ABSTRACT
BACKGROUND: Systemic juvenile idiopathic arthritis (sJIA) is a complex disease with an autoinflammatory component of unknown etiology related to the innate immune system. A major role in the pathogenesis has been ascribed to proinflammatory cytokines like interleukin-6 (IL-6), and effective drugs inhibiting their signaling are being developed. This study evaluates sJIA patients treated with the IL-6 inhibitor tocilizumab (TCZ) concerning clinical response rate, disease course and adverse effects in a real-life clinical setting. METHODS: In 2009 a clinical and research consortium was established, including an online registry for autoinflammatory diseases (AID) ( https://aid-register.de ). Data for this retrospective TCZ study were documented by 13 centers. RESULTS: From 7/2009 to 4/2014, 200 patients with sJIA were recorded in the AID-registry. Out of these, 46 (19 m, 27 f, age 1-18 years) received therapy with TCZ. Long term treatment (median 23 months) has been documented in 24/46 patients who were evaluated according to Wallace criteria (active disease 6/24, inactive disease 5/24, remission 13/24 cases). Under observation co-medication were used in 40/46 cases. Adverse events were reported in 11/46 patients. The clinical response rate (no clinical manifestation, no increased inflammation parameters) within the first 12 weeks of treatment was calculated to be 35%. CONCLUSION: Out of 200 sJIA children reported in the German AID-registry, 46 were treated with TCZ, showing a clinical response rate of 35% during the first 12 weeks, and inactive disease and/or remission under medication in 75% after one year. Adverse events were seen in 24% and severe adverse events in 4%. TRIAL REGISTRATION: The AID-Registry is funded by the BMBF (01GM08104, 01GM1112D, 01GM1512D).
Subject(s)
Antibodies, Monoclonal, Humanized/therapeutic use , Arthritis, Juvenile/drug therapy , Interleukin-6/antagonists & inhibitors , Adolescent , Antibodies, Monoclonal, Humanized/adverse effects , Child , Child, Preschool , Female , Germany , Humans , Male , Registries , Remission Induction , Retrospective Studies , Treatment Outcome , Young AdultABSTRACT
In a nation-wide registration project 38 incident cases of juvenile dermatomyositis were collected in Germany over a 2-year-period. Diagnostic methods as well as the primary treatment for these patients were recorded. Detailed information was available for 25 of these patients. Diagnostic as well as therapeutic decisions varied widely. Steroids were used in almost all of the 25 patients either as oral or as parenteral pulse therapy, additional immunosuppressive drugs were used in 52%. We plan to establish national consensus recommendations for diagnostic and therapeutic standards in JDM. Due to the rarity of JDM clinical trials will have to be performed on an international basis.
Subject(s)
Dermatomyositis/diagnosis , Dermatomyositis/epidemiology , Registries , Administration, Oral , Adolescent , Adrenal Cortex Hormones/administration & dosage , Child , Child, Preschool , Consensus , Cross-Sectional Studies , Dermatomyositis/drug therapy , Drug Therapy, Combination , Female , Germany , Humans , Immunosuppressive Agents/administration & dosage , Incidence , Infusions, Intravenous , Male , Practice Guidelines as Topic , Pulse Therapy, DrugABSTRACT
Antigenic beta-lactoglobulin and alpha-casein were measured in the sera of 45 formula-fed infants of 31 to 41 weeks of gestation at 5 days and at 10 days of age. Quantitation was performed by a sensitive ELISA inhibition assay. On day 5 of life antigenic lactoglobulin was detected in 14 of 19 infants of less than 37 weeks gestation, but in only one of 10 infants of more than 36 weeks gestation. On day 10 of life the sera of all infants contained antigenic lactoglobulin. In contrast, on day 5 antigenic casein was present in four of 17 infants of less than 37 weeks gestation, but in 10 of 12 infants of the more mature group. On day 10 casein was detected in seven of 28 infants, with no difference between groups; anti-casein was found in eight of 12 infants. Infants of less than 37 weeks gestation have different absorption patterns than more mature infants do. "Gut closure" is an unlikely explanation for these findings.
