R21/Matrix-M vaccine well-tolerated and efficacious against malaria in children

A recent study found that R21/Matrix-M vaccine which is low-cost, was well tolerated and offered high efficacy against clinical malaria in children. The results of this study were published in the journal, Lancet.

This double-blind, randomised, phase 3 trial of the R21/Matrix-M malaria vaccine screened 5477 children (aged 5-36 months), out of which 1705 and 3434 children were randomly assigned in a 2:1 ratio to the control vaccine and R21/Matrix-M (5 μg R21 plus 50 μg Matrix-M), respectively. The vaccines were administered 4 weeks apart as 3 doses, with a booster dose administered 12 months after the third dose. Half of the participants were recruited at the seasonal malaria transmission sites and the other half at standard sites with perennial malaria transmission. The primary objective of the study was to assess the protective efficacy of R21/Matrix-M, 14 days following the third vaccination. Vaccine efficacy, safety, and immunogenicity were also assessed.

At the end of the study, it was found that R21/Matrix-M vaccine was well tolerated. The most frequent adverse events were injection site pain (301 out of 1615 participants) and fever (754 out of 1615 participants). At the end of 12 months, the vaccine efficacy at the seasonal sites and standard sites were 75% and 67%, respectively. The effectiveness of the vaccine was correlated with antibodies produced by the vaccination against the conserved central Asn-Ala-Asn-Pro (NANP) repeat sequence of the circumsporozoite protein. The 5-17 month age group of children showed higher NANP-specific antibody titres when compared to the 18-36 month age group.

From the above results, it can be concluded that R21/Matrix-M vaccine which is low-cost, may be well tolerated and may offer high efficacy against clinical malaria in children.

Safety and efficacy of linaclotide in treating functional constipation in paediatric patients

According to a recent study, linaclotide has proven to be an effective and well-tolerated remedy for functional constipation in pediatric patients. This study’s findings were published in the journal, Lancet. Gastroenterology & hepatology.

In this double-blind, randomised,  placebo-controlled, phase 3 study, patients aged 6-17 years were randomly assigned to receive either oral linaclotide 72 μg [n=166] or placebo [n=164] once daily for 12 weeks. The study's primary efficacy endpoint was the change from baseline (CFB) in the frequency rate of spontaneous bowel movements (SBM) per week over a twelve-week period. The change from baseline in stool consistency during the treatment period was the secondary efficacy endpoint. Efficacy and safety were analyzed in all patients who received at least one dose of the study intervention.

The mean frequency rate for SBMs was 1·28 SBMs per week (SD 0·87) with placebo and 1·16 SBMs per week (0·83) with linaclotide at baseline. This rate increased to 2·29 SBMs per week (1·99) with placebo and 3·41 SBMs per week (2·76) with linaclotide during the intervention. Patients treated with linaclotide demonstrated a significant increase in SBM frequency [least-squares mean (LSM) CFB 2·22 SBMs per week (SE 0·19)] compared to those on placebo [LSM CFB 1·05 SBMs per week (0·19)]. Additionally, linaclotide demonstrated a notable improvement in stool consistency compared to the placebo group [LSM CFB 1.11 (SE 0.08) versus 0.69 (0.08)]. Based on the above results, it can be concluded that linaclotide is an efficacious and well-tolerated treatment for functional constipation in paediatric patients.

Dupilumab enhances lung function parameters in children with type 2 asthma

According to a recent study, dupilumab resulted in significant and sustained improvements in lung function across various measures in children (aged 6-11 years) suffering from uncontrolled, moderate-to-severe type 2 asthma. This study’s findings were published in the Journal of Allergy and Clinical Immunology.

In the LIBERTY ASTHMA VOYAGE phase 3 trial, children with asthma were subjected to randomization in a 2:1 ratio to receive either add-on dupilumab 100/200 mg based on bodyweight or a placebo every 2 weeks for a total of 52 weeks. This study analyzed the spirometry parameters among children with type 2 asthma (baseline blood eosinophils ≥150 cells/μL or fractional exhaled nitric oxide [FeNO] ≥20 parts per billion [ppb]), as well as subgroups identified by baseline blood eosinophils or FeNO values.

At baseline, a total of 116 children (49%) who received dupilumab and 59 children (52%) who received placebo had impaired lung function (prebronchodilator percent-predicted forced expiratory volume in 1 second [ppFEV1] less than 80%). The administration of dupilumab resulted in significant improvements in pre- and postbronchodilator ppFEV1 as early as week 2, and these improvements were sustained for up to 52 weeks (the least squares mean difference versus placebo at week 52 was 7.79 percentage points ; 95% confidence interval [CI]: 4.36-11.22; P < .001) and 4.37 points (95% CI: 0.95-7.78; P = .01) for pre- and postbronchodilator measurements, respectively). Additionally, consistent improvements were observed in other lung function parameters, including pre- and postbronchodilator forced vital capacity (FVC), FEV₁/FVC ratio, and prebronchodilator forced expiratory flow, across all populations.

Thus, it can be concluded that dupilumab led to significant and sustained improvements in lung function across various measures in children (aged 6-11 years) with uncontrolled, moderate-to-severe type 2 asthma.

Effect of serum S100B level in the management of pediatric minor head trauma

A recent study demonstrated the efficacy of serum S100B level in managing pediatric minor head trauma through a decrease in the need for cranial computed tomographic (CCT) scans and hospital observation when monitored according to a specific clinical decision algorithm. This study’s findings were published in the journal, JAMA network open.

In this multicenter, prospective, interventional randomized clinical trial, children and adolescents aged 16 years or younger were enrolled. The control group [n= 926] underwent CCT scans or were hospitalized as per the prevailing recommendations. In the S100B biomonitoring group [n=1152], blood sampling was conducted within 3 hours after minor head trauma, and the subsequent management was contingent upon the levels of serum S100B protein. If the S100B level was within the reference range suitable for the child's age, they were discharged from the emergency department. Otherwise, they received the same treatment as the control group. The main outcome of the study was the proportion of patients who underwent CCT scans within 48 hours after experiencing a minor head trauma.

At the end of the study, 299 children (32.3%) in the control group and 112 (9.7%) in the S100B biomonitoring group underwent cranial CT scans. A substantial 50% decline in hospitalizations was observed in the S100B biomonitoring group (479 [41.6%] vs 849 [91.7%]).

Based on the above results, it can be concluded that implementation of S100B biomonitoring resulted in a decline in the number of CCT scans required and the duration of in-hospital monitoring when assessed based on the criteria outlined in a clinical decision algorithm.

Uncovering the future of pediatric research in India by addressing challenges and revealing opportunities

This study examined the present state of pediatric research in India, highlighting challenges like insufficient funding, lack of research facilities, complex regulatory systems, and the increasing prevalence of childhood obesity. Despite these challenges, there are many opportunities for improving child health outcomes through technological advancements. This study was published in the IP International Journal of Medical Paediatrics and Oncology.

This paper provides several applications for improving pediatric health outcomes. Artificial Intelligence (AI) holds very good scope in pediatric research to analyze data, predict disease conditions, and develop individualized treatment plans. It can be beneficial to analyze larger datasets to find out patterns associated with pediatric diseases for early detection and the development of personalized treatment strategies. Integrating AI into general pediatric research and its application can immensely improve healthcare accessibility in India, irrespective of dynamic social and economic scenarios.

Proteomics research allows us to better understand the structure and function of proteins in various diseases; hence, proteomics research is important for investigating disease mechanisms. Similarly, it can be used for monitoring disease susceptibility and progression, monitoring treatment effectiveness, and assessing the likelihood of exacerbations.

Microbiome research can help us to understand the impact of the microbiome on child health and shed light on the relationship between gut bacteria, the immune system, the central nervous system, and metabolic processes.

Nanotechnology opens new possibilities for targeted drug delivery and precision medicine among children. Engineered nanoparticles with increased efficacy and minimal systemic toxicity can be potentially used in the treatment of pediatric cancer and viral infectious diseases.

The use of advanced fetal imaging technology and early treatment can prevent congenital abnormalities. Adolescent health research can help to deal with mental health problems, nutrition deficiency problems, and lifestyle diseases that Indian teenagers are facing today.

Pediatric interventional radiology includes advanced imaging technologies to diagnose and treat various conditions by utilizing minimally invasive procedures with less recovery time and complications. Improving access to interventional radiology in India has the potential to widen treatment options for pediatric cardiac defects, birth defects, and cancer-related issues.

To conclude, pediatric research in India can be transformed by navigating current challenges and seizing emerging opportunities. The integration of AI, microbiome research, nanotechnology, interventional radiology, and the progress in fetal as well as adolescent health will cater for precise diagnosis and personalized treatment plans, leading to improved health outcomes in children and adolescents.