ABSTRACT
Polycythemia vera (PV) is a myeloproliferative neoplasm characterized by unregulated red blood cell production resulting in elevated hemoglobin and/or hematocrit levels. Patients often have symptoms such as fatigue, pruritus, and painful splenomegaly, but are also at risk of thrombosis, both venous and arterial. Ruxolitinib, a selective Janus kinase inhibitor, is approved by the US Food and Drug Administration as second-line cytoreductive treatment after intolerance or inadequate response to hydroxyurea. Although ruxolitinib has been widely used in this setting, limited data exist in the literature on ruxolitinib treatment patterns and outcomes among patients with PV in routine clinical practice. We report a retrospective, observational, cohort study of patients treated for PV with ruxolitinib across three US centers (academic and regional practice) from December 2014-December 2019. The study included 69 patients, with a median follow-up duration of 3.7 years (95% CI, 2.9-4.4). Our data demonstrate very high rates of hematocrit control (88% of patients by three months and 89% by six months); few patients required dose adjustments or suspension. No arterial thromboses were observed; however, the follow-up duration does not allow for the generation of meaningful conclusions from this. Three patients had thrombotic events; one was in the setting of a second malignancy, one post-operative, and a third related to prolonged immobility. We also found that 28% of patients initiated ruxolitinib as a result of poorly controlled platelet counts, second only to hydroxyurea intolerance (46%) as a reason to start therapy. In clinical practice, ruxolitinib continues to be effective in controlling hematocrit levels after three and six months of treatment in patients and is associated with low thrombotic risk.
Subject(s)
Nitriles , Polycythemia Vera , Pyrazoles , Pyrimidines , Thrombosis , Humans , Pyrazoles/therapeutic use , Pyrazoles/adverse effects , Polycythemia Vera/drug therapy , Polycythemia Vera/complications , Polycythemia Vera/blood , Pyrimidines/therapeutic use , Female , Male , Hematocrit , Retrospective Studies , Aged , Middle Aged , Thrombosis/etiology , Thrombosis/prevention & control , Aged, 80 and over , Adult , Follow-Up StudiesABSTRACT
PURPOSE: Fiscal distress or "financial toxicity," in which patients experience challenges in paying for treatment, are becoming dominant problems for patients with cancer because of burgeoning health care costs and strategies implemented by health insurance payers to reduce their level of expenditure. We report the structure and function of the first Financial Toxicity Tumor Board (FTTB). Modeled on the concept of a conventional multidisciplinary tumor board, FTTB functions as a multidisciplinary conference providing broad problem-solving approaches to financial toxicity. METHODS: The FTTB, with participation from physicians, nurses, financial counselors, nurse navigators, social workers, and administrators, meets monthly and is focused on financial toxicity and financial worry experienced by patients with cancer. It is linked to a Patient Assistance Program for oncologic pharmaceutical agents as this domain constitutes a critical area of financial toxicity for many patients. RESULTS: In the first years of function, more than $55-$60 million of personal expenditure has been avoided for 1,749 and 1,819 patients, respectively, as well as more than $1.3 million copay assistance provided for financially challenged patients. Problems addressed have included payer impediments, underinsurance, complexities of certification, coding or billing issues, and inadequate internal standard operating procedures. CONCLUSION: A focus on proactive management of financial toxicity through the function of multidisciplinary FTTBs substantially ameliorates this burgeoning international problem. This concept is presented early as it may be leveraged readily in other centers.