The Myelofibrosis Symptom Assessment Form (MFSAF): An evidence-based brief inventory to measure quality of life and symptomatic response to treatment in myelofibrosis

Abstract

Quality of life (QoL) in patients with myelofibrosis (MF) is severely compromised by severe constitutional symptoms (i.e. fatigue, night sweats, fever, weight loss), pruritus, and symptoms from frequently massive hepatosplenomegaly. Given that no current instrument of patient reported outcomes (PRO) exists that covers the unique spectrum of symptomatology seen in MF patients, we sought to develop a new PRO instrument for MF patients for use in therapeutic clinical trials. Utilizing data from an international internet based survey of 458 patients with MF we created a 20 item instrument (MFSAF: Myelofibrosis Symptom Assessment Form) which measures the symptoms reported by >10% of MF patients, and includes a measure of QoL. We subsequently validated the MFSAF in a prospective trial of MF patients involving patient and provider feedback, as well as comparison to other validated instruments used in cancer patients. The MFSAF results were highly correlated with other instruments, judged comprehensive and understandable by patients, and should be considered for evaluation of MF symptoms in therapeutic trials.

Associations between gender, disease features and symptom burden in patients with myeloproliferative neoplasms: an analysis by the MPN QOL International Working Group

“…Objective examination of symptom heterogeneity has now emerged as a possibility following the development of MPN-specific PRO tools (MF-SAF, MPN-SAF and MPN-10), enhanced precision of risk scoring algorithms and advances in genomic sequencing. 5,11,12 Applying many of these novel instruments, this study represents the first large-scale investigation into the correlates between gender, clinical features and patients' symptoms.…”

Results of a phase 2 study of pacritinib (SB1518), a JAK2/JAK2(V617F) inhibitor, in patients with myelofibrosis

“…MRI for measurement of spleen volume was repeated on day 1 of cycle 4 and every third cycle through cycle 25. The Chronic Idiopathic Myelofibrosis Quality of Life and Symptom Assessment Form (MF-SAF) 13 was completed on day 1 of cycles 1, 2, and 4 and every third cycle through cycle 25. Bone marrow biopsies were required at baseline and to confirm partial responses and disease progression at cycles 6 and 12.…”

“…Other end points included the proportion of patients with a $50% reduction in spleen size by physical examination and the proportion with a $50% reduction in symptom score as assessed by the MF-SAF 13 up to week 24 compared with baseline. Symptom score was computed as the sum of worst fatigue, early satiety, abdominal pain or discomfort, night sweats, itching (pruritus), and bone pain on the MF-SAF.…”

“…Symptom score was computed as the sum of worst fatigue, early satiety, abdominal pain or discomfort, night sweats, itching (pruritus), and bone pain on the MF-SAF. 13 Spleen volume, spleen size, and symptom reductions were also assessed through end of treatment. Duration of spleen response and evaluation of the safety and tolerability of pacritinib were additional secondary end points.…”

Allo-SCT for myelofibrosis: reversing the chronic phase in the JAK inhibitor era?

“…11,12 In an effort to assess the role of patient-reported outcomes in assessing MF patients' candidacy and post-transplant progress, SYMPTOMS, a prospective questionnaire-based study was recently commenced. [100][101][102][103] The study evaluates the quality of life of patients undergoing allo-SCT, using MPN-SAF TSS, the FACT-BMT, Global Impression of Change and a financial questionnaire at baseline, day +30, day +100 and day +365.…”