Gammaglobulina subcutánea en inmunodeficiencia común variable. Primera experiencia en España / Subcutaneous gammaglobulin in common variable immunodeficiency. First experience in Spain

Introducción y objetivo: la autoadministración semanal de gammaglobulina subcutánea (GGSC) domiciliaria es una alternativa en el tratamiento de las inmunodeficiencias primarias con déficit de producción de anticuerpos. El objetivo es comparar y evaluar la eficacia, la seguridad, la calidad de vida y el coste anual de GGSC y gammaglobulina intravenosa (GGIV) en nuestro medio. Material y métodos: estudio observacional y descriptivo de los pacientes pediátricos con inmunodeficiencia común variable (IDCV) que reciben GGSC en nuestro centro (noviembre 2006-abril 2008), en comparación con el último año de GGIV. Resultados: se incluyó a 11 pacientes afectos de IDCV. Mediana de edad, 15 años. Mediana de IgG plasmática valle con GGIV, 622 mg/dl. En los pacientes en que se mantuvo o se disminuyó la dosis de GGSC respecto a la de GGIV previa (7/8), la mediana de IgG fue 850 mg/dl (p<0,0005). Tasa de infección/paciente/año de 2,22, sin diferencias estadísticamente significativas respecto a GGIV (p=0,212). Se produjeron 58 reacciones adversas (45 locales, 13 sistémicas) en 41/506 infusiones. Las reacciones adversas locales más frecuentes fueron dolor y picor y como sistémicas, la cefalea. Todos los pacientes refirieron una mejora en su calidad de vida. El tratamiento con GGSC supuso un importante ahorro económico. Conclusiones: la terapia subcutánea es una alternativa coste-efectiva a la GGIV con una eficacia similar y un aumento de calidad de vida en los pacientes con IDCV. Las concentraciones plasmáticas valle de IgG obtenidas son iguales o mayores. Las reacciones adversas locales son frecuentes, pero leves y autolimitadas (AU)

Introduction and aim: Weekly home-based subcutaneous immunoglobulin (SCIg) therapy is an alternative to intravenous immunoglobulin (IVIg) in the treatment of patients with primary antibody deficiencies. The objective of this study was to investigate the efficacy, safety, related quality of life and cost effectiveness of SCIg in our area. Materials and methods: Observational and descriptive study including paediatric patients with common variable immunodeficiency (CVID) receiving SCIg in our hospital (November 2006 to April 2008). Obtained data were compared with those from the last year with IVIg. Results: Eleven patients with CVID were included. Median age was 15 years. The median trough serum IgG level was 622mg/dl with IVIg. In patients in whom the SCIg dose was maintained or reduced compared to IVIg, the median trough serum IgG level was 850mg/dl (p<0.0005). Annual rate of infection was 2.22 per patient-year, without significant differences to IVIg (p=0.212). There were 58 treatment-related adverse events (AE) reported with SCIg (45 local AE and 13 systemic AE). The most frequent treatment-related adverse event was infusion-site reaction. Switching to home-based subcutaneous IgG treatment led to significant improvements in quality of life and substantial cost savings. Conclusions: We conclude that subcutaneous administration of 16% SCIg is a safe and cost-effective alternative to IVIg for replacement therapy of primary antibody deficiencies. Median trough serum IgG levels were higher with SCIg. Local AE were common but mild and the incidence decreased over time. Quality of life is significantly improved (AU)

Selective high dose gamma-globulin treatment in Kawasaki disease: assessment of clinical aspects and cost effectiveness.

BACKGROUND: High-dose intravenous gamma-globulin (IVGG) plus aspirin (ASA) treatment is effective in preventing coronary artery complications in acute Kawasaki disease (KD). However, gamma-globulin is very expensive, especially in Japan. Furthermore the indication for IVGG treatment and the optimal dose of gamma-globulin remain controversial. OBJECTIVES: To examine these two issues, we used Harada's scoring system to investigate whether a single 2 g/kg dose therapy has any advantage over the 5 day 400 mg/kg per day therapy. METHODS: We studied 203 patients with KD who had no coronary artery complications on admission. Of these, 145 patients scored 4 or more on Harada score within the first 9 days of illness and were treated with IVGG treatment. Using a random number table, 72 patients were selected to receive a single 2 g/kg dose (2 g group), while the remaining 73 patients were treated with 400 mg/kg per day for 5 consecutive days (400 mg group). Those who had a Harada score of three or less received no IVGG (non-IVGG group) treatment (58 patients). RESULTS: The incidence rate of coronary artery complications in the 2 g group was significantly lower than in the 400 mg group. The duration of high fever, positive duration of C-reactive protein and the number of hospital days in the 2 g group were each significantly shorter than in the 400 mg group. The total medical expense in the 2 g group was significantly lower than in the 400 mg group. There were no coronary artery complications in the non-IVGG group. CONCLUSIONS: It was found to be clinically more effective and more cost effective to select a patient by Harada's scoring system and, where a score of four or more was obtained, to administer a single 2 g/kg intravenous dose of gamma-globulin for acute KD.

A comparison of the patient-borne costs of therapy with gamma globulin given at the hospital or at home.

The major aim of this study was to estimate and compare the patient-borne costs of lifelong subcutaneous gamma globulin therapy at the hospital and at home. Thirty patients were included and the data were collected with a questionnaire. The introduction of self-therapy at home reduced the total yearly costs by approximately 50% and the out-of-pocket expenses for the patients by 85%.