Category Archives: Uncategorized

Best Allergy Articles 2021 (Part 5): Allergy Test Ordering

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In Pediatrics, supplement 3 summarizes 76 articles: Synopsis Book: Best Articles Relevant to Pediatric Allergy, Asthma and Immunology

Some of the studies that are most relevant to pediatric GI doctors I am reviewing for this blog over the next/past few days.

MT Kraft et al. Ann Allergy Asthma Immunol 2020; 125: 341-360. Review of Ordering Practices for Single-Allergen and Serum-Specific Immunoglobulin E Panel Tests for Food Allergy

In this study, the author’s examine the ordering of serum IgE food allergy tests at a single hospital in 2018. In total 12,345 tests were ordered by 400 physicians.

Key findings:

  • Allergists ordered 8986 tests, of which only 1.2% were food panels.
  • Nonallergists ordered 3368 tests, of which 37.5% were food panels.
  • Food panel ordering had dropped by 55% in absolute numbers since 2013.

In the commentary, it is noted that food serum IgE panels are not recommended “because more individuals will have detectable IgE sensitization than true symptoms” (aka false positives). “There is still a long way to go regarding educating families and nonallergist provideres on approaches to diagnosis of IgE-mediated food allergies.”

My take: This is a constant struggle. Everyday families want allergy testing on the assumption that it will be useful in treating their GI symptoms. Though dietary changes are frequently helpful in patients with GI problems, food allergy panels are likely to lead to more trouble than benefit.

Related blog posts:

Siesta Key, FL

Best of Allergy Articles 2021 -Cow’s Milk Allergy/Allergic proctocolitis (Part 4)

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In Pediatrics, supplement 3 summarizes 76 articles: Synopsis Book: Best Articles Relevant to Pediatric Allergy, Asthma and Immunology

Some of the studies that are most relevant to pediatric GI doctors I am reviewing for this blog over the next/past few days.

A Lemoine et al. Clin Exp Allergy  2021:51(9):1242-1245.  doi:10.1111/cea.13896. Rectal bleeding and cow’s milk protein-induced allergic proctocolitis: A prospective study

In this prospective cohort of infants (in France) with food protein-induced allergic proctocolitis (FPIAP) (n=76), all infants had rectal bleeding (RB) which resolved with cow’s milk protein (CMP) elimination. After the initial oral food challenge (OFC) which took place 2 to 8 weeks after resolution of rectal bleeding, OFC was repeated every 2 months.

Key findings:

  • Only 31% failed the initial OFC
  • The median age of tolerance, for those with a confirmed FPIAP based on OFC, was 6.8 months, with >75% of the cohort tolerant by 10 months of age

My take:

  1. This study shows that the majority of infants with RB probably do not have FPIAP. In those that do have FPIAP, earlier challenge is reasonable in the majority.
  2. FPIAP is generally mild and self-limited. Diagnosis is hampered by lack of validated criteria.
From Siesta Key, FL

Inconclusive Screening for Cystic Fibrosis and Outcomes

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T Gonska et al. Pediatrics 2021; 148: e2021051740. Outcomes of Cystic Fibrosis Screening-Positive Infants With Inconclusive Diagnosis at School Age

Background: : Cystic fibrosis (CF) screen-positive infants with an inconclusive diagnosis (CFSPID) are infants in whom sweat testing and genetic analysis does not resolve a CF diagnosis

Methods: Prospective, longitudinal, multicenter, Canada-wide cohort study of CFSPID for a mean of 7.7 years

Key findings:

  • A CF diagnosis was established for 24 of the 115 children with CFSPID (21%) — either because of reinterpretation of the cystic fibrosis transmembrane conductance regulator genotype or because of increase in sweat chloride concentration ≥60 mmol/L. Those with initial sweat chloride concentration ≥40 mmol/L were most likely to receive a diagnosis of cystic fibrosis.
  • Children with CFSPID were pancreatic sufficient and showed normal growth until school age and had good pulmonary outcomes (similar to healthy controls)
  • In the associated commentary by P Chakraborty et al (Maximizing Benefits and Minimizing Harms: Diagnostic Uncertainty Arising From Newborn Screening), the authors note that while newborn screening (NBS) offers benefit of early diagnosis, some families can be harmed by false-positive tests or inconclusive results. Furthermore, “these issues of uncertainty are increasingly important to consider as the scope of NBS programs and their use of genomic technologies expands.”

My take: With CF, this study shows the need to monitor those with inconclusive studies. More broadly, the use of genomic testing is leading to more frequent inconclusive results in many areas and sometimes leaving more questions than answers.

Related blog posts:

From NPR, December 2021

Favorite Posts of 2021

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I am happy to say that this is the last nightcall that I will have this year!

Today, I’ve compiled some of my favorite posts from the past year. I started this blog a little more than 10 years ago. I am grateful for the encouragement/suggestions from many people to help make this blog better. Also, I want to wish everyone a Happy New Year.

GI:

IBD:

LIVER:

Nutrition:

Other Topics:

Thanks to Jennifer

Trisomy 18 Trends over the Last 20 Years

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TA Fick et al. J Pediatr 2021; 239: 206-211. Trisomy 18 Trends over the Last 20 Years

Methods: A retrospective analysis using the Kids’ Inpatient Database from 1997 to 2016 (10,151 admissions) was performed for trisomy 18.

Key Findings:

  • Gastrostomy tube placement increased 12-fold during the study period, tracheostomy increased 11-fold, and cardiac intervention increased 5-fold
  • The overall mortality rate (based on inpatient data) decreased in those with trisomy 18 from 32% in 1997 to 21% in 2016
  • Limitations: their data are limited to only hospitalized children and do not include all patients with trisomy 18 or capture population incidence

My take: This study documents a change in the approach to treating children with Trisomy 18. Compared to 20 years ago, these children are being offered more medical/surgical treatments rather than only palliative interventions.

Emerging Data on Risankizumab for Crohn’s Disease

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From Gastroenterology and Endoscopy News: New Anti–IL-23 Therapy Shows Benefit in Crohn’s Disease

An excerpt:

Two phase 3 placebo-controlled trials with the immune modulator risankizumab demonstrated control of Crohn’s disease whether or not patients had previously received a biologic agent.

Rates of clinical remission at 12 weeks with the interleukin (IL)-23 inhibitor risankizumab (Skyrizi, AbbVie), were about 48% in patients without prior exposure to biologic therapy and more than 40% in those with prior exposure…

The two trials, ADVANCE and MOTIVATE were presented together at the 2021 Digestive Disease Week (abstract 775a)…

Only 12% of patients in the placebo group achieved endoscopic remission versus 40.3% of those on the 600-mg dose of risankizumab (P<0.001). [Rates of endoscopic remission were higher in the biologic-naive (50.5%)]

My take: In addition to ustekinumab (already approved), a number of other therapeutic agents that target IL-23 are likely to be available soon to help manage Crohn’s disease. This includes risankizumab but others with phase 3 studies include brazikumab, mirikizumab, and guselkumab..

Slide from David Rubin Twitter Feed (March 2021). Ozanimod now approved.

Ustekinumab vs Adalimumab: Head-to-Head Study

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From Gastroenterology & Endoscopy News: Head-to-Head Trial Shows Similar Efficacy and Safety With Ustekinumab and Adalimumab

An excerpt:

The first head-to-head trial comparing ustekinumab and adalimumab has found the two drugs are similarly safe and effective in patients with moderate to severe Crohn’s disease…

Dr. Scherl and her co-investigators in the SEAVUE trial randomly assigned 386 biologic-naive patients with Crohn’s disease to receive one year of treatment with either ustekinumab or adalimumab at standard on-label doses, with no dose escalation throughout the study period and no concomitant immunomodulators...

The findings, which were presented at the 2021 annual meeting of the European Crohn’s and Colitis Organisation (oral presentation OP02), showed that after one year of treatment, 65% of patients who received ustekinumab and 61% of those who received adalimumab achieved clinical remission, defined as a CDAI below 150...[And] similar additional outcomes, including clinical response at one year (72.3% for ustekinumab vs. 66.2% for adalimumab), corticosteroid-free remission at one year (60.7% vs. 57.4%, respectively), endoscopic remission at one year (28.5% vs. 30.7%) 

My take: This study indicates that ustekinumab likely has similar safety and efficacy as adalimumab (though the study did not allow dose escalation or immunomodulators); thus, it could be positioned as a first-line treatment. It is administered less frequently as well.

Related blog posts:

Is An Unproven Medication Worth More Than the EPA or NASA?

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For those who have not followed the FDA’s controversial decision of approving Aducanumab for the treatment of Alzheimer’s disease, the NEJM has two useful commentaries:

Key points -from 1st article:

  • “Biogen…has announced a list price of $56,000 –10 times the evidence-based benchmark recommended by the independent Institute for Clinical and Economic Review…if even 10% of U.S. patients with Alzheimer’s disease were prescribed aducanumab, drug spending for Medicare Part B would increase from $37 billion to $69 billion per year”
  • The authors note that Medicare Part B payments rely on average sales price (ASP) from private insurers rather than a direct negotiated price; thus, the higher the price for private plans (even if poorly covered), the higher the Medicare rate
  • Hospitals and physicians are incentivized at higher prices due to receiving a 4-6% reimbursement price over the acquisition price
  • “The $56,000 price for aducanumab is a rational manufacturer response to an irrational insurance system.”

Key points -from 2nd article:

  • By one estimate, the potential cost will exceed the budgets of agencies such as EPA or NASA
  • “In granting accelerated approval to aducanumab, the FDA concluded that the drug’s ability to reduce amyloid plaques was reasonably likely to translate into clinical benefits. But this claim is hotly contested and was not presented to the FDA’s advisory committee, which voted against recommending approval of the drug because of the lack of a demonstrated clinical benefit”
  • If Medicare refuses to cover medication, this would leave a burden to state budgets. “As a legal matter,…state Medicaid programs are required to cover nearly all FDA-approved drugs.”
  • “Congress could adopt new legislation specifying that state Medicaid programs need not cover aducanumab…Protecting state budgets shouldn’t require Medicare to cover an expensive drug with unproven clinical benefits.”

My take: This type of huge fiscal burden may provide the rationale for Medicare and Medicaid to reexamine whether/how they cover expensive FDA-approved medications.

Related blog posts:

NASPGHAN Toolbox App

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Patrick Reeves passed along the following helpful information about the NASPGHAN toolbox:

The NASPGHAN Fellows committee, working in close partnership with the NASPGHAN Technology and Training committees, has developed an App named, “The NASPGHAN Toolbox”.

The App is equipped with ready access to: clinical calculators, guidelines and algorithms, medication guides, patient education resources, and more. You can access the Toolbox via its URL (https://toolbox.naspghan.org/) on your phone or computer.

The NASPGHAN team hopes this will enhance your day-to-day patient care of children with gastrointestinal disorders.

Some highlights:

Some screenshots: