Disease Burden and Adherence for Patients With Myelofibrosis

Disease burden is substantial for patients with myelofibrosis, even those with intermediate risk, and a not insubstantial percentage of patients have low or intermediate adherence during treatment, according to 2 abstracts from an Italian clinical trial presented at the 61st American Society of Hematology Annual Meeting & Exposition.

ROMEI (Ruxolitinib Observational study in Myelofibrosis treated patients in Italy) is a prospective observational study on the real-life management of patients with myelofibrosis on ruxolitinib. A total of 215 patients were enrolled, and an evaluation of the 8-item Morisky Medication Adherence Scale (MMAS-8) was the secondary end point.

The first abstract analyzed nonadherence to medication.¹ After patients started on ruxolitinib, they answered the MMAS-8 questionnaire at weeks 4, 8, 12, and 24. Scores lower than 6 indicated low adherence, 6 to 8 indicated medium adherence, and a score of 8 indicated high adherence.

Only questionnaires with all responses provided were analyzed. Of the 215 patients enrolled, 188 were evaluable for the questionnaire, and 163 patients (87%) completed the MMAS-8 at week 4, 156 (83%) at week 8, 146 (78%) at week 12, and 134 patients (71%) at week 24. Only 101 patients (54%) of the 188 completed all 4 questionnaires.

The mean MMAS-8 score was 7.54 at week 4 and 7.67 at week 24. For patients who completed all questionnaires, the total score was stable over the full 24 weeks. The researchers found that 25% to 40% were in the low or intermediate adherence classes over the course of the study.

“Surprisingly, overall, approximately one-third of patients could be exposed to a suboptimal treatment over time and probably clinicians underestimate the number of those [patients] in clinical practice,” the authors wrote.

In the second abstract, the researchers looked at disease manifestations for patients from a range of risk categories.² The primary end points were assessment of the Myeloproliferative Neoplasm 10 (MPN-10) total score and the general health rating on a visual analogue scale (EQ-VAS) assessment of the EuroQol 5 dimensions. Changes in spleen length were a secondary end point.

They analyzed disease burden at the start of ruxolitinib and after 24 weeks of treatment. A total of 183 patients of 215 were evaluable. Of these 183 patients, 96 were male, 34 were of intermediate (INT)-1 risk, 89 of INT-2 risk, and 60 of high risk based on the International Prognostic Scoring System (IPSS).

MPN-10 scores and EQ-VAS values were collected at baseline, as well as weeks 4, 8, 12, and 24. MPN-10 scores improved after starting ruxolitinib, from a mean of 34.86 at baseline to 21.55 at week 24. EQ-VAS values increased from a mean of 59.88 at baseline to 67.36 at week 24. For both, there were no statistically significant differences between IPSS risk categories at baseline. There were still no differences for MPN-10 at week 24, but for EQ-VAS, there were statistically significant differences between INT-1 and high risk (73.70 vs 62.45).

“Our sub analysis suggests that disease burden can be very disabling even in INT-1 pts and comparably as in higher-risk categories,” the authors wrote. “[Although] improvements in symptoms and splenomegaly were comparable across age groups, general health status improvement was significantly better in INT-1 pts.”

References

1. Guglielmelli P, Palandri F, Selleri C, et al. Adherence to treatment in myelofibrosis patients: preliminary results from Italian Romei observational study. Presented at: the 61st American Society of Hematology Annual Meeting & Symposium; December 7-10, 2019; Orlando, FL. Abstract 4179.
2. Palandri F, Breccia M, Selleri C, et al. Impact of disease burden in myelofibrosis patients: a sub analysis from Italian Romei observational study. Presented at the 61st American Society of Hematology Annual Meeting & Symposium; December 7-10, 2019; Orlando, FL. Abstract 4188.