Patient-Centered Approach to Benefit-Risk Characterization Using Number Needed to Benefit and Number Needed to Harm: Advanced Non-Small-Cell Lung Cancer.

This work summarizes the benefit and risk of the results of clinical trials submitted to the US Food and Drug Administration of therapies for the treatment of non-small cell lung cancer (NSCLC) using number needed to benefit (NNB) and number needed to harm (NNH) metrics. NNB and NNH metrics have been reported as potentially being more patient centric and more intuitive to medical practitioners than more common metrics, such as the hazard ratio, and valuable to medical practitioners in complementing other metrics, such as the median time to event. This approach involved the characterization of efficacy and safety results in terms of NNB and NNH of 30 clinical trials in advanced NSCLC supporting US Food and Drug Administration approval decisions from 2003 to 2017. We assessed trends of NNB over time of treatment (eg, for programmed death 1 inhibitors) and variation of NNB across subpopulations (eg, characterized by epidermal growth factor receptor mutation, programmed death ligand 1 expression, Eastern Cooperative Oncology Group performance status, age, and extent of disease progression). Furthermore, the evolution of NNB of treatments for advanced NSCLC was charted from 2003 to 2017. Across subpopulations, NNB, on average, was 4 patients for approved targeted therapies in molecularly enriched populations, 11 patients for approved therapies in nonmolecularly enriched populations, and 23 patients for withdrawn or unapproved therapies. Furthermore, the NNB analysis showed variation for attributes of epidermal growth factor receptor mutations, level of programmed death 1 expression, Eastern Cooperative Oncology Group performance status, etc. When considering the best-case subpopulations and available drugs, the NNB frontier reduced from an estimated value of 7.7 in 2003 to an estimated value of 2.5 in 2017 at the estimated median overall survival-equal to 6 months-of an untreated patient.

Use of metal proximal radial endoprostheses for treatment of non-traumatic disorders: a case series.

We have reviewed five adult patients treated with endoprosthetic reconstruction of the proximal radius following resection of non-traumatic lesions. The patients had a mean age of 33.4 years (20 to 60) at the time of surgery and the mean follow-up was 7.6 years (0.8 to 16). Following surgery, all elbows were clinically stable and there was 100% survivorship of the prosthesis. Evaluation of function was assessed clinically and by the Mayo Elbow Performance Score, achieving a mean of 86% (70 to 100). Results at medium-term follow-up are encouraging with regards to elbow stability, implant survivorship and functional outcome.

Myolipoma affecting the erector spinae: a case report in a child.

Myolipoma is a rare, benign, lipomatous tumour which most commonly occurs in the retroperitoneum, pelvis, and abdomen. A 4-year-old boy presented with a painless enlarging mass in the left paraspinal region. Magnetic Resonance Imaging (MRI) revealed a soft tissue lesion with high fat content and areas of calcification. Excision and histopathological analysis revealed a tumour composed of lobules of mature adipose tissue and broad septa of well-differentiated smooth muscle tissue. The smooth muscle nature of the nonfatty component was demonstrated by a diffuse and strong immunoreactivity for smooth muscle actin and desmin. The mass was reported as a myolipoma. The patient made an unremarkable recovery from surgery and remains healthy with no signs of recurrence at seven years. This paper represents the youngest patient diagnosed with this rare soft tissue tumour which is normally confined to the adult population. A newly reported site of the tumour is also highlighted.