Despite the paucity of evidence supporting chemotherapy in the treatment of node-negative, HER2-positive breast cancer measuring <2 cm, use of trastuzumab-based chemotherapy has increased over the past decade. Therefore, we used the National Cancer Database to evaluate the use and impact of chemotherapy on survival in this population. We identified female patients aged 18 to 70 years with node-negative, HER2-positive breast cancer measuring <2 cm. A propensity-matched cohort model was used to control for risk factors known to influence survival. Primary end points assessed were receipt of chemotherapy and overall survival (OS). In our propensity-matched cohort model (n=8,222), adjuvant chemotherapy (ACT) was associated with a lower 5-year OS rate in T1mi breast cancer (n=626; 89.1% [95% CI, 81.8%-93.5%] vs 99.1% [96.6%-99.8%]), no significant effect in T1a disease (n=2,901; 95.4% [93.2%-96.9%] vs 96.9% [94.1%-98.3%]), and improved 5-year OS in T1b (n=2,340; 97.1% [95.1%-98.4%] vs 92.3% [88.5%-94.9%]) and T1c tumors (n=2,355; 95.9% [93.5%-97.5%] vs 91.5% [88.4%-93.9%]). In the entire cohort of 21,148 patients who met the inclusion criteria, ACT was associated with lower 5-year OS in T1mi (89.6% [83.7%-93.4%] vs 98.1% [96.6%-98.9%]) and T1a tumors (94.9% [92.9%-96.3%] vs 96.5% [94.6%-97.7%]), and improved 5-year OS in T1b (96.8% [95.6%-97.7%] vs 92.3% [88.7%-94.8%]) and T1c tumors (95.8% [94.9%-96.5%] vs 91.6% [88.5%-93.9%]). Increased use of ACT was observed over the study period. From 2010 to 2013, annual treatment rates were 71.5%, 72.4%, 73.3%, and 74.4%, respectively (trend test, <.0001). Our data support the use of ACT for HER2-positive, node-negative T1b and T1c breast cancer, whereas no benefit was observed for ACT in T1mi and T1a HER2-positive, node-negative breast cancer. Although use of ACT is increasing in node-negative, HER2-positive breast cancer <2 cm, our findings caution against its use in the smallest of these tumors (T1mi and T1a) due to lack of survival benefit.
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