Optimal therapy for polycythemia vera and essential thrombocythemia: Preferred use of interferon therapy based on phase 2 trials.

OBJECTIVES: To determine the value of recombinant interferon-alfa (rIFNα) in the treatment of polycythemia vera (PV) and essential thrombocythemia (ET) based on its biological activities and phase 2 clinical studies, pending completion of phase 3 trials; to determine importance of the Internet in patient decision-making in treatment selection.

RESULTS: The value of rIFNα in PV and ET is based upon its biological effects on PV stem cells and megakaryocyte proliferation. Single-arm trials are useful for life-threatening diseases when there are relatively few patients to evaluate endpoints, such as rIFNα treatment of PV and ET. Proper diagnostic criteria are mandatory; for PV, the current World Health Organization criteria emphasizing increased hemoglobin values exclude approximately one-third of eligible patients. Importance of these data: Single-arm studies in diseases exemplified by rIFNα in PV require updated diagnostic criteria for research and for clinical practice. The influence of the Internet on patient decisions for treatment is noteworthy.

CONCLUSION: The biologic basis for selecting therapy is exemplified by rIFNα in treating PV and ET. Current single-arm studies of rIFNα in PV and ET are relevant and acceptable. The importance of the Internet in patient decision-making is important.