Insights into Patient Experiences with Facilitated Subcutaneous Immunoglobulin Therapy in Primary Immune Deficiency: A Prospective Observational Cohort.

BACKGROUND: Immunoglobulin G replacement therapy (IgRT), intravenous (IV) and subcutaneous (SC) routes, is pivotal in treatment of primary immunodeficiencies (PID). In recent years, facilitated subcutaneous immunoglobulin (fSCIG), a combination of rHuPH20 and 10% IgG has emerged as a delivery method to combine advantages of both IV and SC. METHOD: In an observational prospective cohort, we investigated patient experience with fSCIG in PID patients from 5 PID centers for up to 12 months. We assessed the efficacy and safety of this treatment with patient/caregiver- and physician-reported indicators. Additionally, we analyzed patient treatment satisfaction (TSQM-9) and quality of life (QoL). RESULTS: We enrolled 29 patients (22 pediatric and 7 adults; 14 females and 15 males; (median: 15, min-max: 2-40.9 years) who initiated fSCIG as IgRT-naive (n = 1), switched from conventional rapid-push 10% SCIG (n = 6) or IVIG (n = 22). Among the participants, 19 (65%) exhibited antibody deficiencies, 8 (27%) combined immunodeficiencies, and 2 (7%) immune dysregulations. Remarkably, targeted trough immunoglobulin G levels were achieved under all previous IgRTs as well as fSCIG. No severe systemic adverse drug reactions were documented, despite prevalent local (%86.45) and mild systemic (%26.45) adverse reactions were noted with fSCIG. Due to mild systemic symptoms, 2 patients switched from fSCIG to 10% SCIG. The patient satisfaction survey revealed a notable increase at 2-4th (p = 0.102); 5-8th (p = 0.006) and 9-12th (p < 0.001) months compared to the baseline. No significant trends were observed in QoL surveys. CONCLUSION: fSCIG demonstrates admissable tolerability and efficacy in managing PIDs in addition to notable increase of patients' drug satisfaction with IgRT. The identified benefits support the continuation of this therapy despite the local reactions.

Mast cell activation syndrome: An up-to-date review of literature.

Mast cells are a subtype of white blood cells and are involved in the immune system. These cells contain many chemical substances called mediators, which are involved in the allergic response. The fact that mast cells play a role in many events that require urgent intervention, especially anaphylaxis, has led to a more detailed study of these cells. The diseases also caused by dysfunctions of mast cells have been examined in many circumstances. For instance, mast cell activation syndrome is known as an augmented number of cells due to decreased cell death, resulting in clinical symptoms affecting many systems. The main common symptoms include flushing, hypotension, urticaria, angioedema, headache, vomiting and diarrhea. Although the underlying mechanism is not yet clearly known, we aim to review the literature in a broad perspective and bring together the existing knowledge in the light of the literature due to the diversity of its involvement in the body and the fact that it is a little known syndrome.

Heavy Metal and Thiol/Disulfide in Children with Attention-Deficit and Hyperactivity Disorder.

Background: In the etiology of attention-deficit and hyperactivity disorder (ADHD), oxidative stress and heavy metal exposure are still controversial topics. In this study, our goal was to examine heavy metal levels and oxidative balance in newly diagnosed patients with ADHD and reveal whether heavy metal levels have an effect on the oxidation balance. Methods: The study included 35 patients with newly diagnosed ADHD and 31 healthy control groups of similar age and gender. Participants' parents or caregivers completed a semi-structured questionnaire regarding their children's breastfeeding and prenatal and postnatal smoking exposures. The levels of heavy metals lead (Pb), mercury (Hg), and cadmium were measured with inductively coupled plasma optical emission spectroscopy, and a unique automated spectrophotometric approach was used to quantify serum total thiol, native thiol, and disulfide quantities and ratios. Results: The rate of smoking during pregnancy was significantly higher in the ADHD group than in the control group (P = .030). Compared to the control group, the native and total thiol levels of children with ADHD were significantly higher (P < .001). Likewise, the ADHD group had significantly higher Hg levels compared to the control group (P = .002). Cadmium levels were substantially greater in the control group compared to the ADHD group (P < .001). However, there was no significant difference between Pb levels in the ADHD and the control group (P = .844). Conclusion: Exposure to Hg and prenatal smoking may contribute to the development of ADHD in childhood. In response to oxidative stress, the young brains of children with ADHD may enhance their antioxidant levels.

COVID-19 and cardiac complications: Myocarditis and multisystem inflammatory syndrome in children.

Coronavirus is an important pathogen causing disease in humans and animals. At the end of 2019, an investigation into an increase in pneumonia cases in Wuhan, Hubei Province, China, found that the cause was a new coronavirus. This disease, which spread rapidly across China and caused an outbreak worldwide, resulted in a pandemic. Although this virus has previously been referred to as 2019-nCoV, which causes coronavirus disease 2019 (COVID-19), later it was named severe acute respiratory syndrome coronavirus 2. Children were usually asymptomatic and rarely severely affected. In April 2020, reports from the United Kingdom indicated that children may have Kawasaki disease or a clinical condition similar to toxic shock syndrome. This clinical picture was later defined as multisystem inflammatory syndrome in children. Since then, similarly affected children as well as cases with other cardiac complications have been reported in other parts of the world. In this review, we aimed to evaluate COVID-19 in terms of cardiac involvement by reviewing the literature.

COVID-19 and vaccination in hereditary angioedema: Single center experience.

Like many microbial agents, Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) and its vaccination may increase the frequency and/or severity of attacks. We aimed to observe/evaluate patients with hereditary angioedema (HAE) followed by Sakarya University Research/Training Hospital pediatric allergy unit, Sakarya, Türkiye, for the effects of SARS-CoV-2 infection and COVID-19 vaccination. Ten HAE patients-3 males and 7 females-were evaluated retrospectively. Their mean age was 31.80 ± 19.15 (min. 12 - max. 66) years. Four of 10 patients were diagnosed with type 1 HAE and 6 with type 2 HAE. Two out of 6 patients (mother and daughter) diagnosed with type 2 HAE had angioedema attacks during COVID-19 disease. Six out of 10 HAE patients received different COVID-19 vaccines available in Türkiye up to third and fourth doses. There was no increase in COVID-19 vaccine-related attacks during, after 72 hours, and up to the year after vaccination. As a result, we consider it safe to administer inactivated and/or mRNA vaccines in our patients with HAE. In addition, catching SARS-CoV-2 infection was not always associated with disease exacerbation or activation.

Allergic Disease with Selective IgA Deficiency. Comment on Cinicola et al. The Allergic Phenotype of Children and Adolescents with Selective IgA Deficiency: A Longitudinal Monocentric Study. J. Clin. Med. 2022, 11, 5705.

I read the article titled 'The Allergic Phenotype of Children and Adolescents with Selective IgA Deficiency: A Longitudinal Monocentric Study' by Cinicola et al. with great interest [...].

Identification of Paroxysmal Nocturnal Hemoglobinuria-Type Bone Marrow Mast Cells.

Hematopoietic stem cells (HSCs) give rise to mast cells (MCs), and a relative increase in bone marrow (BM) MC is common in various BM failure (BMF) conditions. Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal HSC disorder, frequently associated with BMF, characterized by decreased expression of glycosylphosphatidylinositol membrane anchor for complement regulatory proteins. Eculizumab, a monoclonal antibody that blocks complement factor 5, successfully controls PNH symptoms. In this study, we, for the first time, demonstrated PNH-type culture-grown BM MC (c-BMMC) using flow cytometry in two BMF patients and monitored population size during eculizumab therapy. Further research may unravel the properties of PNH-type c-BMMC.

Omalizumab's efficacy and safety against chronic spontaneous urticaria.

I have read the article titled, "Efficacy and safety of omalizumab against chronic spontaneous urticaria: Real-world study from China", by Wang et al with great interest. Nevertheless, there are a couple of questions raised in my mind. And clarification of some aspects of the study will help the reader benefit and have a better understanding of the study.

Microbiota dysbiosis in hereditary angioedema patients.

I have read the article titled "Throat microbiota alterations in patients with hereditary angioedema" by Wang et al (2022) with great interest. This study examined the change in throat microbiota and its association with laryngeal edema (LE) attacks and attack severity in hereditary angioedema (HAE) patients. This study demonstrated the comparative richness of Bacteroidetes and Prevotellaceae in recent LE attacks and detected positive association between the attack severity scores and Bacteroidetes richness. Nevertheless, I have some questions and concerns about the methodological design of their study. For instance, in the article, the description of HAE and HAE patients is not exactly correct. I do not also agree with the authors on the effect of long-term prophylactic danazol use in HAE patients of this study. It is very important when or how the swab was obtained after the LE attack. The last, not the least, point now is what the authors suggest to improve this dysbiosis in these HAE patients. The discussion to elaborate these points in the study could be helpful and enlightening for readers and future research in this area.