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NICE guidance on inhaled mannitol for treatment of cystic fibrosis On Nov 28, 2012, NICE published guidance recommending mannitol as an option for treatment of cystic fibrosis in adults who cannot use dornase alfa (because of ineligibility, intolerance, or inadequate response), whose lung function—as measured by forced expiratory volume in 1 s (FEV1)—is rapidly decreasing (by greater than 2% yearly), and for whom other osmotic drugs are not judged appropriate.1 NICE appraised mannitol under the single technology appraisal process. The appraisal’s remit was to evaluate the clinical and cost effectiveness of inhaled mannitol dry powder for cystic fibrosis in its licensed indication. The manufacturer of mannitol (Pharmaxis, NSW, Australia) submitted clinical evidence and a health economic model,2 which were reviewed by an independent evidence review group.3 An independent appraisal committee met twice to develop guidance; clinicians and patient experts attended the first meeting and Pharmaxis attended both meetings. Two international trials, DPM-CF-301 and DPM-CF-302,4,5 constituted the key clinical evidence; in both studies, patients with cystic fibrosis not using inhaled hypertonic saline were randomly assigned to receive either mannitol 400 mg or 50 mg (assumed to be subtherapeutic), twice daily. Patients in both groups also received best supportive care with or without dornase alfa. DPM-CF-301 included 295 patients (190 adults) from 40 centres. DPM-CF-302 included 305 patients (151 adults) from 53 centres. Patients were stratified into those taking dornase alfa, and those not taking dornase alfa, including a subgroup of people who cannot take dornase alfa because of ineligibility (because of prescribing regulations), intolerance, or inadequate response. www.thelancet.com/respiratory Vol 1 March 2013

The primary outcome in both trials was change of FEV1 from baseline over 26 weeks. The secondary outcomes included changes in predicted percentage FEV1 and protocol-defined pulmonary exacerbations. Both trials measured quality of life with the Cystic Fibrosis Questionnaire,6 DPMCF-302 also used the Health Utility Index 2.7 Pharmaxis presented outcomes from the trials, separately and pooled. The pooled mean change in FEV1 over 26 weeks was 94·1 mL (95% CI 29·7– 158·42) for adults taking dornase alfa and mannitol and 110·3 mL (95% CI not given, p

NICE guidance on inhaled mannitol for treatment of cystic fibrosis.

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