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ASH 2024 | Real-world efficacy and safety of momelotinib for myelofibrosis: evaluation of a UK-wide study

Alex Rampotas, MBBS, MRCP, University College London, London, UK, comments on the real-world efficacy and safety of momelotinib in patients with myelofibrosis (MF). In patients who received the drug for six months, an encouraging anemia response was observed, and the toxicity profile of the agent was favorable, with the most common adverse event being Grade 1 or 2 peripheral neuropathy that did not significantly impact treatment continuation. Dr Rampotas emphasizes that momelotinib is a valuable therapeutic option for patients with MF. This interview took place at the 66th ASH Annual Meeting and Exposition, held in San Diego, CA.

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Transcript (AI-generated)

Momelotinib is a recently approved drug for myelofibrosis and that gives us a very good other option for patients with myelofibrosis. In the UK we had access to momelotinib via the Compassionate Access Scheme and also very recently it was approved for first-line myelofibrosis with anemia. In the UK we work collaboratively across many centers via the MPN study group and we collect the data of use of momelotinib in the real world...

Momelotinib is a recently approved drug for myelofibrosis and that gives us a very good other option for patients with myelofibrosis. In the UK we had access to momelotinib via the Compassionate Access Scheme and also very recently it was approved for first-line myelofibrosis with anemia. In the UK we work collaboratively across many centers via the MPN study group and we collect the data of use of momelotinib in the real world. 

We are very pleased to say that we have demonstrated good efficacy with momelotinib, good anemia responses. We measure them at six weeks, three months, and six months. And actually, for the patients that were able to stay on momelotinib for six months, we have demonstrated more than 60% responses in anemia. Momelotinib can provide very good options for clinicians treating myelofibrosis patients. 

We also looked into the safety of this drug and we have identified peripheral neuropathy as the most common adverse event in the real world. This is very important, however, in most cases the patients were able to continue the drug. The neuropathy was usually grade 1 or grade 2. And given the response that they had, actually they could tolerate it very well and the clinicians were able to continue the treatment. The second most common adverse event was GI symptoms. But most interestingly, we have identified a certain percentage of patients that they developed thrombocytosis, which was a bit unexpected. We think that this may have to do with the inhibition of the ACVR1 receptor of momelotinib as well as the JAK inhibition that may affect the hepcidin and iron regulation in patients, but this is definitely an area that we continue to study and I think it warrants further exploration. 

In summary in our patient cohort momelotinib was a very good treatment that achieved good anemia responses and as we continue to use it we can learn more and given that in myelofibrosis we have a few drugs available I think it’s very important for patients that will have another option to offer to them with a relatively safe profile and also good efficacy.

 

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