Quality of Life Evaluation in Saudi Arabian Pediatric Patients with Primary Immunodeficiency Diseases Receiving 20% Subcutaneous IgG Infusions at Home.

BACKGROUND AND AIMS: Subcutaneous immunoglobulin (SCIG) home infusion is widely used as an alternative to intravenous immunoglobulin (IVIG). This study aimed to determine the quality of life (QoL) of patients with primary immunodeficiency (PID) after switching to home-based SCIG. METHODS: In this prospective open-label single-center study, QoL was determined using the validated Arabic version of the Child Health Questionnaire at baseline and 3 and 6 months after switching from IVIG to SCIG. RESULTS: Twenty-four patients were recruited from July 2018 to August 2021, including 14 females and 10 males. The median age of the patients was 5 years (range, 0-14 years). The patients' diagnoses included severe combined immunodeficiency, combined immunodeficiency, agammaglobulinemia, Omenn syndrome, immunodysregulation, hyper-IgE syndrome, common variable immunodeficiency, and bare lymphocyte syndrome. The median duration on IVIG before inclusion was 40 months (range, 5-125 months). The QoL score showed a significant improvement in the patients' global health at 3 and 6 months compared with those at baseline and a significant improvement in the patients' general health at 3 and 6 months compared with that at baseline. The mean baseline serum IgG trough level was 8.8 ± 2.1 g/L. The mean serum IgG level was significantly higher on SCIG at both 3 and 6 months (11.7 ± 2.3 and 11.7 ± 2.5 g/L, respectively). CONCLUSIONS: This is the first study involving an Arab population to show improvement in the QoL of patients with PID after switching from hospital-based IVIG to home-based 20% SCIG.

A twenty-two-year experience with Hymenoptera venom immunotherapy in a US pediatric tertiary care center 1996-2018.

BACKGROUND: The rate of systemic reactions (SRs) to venom immunotherapy (VIT) in children has not been well evaluated. OBJECTIVE: To evaluate the rate of SRs to VIT in pediatric patients age 5 to 18 years who were treated with a standard protocol. METHODS: A retrospective chart review was conducted to identify patients who received VIT at Boston Children's Hospital from 1996 through 2018. Information on venom testing, severity of reaction to insect field sting, and SRs to VIT were retrieved. RESULTS: A total of 78 patients were included. Most had moderate to severe reactions to insect sting before VIT. The rate of SRs was 0.2% of injection visits, occurring in 9% of patients. The SRs from VIT were mild (mostly grade 1 and some grade 2), and no grades 3, 4, or 5 reactions were seen. Male sex was a significant risk factor for moderate to severe reactions to insect sting. Positive testing to vespinae was seen in 98.7% of patients, and none had exclusive sensitivity to honeybee. The severity of the initial, pre-VIT insect sting reactions in our patients did not correlate with the occurrence of SRs from VIT. Twenty-seven percent of the patients were subsequently stung while on VIT. Only 1 patient (5%) had a mild SR, while all others had only local or no reaction at all. CONCLUSION: In the largest US study evaluating the safety of VIT in children, SRs to VIT were mild, and none required epinephrine. Male sex was significantly associated with higher risk of moderate to severe reactions to insect sting. Larger multicenter studies are needed to further evaluate the rate of SRs to VIT in pediatric patients.

The use of adjunctive therapies during oral immunotherapy: A focus on biologics.

Oral immunotherapy (OIT), thus far, is the most evaluated therapeutic approach for food allergy. However, OIT is not known to lead to a cure, and it carries a risk for allergic reactions. Adjunct therapies to OIT are currently being investigated to evaluate their effect on safety and outcome. Of these therapies, omalizumab is the most evaluated biologic. There is mounting evidence that omalizumab is effective in inducing rapid desensitization of OIT in both single-food and multiallergen OIT, while diminishing the rate of adverse reactions. Evaluation of other adjunct biologics, such as dupilumab and bacterial therapy, is underway.

Biologics and Novel Therapies for Food Allergy.

Food allergy is a significant public health burden affecting around 10% of adults and 8% of children. Although the first peanut oral immunotherapy product received Food and Drug Administration approval in 2020, there is still an unmet need for more effective therapeutic options that minimize the risk of anaphylaxis, nutritional deficiencies, and patient's quality of life. Biologics are promising modalities, as they may improve compliance, target multiple food allergies, and treat other concomitant atopic diseases. Although omalizumab has been evaluated extensively, most biologics are more novel and have broader immunologic impact. Careful evaluation of their safety profile should therefore be conducted.

Adherence to Asthma Controller Therapy Among Children in Majmaah City, Saudi Arabia.

Background and objective Improving adherence to asthma medications may prevent asthma exacerbation, which is associated with a decline in lung function. The purpose of this study was to assess the adherence to asthma controller therapy and the factors that might influence the level of adherence among asthmatic children. Materials and methods We conducted a prospective observational study at the King Khalid Hospital in Majmaah, Saudi Arabia between January and April 2020; the study was conducted among children aged 1-14 years with a diagnosis of asthma. The data collected when available included age and gender by using a pre-tested questionnaire that contained four validated items, and the respondents were the parents of the affected children. Due to the coronavirus disease 2019 (COVID-19) pandemic, the data collection was performed via phone calls to ensure safety. Informed consent was obtained from the parents. Results We analyzed 152 asthmatic children to evaluate their adherence to asthma controller therapy. The majority of the children were males (60%). Asthma was most prevalent in the age group of 6-10 years (40.1%), followed by the age group of two to five years (32.9%). We found that the majority of the patients had poor adherence to asthma medication (83.6%) while the remaining had good adherence (16.4%). The total mean score for adherence to medication therapy was 5.16 (SD: 2.3). Conclusions The present study showed that the overall adherence level to the asthma controller therapy was poor among children. Larger, nationwide studies are needed to assess the adherence to asthma medications and implement interventions that can improve the same.

Antibiotic Prescribing Patterns for Hospitalized children with Community-Acquired Pneumonia in a Secondary Care Center.

BACKGROUND: Narrow-spectrum antibiotics are recommended as the first-line therapy for management of children hospitalized with community-acquired pneumonia (CAP). There are limited data evaluating the antibiotic prescription patterns for CAP in Saudi Arabia. The goal of this study to report on the antibiotic patterns in children hospitalized with CAP. METHODS: A prospective cross-sectional study was conducted in children aged 1 month to 13 years who were hospitalized with a diagnosis of CAP at King Khalid Hospital in Majmaah, Saudi Arabia, between January 2019 and January 2020. RESULTS: Data from 233 patients were collected. The majority of patients received amoxicillin clavulanate (57.9%), followed by ceftriaxone (30%), azithromycin (20.6%), cefuroxime (6%), ampicillin (2.1%), and piperacillin-tazobactam (2.1%). None of our patients were started on narrow-spectrum therapy. The younger age group (1-3 months) and patients with severe pneumonia were more likely to receive broad-spectrum cephalosporin and have a longer hospital stay (P = .000 and P = .002, respectively). However, the 4 months to 5 years age group was more likely to receive amoxicillin clavulanate (P = .001). Male gender was a significate risk factor for patients with severe pneumonia (P=.013) CONCLUSIONS: We demonstrated the inappropriate use of broad-spectrum therapy in children hospitalized with CAP. Further large multicenter studies are necessary to evaluate the patterns of antibiotic use and implement antimicrobial stewardship programs or quality-improvement projects to improve adherence to guidelines.

Novel Therapies for Treatment of Food Allergy.

Food allergy prevalence has increased over the past 2 decades and is estimated to affect 8% of children and 4% to 10% of adults. There is an unmet need to evaluate new therapeutic modalities that may decrease the risk of food-induced anaphylaxis and improve patients' quality of life. Oral, epicutaneous, and sublingual food immunotherapies have different safety and efficacy profiles, and their long-term outcome and applicability are unclear. Food allergy trials are currently evaluating different biologics (given as monotherapy or adjunct to immunotherapy), modified food proteins, DNA vaccines, and fecal microbiota transplantation.

Acquired Cold-Induced Urticaria in Pediatric Patients: A 22-Year Experience in a Tertiary Care Center (1996-2017).

BACKGROUND: Acquired cold-induced urticaria (ACU) has not been well evaluated in pediatrics. OBJECTIVE: To further evaluate the presentation of ACU in children and associated risk of anaphylaxis. METHODS: A retrospective chart review was performed in children 18 years or younger diagnosed with ACU at Boston Children's Hospital (US, Northeast) from 1996 to 2017. RESULTS: A total of 415 patients with ACU were identified, aged 4 months to 18.3 years at the time of diagnosis, with similar male:female distribution. Most patients had a history of atopic disease (78.3%), and 25.8% had other urticaria. Around two-third of patients experienced only localized cold-induced symptoms (grade 1), whereas 14.0% had diffuse cutaneous symptoms (grade 2) as the most severe reaction, and 18.6% experienced anaphylaxis (grade 3). Swimming triggered 77.6% of grade 3 reactions, whereas the rest were secondary to ingestion of cold food or beverages, or cold air or cold water exposure. Seven percent of subjects had more than 1 episode of anaphylaxis. Cold stimulation test (CST) was performed in 61.7% of patients, and the result was positive in 69.9% of those tested. Positive CST result was significantly associated with increased risk of anaphylaxis. There was a 11.7% rate of anaphylaxis among patients with negative CST result. Disease resolution at any point in the study period was documented in 8.9% of patients and was associated with a negative history of anaphylaxis. CONCLUSIONS: In the largest study to date on ACU, grade 3 reactions occurred in about a fifth of patients. Positive CST result was associated with a higher risk for anaphylaxis from ACU. Epinephrine prescription and patient/family counseling about risk factors for grade 3 reactions are recommended.

Long-Term Outcome of Peanut Oral Immunotherapy Facilitated Initially by Omalizumab.

BACKGROUND: We successfully used omalizumab to facilitate peanut oral immunotherapy (OIT) in children with reactivity to ≤50mg peanut protein and with high peanut IgE (median, 229 kU/L). OBJECTIVE: We report on long-term OIT outcomes in these patients, including dosing changes, adverse events, peanut immunoglobulin changes, and quality of life (QoL). METHODS: Patients were followed for up to 72 months (67 months of maintenance). Outcomes were collected on peanut dose amount, form, and frequency, as well as adverse events, (QoL), and laboratory studies. RESULTS: Of 13 patients initially enrolled, 7 patients (54%) continued on peanut OIT through month 72; 6 (46%) discontinued therapy because of adverse reactions. Maintenance peanut protein dose varied between 500 and 3500mg. Most patients consumed different peanut-containing products. All patients experienced at least 1 adverse event, and 1 patient developed eosinophilic esophagitis. Peanut-IgE, Arah1-IgE and Arah2-IgE, peanut-SPT, peanut-IgE:IgE ratio, and Arah2-IgE:Arah2-IgG4 ratio decreased on OIT. Peanut-IgG4, Arah1-IgG4, and Arah2-IgG4 initially increased on OIT and then decreased, though not falling to baseline levels. In patients who stopped OIT, there was a trend for reversal of these biomarker changes. Higher peanut-IgE and Arah2-IgE at study month 12 were associated with discontinuation. Patient and parent QoL improved from baseline, even in patients who discontinued OIT. CONCLUSIONS: Although adjunctive omalizumab allowed for faster and successful desensitization in patients with high peanut-IgE, almost half of patients discontinued OIT within 72 months because of reactions. Patients who stopped therapy had higher month 12 peanut-IgE and Arah2-IgE. It is possible that these patients might benefit from longer omalizumab administration.

Systemic Reactions in Pediatric Patients Receiving Standardized Allergen Subcutaneous Immunotherapy with and without Seasonal Dose Adjustment.

BACKGROUND: The 2003 Joint Task Force on Practice Parameters recommended standardizing allergen subcutaneous immunotherapy (SCIT). Data from longitudinal surveillance survey in North America reported a systemic reaction (SR) rate of 0.1% to 0.2% of injection visits. The rate of SR to standardized SCIT in pediatric patients has not been well evaluated. OBJECTIVE: The objective of this study was to evaluate the rate of SRs to standardized SCIT in pediatric patients aged 5 to 18 years in a single tertiary care center in the United States. METHODS: A retrospective chart review was conducted in 2 groups: group 1 started SCIT within a period extending from January 2009 to June 2012, whereas group 2 started SCIT within a period extending from January 2013 to June 2016. The protocol was modified in group 2 such that updosing and maintenance doses were adjusted in the spring for tree and grass pollen and in the fall for weed pollen. RESULTS: There were a total of 128 patients in group 1 and 118 patients in group 2. The rate of SR was 0.429% in group 1 and 0.364% in group 2, which was not significant. There was no difference in the severity of SR in the 2 groups with no-fatal or near-fatal SR noted. Asthma was a significant risk factor in the younger age subgroup aged 5 to 11 years. CONCLUSIONS: Standardized SCIT appears to be associated with an SR rate of 0.429% to 0.364% of visits in pediatric patients. Protocol modification did not lead to a significant drop in SR. Larger multicenter studies are required to further evaluate the rate of SRs from standardized SCIT.