Patients with heavily pretreated advanced cancer or with rare tumors are difficult to treat. Molecular profiling (MP) of tumors to identify biomarkers that predict potential outcomes with individual therapies is an emerging strategy to guide treatment decisions. Patients with rare tumors for which standard-of-care therapy was unavailable or more common tumors for which standard-of-care options had been exhausted underwent MP at a single Australian center. Data regarding treating physicians' choice of therapy, MP results and recommendations, and patient outcomes were collected. Seven patients had received prior standard first-line therapy (PST), 16 had rare tumors, and 31 had been heavily pretreated (HPT; ≥2 prior lines). Most treatments suggested by MP (541/594; 91.1%) were common chemotherapy drugs available in generic formulations. MP-guided therapy recommendations differed from physician's recommendations in 48 patients (88.9%). MP-guided therapy produced clinical benefit (improved QOL and/or performance status, symptoms, bodyweight, or RECIST) in 19/31 (61.3%), 11/16 (68.8%), and 3/7 (42.9%) patients with HPTs, rare tumors, and PSTs, respectively, and had a PFS ratio ≥1.3 in 22/37 evaluable patients (59.5%; 95% confidence interval 44–76%). The null hypothesis that ≤15% of these patients would have a PFS ratio ≥1.3 was rejected (one-sided p < 0.0001). In conclusion, using MP to guide therapy selection is feasible in clinical practice and may improve patient outcomes.
e17586 Background: Advanced anaplastic thyroid cancer is a disease with very few treatment options. Poor response rates have been reported with cisplatin / doxorubicin and modest response noted in a single phase 2 trial with 24 hour paclitaxel infusion. The 50% response rate has not been reproduced by other investigators but raises questions about the possible utility of taxanes. Although paclitaxel is not approved for this indication in Australia, nab-Paclitaxel is available on a compassionate access scheme. We report 6 consecutive cases treated with nab-paclitaxel (as an alternative to paclitaxel infusion) who all exhibited a response to treatment. Methods: 6 consecutive patients with histologically proven advanced anaplastic thyroid cancer were assessed and imaged to document extent of disease. They were all treated with nab-paclitaxel 100mg/M2 given weekly either continuously or for 3 weeks out of every 4. Clinical and radiological response to treatment was documented. Results: The number of cycles given ranged from 3 to 8. Two patients required dose reductions due to neuropathy. Of the 6 patients; 5 had measurable metastatic disease and 5 had measurable local disease. Four patients showed clinical response to treatment with shrinkage of measurable local disease. One patient showed response to treatment with shrinkage of the primary tumour. Three patients with stridor developed considerable relief from this distressing symptom. Three patients with inoperable disease subsequently became operable and were resected to obtain local disease control. One patient with no evaluable disease after resection (Stage 4B) who received adjuvant nab-paclitaxel remains free of recurrence at 4 years. Conclusions: Anaplastic thyroid cancer is generally regarded as malignancy untreatable by chemotherapy. Our pilot series suggests nab-Paclitaxel warrants further evaluation and should be considered as a chemotherapy backline for combination with targeted agents.
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