News
EU (European Union) Consensus Conference
(Peter Vickers
and Ann Gardulf)
Summary of Presentations
Nurse Presentations
Dr. Ann Gardulf - Quality of Life and Healthservice Costs:
Why Diagnosis and Optimal Treatment is Good for the Patient and Good for the Healthcare Systems
PIDs are currently an un-addressed public health issue in the EU, with a large.number of children and adults suffering from recurrent infections within the member states without being diagnosed and so being offered treatment
PID sufferers in Europe:
No. of people with PID
- Sweden (population 9 million) 36 000
- Enlarged European Union 1 940 000
(population 485 million)
To suffer from recurrent infections means:
- to live with symptoms and the sense of being ill every day
- to be very tired and to feel exhausted
- to have difficulties in being able to work or study
- to perceive several restrictions in everyday life and not being able to cope with family, social and leisure activities
- and always having to live with the fear of becoming infected leading to coping behaviours based on withdrawals to avoid persons and situations with the risk of acquire an infection.
- Antibody Replacement Therapy improves the Quality of Life (QoL) by preventing infections.
- If not diagnosed and therefore treated, adults with PID have a significantly worse QoL as compared to healthy individuals.
- In the graph below, the higher the score, the poorer the Q0L.
Gardulf, Björvell, Gustafson et al
Clin Exp Immunol 1993
- But with Antibody Replacement Therapy, the QoL significantly improves
- The higher the score, the worse the QoL
Gardulf, Björvell, Gustafson et al
Clin Exp Immunol 1993
Patient Stories:
- The National Insurance Company wanted an early retirement for a 32-year old woman. She was given antibody replacement therapy (subcutaneous IgG) – today she is working full-time.
- A 44-year old man worked full-time but was completely exhausted at the end of the day. He had difficulties participating in family and social life, and he could not participate in recreational activites. But now, following trsatment, he says, ”I have gained a new life”
Adult patients’ opinions of the home-therapy categorised in five groups/areas:
- Independence/freedom/flexibility
- No travelling to hospitals
- Less time away from work or school
- A sense of being less sick or disabled
- Resources made availiable to other patients
Gardulf, Björvell, Andersen, Björkander et al.
J Adv Nurs, 1995
Independence/freedom/flexibility
- I can travel without any problems. Now I feel I can contribute to my own well-being, I am no longer just a ’patient’ or a ’case’.” (Woman , aged 27)
- You feel less sick when you don’t have to visit the hospital so often. Freedom!” (Man, aged 29)
- The home treatment means greater independence and flexibility.” (Man, aged 45)
A sense of being less sick or disabled
- I find the home treatment outstanding. Now I live a normal life and I don’t feel disabled any longer. It makes me so happy.” (Woman , aged 51)
- Home treatment gives me the opportunity to live like a ’normal’ human being. If anyone feels disabled by the antibody deficiency, home treatment reduces this feeling to a minimum.” (Man, aged 62)
The parents to children with PID reported significantly:
- Fewer limitations in personal time and less emotional distress due to the child's PID
- Fewer limitations in family activities, school, work & social activities
- Fewer disruptions in family activities and fewer cancellations or changed plans at the last minute affecting work and social life
- Less tension or fewer conflicts at home
- Better therapy convenience, comfort, treatment flexibility and pleasantness of treatment atmosphere
Gardulf, Nicolay, Asensio, et al. J Allergy Clin Immunol 2004
Conclusions;
- Scientific studies show patient-reported health and QoL are significantly improved by optimal antibody treatment
- Home-therapy has been shown to further increase the QoL for patients with PID and their families
- The introduction of home-therapy programs leads to substantial savings for healthcare services, patients and their families
- Mechanisms are needed to ensure that the awareness regarding PID is increased within the EU member states and that these conditions are recognised as an important public health issue so that children and adults can be helped to a life in dignity and a life with a high quality of life
Mrs Amena Warner - The Role of Nurses in recognising Primary immunodeficiencies (PIDs)
ICN definition of Nursing:
Nursing: encompasses autonomous and collaborative care of individuals of all ages, families, groups and communities, sick or well and in all settings.
Nursing: includes the promotion of health, prevention of illness, and the care of ill, disabled and dying people.
Advocacy, promotion of a safe environment, research, participation in shaping health policy and in patient and health systems management, and education are also key nursing roles.
The UK Royal College of Nursing definition:
The use of clinical judgement in the provision of care to enable people to improve, maintain, or recover health, to cope with health problems, and to achieve the best possible quality of life, whatever their disease or disability, until death.
Defining Characteristics:
The purpose of nursing is to:
- promote health, healing, growth and development,
- prevent disease, illness, injury, and disability.
When people become ill or disabled, the purpose of nursing is, in addition, to:
- minimise distress and suffering
- enable people to understand and cope with their disease or disability
- its treatment and its consequences.
Domains of Practice:
- Nursing interventions are concerned with empowering people, and helping them to achieve, maintain or recover independence.
- In addition to direct patient care, nursing practice includes management, teaching, as well as policy and knowledge development.
Focus and Values of Nursing:
- Nursing is focussed on the whole person rather than a particular aspect or pathological condition…Holistic Healthcare
- It is based on Ethical values which respect the dignity, autonomy and uniqueness of human beings
- There is a privileged nurse - patient relationship
Exploration of the privileged Nurse-Patient Relationship:
- Nurses in all areas of the healthcare sector play a vital role in the direction of patients with PID. The nurses are often the first ones to hear a long-lasting history of recurrent infections (Gardulf 1994)
- Nurses spend more time with patients and their families than any other group of healthcare professionals
- As the first healthcare professionals to come into contact with a patient or to see infants and toddlers in hospital, the observant, well informed nurse can play a vital role in the early detection of antibody deficiency (Cochrane S, Salomé-Bentley N, Toolan J. 2006)
- Nurses have the ability to evaluate and guarantee a high quality and cost effective care (Ibid.)
Diversity of Nursing Practice:
Student Nurses
Ward Nurse
Ward Sister
Nurse Specialist
Nurse Consultant
Nurse Researcher
Lecturer
Midwife
Health Visitor
School Nurse
Public Health Nurse
Community Nurse
District Nurse
Practice Nurse
Nurse Practitioners
Nurse Specialists and the International Nurses Group in Immunodeficiency (INGID):
Nurse specialists play a central role in disease education and monitoring compliance of treatment
In the UK alone we have 104 members of the Royal College of Nursing, Immunology and Allergy Nurses group, whose purpose is to improve the nursing care and management of patients with Primary Immunodeficiency
On an international scale we have a network of nurses worldwide who through our database we can contact and circulate our e-journal and e-newsletter to help keep them updated with latest developments. This helps international collaboration between nurses in research and quality assurance projects. It also provides advice and support with other nurses often sharing thoughts and experiences.
Note:
Several national and international nursing organisations are supporting this PID consensus conference. Nurses can take a very proactive role in recognising patients with signs and symptoms that may indicate a Primary Immunodeficiency. Therefore helping to support an early diagnosis
Examples of potential nursing diagnosis related to PID are altered growth and development, body image disturbance and ineffective individual or family coping, knowledge deficit (Winkelstein 1992)
In areas where Nurse Specialists are not available, home care provision is either limited or it is absent.
Conclusion:
The ability of nursing to respond to people’s need for nursing within the rapidly changing environment of health care depends on:
- the way in which nursing work is organised in health care delivery systems
- the way in which nursing practice is regulated and the quality of care is assured
- the way in which practitioners are prepared.
Dr Peter Vickers - Educational Outreach Efforts by Nurses
Why educate nurses?
- Nurses are extremely important for the health of a population as they have the qualifications to find ‘ill-health because they make up the largest single professional group in the health care system and can be found in all societal sectors and areas. (WHO [1982] Nurses in Support of the Goal of Health for All by the year 2000)
- Nurses have the ability to evaluate and guarantee a high-quality and cost-effective care. (ibid.)
- Nurses in all areas of the health care sector play a vital role in the detection of patients with PID. The nurses are often the first ones to hear a long-lasting history of recurrent infections. (Gardulf 1994)
- For example, potential nursing diagnoses related to PID are altered growth and development, body image disturbance, ineffective individual and/or family coping, and knowledge deficit. (Winkelstein 1992)
“Knowledge itself is power” (Francis Bacon 1561-1626)
This power through knowledge can be used to:
- Empower the nurses to become knowledgeable, thoughtful, caring and efficient professionals and practitioners.
- Improve the early suspicions a nurse may have of a Primary Immunodeficiency (PID), and in turn help to improve the early diagnosis rate.
- Along with other health care professionals, improve the care of patients with PIDs and their families.
- Communicate with the patients and their families, and other health professionals, as equal partners in their care and treatment.
- Help to empower the patients and their families to come to terms with, and to live with, their condition.
Nurse Outreach Education: What is Available?
- Masters Degree in Advancing Practice: Immunology (University of Hertfordshire, UK)
- Post-Registration Certificate in Immunology Nursing (University of Hertfordshire, UK)
- Two ‘stand-alone’ specialist modules at undergraduate or Master’s level (University of Hertfordshire, UK)
- Education regarding PID for some nursing students during their basic education at some Swedish and UK universities
- Immunology/Immunodeficiencies - antibody deficiency: Nurse Education Module (UK Nurses/Baxter)
- Planned 10-weeks education in immunology and PID at the Karolinska Institut, Sweden
- Planned basic-level introduction to the immune system and PIDs organised as a web-based learning package in the UK (RCN Immunology and Allergy Nurses Group)
- Informally:
- Education at Conferences and Meetings (INGID, RCN)
- Education section in the INGID eJournal
Problems:
- There are difficulties in recruiting young nurses into the specialty because of a poor awareness of immunology (and PIDs in particular) because these 2 subjects are not taught in all basic nursing education programmes - lack of qualified and experienced teachers
- Apart from the Swedish initiatives, these educational opportunities are only in English
- At the moment, there is no entrance level education - other than local provision in the UK
- University of Hertfordshire courses are at advanced level
- CD Rom is intermediate level
Conclusion:
- The specialties of Immunology and Primary Immunodeficiency Disorders are complicated and not easily understood, and therefore resources are needed in both theory and practice to improve the knowledge and experience of nurses.
- Our knowledge of these subjects and illnesses is increasing at an exponential rate.
- People with PIDs and their families need and deserve the best treatment and care possible.
- Nurses are a major component of the health care workforce.
- Nurses spend more time with patients and families than any other health care professional.
- Consequently, nurses need to be well educated in the discipline and specialty in order to play their part in the care and treatment of people with PIDs and their families.
- For too many years, nurses throughout Europe have had to try and learn about these disorders on their own in order to be able to give the optimum care.
- Now, mechanisms are needed to ensure that education reaches out to all nurses within the EU member states.
Members of the INGID Board invited to the EU Consensus Conference.
From left to right, Peter Vickers, Ann Gardulf, Amena Warner, Nina Berger.
Presenters at the Consensus Conference
(Ann Gardulf and Amena Warner in the centre, Peter Vickers in his usual position - hiding at the back)
Other Presentations:
François Houÿez (Health Policy Officer, EORORDIS) - Addressing Inequalities of Access to Health Care in an Expanded Europe
First of all M. Houÿez discussed EURORDIS, which was created in 1997 by a Treaty, and which is a patient driven initiative. Over 1 000 rare diseases are represented by EURORDIS - obviously not all primary immunodeficiencies, but they do make up just over 10% of these rare diseases.
The Treaty that set up EURORDIS stated that there is a national responsibility to ensue competence throughout Europe. One of the most important responsibilities is towards the quality of care, although M. Houÿez did point out that this is not so much an issue in the United Kingdom.
M. Houÿez pointed out that the main factors leading to inequality, not only in Europe but in the whole world, include:
- Income
- Education level
- Activity status
- Regime disparities
- Private health insurance
- Lack of information
He stated that much of the financing of care and treatment depends upon the size of the patient population within a particular disease/disorder. Therefore, this use of size of patient population to determine financing does not favour the rarest of the rare diseases.
M. Houÿez also discussed other aspects of coping with the rarest of rare diseases, for example, when discussing the effects upon the family:
- Care is a full-time activity, therefore often one parent needs to stop working.
- There is limited financial compensation.
- There are unrealistic health insurance premiums.
The European Union (EU) strategy is to compare health policies across member states, which will then lead to the creation of reference care guidelines.
Since the advent of EURORDIS, there is now the possibility of complaints been made to the European Commission against member states for violating community law (and under this can be included access to appropriate health care). Initially these complaints will be made to the national courts within the member nations, but there is or is the possibility of referring to the European Convention for Human Rights.
Professor Edvard Smith (Karolinska Institut, Stockholm) - PIDs - a Public Health Issue?
Professor Edvard Smith & Amena Warner
Professor Smith gave the background to the issue of primary immunodeficiency disorders in Europe.
Primary Immunodeficiency Disorders:
- This is a currently on addressed public health issue in the EU.
- It affects up to 1.5 million people across Europe, with an estimated 60 000 severe cases.
- Large numbers of children and adults are suffering from recurrent infections, as well is or to immunity, tumours, etc.
What is the need?
- An increase in quality of life
- a decrease in costs
How many types or of primary immunodeficiencies are there?
- 1986 - the first gene defect for a primary immunodeficiency disorder was discovered
- 1999 - 74 primary immunodeficiency disorders identified
- 2006 - 128 primary immunodeficiency disorders identified
- 2020 - more than 300 primary immunodeficiency disorders predicted!
Other Points:
- European primary immunodeficiency disorder research is world leading
- There are many common themes between the different rare diseases, for example:
- basic biology
- instruments for patient data handling
- Collaboration channels, e.g. ESID, INGID, IPOPI.
- An example of collaboration is ESID (www.esid.org) where there is collaborative research between member countries
To conclude, Professor Smith gave an example of how one gene can cause many diseases. For this, he looked at the gene that causes Wiskott-Aldrich Disease. The same gene dysfunction can cause:
- The classical syndrome of Wiskott-Aldrich disease.
- A milder disease - bleeding disorder
- Granulocyte disease.
Brian O'Mahony (Irish Haemophilia Society) - The Importance of Patient Registries in Rare Diseases
Mr. O'Mahony used his experience working within the field of haemophilia to explain about the importance of compiling and maintaining patient registries.
First of all, he defined a Registry as a record of demographics, type and amount of disease.
Benefits of Patient Registries:
- Advisability for health.
- Allocation of resources.
- Monitoring trends in health.
- Distribution mechanisms.
- Communication
Advisability for health
Under this heading, Mr. O'Mahony discussed:
- Organisation of care
- Improvement of care
- Requirement of treatment products
These need to be planned, depending upon demographics, i.e.
- number of patients
- level of care and unmet medical needs
- location of patients
Healthcare budgets are allocated on the basis of priority, therefore governments need to know about primary immunodeficiency disorders, such as:
- How many patients?
- How much does treatment cost?
Expensive chronic diseases often have a low priority, therefore there is a:
- Need to demonstrate the requirements of the patient population.
- Need to demonstrate the benefits of government intervention.
Allocation of resources
Under this heading our included:
- Location of Treatment Centres.
- Requirement of the different treatment products.
- Providing/defending budgets -obtainable from the Registry data.
- Impact of treatment on outcomes.
- Low-cost activities to increased life expectancy, including:
- modification of structure and organisation of care delivery
- education are health professionals and patients
- modification of blood bank products
Monitoring trends in health
Identifies:
- Changing requirements of patient populations.
- Problems.
- Documented impact of changes.
Distribution mechanisms
This can improve tendering/distribution, by identifying and analysing:
- Quantities required.
- Organisation and monitoring of distribution and usage.
- Product recall.
- Patterns of use.
Communication
Provides patients with opportunities to becoming for in various initiatives, such as:
- Access to educational material.
- Planning of services to/by patient organisations.
- Priorities/strategies - from demographic information.
Global Data
With the use of data from registries from around the world, there are many improvements that can be made, for example:
- Advancements leading to improvements to care.
- Comparative data is very useful for individual National Organisations, and again leading to improvements to treatment and care.
- It is possible to document the effectiveness of provision of care in different environments and sociological groups.
PostScript
For further information, check the Hemophilia Web site for the document "World Federation of Hemophilia Guide to Developing a National Patient Registry" (Bruce Evatt).
Dr Helen Chapel (John Radcliffe Infirmary, Oxford) - Overview of PIDs in Adults and Children
Dr Chapel began by discussing the importance of immunity, mainly that it protects against infection.
In particular, she looked at the development of treatment and care for primary immunodeficiency disorders:
Antibody deficiencies:
- 1950-1970 life expectancy = 12 years treatment = IMIG
- 1970-1990 life expectancy = 45 years treatment = IVIG
- 1990-2005 life expectancy = 70 years treatment = IVIG/SCIG
Severe combined immunodeficiencies:
- 1950-1970 life expectancy = <2 years treatment = none
- 1970-1990 life expectancy = 30% = 10 years treatment = BMT
- 1990-2005 life expectancy = 80% = 20 years treatment = BMT
Professor Lennart Hammarström (Karolinska Institut, Stockholm) - Prevalence of PIDs in the European Union (EU)
Professor Edvard Smith, Dr. Ann Gardulf & Professor Lennart Hammarström (The Swedish/Karolinska Institut Contingent)
Professor Hammarström began his presentation by looking at the different classes of primary immunodeficiencies. He gave the following statistics:
- 90% of patients have B-cell deficiencies in line
- 5% of patients have T-cell and combined immunodeficiencies
- 5% of patients have Complement deficiencies
Professor Hammarström then gave statistics for the prevalence of antibody deficiencies in any population. These are:
- HyperIgM deficiency 1:500 000 (i.e. 1 in 500 000)
- CVID 1:20 000
- IgA deficiency 1:600
- IgG subclass deficiency 1:600?
Professor Hammarström was then able to give the prevalence of other diseases as well as primary immunodeficiency disorders (PIDs) for comparison:
- Rheumatoid arthritis 1:100-200
- PID 1:250-500
- Gluten enteropathy 1:200-800
- Type 1 diabetes (insulin-dependent) 1:600-800
- Multiple Sclerosis 1:800-1 000
Professor Hammarström followed this up by looking at the number of people with primary Munich deficiencies that had been diagnosed in Sweden, and comparing it with the number that would be expected from the statistics above:
- HyperIgM 11 expected = 15-20
- CVID 250 expected = 400-500
- IgA deficiency 1600 expected = 15 000
- IgG subclass deficiency 500 expected = 15 000
- Complement 2 deficiency 41 expected = 200-600
This demonstrates that far too many people with primary immunodeficiency disorders are just not been diagnosed, and this is in a European country that is relatively sophisticated in its identification, care and treatment of people with primary immunodeficiencies.
Finally, in the presentation of statistics, Professor Hammarström then extrapolated the statistics for the prevalence of several primary immunodeficiencies to the whole of the European Union:
- HyperIgM 600 patients
- CVID 25 000 patients
- IgA deficiency 750 000 patients
- IgG subclass deficiency 75 000 patients
- Complement 2 deficiency 20 000 patients
This would make approximately 1 500 000 patients in the EU with one of the primary immunodeficiency disorders, and of these, approximately 60 000 what have a severe form of primary immunodeficiency disorder.
So what are the diagnostic problems and potentials? Professor Hammarström mentioned three of them:
- Awareness
- Diagnostic tools/Centres
- Screening
He also pointed out that although the symptoms may be the same for many different primary immunodeficiency disorders, the actual cause can be very different, and this may cause problems with diagnosis. In addition, primary immunodeficiency disorders can be associated with other disorders, and Professor Hammarström gave the example of primary immunodeficiency disorders and autoimmunity.
Screening for PID
- We could screen populations, or
- we could screen neonates (this would identify most patients)
Neonatal screening could use the same samples as we obtain for phenylketonurea, i.e. blood from a heel prick. We now have the tools from making a neonatal diagnosis.
From neonates, at the moment, we can diagnose for:
- All IgA deficiencies, including Hyper IgM, CVID, IgA deficiency
- Complements deficiencies (C2 and C3)
- 2 different types of SCID
Dr. Hilary Longhurst (Barts & The London NHS Trust, London) - Treatment Options: Immunoglobulin Therapy - The Major Therapy Choice in PID.
Dr, Longhurst first of four posed the question as to whether or not immunoglobulin therapy works. She looked at three reports:
- Medical Research Council (UK) in a study from 1955-1970 of low-dose immunoglobulin therapy, found that there was a 10-year survival rate in 30% of males and 36% of females.
- Cunningham-Rundle (USA) looking at the years 1973-1998 found that with high dose immunoglobulin therapy, there was a 10% survival rate in 78% of males and 78 of females, and a 20 year survival rate of 64% in males and 67% females.
- In the United States, a report in 1999, found that with high dose immunoglobulins therapy, there was a 10 year survival rate in 97% of males and 98 of females, and a 20 year survival rate of 92% in males and 94% in females.
Therefore, we can be certain that immunoglobulin therapy does work, and also that:
- high dose therapy is better than low-dose therapy
- we also know that I V therapy is better than IM therapy
- it improves the quality of life of the patients (and their families)
- it decreases overall health care costs.
Is there a risk of getting infections with immunoglobulin therapy?
- There has been mission of HIV or hepatitis B
- Hepatitis C has been transmitted by some batches, but now there is more rigorous testing this is no longer seen as a problem.
What about adverse reactions?
- These are minute now because of:
- highly purified preparations
- slow infusion times
- better choice in preparation of immunoglobulins
It takes 12-36 000 donors per batch of immunoglobulin, and this gives a good general protection against most common infections.
A solution for immunoglobulin therapy is made up of 95-99% IgG, whilst there are low levels of aggregated immunoglobulin, and other immunoglobulins.
Professor Christine Kinnon (Institute of Child Health, London) - Treatment Options: Bone Marrow Transplants/Gene Therapy
In this presentation, Professor Kinnon discussed bone marrow transplants (BMT) and haemopoietic stem cell transplants (HSCT).
The use of matched donors for these transplants is very successful, with 90% being successful. However, for 60% of patients, there is no matched bone and therefore it is still necessary to use a mismatched bone (i.e. one of the parents usually). The success rate for mismatched transplants is less than 60%. Therefore the challenge is to improve the success rate for children who do not have a matched donor.
Mismatched Transplants
- There is a mortality rate of 20% in the first year after the transplant.
- There are long-term effects that are related to the chemotherapy required to ablate the small amount of T-cells the child possesses.
- Sometimes there is incomplete immunological reconstitution.
Gene Therapy
- This is a good option for X-linked SCID, with 9 children so far successfully treated.
- All 9 of the children are showing signs of immunological reconstitution, with a satisfactory immune cell function and normal immune responses.
- 5 out of these 9 children are at home and off all therapy.
- In France there are 10 out of 11 will have had a good result from the gene therapy.
- However, out of these 10 children, 3 developed a leukaemia-like disease, which was caused by the transplanted gene in asserting itself into the wrong place in the DNA. All 3 were successfully treated this disease, and procedures have been put in place to try to make sure that it does not happen again.
The Future
Attempts are ongoing for the development of safer viruses as vectors for Gene therapy:
- One line of research is looking at the use of self-inactivating (SIN) retroviruses.
- A second route is the development of SIN lentiviral vectors, which use similar properties to HIV to insert genes into cells.
Other Gene Therapies:
- ADA SCID 7 children treated in Italy 2 children treated in the UK
- CGD 2 children treated in Germany 2 children treated in the UK
- WAS trials have been planned using lentiviral viruses in 2007
Similar techniques could be used to treat other diseases, for example:
- leukaemias
- haemopathies, such as sickle cell anaemia, and others.
Professor Anna Sediva (Charles University, Prague) - Disparity of Diagnosis, Care, andTreatment for PIDs between member states of the EU.
To give an idea of the disparity of diagnosis of primary immune deficiencies between member states of the EU, Professor Sediva explained that the rate per million population in 2004 of primary new no deficiency disorders in the EU was from 3 - 47 depending upon the member state.
For example,
- Yugoslavia = 3 per million population
- Sweden = 47 per million population
- UK =24 per million population
Professor Sediva explained that there are socio-economic reasons for these disparities, as well as basic 'biological' reasons for them.
In terms of the basic 'biological' reasons, Professor Sediva pointed out that there are different spectrum are of diseases and different genetic backgrounds predominating within different member states.
Professor Sediva also explained that home care (IVIG/SCIG) is available in most countries of the EU, but not in the old Eastern European countries that have recently joined the union.
Professor Thomas Szucs (Health Economics, Institut of Social & Preventative Medicine, University of Zurich) - Consequences of Delayed Diagnosis: Health-Care Costs.
Professor Szucs explained that there were two aspects of health-care costs to be taken into account:
- economics (macroeconomics)
- biopharmaceutical (microeconomics)
Professor Szucs then pointed out that 1 additional life year expectancy equals a 4% increase in GDP.
Medical Care Costs
These are made up of:
- Patient/disease related factors.
- Treatment related factors.
- Provider/hospital related factors.
- Environmental factors
Professor Szucs also presented a formula, namely,
He also distinguished between efficacy and effectiveness in treatment.
- Efficacy = performance and are ideal conditions (RCT).
- Effectiveness = performance and are usual conditions.
Other definitions that Professor Szucs gave were:
- Cost effectiveness analysis includes clinical parameters and survival.
- Cost utility analysis = quality adjusted life analysis.
Professor Reinholdt Smith (EFIS) - Evaluating Strategies for Early Identification of People with PIDs.
Professor Smith introduced the idea that the main factor in reducing the disparity between member states of the EU regarding the diagnosis of PIDs is early diagnosis.
Professor Smith discussed the fact that these strategies for the early identification of people with PIDs can only be affected by increasing the awareness of these disorders.
He determined that 3 groups needed to have an increased awareness of primary new no deficiency disorders. These 3 groups are:
- The general public.
- Health-care professionals.
- Health-care policymakers and implementers.
The General Public
How can awareness be increased in this group? Professor Smith came up with a number of ideas,
- Campaigns
- 10 warning signs
- Day of immunology - to take place on the 29th April 2007 (more information about this day of immunology can be obtained from EFIS)
- TV shows & films
- Other media activities, e.g. advertisements, radio, newspapers.
Health-Care Professionals
Ideas put forward by Professor Smith included,
- Studies on disease impact.
- Studies on therapy efficacy.
- Epidemiology & public health input.
- Cost effectiveness studies.
- Education:
- Health education in primary school communities
- Students and nurses about PIDs.
- Patient organisations at society meetings
- EU should provide education to member states (courses, exchanges, etc.)
- Education of related health-care professionals.
Professor László Maródi (University of Debrecen Medical & Health Science Centre, Hungary) - Educational Outreach Efforts by Physicians - J-Project.
This was commenced in 2002, and is an Eastern European Network.
Professor Maródi in his presentation sent out the message to doctors that they have to initiate education. He discussed the J-Project and its aims, which are:
- To improve the awareness of primary immune deficiency disorders in Eastern Europe.
- To have a PID symposium in Debrecen every year for ECE delegates.
- To organise local meetings.
- The discussed diagnostic and therapeutic problems.
- To update National PID registries.
- To have in existence a PID professional working group.
- To have in existence a PID patient working group.
Financial support comes from Biotest and the Jeffrey Modell Foundation.
Professional support comes from ESID.
Mr. Fred Modell (Jeffrey Modell Foundation, USA) & Mrs Anneli Larsson (PIO) - Education Outreach Efforts by Patients.
59 patient groups participate in the Jeffrey Modell Foundation's World Immunodeficiency Network (WIN).
The Jeffrey Modell Foundation National Hotline and the web site (which has had 665,617 hits) offers referrals to 118 Jeffrey Modell Foundation physician-referral centres.
A USA consensus conference in 2001 to assess the public health impact of PIDs concluded that:
- PID is a serious public health issue.
- The message of the campaign is that the earliest possible diagnosis of a PID should be made.
The 10 warning signs of a Primary Immunodeficiency checklist, and the 4 stages of immunologic testing, is distributed to 25 countries and in 20 languages.
There is a CD-ROM starter kit for new patients available.
Public Service Advertising campaigns include:
- television
- radio
- newspapers
- magazines
- airports
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