Flemish network on rare connective tissue diseases (CTD): patient pathways in systemic sclerosis. First steps taken.

Despite the low prevalence of each rare disease, the total burden is high. Patients with rare diseases encounter numerous barriers, including delayed diagnosis and limited access to high-quality treatments. In order to tackle these challenges, the European Commission launched the European Reference Networks (ERNs), cross-border networks of healthcare providers and patients representatives. In parallel, the aims and structure of these ERNs were translated at the federal and regional levels, resulting in the creation of the Flemish Network of Rare Diseases. In line with the mission of the ERNs and to ensure equal access to care, we describe as first patient pathways for systemic sclerosis (SSc), as a pilot model for other rare connective and musculoskeletal diseases. Consensus was reached on following key messages: 1. Patients with SSc should have multidisciplinary clinical and investigational evaluations in a tertiary reference expert centre at baseline, and subsequently every three to 5 years. Intermediately, a yearly clinical evaluation should be provided in the reference centre, whilst SSc technical evaluations are permissionably executed in a centre that follows SSc-specific clinical practice guidelines. In between, monitoring can take place in secondary care units, under the condition that qualitative examinations and care including interactive multidisciplinary consultations can be provided. 2. Patients with early diffuse cutaneous SSc, (progressive) interstitial lung disease and/or pulmonary arterial hypertension should undergo regular evaluations in specialised tertiary care reference institutions. 3. Monitoring of patients with progressive interstitial lung disease and/or pulmonary (arterial) hypertension will be done in agreement with experts of ERN LUNG.

Breast cancer and fat grafting: efficacy, safety and complications-a systematic review.

Autologous fat grafting (AFG) or lipofilling is nowadays a popular technique for breast reconstruction after breast cancer surgery. There is debate regarding the oncological safety and risks of this procedure in breast cancer patients. A systematic review of the literature published between January first 1995 and October first 2016 was conducted regarding the efficacy, safety and complications of this technique in breast cancer patients after their cancer treatment. The databases PubMed, Science Direct and Thomson Reuters Web of Science were used to search for qualified articles. Inclusion criteria were women with a personal history of breast cancer and at least one lipofilling procedure. Only studies containing a minimum of 20 patients were included in this systematic review. The search yielded a total of 23 suitable articles: 18 case series, 4 retrospective cohort studies and one prospective cohort study. The systematic review encompassed a total of 2419 patients. Medical imaging was used in the majority of the studies to assess the follow-up. Mammography was the most popular technique (65.2%), followed by ultrasound (47.8%) and MRI (30.4%). The prevalence of complications was the following: fat necrosis in 5.31%, benign lesions, like cysts or calcifications in 8.78%, infections in 0.96% and local cancer recurrence in 1.69%. AFG or lipofilling appears to be an oncological safe technique with a low morbidity in women with a history of breast cancer. In order to have a better understanding and evidence of the oncological safety a randomised controlled trial is urgently needed. We further recommend that all AFG be registered in the cancer register.

Effect of the cathepsin K inhibitor odanacatib on bone resorption biomarkers in healthy postmenopausal women: two double-blind, randomized, placebo-controlled phase I studies.

Inhibition of cathepsin K (CatK) is a potential new treatment for osteoporosis. In two double-blind, randomized, placebo-controlled phase I studies, postmenopausal female subjects received odanacatib (ODN), an orally active, potent, and selective CatK inhibitor, once weekly for 3 weeks or once daily for 21 days. Bone turnover biomarkers, safety monitoring, and plasma ODN concentrations were assessed. These studies showed ODN to be well tolerated. Pharmacokinetic (PK) analysis revealed a long half-life (t(1/2); 66-93 h) consistent with once-weekly dosing. Pronounced reductions in C-terminal telopeptide of type I collagen (approximately 62%) and N-terminal telopeptide of type I collagen normalized to creatinine (NTx/Cr) (approximately 62%) at trough (C(168 h)) were seen following weekly administration. Robust reductions in CTx (up to 81%) and NTx/Cr (up to 81%) were seen following daily administration. ODN exhibits robust and sustained suppression of bone resorption biomarkers (CTx and NTx/Cr) at weekly doses > or = 25 mg and daily doses > or = 2.5 mg.