The Impact of Inoperable Advanced Basal Cell Carcinoma: the Economic, Physical, and Psychological Burden of the Disease
October 2013 | Volume 12 | Issue 10 | Supplement | s151 | Copyright © 2013
Arielle W. Haves BA, Panta Rouhani Schaffer MD PhD MPH, and John A. Carucci MD PhD
Ronald O. Perelman Department of Dermatology, New York University, New York, NY
The development of vismodegib and its recent approval by the United States Food and Drug Administration for use in patients with locally advanced or metastatic basal cell carcinoma (BCC) carries with it a renewed sense of optimism. Once BCC has progressed to an advanced, or so-called inoperable stage, there has been a paucity of effective therapies, making the new small molecule inhibitors targeting the hedgehog pathway particularly hopeful prospects. In order to better understand the utility of these new treatments, it is important to assess the existing economic, physical, and psychological burden of advanced BCC. This review aims to recognize the impact of inoperable and metastatic BCC, as well as to better characterize the various types of advanced BCC. The use of vismodegib as a prophylactic treatment in patients with basal cell nevus syndrome is also addressed, including possible adverse events, tumor resistance, and new onset malignancies.
J Drugs Dermatol. 2013;12(suppl 10):s151-s153.
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While the overwhelming majority of basal cell carcinoma (BCC) is effectively managed with surgery, causing minimal distress to the patient, individuals whose BCC has metastasized or progressed to an inoperable state are severely affected by their disease.1 The physical disfigurement from advanced lesions as well as concern about life expectancy affect both quality of life (QOL) and the psychological state of the patient.2 In interviews with clinicians, Shingler et al found that the most common symptom of patients with advanced BCC was embarrassment from either the dressings or the lesions themselves. Metastatic lesions may weep or bleed, have the potential to become infected, and consequently can become malodorous.2 As a result, patients with advanced BCC may isolate themselves from family, friends, and the workplace, despite the fact that they may be physically able to carry on with the normal activities of daily life. Anxiety and depression can also often accompany this social isolation.2
Because BCC metastasis is extremely rare, with a reported incidence of 0.0028% to 0.5%, it is difficult to quantify the economic, physical, and psychological impact of the disease.3,4 There has been very little in the medical literature that attempts to investigate how and to what extent the QOL is affected in patients with advanced BCC, as case reports only offer qualitative information regarding the burden of the disease. In an attempt to capture social utility values associated with different levels of advanced BCC, Shingler et al employed a newer methodology known as the time trade-off (TTO) measurement.2 A representative sample of the general public in the United Kingdom was asked to choose between living in a particular health state with advanced BCC for 10 years vs living in a state of full health for 10-x years.2 The TTO method was used to calculate utility values based upon the responses to these scenarios.2 The health states of advanced BCC used in the valuation exercise included the following: complete response (CR), post-surgical state, partial response (PR) with small growth, PR with large growth, stable disease (SD) with small growth, SD with multiple growths (at 2 cm), SD with large growth, progressed disease (PD) with small growth, and PD with large growth.2 Small growth was defined as 2 cm and large growth as 6 cm. While the study was limited in its ability to capture all possible presentations of advanced BCC, several important findings did emerge.
Not unexpectedly, the highest mean utility value, or amount of time participants were willing to trade for a full state of health, was for the complete response state (94%). The lowest utility value was progressed disease with large growth (67%).2 The size and number of lesions was also found to be an important influence on QOL, and those states were accordingly valued. The most interesting finding of all was that the post-surgical state was valued second to last at 74%.2 The post-surgical state was found to have even more impact on QOL than progressed disease with small growth, suggesting that the general public perceives the impact of disfigurement from extensive surgery for advanced BCC just as debilitating as the experience of progressed disease. From these data, Shingler et al concluded that patients with larger lesions as well as those with numerous lesions would benefit from non-surgical intervention.2 Additionally, treatment efforts to reduce the size and number of lesions were also highly valued by patients.
In addition to the physical and psychological impact of advanced BCC, the financial burden of non-melanoma skin cancer