videos
Related Content