Reuters Health Information
Ruxolitinib Improves Patient-Reported Outcomes in Myelofibrosis
Feb 22, 2013
Survival Improving in Primary Myelofibrosis
Ruxolitinib Approved for Myelofibrosis in European Union
Drug & Reference Information
Agnogenic Myeloid Metaplasia With Myelofibrosis
Acute Myeloid Leukemia, Not Otherwise Categorized
By Will Boggs, MD
NEW YORK (Reuters Health) Feb 22 - The modified Myelofibrosis Symptom Assessment Form (MFSAF) v2.0 is a good tool for monitoring patient-reported symptoms of myelofibrosis, according to a secondary analysis of data from the COMFORT-I trial.
The initial data from the trial, reported last year in the New England Journal of Medicine, showed that compared to a placebo, ruxolitinib reduced spleen size, ameliorated debilitating myelofibrosis-related symptoms, and improved overall survival.
The authors of the new study, published online February 19 in the Journal of Clinical Oncology, evaluated the usefulness of the MFSAF for symptom assessment.
Physical signs (lab value or images) are not the only important end points, according to Dr. Rubin A. Mesa from Mayo Clinic, Scottsdale, Arizona, who led the new study. He told Reuters Health in an email, "In the past, symptomatic status was brushed aside given limited abilities to measure in an accurate way."
Dr. Mesa sees a role for the MFSAF in daily clinical practice: "Very brief, serial assessment of symptomatic improvement with medical therapy can aid the physician in assessing the response of a myelofibrosis patient to therapy. In a time-constrained practice these data brought in with a visit are very enlightening."
Dr. Mesa and colleagues studied 309 patients with intermediate-2 or high-risk myelofibrosis. Exploratory analyses were also conducted on the Total Symptom Score (TSS), European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30), Pat
The affiliation of Dr Mesa is also interesting--Mayo clinic (although Arizona). I'll have to hit the library for the full paper, but the abstract looks very good (maybe a little more weaseling about subsets than I'd like--when a percentage of a subset is reported, I prefer to have the percentage of the full population in parentheses next to it). Whatever it says about treatment of the underlying disease, it certainly demonstrates that patients like the effect of the drug.
This speaks to a distinction I like to bring up: that between treating the patient vs treating the chart. Scientific medicine can easily drift toward the latter. Sometimes dramatic changes in test results seem to have no effect on patient experience (hydroxyurea improves blood counts in many patients with MPDs without improving either quality of life or prognosis), while at other times infeasible-to-quantify effects make a treatment highly desirable (this is an example).