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A highly concentrated (20%) immunoglobulin (Ig)G preparation for subcutaneous administration (IGSC 20%), would offer a new option for antibody replacement therapy in patients with primary immunodeficiency diseases (PIDD). The efficacy, safety, tolerability and pharmacokinetics of IGSC 20% were evaluated in a prospective trial in Europe in 49 patients with PIDD aged 2-67 years. Over a median of 358 days, patients received 2349 IGSC 20% infusions at monthly doses equivalent to those administered for previous intravenous or subcutaneous IgG treatment. The rate of validated acute bacterial infections (VASBIs) was significantly lower than 1 per year (0·022/patient-year, P < 0·0001); the rate of all infections was 4·38/patient-year. Median trough IgG concentrations were ≥ 8 g/l. There was no serious adverse event (AE) deemed related to IGSC 20% treatment; related non-serious AEs occurred at a rate of 0·101 event/infusion. The incidence of local related AEs was 0·069 event/infusion (0·036 event/infusion, when excluding a 13-year-old patient who reported 79 of 162 total related local AEs). The incidence of related systemic AEs was 0·032 event/infusion. Most related AEs were mild, none were severe. For 64·6% of patients and in 94·8% of IGSC 20% infusions, no local related AE occurred. The median infusion duration was 0·95 (range = 0·3-4·1) h using mainly one to two administration sites [median = 2 sites (range = 1-5)]. Almost all infusions (99·8%) were administered without interruption/stopping or rate reduction. These results demonstrate that IGSC 20% provides an effective and well-tolerated therapy for patients previously on intravenous or subcutaneous treatment, without the need for dose adjustment. [ABSTRACT FROM AUTHOR]

The Langerhans cell histiocytosis (LCH) in children is relatively rare and the long-term analysis of therapy results has not been done yet in Hungary. The aim of this study was to investigate the incidence, clinical features, prognostic risk factors, and treatment results of children's LCH in Hungary in a 20-year period. Children less than 18 years of age with newly diagnosed LCH in Hungary were entered in this study. Clinical data of all children with LCH were reported to the National Childhood Cancer Registry in Hungary from 1981 to 2000. The clinical files were collected and abstracted for information regarding age at diagnosis, gender, disease characteristics, treatment, and outcome of treatment. Median follow-up duration of surviving patients is 10.98 years. Between January 1981 and December 2000, 111 children under 18 years of age were newly diagnosed with LCH in Hungary. The annual incidence of LCH in children younger than 18 years of age was 2.24/million children. The male–female ratio was 1.36:1; the mean age was 4 years 11 months. Thirty-eight children had localized disease and in 73 cases systemic dissemination was found already at the time of diagnosis. Twenty-two patients were treated only by local surgery, 7 by surgery with local irradiation, and 5 children got only local irradiation. In 2 cases remission was achieved with local steroid administration. Seventy-five patients received chemotherapy. In the 20 years of the study 14 children died, 9 due to the progression of the disease. Sixteen patients had relapse with a mean of 2.16 ± 1.29 years after the first diagnosis. Three patients with relapse got chemotherapy generally used in lymphoma and remission was achieved. The overall survival of all patients (n = 111) was 88.3 ± 3.1% at 5 years and 87.3 ± 3.2% at 10 and 20 years. Childhood LCH is a well-treatable disease and the survival rate is high. Even disseminated diseases have a quite good prognosis in childhood. [ABSTRACT FROM AUTHOR]

The article presents a case study of a 52-year-old woman with deep-vein thrombosis (DVT). Rheumatoid arthritis (RA) was diagnosed and methotrexate was initiated, and later due to permanent disease activity leflunomide was administered. However, the patient developed further complications and finally plasmapheresis synchronized with intravenous Ig and cyclophosphamide was subsequently administered and the vasculitis and necrosis went into remission.

Introduction L'immunoglobuline humaine sous-cutanée à 20 % (Ig20Gly) est une préparation liquide d'immunoglobuline humaine G (IgG) hautement purifiée et prête à l'emploi pour une administration sous-cutanée. Nous rapportons ici, les analyses finales d'une étude de phase 2/3 sur l'IGSC à 20 % chez des patients âgés de ≥ 2 ans en Europe souffrant de déficits immunitaires primitifs (DIP). Patients et méthodes Dans Epoch 1, une IgG à 16 % par voie sous-cutanée ou une IgG à 10 % par voie intraveineuse a été administrée à des doses de pré-étude pendant 3 mois. Dans Epoch 2, l'Ig20Gly a été administrée une fois par semaine pendant 12 mois aux doses utilisées durant Epoch 1. Le critère de jugement principal était le taux d'infections bactériennes graves validées (VASBI). Résultats Quarante-neuf patients (âgés de 2–67 ans) ont débuté l'étude ; 1 patient est sorti d'étude pour cause de grossesse dans Epoch 1 ; 45 patients ont terminé l'étude jusqu'à la fin de Epoch 2 (arrêts dus à des douleurs locales [ n = 1] ; raisons personnelles [ n = 2]). Durant le traitement par Ig20Gly (n = 48), 1 VASBI (pneumonie, d'une sévérité modérée) a été rapportée ; le taux d'infections était de 4,38/patient-année. Aucun événement indésirable grave (EI) n'a été considéré comme étant lié à un quelconque traitement. Durant le traitement par Ig20Gly, 121 des 2 349 (5,2 %) perfusions ont été associées à ≥ 1 EI local lié au traitement (chez 17/48 [35 %] patients)—tous étaient d'une sévérité légère à modérée. Aucun EI systémique grave lié au traitement n'a été rapporté, et 99,8 % des perfusions ont été dispensées sans interruption, arrêt ou ralentissement du débit (médiane sites/perfusion = 2 ; intervalle 1–5). Conclusion L'Ig20Gly est un traitement efficace et bien toléré, n'ayant pas nécessité d'ajustement de la dose initiée durant la période de pré-étude. [ABSTRACT FROM AUTHOR]