Symptom burden and splenomegaly in patients with myelofibrosis in the United States: a retrospective medical record review

Abstract

Myelofibrosis (MF) is a clonal hematopoietic malignancy characterized by constitutional and localized symptoms, progressive splenomegaly, bone marrow fibrosis, and cytopenias. Although MF is well studied, few studies exist regarding its symptomatic burden in routine clinical practice. This study aimed to characterize symptoms and other clinical features of MF among patients in the United States. We conducted a retrospective medical record review of adult patients with an MF diagnosis between 1 January 2005 and 31 March 2010, stratified by the presence of palpable splenomegaly. Eligible patients had 12 months or more of follow-up after diagnosis (or after detection of splenomegaly, if present) unless death occurred. Demographic and clinical characteristics, MF-related symptoms, and treatments were reported by treating physicians. We report on 180 MF patients: 102 with splenomegaly, 78 without. Median age was 66 years, 63% were male, and 82% had intermediate-2 or high-risk MF (International Prognostic Scoring System). Fatigue was reported by ∼85% of patients; weight loss, night sweats, and fever (any grade) were each reported by 50% or more of patients. Generalized abdominal pain, left subcostal pain, and early satiety occurred more frequently among patients with splenomegaly. Multiple symptoms were reported by 95% of patients. Common comorbidities were hypertension, diabetes, and chronic pulmonary disease. Symptoms are common in MF patients, regardless of the presence of palpable splenomegaly. Careful assessment of symptom burden is an important aspect of the clinical evaluation of patients with MF.

Myeloproliferative neoplasm patient symptom burden and quality of life: Evidence of significant impairment compared to controls

“…MPN patients have been shown to have levels of fatigue far in excess of that of published norms (Brief fatigue index score of 4.9 vs. 2.2 out of 10, respectively) [4] but to date no other studies have compared the broad spectrum of MPN symptoms, evaluated by the MPN-SAF, with rates in the general population. Fatigue was the most commonly reported symptom among MPN-UK patients consistent with other reports in the literature [4,5,10,12,15,16]. Fatigue is a common symptom reported in clinical practice with 58% of patients in primary care reporting chronic fatigue [17].…”

“…Early satiety is more common in patients with splenomegaly [12], which can be present at MPN diagnosis particularly in patients with PMF. A higher proportion of PMF patients were included in the Mayo Clinic survey (20.4%) compared to the MOSAICC study (13.2%) which may explain the observed difference.…”

Primary analysis of a phase II open-label trial of INCB039110, a selective JAK1 inhibitor, in patients with myelofibrosis

“…3 Although the symptoms of myelofibrosis vary among patients, for many the overall symptom burden is substantial and often has a debilitating effect on quality of life. [4][5][6] Although most patients with myelofibrosis have primary disease, some develop myelofibrosis secondary to other myeloproliferative neoplasms, including poly-C ombined Janus kinase 1 (JAK1) and JAK2 inhibition therapy effectively reduces splenomegaly and symptom burden related to myelofibrosis but is associated with dose-dependent anemia and thrombocytopenia. In this open-label phase II study, we evaluated the efficacy and safety of three dose levels of INCB039110, a potent and selective oral JAK1 inhibitor, in patients with intermediate-or high-risk myelofibrosis and a platelet count ≥50×10 9 /L.…”

Comprehensively understanding fatigue in patients with myeloproliferative neoplasms

“…Previous studies of MPN-related fatigue indicate that it occurs in 81% 2 to 95% 3 of patients with MPNs and is the predominant symptom regardless of MPN subtype. [2][3][4][5][6][7] This fatigue reduces both physical and social functioning and substantially impairs quality of life. 2 Fatigue is a particular challenge for clinicians and patients due to its multifactorial nature.…”