Tag Archives: achalasia

Botox for Pediatric Gastrointestinal Disorders

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M Homan et al. J Pediatr Gastroenterol Nutr. 2024;79:1096–1105. Open Access! Drugs in focus: Botulinum toxin in the therapy of gastrointestinal disorders in children (Review)

Botox has been used for the following:

  • Achalasia
  • Cricopharyngeal achalasia
  • Retrograde cricopharyngeal dysfunction
  • Delayed gastric emptying
  • Anal achalasia
  • Constipation after Hirschsprung disease surgery
  • Selected functional constipation
  • Chronic anal fissure

With regard to achalasia, “Botox can be considered only in patients in whom rapid weight gain is important to improve surgical outcomes.”

With regard to gastroparesis/delayed gastric emptying in children, the authors note the following:

‘A recent retrospective study from the Mayo Clinic analysed the response to intrapyloric Botox in children (n = 20) with gastroparesis and concluded that intrapyloric Botox injection in children is safe and can provide temporary relief for patients with refractory upper gastrointestinal symptoms with and without gastroparesis.43 [This was] a meta-analysis, including six studies, 160 patients, which showed that 68% of patients responded to intrapyloric Botox irrespective of the presence of gastroparesis, while among patients diagnosed with gastroparesis the therapeutic response was 66%.43 These results suggest that intrapyloric Botox can be effective not only in children with gastroparesis but also in children with refractory functional upper gastrointestinal symptoms.’ In their conclusion, the authors note “Intrapyloric Botox injection is increasingly used for the treatment of gastroparesis but evidence supporting its use in children is still scarce.”

My take: Overall, this is a helpful review.

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Manometry for Persistent Dysphagia in Eosinophilic Esophagitis

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D Yogev et al. JPGN Reports; 2024 https://doi.org/10.1002/jpr3.12083. Open Access! Manometric findings in children with eosinophilic esophagitis and persistent post-remission dysphagia

In this 10-year retrospective review (2013-2023), the authors reviewed children with EoE referred for high-resolution impedance manometry (HRIM) due to persistent dysphagia despite histologic healing (i.e., <15 Eos/hpf).

Key findings:

  • Among a cohort of ~1500 children, only 4 patients met inclusion criteria:  histologic remission (<15 eos/hpf) and absence of fibrostenotic features on endoscopic evaluation
  • Thus, the estimated prevalence of post-remission dysphagia in this cohort was exceedingly rare (<0.05%).
  • On HRIM, all four patients had a hypomotile esophagus and abnormal bolus clearance. Lower esophageal sphincter integrated relaxation pressure values were normal in three patients and elevated in one. Two patients were diagnosed with ineffective esophageal motility, one with aperistalsis and one with achalasia type 1.
Manometry findings from 12 yo diagnosed with Achalasia Type 1

Discussion:

  • “Endoscopic evaluation of fibrosis is somewhat limited as less than 50% of biopsies contain an adequate sample of the lamina propria for evaluation..[Also, these] patients did not undergo endoluminal functional lumen imaging (Endoflip) which has been recently shown to correlate with fibrotic changes of the esophagus in pediatric patients…Nonetheless, this case series highlights the fact that esophageal dysmotility can persist, even in the absence of endoscopic or histologic findings”
  • The authors “did not study all EoE patients treated in our facility who had persistent dysphagia despite histologic remission of EoE, but rather explored just those who were referred for manometry. This methodology creates a risk of referral bias.”

My take: Though there is a referral bias due to the methodology, this study suggests that persistent dysphagia is rare in children who achieve EoE histologic remission. In addition, in those with significant dysphagia despite improvement in EoE, manometry is worthwhile.

A related study suggests that achalasia may be more common in patients with Klinefelter syndrome, though still quite rare: L Miller et al. JPGN Reports 2024: https://doi.org/10.1002/jpr3.12084 Open Access! Achalasia in Klinefelter syndrome: A suspected pediatric case as well as prevalence analysis suggesting increased risk in this population

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Who is Going to do POEM (Peroral Endoscopic Myotomy) in Children?

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AA Mencin et al. JPGN 2022; 75: 231-236. Peroral Endoscopic Myotomy (POEM) in Children: A State of the Art Review

This is a terrific review of POEM; it explains the procedure technique (with pictures), outcomes, indications and adverse events. The paper indicates that more than 300 children (as young as 11 months) have had POEM in published studies with good outcomes.

Interestingly, the authors state that 20-40 procedures “should be performed for competency and that 60 are required to achieve mastery.” Also, long-term data are lacking.

This paper extensively references a White Paper summary: DJ Scott et al. Gastrointestinal Endoscopy 2014; 80: 1-15. Open Access! Per-oral endoscopic myotomy white paper summary

My take: POEM has good data supporting its use, especially in adults. Still, it will be quite difficult for pediatric gastroenterologists to acquire sufficient expertise to do this procedure.

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“Exit Glacier” in Kenai Fjords National Park. This picture shows how much the glacier has retreated since 2005 due to global warming (the ice used to extend to this sign).

Briefly Noted: Pediatric Achalasia

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S Woodhull, DR Segar N Engl J Med 2021; 385:e64. DOI: 10.1056/NEJMicm2108193

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Achalasia Frequent in Patients with Eosinophilic Esophagitis

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M Ghisa et al. Clin Gastroenterol Hepatol 2021; 19: 1554-1563. Achalasia and Obstructive Motor Disorders Are Not Uncommon in Patients With Eosinophilic Esophagitis

In this study with 109 adults who were newly diagnosed with eosinophilic esophagitis (EoE), the authors consecutively performed high-resolution manometry (HRM). Key findings:

  • 68 (62%) had normal findings from HRM
  • 8 (7.3%) had achalasia (1 with type 1, 4 with type 2, and 3 with type 3)
  • 9 (8.3%) had major motor disorders of esophagus (& not achalasia) and 24 (15.6%) had minor motor disorders

These findings are important because the diagnosis of EoE could result in a diagnostic delay of concurring achalasia and because the presence of esophageal eosinophilia could perhaps play a role in the pathogenesis of achalasia (or vice versa). The finding of achalasia in 7.3% of this population is exponentially higher than the estimated prevalence of achalasia in the general population (10-16 cases per 100,000).

My take: In patients with EoE, further diagnostic workup is indicated if there are persistent symptoms.

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Watersound Beach, FL

Achalasia -More Complexity to Pathophysiology?

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RK Mittal et al. Gastroenterol 2020; 159: 864-72. Three-Dimensional Pressure Profile of the Lower Esophageal Sphincter and Crural Diaphragm in Patients with Achalasia Esophagus

This prospective study combined 3-dimensional high-resolution manometry (3D-HRM) with detailed analysis of computed tomography images to study the EGJ (esophagogastric junction) in patients with achalasia (n=12) and healthy controls (n=10). Key finding: Besides LES, the 3D pressure profile of the EGJ can indicate anatomic and functional abnormalities of the crural diaphragm muscle in patients with achalasia esophagus

Full text editorial: DA Katzka, M Fox. Gastroenterol 2020: 159: 821- 23. Achalasia: When a Simple Disease Becomes Complex

“In this edition of Gastroenterology, researchers led by Ravi Mittal …demonstrated an asymmetric, extrinsic CD (crural diaphragm) component to EGJ pressure superimposed on the intrinsic LES.”

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

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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)

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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.

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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.

Lincoln Park, Chicago

#NASPGHAN19 Postgraduate Course (Part 3)

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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

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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.”

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Amber Cove, Dominican Republic

Achalasia -Updated Epidemiology

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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.

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