The Pediatrician’s Role in Vaccination and Trust

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Two recent commentaries on “hot button” issues:

  • ER Menzin: The Pediatrician’s Lament. NEJM 2025; 392: 320-321
  • PMG Santos et al. Texas Executive Order GA-46 and the Erosion of Trust in Health Care. NEJM 2025; 392: 108-109

An excerpt from the first commentary:

“Throughout my career, I have seen new vaccines approved: pneumococcus, rotavirus, meningococcus, and human papillomavirus. In each case, I have studied the data, reviewed published recommendations, and adjusted my language to encourage vaccination. I consider the high immunization rate in my patient panel to be one of my greatest professional accomplishments — a quantitative metric of the benefit I provide…

Every so often, parents will look at me over a smiling infant and tell me they want their child to have only one or two recommended vaccines. Can I choose the most important? I tell them the question is akin to asking me to pick my favorite child — an impossible task…

Some will ask, “Can you recommend a good pediatrician who does not believe in vaccines?” No, I say, no more than I can recommend a good physicist who does not believe in gravity…

Even if patients are skeptical of the alphabet soup of institutions designed to protect and safeguard their health, they still have confidence in the long-standing relationships with their clinicians. To deserve that trust, we are obligated to raise our collective voice in defense of science, health, and vaccines.”

From the 2nd Commentary:

“On Halloween morning, 2024, Texas physicians received disturbing news about hospital policies set in compliance with Governor Greg Abbott’s Executive Order GA-46 — a rule that mandates the collection and reporting of information on patient citizenship status during intake…

Throughout medical training, physicians learn that trust is a cornerstone of patient care: we ask patients to share deeply personal information about themselves and their loved ones, with the assurance that legal and ethical safeguards protect against the misuse of such information. Chief among these safeguards is the Health Insurance Portability and Accountability Act (HIPAA), which restricts physicians from disclosing protected health information (PHI) without a person’s consent. Citizenship status, though not traditionally considered PHI, may be treated as such when it is paired with medical information obtained during patient encounters…

GA-46 … will deter immigrants, both documented and undocumented, from seeking help for serious medical concerns… Under the Emergency Medical Treatment and Labor Act (EMTALA), hospitals are required to provide emergency care to all patients, regardless of citizenship status. GA-46 indirectly conflicts with EMTALA’s intent to guarantee access to emergency services for all people by discouraging undocumented people from seeking care…

From an economic standpoint, Texas officials have stated that the goal of GA-46 is to protect the financial solvency of public hospitals; however, federal financial support covers most uncompensated care costs. Moreover, contributions from immigrants help sustain the viability of public health insurance programs. In 2017, immigrants helped offset a $67 billion deficit in health care costs for U.S.-born citizens by paying $58 billion more in taxes and premiums than was spent on their health care; 89% of this surplus was attributed to contributions made by undocumented immigrants…

Physicians in Texas and Florida must continue to inform patients of their right to refuse disclosure of their citizenship status.”

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IBD Briefs: Upadacitinib in Children, Predicting Crohn’s Disease, and Autoimmune Diseases Associated with IBD

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J Runde et al. J Pediatr Gastroenterol Nutr. 2025;80:133–140. Upadacitinib is associated with clinical response and steroid-free remission for children and adolescents with inflammatory bowel disease

In this single-center retrospective study, n=20 (3 CD, 13 UC, 4 IBD-U), steroid-free clinical remission (SF-CR) was seen in 75% (16/20) following induction and maintained in 65% (11/17) reaching Week 24 of therapy

J Gaifem et al. Nature Immunology 2024; 25: 1692-1703. Open Access! A unique serum IgG glycosylation signature predicts development of Crohn’s disease and is associated with pathogenic antibodies to mannose glycan.

“Analysis of preclinical serum samples, up to 6 years before IBD diagnosis (from the PREDICTS cohort), revealed the identification of a unique glycosylation signature on circulating antibodies (IgGs)…[which] elicits a proinflammatory immune pathway through the activation and reprogramming of innate immune cells.”

LR Jolving et al. Inflamm Bowel Dis 2025; 31: 87-94. Children and Adolescents Diagnosed With Inflammatory Bowel Disease Are at Increased Risk of Developing Diseases With a Possible Autoimmune Pathogenesis

Using Danish registry and 50-fold matched controls, there was a significant increase for a large number of autoimmune diseases: The adjusted hazard ratio after full follow-up was 4.72 for psoriatic arthritis, 5.21 for spondyloarthritis, 2.77 for celiac disease, 2.15 for rheumatoid arthritis, 1.69 and 1.64 for type 1 and type 2 diabetes, respectively. For thyroid disease, it was 1.16.

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La Fortuna, Costa Rica

Gastrointestinal Issues in Rett Syndrome: Key Findings

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FD Ihekweazu, KJ Motil. J Pediatr Gastroenterol Nutr. 2025;80:46–56. Gastrointestinal manifestations of Rett syndrome: An updated analysis using the Gastrointestinal Health Questionnaire

Methods: Parents of 118 females with Rett syndrome (RTT) and 27 unaffected females completed the GHQ.

Key findings:

  • GI symptoms were common in females with RTT, including constipation (81%), gas and bloating (70%), issues with eating, chewing and swallowing (73%), and irritability because of stomach or intestinal problems (53%).
  • Females with RTT commonly used proton pump inhibitors (52%) and laxatives (64%). 
All with p values of <0.001 with the exception of has >3 BM/day which had p value of 0.004

My take: “GI problems are common in RTT and pose a significant medical burden to caregivers.” As such, it is a good idea to screen for treatable disorders including swallow dysfunction, constipation, and reflux.

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Pharmacological Management of Pediatric Steatotic Liver Disease

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RARA Jaoudeh et al. J Pediatr Gastroenterol Nutr. 2025;80:14–24; Pharmacological management of pediatric metabolic dysfunction-associated steatotic liver disease

Key points:

  • Glucagon-like peptide-1 receptor agonists (GLP-1RAs) have demonstrated efficacy against adult MASLD. “In patients ≥12 years of age with obesity (BMI ≥ 95th percentile), we recommend the use of GLP-1RA—along with dietary and lifestyle modifications—in those who have MASLD or MASH, and 1 additional metabolic comorbidity (hypertension, prediabetes, polycystic ovary syndrome, dyslipidemia, and obstructive sleep apnea) (Figures 1 and 2). Treatment with these agents should only be initiated after other causes of hepatic steatosis are ruled out.”
  • “GLP-1RA are effective in treating pediatric obesity and have shown to decrease liver enzyme levels which is likely indicative of their effect on MASLD.”
  • “It is important to note that phentermine/topiramate combination drug is approved for patients 12 years and older with obesity15 and can be used as a bridge for GLP-1RA therapy in cases where access to GLP-1RA is limited.”
  • GLP-1RA Dosing regimens are provided in Table 1. For example, “Semaglutide for weight loss is initiated at 0.25 mg once weekly subcutaneously and increased every 4 weeks in a stepwise fashion up to a maximum of 2.4 mg once weekly dose. The most common side effects are nausea and/or vomiting and can be worse the first few days a after dose increase. It is acceptable to delay dose escalation or reduce the target dose based on patient tolerance. Medication therapy should be evaluated for effectiveness after 12 weeks on a maximally tolerated dose.” And, “Liraglutide for weight loss is initiated at 0.6 mg daily subcutaneously and increased weekly in 0.6 mg increments up to a maximum 3 mg daily dose.”
  • Adverse effects: “Both liraglutide and semaglutide have been associated with thyroid C-cell tumors in animal studies37 and are contraindicated in patients with a personal or familial history of multiple endocrine neoplasia 2A and 2B and medullary thyroid carcinoma. Patients should be educated on symptoms of thyroid tumors—lump in the neck, difficulty breathing or swallowing, or persistent hoarseness—and treatment should be discontinued if these occur.37 GLP-1RA also increase the risk of pancreatitis and gallbladder disease especially with rapid weight loss.37 Liraglutide is contraindicated in pregnancy due to potential embryo-fetal defects shown in animal studies, and semaglutide should be discontinued if pregnancy occurs.10, 37“

Useful algorithm:

My take: GLP-RAs are likely to be used increasingly in adolescents with MASLD despite issues with insurance/affordability and need for chronic treatment. This is a helpful review.

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Disclaimer: This blog, gutsandgrowth, assumes no responsibility for any use or operation of any method, product, instruction, concept or idea contained in the material herein or for any injury or damage to persons or property (whether products liability, negligence or otherwise) resulting from such use or operation. These blog posts are for educational purposes only. Specific dosing of medications (along with potential adverse effects) should be confirmed by prescribing physician.  Because of rapid advances in the medical sciences, the gutsandgrowth blog cautions that independent verification should be made of diagnosis and drug dosages. The reader is solely responsible for the conduct of any suggested test or procedure.  This content is not a substitute for medical advice, diagnosis or treatment provided by a qualified healthcare provider. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a condition.

Improving Laryngopharyngeal Symptoms with Laryngeal Recalibration

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E Walsh et al. Am J Gastroenterol 2024; 119: 2198-2205. Laryngeal Recalibration Therapy Improves Laryngopharyngeal Symptoms in Patients With Suspected Laryngopharyngeal Reflux Disease (Thanks to Ben Gold for this reference)

For a lot of patients with rheumatologic complaints like joint pain, treatment often consists of sending patients to physical therapy rather than using pharmaceuticals. This type of approach is under-utilized in gastroenterology. A recent study, however, suggests that an analogous approach is likely beneficial in patients with chronic laryngopharyngeal symptoms.

Background: Laryngopharyngeal symptoms such as cough, throat clearing, voice change, paradoxic vocal fold movement, or laryngospasm are hyper-responsive behaviors resulting from local irritation (e.g., refluxate) and heightened sympathetic tone. Laryngeal recalibration therapy (LRT) guided by a speech-language pathologist (SLP) provides mechanical desensitization and cognitive recalibration to suppress hyper-responsive laryngeal patterns.

Methods: Adults (n=65, mean age 55 years) with chronic laryngopharyngeal symptoms referred for evaluation of GERD to a single center were prospectively followed. Inclusion criteria included ≥2 SLP-directed LRT sessions (60 minutes sessions). “Mechanical desensitization focuses on well-known laryngeal suppression techniques (i.e. pursed lip breathing to suppress throat clearing or cough) or changing voice production by means of acoustic and aerodynamic techniques…Cognitive recalibration uses relaxation and conceptualization of symptoms to rework thought patterns around chronic laryngeal behaviors.”

Key findings:

  • Overall, 55 participants (85%) met criteria for symptom response. 17 (26%) had complete resolution, 19 (29%) had near-complete resolution, and 19 (29%) had a moderate response
  • Specifically, symptom response was similar between those with isolated laryngopharyngeal symptoms (13/15, 87%) and concomitant laryngopharyngeal/esophageal symptoms (42/50, 84%)

My take: Historically, patients with laryngopharyngeal symptoms have been difficult to treat. Many do not respond to reflux therapies. This study highlights a different approach and shows that the benefit of working with highly-skilled SLPs.

Snow day in Atlanta (Jan 10, 2025)

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Pivotal Study: Guselkumab Efficacy in Ulcerative Colitis (QUASAR study)

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DT Rubin et al. Lancet 2024; 405: 33-49. Guselkumab in patients with moderately to severely active ulcerative colitis (QUASAR): phase 3 double-blind, randomised, placebo-controlled induction and maintenance studies

Background: “Guselkumab is a dual-acting, human IgG1, interleukin-23p19 subunit inhibitor that potently neutralises interleukin-23 and can bind to CD64.”

Methods:  “Two phase 3, randomised, double-blind, placebo-controlled studies (QUASAR phase 3 induction and maintenance) included randomised and treated adults with moderately to severely active ulcerative colitis (induction baseline modified Mayo score from 5 to 9) with inadequate response or intolerance to conventional or advanced ulcerative colitis therapy.”

“The induction study primary population included 701 patients (guselkumab 200 mg given intravenously 60% [421 patients]; placebo 40% [280 patients]). The maintenance study primary population included 568 guselkumab induction responders randomly assigned to receive guselkumab 200 mg given subcutaneously every 4 weeks (190 [33%] patients) or 100 mg every 8 weeks (188 [33%] patients) or placebo (guselkumab withdrawal 190 [33%] patients).”

Key findings:

  • INDUCTION DATA AT 12 WEEKS:
At induction week 12
At induction week 12
Induction symptomatic endpoints -response noted in majority of guselkumab-treated patients by 4 weeks

MAINTENANCE DATA AT 44 WEEKS

Results and Discussion points:

  • “Symptomatic improvement was observed as early as induction week 1 (first assessed timepoint)”
  • “Greater reductions in C-reactive protein and faecal calprotectin concentrations with guselkumab induction compared with placebo were observed as early as induction week 4 (first assessed timepoint)”
  • “Guselkumab efficacy was shown in both biologic naive and JAK inhibitor-naive patients, and in patients with a history of inadequate response or intolerance to biologics or JAK inhibitors”
  • “Overall, 34% (129 of 378) of patients in the guselkumab groups achieved endoscopic
    remission     at maintenance week 44. Among the 180 patients in the guselkumab groups in clinical remission at maintenance week 44, 124 (69%) of 180 were in endoscopic remission”
  • “Symptomatic remission and deep symptomatic remission achieved with guselkumab induction was generally maintained to maintenance week 44 with guselkumab relative to placebo”
  • “The incidences of anti-guselkumab antibodies and NAbs were low in both the induction and maintenance studies…titres were low and did not affect serum concentration, efficacy, or safety”
  • “Head-to-head comparison data with other IL-23 antagonists are currently not available”
  • “Safety results were consistent with the known and favourable safety profile of guselkumab in its approved indications. Rates of adverse events, serious adverse events, and adverse events leading to treatment discontinuation generally did not occur more frequently in patients treated with guselkumab versus placebo-treated patients”
  • Limitation: The primary analysis population for the maintenance study included only guselkumab induction responders following 12 weeks of intravenous treatment

My take: Overall, this is a pivotal study showing that guselkumab is an effective agent for moderately to severely active ulcerative colitis in those with and without prior treatments. More head-to-head studies are needed to determine the optimal positioning of therapies for UC. Currently, AGA guidelines (AGA Living Guideline for Moderate-to-Severe Ulcerative Colitis –The Good and The Bad) suggest that guselkumab should be considered in the top tier of medications used in patients naive to biologics/advanced therapies and in the second tier for those with prior biologic treatments.

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Adverse Childhood Experiences Increase Anxiety and Disorders of Gut-Brain Interaction

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J Fritz et al. J Pediatr Gastroenterol Nutr. 2025;80:100–107. The relationship between adverse childhood experiences and disorders of the gut–brain interaction

Methods: Retrospective review of patients aged 3–18 years with ACE scores documented between October 1, 2019 and April 30, 2022. from two large primary care medical groups in Southern Maine within the MaineHealth system. ACE screening tool was taken at a routine WCC at a primary care office. From these patients, we identified from the medical record whether patients were referred to a general pediatric GI clinic in Southern Maine. 

Key findings:

  • Four hundred and one (44.7%) were diagnosed with a DGBI.
  • With each additional adverse experience, patients were 1.09 times more likely to have a DGBI diagnosis (p ≤ 0.001). An anxiety diagnosis mediated 73% of this relationship (p = 0.012).

Limitations:

There are many limitations to this study. The vast majority of patients with DGBIs were likely never referred to a pediatric GI clinic. While anxiety was found to mediate the relationship between ACEs and DGBIs, perhaps an alternative explanation was that anxiety increased the likelihood of GI referral. In addition, the authors note that” patients who are perceived as anxious by their gastroenterologist are more likely to receive a DGBI diagnosis.27 The nature of a symptom-based diagnosis for DGBI was also a limitation as not all patients had sufficient documentation to apply Rome criteria for inclusion.”

My take: Despite the limitations of this study, it appears that ACEs are associated with DGBIs mediated mainly by anxiety.

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From Manuel Antonio Park
View from Manuel Antonio

Understanding Patient-Specific Blood Count Setpoints

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BH Foy et al. Nature (2024). https://doi.org/10.1038/s41586-024-08264-5. Haematological setpoints are a stable and patient-specific deep phenotype

Background: The complete blood count (CBC) is an important screening tool for healthy adults and a common test at periodic exams. However, results are usually interpreted relative to one-size-fits-all reference intervals1,2, undermining the precision medicine goal to tailor care for patients on the basis of their unique characteristics3,4.

Key findings:

  • Routine CBC indices fluctuate around stable values or setpoints5, and setpoints are patient-specific, with the typical healthy adult’s nine CBC setpoints distinguishable as a group from those of 98% of other healthy adults, and setpoint differences persist for at least 20 years.
  • Setpoints also define patient-specific reference intervals and personalize the interpretation of subsequent test results. In retrospective analysis, setpoints improved sensitivity and specificity for evaluation of some common conditions including diabetes, kidney disease, thyroid dysfunction, iron deficiency and myeloproliferative neoplasms

This study is discussed by Eric Topol: Your Lab Tests. “This [individualized setpoints] enabled the ability to differentiate increased risk of progression to Type 2 diabetes in people as a function of MCHC setpoint, likelihood of hypothyroid status (elevated thyroid stimulating hormone (TSH) from the current MCV, low iron storage (ferritin) from the drop in hemoglobin, and even a mutation in Janus Kinase (JAK2) in people with high platelet setpoints (>9-fold higher rate of mutations, which are associated with several blood cancers.).”

My take: This study indicates that for each individual there past test results are more useful than establised normative values. Deviation from a person’s setpoint, even if in the normal range, is more indicative of development of a medical problem. However, this type of data is not always utilized. This study focused on CBC values, but there may be setpoints with other common labs as well; more studies are needed.

Position Paper: Pediatric Refractory Constipation Management

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AL Kilgore et al. JPGN 2024; https://doi.org/10.1002/jpn3.12390. Open Access! Evaluation and management of pediatric refractory constipation: Recommendations from the NASPGHAN neurogastroenterology and motility committee

Selected Recommendations:

Evaluations:

  • Screen for thyroid disease and celiac disease (though acknowledges that the data regarding an association between celiac disease and constipation are inconsistent)
  • The use of an AXR in RC should be reserved for those patients unable to provide a reliable medical history and/or unable to allow for a physical exam (including a DRE), or to evaluate for mechanical obstruction or colonic distention when considering surgical interventions
  • A contrast enema (CE) can be used to screen for HD or to assess colorectal anatomy
  • There is no evidence to recommend the routine use of defecography in children
  • Abdominal ultrasound has a good agreement with digital rectal exam (DRE) to evaluate for fecal impaction but should not be performed in place of DRE
  • ARM should be used to screen for the presence of a RAIR. If anal spasms and prolonged sphincter relaxation are detected during ARM, an assessment for spinal abnormalities can be considered
  • An LS MRI should be performed in pediatric patients with RC associated with physical or neurological signs of spinal anomalies, signs of neurogenic bladder on urodynamics, or when the anorectal manometry (ARM) is abnormal suggesting spinal cord abnormalities
  • Colonic transit time (CTT) via radiopaque markers should be completed for patients with RC with equivocal medical history and to screen for the need to perform colonic manometry (CM)
  • Colonic manometry (CM) should be performed only after medical therapy has been exhausted and surgical therapy is being considered. CM should be used to guide the timing and type of surgery to address RC. CM should be used to guide when to perform an ostomy takedown
  • Rectal biopsies should not be used routinely in patients with RC and are indicated exclusively in patients with a suspected diagnosis of HD

Pharmaceuticals:

  • High-dose sennoside (or Bisacodyl) is a mainstay of management of RC and should be optimized for the individual patient before considering further management options
  • A secretagogue (or prucalopride) should be considered as an adjunct to a high-dose stimulant laxative when treating RC with poor response to optimized high-dose stimulant laxatives or when high-dose stimulant laxatives are not tolerated
  • There is no clear role of anal botox in the treatment of patients with RC without a diagnosis of IAS achalasia
  • Early intervention with daily stimulant laxatives in the treatment of FC is encouraged to try to prevent the disease progression from functional constipation (FC) to RC
  • Antegrade and Retrograde Treatments:
Routine dosages of frequently used antegrade and retrograde solutions and additives
  • The last part of the recommendations include antegrade continence enemas, surgical approaches, and complicated algorithms (see Figure 1 and Figure 2)

My take: These recommendations address a widespread problem for pediatric gastroenterologists and are useful for those with and without an interest in motility disorders.

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Disclaimer: This blog, gutsandgrowth, assumes no responsibility for any use or operation of any method, product, instruction, concept or idea contained in the material herein or for any injury or damage to persons or property (whether products liability, negligence or otherwise) resulting from such use or operation. These blog posts are for educational purposes only. Specific dosing of medications (along with potential adverse effects) should be confirmed by prescribing physician.  Because of rapid advances in the medical sciences, the gutsandgrowth blog cautions that independent verification should be made of diagnosis and drug dosages. The reader is solely responsible for the conduct of any suggested test or procedure.  This content is not a substitute for medical advice, diagnosis or treatment provided by a qualified healthcare provider. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a condition.

ESPGHAN Guidelines for PSC in Children

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PF van Rheenen et al. JPGN 2024; DOI: 10.1002/jpn3.12378. Open Access! Primary sclerosing cholangitis in children with inflammatory bowel disease: An ESPGHAN position paper from the Hepatology Committee and the IBD Porto group

Recommendations:

  • In children with suspected or confirmed IBD, screening for liver disease is usually performed at 3 to 6 months intervals and a work‐up for underlying liver disease is most commonly initiated when liver enzymes exceed 2x the upper limit of normal
  • Use MRCP as the radiological modality of choice for diagnosing PSC
  • Consider performing a liver biopsy in children with IBD and suspected PSC in the following circumstances: i) Normal biliary tree at MRCP, ii) raised immunoglobulin G and the presence of liver-specific autoantibodies, or iii) clinical uncertainty before steroid induction therapy for IBD
  • Perform fecal calprotectin screening at least once yearly in children with isolated PSC and/or AIH to select patients for diagnostic endoscopy for suspected inflammatory bowel disease (panel recommends cutoff of >150 indicating need for ileocolonoscopy)
  • Surveillance colonoscopy should be considered in children with PSC–IBD and the following risk factors of colorectal cancer: i) persistent active colonic inflammation, ii) longstanding colitis (≥8 years), or iii)  a family history of colorectal cancer in a first-degree relative <50 years. (The overall risk of colon cancer in those <18 yrs of age is very low)
  • UDCA may be prescribed at doses of 15–20 mg/kg/day. Despite evidence of improvement of liver enzymes, its long-term effect on disease progression has not been demonstrated. Consider a 6-months therapeutic trial of UDCA, either immediately after PSC diagnosis or when spontaneous normalization of GGT does not occur in the first 6 months postdiagnosis. Continue UDCA treatment if there is a meaningful reduction or normalization of GGT or improvement of symptoms
  • Oral vancomycin may be prescribed for a potential improvement in liver biochemistry as well as bowel inflammation. Its long-term effect on disease progression has not been demonstrated
  • In children with PSC–IBD and biochemical, serological, and histological features of AIH, the use of corticosteroids and antimetabolites may suppress immune-mediated hepatitis. In the absence of convincing AIH features, the use of corticosteroids and antimetabolites is not indicated to manage PSC
  • Children with PSC, relevant bile-duct strictures and cholestatic symptoms should be assessed for liver transplantation. When their symptoms are likely to improve following biliary intervention, ERCP can be considered
  • Recommended blood testing for children with PSC: At diagnosis: Autoantibodies (ANA, anti-SMA, anti-LKM-1, anti-LC1, and anti-SLA), Every 3-6 months: ALT, AST, GGT, Albumin, INR, Platelets, CRP. Every 12 months: IgG, AFP, and Fat Soluble vitamins. Consider f/u autoantibodies in those with elevated IgG at f/u lab testing

My take: This is a useful position paper; it does not have a zillion recommendations like some other ESPGHAN positions papers. Given the frequency of liver enzyme elevation in patients with IBD, mild to modest elevations may need to be observed before launching an extensive evaluation (see related blog posts below).

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