Long-term safety of facilitated subcutaneous immunoglobulin treatment in pregnant women with primary immunodeficiency diseases: results from a registry study.

Aim: Clinical outcomes of women who become pregnant during/after facilitated subcutaneous immunoglobulin (fSCIG) treatment are not well characterized. Materials & methods: This noninterventional, prospective, open-label, post authorization, pregnancy registry study assessed safety outcomes in mothers with primary immunodeficiency diseases who had ever received fSCIG before/during pregnancy and their infants (n = 7). Enrolled women received alternative treatment (arm 1: n = 2) or continued fSCIG (arm 2: n = 7) during pregnancy. Results: No treatment-related adverse events (AEs)/serious AEs (SAEs) were reported. 13 AEs occurred in mothers, including two SAEs (thrombocytopenia, pre-eclampsia; arm 2). A total of 17 AEs occurred in infants, including two SAEs (cleft lip, talipes calcaneovalgus; arm 2) with normal growth/development. Conclusion: Findings provide limited but useful safety data regarding women who received fSCIG before/during pregnancy and the growth/development of their infants. Clinical Trial registration: NCT02556775 (ClinicalTrials.gov); EUPAS5798.

Alveolar Proteinosis, Infectious Complications and Monocytopenia Associated with GATA2 Deficiency.

The authors describe a case of a rare disorder associated with GATA 2 deficiency, which is an important hematopoietic transcription factor for the development of monocytes. The clinical hallmarks of GATA2 deficiency include monocytopenia, cellular immunodeficiency with a resultant marked susceptibility to infections (mycobacterial, fungal, and viral), predisposition to myelodysplasia, alveolar proteinosis of the lungs and congenital lymphoedema. These features overlap with other genetic and acquired syndromes. It is therefore important to establish a genetic diagnosis early. The only known curative treatment is hematopoietic stem cell transplantation and is integral to the appropriate management of these patients.

Detection of anti-infliximab antibodies in Slovak IBD patients and its costs saving effect.

OBJECTIVE: Management chronic inflammatory bowel disease (IBD) patients is associated with diagnosis, targeted treatment and and individual approach. There is a group of patients which loss the response to the biologic treatment caused by insufficient levels of biologics or positive antibodies against these drugs. This study was aimed to determine the prevalence of patients with positive antibodies against the biological treatment and the costs saving probabilities of the antibodies detection during the treatment. STUDY DESIGN: This retrospective study was based on examination of 183 IBD patients' sera (72 with Crohn's disease (CD) and 111 ulcerative colitis (UC)) treated with infiliximab. METHODS: Circulating serum infliximab concentrations and anti-infliximab antibodies (ATI) were quantified by ELISA methods. Costs associated with the treatment were analysed from the data of General Health Insurance Company, Slovakia. RESULTS: The average infliximab concentrations in groups of CD were 2.9 µg/mL, 38.9% of samples had a concentration ≤1 µg/mL. Group with UC had average infliximab levels of 3.19 µg/mL, 32.4% bellow ≤1 µg/mL. Positive ATI levels were detected in 52 patients, in 28 patients with CD (38.8%) and 24 patients with UC (21.6%). The average values of the antibodies were 387.75 U/ml in CD and 391.94 U/ml in UC group. More than 28% IBD patients were positive for ATI. After application of the results to the database of all IBD patients, finishing of the treatment with ATI could lead (after considering the ATI quantification costs) to possible annual savings of more than €2 million in Slovakian health-care system. CONCLUSIONS: Monitoring of infliximab and antibodies against infliximab and anti-TNF-α biologics may help optimize treatment strategies and costs for biological treatment.