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Georgina L Jones,1 Kate Williams,2 Mark Edmondson-Jones,2 Johan Prevot,3 Jose Drabwell,3 Leire Solis,3 Anna Shrimpton,4 Nizar Mahlaoui5– 7 1Department of Psychology, School of Social Sciences, Leeds Beckett University, Leeds, UK; 2PAREXEL International, London, UK; 3International Patient Organisation for Primary Immunodeficiencies, Downderry, Cornwall PL11 3LY, UK; 4Clinical Immunology and Allergy Unit, Northern General Hospital, Sheffield Teaching Hospitals and NHS Foundation Trust, Sheffield, UK; 5French National Reference Center for Primary Immune Deficiencies (CEREDIH), Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux De Paris, Paris, France; 6Pediatric Immuno-Haematology and Rheumatology Unit, Necker Enfants Malades University Hospital, Assistance Publique-Hôpitaux De Paris, Paris, France; 7IPOPI Medical Advisory Board, ChairCorrespondence: Georgina L JonesDepartment of Psychology, School of Social Sciences, Leeds Beckett University, Calverley Building, City Campus, Leeds, UKTel +44 113 8125106Email g.l.jones@leedsbeckett.ac.ukPurpose: To describe the development and psychometric testing of a new questionnaire to measure the burden of immunoglobulin treatment (Ig) from the perspective of patients with primary immunodeficiencies (PID).Patients and Methods: An online, cross-sectional survey was administered to PID patients across 10 countries (nine European and Canada) who were receiving either intravenous (IVIg) or subcutaneous (SCIg) immunoglobulin therapy. The range and distribution of the responses (ie, levels of missing data, floor and ceiling effects), exploratory factor analysis (using factor loadings of 0.4 or greater) and measures of internal consistency reliability (ie, Cronbach’s alpha coefficient, inter-item and item-total correlations) were used to identify the domain and item pool.Results: In total, 472 patients completed the questionnaire, of which 395 were included in the analysis (32% underwent IVIg and 67% underwent SCIg). The final instrument contained 34 items across eight domains of treatment burden (time, organisation and planning, leisure and social, interpersonal relationships, employment and education, travel, consequences of treatment and emotional) and an additional Ig treatment burden global question at the end of the measure. All the scales achieved good internal reliability (Cronbach’s alpha coefficient ranged from 0.70 to 0.85) and, with the exception of one item exceeded the minimum threshold of 0.35 for item-total correlations. Treatment burden was lower than anticipated across the different treatment routes and countries, although overall was more burdensome for patients undergoing IVIg compared to SCIg treatment.Conclusion: The IgBoT-35 appears to be a reliable, patient-generated questionnaire and may help to identify more individualised and preferred therapies for the PID patient when used in clinical practice. A new survey with a sample of US patients is currently being undertaken to further establish its validity and conceptual model. The overall Ig burden of treatment scores appeared to be low. PID patient preferences are important to guide treatment decisions and ensuring patients receive the right treatment at the right time.Keywords: intravenous immunoglobulins, subcutaneous immunoglobulins, primary immunodeficiency, treatment burden, patient preference, quality of life

This paper describes the burden of receiving immunoglobulin (Ig) treatment from the perspective of patients diagnosed with a Primary Immunodeficiency (PID). Thirty semi-structured interviews with patients receiving intravenous (n=21) and subcutaneous immunoglobulin (n=9) therapy, either at home or in hospital were undertaken. Underpinned by a phenomenological theoretical framework, and using a qualitative, inductive thematic approach to prioritise patients’ concerns, we identified that Ig treatment requires considerable effort by the patient, particularly in relation to the amount of time, organization and planning that is needed. They also face numerous physical, social, relationship, emotional, role functioning, travelling, and financial challenges in their effort to undergo and maintain their infusions and care for their health. Some qualitative differences in treatment burden were noted between home and hospital settings which contributed to non-adherence to those regimes. Immunoglobulin treatment burden is complex and influenced by therapeutic mode and setting and the personal circumstances of the patient. As choice over treatment method appears to be mainly informed by lifestyle needs, PID patients may benefit from more information about these potential Ig lifestyle influences when selecting which form of treatment to take together with their health professional.

Teresa Espanol,1 Johan Prevot,2 Jose Drabwell,2 Seema Sondhi,3 Laurence Olding4 1Immunology Unit, Vall d’Hebron University Hospital, Barcelona, Spain; 2International Patient Organisation for Primary Immunodeficiencies, Cornwall, UK; 3Baxter Healthcare SA, Zurich, Switzerland; 4Bryter, London, UK Background: Subcutaneous or intravenous immunoglobulin replacement is the mainstay of treatment for most patients with primary immunodeficiency disease (PID). The purpose of this study was to gain an understanding of how existing PID therapies affect patient lives and to identify desired improvements to immunoglobulin treatments. Methods: An online questionnaire was made available through the International Patient Organisation for Primary Immunodeficiencies to patients with PID and their caregivers regarding current treatment satisfaction, living with PID, and patient preferences using a conjoint approach. Health-related quality of life was canvassed via questionnaires using the Short Form12Health Survey and EuroQoL5Dimensions. Results: A total of300responded to the survey (72% patients with PID and28% caregivers) from across21countries, mostly the UK, Sweden, Canada, France, Germany, and Spain. Fifty-three percent and45% of patients received intravenous and subcutaneous therapy, respectively. Most respondents (76%) were satisfied with their current treatment, reflecting the benefits that immunoglobulin therapy provides for patient health and well-being. However, patients remained below the physical and mental well-being norms for health-related quality of life as determined by the questionnaire. All respondents expressed a desire for4-weekly infusions, the ability to administer these at home, self-administration, shorter duration of administration, and fewer needle sticks. Conclusion: The results of this survey highlight the importance of providing access to different treatment options and modes of administration to ensure individual patient needs are best met. Keywords: primary immunodeficiency, immunoglobulins, quality of life, patient needs, patient satisfaction, conjoint analysis

Changes take place all the time in the world of primary immunodeficiency. It really is an evolutionary path and in the developed western world the advances in technologies are looked upon as a near normal way of life. However in the areas of the world where there are so many challenges just to stay alive, the emphasis has to shift to the more pressing needs of the entire population. There are countries in Africa where there is not enough water. And if there is not enough water nor wood to start a fire how will it be possible to achieve even the most basic hygiene. Boiled water? So diagnosis and treatment of people with a primary immunodeficiency are not going to get a priority in the severely restricted health services of those countries. Conflicts, AIDS and starvation are now part of life in some African and Asian countries.When an important advancement has taken place, such as the formation of the African Society for Immunodeficiencies (ASID), it can have a huge impact on the lives of people with a primary immunodeficiency. Suddenly immunologists, doctors, nurses, parents of children with a PID and patients themselves have a voice and a conduit to much information and assistance from the world wide PID community. Isolation, often associated with rare disorders, is no longer the issue. There is a constant need to raise awareness and promote the urgency of diagnosis, but also to increase the visibility of this rare condition and to dispel the suspicion and so often the mistaken identification and stigma of HIV. Sadly this is not confined to underdeveloped countries, it still happens in London, Paris and many other sophisticated cities. Raising awareness amongst the first line of medical practitioners, such as GPs, lung specialists, ear, nose and throat specialists and gastroenterologists still has to be a matter of the utmost urgency. However dentists, ophthalmic surgeons, allergy and skin specialists should be made aware of these conditions as well, because blocked tearducts, skin lesions that will not heal, and receeding gums all could indicate a problem with an impaired immune system. Once diagnosed there should follow a life saving programme of replacement immunoglobulin therapy. But what happens when such treatments are not readily available? In many countries there are inadequate diagnostic services available to obtain a diagnosis and that problem can be compounded when there is no health budget in place to buy the life saving therapies that could be available or to facilitate