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BackgroundPanitumumab is a fully human monoclonal antibody Ig G2 whose target is the epidermal growth factor receptor (EGFR). It is indicated as monotherapy to treat patients with metastatic colorectal cancer (mCRC) that show EGFR with wild-type KRAS, after failure of chemotherapy regimens containing fluoropyrimidine, oxaliplatin and/or irinotecan.PurposeThe purpose of this study was to evaluate the effectiveness and safety of panitumumab monotherapy in patients with mCRC.Material and methodsThis was a retrospective observational study of patients with mCRC treated with panitumumab as monotherapy from June 2008 to September 2015. Demographic, clinical and pharmacotherapeutic information was collected from the computerised medical records. The main effectiveness variables were: type of response to treatment (following RECIST criteria), progression free survival (PFS) and overall survival (OS). Frequency of adverse effects and severity (according to CTCAE V.4.0) established the safety profile of the treatment.Results30 patients were included: 73% men (n=22), average age 65.4 years (SD=10.7), 56.6% (n=17) ECOG PS 2 at the beginning of treatment and 46.7% (n=14) stage IV diagnosed. Panitumumab was used as a second-(n=10), third- (n=11) and fourth-line (n=9) treatment. Median number of cycles was 6 (IQR 4–10) and the average treatment period was 3.9 months (SD=2.6). Objective response rate was 10% (n=3), all being partial responses. 10% (n=3) showed stabilisation of disease and 63.3% (n=19) progression. In 5 patients the response was not evaluable (2 treatment cycles until death). The SLP median was 3 months (95% CI 1.7–4.2) and the SG median was 8 months (95% CI 3.6–12.3). Dermatologic toxicities occurred in 70% (n=21) of patients, and were severe (grade 3 and higher) in 15% of patients receiving panitumumab monotherapy. It was necessary to reduce the drug dose in 3 patients (due to dermal toxicity), with an average reduction of 26% (SD=11.5, range 20–40).ConclusionPanitumumab as monotherapy showed adequate effectiveness (SLP median 3 months and SG median 8 months) in patients with mCRC: pretreated, KRAS wild-type and poor performance status. It should be noted that dermal toxicity was observed in 70% of patients, characteristic of the EGFR inhibitor family. Future guidelines for mCRC treatment will have to establish the optimum sequence of use of the available therapies with the aim of achieving the greatest clinical benefit in patients.References and/or acknowledgementsI want to thank Beatriz Sánchez Castellanos for her support and time.No conflict of interest

BackgroundTuberous sclerosis complex (TSC) is an autosomal dominant disease with variable expressiveness and multisystem involvement. Everolimus, an mTOR inhibitor, is indicated for the treatment of kidney angiomyolipoma and subependymal giant cell astrocytoma (SEGA) associated with TSC.PurposeThe objectives of the study were to evaluate the effectiveness and safety of treatment in TSC.Material and methodsRetrospective observational study of patients treated with everolimus from July 2013 to April 2014.The collected variables were: sex, age, affected organs, dose, duration and reason for treatment.The effectiveness variables were, in each case: reduction in size of SEGA equal to or greater than 30%, reduction in size of the kidney angiomyolipomas in at least 25%, improvement of dyspnoea and/or absence of lung acute episodes.The safety profile of the drug was determined by the number of adverse reactions.Results4 patients were included:Patient No 1: female, 32 years old. Skin and neurological involvement. Everolimus was initiated at 7.5 mg four times daily for SEGA. No response to treatment was noted. Skin lesions disappeared and absence of epileptic seizures was observed. At the beginning of the treatment, the patient suffered grade 1 stomatitis.Patient No 2: female, 38 years old. Cerebral, skin, bone, heart and pulmonary involvement. Everolimus was initiated at 7.5 mg four times daily for pulmonary lymphangioleiomyomatosis. Response to treatment was achieved. There was also an improvement in osteomas and skin lesions. Grade 2 non-infectious pneumonitis was reported; this adverse event was resolved after dose reduction of everolimul to 5 mg four times daily.Patient No 3: male, 21 years old. Skin, ocular and neurological involvement. The treatment was initiated at 7.5 mg four times daily for SEGA. Reduction in size of SEGA of 30% was observed (response to treatment). At the beginning of the treatment the patient presented stomatitis and mild microalbuminuria (169 mg/g), which improved with enalapril treatment (63 mg/g).Patient No 4: female,15 years old. Skin, heart, kidney and brain involvement. Everolimus treatment was initiated at 10 mg four times daily due to kidney angiomyolipomas and SEGA. Neither response nor side effects were observed.Currently, all patients continue with the treatment; follow-up (median, range) is 17 (12–27) months.ConclusionEverolimus is the only well tolerated treatment for TSC, but its effectiveness is variable. In the cases where no response was observed, the lesions were stabilised.The number of patients is limited due to the low prevalence of this disease and to the restrictive criteria for initiating everolimus treatment.More studies are needed to determine the optimal dose and duration of treatment.No conflict of interest.