Esophageal Disorders: POEM in Kids, Mitomycin C for Refractory Strictures

At our recent national meeting, Dr. Peter Kahrilas indicated that POEM (Per-oral Endoscopic Myotomy) was now the treatment of choice for most adults with achalasia (#NASPGAN19 Postgraduate Course -Part 3).

A Chone et al (JPGN 2019; 69: 523-7) provide recent multicenter retrospective data on POEM in the pediatric age group (mean age 14 years), n=117.

Key findings:

  • Clinical success, defined as Eckardt score ≤3 during followup, was achieved in 90.6% of cases. The Eckardt score was >3 in 5 (4.3%) and data was missing in 6 (5.1%)
  • Adverse events included 1 case with significant bleeding, 2 cases of aspiration pneumonia (related to anesthesia), 1 esopleural fistula (managed endoscopically), and 6 mild AEs (4 mucosomtomies, 2 subcutaneous emphysema)

Additional related blog posts:

D Ley et al (JPGN 2019; 69: 528-32) provide retrospective data on 39 patients, median age 19 months, with refractory esophageal strictures which were treated with mitomycin C.  The authors considered mitomycin C after a minimum of two previous dilatations.

Key findings:

  • Etiology: The majority had strictures/stenosis associated with esophageal atresia (n=25) followed by caustic ingestion in 9.
  • Number of stenosis: The majority (n=35) had a single stenosis.
  • In 26 patients (67%), topical application of mitomycin C was considered a success based on a reduction in the number of dilatations.  In this group, the number of dilatations dropped from 102 to 17 over a comparable period.
  • 16 (41%) never required further dilatation following mitomycin C application

My take: This study provides some of the best evidence that mitomycin C may be helpful.  Long-term followup and more studies are needed.

Related blog posts:

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.

Lincoln Park, Chicago

#NASPGHAN19 Postgraduate Course (Part 3)

Here are some selected slides and notes from this year’s NASPGHAN’s postrgraduate course. There may be some errors of omission or transcription.

Link to the full NASPGHAN PG Syllabus 2019 (Borrowed with permission)

Functional/Motility Session

95 Carlo Di Lorenzo, MD, Nationwide Children’s Hospital. Evaluation Testing for functional disorders: The indispensable, the useless, the dangerous and treatment strategies in NERD and functional dyspepsia.

This was the best lecture of the day!!! (Hence a lot of slides follow)

  • Families never complain about doctors missing irritable bowel syndrome and anxiety. They may complain about missing diagnosis which are controversial with regarding to chronic pain (‘chronic appendicitis, gallbladder dyskinesia, ‘mild’ IBD, median arcuate ligament syndrome, and food allergies)
  • Functional disorders, but not organic disorders, can cause ‘constant’ pain. “Tried everything.”  Functional disorder patients frequently have side effects with everything.
  • Listen to patient and sit while listening.
  • Early diagnosis of functional disorder associated with higher long-term resolution
  • Testing –only tests that are cost-effective: celiac disease and stool calprotectin.  “Don’t get KUB for constipation.”
  • Endoscopy does not improve outcomes in children with functional GI disorder (FGID)
  • Eosinophilic esophagitis (EoE) treatment does not help abdominal pain but can help if patient has dysphagia
  • Abdominal wall pain is often overlooked.  Check Carnett sign.

 

112 Peter Kahrilas, MD, Northwestern Medicine  Achalasia

  • Achalasia likely develops after an infection in a susceptible host
  • Discussed POEM as newer treatment. It appears to be more effective than either Heller myotomy or pneumatic dilatation in adults.  So far, there is limited experience in pediatrics though it appears to mirror adult experience

124 Julie Khlevner, MD, Morgan Stanley Children’s Hospital Evaluation and treatment strategies in NERD and functional dyspepsia

  • In patients with NERD, hypermetabolizers of PPIs may need higher dosing.
  • Neuromodulators (not FDA approved) used for PPI-nonresponders.  Cognitive behavioral therapies may be helpful as well.
  • Functional dyspepsia with reflux symptoms are more likely to respond to PPIs than those with dyspepsia symptoms
  • A Japanese herb, rikkunshito, may be helpful for functional dyspepsia

136 Robert J. Shulman, MD, Children’s Nutrition Research Center Role of diet in managing of IBS

Key points:

  • Vast majority of low FODMAPs studies show “too much bias” due to lack of blinding in study designs.
  • Nutritionists are needed to guide diet.  Kids (families) do not follow these diets well.
  • Most who are going to respond to diet will do so within 7-10 days.

Disclaimer: NASPGHAN/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. The discussion, views, and recommendations as to medical procedures, choice of drugs and drug dosages herein are the sole responsibility of the authors. Because of rapid advances in the medical sciences, the Society 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. Some of the slides reproduced in this syllabus contain animation in the power point version. This cannot be seen in the printed version.

 

POEM for Achalasia in Children

Briefly noted: S Miao et al. JPGN 2018; 66: 257-62.  In this retrospective study, the authors examined the use of peroral endoscopic myotomy (POEM) in children.  They , describe a successful outcome in all 21 patients (range 11 months to 18 years).  Complications included subcutaneous emphysema (n=4), pneumoperitoneum (n=1), mediastinal emphysema ((n=4), pneumonia (n=1) and mucosal injury (n=1). The authors: “although Heller myotomy is still widely accepted as the standard treatment for achalasia in children, POEM …may provide a better treatment …due to less hospitalization, less trauma, …and long-term efficacy.”

Related blog posts:

Amber Cove, Dominican Republic

Achalasia -Updated Epidemiology

In this new era of high resolution manometry, there is an increasing incidence of achalasia.

Briefly noted:

JA Duffield et al. Clin Gastroenterol Hepatol 2017; 15: 360-5. In this study from South Australia, using a large database (2004-2013), the annual incidence of achalasia was between 2.3 and 2.8 per 100,000 persons. Mean age at diagnosis was 62 years.

S Samo et al. Clin Gastroenterol Hepatol 2017; 15: 366-73. In a similar study from Chicago, the authors estimated that the yearly city-wide incidence averaged 1.07 per 100,000; however the average in the neighborhood closest to the hospital (and possibly with better case capture) was 2.92 per 100,000.

My take: These studies identified incidence rates that are about double the rates that were reported prior to the availability of high resolution manometry.

Related blog posts:

New Mutations: Achalasia, Pseudoobstruction, & IBD

The ability to use whole exome sequencing and widely available genetic testing is yielding a plethora of new information regarding the genetic causes for many conditions.  In gastroenterology, here are a few recent examples:

  • Shteyer E, et al. “Truncating mutation in the nitric oxide synthase 1 gene is associated with infantile achalasia.” Gastroenterology. 2015 Mar;148(3):533-536.e4. doi: 10.1053/j.gastro.2014.11.044. Epub 2014 Dec 3.
  • Bonora E, et al. “Mutations in RAD21 Disrupt Regulation of APOB in Patients with Chronic Intestinal Pseudo-Obstruction” Gastroenterology 2015; 148: 771-82.  Genetic defect in RAD21 identified in Turkish family with consanguinity; in addition, APOB48 serum levels was identified as a potential biomarker for intestinal pseudo-obstruction and intestinal ganglion numbers.
  • Alonso A, et al. “Identification of Loci for Crohn’s Disease Phenotypes Using a Genome-Wide Association Study.” Gastroenterology 2015; 148: 794-805. Variants in MAG11, CLCA2, 2q24.1, LY75 identified as associated with Crohn’s phenotypes.

For me, I am not sure whether these findings should be considered mundane or amazing. On the one hand, each of the findings helps understand these diseases; yet, I came across all of these articles in the span of 24 hours and from the same journal.

What does the law require with regard to food allergen labeling? (plus one)

From APFED twitter feed: What does the Food Allergen Labeling and Consumer Protection Act require?: http://youtu.be/nhBd9iTYkUQ?a (<2 minute video)

Plus one more reference on Achalasia:

A recent review on achalasia highlights the recent advances in our understanding of this disorder (Gastroenterol 2013; 145: 954-65). This post is mainly to note it as a useful reference.

Specific topics covered include the following:

  • High-resolution manometry
  • Criteria for achalasia diagnosis
  • Physiology/pathogenesis/pathophysiology.  Achalasia is “an autoimmune disease targeting esophageal myenteric neurons with both a cell-mediated and antibody-mediated attack directed against an as yet unidentified antigen.”
  • Subtypes (Figure 2 shows, in color, manometric pattens with each subtype)

Related posts: