Question

What are the evidence-based second-line therapies for PV patients?

Hint:

Ruxolitinib is a first-line second-line therapy for PV patients who are resistant to or intolerant of hydroxyurea. Busulfan and pomalidomide are also options for refractory disease.

Answer 1

Step 1: Second-Line Therapies in PV.
Second-line therapies for polycythemia vera (PV) are considered when patients are refractory to first-line treatments or cannot tolerate them. These therapies are often used for patients with high-risk features or inadequate response to standard treatments.

Step 2: Available Second-Line Therapies.
1. Ruxolitinib: Ruxolitinib is a JAK1/2 inhibitor that has shown efficacy in reducing symptoms, spleen size, and improving hematocrit control in patients with PV, especially those with resistance or intolerance to hydroxyurea. It is now considered an important option for PV patients with resistant or refractory disease.
2. Busulfan: Busulfan is an alkylating agent used in patients who cannot tolerate hydroxyurea or ruxolitinib. It can be effective in controlling PV but carries a risk of myelosuppression and long-term complications such as secondary malignancies.
3. Pomalidomide: Pomalidomide, an IMiD, is another treatment option for patients with relapsed or refractory PV. It is used in selected cases, particularly when other therapies are ineffective.

Step 3: Considerations for Treatment Choice.
The choice of second-line therapy depends on patient factors, such as age, comorbidities, and previous treatment history. Ruxolitinib is often preferred for patients with splenomegaly and those who have failed other therapies.