Monoclonal Antibody Therapy in Childhood Asthma.

PURPOSE OF REVIEW: There has been an explosion of monoclonal antibodies in the treatment of severe uncontrolled adult asthma. Studies have now been published in severe pediatric asthma. There are numerous questions that need to be answered in determining whether these modalities are appropriate and safe in children. RECENT FINDINGS: This is a narrative review examining the latest pediatric literature on monoclonal antibodies, both approved and in the pipeline, for uncontrolled asthma. Presently, all of the biologics are positioned to treat patients with underlying type 2 high disease. Two monoclonal antibodies are approved for children 6 years of age and older, omalizumab and mepolizumab, with more likely approved in the near future. The effect of these agents in controlling severe pediatric asthma is promising. Data is limited to long-term efficacy and safety, and whether any agent has an effect on the natural history of asthma.

Peanut Oral Immunotherapy: a Current Perspective.

PURPOSE OF THE REVIEW: Peanut oral immunotherapy (OIT) is one of the most studied experimental therapies for food allergy. With the recently FDA-approved peanut product, Palforzia, the goal of this article is to review the most recent data from clinical trials, discuss recent trends, and anticipate future developments. RECENT FINDINGS: The latest research suggests that peanut OIT could be a promising option for peanut-allergic patients, with the majority of participants in research studies achieving the primary efficacy endpoint of desensitization, as well as sustained unresponsiveness in select populations. Some studies also showed improvements in food allergy-related quality of life. However, peanut OIT is not without risk or side effects, including potentially serious allergic reactions. Future research will need to evaluate the short- and long-term effectiveness of the therapy in the real-world setting, predictors of important treatment outcomes, and the use of adjunctive therapies that may mitigate some of these allergic reactions.

Successful desensitization of Pegvaliase (Palynziq®) in a patient with phenylketonuria.

Pegvaliase (Palynziq®) was FDA approved in 2018 as an enzyme substitution therapy in patients with Phenylketonuria. However, various drug induced hypersensitivity adverse events (HAEs) have been reported. We present a case of Pegvaliase (Palynziq®) induced anaphylaxis and successful desensitization. A 13-step desensitization protocol was performed using three solution concentrations of Palynziq with premedication of diphenhydramine and prednisone in an outpatient setting. The patient tolerated the desensitization and was able to continue Palynziq.

Pulmonary Disease Burden in Primary Immune Deficiency Disorders: Data from USIDNET Registry.

PURPOSE: Pulmonary manifestations are common in patients with primary immunodeficiency disorders (PIDs) but the prevalence, specific diseases, and their patterns are not well characterized. METHODS: We conducted a retrospective analysis of pulmonary diseases reported in the database of the United States Immunodeficiency Network (USIDNET), a program of the Immune Deficiency Foundation. PIDs were categorized into 10 groups and their demographics, pulmonary diagnoses and procedures, infections, prophylaxis regimens, and laboratory findings were analyzed. RESULTS: A total of 1937 patients with various PIDs (39.3% of total patients, 49.6% male, average age 37.9 years (SD = 22.4 years)) were noted to have a pulmonary disease comorbidity. Pulmonary diseases were categorized into broad categories: airway (86.8%), parenchymal (18.5%), pleural (4.6%), vascular (4.3%), and other (13.9%) disorders. Common variable immune deficiency (CVID) accounted for almost half of PIDs associated with airway, parenchymal, and other pulmonary disorders. Pulmonary procedures performed in 392 patients were mostly diagnostic (77.3%) or therapeutic (16.3%). These patients were receiving a wide variety of treatments, which included immunoglobulin replacement (82.1%), immunosuppressive (32.2%), anti-inflammatory (12.7%), biologic (9.3%), and cytokine (7.6%)-based therapies. Prophylactic therapy was being given with antibiotics (18.1%), antifungal (3.3%), and antiviral (2.2%) medications, and 7.1% of patients were on long-term oxygen therapy due to advanced lung disease. CONCLUSIONS: Pulmonary manifestations are common in individuals with PID, but long-term pulmonary outcomes are not well known in this group of patients. Further longitudinal follow-up will help to define long-term prognosis of respiratory comorbidities and optimal treatment modalities.

Nonsevere combined immunodeficiency T-cell lymphopenia identified through newborn screening.

PURPOSE OF REVIEW: Although severe combined immunodeficiency (SCID) is the primary target condition for newborn screening (NBS), over 25 secondary targets, conditions other than SCID, have been identified. There is no standard method for evaluating neonates with non-SCID T-cell lymphopenia (TCL) and no standard approaches to treatment. We will describe these conditions and discuss recommendations for evaluating and follow-up of non-SCID TCL detected by NBS. RECENT FINDINGS: The birth prevalence of non-SCID TCL detected through SCID NBS is higher than SCID and can be a burden on NBS programs. We will present some publications discussing outcomes and comorbidities in these patients. SUMMARY: NBS for SCID has been very successful in identifying infants with SCID at birth to institute early life saving therapies. TCL due to other conditions can cause significant immune deficiency and treatment is dependent on the cause of the defect, as well as the magnitude of the immunodeficiency. Data collection from NBS programs should include assessment of various therapies and clinical outcomes. Better systems for recording long-term outcomes of SCID NBS including both SCID and non-SCID conditions should become a priority for NBS programs. This will help to advance the goal of NBS programs: improve outcomes in the most cost-effective manner.