AbstractBackgroundPediatric patients with relapsed/refractory sarcomas have poor outcomes and need novel therapies that provide disease control while maintaining an acceptable quality of life. The activity and toxicity of gemcitabine and nab‐paclitaxel in combination has not been reported in pediatrics.ProcedureWe reviewed the records of fifteen relapsed/refractory patients and one treatment‐naïve patient who received gemcitabine/nab‐paclitaxel at our institution.ResultsSixteen patients (median age 13.5 years, range 3–19 years) received 53 cycles of gemcitabine/nab‐paclitaxel. Twenty‐nine cycles (55%) resulted in ≥Grade 3 toxicity, with nonhematologic Grade ≥3 toxicities occurring in only eight of 53 cycles (15%). Patients received red blood cell and platelet transfusions in 23% and 4% of cycles, respectively. Grade ≥3 infectious toxicities occurred in 4% of cycles. Of 14 patients with measurable disease, there were no complete responses (CR), one partial response (PR; 7%), and six patients (43%) with stable disease (SD; median SD: 4.5 months, range: 2–19 months). In total, 31% of the patients derived clinical benefit (CR + PR + SD ≥ 4 months). Median time to progression was 72 days with a 4‐month progression‐free survival of 31% ± 12% and 1‐year overall survival of 19% ± 10%. With a median follow‐up for all 16 patients of 21 months from the first treatment with gemcitabine/nab‐paclitaxel, one (6%) remains alive with disease.ConclusionsGemcitabine/nab‐paclitaxel is a relatively safe regimen with mainly hematologic toxicities. It offers a well‐tolerated, palliative option providing clinical benefit in a subset of patients. A phase I trial of this combination is underway.