Standardizing the Care of Children Receiving Chronic Glucocorticoid Therapy

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A recent study (ML Basiaga et al. J Pediatr 2016; 179: 226-32) highlights the large variation in care for 701 children receiving  steroids (for at least 15 days) at a leading children’s hospital (CHOP).  I think, given the fact that this is a retrospective study and the huge variation in steroid exposure, the message regarding variation should not be taken that seriously.  But, the article does suggest that in children with chronic glucocorticoid therapy, several measures should be considered:

  • Bone health -particularly Vitamin D (25-OH) levels
  • Immunity -particularly assuring pneumococcal and influenza vaccines
  • Lipid screening
  • Stress steroid plan.  The authors indicate that the endocrinology society recommendations have included instructing parents in intramuscular hydrocortisone in case of vomiting or severe stress.

My take: Having a standard approach to an at-risk group makes sense, however, “whether implementation of preventive care guidelines improves outcomes in children” is not known.

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Is It OK for Pediatricians to try to Prevent Firearm Injuries? Focus on Child Safety –Not on Gun Safety

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A recent study (JM Garbutt et al. J Pediatr 2016; 179: 166-71 and related editorial by MD Dowd, pg 15-17) provide relevant information on the issue of firearm injury prevention.

The study describes the results of a survey provided to 1246 parents at a diverse group of practices around St Louis.

Key findings:

  • 36% reported being owners of firearms
  • Of the owners, 25% reported ≥1 firearm was stored loaded and 17.9% carried a firearm when leaving the house.
  • 75% of all parents thought pediatricians should provide advise on safe storage of firearms (71% of owners); however, only 12.8% of all parents reported a discussion about firearms with the pediatrician

The discussion and commentary on this study are more interesting than the actual results. Key points:

  • The AAP has recommended that pediatricians screen for the presence of household firearms and has stated that a “home without guns is the safest option…Advising safe storage is also encouraged.”  Prior surveys have echoed this study that few pediatricians counsel families about firearm safety.
  • Despite AAP recommendations, over “60% of Americans believe that a ‘gun in the house makes it safer’ which is a more common attitude than in 2004 when 42% of Americans held that view.
  • Providing a child with firearm avoidance educational programs (eg. NRA’s “Eddie Eagle”) “is unlikely to lead to safe behaviors…[and] may give parents a false sense of security.”
  • “Children cannot distinguish real guns from toy guns and are strong enough to pull a trigger as early as 3 years of age.”
  • Approaching the topic of safe storage “as an expert in child development” and children’s unpredictable behavior rather than in firearm safety “may be acceptable to both pediatricians and parents.”
  • The authors advocate keeping firearm storage on a checklist of hazards (eg. medication storage, avoiding household poisons) –though this has not been well-studied.
  • From editorial: “When compared with other developed nations, US children under 15 years of age are 12 times more likely to be killed by a gun…We know that nearly 1 in 10 families with guns admit to keeping at least 1 gun loaded and unlocked, and nearly one-half keep at least 1 gun unlocked.”

So, in fact, having a gun in the home does not make a home safer, just the opposite.  But delving into this topic is probably not productive due to strong feelings tangential to gun ownership.  There have been unsuccessful legislative efforts in over 10 states to prevent physicians from discussing the topic as well as a protracted legal battle in Florida.

My take (borrowed from editorial): “Although the difference between “gun safety” and “child safety” may seem subtle, such a shift allows a consistent approach to home injury prevention across mechanisms of injury with the focus on the child, not the gun.” “Little children are curious and big children (teens) are impulsive, so exposure to unsecured guns can lead to tragic outcomes that cannot be prevented by child education.  Who better to deliver this message than pediatricians?”

Related blog posts:

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New HCV Treatment Effective in Adolescents –Important Study Now Published Online

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A soon-to-be-published study (available online Hepatology) from WF Balistreri et al shows that the combination of Ledipasvir-Sofosbuvir is highly effective in pediatric patients aged 12-17 years.

Here is the abstract:

ABSTRACT

No all-oral, direct-acting antiviral regimens have been approved for children with chronic hepatitis C virus (HCV) infection. We conducted a Phase 2, multi-center, open-label study to evaluate the efficacy and safety of ledipasvir–sofosbuvir in adolescents with chronic HCV genotype 1 infection. One hundred patients ages 12 to 17 years received a combination tablet of 90 mg ledipasvir and 400 mg sofosbuvir once daily for 12 weeks. On the 10th day following initiation of dosing, 10 patients underwent an intensive pharmacokinetic evaluation of the concentrations of sofosbuvir, ledipasvir, and the sofosbuvir metabolite GS-331007. The primary efficacy endpoint was the percentage of patients with a sustained virologic response 12 weeks posttreatment (SVR12). Median age of patients was 15 years (range, 12-17 years). A majority (80%) were HCV treatment naïve, and 84% were infected through perinatal transmission. One patient had cirrhosis and 42 did not; in 57 patients the degree of fibrosis was unknown. Overall, 98% (98/100; 95% CI, 93% to 100%) of patients reached SVR12. No patient had virologic failure. The 2 patients who did not achieve SVR12 were lost to follow-up either during or after treatment. The 3 most commonly reported adverse events were headache (27% of patients), diarrhea (14%), and fatigue (13%). No serious adverse events were reported. AUCtau and Cmax values for sofosbuvir, ledipasvir, and GS-331007 were within the predefined pharmacokinetic equivalence boundaries of 50% to 200% when compared with adults from Phase 2 and 3 studies of ledipasvir and sofosbuvir. Conclusion: Ledipasvir−sofosbuvir was highly effective in treating adolescents with chronic HCV genotype 1 infection. The dose of ledipasvir−sofosbuvir currently used in adults was well tolerated in adolescents and had an appropriate pharmacokinetic profile.

My take: This is great news for pediatric patients.  This study indicates that this breakthrough therapy (and likely others) for adults will become more widely available for pediatric patients soon.

TV sports analysts have tremendous insight!

TV sports analysts have tremendous insight!  Virginia needs to score enough points to win.

Understanding the “Rashomon Effect”

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An interesting commentary (GM Ronen, DL Streiner. J Pediatr 2016; 179: 17-18) discusses the “Rashomon” effect and how this can relate to studies which show differences between children with health problems and their parents’ perception of how they are doing.

“In this famous Japanese tale, set in the 12th century, a notorious bandit attacked a samurai and his wife in the woods.”  Afterwards, all of the accounts of the incident by the participants were widely discrepant. “When the tale is over, the reader realizes that even though none of the version is a truthful objective account, all must be true at least from the character’s own unique perspective.”

In medical studies with children and their parents, different versions of the truth can be due to many factors:

  • Depression distortion hypothesis –raters with depression tend to score poorer on numerous health variables
  • Disability paradox –“some persons with impairments, against all odds, are satisfied with their life and rate their health similar to typical children”
  • Parents may also be affected by the emotional impact of their child’s health problem even when the problem is well-controlled

My take: This short commentary has a lot to say about understanding why a person with a medical problem may rate their health much better or much worse than an outside observer would expect.

Penobscot Narrows Bridge, Maine

Penobscot Narrows Bridge, Maine

 

Blind Men and The Elephant: Lasting Consequences of Enteric Infections

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Recently, Ben Gold handed me a supplement which alluded to the case of “the blind men and the elephant.”  So, of course, I wanted to know more about this.

According to Wikipedia:

In various versions of the tale, a group of blind men (or men in the dark) touch an elephant to learn what it is like. Each one feels a different part, but only one part, such as the side or the tusk. They then compare notes and learn that they are in complete disagreement. The stories differ primarily in how the elephant’s body parts are described, how violent the conflict becomes and how (or if) the conflict among the men and their perspectives is resolved.  In some versions, they stop talking, start listening and collaborate to “see” the full elephant. When a sighted man walks by and sees the entire elephant all at once, the blind men also learn they are all blind. While one’s subjective experience is true, it may not be the totality of truth. If the sighted man were deaf, he would not hear the elephant bellow.

It has been used to illustrate a range of truths and fallacies; broadly, the parable implies that one’s subjective experience can be true, but that such experience is inherently limited by its failure to account for other truths or a totality of truth. At various times the parable has provided insight into the relativism, opaqueness or inexpressible nature of truth, the behavior of experts in fields where there is a deficit or inaccessibility of information, the need for communication, and respect for different perspectives.

The rest of the supplement regarding chronic health consequences following acute enteric infections was less interesting but probably more important than learning a new anecdote.

The introduction notes that nearly 600,000 children under 5 years die from dehydrating diarrhea each year.  Many more suffer from consequences of disease-associated malnutrition with both physical and cognitive deficits.

Articles in supplement:

  • Am J Gastroenterol Suppl 2016; 3: 4-11. –details diarrhea-associated years lived with disability 51 per 100,000 in developed regions compared with 685 in developing regions.
  • Am J Gastroenterol Suppl 2016; 3: 12-23. –details the likelihood of consequences following enteric infections, including functional GI disorders, inflammatory bowel disease, celiac disease (data limited), Guillain-Barré syndrome, hemolytic uremic syndrome, chronic fatigue, and neurologic sequelae.
  • Other articles in the supplement describe changes in the microbiome, the micorbiome-gut-brain axis, and the relationship between autoimmunity and irritable bowel.

 

Nutrition Guidelines for Cystic Fibrosis

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Wilschanski et al (JPGN 2016; 63: 671-5) provide a summary (“highlights”) of a full report (Turck D et al. Clin Nutr 2016; 35: 557-77) on nutritional recommendations for infant and children with cystic fibrosis.

What’s in here:

Table 1: criteria for adequate nutritional status including

  • Age <2 yrs: 50% for weight & height compared to healthy-age peers
  • Age 2-18 yrs: 50% BMI compared to healthy peers

Table 2: nutritional assessment and followup

  • Assess elastase-1 annually if pancreatic sufficient
  • Assess pancreatic enzyme supplementation
  • Annual blood tests: CBC/d, iron status, fat-soluble vitamins, LFTs.  Possibly: fatty acids
  • If older than 10 years, annual glucose tolerance
  • Dietary review every 3 months
  • Bone density assessment between 8-10 yrs and then every 1-5 yrs

Table 3: Energy requirements

  • Anticipate need for 110-200% compared with healthy peers

Table 4: Pancreatic enzyme replacement therapy (PERT)

  • 0-1 yr: 2000-4000 units lipase/120 mL of formula/breast milk & 2000 units lipase/gram of dietary fat
  • 1-4 yrs: 2000-4000 units of lipase/gram of dietary fat (max 10,000 units lipase/kg/day)
  • >4 yrs: starting dose; 500 units lipase/kg/meal -titrate up to 1000-2500 units lipase/kg/meal (max 10,000 units lipase/kg/day)

Table 5: Fat-soluble vitamin/vitamin guidelines

Table 6: Sodium supplementation

  • 0-6 months: 1-2 mmol/kg/day –give salt in small portions throughout the day, “diluted in water or fruit juice”.  In some infants, up to 4 mmol/kg/day if increased losses (eg. due to heat, gastrointestinal losses)
  • Older children: anticipate need for additional salty foods or use sodium chloride capsules, especially when excessive sweating (eg. fever, sports, hot weather)

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.

Maine Coast, near Acadia

Maine Coast, near Acadia

 

IBD Updates -January 2017

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L Beswick et al. Inflamm Bowel Dis 2016; 22: 2966-2976. The authors provide an algorithm for diagnosis and management of CMV in the setting of inflammatory bowel disease which primarily in the setting of steroid-refractory colitis.  Despite conferring a worse prognosis, the authors note that in most cases the virus is nonpathogenic and thus antiviral is usually ineffective.  Figure 1 outlines their algorithm, in those with high density inclusions on tissue (≥5 per biopsy) and/or high blood CMV PCR, the authors recommend treatment (including ganciclovir).  The details of this figure are too complex to easily summarize and if faced with this clinical scenario, I recommend reviewing source article.

KA Dunn et al.  Inflamm Bowel Dis 2016; 22: 2853-62. Fecal samples from 10 patients & 5 controls (age 10-16) showed that microbial diversity was lower in Crohn’s and lowest in patients who did not achieve SR.

EL Barnes, R Burakoff. Inflamm Bowel Dis 2016; 22: 2956-65. New biomarkers for diagnosing inflammatory bowel disease: biomarker signatures, gene expression analysis, protein profiling and microRNA

Does a Healthy Lifestyle Result in Better Outcomes?

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It’s easy to become discouraged that sensible actions may not be effective due to general pessimism and sometimes conflicting medical reports.  On the positive side of the ledger, a recent study (AV Khera et al. NEJM 2016; 375: 2349-58) provides compelling data that a combination of healthy lifestyle changes make a BIG difference.

The study focused on 4 healthy lifestyle factors: no smoking, no obesity, regular physical activity, and a healthy diet.  The study examined three large prospective cohorts with a total of more than 55,000 patients.

Key finding:

  • Among participants with high genetic risk, a favorable lifestyle was associated with a 46% lower relative risk of coronary events compared to those with an unfavorable lifestyle over the 10-year study period.

In the same issue, a review of the human intestinal microbiome (pages 2369-79) notes that “dietary intake appears to be a major short-term and long-term regulator of the structure and function of gut microbiota.  Still, only a relatively small number of randomized, clinically controlled dietary interventions targeting the gut microbiota have been reported in humans and these show that energy restriction and diets rich in fiber and vegetables are associated with gut microbial changes that, in turn, are associated with a health benefit.”

My take: To enhance your odds of good health, avoid smoking, stay fit, and eat your fruits/veggies.

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Care Coordination and Magical Thinking

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One myth that has been promulgated has been that care coordination will lead to cost containment. A commentary on this topic (JM McWilliams. NEJM 2016; 375: 2218-20) explains the fallacy of this thinking.  While care coordination can improve medical care, “conflating cost containment and care coordination poses many potential dangers.”  Good care is worthy goal even in the absence of saving money.

Key points:

  • Care coordination often improves outcomes but typically involve interventions to correct underuse of care
  • For every costly complicated prevent, “a care coordination program must manage care for multiple patients…[which] is costly.”
  • Early evaluation of care coordination in accountable care organizations (ACOs) have shown the efforts “have meaningfully improved patient experiences but not rates of hospitalizations for ambulatory care-sensitive conditions.” There has not been evidence of fewer readmissions or fewer preventable hospitalizations with care coordination.
  • Other strategies to reduce cost are now being targeted, like steering patients to lower-priced providers

My take (from author): “We should coordinate care not to save money but because coordinated care is better care.”

Yosemite

Yellowstone (I took this picture!)