Comparative Effectiveness Analysis of Treatment Options for Single Brain Metastasis.

BACKGROUND: Brain metastases (BMs) occur in up to 30% of patients with cancer. Treatments include surgery, whole-brain radiotherapy (WBRT), and stereotactic radiosurgery (SRS), alone or in combination. Although guidelines exist, data to inform individualized approaches to therapy remain sparse. We sought to compare semiquantitatively the effectiveness of various modalities in the treatment of single brain metastasis. METHODS: We performed a comparative effectiveness analysis (CEA) that integrated efficacy, cost, and quality of life (QoL) data for alternate BM treatments. Efficacy data were obtained from a comprehensive review of current literature. Cost estimates were based on publicly available data. QoL data included the Karnofsky Performance Status (KPS) and other questionnaires. Six treatment strategies using combinations of surgery, WBRT, and SRS were compared with decision tree software. RESULTS: The clinical efficacy, cost, and QoL effects of each strategy were scored semiquantitatively. We constructed a model to integrate individual preferences regarding the relative importance of efficacy, QoL, and cost to provide personalized rankings of the effectiveness of each strategy. CONCLUSION: The choice of strategy must be individualized for patients with a single BM. Our CEA and decision model combines empirical data with patient priorities to produce a ranking of alternate management strategies.

Comparative effectiveness review of treatment options for pituitary microadenomas in acromegaly.

OBJECT: Acromegaly, a syndrome of excess growth hormone (GH) secretion typically caused by a GH-secreting pituitary adenoma, reduces life expectancy by approximately 10 years when left untreated. Treatment of acromegaly involves combinations of one or more discrete therapeutic modalities to achieve biochemical control. Unfortunately, data capable of informing decisions among alternate management strategies are presently lacking. METHODS: The authors performed a comparative effectiveness research (CER) review integrating efficacy, cost, and quality of life (QOL) analysis for treatment strategies comprising various combinations of surgery, radiotherapy, stereotactic radiosurgery, and pharmacotherapy in patients with acromegaly caused by a pituitary microadenoma. A management decision tree was used to identify 5 treatment strategies, each with up to 4 potential treatment steps. Efficacy was assessed using recent literature reports of biochemical control rates for each modality. Cost estimations were derived from wholesale drug prices and from the Healthcare Cost and Utility Project. Quality of life data were obtained from studies utilizing the Acromegaly Quality of Life Questionnaire. RESULTS: Individual treatment modalities were analyzed and ranked in each of 3 domains: highest rate of success, lowest cost, and highest QOL, and these scores were combined to facilitate comparison of overall effectiveness of each of the management strategies. These aggregate effectiveness scores were used to compare the 5 strategies from the decision tree, and a novel strategy was also proposed. CONCLUSIONS: The choice of management strategy must be individualized for each patient with acromegaly. This CER analysis provides a comprehensive framework to inform clinical decisions among alternate management strategies in patients with GH-secreting pituitary microadenomas.