Category Archives: Pediatric Gastroenterology Intestinal Disorder

CCFA Conference Notes 2014 (part 1)

Posted on by

Each year our local CCFA chapter holds a one day seminar with separate lectures for health care providers and families.  Overall, it is a good opportunity to hear ‘cutting edge’ material.  I did not pick up as much at this year’s seminar as in previous years, but will highlight what I thought was most important.

Key points:

  1. Symptoms are not accurate at determining effectiveness of IBD therapy.
  2. More frequent use of objective markers are needed to optimize treatment.  Mucosal healing is starting to be a target in clinical practice, but limited by number of medications available.
  3. Stricture classification and operative techniques were reviewed.
  4. IBD frequently results in psychological problems: anxiety, depression, pain, sleep. 15% of kids and 25% of adults are having thoughts of death on screening tool intake.
  5. Fecal microbiota transplantation (FMT) –not enough data to recommend for IBD.  Clinical trials ongoing.

Debate: What should be the End Points in Therapy? 

  • Tanvi Dhere (Emory): Goal: clinical symptoms
  • Cary Sauer (Emory Pediatrics): Goal: mucosal healing and normal bloodwork

In my opinion, this was the most thought-provoking and best presentation

Mucosal healing (MH) consensus definition –normal mucosa after previously abnormal with complete absence of ulceration, macroscopic and histologic signs of inflammation.  In practice MH = absence of ulcerations.

Reasons why mucosal healing as a target is problematic:

  • Problems with MH –not validated.  No long-term data utilizing endoscopic scoring indices of MH.
  • MH relies on a binomial endpoint –Yes or no, but there may be intermediate endpoints.
  • How likely is MH (different definitions in these studies)?  SONIC –MH in 43.9% of combination Rx (30.1% in those with infliximab monotherapy); EXTEND (Adalimumab) 27% and 24.2% 12/52 weeks; MUSIC (certolizumab at 10/54 weeks) 11.5% and 18.9%.

In practice, Mayo Score 0-1 both considered to have MH.

MayoScore Visual

Images above online at www.nature.com

In small study, MH at 1 year were not associated with improved outcomes at 5 years.  Risks of MH: more procedures, more costs of treatment, and potential for more complications.

Dr. Sauer’s reply.  Three simple questions –why should I try to target MH, is it possible, what is needed to get this done?

  1. If the goal were only an asymptomatic patient – why do screening colonoscopy in the general population, much less in IBD?
  2. In IBD, long-term evolution of IBD (Cosnes J et al. Inflamm Bowel Dis. 2002 Jul;8(4):244-50) is toward structuring and penetrating disease. CD Evolution This needs to be modified if possible.

Why MH? Improved symptoms, better quality of life, less likely to develop colon cancer, and it is an objective measure of treatment response.

  • In MH patients, less steroids and fewer flares over 2 year period.
  • MH healing patients have sustained clinical benefit over 96 months.
  • With MH, there is a decreased colectomy in UC.  In one study, there was a lower  colectomy rate at 8 years if colonic CD (62% vs 8%), decreased steroids in CD, decreased hospitalizations, & decreased fistulae.

Is MH possible in clinical practice?  The accuracy of CDAI to detect endoscopic healing is low in patients with CD. (Bouguen G et al Clin Gastrohep 2014).  More frequent adjustments in medical therapy –could lead to MH in up to 80% over 80 week study period.  Same story in UC (Bouguen G et al IBD 2014).

What do I need to do to obtain MH? Endoscopy (or MRE), maximize medications (checking levels), change medications, and most important –set a target. “Adjusting infliximab dose alone could lead to MH in up to 60%.”

When to assess for MH?  Consider endoscopy at 6 months into treatment if symptoms and at 12 months if in clinical remission.

Other viewpoints on MH from panel:

Dr. Loftus –“I think of this like oncology.” He agreed with using the best evaluating tool 6 months into treatment.  Cross-sectional imaging is often more helpful, but may need more than one tool.

Dr. Long—“Are we going to check every 6 months?” No.  She stated that she does not do this and tries to avoid repeated endoscopic procedures if this will not change treatment.  Goal is to make sure patient is headed in right direction, often after starting therapy.  Dr. Long stated that stool biomarkers most useful for colonic disease.

Dr. Dhere—documenting MH is important for deescalating treatment.

Millie Long  “Quality of Care in IBD”

  • 75% of Crohn disease patients will need surgery, 10% in 1st year
  • “One way to gauge quality of care is to examine the degree of consistency in care”
  • High variability in care in IBD (Aliment Pham Ther 2007; 26: 1005-18)
  • “Over half of institutions with worst quality have mortality in normal range.” Outcomes may not occur until several years after treatment, thus more useful to measure process measures

PQRS IBD Quality Measures in Adults: 10 Measures

  • #1 Establishing/documenting IBD type, anatomic location, and activity
  • #2 Preventive care: corticosteroid sparing.  Steroids associated with mortality (OR 2.1 in TREAT registry)
  • #3 Preventive care:  Preventing bone loss.  Limiting steroid use.  Recommend weight-bearing exercise, Quit Smoking, Measure DEXA, added Calicum/Vit D/Bisphosphonates
  • #4: Vaccination –pneumococcal vaccine.  Avoid live virus vaccines
  • #5 Vaccination –influenza vaccine, zoster vaccine
  • #6 Testing for latent TB prior to anti-TNF
  • #7 Testing for hepatitis B virus
  • #8 Testing for C diff with patients hospitalized with IBD
  • #9 VTE prophylaxis in adult IBD patients.  Risk assessment on admission to hospital is recommended.  IBD patients have 1.5-3.5-fold higher risk of VTE àwhich can increase mortality risk
  • #10 Screening for tobacco.  Tobacco use after surgery increases recurrence by 2.5-fold.  It also increases risk for reoperation.

Last year’s notes:

Disclaimer: These blog posts are for educational purposes only. Specific dosing of medications (along with potential adverse effects) should be confirmed by prescribing physician.  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.

Think Like a Doctor –Another Reason for Cyclic Vomiting

Posted on by

When cyclic vomiting pattern starts in young adults, the differential diagnosis is different than in toddlers.  A case in point is a recent think like a doctor column from NY Times.

The initial presentation described a 25-year-old man who gets sweaty and nauseated and starts vomiting uncontrollably every few weeks.

1st the link with the case challenge: challenged Well readers to figure out

A big clue in this case was the fact that hot showers ameliorated his symptoms.

Now the answer, full link: http://nyti.ms/1lcPJKj 

“The correct diagnosis is…

 

 

 

Cannabinoid hyperemesis caused by smoking synthetic marijuana.”

Related blog post: Diet or drugs for cyclic vomiting syndrome | gutsandgrowth

Briefly Noted…Scoliosis, Cystic Fibrosis, Specific Carbohydrate Diet, GGT, Surgeon General

Posted on by

“Net effect of scoliosis surgery on gastric emptying, upper gastrointestinal symptoms, and clinical nutritional status was minimal”  according to this prospective study of 31 children: JPGN 2014; 58: 38-45.

Lactobacillus reuteri ATCC55730 was associated with reduced pulmonary exacerbations (but not improved FEV1) in 61 patients with cystic fibrosis who were enrolled in a prospective randomized, double-blind, placebo-controlled study over 6 months between 2007-2009 (JPGN 2014; 58: 81-86).

A retrospective chart review of seven children with Crohn disease who were treated with the specific carbohydrate diet with an average diet duration of 14.6 months showed that all symptoms resolved and laboratory studies improved or normalized.  (JPGN 2014; 58: 87-91). More studies on this diet are expected to be published soon.

Data from 200 preterm infants and 383 term infants help provide updated GGT (gamma-glutamyl transferase) reference values.  In preterm infants during 1st 7 days: 141 ± 89 U/L, then between days 8-28: 131 ± 85 U/L.  In term infants the values were similar 140 ±86 U/L and 145 ± 87 U/L respectively. (JPGN 2014; 58: 99-101).

And from NEJM twitter feed, Congress may allow the NRA veto power in the selection the next surgeon general: http://nej.md/1nECFlM.

Training Not Meeting Procedure Thresholds for Fellows

Posted on by

With expanding numbers of pediatric GI trainees, it is even more concerning that training centers have had difficulty providing adequate experience for their trainees.  In a recent study (JPGN 2014; 58: 27-33), not one of 12 centers was able to meet the threshold for all of the procedures.

The authors provide NASPGHAN procedural competency guidelines in Table 2. The 2013 guidelines lowered the threshold for almost all procedures in comparison to the 1999 guidelines.  One notable exception was foreign bodies which was increased to 10. Then, the authors examined the frequency of procedures actually performed between 2009-2011.

Key findings (with the lower thresholds)

  • Polypectomy (threshold, n=10) 67% of programs meeting 2013 NASPGHAN guidelines
  • Control of nonvariceal bleeding/Sclerotherpy/Variceal band ligation (threshold, n=15) 17% of programs meeting 2013 NASPGHAN guidelines. Mean number per fellow of nonvariceal bleeding cases was 2 procedures. Mean number per fellow of banding/sclerotherapy was 7 procedures. With the 1999 guidelines, the threshold had been 35 for these procedures combined.
  • Esophageal, pyloric and duodenal stricture dilatation (threshold, n=15) 42% of programs meeting 2013 NASPGHAN guidelines
  • PEG (threshold, n=10) 42% of programs meeting 2013 NASPGHAN guidelines (training not available at 42% of these institutions)
  • Percutaneous liver biopsy (threshold, n=15) 67% of programs meeting 2013 NASPGHAN guidelines
  • Foreign-body removal (threshold, n=10) 58% of programs meeting 2013 NASPGHAN guidelines

It is noted that the 2013 guidelines “reclassified control of variceal and nonvariceal bleeding from a level 2 to a level 1 procedure, stressing that expertise in hemostasis should be achieved during fellowship.” “Without supplemental training, none of these programs, which are likely representative of most programs, were able to provide sufficient opportunities to meet the NASPGHAN guidelines.” Besides spending more time working with adult gastroenterologists, other potential ways to gain more experience includes simulators and hands-on training courses.

Take-home message: Pediatric gastroenterology training centers are not providing enough procedural experience and need to make rotating with adult gastroenterologists mandatory. It is unfair to trainees and vulnerable patients to consider their training complete with these obvious deficiencies.

When families ask the newly trained pediatric gastroenterologist how many cases like this that they have completed, how will they feel if the honest answer is two?

Related blog post:

Training for Tomorrow | gutsandgrowth

More on Gut Microbiome and Crohn Disease

Posted on by

Earlier this week on NPR there was a story summarizing the altered microbiome in Crohn disease and a related recent paper; here’s the link: Mix Of Gut Microbes May Play Role In Crohn’s Disease.  Other media outlets covered the story too:

The graphical abstract (Cell Host & Microbe, Volume 15, Issue 3, 382-392, 12 March 2014) is noted below:

Graphical Abstract

The link to the full study is listed below if you want to see the source article.  The amount of data that is presented is impressive but easy to follow with the figures:

Full link to article (from Kipp Ellsworth twitter feed): http://goo.gl/603Rbz 

Related blog posts:

“If You Never Give Up, You Cannot Possibly Lose”

Posted on by

Recently I was reviewing the “Black Knight scene” from Monty Python’s Holy Grail.  This scene in which the Black Knight continues to insist on fighting King Arthur even after losing all of his limbs came to mind as I was reading a recent study (JPGN 2014; 58: 226-36).  The blog title comes from an explanation of the scene from John Cleese who intended the scene to mock this philosophy. (Wikipedia link: Black Knight (Monty Python) – Wikipedia, the free encyclopedia)

The study is another trial of a proton pump inhibitor (rabeprazole) for 1- to 11-month old infants with symptomatic GERD.  In the discussion the authors note that this is the “fourth DB randomized placebo-controlled study published in the last 4 years that fails to show the efficacy of PPIs to treat symptomatic GERD in infants younger than 1 year.”

If anything, the design of this study should have allowed a therapeutic effect to be witnessed if present.  Infants selected for participation (n=268 in the double-blind phase) had been responsive to a 10 mg open-label usage of rabeprazole before randomization.  Yet, those infants who continued to receive 5 mg or 10 mg daily fared no better than placebo-treated patients.

The good news: no new safety signals in those who were treated compared to placebo.

The findings of this study are in marked distinction to clinical practice which has embraced PPIs in all age groups. In the same issue of JPGN, using a national database, De Bruyne et al (JPGN 2014; 58: 220-25) show a huge increase in PPI usage over the past decade in the Netherlands, especially in children ages 2 years and younger.  From 2004 to 2008, use of PPIs nearly doubled in this population.

The rabeprazole study manuscript which had nearly as many pediatric GI investigators as enrolled patients discusses the potential drawbacks of PPI therapy in infants including enteric infections like Clostridium difficile, lower respiratory infections (e.g.. pneumonia), and “perhaps even an increased incidence of necrotizing enterocolitis in premature infants.”  Unlike the Black Knight, after four blows to the PPI cause, the authors recommend yielding on PPIs except under much more stringent criteria (1 of 3):

  • Nonimproving symptoms at 1 year of age & resistant to conservative measures
  • Presence of underlying conditions that predispose to a natural history of severe chronic unremitting reflux
  • Erosive reflux esophagitis proven on endoscopy

Take-home message: PPIs have not been shown to be effective in infants…again!

Related blog posts:

Even the Experts Agree: pH-MII is a “Flawed Test”

Posted on by

A recent study (JPGN 2014; 58: 22-26) reports on the combination of a new technique of intraesophageal pressure recording (IEPR) along with multichannel intraluminal impedance with pH (pH-MII).  While this prospective study is small with only 20 children who had a history of chronic intractable cough, some of its observations are important, especially for those who have embraced pH-MII.

In determining whether the pH-MII studies were abnormal the authors relied on symptom index (SI) defined as the number of symptoms associated with reflux/total number of symptoms.  SI is considered positive if >50%.  In addition, the authors calculated the symptoms sensitivity index (SSI) which is defined as the total number of reflux episodes associated with symptoms/total number of reflux episodes; it is considered positive if it is >10%.  The authors note SAP and SI have a comparable positive predictive value and “our experience suggests that SAP calculation using software is unreliable.”

Key Results/Discussion:

  • IEPR changed the diagnosis in 15-20% of patients depending of scoring index used.  That is, IEPR assisted the detection of reflux-associated cough.
  • IEPR detected 106% more coughs than patient report alone.  Thus, this study, if accurate, indicates that “symptom reporting during pH or pH-MII testing is significantly flawed and, if possible, should not be used alone for clinical decision making.”
  • “We did not find a significant association between cough production and the height of the refluxate.”
  • The authors argue that since nonacid reflux can be associated with cough and is not always detected with pH-MII, that this could “explain why studies that have tried to use pH criteria to predict clinical outcome after acid suppression therapy have been negative.”  The two studies cited at that point by the authors were landmark studies (referenced below) showing that proton pump inhibitors are not effective in children or adults in improving asthma.  I think the authors’ comment misses the importance of these studies entirely.  There are no proven effective GERD (acid or nonacid) therapies that alter the course of asthma.

Take-home message from authors: “Studies are now needed to determine whether this increased detection improves therapeutic outcomes, but clearly, relying on symptom reporting by patients is flawed and clinical decision making based on patient report alone should be done with caution.”

Referenced studies:

  • JAMA 2012; 307: 373-81
  • NEJM 2009; 360: 1487-99

Related blog posts:

Calprotectin: Part of diagnostic algorithm for IBD?

Posted on by

Full text available at this link from Jeremy Adler: cghjournal.org/article/S1542-3565(13)01044-6/abstract#.Ut5vnV5z8Cw.twitter …

This article describes the use of fecal calprotectin (FC) levels as a screen for inflammatory bowel disease.  The false-negative rate for this assay is related to pre-test probability of having IBD.  Thus, in patients with a low probability of IBD, a normal calprotectin may allow avoidance of endoscopic evaluation and may be “particularly cost-effective when baseline clinical suspicion for IBD is low to moderate…the low FC cutoff value of 50 μg/g would substantially reduce the likelihood of false-negative FC, minimizing delayed diagnosis of true IBD.” In those with persistent symptoms, the article’s algorithm recommends proceeding with endoscopic evaluation.

Excerpt from abstract:

Conclusions

Screening adults and children to measure fecal levels of calprotectin is effective and cost-effective in identifying those with IBD on a per-case basis when the pre-test probability is ≤75% for adults and ≤65% for children. The utility of the test is greater for adults than children. Increasing the FC cutoff level to ≥50 μg/g increases diagnostic accuracy without substantially increasing total cost.

Related blog posts:

Missing ingredients in TPN -Case Report

Posted on by

Recently one of my radiology colleagues, Dr. Laura Hayes, put together (lead author) a presentation (poster) for an upcoming meeting.  The main focus of the presentation is a TPN-dependent toddler who presented with refusal to walk due to copper deficiency.

Attached is a link to the presentation: TPN Copper.  This link is a power point presentation with numerous radiographs and even bone scan images.

Key points:

  • All TPN components except dextrose have been in periods of shortage over the last few years.
  • TPN-dependent patients may not be receiving all the needed components and their physicians may not have been notified of the specific shortage(s).
  • Copper deficiency leads to reduced activity of numerous enzymes important for function of bone, blood, skin, nervous system and hair.
  • Subperiosteal hemorrhage leads to the periosteal thickening seen in this case and is associated with the bone pain our patient experienced.
  • Increased losses of bilious fluid can increase the risk of copper deficiency due to the excretion of copper in bile.
  • Other TPN-related deficiencies reviewed include thiamine deficiency (Wernicke’s encephalopathy), Vitamin D deficiency (Rickets), and Vitamin C deficiency (Scurvy).

Another recent case report:

Oestreich AE, Cole CR. Vigorous periosteal reaction secondary to copper deficiency in an infant on total parenteral nutrition. (2013) Pediatr Radiol 43:1411-1413.

Related Blog Posts: