Agreed Statements and Actions

1. DIAGNOSIS
Proposed Champions: Martine Pergent, Vicki Modell & Dr Jose Franco

Primary immunodeficiencies (PID) are often misdiagnosed, leading to unnecessary illness and suffering. To increase the rate of diagnosis of these disorders there must be well orchestrated awareness programmes to match the needs of each individual country at a political and social level. Those campaigns must be easily understood both by family doctors and specialists.

Develop Guidelines to enable news of diagnosis to be delivered sensitively and appropriately with due regard to cultural circumstances
Develop Guidelines for adult care and diagnosis on the same basis as paediatric care and diagnosis
Develop Diagnostic Guidelines for cost effective front line testing for PID in emerging countries

2. TREATMENT
Proposed Champions: Dr Monika Esser, Dragana Koruga, Roberta Pena

Once diagnosed, most PID patients need access to a number of therapies, depending on the condition. The majority of patients have an antibody deficiency and require life-long immunoglobulin (Ig) replacement therapy, which is available from human blood and plasma donations. Ig is a proven treatment throughout the world and enables most people receiving it to lead full and productive lives. Failure to receive this treatment results in unnecessary hospitalisation and major reduction of quality of life and life expectancy. In addition each country needs to provide infrastructure support for bone marrow transplant and stem cell therapy to save the lives of severely affected patients.

Campaign to ensure that effective treatments are made available as a basic human right
Obtain a global consensus on viable Ig in emerging countries (refer to the process for distribution of vaccines in emerging countries)
Track the progress of ICD11 (International Classification of Diseases due to publish in 2011) and work with the expert Committee at IUIS as necessary
Encourage the work of the WHO Achilles Project
Campaign globally for adult care and diagnosis on the same basis as paediatric care and diagnosis

3. COMMUNICATION
Proposed Champions: Sven Fandrup, Stephen Baxter & Bob Perry

There needs to be sustained, regular and effective communication between all those committed to patient care including patients, caregivers, clinicians and researchers, patient group representatives, regulators, manufacturers and political decision makers.

Consider the use of language and the use of prevalence statistics
Consider the instigation of a worldwide Primary Immunodeficiency Awareness Day (or Week)
Be aware of all awareness raising campaigns and launch them simultaneously when possible
Campaign globally for adult care and diagnosis on the same basis as paediatric care and diagnosis

4. DATA COLLECTION
Proposed Champions: Marcia Boyle, Joy Rosario & Charles Waller

Data from patient registries and patient and physician surveys is invaluable in providing the information needed to demonstrate to those who make policy decisions the best strategies for success and the consequences of non-action.

Collect data from various countries regarding the efficacy of treatment and the health and experience of patients, as well as the ability to collect plasma for fractionation
Provide the data to convince payers of the appropriateness of treatment

5. GUIDELINES
Proposed Champions: Prof Helen Chapel, Dr Espanol & Dr Klaus Warnatz

Diagnosis and management need to be directed by frequently updated Guidelines, as well as Guidelines that emphasise priority usage of Ig and other valuable plasma-derived products.

Develop Guidelines to enable news of diagnosis to be delivered sensitively and appropriately with due regard to cultural circumstances
Develop Guidelines on the infrastructure for the transition from paediatric to adult treatment
Obtain global support for the development of a Guideline for the use and prioritization of Ig

6. SUMMARY

Primary immunodeficiency diseases are under-diagnosed, life threatening, but treatable disorders and patients globally face similar issues of diagnosis and access to appropriate specialists and optimal treatment.
Because the needs of the patients are great and resources are limited, it is imperative that we work even more closely together by having open, honest, productive, relationships as stakeholders in our efforts to educate physicians and health policy makers. Only then, by sharing and understanding goals, will we achieve the optimum for each other.