Platinum compounds have activity in triple-negative breast cancer (TNBC) in germline mutation carriers ( carriers). Limited data exist for estrogen receptor (ER)-positive (+) breast cancer among carriers. INFORM is a randomized, multicenter, phase II trial comparing pathologic complete response (pCR) rates (ypT0/is, N0) after neoadjuvant single-agent cisplatin (CDDP) versus doxorubicin-cyclophosphamide (AC) in carriers with stage I-III human epidermal growth factor receptor 2 (HER2)-negative breast cancer. Secondary objectives included residual cancer burden scores (RCB) of 0 or 1 (combined) and toxicity. The goal was to determine whether pCR was ≥ 20% higher with CDDP than AC.

carriers with cT1-3 (≥ 1.5 cm), cN0-3 HER2-negative breast cancer were randomly assigned to preoperative CDDP (75 mg/m every 3 weeks × 4 doses) or AC (doxorubicin 60 mg/m; cyclophosphamide 600 mg/m every 2-3 weeks × 4 doses) followed by surgery. Pathologic responses were confirmed by central review.

A total of 118 patients were randomly assigned; 117 were included in outcome analyses. Mean age was 42 years (range, 24-73 years); 69% were +, 30% were +, and 2% had both mutations. Clinical stage was I for 19%, II for 63%, and III for 18%; 45% had nodal involvement at baseline. Seventy percent had TNBC. Clinical and tumor characteristics were well matched between treatment arms. The pCR rate was 18% with CDDP and 26% with AC, yielding a risk ratio (RR) of 0.70 (90% CI, 0.39 to 1.2). The risk of RCB 0 or 1 (RCB 0/1) was 33% with CDDP and 46% with AC (RR, 0.73; 90% CI, 0.50 to 1.1). Both regimens were generally well tolerated without unexpected toxicities.

pCR or RCB 0/1 is not significantly higher with CDDP than with AC in carriers with stage I-III HER2-negative breast cancer for both TNBC and ER+/HER2-negative disease.