Consequences of Individual Mandate Repeal

Link: NY Times: Obamacare’s Insurance Mandate Is Unpopular. So Why Not Just Get Rid of It?

An excerpt: Without the mandate, the C.B.O. has said for years, premiums would spike, and millions fewer Americans would have health insurance. The budget office’s most recent estimate, published last week, said that the ranks of the uninsured would rise by 13 million over 10 years, and that average premiums would be 10 percent higher than under current law.

 

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#NASPGHAN17 Annual Meeting Notes (Part 1): Neurostimulation for RAP, PSC-IBD, Organoids

This blog entry has abbreviated/summarized these presentations. Though not intentional, some important material is likely to have been omitted; in addition, transcription errors are possible as well.

William Balistreri Prize: Katja Kovaic et al. Neurostimulation for functional abdominal pain disorders in children –a randomized, double-blind, sham-controlled trial. This study enrolled 104 patients.  Lancet Gastroenterol Hepatol 2017; 2: 7272-37.

Summary slide:

Fellow Research Award: Symptoms Underestimate Endoscopic Activity in PSC-IBD. Amanda Ricciuto et al. Hospital for Sick Children.

Key points:

  • In patients with IBD-PSC, clinical remission based on clinical symptoms is not reliable indicator of histologic remission.
  • Patients with PSC-IBD are more likely to have active endoscopic disease even when in “clinical remission”
  • Calprotectin levels (not PUCAIs) are helpful in confirming clinical remission.  A calprotectin <93 mcg/g was optimal level in determining clinical remission
  • Better control of disease could improve clinical outcomes (eg. colon cancer, liver progression)

Keynote Address: Organoids: Current and future promise for changing treatment of gastrointestinal and liver disorders.  James Wells Cincinnati Children’s Hospital Medical Center.

This was a terrific lecture.

  • Example of use of pluripotent stem cell usage: Diabetes. Phase 1 study has been started.
  • Organoids are in essence miniature versions of organs in a dish and with complex combination of cell types.
  • Organoids allow easier testing on these tissues for treatment and diagnosis of diseases
  • Organoids will allow for personalized testing of medications.  Some patients will respond differently.  This technology could be used to grow a specific organoid for a specific person and determine response on the organoid before giving to the patient.
  • Can engraft organoids into mice which can provide blood supply and allow larger organoids.
  • Clinical projects for organoids: Hirschsprung’s,  H pylori, Clostridium difficile, Short bowel syndrome, Fatty liver disease

#NASPGHAN17 IBD Treat to Target and Tight Control

More information from this year’s annual NASPGHAN meeting.

This blog entry has abbreviated/summarized these presentations. Though not intentional, some important material is likely to have been omitted; in addition, transcription errors are possible as well.

IBD Treat to Target: Treat the Patient or Treat the Disease

Robert Baldassano  Children’s Hospital of Philadelphia

I missed the first few minutes of this presentation, even though I had highlighted this as one of my top priorities.  So, if anyone reading this post has some additional comments, they are certainly welcome.

Key points:

  • Do not rely on symptoms alone to assess patient improvement.
  • Best surrogate marker: calprotectin.  Frequent calprotectin levels can help determine objective improvement; it is much more helpful than CRP as ~25% of patients do not elevate their CRP levels
  • Therapeutic drug monitoring is important in improving outcomes. Dose optimization improves response rate and durability of infliximab response.
  • Evolving targets in ulcerative colitis.  Even histologic activity, in the absence of endoscopic activity, is associated with relapsing disease
  • Dr. Baldassano indicated that he no longer is starting patients on thiopurine therapy. There are “36 phase 3 trials underway.” Thus, many promising options for those who may burn through current treatments
  • This lecture reviewed data from the RISK study showing that early (1st 90 days w/in diagnosis) TNF therapy helps prevent penetrating disease (related post: CCFA Update 2017/RISK study)

Another presentation by Philip Minar et al (Cincinnati Children’s Hospital Medical Center) shows that CD64 suppression is an early biomarker of response to infliximab therapy.

#NASPGHAN17 Is it time to stop using thiopurine therapy?

This blog entry has abbreviated/summarized this presentation. Though not intentional, some important material is likely to have been omitted; in addition, transcription errors are possible as well.

Safety in Pediatric IBD Therapy: Is it time to stop using thiopurines?

Jeffrey Hyams  Connecticut Children’s Medical Center

Key points from this lecture:

  • Dr. Hyams:  “There are better options than thiopurines in 2017 due to infrequent but serious risks”
  • The DEVEVOP study showed that anti-TNF agents did NOT increase the risk of lymphoma or hemophagocytic lymphohistiocytosis (HLH).  In contrast, these risks do occur with thiopurines –this is infrequent but remains significant.
  • Therapeutic drug monitoring may obviate the need for combination/dual therapy which has been shown to improve response rates to anti-TNF agents; methotrexate may work for combination therapy and may be safer than thiopurines
  • If a thiopurine is used as part of combination therapy, short duration (~6 months) is likely to have low risks
  • In addition to Dr. Hyams, Dr. Baldassano, in his discussion of treat to target (discussed in subsequent post), echoed the sentiment that he no longer recommends thiopurine therapy

Dr. Hyams slides list some of the relative risks of thiopurine therapy.  To understand these risks, the absolute risk is probably more helpful.

My take: This lecture did not focus on the main benefit of thiopurines which is its use in combination therapy. Many experts consider combination therapy to be the standard of care for adults with Crohn’s disease.  The advantages of combination therapy are mainly due to improved durability of anti-TNF therapy and lower antidrug antibodies.  How this benefit stacks up against the risks discussed in this lecture and whether this benefit can be supplanted by the use of therapeutic drug monitoring is uncertain.

 

Related blog posts:

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.

NASPGHAN Postgraduate Course 2017 (Part 5): Refractory constipation, Extraesophageal GERD, POTS, Recurrent Abdominal Pain

This blog entry has abbreviated/summarized these presentations. Though not intentional, some important material is likely to have been omitted; in addition, transcription errors are possible as well.

Here is a link to postgraduate course syllabus: NASPGHAN PG Syllabus – 2017

The child with refractory constipation

Jose Garza   GI Care for Kids & Children’s Healthcare of Atlanta

Key points:

  • Polyethylene glycol is a first-line agent and many patients require cleanout at start of therapy
  • Adequate dose of laxative is needed for sure regular painless stools
  • Don’t stop medicines before toilet training and until pattern of regular stooling established. “All symptoms of constipation should resolve for at least one month before discontinuation of treatment”
  • Gradually reduce laxatives when improved
  • An abdominal xray is NOT recommended to make the diagnosis of constipation
  • Do you have the right diagnosis? Irritable bowel is often confused with constipation.  With constipation, the pain is relieved after resolution of constipation.
  • Outlet dysfunction. Stimulant laxatives (eg. Senna) are probably underutilized. Biofeedback may help in older children.
  • Slow transit constipation. Newer prosecretory agents may be helpful –lubiprostone and linaclotide.
  • Organic constipation. Hirschsprung’s, Spina bifida, anorectal malformations etc. Testing: anorectal manometry, rectal biopsy (for Hirschsprung’s)
  • For refractory disease, consider rectal therapy –suppositories, transanal irritagations/enemas (~78% success for fecal incontinence/constipation). These treatments should be used prior to surgical therapy (eg. Malone antegrade continence enema/cecostomy)

 

 

The quest for the holy grail: Accurately diagnosing and treating extraesophageal reflux

Rachel Rosen   Boston Children’s Hospital

Key points:

  • It is frequent that EGD or impedance study will be abnormal, though this may not be causally-related.
  • No correlation with ENT exams/red airways and reflux parameters
  • No correlation with lipid laden macrophages and reflux parameters
  • No correlation with salivary pepsin and reflux parameters

Treatment:

  • Lansoprazole was not effective for colic or extraesophageal symptoms (Orenstein et al J Pediatrics 2009)
  • PPIs can increase risk of pneumonia/respiratory infections
  • Macrolides have been associated with increased risk of asthma but may be helpful for pulmonary symptoms
  • Fundoplication has not been shown to be effective for reducing aspiration pneumonia.  Fundoplication could increase risk due to worsened esophageal drainage.
  • ALTEs (BRUEs -brief resolved undefined events) need swallow study NOT PPIs

POTS and Joint Hypermobility: what do they have to do with functional abdominal pain?

Miguel Saps  University of Miami

Key points:

  • Patients with POTS and joint hypermobility have frequent functional abdominal pain as well as other comorbidities
  • Beighton Score can determine if joint hypermobility is present
  • Brighton Score determines if hypermobile Ehlers-Danlos syndrome is present
  • Patients with frequent fatigue.  Gradual progressive and regular exercise is important part of therapy.  Can start with recombant exercise – training bicycle exercise, swimming
  • Need to push salt intake and fluds

Do I need to test that C.R.A.P.?

Rina Sanghavi   Children’s Medical Center Dallas

This basic talk reviewed a broad range of issues related to functional abdominal pain.

Key points:

  • Carnett’s sign can help establish abdominal pain as due to abdominal wall pain rather than visceral pain
  • What is an appropriate evaluation?  Limited diagnostic testing for most patients.
  • Alarm symptoms include: Fevers, Nocturnal diarrhea, Dysphagia, Significant vomiting, Weight Loss/poor growth, Delayed Puberty, and Family history of IBD

NASPGHAN Postgraduate Course 2017 (Part 4): Therapeutic drug monitoring, Anti-TNF management, Postoperative Crohn’s disease

This blog entry has abbreviated/summarized these presentations. Though not intentional, some important material is likely to have been omitted; in addition, transcription errors are possible as well.

Here is a link to postgraduate course syllabus: NASPGHAN PG Syllabus – 2017

Therapeutic Drug Monitoring

Andrew Grossman  Children’s Hospital of Philadelphia

The topic of therapeutic drug monitoring, both reactive and proactive, has been discussed numerous times on this blog.  This talk provided a good review of this topic.

Key points:

  • Greatest predictor of infliximab treatment failure was a low infliximab (<0.9 mcg/mL at anytime or <2.2 mcg/mL at 14 weeks) (Castelle et al Am J Gastro 2013; 108: 962-71)
  • Low level antibodies to infliximab may be transient in ~28% and may be overcome with escalation of therapy
  • Tissue levels of infliximab (and other agents) may be inadequate despite good serum levels

What if anti-TNF fails

Maria Oliva-Hemker   Johns Hopkins University School of Medicine

Key points:

  • Discussed prevalence of problem with anti-TNF failures and main options: vedolizumab, ustekinumab, and surgery
  • Vedolizumab can take a while to work, particularly for Crohn’s disease
  • Limited data in pediatrics for these newer agents
  • Ustekinumab has some preliminary data indicating benefit with anti-TNF induced psoriaform rashes
  • Newer agents also likely to need therapeutic drug monitoring
  • Overall, ustekinumab and vedolizumab have good safety profiles at this point

 

Prevention of postoperative Crohn’s disease

Miguel Regueiro   University of Pittsburgh

  • Rationale for postoperative preventative treatment: high rate of recurrent disease which can be silent for several years despite progressive damage to GI tract
  • Large study (PREVENT) to compare infliximab and placebo after surgery.  Primary endpoint was clinical recurrence (was endpoint demanded by FDA) even though clinical recurrence can be a late finding.  Endoscopic recurrence rate was a secondary endpoint.

Dr. Regueiro’s approach

  • Low risk patient –repeat scope at 6 months post-op, then every 1-3 yrs if no disease and Rx with anti-TNF or immunomodulator in those with endoscopic recurrence
  • Moderate risk patient -possible use of thiopurine or use the ‘low risk’ approach
  • High risk patient-combination therapy and if doing well for several years, consider monotherapy
  • In pediatrics, the postoperative management is unclear due to difficulty with risk stratification.  If postoperative treatment is not given, consider colonoscopy 3-4 months afterwards and treat if recurrence.  Then could use calprotectin every 3 months to monitor and when >50, likely will need to be treated

PREVENT Trial Data:

 

 

 

NASPGHAN Postgraduate Course 2017 (Part 3): Biliary Atresia, NAFLD, SMOFlipid, Pancreatic Pain

This blog entry has abbreviated/summarized these presentations. Though not intentional, some important material is likely to have been omitted; in addition, transcription errors are possible as well.

Here is a link to postgraduate course syllabus: NASPGHAN PG Syllabus – 2017

Biliary Atresia: Update on diagnostic and prognostic biomarkers and therapeutic interventions

Cara Mack    Children’s Hospital of Colorado

Key points:

  • 84% of biliary atresia is isolated; 16% are syndromic with other defects
  • Direct bilirubin is (mildly) elevated at birth in patients with biliary atresia
  • Total bilirubin 3 months after Kasai predicts outcome. If <2 mg/dL, then unlikely to need a transplant in the first 2 years of life.
  • Reviewed biomarkers including Th1, Autotaxin, IL-8

Therapeutic interventions:

  • Nutritional support. Better nutrition improves outcomes after liver transplantation.
  • Fat soluble vitamin supplementation
  • Cholangitis prevention. Some studies have shown that prophylactic antibiotics may reduce incidence of cholangitis.
  • No therapeutic interventions that delay progression of this disease

 

 

CHILDREN Cohort Mgt of Vitamin Supplementation

Steroids are not helpful after Kasai procedure

Diagnosis and Management of Pediatric NAFLD 2017

Stavra Xanthokos   Cincinnati Children’s Hospital Medical Center

Key points:

  • NAFLD is #2 cause of liver transplantation in adults and on its way to becoming #1
  • ALT is still the best screening tool; NASPGHAN guidelines recommends screening overweight/obese children 9-11 years of age
  • Ultrasound has poor sensitivity and specificity for NAFLD; it is helpful for detecting gallbladder disease
  • Bariatric surgery has been effective for NAFLD

 

SMOFlipid and the Pediatric Patient

Peter Wales  Hospital for Sick Children (Toronto)

Slides are not available in syllabus

Key points:

  • Improving outcomes noted in the intestinal failure population
  • Dr. Wales reviewed proposed improvements with Omega-3 lipids -less cholestasis, less hepatitis, and less fibrosis
  • Compared improvements with lipid minimization (1 g/kg/day) compared to newer agents: omegaven and SMOFlipid. Additional studies are needed due to limitations of previous studies
  • Discussed SMOFlipid vs. Intralipid trial at 5 centers in Canada. N=24.
  • At SickKids: SMOFlipid for all preterms at admission & for term infants after 2 weeks of PN. Dosing 2-2.5 g/kg & now accounts for 85% of lipid usage at institution
  • None of the lipid products were designed for preterm infants. Intralipid has a pediatric indication and other products are used off label
  • Lipid restriction probably affects brain size/development; thus, a lipid agent that allows for higher doses likely will be beneficial for developmental outcomes.  The retina can be used as a biomarker of the brain affects of lipids.

Painful Chronic Pancreatitis: Management/therapeutic interventions

Vikesh Singh  Johns Hopkins University School of Medicine

Slides are not available in syllabus