Patient Preferences on Treatment Attributes in MM
Patient preference is an important factor to consider when making treatment decisions. To date, few studies have examined multiple myeloma (MM) patients’ degree of preference for method of administration and for avoiding specific adverse events (AEs).
When designing the study, AEs representative of MM patient experience were selected from clinical trials of lenalidomide, bortezomib, thalidomide, pomalidomide, and carfilzomib. After consultation with MM patients, a total of 12 AEs, including bone pain, febrile neutropenia, hypokalemia, hyponatremia, infection, lymphopenia, neuralgia, neutropenia, peripheral neuropathy, renal adverse reaction, thrombocytopenia, and thromboembolic events, were selected for evaluation. Patients were asked to base their responses on desire to avoid grade 3/4 AEs. Using an online survey format, patients were asked to complete 14 discrete choice cards on which they selected their preferred option between 2 hypothetical treatments with varying combinations of AEs, route of administration, and progression-free survival. Strength of preference was converted into a willingness to trade progression-free survival months to receive preferred choice of treatment. Four hundred patients from 8 countries participated in the survey. The majority (58.7%) of patients were male, with a mean age of 40 years. Of the respondents, 28.8% were on their first treatment, and 70.0% of patients reported having switched treatment.
There was a statistically significant preference for oral versus intravenous administration (P = .020), and there was a numerical but nonsignificant trend toward preference of oral versus subcutaneous administration (P = .067). Strength of preference declined in patients with prior treatments. In addition, patients expressed a significant preference (P <.01) to avoid all presented grade 3/4 AEs, except for nonsymptomatic hematologic AEs (thrombocytopenia, neutropenia, and lymphopenia) for first treatment patients, and neutropenia for patients with prior therapy. The relative importance of grade 3/4 febrile neutropenia and renal adverse reaction increased with prior treatments.
The study yielded interesting findings concerning the preferences of younger, working-age MM patients regarding individual AEs and methods of administration. Patients expressed a greater preference for oral versus infusion therapy; they also expressed a preference to avoid certain symptomatic AEs versus nonsymptomatic hematologic AEs. Researchers concluded that patient preference should be considered when making treatment decisions. Given the median age of the study population, these findings have limited generalizability to the broader population of patients with MM.