Dose and outcomes in primary immunodeficiency disorders.

摘要: Currently, the principal form of treatment for patients with primary immunodeficiency disorders (PID) in Europe is subcutaneous immunoglobulin (SCIg) replacement therapy. Conversely, until relatively recently, intravenous (IVIg) was preferred option PID United States. Patients are more susceptible to recurrent or severe infections than individuals without PID. A retrospective analysis data from European Society Immunodeficiencies (ESID) registry, between 2004 and 2010, showed marked regional variability clinical outcomes receiving SCIg IVIg therapy 1. In this analysis, appeared present serious bacterial spend days hospital SCIg. Furthermore, a subgroup patients, those who switched spent fewer after switch 1. However, there number considerations when deciding on route G (IgG) administration. The volume (Ig)G that can be given into (s.c.) space limited compared delivered intravenously (i.v.); thus, must administered frequently IVIg. regimens include weekly, bi-weekly daily push doses self-administered. Typical i.v. administration once every 3–4 weeks at home clinic setting. contrast cycle peak trough serum IgG levels following infusion, produce minimal variation 2. Systemic side effects common SCIg, infusion site reactions particularly during early phase s.c. 2,3. An alternative method facilitate pre-infusion recombinant hyaluronidase followed by monthly dose IgG. This has been reported have low rate systemic effects, similar conventional 4. The impact home-based weekly self-infusions health-related quality life (HRQoL), satisfaction patient preference either venue investigated treated previously 5. hospital-based significantly limitations work/daily activities, improved (e.g. feeling energy, less tiredness fatigue) better general health. While were over infusions overall linked Specifically, effect improving (QoL) striking home; major factor QoL 5. Administration via becoming increasingly popular States, survey US immunologists majority respondents agreed as effective 6. However, question still remains optimal dosage regimen provides best outcome preventing and/or Progressive increments linear increases levels, leading 27% decline incidence pneumonia 100 mg/dl increase up least 1000 mg/dl 7. Similarly, higher shown also correlate levels. although achieved correlated lower rates non-serious infections, no correlation infection study 8. Nevertheless, provide improvements outcomes, including hospital, antibiotics reduced annual 8. Although targeting serum/trough level raises above 500 mg/dl useful guide beginning 7,8, healthy wide well ‘targeted’ 9. protects against likely vary widely each patient, it does suggested goal should identify individual ‘biological’ required maintain infection-free possible 10. obtained charting patient's their time, ‘moving target’ because altered changes status, such body weight, pregnancy development co-morbid conditions, renal 10 gastrointestinal disease. concept an supported large cohort variable (CVID) X-linked agammaglobulinaemia (XLA) followed-up period 22 years 11. clearly range keep these possible, only some need largest reduce pneumonias addition, amount CVID versus XLA healthy, suggesting original repertoire may important determining two different populations healthy. Moreover, used did not exceed found age-appropriate individuals. A 5-year prospective identified subgroups high risk 12. had if they IgA diagnosis decrease progression bronchiectasis 13. another 2-year study, >600 mg/dl controlling pulmonary complications 14. 800–>1100 mg/dl slower age-associated function tests adults 15. In conclusion, individualizing administration, together identification ‘biological level’, helps prevent infections. individualization improve adherence allowing choose suits lifestyle. Finally, sufficient amounts IgG, route, minimize infection. identifying then maintaining level’ time depending conditions.