The use of lumacaftor/ivacaftor to treat acute deterioration in paediatric cystic fibrosis.

Lumacaftor/ivacaftor is a precision medicine targeting the defective cystic fibrosis transmembrane regulator (CFTR) protein in cystic fibrosis (CF) patients homozygous for Phe508del genotype. Whilst there is evidence for efficacy in children aged 6-11 years who are stable with good lung function, there are little data about the use of this medication for children with acute deterioration in this age group. We describe the use of this drug to treat a child with an unusually severe exacerbation of CF lung disease and review the potential of lumacaftor/ivacaftor as a rescue therapy in the paediatric CF population.

Results in the management of malignant submandibular tumours and guidelines for elective neck treatment.

BACKGROUND: Malignant submandibular gland tumours are rare tumours treated primarily with surgery. PURPOSE: This retrospective study was done to determine the effectiveness of adjuvant radiation therapy (RT), to document the patterns of failure, and to define the prognostic variables for treatment outcome. MATERIALS AND METHODS: Between 1960 and 2002, 74 patients diagnosed as having malignant submandibular gland tumours were treated with radical surgery. The surgery included neck dissection in 37 patients and adjuvant RT in 50 patients with high-risk features. RESULTS: Forty-one (54%) patients experienced disease relapse. The local recurrence rates were 35% in those treated only with surgery and 21% in patients who underwent postoperative RT (p  =  .20). The risk of subclinical nodal metastases in the clinical N0 neck was 21%. CONCLUSIONS: The American Joint Committee on Cancer stage approached statistical significance for cause-specific survival. Although not statistically significant, RT probably reduced local recurrence. All patients with malignant submandibular tumours except T1 need elective neck treatment.

Brachytherapy with permanent gold grain seeds for squamous cell carcinoma of the lip.

PURPOSE: To describe the use of radioactive gold grain implantation for squamous cell carcinoma of the lip. METHODS: Retrospective review of 51 patients treated with permanent gold ((198)Au) grain implant brachytherapy. The seed arrangement delivered a dose of 5500 cGy at 0.5 cm from a single plane. Primary endpoints were local recurrence and cosmetic outcome. RESULTS: Median follow-up was 27 months. Median age was 69 years. The majority (90%) were T1 lesions. None of the patients had evidence of regional lymph node or distant metastasis. Twelve patients had recurrent disease with prior surgery and five patients had previous head and neck radiation. Local control was achieved in 49 patients. Good cosmesis was achieved in 48 patients. Two-year actuarial estimates for local failure-free survival, disease-free survival and overall survival were 97.9%, 94.1% and 87.9%, respectively; no deaths were attributable to lip cancer. CONCLUSIONS: Gold grain interstitial low-dose rate brachytherapy provides excellent local control and cosmesis in patients with squamous cell carcinoma of the lip. This technique provides an excellent option for patients that are elder or live remotely. It is particularly useful for lesions that are small, in previously radiated areas, or treated with prior surgery.