Home-based subcutaneous immunoglobulin G replacement therapy under real-life conditions in children and adults with antibody deficiency

F Hoffmann, B Grimbacher, J Thiel, H-H Peter, B H Belohradsky, Vivaglobin Study Group, Woldram Eball, Bernd Gruhn, Georg Jacobs, Wilma Mannhardt-Laakmann, Ove Peters, Ilka Schulze, F Hoffmann, B Grimbacher, J Thiel, H-H Peter, B H Belohradsky, Vivaglobin Study Group, Woldram Eball, Bernd Gruhn, Georg Jacobs, Wilma Mannhardt-Laakmann, Ove Peters, Ilka Schulze

Abstract

Background: Subcutaneous immunoglobulin (SCIG) therapy is an alternative to intravenous immunoglobulin (IVIG) therapy.

Methods: We evaluated the efficacy and safety of the SCIG Vivaglobin(formerly known as Beriglobin SC) under real-life conditions in a post-marketing observational study in 82 patients with primary or secondary antibody deficiencies. Health-related quality of life (HRQoL) was evaluated in a subset of 30 patients previously treated with IVIG (including 11 children <14 years) using the Short Form 36 (SF-36) for patients > or = 14 years of age (adults) and the Child Health Questionnaire - Parental Form 50 (CHQ-PF50) for children <14 years of age. Treatment preferences were assessed in adults.

Results: The mean serum immunoglobulin G (IgG) trough level during SCIG treatment (7.5 g/L) was higher than during previous IVIG treatment (6.6 g/L; p<0.01). The investigators assessed the efficacy of SCIG therapy as "excellent" in 89% of patients. No systemic adverse drug reactions were observed. Improvements by > or = 5 points were observed in 5 of 8 SF-36 subscales and in 6 of 12 CHQ-PF50 subscales. Statistically significant improvements (p< or =0.05) were observed for the SF-36 subscales of bodily pain, general health perceptions, and vitality (adults), and for the CHQ-PF50 subscales of general health perceptions, parental impact - time, parental impact - emotional, and family activities (children). Patients preferred SCIG over IVIG therapy (92%) and home therapy over therapy at the clinic/physician (83%).

Conclusion: This study confirms that therapy with Vivaglobin at home is effective, safe, well tolerated, and improves quality of life in patients with antibody deficiency.

Figures

Figure 1
Figure 1
Serum IgG trough levels (means and standard errors). For this analysis, 20 patients were considered who had prestudy serum IgG concentrations which fulfilled both of the following conditions: (i) measured within 2 months before study start and (ii) measured 3 to 4 weeks after an IVIG infusion. On average, visit 4 was 100 days and visit 7 was 290 days after baseline.
Figure 2
Figure 2
SF-36 Scores for adults (≥ 14 years) in the HRQoL subpopulation at baseline (visit 1) and follow-up (visit 7). Mean and standard error data are shown (N = 19, except for the scales general health perceptions and role emotional: N = 18). A score of 0 indicates the worst possible health state and 100 the best possible health state. * Statistically significant difference between baseline and follow-up (p ≤ 0.05). a) Physical health subscales. b) Mental health subscales.
Figure 3
Figure 3
CHQ-PF50 Scores for children < 14 years in the HRQoL subpopulation at baseline (visit 1) and follow-up (visit 7). Mean and standard error data are shown (N = 11, except for the scale self esteem: N = 10). A score of 0 indicates the worst possible health state and 100 the best possible health state. * Statistically significant difference between baseline and follow-up (p ≤ 0.05). a) Physical subscales. b) Psychological subscales. c) Parental impact and family subscales.

References

    1. Yong PF, Tarzi M, Chua I. et al.Common variable immunodeficiency: an update on etiology and management. Immunol Allergy Clin North Am. 2008;28(2):367–86. doi: 10.1016/j.iac.2008.01.001. ix-x.
    1. Pan-Hammarström Q, Hammarström L. Antibody deficiency diseases. Eur J Immunol. 2008;38(2):327–33. doi: 10.1002/eji.200737927.
    1. Cunningham-Rundles C, Bodian C. Common variable immunodeficiency: clinical and immunological features of 248 patients. Clin Immunol. 1999;92(1):34–48. doi: 10.1006/clim.1999.4725.
    1. Grimbacher B, Warnatz K, Peter HH. Der variable Immundefekt (Common variable immunodeficiency) 2003. (Accessed: 26 Nov 2009)
    1. Buckley RH. Primary immunodeficiency diseases: dissectors of the immune system. Immunol Rev. 2002;185:206–19. doi: 10.1034/j.1600-065X.2002.18517.x.
    1. Kirmse J. Subcutaneous administration of immunoglobulin. J Infus Nurs. 2006;29(3 Suppl):S15–20.
    1. Darabi K, Abdel-Wahab O, Dzik WH. Current usage of intravenous immune globulin and the rationale behind it: the Massachusetts General Hospital data and a review of the literature. Transfusion. 2006;46(5):741–53. doi: 10.1111/j.1537-2995.2006.00792.x.
    1. Radinsky S, Bonagura VR. Subcutaneous immunoglobulin infusion as an alternative to intravenous immunoglobulin. J Allergy Clin Immunol. 2003;112(3):630–3. doi: 10.1016/S0091-6749(03)01781-0.
    1. Gardulf A, Nicolay U, Asensio O. et al.Rapid subcutaneous IgG replacement therapy is effective and safe in children and adults with primary immunodeficiencies - a prospective, multi-national study. J Clinical Immunol. 2006;26:177–85. doi: 10.1007/s10875-006-9002-x.
    1. Weiler CR. Immunoglobulin therapy: history, indications, and routes of administration. Int J Dermatol. 2004;43:163–6. doi: 10.1111/j.1365-4632.2004.02236.x.
    1. Gardulf A, Andersen V, Björkander J. et al.Subcutaneous immunoglobulin replacement in patients with primary antibody deficiencies: safety and costs. Lancet. 1995;345:365–9. doi: 10.1016/S0140-6736(95)90346-1.
    1. Berger M. Subcutaneous immunoglobulin replacement in primary immunodeficiencies. Clin Immunol. 2004;112:1–7. doi: 10.1016/j.clim.2004.02.002.
    1. Gustafson R, Hammarström L. Subcutaneous immunoglobulin replacement therapy. Ellipse. 2002;18:45–8.
    1. Gardulf A. Immunoglobulin treatment for primary antibody deficiencies: advantages of the subcutaneous route. Bio Drugs. 2007;21(2):105–16.
    1. Gaspar J, Gerritsen B, Jones A. Immunoglobulin replacement treatment by rapid subcutaneous infusion. Arch Dis Child. 1998;79:48–51. doi: 10.1136/adc.79.1.48.
    1. Gardulf A, Björvell H, Andersen V. et al.Lifelong treatment with gammaglobulin to patients with primary antibody deficiencies: The patients' experiences of subcutaneous self-infusions and home therapy. J Adv Nurs. 1995;21:917–27. doi: 10.1046/j.1365-2648.1995.21050917.x.
    1. Sigstad HM, Stray-Pedersen A, Frøland SS. Coping, quality of life, and hope in adults with primary antibody deficiencies. Health Qual Life Outcomes. 2005;3:31. doi: 10.1186/1477-7525-3-31.
    1. Fasth A, Nyström J. Quality of life and health-care resource utilization among children with primary immunodeficiency receiving home treatment with subcutaneous human immunoglobulin. J Clin Immunol. 2008;28(4):370–8. doi: 10.1007/s10875-008-9180-9.
    1. Gardulf A, Nicolay U, Asensio O. et al.Children and adults with primary antibody deficiencies gain quality of life by subcutaneous IgG self-infusions at home. J Allergy Clin Immunol. 2004;114:936–42. doi: 10.1016/j.jaci.2004.06.053.
    1. Gardulf A, Nicolay U. Replacement IgG therapy and self-therapy at home improve the health-related quality of life in patients with primary antibody deficiencies. Curr Opin Allergy Clin Immunol. 2006;6:434–42. doi: 10.1097/01.all.0000246619.49494.41.
    1. Gardulf A, Borte M, Ochs HD, Prognostic factors for health-related quality of life in adults and children with primary antibody deficiencies receiving SCIG home therapy. 2008. pp. 81–88.
    1. Conference Report, Network of Rare Blood Disorder Organizations, Conference on Comprehensive Care for Rare Blood Disorders, February 3-5, 2006, Toronto, Ontario
    1. Abrahamsen TG, Sandersen H, Bustnes A. Home therapy with subcutaneous immunoglobulin infusions in children with congenital immunodeficiencies. Pediatrics. 1996;98:1127–31.
    1. Ware JE Jr, Sherbourne CD. The MOS 36-item short form health survey (SF-36). I. Conceptual framework and item selection. Med Care. 1992;30:473–83. doi: 10.1097/00005650-199206000-00002.
    1. Ware JE, Jr, Snow KK, Kosinski M. SF-36 health survey: manual and interpretation guide. Lincoln (RI): Quality-Metric Inc; 2000.
    1. Landgraf J, Abetz L, Ware JE. , Jr. The Child Health Questionnaire (CHQ), a user's manual. 2. Boston: The Health Institute; 1999.
    1. Gardulf A, Björvell H, Gustafson R. et al.The life situations of patients with primary antibody deficiency untreated or treated with subcutaneous gammaglobulin infusions. Clin Exp Immunol. 1993;92(2):200–4.
    1. Nicolay U, Kiessling P, Berger M. et al.Health-related quality of life and treatment satisfaction in North American patients with primary immunodeficiency diseases receiving subcutaneous IgG self-infusions at home. J Clin Immunol. 2006;26(1):65–72. doi: 10.1007/s10875-006-8905-x.
    1. Chapel HM, Spickett GP, Ericson D. et al.The comparison of the efficacy and safety of intravenous versus subcutaneous immunoglobulin replacement therapy. J Clin Immunol. 2000;20(2):94–100. doi: 10.1023/A:1006678312925.
    1. Singh-Grewal D, Kemp A, Wong M. A prospective study of the immediate and delayed adverse events following intravenous immunoglobulin infusions. Arch Dis Child. 2006;91(8):651–4. doi: 10.1136/adc.2005.078733.
    1. Kittner JM, Grimbacher B, Wulff W. et al.Patients' attitude to subcutaneous immunoglobulin substitution as home therapy. J Clin Immunol. 2006;26(4):400–5. doi: 10.1007/s10875-006-9031-5.
    1. Fasth A, Nyström J. Safety and efficacy of subcutaneous human immunoglobulin in children with primary immunodeficiency. Acta Paediatr. 2007;96(10):1474–8. doi: 10.1111/j.1651-2227.2007.00485.x.

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