Briefly noted: PFAPA Study

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U Lantto et al. J Pediatr 2016; 179: 172-7.  This retrospective study showed that tonsillectomy was highly effective in children with PFAPA (periodic fever, aphthous stomatitis, pharyngitis, and adenitis) -both those with classic features and incomplete features.

“In the group that met Thomas criteria, 97% (56/58) had complete resolution of fever episodes after tonsillectomy; in the group that did not meet Thomas criteria (50/50) had complete resolution.”

Thomas criteria:

  1. Recurring fevers with early age of onset (<5 years) [the authors suggest a minimum of 5 episodes]
  2. Constitutional symptoms in the absence of over upper respiratory infection with at least one of the following: aphthae, cervical adenitis, pharyngitis
  3. Exclusion of cyclic neutropenia
  4. Completely asymptomatic interval between episodes
  5. Normal growth and development

whater

 

Quick Take on Migraine Study

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For those who missed this important pediatric study, a quick take ~2 min video link: Pediatric Migraine CHAMP Study

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Related blog post: Topamax and Amitriptyline Did Not Work for Pediatric Migraines

My take: It looks like placebo did pretty well with >60% response rate.  In a commentary on this subject, the authors noted that when patients are seen by the physician, the symptoms are often severe.  So, some improvement is expected, in part, due to regression to the mean; that is, it is common to return to their baseline level of symptoms.

How Rudeness Affects Performance in Medicine (and probably elsewhere)

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From A Riskin et al. Pediatrics Jan 2017 (Thanks to Seth Marcus for pointing out this study), Link: Rudeness and Medical Team Performance

Abstract

OBJECTIVES: Rudeness is routinely experienced by medical teams. We sought to explore the impact of rudeness on medical teams’ performance and test interventions that might mitigate its negative consequences.

METHODS: Thirty-nine NICU teams participated in a training workshop including simulations of acute care of term and preterm newborns. In each workshop, 2 teams were randomly assigned to either an exposure to rudeness (in which the comments of the patient’s mother included rude statements completely unrelated to the teams’ performance) or control (neutral comments) condition, and 2 additional teams were assigned to rudeness with either a preventative (cognitive bias modification [CBM]) or therapeutic (narrative) intervention. Simulation sessions were evaluated by 2 independent judges, blind to team exposure, who used structured questionnaires to assess team performance.

RESULTS: Rudeness had adverse consequences not only on diagnostic and intervention parameters (mean therapeutic score 3.81 ± 0.36 vs 4.31 ± 0.35 in controls, P < .01), but also on team processes (such as information and workload sharing, helping and communication) central to patient care (mean teamwork score 4.04 ± 0.34 vs 4.43 ± 0.37, P < .05). CBM mitigated most of these adverse effects of rudeness, but the postexposure narrative intervention had no significant effect.

CONCLUSIONS: Rudeness has robust, deleterious effects on the performance of medical teams. Moreover, exposure to rudeness debilitated the very collaborative mechanisms recognized as essential for patient care and safety. Interventions focusing on teaching medical professionals to implicitly avoid cognitive distraction such as CBM may offer a means to mitigate the adverse consequences of behaviors that, unfortunately, cannot be prevented.

This same group had a related study in 2015:The Impact of Rudeness on Medical Team Performance: A Randomized Trial

My take:The saying “you catch more flies with honey than you do with vinegar” is probably accurate.

Costa Maya, Mexico

Costa Maya, Mexico

 

Role of Biosimilars in Inflammatory Bowel Disease

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A cautionary note on biosimilars has been discussed in a recent review (DT Rubin et al. Gastroenterol & Hepatol 2016; 12: 741-51)

In the recently completed NOR-SWITCH study presented at the United European Gastroenterology Week 2016 meeting, “a total of 481 patients were recruited across 40 centers: all patients had been on stable treatment with the originator infliximab for at least 6 months…When looking specifically at IBD patients, disease worsening was noted in 21.2% of originator infliximab-treated patients and 36.5% of CT-P13-treated Crohn’s disease patients (n=155).”  The 15% difference did not reach statistical significance, but is concerning.  The authors state that “subtle postranslational modifications unique to the biosimilars may be sufficient to lead to antidrug antibody formation with associated loss of response.  Also, it is noted that this study did not include endoscopic evaluation.

The authors note that therapeutic monitoring worked with biosimilar product using available infliximab assays.

My take: We still have a lot to learn.  The preliminary message, until more studies are available, indicate that switching stable patients could increase risk of losing response.

Related blog posts:

Puerto Rico

Puerto Rico

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.