Subcutaneous immunoglobulin replacement therapy for primary immunodeficiency in children and young adults. Radana Zachová Department of Immunology University hospital Motol, Charles University Prague, Czech Republic ESID Prague Spring Meeting 2009

Immunoglobulin replacement therapy in primary immunodeficiencies Life –long therapy for patients with PID Possible administration ways – intravenous, subcutaneous Historically - intramuscular Disorders: CVID X- linked agammaglobulinemia Specific antibody deficiencies ESID Prague Spring Meeting 2009

Subcutaneous immunoglobulin replacement therapy Patient Diagnosis Age (years) Subcutaneous therapy started Age at beginnig of therapy Results ZP CVID 29 2007 27 Home therapy MK 16 14 JJ 15 13 SM 2009 XLA 2 FV 52 2008 51 therapy in hospital SV XLP 19 17 refused s.c.therapy

Small infusion pump for scig administration NOT refunded from insurance ! WE lend this pump from company

Subcutaneous immunoglobulin replacement therapy in 1,5 year old boy. Case report: MK *2007 History: boy, 1. pregnancy, unrelative parents, born in term, 3100g, anorectal atresia, surgical treatment after delivery , 3 month - pyelonephritis 11 month - bilateral bronchopneumonia Immunoglobulin levels IgG, IgA, IgM – very low CD 19-1,1% CD 20-0,5% BTK mutation –pR28C missence mutation Family history: grandmother´s brother ( mother´s side) died at 18 years ( cerebral absces, recurrent bronchopneumonias)

Subcutaneous immunoglobulin replacement therapy in 1,5 year old boy. MK *2007 Therapy: Immunolgobulin substitution therapy was indicated Weight : 10 kg after first application of IVIG mother refused intravenous application of immunoglobulins Intramuscular administration was started 1x in 14 days - painfull, uncomfortable - mother refused I.M. application too ↓ We started subcutaneous application of immunoglobulins

Subcutaneous immunoglobulin replacement therapy in 1,5 year old boy. Thinks taken into consideration parents reffused IV and IM immunoglobulin substitution completelly subcutaneous therapy ? Disadvantages for SCIG low BMI – small subcutaneous tissue - big local swelling??? scar on abdominal wall after surgical treatment - only one side of abdominal wall can be used for replacement therapy 2 adult persons have to apply the therapy ( 1 has to fix the child) Actually: subcutaneous replacement therapy in home administration

Subcutaneous immunoglobulin replacement therapy in 1,5 year old boy. MK *2007

Subcutaneous immunoglobulin replacement therapy in 1,5 year old boy. MK *2007

Our experience with SCIG therapy SCIG replacement therapy is not limited by age Patients pretreatet with IVIG may refuse SCIG SCIG is suitable for patients with previous adverse reactions to IVIG

Thank you for your attention! ESID Prague Spring Meeting 2009