1. SCIg vs IVIg: Let’s Give Patients the Choice! Marie-Claude Levasseur inf. B.Sc, D.E.S.S Bioethic Immunology-Rhumatology ESID 30 octobre 2014

2. Presentation of the context of the study Detailed presentation of the research Analyse of the result and interpretation Plans for the future Plan Marie-Claude Levasseur, ESID 2014

3. CHU Sainte Justine’s situation SCIG home therapy programm since 2007 Approximately 185 patients on SCIG Consultant for SCIG teaching programm with adults from other health care centers Marie-Claude Levasseur, ESID 2014

4. IVIg and SCIg are equally efficient in patients with PID (1-5) Use is often base on physician’s and nurse’s opinion on the “idealness” of the candidate. Marie-Claude Levasseur, ESID 2014

5. The “ideal” patient - Accept his diagnosis - Active in his treatment - Responsable - Support - Honnest - Good comprenhension capacity - Always on time - Know exactly what he wants - Looks good - Looks “intelligent” - Smells good - E.t.c Criteria based on “opinion” that sometimes do not match between the perception of the doctor and the nurse! Marie-Claude Levasseur, ESID 2014

6. We realize that this perception was changing the way medical team explain the two treatment and was probably influencing the “choice” of the patient. Not neutral! Marie-Claude Levasseur, ESID 2014

7. Retrospective study Tertiary center with pediatric cohort 143 patients with PID on Ig remplacement Present our experience regarding patient’s behavior when given choice of hospital-based IVIg versus home-based SCIg. Marie-Claude Levasseur, ESID 2014

8. All patients, regardless of the physician and nurse’s impression of the “idealness”of the candidates were offered the choice between hospital-based IVIg and home-based SCIg. Switch cohort Patients already on IVIg when the choice of route of administration was given. New cohort Patients diagnosed after, given the choice at the beginning of Ig replacement. SCIg availale in Quebec since 2007 Marie-Claude Levasseur, ESID 2014

9. 1- Physician explained both treatment options in general terms. 2- Clinical nurse provided technical explanation on the methods with written information describing modalities and side effects. 2 Steps: Marie-Claude Levasseur, ESID 2014

10. Clearly indicated that both routes were equally effective Patient had the choice to change their mind at a later point in time if desired and as many times has desired Marie-Claude Levasseur, ESID 2014

11. Options: 400mg/kg/months IVIG Every 4 weeks in our day care center 4-6 hours infusion No fees Marie-Claude Levasseur, ESID 2014

12. Options: 400mg/kg/months SCIg 3 weeks of 2h teaching sessions 15 min-60 min infusion once a week No fees Pump provide by the company 1:1 IV dose Begin 1-2 weeks after IVIG Marie-Claude Levasseur, ESID 2014

13. Demographic characteristics of patients in each cohorts

14. Result Analysis of the patient`s choice “Switch cohort” “De Novo cohort”

15. With same informations half choose SCIg, half IVIg, for first infusion. If they choose IVIg hospital base: want to begin with support afraid to make mistakes want the child to “get use to it”... are not ready to prick still on “shock "of the diagnostic Result Marie-Claude Levasseur, ESID 2014

16. Tolerability SCIg No systemic reaction N=5 Sites reaction (resolved by 3 months of treatment). N=2 Developed hypersensitive nodule at injection sites (resolved by rotating the sites) Steady-state level of IgG 900mg/dl (range 530-1470) IVIg No anaphylaxis Sides effects similar than in the literature Steady-state level of IgG 920mg/dl (range 600-1250) Marie-Claude Levasseur, ESID 2014

17. Together, only a total of 13 patients switched from SCIg to IVIg Reasons noted: Change in family situation (parental medical difficulties) Pain associated with frequent injection Compliance issues Result Marie-Claude Levasseur, ESID 2014

18. Definition of compliance: “the extend to which a person’s behaviour coincides with medical advice”. North America> 50% of patient with chronic disease don’t respect their medical prescription of treatment >80% search for health information on the web With SCIg therapy, how can we measure compliance/adherence? Log sheet verification? Patient questionnaire? IgG monitoring? Let the patient decide what’s best for him at this point in his life Analysis of compliance Marie-Claude Levasseur, ESID 2014

19. “Patient partner” Marie-Claude Levasseur, ESID 2014

20. Analysis of compliance SCIg Poor compliance was noted in 3 pts (6%) in the “switch cohort” and 7 pts (9%) in the new cohort monoparental families and poor supervision in pre-teenagers and teenagers attention-deficit disorder in parents or child parental stress regarding the SCIg modality We proposed to switch back to IVIg. Two of these returned to SCIg treatment 2 and 23 months later without any relapse of poor compliance. Marie-Claude Levasseur, ESID 2014

21. Analysis of patient’s behavior when the choice was offered Stress in relation to the need to make a choice Seems to be more important with the patient from the new cohort, (30%) mention “mild stress”. Despite this stress, patients mentioned that they were reassured by being offered the possibility of changing treatment modality as they wanted. Marie-Claude Levasseur, ESID 2014

22. Discussion 5/6 patient showed a perfect compliance Demonstrate that the medical “a priori” can be false. Providing patients with the opportunity to be responsible for their own health could be a factor that diminishes poor compliance. Patients seems to have strong preference for SCIg, suggestion association with better quality of life (QoL). Marie-Claude Levasseur, ESID 2014

23. Discussion IVIg can not be delivered at home in Quebec Hospital-administered Ig may be unpleasant (set-up, waiting, comfort) Because SCIg is free in our center, the cost of material was offset by the saving in loss of productivity and cost of travel. Marie-Claude Levasseur, ESID 2014

24. Conclusion We believe there are no ideal candidates for SCIg and IVIg therapy Patients is a partner Giving the choice is a feasible, safe and efficient strategy. Marie-Claude Levasseur, ESID 2014

25. REFERENCES 1.Ochs HD, Gupta S, Kiessling P, Nicolay U, Berger M, Subcutaneous Ig GSG. Safety and efficacy of self-administrered subcutaneous immunoglobulin in patient with primary immunodeficiency diseases. J Clin Immunol. 2006;26(3):265-73 2.Berger M Choices in IgG remplacement therapy for primary immune deficiency diseases:subcutaneous IgG vs. Intravenous IgG and selecting and optimal dose. Curr Opin Allergy Clin Immunol.2011;11(6)532-8 3.Chapel HM, Spickett GP, Ericson D, Engl W, Eibls MM, Bjorkander J. The comparison of the efficacy and safety of intravenous versus subcutaneous immunoglobulin replacement therapy. J Clin Immunol. 2000;20(2):94-100. 4.Ballow M.mImmunoglobulin therapy:methods of delivery. The J of allergy and clin immunol. 2008;122(5):1038-9 5.Haddad E, Bames d, Kafal A. Home therapy with subcutaneous immunoglobulins for patients wit primary immunodeficiency disease. Transfusion and apheresis science:official j of the World Apher Asso : official j of the Eur Soc for Haemapheresis 2012;46(3):315-21. 6.Wasserman RL. Progress in gammaglobulin therapy for immunodeficiency: from subcutaneous to intravenous infusion and back again. J Clin Immunol. 2012;32(6):1153-64

26. MERCI! Collaborators: Kathryn Samaan Hugo Chapdelaine Anne Des Roches Helene Decaluwe Elie Haddad Marie-Claude Levasseur, ESID 2014