Symptom Burden and Quality of Life With Cytoreductive Therapy in Essential Thrombocythemia and Polycythemia Vera

Treatment of patients with essential thrombocythemia (ET) or polycythemia vera (PV) with pegylated interferon alfa-2a may actually increase the symptom burden in patients with low baseline symptoms.

However, treatment with either pegylated interferon alfa-2a or hydroxyurea was associated with a reduction in symptom burden within 3 to 12 months in those with high baseline symptoms, according to a post-hoc analysis of 2 trials published by the Myeloproliferative Neoplasms Research Consortium 111 and 112 trial teams.

“Results can inform treatment decisions, including treatment timing and goals in managing ET and PV, because measuring symptom burden from the patient perspective is crucial to understanding treatment efficacy and tolerability,” wrote lead author Gina Mazza, PhD, department of quantitative health sciences, Mayo Clinic, Scottsdale, AZ, and co-authors.

MPN-RC 111 was a single-arm, open-label, phase 2 trial at 17 hospitals and cancer centers in Italy and the US, and enrolled 135 patients between February 15, 2012, and December 23, 2015 to evaluate the clinical-hematological response to pegylated interferon alfa-2a in patients who were resistant or intolerant to hydroxyurea.

MPN-RC 112, on the other hand, was a randomized, open-label, phase 3 trial at 25 hospitals and cancer centers in France, Germany, Israel, Italy, the UK, and the US, and enrolled 168 therapy-naive patients with high-risk ET or PV between September 24, 2011, and June 30, 2016, to compare the clinical-hematological response to pegylated interferon alfa-2a versus hydroxyurea.

Patients completed the Myeloproliferative Neoplasm Symptom Assessment Form and the European Organization for the Research and Treatment of Cancer Core Quality of Life Questionnaire for 12 months as secondary endpoints.

At 12 months, a clinically significant improvement in symptom burden was reported by 32% of complete responders (CRs) and 20% of partial responders (PRs) treated with pegylated interferon alfa-2a in MPN-RC 111; 19% of CRs and 18% of PRs treated with pegylated interferon alfa-2a in MPN-112, and 27% of CRs and 22% of PRs treated with hydroxyurea in MPN-112.

Source:

Mazza G, Mead-Harvey C, Mascarenhas J, et al. Symptom burden and quality of life in patients with high-risk essential thrombocythaemia and polycythaemia vera receiving hydroxyurea or pegylated interferon alfa-2a: a post-hoc analysis of the MPN-RC 111 and 112 trials. Lancet Haematol. 2022 Jan;9(1):e38-e48.