R. Rashid

67 Clinical outcomes and patient satisfaction following initiation of the TOBI Podhaler in CF adults

In CF patients with the F508del mutation, vertebral fractures and subsequent dorsal kyphosis decrease pulmonary function, thus accelerating the course of the disease. We discovered a defective CFTR-mediated Cl− channel activity and deficit of osteoprotegerin production by primary osteoblasts (the cells that form bone) of a 25-yr-old CF male (Gimenez A., 2012). Homozygous F508del-CFTR mice exhibit a severe osteopenic phenotype (Le Henaff C., 2012). Miglustat (N butyldeoxynojyrimicin) was reported to normalize CFTR-dependent chloride transport in nasal mucosa in F508del CF mice. We evaluated the efficacy of oral miglustat treatment in restoring bone mass in CF mice. The bone microarchitecture of CF mice, relative to wild type (WT) littermates was evaluated after an administration of 120mg/kg/day miglustat for 28 days using in vivo micro-CT, bone histomorphometry and dynamic parameters of bone formation. Levels of two serum growth factors, insulin-like growth factor 1 (IGF-1) and 17b-estradiol (E2) were determined. A once-a-day oral treatment with miglustat normalized bone volume and improved bone micro-architecture of the lumbar spine in CF mice after 4 weeks. This increase of bone volume was accompanied to both an increased bone formation and serum E2 level with no changes in IGF-1 level in miglustat-treated F508del mice. This study provides first evidence that oral administration of miglustat increases bone mass and formation in CF mice; these findings support the therapeutic potential of miglustat in patients with CF-related bone disease. Supported by Association Vaincre la Mucoviscidose, France; miglustat kindly provided by Actelion Pharmaceuticals, Switzerland.

39 ‘Real world’ tolerability, ease of use, patient satisfaction and reported adherence in CF adults commencing Colobreathe®

 “It‟s a very good product . I can‟t believe how much of a difference it has made to me in terms of convenience and time.”  “It‟s easy to use. I can take it in my handbag and use it on the move.”  “It saves time and it‟s easier to organise within my treatment plan. I‟m excited about when the manufacturers get the inhaler design right.”  “I tolerated Promixin® better but the ease of doing Colobreathe® outweighs the chest tightness I get with it.”  “I‟ve sometimes breathed in bits of capsule and I‟m concerned that they might be going onto my lungs.”  “I‟m very disappointed with the design of the device and the way the capsules fracture. When this happens there can be a large influx of powder all at once which causes coughing – I then feel I‟m losing the powder that I‟ve breathed in.”  “I‟m worried that I may not always be getting a full dose. Sometimes when the capsules crack, powder is left in the inhaler or it gets stuck behind the pieces of broken capsule and I have to try and shake/open the capsule to free the trapped powder.”  “Powder starts to build up on the inside of the inhaler. This stops the capsules from spinning properly and sometimes I get a „gush‟ of powder and sometimes very little. A supply of two inhalers rather than one a month would be better.”  “Sometimes bits of the capsule get stuck in the inhaler and I have to use a pencil to get them out.”

137 Transcutaneous ear probe oxygenation readings are significantly higher than finger probe oxygenation readings in CF adults

Objectives: Accurate assessment of oxygen saturations (SaO2) is vital in the management of people with CF. Capillary blood gases (CBGs) are relatively painless and correlate well with arterial blood gases (ABGs) when estimating SaO2 in chronic lung disease [1]. Finger probe pulse oximetry (SpO2) is routinely used to estimate SaO2, but over the last 12 months we have started using ear probe pulse oximetry in our large regional CF centre. Our objective was to compare SaO2 by CBG or ABG with finger and ear probe SpO2 in adults with CF and normal controls. Methods:We performed simultaneous CBG or ABG, finger and ear probe SpO2 in adults with CF and normal controls. Finger and ear probe SpO2 were recorded by the same oximeter (Nonin Medical Inc.) using the appropriate sensors. Results: 21 sets of observations (19/21 CBGs) were recorded in 14 patients (6 male, mean age 29yrs, mean FEV1 30% predicted). 3 sets of observations (3/3 CBGs) were performed in 3 normal controls (1 male, mean age 31yrs). In patients ear probe SpO2 was significantly higher than finger probe SpO2 (*p < 0.001) and finger probe SpO2 was significantly lower than SaO2 (^p =0.01).

316* Cardiac autonomie neuropathy (CAN) is related to lung function in adults with cystic fibrosis related diabetes (CFRD)

315* Cardiac autonomic neuropathy (CAN) is more likely to occur in cystic fibrosis (CF) patients with or without diabetes than in patients with type 1 diabetes (T1DM) R. Rashid1, A.A. Tahrani2, M. Piya2, N. Edward1, D. Honeybourne1, M. Newnham3, M.J. Stevens2, J. Whitehouse1. 1Heart of England NHS Trust, Respiratory and CF, Birmingham, United Kingdom; 2Heart of England NHS Trust, Diabetes, Birmingham, United Kingdom; 3Heart of England NHS Trust, Birmingham, United Kingdom