1. ECFC Brussels - June 2015 A nurse specific monitoring for CF patients taking ivacaftor (Kalydeco) A nurse specific monitoring for CF patients taking ivacaftor (Kalydeco TM ) M.Kerbrat 1, R.Fretay 2, D.Chevalier 3, F.You 3, M.Chiffoleau 4, A.Hubault 4, C.Matras 5,I.Berhault 6, MT.Jousseaume 6, MC.L’Abbé 6, C.Pingon 6, MC.Quillevere 7, S.Paul 7, K.Sery 8, M.Idres 9 1 CF reference centre Nantes-Roscoff, France; 2 CF centre Vannes-Lorient, France; 3 CF centre Nantes, France; 4 CF centre Angers, France; 5 CF centre Tours France; 6 CF centre Rennes, France; 7 CF centre Roscoff, France; 8 St Nazaire Hospital, France; 9 St Brieuc Hospital, France Acknowledgments to G.Rault 1 and S.Hugé 2 Email: marythe.kerbrat@perharidy.frmarythe.kerbrat@perharidy.fr ECFC Brussels - June 2015

2. Context ► A French coordinator nurses’ CF network group representing 9 CF centres (average 1000 patients) ► 4 meetings a year to discuss nurses' practices, improve patient support, build and innovate together ► One in 2014, focused on ivacaftor (Kalydeco TM )  25 CF patients, children (>6 years old) and adults with gating mutations, concerned by ivacaftor  Represents a significant breakthrough  Needs some administration conditions and long term compliance

3. ECFC Brussels - June 2015 Objectives ► To evaluate the ivacaftor-taking in daily life:  feeling of efficacy  compliance to ivacaftor  drug administration conditions, side effects ► To highlight the potentials changes with ivacaftor in patients’ life:  quality of life  other treatments ► To improve nurses’ knowledge about ivacaftor and the optimal administration conditions

4. ECFC Brussels - June 2015 Methodology ► 1. Brainstorming: « What do we really know about ivacaftor?” ► 2. Material  Quality of life questionnaires (CFQ-R and Satmed-Q Inserm)  French questionnaire about compliance (« Auto questionnaire ACGT comment dire »)  Booklets about Kalydeco TM ► 3. Creation of a nurse specific monitoring for patients taking ivacaftor and instructions (when, how to use it …) ► 4.Test, feedback and adjustments ► 5.Effective establishing on the network

5. ECFC Brussels - June 2015 The nurse specific monitoring for patients taking ivacaftor 2 questionnaires and Instructions

6. ECFC Brussels - June 2015

9. Results ► 18 completed questionnaires until now:  Feeling of efficacy: ► And 12 feel good with it (12) ► But 8 do not see any difference (Young patients already with few symptoms)  Compliance: ► They rarely forget the intake: 15/rarely and 3/sometimes ► All the patients want to continue the treatment  Side effects: only a few (5)  Changes: ► Some of them change usually treatment: decrease physiotherapy (4), pancreatic enzyme supplements (1), antibiotherapy (2), stop salt (2) and aerosol (1)  Drug administration conditions: ► A patient does not eat in the morning and take on an empty stomach!

10. ECFC Brussels - June 2015 Discussion and conclusion ► Those questionnaires give clues for a long-term monitoring:  Not to trivialize the importance of treatment, to recall the modalities  To exchange and adapt the beside treatment  To evaluate and preserve the long term compliance  To appreciate globally the effects and the impact on patient life ► An approach, for the nurses, of educational tools of compliance, self-efficacy and quality of life ► A question: How asymptomatic children will manage this long- lasting treatment without feeling of improvement? ► Those questionnaires could be improved and used for other specific upcoming treatments