Fedratinib Helps Myelofibrosis Patients with Low Platelet Counts | Haifa Katrhin Al-Ali, MD | #ASH24

My name is Haifa Kathrin Al-Ali. I'm a hematologist from Germany working at the University Hospital of Halle in Germany, and I am the head of the cancer center there. We presented a sub analysis of a phase three clinical trial called freedom two that was recently published. This trial, dealt with the role of a Jack inhibitor fedratinib.

That is approved in the treatment of patients with myelofibrosis. And in this trial, we made mainly the point could also patients with low platelets, those below 100 between 50 and 99 thousand also benefits from this treatment, because we know that the low platelet counts is not a good thing for patients with myelofibrosis, and it has a negative impact on survival.

So in this sub analysis we just checked the patients included in this study, whether they have platelets between 50 to 99 versus the patients with the higher platelets, and looked at the impact of the drug on safety and efficacy. All patients had to be refractory for a firstline Jack inhibitor, usually ruxolitinib which is second line treatment.

And what we could see from the data is that no matter how high or low the platelets are, all patients could benefit very well from a full dose of the drug 400mg once daily, irrespective of the platelet count. Before the start of the trial. This is not only in terms of the platelets counts remained stable over months, but also response.

Interestingly, the response in the patients with the low platelets was even better than the response in the patients with higher platelet counts. So on the bottom line, the spleen, the symptoms improved in almost half of the patients treated, even those with the low platelet counts. Sure, we have side effects. Platelet counts have to be checked. And they did go down in about 30% of patients.

But only one patient had to be discontinued from the treatment. Otherwise we tried to modify the dosing so patients were able to continue with treatment. Gastrointestinal side effects are also present, but I think we have all learned how to deal with this drug. Giving it in the evening after meals so that patients can tolerate their medication much better infections.

Some Covid 19 infections were present because this trial recruited at the time of the Covid 19 pandemic. So in summary, patients with moderate thrombocytopenia with the platelet counts between 50 and 99, they could very well profit from the Jak inhibitor treatment with Fedratinib as second line, because there is this so-called platelet sparing effect with very nice responses in terms of spleen and symptoms, which is very, very important for the quality of life of patients.

The drug is already approved for the first line setting, and indeed, the data for the low platelets have been already examined in the initial trial that led to the approval of the drug where again, the same finding was observed, namely that those patients with platelets between 50 to 99. They do have the same benefit compared to patients with higher platelets.

Although, to be honest, the number of patients included in this initial trial was small. But the findings are quite similar. And, you know, the second line treatment is always a bigger challenge in the first line setting.