Stakeholder Opinions: Alpha 1 Antitrypsin Deficiency

Table of Contents

• ABOUT DATAMONITOR HEALTHCARE
  • About the Respiratory & Infectious Disease (RID) analysis team
• CHAPTER 1 EXECUTIVE SUMMARY
  • Scope of the analysis
  • Datamonitor insight into the disease market
  • Related reports
• CHAPTER 2 DISEASE BACKGROUND
  • Alpha-1 antitrypsin deficiency is a hereditary disease found mainly in Caucasians
  • Etiology of alpha-1 antitrypsin deficiency
    • Genetics of Alpha-1 antitrypsin deficiency
    • Epidemiology
  • Emphysema
  • Liver disease
  • Unmet needs
  • Risk factors
    • Smoking
      • Environmental tobacco smoke exposure
    • Occupational exposure
    • Bacterial infections
    • Body mass index
• CHAPTER 3 DIAGNOSIS
  • Diagnosis of alpha-1 antitrypsin deficiency
    • Lung function
    • Imaging
    • Serum alpha-1 antitrypsin levels
    • Biochemical markers
    • Phenotyping
  • Diagnostic guidelines
  • Genetic screening
  • Increase in age at diagnosis and delay in diagnosis of alpha-1 antitrypsin deficiency
    • Problems with physician knowledge
  • Difficulty in conducting clinical trials in COPD
  • Declining smoking rates
• CHAPTER 4 TREATMENT OPTIONS
  • Standard COPD therapy
  • Augmentation therapy
    • Therapeutic rationale
    • Market analysis
    • Cost and reimbursement
  • Antibiotic therapy
  • Organ transplant
  • Pulmonary rehabilitation, supplementary oxygen and genetic counseling
• CHAPTER 5 FUTURE TRENDS
  • Inhaled alpha-1 antitrypsin augmentation therapy
    • Arriva/Hyland
    • Kamada
    • Talecris
  • Recombinant alpha-1 antitrypsin augmentation therapy
  • Gamma retinoid agonists
  • Gene therapy is in the far future
  • Alpha-1 antitrypsin replacement therapy in cystic fibrosis
  • Clinical trial endpoints
  • Continuing medical education
  • The role of patient support groups
  • Neonatal genetic screening
  • Transfer of treatments to the general emphysema population
• CHAPTER 6 BIBLIOGRAPHY
  • Articles
  • Websites
  • List of Tables
    • Table 1: Alpha-1 antitrypsin levels in common genotypes
    • Table 2: Estimated prevalence of the five main phenotypes of alpha-1 antitrypsin deficiency in selected countries
    • Table 3: Estimated numbers of each of the five main phenotypes of alpha-1 antitrypsin deficiency in selected countries
    • Table 4: Classification of recommendations for genetic testing
    • Table 5: Comparison of augmentation therapies in the US
  • List of Figures
    • Figure 1: Alpha-1 antitrypsin production and activity
    • Figure 2: An example of three-generation pedigree with alpha-1 antitrypsin deficiency
    • Figure 3: Estimated numbers of PiZZ individuals in selected European countries
    • Figure 4: Liver disease in PiZZ patients by age
    • Figure 5: Association between liver dysfunction and age in PiZZ infants
    • Figure 6: Flow diagram of anticipated liver disease outcomes in PiZZ infants
    • Figure 7: Decline in lung function by smoking status
    • Figure 8: Hospital admissions for alpha-1 antitrypsin deficient patients in Norway, 2005
    • Figure 9: SWOT analysis of human, plasma derived augmentation therapy