Otolaryngologic Manifestations of Gastroesophageal Reflux
Opinion statement
Gastroesophageal reflux disease (GERD) is a common problem
that has been linked to multiple extraesophageal manifestations relevant to the
otolaryngologist. Finding evidence for a causal relationship between reflux and
manifestations of otolaryngologic disease is often difficult, however, due in
part to the non-specific nature of extraesophageal symptoms and the lack of
pathognomonic endoscopic or laryngoscopic signs. This poses an even greater
challenge in pediatrics given the inherent unreliability when obtaining
subjectively reported data from younger aged children. Numerous studies have
drawn a correlation between GERD and specific extraesophageal symptoms such as
otitis media, sinusitis, chronic cough, and various laryngeal findings; however,
determining a clear causative relationship has proven to be much more difficult.
Further studies must be done to elucidate the true pathophysiologic mechanisms
behind these disease processes. Diagnosis of GERD is challenging and is
typically over-diagnosed among otolaryngologists based on laryngoscopic
findings. This method has been proven to be both inconsistent and non-specific
when evaluating for the presence of GERD. Impedance monitoring is supplanting pH
probes as the new gold standard; the clinical utility of impedance testing among
patients with extraesophageal symptoms remains unclear however and there are
practical limitations in performing these studies in children. The accuracy of
diagnostic tests (laryngoscopy, endoscopy, and pH- or pH-impedance monitoring)
for patients with suspected extraesophageal manifestations of gastroesophageal
reflux disease remains suboptimal at this time. Management options for GERD
include lifestyle changes, pharmacologic therapy, and surgical intervention. H2
blockers and proton pump inhibitors are effective treatment options in children
but must be used judiciously given their potential side-effect profiles.
Surgical options remain for those cases which are refractory to medical
management. Multidisciplinary approaches and close collaboration between
otolaryngologists and gastroenterologists is recommended to ensure application
of best practice guidelines and for continued improvements in this
area.
Treatment Options for the Adolescent Patient Experiencing Abnormal Uterine Bleeding
Opinion statement
Abnormal uterine bleeding (AUB) is one of the most common
reasons adolescent patients present for gynecologic care. A new classification
system has been created to provide a universally accepted system of nomenclature
to describe uterine bleeding causes in reproductive-aged women. The acronym
PALM-COEIN (polyp, adenomyosis, leiomyoma, malignancy and hyperplasia,
coagulopathy, ovulatory dysfunction, endometrial, iatrogenic and not yet
classified) was introduced in 2011. This classification will help to target not
only treatment but also future research into treatment options in the AUB
population. In order for treatments to be effective, an accurate diagnosis is
paramount. Adolescents tend to have anovulatory, infectious, or
pregnancy-related causes of AUB. Traditional treatment modalities have been
hormonal in nature, the most common being the use of oral contraceptive pills.
The latest development has been the recognition of the effective use of the
levonorgestrel intrauterine systems (LNG-IUS) in successful treatment of AUB in
the adolescent population.
Diagnosis and Management of Congenital Sensorineural Hearing Loss
Opinion statement
Hearing loss is the most common sensory disorder in the
USA. The diagnosis of congenital hearing loss starts with newborn hearing
screening, which is best performed with auditory brainstem evoked responses in
order to avoid the risk of missing auditory neuropathy spectrum disorder. A
careful history and physical exam can occasionally help reveal the etiology for
congenital hearing loss. Imaging studies, either CT temporal bones or MRI of the
internal auditory canals without gadolinium, and genetic testing, in particular
for connexin 26, connexin 30, and Pendred syndrome, are the most useful
diagnostic tests. Management of congenital hearing loss involves early fitting
of amplification. Early cochlear implantation, preferably before 2 years of age,
should be strongly considered for children with bilateral severe hearing
loss.
Gynecologic Pain in Adolescents
Opinion statement
Gynecologic pain affects a significant number of
adolescents. While the differential diagnosis for such pain is often extensive,
the treatment goals are the same—to relieve pain and minimize the effect it has
on an adolescent’s daily functioning, and to maximize their future reproductive
potential. For management of acute gynecologic pain—such as in cases of adnexal
torsion or obstructive anomalies—surgical intervention is often warranted. Pain
related to more chronic processes—such as endometriosis—is treated both
medically and surgically. Hormonal therapy (either combined estrogen/progestin
or progestin-only) is the mainstay of medical therapy. Newer methods, such as
the levonorgestrel intrauterine devices (LNG-IUD), show promise as a management
option for chronic endometriosis-related pain. The LNG-IUD also provides highly
effective long-acting reversible contraception, and is a first-line recommended
contraceptive method for adolescents. Other etiologies of gynecologic pain may
be infectious and are therefore treated by antibiotics. Providers must perform a
thorough history and physical exam—with attention to patient confidentiality.
Establishing a good rapport with both patients and their caregivers is crucial.
Regular follow-up to assess treatment efficacy is necessary—as certain
conditions may require a stepwise progression of treatment modalities for
resolution of symptoms. While it is often challenging caring for an adolescent
with chronic gynecologic pain, it is also extremely important and rewarding.
Optimizing the gynecologic health of adolescent females will allow them to
function at their full potential and lead productive, enjoyable
lives.
Tongue Tie in Infancy
Opinion statement
Ankyloglossia, or “tongue tie,” classically involves a
short or thickened lingual frenulum that may prohibit tongue protrusion.
However, the diagnosis, evaluation, clinical significance, and management of
ankyloglossia are widely variable and controversial. Despite attempts to create
standardized diagnostic criteria for ankyloglossia, there has yet to be a
universally accepted system. Management of ankyloglossia often includes a
multidisciplinary approach including lactation consultants, speech language
pathologists, pediatricians, and otolaryngologists. Observation or conservative
management for asymptomatic infants or infants with minimal or well-compensated
symptoms is a reasonable option, whereas surgical intervention may be warranted
for infants and children with ankyloglossia that has significant impact on
breastfeeding or speech. Frenotomy (also known as frenulotomy) is a relatively
simple procedure that can be performed at the bedside or office setting in very
young infants, precluding the need for general anesthesia. Frenuloplasty is
usually performed on older children and should be performed in the operating
room with general anesthesia. Overall, there is insufficient evidence to
definitively associate ankyloglossia with breastfeeding or speech deficits.
Surgical intervention for ankyloglossia should be recommended with caution and
performed only on infants or children with clear findings of ankyloglossia on
physical exam and a documented history of breastfeeding or speech difficulties;
the timing and method of treatment should be tailored to the individual infant
or child. Frenotomy or frenuloplasty should only be performed by providers with
adequate training and experience in order to minimize
complications.
Contemporary Management of Vesicoureteral Reflux
Opinion statement
The past 30 years have seen broad changes in the diagnosis
and management of vesicoureteral reflux (VUR). Recently, a clinical debate has
generated an open discussion in academic circles. New evidence has shifted
treatment patterns away from widespread surgical management and recently brought
into question some pharmacologic treatments. VUR is usually not hazardous by
itself but is a significant risk factor for urinary tract infection (UTI) and
less commonly, renal scarring and insufficiency. Given the costs and morbidity
of UTI as well as the potential for significant renal injury, our approach
remains conservative. Careful follow-up, parental education about
pathophysiology and management of VUR and UTI, and management of bowel and
bladder dysfunction (BBD), when present, are the foundations of treatment.
Additionally, though we recognize the limitation of continuous antibiotic
prophylaxis (CAP), we believe that the benefits outweigh the risks and costs for
many patients. Careful observation can be considered in patients with a single
medical home, parental understanding of what UTI signs and symptoms are, low
grade VUR, no history of complicated UTIs, and close follow-up. Surgical
management remains a relevant option for select patients who fail conservative
measures with breakthrough UTIs or failure to resolve. Minimally invasive
surgical options are available with acceptable outcomes though open
ureteroneocystostomy still carries the highest success rate.
Nephrotic Syndrome: Updates and Approaches to Treatment
Opinion statement
Nephrotic syndrome (NS) is among the most common pediatric
kidney diseases with a high risk of morbidity and mortality due to infection and
thrombosis. Goals of treatment are to reduce proteinuria to normal levels
thereby reducing symptoms and risk of complications. Children with NS should
initially be treated with prednisone or prednisolone at a dose of 60 mg/m2/day daily for 6 weeks followed by 40 mg/m2/day given every other day for an additional 6 weeks.
While most children are steroid responsive, approximately 20 % of children with
NS do not go into remission with steroids and should be treated with a
calcineurin inhibitor such as cyclosporine or tacrolimus. Some children with NS
who respond to steroids eventually have a frequently relapsing or
steroid-dependent course and may have significant side effects from cumulative
corticosteroid therapy. For these children, steroid-sparing medications are
required. Treatment with mycophenolate mofetil is recommended as first-line
therapy for treatment of frequently relapsing or steroid-dependent NS with
steroid toxicity due to its favorable side effect profile compared to
alternatives. If this is not effective, alternate agents such as
cyclophosphamide, calcineurin inhibitors, or rituximab could be considered after
careful review of the pros and cons of each medication with the child’s family.
Further randomized controlled trials are necessary to determine which agents are
most effective and to determine methods to predict medication response in
individual children.
Pediatric Kidney Stones—Avoidance and Treatment
Opinion statement
Pediatric stone disease is increasing in incidence and
healthcare costs. With more years at-risk for stone recurrence during their
lifetimes, children with nephrolithiasis constitute a high-risk patient
population that requires focused intervention through both medical and surgical
means. Through high-quality future studies to compare methods of stone
prevention and treatment, the burden of stone disease on the youngest members of
society may be ameliorated.
The Renal Transplant Patient—Updates for the Pediatrician
Opinion statement
Kidney transplantation remains superior to chronic dialysis
as the treatment option for end-stage renal disease (ESRD) in children and
adults. Recent refinements in pediatric kidney transplantation have mainly
sharpened our focus on the major cardiovascular (CV) morbidity risk for children
with kidney transplants, unfortunately including our recognition of how poorly
we are managing this risk, and on new approaches to gauge and manage
immunological risk, specifically monitoring our patients for evidence of too
much (BK virus) or not enough (donor-specific antibodies, DSAs, and
antibody-mediated rejection, AMR) immunosuppression.
Therapeutic Options for Neonatal Acute Kidney Injury (AKI)
Opinion statement
Acute kidney injury (AKI) occurs commonly in neonates and
is associated with increased mortality. Recent advances in neonatal intensive
care have resulted in improved patient survival, but this had not been
paralleled by improved outcomes for neonates with AKI. Management of AKI remains
supportive with no effective pharmacologic therapy to hasten the recovery
process. Emphasis is placed on the prevention and minimization of further renal
insults once AKI is established. Routine, regular assessments of renal function
with identification of high risk patients and those with incipient AKI are
essential so prevention strategies can be promptly initiated. Effective
prevention strategies include prompt restoration/preservation of organ perfusion
with fluid resuscitation to ensure an adequate effective circulating volume and
inotropic support as needed to maintain hemodynamic stability while also
avoiding/minimizing nephrotoxin exposure. In established AKI, efforts are
focused on maintaining organ perfusion while closely monitoring cumulative fluid
balance from admission and remaining cognizant of the need to minimize the
degree of fluid overload as this has also been associated with poor outcomes.
Continuous renal replacement therapy (CRRT) has become the preferred therapy for
managing fluid overload and metabolic disturbances in critically ill children
and adults but is still considered by many to be a heroic intervention for
neonates due in large part to numerous technical challenges. The recent
development of two miniaturized machines specifically designed to deliver CRRT
and hemodialysis (HD) to neonates will hopefully change this paradigm making it
easier for nephrologists and intensivists to safely offer and provide this
therapy to neonates. Prospective studies are needed to assess whether early
initiation of RRT with correction of fluid imbalance results in improved patient
survival and renal recovery.
Diagnosis and Treatment of Acute Kidney Injury in Pediatrics
Opinion statement
The term acute kidney injury
(AKI) has replaced the outdated term acute renal failure throughout the
literature and clinical practice. The term “injury” highlights the spectrum of
organ injury that may occur and reflects the fact that even small changes in
serum creatinine (rise of 0.3 mg/dL) can be associated with adverse outcomes. A
major advance in the field of AKI research has been the development of
standardized staged definitions of AKI that allow for comparison of incidence,
prevalence, and outcomes across studies. The Kidney Disease: Improving Global
Outcomes (KDIGO) AKI definition represents the most recent consensus definition
which is currently recommended for use in pediatric populations. Utilization of
standard AKI definitions has made it clear that AKI occurs often in hospitalized
patients and is associated with adverse short-term and long-term outcomes
(hospital length of stay, mortality, subsequent chronic kidney disease).
Awareness of the impact of AKI has resulted in increased efforts to understand,
diagnose, prevent, and manage AKI earlier in the course of illness. While
attempts at finding a treatment for AKI have been unsuccessful, largely due to
the lack of sensitivity of the primary biomarker, serum creatinine, there have
been many major advances in this field over the last 15 years. The development
of novel biomarkers to predict the development of AKI in a timely manner and
improve diagnostic accuracy is being pioneered by pediatric AKI researchers. The
development of risk stratification scores (renal angina) and functional bedside
tests (furosemide stress test) is enhancing our use of these biomarkers and our
ability to predict those patients most likely to develop severe AKI. The
recognition of the impact of fluid overload on mortality and hospital length of
stay in patients with severe AKI has prompted more timely and frequent use of
renal replacement therapy in critically ill children. Finally, we are
recognizing that children who suffer AKI are at long-term risk for the
development of chronic kidney disease and warrant follow-up.
Managing Vesicoureteral Reflux in the Pediatric Patient: a Spectrum of Treatment Options for a Spectrum of Disease
Opinion statement
Vesicoureteral reflux (VUR), or the reverse flow of urine
from the bladder into the ureter or renal collecting system, is characterized by
a wide spectrum of severity. Consequently, a spectrum of treatment options
exists that can be broadly divided into non-operative and surgical management.
Non-operative management is based on the natural history of reflux which
suggests that the vast majority of VUR, and in particular low grades of reflux,
will resolve spontaneously. Furthermore, most patients with lower grades of VUR
are at relatively low risk for recurrent pyelonephritis. The focus of
non-operative management is to prevent urinary tract infections that, when
combined with VUR, place a child’s kidney at risk for renal damage and potential
loss of renal function. This is typically achieved by optimizing bladder and
bowel function through a combination of dietary, behavioral, or pharmacologic
therapies and in some cases may include the use of antibiotic prophylaxis.
Surgical management seeks to mechanically correct VUR, either by endoscopic
injection of the intravesical ureteral tunnel with bulking agents (Deflux) or
with open or minimally invasive surgical ureteral reimplantation. Deflux
provides a less invasive but comparatively less successful alternative to
surgical reimplantation, and therefore, surgical reimplantation is more
frequently utilized in children with persistent high-grade VUR, known renal
damage, and associated bladder and bowel dysfunction. These approaches are not
mutually exclusive, and the management of VUR is highly individualized, taking
into consideration a litany of factors including a child’s age, sex, severity of
reflux, response to previous therapy options, the presence and severity of renal
damage, and concomitant bladder and bowel dysfunction. These considerations must
also be balanced with patient/parent preference and the potential consequences
of choosing a particular treatment strategy.
Current Management of Undescended Testes
Opinion statement
Undescended testes (UDTs) are a relatively common finding
in newborn males, especially in those born prematurely. Upon discovering a
non-intrascrotal testis, it is important to determine whether the testis is
palpable or non-palpable and whether the finding is unilateral or bilateral.
Imaging should not be used in this workup, as no current modality has been shown
to be adequately sensitive or specific to aid in management decisions. Patients
with UDTs diagnosed after 6 months of age should be referred to a specialist for
correction so that surgery may be performed within 1 year thereafter. This
allows testes to descend spontaneously if they are to do so while facilitating
early intervention to decrease the risk of subfertility and testicular
malignancy for those patients in whom spontaneous descent does not occur. The
surgical approach is often dependent on the location of the testis on physical
exam. Most orchiopexies for palpable testes are performed through an inguinal
incision, although a scrotal approach can be safely utilized depending on the
testis position. Diagnostic laparoscopy is most often used for non-palpable
testes, as it not only allows for the identification of an atrophic or absent
testicle, but it also provides an opportunity to perform an orchiopexy
simultaneously should a viable testis be found. Hormonal therapy is not
recommended for treatment of UDTs due to its low success rate, the incidence of
secondary re-ascent, and the possible detrimental effects on spermatogenesis.
Finally, patients with bilateral non-palpable UDTs require a more extensive
preliminary evaluation to rule out congenital adrenal hyperplasia (CAH) and
disorders of sexual development (DSD). This involves serum electrolytes,
karyotype analysis, and hormonal testing including a serum müllerian inhibiting
substance (MIS), in order to determine if testicular tissue is present and
functional.
Transition of Care for Adults with Congenital Urological Conditions
Opinion statement
Patients with congenital urologic conditions require
complex chronic care from birth to adulthood. Early in life, these patients may
establish close relationships with their specialized pediatric providers and
commonly undergo complex reconstructive procedures. When these patients approach
adulthood, they are faced with an informal transition to an adult urologist and
may become lost to follow-up. Goals of care during this transition period must
be identified and addressed by these patients’ future providers. The concept of
transition care is now emerging in urology. We propose that the most effective
model for successful transfer of care involves joint involvement from pediatric
and adult providers in the same setting.
An Update on Current Treatment Options for Pediatric Genitourinary Tract Tumors
Opinion statement
Most of the success and advances made in the care of
children with genitourinary (GU) cancer over the last 50 years are thanks in
large part to the efforts of the cooperative pediatric oncology groups both in
North America and Europe. Currently, children with tumors of the kidney,
bladder, prostate, and testis/para-testis largely enjoy a good prognosis thanks
to the outstanding research into these relatively rare diseases. Evidence-based
and protocol-driven care has resulted in outstanding rates of cure. The future
of the care for these children with GU malignancy is to preserve these outcomes
while minimizing the morbidity of therapy. One area of great interest across
these entities is risk stratification which holds the potential to reduce the
burden of therapy in those who can afford to do so and reserve more aggressive
treatments for those with risk factors for poor prognosis. From a surgical
standpoint, there is an increasing interest in reducing morbidity as well. For
renal tumors, this includes nephron-sparing and minimally invasive surgery when
possible. For bladder and prostate tumors, there is an increasing emphasis on
organ preservation and non-exenterative surgery. Regardless of these surgical
advances, the future of pediatric GU cancer therapy likely rests more in
tumor-biology-driven risk stratification and personalized therapy. Thus, from a
surgical standpoint, there is motivation for surgeons to think beyond just the
surgery and to be involved and be knowledgeable about all of the
multidisciplinary aspects of pediatric cancer care.
Urolithiasis in Children—Treatment and Prevention
Opinion statement
The incidence of stones is increasing in children
especially among Caucasian adolescents. Every child with stones deserves an
evaluation because the majority has a diagnosable metabolic defect and 50 % will
have a recurrence of stones. Diet, sedentary lifestyle, and climate change
contribute to the changing frequency of stones. There is some evidence to
support the following lifestyle changes: high fluid intake, low sugar intake,
low sodium intake, higher plant protein intake and lower animal protein intake,
normal calcium intake, high potassium citrate intake, moderate exercise, and
reduced environmental temperature. Our challenge is to help our patients commit
to and maintain a healthy lifestyle. After dietary influences, having a family
member with nephrolithiasis poses the greatest risk. Identifying the underlying
defect that permits stones to form in some members of the family will permit
targeted therapy. For instance there is a “gain of function” mutation in the
calcium-sensing receptor gene in families with autosomal dominant hypocalcemic
hypercalciuria. Treating these patients with vitamin D to increase the blood
calcium results in marked hypercalciuria, nephrocalcinosis, and nephrolithiasis.
Thus, the second challenge in addition to lifestyle changes is to identify the
gene defects permitting stone formation.
Creating a Clinical Systems Integration Strategy to Drive Improvement
Opinion statement
Health care as an industry has lagged behind other
industries in leveraging its data for improvement. Nevertheless, exemplary
health care organizations have demonstrated substantial improvements for their
patients by integrating data across their clinical, operational, and financial
systems. We believe that all providers can use their data to drive improvements
within the systems in which they practice. Here we explain the strides that
Texas Children’s Hospital has taken to improve care through the power of data.
The foundation of clinical systems integration is converting the plethora of
data acquired during the daily operations of an entire enterprise into analytics
for meaningful interpretations of disease spectrums across the enterprise. Texas
Children’s Hospital focused on three domains of this foundation: (1) science-
and evidence-based practice, (2) quality improvement education and
implementation, and (3) data analytics and predictive analytics. As a result, we
have seen improvements in the care for populations of children—which will be
described at length for asthma, appendicitis, and cross-cutting payment reform
models. Clinical systems integration requires investments in technical resources
(i.e., software and hardware), re-engineering workflows, and human capital. The
investments must be matched with leadership that understands and participates in
the transformation—along with a governance structure that can help prevent
fragmentation and inefficiencies that can occur in complex systems. Finally, the
system should also be nimble enough to respond to changing internal and external
quality demands.
A Practical Guide to Conducting Quality Improvement in the Health Care Setting
Opinion statement
Quality improvement uses rigorous methodology to evaluate
systemic changes to patient care processes in an effort to improve patient
outcomes, the patient and family experience of care, and the safety and value of
the care delivered. This article introduces the Model for Improvement, which was
developed by the Associates for Process Improvement in the early 1990s using an
adaptation of a real-life improvement project. The example will explore how a
primary care practice uses the Model for Improvement to maximize the value and
safety of care they deliver for children presenting with community-acquired
pneumonia with an initial focus on appropriate first-line antibiotic treatment.
The three fundamental questions which form the foundation of this approach are
explored through the case example: (1) What are we trying to accomplish? (2) How
will we know that a change is an improvement? (3) What changes can we make that
will result in improvement? Examples of many of the fundamental tools used in
the course of quality improvement work, such as a key driver diagram, run chart,
and plan-do-study-act (PDSA) cycle, are explored throughout the text. Finally, a
discussion of implementation and sustainability of improvement gains is
introduced. This article serves as a primer on quality improvement in health
care and serves as a foundation for subsequent articles in this issue as well as
future learning.
Partnering with Parents and Families to Provide Safer Care: Seeing and Achieving Safer Care through the Lens of Patients and Families
Opinion statement
Humans function best within relationships that are
supportive, nurturing, collaborative, and filled with mutual respect. Why, then,
do these relationships seem to disappear at the clinical bedside? For both
clinicians and patients and their families, the need for empathy and compassion
is never greater than during a time of sickness and tragedy. This is especially
true in pediatric care, among some of our most vulnerable patients. The best
medical care and the safest outcomes derive from collaborative relationships. In
true partnerships, clinicians feel comfortable expressing their own humanity,
and patients and their families are satisfied that they are being heard. With
empathetic communication to family-centered care process, we can create a
profound setting for healing.
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