New Treatment Option for Pre-Fibrotic and Low-Risk Myelofibrosis

Ropeginterferon Alfa-2b Shows Benefit in Early-Stage and Low-Risk Myelofibrosis

There is currently no standard approach for treating pre-fibrotic primary myelofibrosis (pre-PMF) or low/intermediate-1 risk myelofibrosis (MF) . 

A recent ongoing study evaluated ropeginterferon alfa-2b (Besremi, PharmaEssentia)), a long-acting form of interferon, in this patient group. This therapy was developed specifically to treat myeloproliferative neoplasms (MPNs), including early and lower-risk stages of myelofibrosis.

Who was included in the study?

The study included 71 patients with confirmed pre-PMF, or low/intermediate-1 risk overt myelofibrosis. This includes cases that developed from polycythemia vera (PPV-MF) or essential thrombocythemia (PET-MF). All patients needed to reduce their high blood cell counts. The study’s main goals were to see if blood counts could be brought into normal ranges by week 24 and week 52. Other goals included tracking side effects, spleen size, symptoms, genetic mutations, and bone marrow changes.

Blood count improvements seen in most 

Most people showed improved blood counts within the first year of therapy. At week 24, 74% reached a normal hemoglobin rate and 83% normalized their white blood cell count. All participants had platelet counts within the desired range. These results were sustained at week 52. 

Researchers also looked at specific genetic mutations. At 52 weeks, 16 of the 47 people with the JAK2V617F mutation and 20 of 41 people with the CALR mutation showed a drop in mutation levels. These molecular changes are one way to monitor disease control. A reduction in the burden of these mutations may show a deeper biological response to treatment.

Spleen size and symptom relief

Nearly half of the patients experienced a reduction in spleen size by physical exam. An enlarged spleen is common in MF and can lead to abdominal discomfort. The treatment also reduced the symptoms participants experienced. Over 40% of patients reported at least a 50% improvement in their symptoms. This was measured using a scoring tool called the Myeloproliferative Neoplasm Symptom Assessment Form (MPNSAF-TSS). This tool tracks fatigue, itching, bone pain, and other common MF symptoms.

Tolerability and side effects

Ropeginterferon alfa-2b was generally well-tolerated. The most frequent non-blood-related side effects were mild liver enzyme elevations, fatigue, and hair thinning. Blood-related side effects included mild to moderate anemia (low red blood cells), neutropenia (low white cells), and thrombocytopenia (low platelets). Few participants experienced more serious side effects. Importantly, none of the participants developed acute leukemia or experienced major clotting or bleeding events during the study period.

A potential option for early and low-risk MF

This study suggests that ropeginterferon alfa-2b could offer a safe and effective treatment option for patients with early-stage or low-risk MF who need cytoreduction. It led to improvements in blood counts, symptoms, and underlying disease markers. 

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Source: 

• Ropeginterferon alfa-2b for pre-fibrotic primary myelofibrosis and DIPSS low/intermediate-risk myelofibrosis.