Efficacy and tolerability of an argentine intravenous immunoglobulin in pediatric patients with primary immunodeficiency diseases.

Inmunoglobulina G Endovenosa UNC is a 5% liquid Argentine intravenous immunoglobulin obtained from South American donors. This prospective trial was designed to evaluate if the product meets the minimal efficacy requirement of the US Food and Drug Administration of <1 serious infection/subject/year as well as its safety in pediatric patients with Primary Immunodeficiency Diseases. Thirty patients under the age of 18, with well-defined Primary Immunodeficiency Diseases received Inmunoglobulina G Endovenosa UNC (330-700 mg/kg every 3-4 weeks) for 6 months. Vital signs, laboratory abnormalities, adverse events and viral tests were assessed to evaluate safety. Two serious infections occurred (pneumonia and bacteriemia). The estimated infection rate was 0.114 serious infection/subject/year (95% CI, 0.003-0.2277). Minor adverse events occurred in 5.5% of infusions; fever and headache were the most common. Neither severe adverse events, nor abnormal laboratory values were observed. All viral assessments were negative. Inmunoglobulina G Endovenosa UNC meets the minimal efficacy requirement of the US Food and Drug Administration for pediatric Primary Immunodeficiency Diseases patients and showed efficacy and safety data comparable with other data published.

Thirteen years of culture-positive M. bovis-BCG infection in an IL-12Rbeta1 deficient patient: treatment and outcome.

Patients with mutations in the IFNgamma/IL-12 pathway show an exquisite susceptibility to mycobacterial diseases. An IL-12Rbeta1 deficient patient with impaired intestinal absorption suffered from a 13 year culture-positive Mycobacterium bovis-BCG infection with acquired multidrug resistance. A combined parenteral and enteral anti-mycobacterial treatment, including recombinant IFNgamma, helped to clear his infection.

[Kawasaki disease. Immunological evaluation of 26 cases]. / Enfermedad de Kawasaki. Evaluación inmunológica de 26 casos.

Kawasaki disease (KD) is an acute febrile vasculitis of childhood, characterized by multiple clinical and biochemical features of inflammation with special involvement of the heart. The activation of lymphocytes and monocytes/macrophages and their secreted soluble products, cytokines, play a central role in the pathogenesis of the disease. In this study we performed immunologic studies in 26 patients with KD. No constant pattern of serum levels of IgG, IgA, IgM, C3 and C4 fractions of complement measured by Nephelometry and neither autoantibodies, FAN and ANCA performed by indirect immunofluorescence were found in 22 patients in the acute stage. Variable percentages of CD3, CD4, CD8, CD20, CD56 and DR in peripheral mononuclear cells specifically stained and analysed by flow cytometry were seen among 25 patients in the acute stage. CD25 was elevated in 17/25 cases. Serum levels of TNF alpha performed by ELISA in 12 patients in acute stage were low. Intracellular cytokines such as TNF alpha, IL1 beta, IL2 and IFN gamma were measured in peripheral mononuclear cells of 15 patients in acute stage, in 5th and 30th days after gammaglobulin treatment, utilizing specific staining and analysis by flow cytometry showing no sole characteristic profile. In 2 patients there was an elevated percentage of TNF alpha and IL1 beta in monocytes during the convalescent stage; both had coronary sequelae. More research on this question is needed. In conclusion, immunologic studies showed an heterogeneous profile and no laboratory finding was registered in the acute stage that could be used as predictive factor of cardiovascular involvement.

Enfermedad de Kawasaki. Evaluación inmunológica de 26 casos. / [Kawasaki disease. Immunological evaluation of 26 cases]

Kawasaki disease (KD) is an acute febrile vasculitis of childhood, characterized by multiple clinical and biochemical features of inflammation with special involvement of the heart. The activation of lymphocytes and monocytes/macrophages and their secreted soluble products, cytokines, play a central role in the pathogenesis of the disease. In this study we performed immunologic studies in 26 patients with KD. No constant pattern of serum levels of IgG, IgA, IgM, C3 and C4 fractions of complement measured by Nephelometry and neither autoantibodies, FAN and ANCA performed by indirect immunofluorescence were found in 22 patients in the acute stage. Variable percentages of CD3, CD4, CD8, CD20, CD56 and DR in peripheral mononuclear cells specifically stained and analysed by flow cytometry were seen among 25 patients in the acute stage. CD25 was elevated in 17/25 cases. Serum levels of TNF alpha performed by ELISA in 12 patients in acute stage were low. Intracellular cytokines such as TNF alpha, IL1 beta, IL2 and IFN gamma were measured in peripheral mononuclear cells of 15 patients in acute stage, in 5th and 30th days after gammaglobulin treatment, utilizing specific staining and analysis by flow cytometry showing no sole characteristic profile. In 2 patients there was an elevated percentage of TNF alpha and IL1 beta in monocytes during the convalescent stage; both had coronary sequelae. More research on this question is needed. In conclusion, immunologic studies showed an heterogeneous profile and no laboratory finding was registered in the acute stage that could be used as predictive factor of cardiovascular involvement.

Progressive multifocal leukoencephalopathy in a patient with hypogammaglobulinemia.

We describe a child with congenital hypogammaglobulinemia that was diagnosed at 13 months of age. When he was 4 years old, gait disturbances began. The main neurological manifestations were progressive spastic tetraparesis and intellectual and speech deterioration. No infectious agent was identified. A magnetic resonance imaging scan of the central nervous system revealed periventricular demyelinating areas in the frontal, temporal, and parietal lobes with cortical atrophy. Stereotactic brain biopsy confirmed the diagnosis of progressive multifocal leukoencephalopathy caused by JC virus. He was treated with intravenous and intraventricular cytarabine and interferon-alpha, and there was clinical improvement. We emphasize the need for brain biopsy as soon as a neurological complication is suspected in patients with congenital hypogammaglobulinemia for whom cerebrospinal cultures or polymerase chain reaction analyses are negative.