Facilitated subcutaneous immunoglobulin treatment patterns in pediatric patients with primary immunodeficiency diseases.

Aim: This retrospective study investigated real-world hyaluronidase-facilitated subcutaneous immunoglobulin (fSCIG) treatment patterns in pediatric patients with primary immunodeficiency diseases (PIDs) in Poland. Methods: Clinical and demographic information, fSCIG treatment parameters and clinical outcomes were extracted from medical records of 28 participants (aged ≤18 years) with PIDs who received fSCIG. Results: 18 participants (64.3%) started fSCIG with a ramp-up (median duration: 35.5 days). 27 patients (96.4%) were administered fSCIG every 4 weeks and one patient every 3 weeks. 25 patients (89.3%) used one infusion site. No serious bacterial infections occurred. Conclusion: Data support the feasibility of administering fSCIG to children and adolescents with PIDs every 3-4 weeks using a single infusion site and indicate flexibility in modifying fSCIG infusion parameters. Clinical Trial Registration: NCT04636502 (ClinicalTrials.gov).

Antibodies, also known as immunoglobulins, are proteins that are made by the immune system to help fight infections. In primary immunodeficiency diseases (PIDs), part of the immune system may be missing or not working properly. This study looked at the use of an antibody treatment called hyaluronidase-facilitated subcutaneous immunoglobulin (or fSCIG) in Polish children aged 18 years or younger with PIDs. Information on patients, their disease, how fSCIG was being used and how patients responded to treatment was taken from medical records. Out of 28 patients, 18/28 (64.3%) had their fSCIG dose slowly increased, which took an average of 35.5 days. Overall, 27/28 patients were treated with fSCIG every 4 weeks (96.4%), and 25/28 patients used one place to inject fSCIG (89.3%). No serious infections caused by bacteria happened during the study. The study results suggest that children with PIDs could be treated every 3 to 4 weeks with fSCIG, and that flexibility in how fSCIG is injected may offer options suited to individual patients.

Subcutaneous immunoglobulin 20% (Ig20Gly) treatment regimens in pediatric patients with primary immunodeficiencies - real-world data from the IG TATRY study.

BACKGROUND: Subcutaneous administration of immunoglobulins is associated with fewer systemic adverse events and easier infusion compared to intravenous administration. Ig20Gly is a 20% immunoglobulin formulation effective and safe in patients with primary immune deficiency diseases (PIDDs). Real-world data are scarce, therefore our study aimed to examine the real-life treatment regimen and clinical outcomes of Ig20Gly in Polish children with PIDDs. RESEARCHDESIGN: We retrospectively analyzed the medical documentation of 75 pediatric patients aged 0-17 years (mean 9.9) who received Ig20Gly (Cuvitru®; Baxalta US, Inc.; part of Takeda, MA, U.S.A.). RESULTS: The median exposure to treatment of the study population was 22.3 months. At the end of the study, 59 (78.7%) were still on Ig20Gly. The median monthly dose was 0.40 g/kg. The median treatment interval was 7.7 days. Most patients (96%) used one infusion site. The median infusion rate increased with patient age. The median IgG level in the study population, 8.0 g/L, was stable. There was one case of serious bacterial infection. CONCLUSION: This is the largest, long-term real-world study to date on the treatment patterns of Ig20Gly in pediatric patients with PIDDs. The results of this study support the feasibility and tolerability of Ig20Gly usage in PIDD patients across the pediatric age spectrum. TRIAL REGISTRATION: The trial is registered at ClinicalTrials.gov (NCT04636502).

Selected CC and CXC chemokines in children with atopic asthma.

INTRODUCTION: There are only limited data on CC and CXC chemokines regulation in children with asthma. AIM: We compared the serum profile of selected CC and CXC chemokines in patients with atopic asthma and healthy children. MATERIAL AND METHODS: Serum concentration of CC chemokines RANTES, MCP-1, and CXC chemokines IP-10, MIG, IL-8, RANTES was measured using cytometric bead array in 44 children with atopic asthma and 17 healthy subjects. RESULTS: The concentration of RANTES was significantly higher and the MIG level was lower in all children with asthma as compared to their control counterparts. We observed increased RANTES and decreased MIG levels also in patients with stable asthma when compared with children in the control group. The IP-10 concentration was similar between the whole asthma group and healthy controls, while significantly increased levels of this chemokine in acute asthma have been observed when compared to stable asthma. For MCP-1 and IL-8, the serum concentration was similar in all compared groups. The MIG concentration correlated positively with IP-10, IL-8, and CRP levels and negatively with the eosinophil count. A negative correlation between the IP-10 and eosinophil count and a negative correlation between FEV1 and IP-10 were found. CONCLUSIONS: An increased serum RANTES level in children with asthma may result in enhancement of Th2 lymphocyte recruitment into the airway. A decreased expression of Th1 chemokine MIG in children with stable asthma may contribute to a diminished antagonizing effect on Th2 cytokine production and hence intensify Th2 predominance. An increased IP-10 level in children during an asthma attack suggest that this chemokine is a serological marker of disease exacerbation.

Atopic allergy and chronic inflammation of the oral mucosa in a 3-year-old boy after heart transplantation - diagnostic and therapeutic difficulties.

In recent years, we have been observing an increased proportion of atopic diseases in children after solid organ transplantation. The pathogenesis of post-transplantation allergy is not completely understood and probably involves several factors, including immunosuppressive therapy. In this paper we present a case of 3-year old boy, after orthotopic heart transplantation at 6 months of age, with symptoms of food allergy associated with atopic dermatitis and changes in the orofacial area. The mentioned symptoms and elevated levels of total and specific IgE occurred with a year of transplant. Because of failure to achieve remission after typical allergy therapy we suspected that the reason of allergy in this case can be immunosuppressive therapy.

[Assessment of the natural history and clinical presentation of acetonemic vomiting]. / Ocena przebiegu i obrazu klinicznego wymiotów acetonemicznych u dzieci.

INTRODUCTION: Ketosis in children may result from physiological adaptation to situations like fasting, fat-rich diet, straining physical activity, as well as from serious endocrine or metabolic disorders. The most frequently diagnosed cause of ketoacidosis are states of acetonemia and acetonuria with vomiting, during airways infections. GOAL: Assessment of the natural history and clinical presentation of acetonemic vomiting in children. PATIENTS AND METHODS: 85 children from 18 months to 12 years of age with acetonemic vomiting were incorporated in this study. Detailed anamnesis, clinical examination, and chosen laboratory parameters were analyzed. RESULTS: In 18% of the children a familial pattern of the disease was observed, 75% of the parents declared that their children had fat-rich meals on a regular basis, in 47% there was a tendency to recurrent respiratory tract. The most frequently observed symptoms were incoercible vomiting with nausea (100%), abdominal pain (87%), headaches (35%) and febrile states (62%). Ketosis triggers were: infections with insufficient fluid and food intake (68%), and child overfeeding with fat-rich products (23%). Observed biochemical disturbances were ketosis (mean J3-hydroxybutyric acid serum concentration--1.03 mmol/l, SD +/- 0.83), acetonuria, hypoglycemia (15%), metabolic acidosis (17%) and dyselectrolytemia (14%). The treatment of the children consisted in intravenous and oral rehydration, managing acid-base and electrolyte disturbances. CONCLUSION: In some children acetonemic vomiting is recurrent, and thus prophylactic management is im- portant in children who are at risk.

[RSV infection course in infants and young children during hospitalization]. / Przebieg zakazen RSV u hospitalizowanych niemowlat i malych dzieci.

Respiratory syncytial virus (RSV) infection is a widespread among the youngest group of pediatric patients, especially in the young infant, when the immune system is just being formed. A lot of children up to 5 year-old suffer infection many times, not only because of easy pathogen transfer, but also primary incidence of disease doesn't give permanent immunity. This study retrospectively analyses a group of children with confirmed during epidemic period RSV infection. Particular attention was paid to the infection risk factors, varied clinical manifestations of infection, abnormal additional studies and accessible prophylactic action. The treatment and correlation between RSV, recurrent wheezing and asthma were discussed.