Improved transplant survival and long-term disease outcome in children with MHC class II deficiency.

MHC class II deficiency is a rare, but life-threatening, primary combined immunodeficiency. Hematopoietic cell transplantation (HCT) remains the only curative treatment for this condition, but transplant survival in the previously published result was poor. We analyzed the outcome of 25 patients with MHC class II deficiency undergoing first HCT at Great North Children's Hospital between 1995 and 2018. Median age at diagnosis was 6.5 months (birth to 7.5 years). Median age at transplant was 21.4 months (0.1-7.8 years). Donors were matched family donors (MFDs; n = 6), unrelated donors (UDs; n = 12), and haploidentical donors (HIDs; n = 7). Peripheral blood stem cells were the stem cell source in 68% of patients. Conditioning was treosulfanbased in 84% of patients; 84% received alemtuzumab (n = 14) or anti-thymocyte globulin (n = 8) as serotherapy. With a 2.9-year median follow-up, OS improved from 33% (46-68%) for HCT before 2008 (n = 6) to 94% (66-99%) for HCT after 2008 (n = 19; P = .003). For HCT after 2008, OS according to donor was 100% for MFDs and UDs and 85% for HIDs (P = .40). None had grade III-IV acute or chronic graft-versus-host disease. Latest median donor myeloid and lymphocyte chimerism were 100% (range, 0-100) and 100% (range, 64-100), respectively. Latest CD4+ T-lymphocyte number was significantly lower in transplant survivors (n = 14) compared with posttransplant disease controls (P = .01). All survivors were off immunoglobulin replacement and had protective vaccine responses to tetanus and Haemophilus influenzae. None had any significant infection or autoimmunity. Changing transplant strategy in Great North Children's Hospital has significantly improved outcomes for MHC class II deficiency.

Off-Label Drug Use in Pediatric Out-Patient Care: A Multi-Center Observational Study.

OBJECTIVE: Prescribing a drug for a child is not an easy task and requires using the best available evidence as a guide, especially when a drug is used off-label. The practice of prescribing a drug for off-label use is fairly widespread worldwide. The FDA does not regulate prescribing patterns or practices of individual practitioners and, therefore, allows off-label use. The main objective of this study is to evaluate off-label prescribing among the pediatric population in the Kingdom of Saudi Arabia (KSA). METHOD: This is a retrospective, simple random selection observational study of children (≤15 years) who visited pediatric clinics and had at least 1 drug prescribed over a 12-month period (January to December 2018). RESULTS: A total of 865 drugs (mean 1 and SD 0.24) were prescribed to 326 children. Off-label was identified in 39.4% of the drugs with a frequency of 512 (as 1 drug may belong to more than 1 off-label category). The most common reason for off-label prescribing was related to doses that were "higher or lower than the recommended use" (48.6%), and the most frequently identified drug class prescribed for off-label use was anti-infective drugs for systemic use (39.9%). The percentage of off-label drug use was found to be higher in girls and in the age group of 1 month to 2 years (P = .001) for both variables. In addition, a significant association was found between off label drug use and the total number of drugs prescribed, P < .001. CONCLUSION: The findings of this study showed a high incidence of off-label prescribing mainly related to dosing and indication. The results of this observational study support the need to establish a unified national pediatric dosing formulary guide to ensure safe drug use in pediatrics.

The Prevalence of Atopic Diseases Among Pediatric Food Allergic Patients.

Introduction Food allergy is an immunological reaction that occurs in response to particular proteins in food. As food allergy can affect multiple body organs, symptoms usually include pruritus, urticaria, rash, cough, dyspnea, and wheezing. Atopic diseases are common in patients with food allergies, and multiple significant associations have been made between them. As such, the presence of food allergy in atopic patients can be used as an indicator of disease severity. The aim of this study was to estimate the prevalence of atopic diseases in food allergy patients. Methodology This was a retrospective cohort study that included pediatric patients aged 0-14 with confirmed food allergies between January 2016 and June 2022. Data was retrieved from electronic medical records and included patients' demographics, type of food allergens, symptoms of food allergy, presence of atopic diseases, and the utilization of healthcare services. Categorical variables were reported as frequencies and percentages, with Chi-squared being used for comparison. Results A total of 228 patients were included in the study. Half of them (114, 50%) were diagnosed with an atopic disease. Seventy-four (64.9%) had asthma, 57 (50%) had atopic dermatitis, and 45 (39.5%) had allergic rhinitis. The most common food allergens in atopic patients were eggs and milk products, in 53 (46.4%) and 42 (36.8%), respectively. There was a significant association between allergy to eggs (p=0.0005), bananas (p=0.0242), and strawberries (p=0.0393), and the presence of an atopic disease. No significant difference was found between atopic and non-atopic patients in terms of utilization of outpatient (p=0.09), inpatient (p=0.50), or ER visits (p=0.31) due to food allergy. Conclusion The current study demonstrates the prevalence of atopic disease in patients with food allergies. Although certain foods were associated with the prevalence of atopic diseases, both atopic and non-atopic patients have similar utilization of health care services such as outpatient, inpatient, and ER.

Budget Impact of Early Introduction of Amino Acid Formula in Managing Infants with Cow Milk Protein Allergy: Arabian Gulf Countries' Experience.

Objective: This study estimates the budgetary impact of the introduction of amino-acid formula (AAF) as first-line management in the elimination diet of infants with suspected cow milk protein allergy (CMPA) in the Kingdom of Saudi Arabia (KSA), Kuwait, and the United Arab Emirates (UAE) from a health-care payer's perspective. Methods: A global decision tree model was adapted to compare estimated costs in current practice (extensively hydrolyzed formula [eHF] or soy formula [SF] with the proposed approach of early introduction of AAF as first-line treatment of CMPA in non-breast-fed infants). Model inputs were derived from explorative literature reviews and medical experts' opinions. All costs were reported in local currency, ie, Saudi Riyal (SAR) for KSA, Kuwaiti Dinar (KWD) for Kuwait, and United Arab Emirates Dirham (AED) for the UAE. Results: Cost savings with the early introduction of AAF were 10% (SAR 15102542) in KSA, 10% (KWD 306565) in Kuwait, 17% (AED 1842018) in the UAE government sector and 13% (AED 4232932) in the UAE private sector. The highest cost reduction was observed in the cost of soy formula (SF), with a 58% reduction both in KSA (SAR 4204540) and UAE public sector (AED 110331). A significant cost reduction in medication costs in Kuwait (37%; KWD 5630) and medical examination costs in the UAE private sector (50%; AED 1508918) was observed. Conclusion: Results indicated that the introduction of AAF as the first line in the management of CMPA is a cost-saving strategy for the Gulf Cooperation Council (GCC) countries-KSA, Kuwait, and UAE- from a health-care payer's perspective.

Comparative responses to nasal allergen challenge in allergic rhinitic subjects with or without asthma.

BACKGROUND: Nasal allergen challenge (NAC) is useful to study the pathophysiology of rhinitis, and multiple challenges may more adequately approximate natural exposure. OBJECTIVE: To determine the effect of 4 consecutive daily NAC, on clinical and inflammatory parameters in rhinitics with or without asthma. METHODS: Rhinitic subjects were recruited: 19 with mild asthma and 13 without asthma. Subjects underwent a control challenge (normal saline) followed by 4 consecutive daily NAC. Allergen challenge consisted of spraying the chosen allergen extract into each nostril until a positive nasal response occurred. Symptoms were recorded on a Likert scale, and oral peak expiratory and nasal peak inspiratory flows allowed assessment of a nasal blockage index (NBI), for a period of 7 hours. Induced sputum and nasal lavage were performed on control day and after 1 and 4 days of NAC. RESULTS: Compared with the control day, there was a significant increase in symptom scores and NBI 10 minutes after each last daily NAC in both groups (p < 0.05). Symptom scores and NBI were similar for the 2 groups, except for nasal obstruction and rhinorrhea, which were more marked in subjects with asthma and rhinitis, respectively. Nasal lavage eosinophils were increased after 4 days of challenges in both groups, but there was no change in sputum eosinophils. No cumulative effect or any late response were observed in any of the groups over the challenge period. CONCLUSION: Multiple NAC may be a useful tool to study the pathophysiology of allergic rhinitis or its relationships with asthma. TRIAL REGISTRATION: ClinicalTrials.gov NCT01286129.

The Effectiveness of Written Asthma Action Plan at the National Guard Health Affairs' Asthma Clinic.

Introduction Bronchial asthma has grown in epidemic proportions, and it is one of the most chronic diseases that affect many societies in the world. As managing asthma is complex, new management approaches have been developed, such as the written asthma action plan. This study aims to assign a baseline for the patients' knowledge about asthma and its management and to assess their need for an asthma action plan. Then, to identify the effect of the written asthma action plan on the following parameters: exacerbations rate, and the frequency of using rescue mediations. Also, to compare the quality of life, functional limitations, and the level of patients' self-confidence in treating their asthma before and after using the written asthma action plan. Method This study is a cross-sectional and interventional mixed-method study design. It was conducted at the National Guard Health Affairs (NGHA) asthma clinics between October 2017 to November 2017. Asthmatic patients who were above five years old and had no other lung comorbidities were evaluated before and after following the written asthma action plan by using three previously validated and published surveys that consist of five sections: demography, knowledge, quality of life, exacerbation rate, and overall evaluation. Results This study enrolled 58% (209) males and 42% (154) females. Regarding asthma medication knowledge and attack management, 62.3% of the patients do not adhere to their maintenance medications when they do not have asthma symptoms. Also, only 24.9% were very confident about knowing how to use their inhalers. For the impact of asthma on patients' quality of life and the functional limitations, we found that 42.1% of patients were absent from school or work more than once a week because of asthma. While 61.0% of patients did not attend social events twice or less per week because of their asthma. The third section of the survey was about asthma exacerbation related events during the past year, we found that 39.0% of patients had one or more asthma attacks, 41.6% visited the emergency room (ER) once or more during the past year, and 28.1% of patients have been hospitalized because of their asthma. Finally, the section about patients' evaluation of their asthma and their confidence about managing their condition, we found that around 20% of patients had poor or no control over their condition. In the second phase of the study, which includes 60 subjects, we found that following the action plan helps in increasing the patients' knowledge about their condition, and improves their quality of life and functional limitations as they learned how to cope with their symptoms. In addition, it has increased the confidence level of patients about controlling their asthma and decreasing the asthma exacerbation related events rates. Overall, the patients' evaluation of their asthma has been increased significantly (p-value= 0.023). Conclusion Most of the asthmatic patients had insufficient knowledge and/or poor adherence to their treatment which impacted their quality of life. The written asthma action plan was effective in increasing the patients' knowledge about their condition, improving their quality of life and functional limitations, and increasing their confidence level about controlling their asthma.