Gastroenterology and GI Surgery

audience members at the 2018 Gluten Free Expo keynote

Dispelling gluten-free myths and patient education headline the 2018 Washington DC Gluten-Free Education Day and Expo

audience members at the 2018 Gluten Free Expo keynote

On June 10, 2018, some of the brightest minds in the field gathered at the 2018 Washington DC Gluten-Free Education Day and Expo to discuss opportunities and challenges associated with living a gluten-free life.

The one-day expo focused on how the food we eat affects our brains, how to dispel gluten-free myths and how to think outside the box with new recipes and cooking demonstrations from local restaurants, bakeries and chefs.

Keynote speakers for this year’s symposium included Benny Kerzner, M.D., medical director, Celiac Disease Program at Children’s National, Jocelyn Silvester, M.D., director of Research, Celiac Disease Program at Boston Children’s Hospital and Ian Liebowitz, M.D. from Pediatric Specialists of Virginia.

Additionally, Edwin Liu, M.D., from Colorado Children’s Hospital and Ilana Kahn, M.D., from Children’s National gave a joint keynote on the autoimmune connection, focusing on conditions related to celiac disease and the gut-brain connection.

Teen mentors gave a panel discussion for the 13+ age group attending the conference in a session called “For Teens from Teens!” during which they discussed the challenges of navigating a gluten-free lifestyle as a teen.

In addition to the useful educational sessions, attendees visited many of the 57 vendor tables with gluten-free product samples voting for the best sweet and savory winners.

The Celiac Disease Program at Children’s National Health System started in 2009 to improve the way pediatric celiac disease is diagnosed and treated. Working in partnership with concerned members of our community, our Celiac Disease Program brings together a team of expert physicians, nurses, nutritional consultants and professional counselors dedicated to developing a national model for detecting and treating celiac disease in children.

Making the grade: Children’s National is nation’s Top 5 children’s hospital

Children’s National rose in rankings to become the nation’s Top 5 children’s hospital according to the 2018-19 Best Children’s Hospitals Honor Roll released June 26, 2018, by U.S. News & World Report. Additionally, for the second straight year, Children’s Neonatology division led by Billie Lou Short, M.D., ranked No. 1 among 50 neonatal intensive care units ranked across the nation.

Children’s National also ranked in the Top 10 in six additional services:

For the eighth year running, Children’s National ranked in all 10 specialty services, which underscores its unwavering commitment to excellence, continuous quality improvement and unmatched pediatric expertise throughout the organization.

“It’s a distinct honor for Children’s physicians, nurses and employees to be recognized as the nation’s Top 5 pediatric hospital. Children’s National provides the nation’s best care for kids and our dedicated physicians, neonatologists, surgeons, neuroscientists and other specialists, nurses and other clinical support teams are the reason why,” says Kurt Newman, M.D., Children’s President and CEO. “All of the Children’s staff is committed to ensuring that our kids and families enjoy the very best health outcomes today and for the rest of their lives.”

The excellence of Children’s care is made possible by our research insights and clinical innovations. In addition to being named to the U.S. News Honor Roll, a distinction awarded to just 10 children’s centers around the nation, Children’s National is a two-time Magnet® designated hospital for excellence in nursing and is a Leapfrog Group Top Hospital. Children’s ranks seventh among pediatric hospitals in funding from the National Institutes of Health, with a combined $40 million in direct and indirect funding, and transfers the latest research insights from the bench to patients’ bedsides.

“The 10 pediatric centers on this year’s Best Children’s Hospitals Honor Roll deliver exceptional care across a range of specialties and deserve to be highlighted,” says Ben Harder, chief of health analysis at U.S. News. “Day after day, these hospitals provide state-of-the-art medical expertise to children with complex conditions. Their U.S. News’ rankings reflect their commitment to providing high-quality care.”

The 12th annual rankings recognize the top 50 pediatric facilities across the U.S. in 10 pediatric specialties: cancer, cardiology and heart surgery, diabetes and endocrinology, gastroenterology and gastrointestinal surgery, neonatology, nephrology, neurology and neurosurgery, orthopedics, pulmonology and urology. Hospitals received points for being ranked in a specialty, and higher-ranking hospitals receive more points. The Best Children’s Hospitals Honor Roll recognizes the 10 hospitals that received the most points overall.

This year’s rankings will be published in the U.S. News & World Report’s “Best Hospitals 2019” guidebook, available for purchase in late September.

child measuring belly with tape measure

Children’s obesity research team presents compelling new findings

child measuring belly with tape measure

Faculty from Children’s National Health System’s Department of Psychology & Behavioral Health set out to learn if any demographic, psychiatric, or cognitive factors play a role in determining if an adolescent should be eligible for bariatric surgery, and what their weight loss outcomes might be. Presenting at the Society for Pediatric Psychology Annual Conference earlier this month, a group of researchers, fellows and clinicians, including surgeons from Children’s National showcased their findings. One of the posters developed by Meredith Rose, LGSW, ML, who works as an interventionist on a Children’s National clinical research team, received special recognition in the Obesity Special Interest Group category.

One presentation reported on a total of 222 pediatric patients with severe obesity, which is defined as 120 percent of the 95th percentile for Body Mass Index. Mean age of the participants was 16 years of age, 71 percent were female and 80 percent where Hispanic or non-White. As part of their preparation for surgery, all patients were required to complete a pre-bariatric surgery psychological evaluation, including a clinical interview and Schedule for Affective Disorders and Schizophrenia (KSADS-PL) screening. The studies by the Children’s teams were based on a medical record review of the pre-screening information. Adolescents being evaluated for surgery had high rates of mental health diagnoses, particularly anxiety and depression, but also included Attention Deficit Hyperactivity Disorder, eating disorders, and intellectual disability.

Another Children’s presentation at the conference looked at weight loss outcomes for adolescents based on IQ and intellectual disability. Overall, neither Full Scale IQ from the Wechsler Abbreviated Intelligence Scale – 2nd edition, nor the presence of an intellectual disability predicted weight loss following surgery.

“The sum of our research found that kids do really well with surgery,” said Eleanor Mackey, PhD, assistant professor of psychology and behavioral health. “Adolescents, regardless of the presence of intellectual disability areas are likely to lose a significant amount of weight following surgery,” added Dr. Mackey.

“This is a particularly important fact to note because many programs and insurers restrict weight loss surgery to ‘perfect’ candidates, while these data points demonstrate that our institution does not offer or deny surgery on the basis of any cognitive characteristics,” says Evan P. Nadler, M.D., associate professor of surgery and pediatrics. “Without giving these kids a chance with surgery, we know they face a lifetime of obesity, as no other intervention has shown to work long-term in this patient population. Our research should empower psychologists and physicians to feel more confident recommending bariatric surgery for children who have exhausted all other weight loss options.”

The research team concluded that examining how individual factors, such as intellectual disability, psychiatric diagnoses, and demographic factors are associated with the surgery process is essential to ensuring adequate and empirically supported guidelines for referral for, and provision of bariatric surgery in adolescents. Next steps by the team will include looking into additional indicators of health improvement, like glucose tolerance, quality of life, or other lab values, to continue evaluating the benefits of surgery for this population.

Janelle Vaughns

Few prescribing options exist for obese kids

Janelle Vaughns

“We are making progress in expanding the number of medicines with pediatric labeling, but we need to do more concerning providing dosing guidelines for children with obesity,” says Janelle D. Vaughns, M.D., director of bariatric anesthesia at Children’s National and the lead study author.

Despite years of study and numerous public health interventions, overweight and obesity continue to grow in the U.S. Currently, more than two-thirds of adults have these issues, according to data from the Centers for Disease Control and Prevention. Children and adolescents also are being affected at an increasing rate: About one in five is obese. Obesity and overweight have been linked with a bevy of health problems, including Type 2 diabetes, high blood pressure, coronary heart disease and stroke.

Additionally, because obesity increases the percentage of fat tissue in relation to lean tissue and enlarges kidney size, it can affect how readily the body takes up, metabolizes and excretes medicines.

This latter issue can be particularly problematic in children, a population for whom relatively few drug studies exist. Now, a study team that includes Children’s National Health System researchers suggests that, despite the U.S. Congress providing incentives to drug manufacturers to encourage the study of medications in children, few approved drugs include safe dosing information for obese kids.

The study, performed in conjunction with the Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research, surveyed pediatric medical and clinical pharmacology reviews under the FDA Amendments Act of 2007 and the FDA Safety and Innovation Act of 2012. The researchers used search terms related to weight and size to determine the current incorporation of obesity as a covariate in pediatric drug development.

Of the 89 product labels identified, none provided dosing information related to obesity. The effect of body mass index on drug pharmacokinetics was mentioned in only four labels, according to the study “Obesity and Pediatric Drug Development,” published online Jan. 19, 2018, in The Journal of Clinical Pharmacology.

“We are making progress in expanding the number of medicines with pediatric labeling, but we need to do more concerning providing dosing guidelines for children with obesity,” says Janelle D. Vaughns, M.D., director of bariatric anesthesia at Children’s National and the lead study author. “Moving forward, regulators, clinicians and the pharmaceutical industry should consider enrolling more obese patients in pediatric clinical trials to facilitate the safe and effective use of the next generation of medicines by obese children and adolescents.”

Study co-authors include Children’s Gastroenterologist Laurie Conklin, M.D., and Children’s Division Chief of Clinical Pharmacology Johannes N. van den Anker, M.D., Ph.D.; Ying Long, Pharm.D., University of Southern California; Panli Zheng, Pharm.D., University of North Carolina at Chapel Hill; Fahim Faruque, Pharm.D., University of Maryland; and Dionna Green, M.D., and Gilbert Burckart, Pharm.D., both of the FDA.

Research reported in this news release was supported by the Eunice Kennedy Shriver National Institute of Child Health and Human Development of the National Institutes of Health under award number 5T32HD087969.

gluten-free diet app

Celiac Program offers gluten-free diet app

gluten-free diet app

The Celiac Disease Program at Children’s National has created a new digital app for celiac disease and gluten-free diet management.

Celiac disease affects approximately one in 100 children, making it one of the most common conditions in children. To help patients and their families understand more about the disease and live a safe, gluten-free lifestyle, the Celiac Disease Program at Children’s National has created a gluten-free diet app.

The Celiac Disease and Gluten-Free Diet Digital Resource Center app was designed to offer all of the Celiac Disease Program’s educational tools in one place. “We have so many incredibly valuable resources, but all were housed in different places, making it very difficult to show people where to find them,” explains Vanessa Weisbrod, education director of the Celiac Disease program. “We created the app as a way to put everything in one place, but also as a mechanism for sharing our tools with the rest of the world.”

Available through the Apple App Store and Android Marketplace, the app gives users access to a variety of resources, including:

  • Safe and unsafe ingredient lists
  • Grocery store shopping tips
  • Gluten-free recipes accompanied by instructional cooking videos
  • Nutrition education
  • A monthly podcast
  • News feed of hot topics in the celiac and gluten-free community
  • Continuing education seminars led by celiac disease and gluten-free diet experts

“We are one of the few celiac programs in the country truly dedicated to developing high quality in-house patient education tools for families living with celiac disease,” says Weisbrod. “As we’ve shown our materials to other programs, they always ask us to share them. Through the app, anyone living a gluten-free lifestyle now has access to these remarkable tools.”

Providing more treatment options for kids with hep C

Parvathi Mohan

The Children’s National Hepatology Program, led by Parvathi Mohan, M.D., is one of the few centers on the East Coast participating in clinical trials offering regimens using the direct acting antiviral agent sofosbuvir to treat children and adolescents with chronic hepatitis C virus (HCV) infection.

The Children’s National Hepatology Program, led by Parvathi Mohan, M.D., is one of a few such centers on the East Coast of the U.S. and one of about 30 Hepatology programs throughout the U.S. and Europe, participating in clinical trials offering regimens using the direct acting antiviral agent sofosbuvir to treat children and adolescents with chronic hepatitis C virus (HCV) infection. Sofosbuvir-based treatments have been approved for adults with chronic HCV infection and proven to be highly successful in viral clearance close to 95 percent with minimal side effects, but they are not yet FDA approved for use in pediatrics.

Current HCV therapies available for children use pegylated-interferon and weight-based ribavirin but are unsatisfactory with only about 50 percent viral clearance in subjects with genotype 1, which is the most prevalent in the country. Most children treated with these therapies experience adverse events such as influenza-like illness, headache and a more concerning effect on growth and development.

Children’s is actively participating in several clinical trials to treat HCV with the ultimate goal of eradicating the virus, and these current trials with sofosbuvir are accepting children and adolescents ages 3-17 infected with all genotypes of the virus.

Scientist with centrifuge

Giving fat cell messages a positive spin

Woman on a scale

Study findings offer hope to the nearly 2 billion adults who are overweight or obese worldwide that detrimental effects of carrying too much weight can recede. (Image source: Centers for Disease Control and Prevention)

Losing weight appears to reset the chemical messages that fat cells send to other parts of the body that otherwise would encourage the development of Type 2 diabetes, substantially reducing the risk of that disease, a team led by Children’s National Health System researchers report in a new study. The findings offer hope to the nearly 2 billion adults who are overweight or obese worldwide that many of the detrimental effects of carrying too much weight can recede, even on the molecular level, once they lose weight.

In 2015, Robert J. Freishtat, M.D., M.P.H., Chief of Emergency Medicine at Children’s National and Associate Professor of Pediatrics, Emergency Medicine and Integrative Systems Biology at The George Washington University School of Medicine & Health Sciences, and colleagues showed that fat cells (also known as adipocytes) from people who are obese send messages to other cells that worsen metabolic function. These messages are in the form of exosomes, nanosized blobs whose contents regulate which proteins are produced by genes. Exosomes are like “biological tweets,” Dr. Freishtat explains — short signals designed to travel long distances throughout the body.

Dr. Freishtat’s earlier research showed that the messages contained in exosomes from patients who are obese alter how the body processes insulin, setting the stage for Type 2 diabetes. However, says Dr. Freishtat, it has remained unclear since that publication whether these aberrant messages from adipocytes improve after weight loss.

“We’ve known for a long time that too much adipose tissue is bad for you, but it’s all moot if you lose the weight and it’s still bad for you,” he explains. “We wanted to know whether these negative changes are reversible. If you reduce fat, does the disease risk that goes along with excess fat also go away?”

Details of the study

To investigate this question, Dr. Freishtat and colleagues worked with six African American adults scheduled to receive gastric bypass surgery — a nearly surefire way to quickly lose a large amount of weight. The volunteers, whose average age was 38 years, started out with an average body mass index (BMI) of 51.2 kg/m2. (The Centers for Disease Control and Prevention considers a healthy BMI to range between 18.5 to 24.9.)

Two weeks before these volunteers underwent surgery, researchers collected blood samples and took a variety of measurements. The researchers then performed a repeat blood draw and measurements one year after the surgery took place, when the volunteers’ average BMI had dropped to 32.6.

Dr. Freishtat and colleagues drew out the adipocyte-derived exosomes from both sets of blood samples and analyzed their contents. The team reports in the January 2017 issue of Obesity that at least 168 microRNAs — the molecules responsible for sending specific messages — had changed before and after surgery. Further analyses showed that many of these microRNAs were involved in insulin signaling, the pathways that the body uses to regulate blood sugar. By changing these outgoing microRNAs for the better, Dr. Freishtat says, adipocytes actively were encouraging higher insulin sensitivity in other cells, warding off Type 2 diabetes.

Sure enough, each volunteer had better insulin sensitivity and other improved markers of metabolic health post-surgery, including lower branched chain amino acids and a two-fold reduction in their glutamate to glutamine ratio.

“These volunteers were essentially cured of their diabetes after surgery. The changes we saw in their surgery-responsive microRNAS correlated with the changes we saw in their metabolic health,” Dr. Freishtat says.

A glimpse into the future

Dr. Freishtat and colleagues plan to study this phenomenon in other types of weight loss, including the slower and steadier paths that most individuals take, such as improving diet and doing more exercise. The team expects to see similar changes in exosomes of patients who lose weight in non-surgical ways.

By further examining the aberrant messages in microRNAs being sent out from adipocytes, he says, researchers eventually might be able to develop treatments to reverse metabolic problems in overweight and obese patients before they lose the weight, improving their health even before the often challenging process of weight loss begins.

“Then, if you can disrupt this harmful signaling in combination with weight-loss strategies,” Dr. Freishtat says, “you’re really getting the best of both worlds.”

Eventually, he adds, tests might be available so that doctors can warn patients that their fat cells are sending out harmful messages before disease symptoms start. By giving patients an early heads up, Dr. Freishtat says, patients might be more likely to heed advice from physicians and make changes before it’s too late.

“If doctors could warn patients that their fat is telling their blood vessels to fill up with plaque and trigger a heart attack in 10 to 20 years,” he says, “patients might be more compliant with treatment regimens.”

Photo of patient walking through the hallways

Cardiovascular and GI symptom relief

Photo of patient walking through the hallways

By adding antroduodenal manometry to the cardiovascular tilt table test, doctors in the GI Motility Program are able to collaborate with cardiologists to treat both cardiovascular and gastroenterological symptoms in children with complex orthostatic intolerance.

Physicians treating pediatric patients with complex orthostatic intolerance issues often face a double whammy – the children exhibit symptoms of their cardiovascular condition, but secondary issues relating to gastrointestinal functions are also common. That’s why cardiologists and gastroenterologists at Children’s National Health System have collaborated in recent years to diagnose and find comprehensive treatment solutions for patients with conditions such as neurally mediated hypotension (NMH) and postural orthostatic tachycardia syndrome (POTS).

Their innovative team approach was highlighted in a study of their first 35 patients from age 10-23, published in the Journal of Pediatric Gastroenterology and Nutrition last fall – patients who experienced relief for multiple symptoms thanks to treatment at Children’s National.

The collaboration is possible because of the advancements in gastroenterological diagnostics here – specifically related to the comprehensive GI Motility testing. While patients with POTS can exhibit an abnormal heart rate, low blood pressure, headaches, fatigue or weight loss, their gastroenterological symptoms like nausea and constipation are often under recognized, says Children’s Director of the Comprehensive GI Motility Program Anil Darbari, M.D., MBA, who co-authored the study with a team including Jeffrey Moak, M.D., Director of the Electrophysiology Program at Children’s.

Through antroduodenal manometry of the stomach and upper small intestine, in combination with the cardiovascular tilt table test, the team has been able to more accurately diagnose and treat these patients.

“We have the ability to do them together, thereby making the connection between these two conditions and providing a path for management or treatment,” Dr. Darbari said.

Typically, by the time he sees these patients, those with complex medical issues have often seen several other gastroenterologists at multiple centers, and have been through a lot. The study found that overall, antroduodenal manometry was abnormal in 26 of 35 (74%) patients either at baseline or during tilt table testing in subjects with orthostatic intolerance. Darbari and his colleagues concluded that upper GI motility studies should always be part of the comprehensive evaluation for this population of patients, because treating the autonomic condition improved their gastroenterological symptoms as well.

What does the future look like for this double-whammy approach?

“We have a cohort of over 100 patients with these issues who have been evaluated using this combined diagnostic approach,” Dr. Darbari said. “This gives the team the knowledge and opportunity to help even more kids, which is very rewarding.”

In addition to comprehensive assessment and medical approaches, Children’s National is home to leaders in minimally invasive laparoscopic and endoscopic diagnostic and corrective procedures that have enabled Children’s GI motility specialists and the teams they collaborate with to offer the next level of comprehensive pediatric medical care.

Drug dosing guidelines poor fit for obese patients

Children’s National researchers are among the top teams examining how obesity alters pharmacokinetics and the effect of body mass index on drug dosing and treatment outcomes specifically for pediatric and adolescent patients.

Obesity affects about 12.7 million U.S. children and adolescents – or about 1 in 6 kids across the nation, according to the Centers for Disease Control and Prevention. Despite this, there is a significant dearth of dosing guidelines for practitioners, for example pediatric anesthesiologists, to follow when administering potent anesthetics to pediatric patients who are obese.

Janelle D. Vaughns, M.D., director of bariatric anesthesia within the Division of Anesthesiology, Pain and Perioperative Medicine, says Children’s National Health System sees pediatric and adolescent patients of extreme weight (as much as 450 pounds) presenting for weight-loss surgery. In order to ensure that patients remain anesthetized during their surgical procedures, anesthesiologists use various classes of drugs, including hypnotics, muscle relaxants and pain medications. Dr. Vaughns says providers across the nation face similar challenges when determining accurate and precise dosing of drugs for obese pediatric patients.

“Medical guidelines calibrated for a 13-year-old of typical weight cannot be applied to a 13-year-old who weighs 400 pounds. Because morbid obesity in kids is a relatively new phenomenon in our country and globally, there are no formal guidelines to aid with dosing. In this scenario, most doctors extrapolate from guidelines written for lean patients. Because anesthetic drugs are so strong, it is essential to use the correct dose in all patients,” she says.

A recent brief report that Dr. Vaughns co-authored examines this issue. Researchers at Children’s National and the Washington Hospital Center conducted a retrospective review for 440 adult patients who received rapid sequence endotracheal intubation (RSI) in an urban, tertiary care academic Emergency Department. The patients received succinylcholine (a muscle relaxant) and etomidate (a short-acting anesthetic), whose doses are ideally calculated in milligrams per kilogram of total body weight.

The work, published in the December 2016 issue of American Journal of Emergency Medicine, reinforced the importance of data-driven guidelines for all patients. The research team found that the 129 obese patients included in the study were more likely to receive too little of the studied drugs while the 311 non-obese patients studied were more likely to receive too much medicine.

“Our single-center study demonstrates that obesity is a significant risk factor for underdosing RSI medications, whereas non-obesity is a risk factor for overdosing of these medications,” the research team concludes. This study also was reviewed and featured by the New England Journal of Medicine “Journal Watch” in October 2016.

Broadly, the issue of dosing potent medicines for pediatric obese patients is a national public health concern, Dr. Vaughns says. Research teams across the nation have made a concerted effort to publish papers on topics such as how obesity alters pharmacokinetics – how the body takes up, distributes and disposes of powerful medicines – and the deleterious effect of unhealthy body mass index on treatment outcomes for children with diseases such as acute myeloid leukemia.

Dr. Vaughns is among the clinician researchers working with the Pediatric Trials Network (PTN), sponsored by the Eunice Kennedy Shriver National Institute of Child Health and Human Development, to fill this research gap. Working as a team, she, Evan Nadler, M.D., a bariatric surgeon, and Johannes N. van den Anker, M.D., Ph.D., division chief of Clinical Pharmacology, enroll pediatric patients in ongoing trials with a special focus on surgical patients who are obese.

The network is currently conducting pediatric studies at a number of locations, including Children’s National, leveraging blood samples and other specimens drawn during regular care to better understand how medicines routinely used in pediatric patients actually work in kids and to determine appropriate dosing.

Ultimately, the information PTN researchers discover from their multi-year studies will help the Food and Drug Administration update medicine labels to reflect safer, more accurate and more effective dosing for all pediatric patients.

Children’s National gastroenterologist honored with Excellence of Healthcare Award

Benny Kerzner, M.D., gastroenterologist and creator of the Division of Gastroenterology, Hepatology, and Nutrition at Children’s National Health System, received the Excellence of Healthcare award from the Crohn’s & Colitis Foundation of America (CFFA) – Greater Washington, D.C. and Virginia Chapters during the organization’s Evening of Hope Gala on November 17, 2016. The award is presented to a medical professional who has positively impacted patient lives and furthered the mission of the CCFA to cure Crohn’s disease and ulcerative colitis and to improve the quality of life of children and adults affected by these diseases. Learn more here.

Minimally invasive surgery brings lasting relief to pediatric achalasia patients

tkane_atmospheric_2015

Achalasia affects only a small number of people around the world, estimated at 1.6 per 100,000, and children make up fewer than 5 percent of that total. In most cases, the causes are unknown, but it is attributed to a combination of heredity and autoimmune or nerve cell disorders. For adults, treatment might include oral medication to prevent narrowing, balloon dilation, or botulinum toxin injections to relax the muscle at the end of the esophagus. For a growing child, who faces not just months but a lifetime of injections and potential repeat procedures, these methods aren’t viable. Instead, surgical correction is the standard of care. In the past 10 years, the surgical option evolved from a traditional open procedure with weeks of recovery and pain to less-invasive approaches.

“The total number of children with achalasia is small,” says Timothy D. Kane, M.D., Division Chief of General and Thoracic Surgery at Children’s National Health System. “But Children’s National treats more of these cases than most other children’s hospitals around the world, and that gives us the ability to look at a larger population and see what works.”

Dr. Kane is senior author of a study recently published in the Journal of Pediatric Surgery that analyzed the outcomes from nearly a decade’s worth of these cases to gauge the effectiveness of two different minimally invasive surgical approaches for children with achalasia.

A look at the two surgical options

The most common surgical intervention is laparoscopic Heller myotomy, performed through small incisions in the belly. Additionally, Dr. Kane and the Children’s surgical team are one of only two teams in the country who perform a different procedure called peroral endoscopic myotomy (POEM) on children. The POEM procedure is completed entirely through the mouth using an endoscope, with no additional incision needed. The procedure is commonly used for adult achalasia cases, but is not widely available for children elsewhere as it requires specialized training and practice to perform.

“Heller myotomy works very well for most kids — that’s why it’s the standard of care,” Dr. Kane says. “Our study found that patients who underwent the POEM procedure experienced the same successful outcomes as Heller patients, and we already knew from adult data that POEM patients reported less pain following surgery — a win-win for children.”

The retrospective study included all children who had undergone surgical treatment for achalasia at Children’s from 2006 to 2015. Since achalasia cases are few and far between, with most children’s hospitals seeing maybe one to five cases over 10 years, collecting reliable data on outcomes is challenging. This study provides a large enough sample to allow doctors to use the findings as a guide to find the interventions that are the best fit for each patient.

“Now we’re very comfortable presenting families with two really good options and letting them choose the one that works best for them,” he concludes.

Imagine the feeling of food stuck in your throat. For children with esophageal achalasia, that feeling is a constant truth: The muscles in the esophagus fail to function properly and the lower valve, or sphincter, of the esophagus controlling the flow of food into the stomach doesn’t relax enough to allow in food — causing a backup, heartburn, chest pain, and many other painful symptoms. For children, surgery is the best hope for permanent relief.

Unbelievable survivability rates for short bowel patients

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When other doctors ask Clarivet Torres, M.D., how she is getting the best survivability rates for patients with Short Bowel Syndrome (SBS), she says her success is because of teamwork.

The Intestinal Rehabilitation Program (IRP) at Children’s National, started in 2007 when Dr. Torres joined the health system and became the program’s director, has shown 98 percent survivability for patients with SBS over a period of nine years. That’s compared with a recent study from the Pediatric Intestinal failure consortium (Predictors of Enteral Autonomy in Children’s with Intestinal Failure: a Multicenter Cohort Study), which showed that 43 percent of the patients died or underwent transplantation over a median follow-up of 33.5 months.

Intestinal failure often prevents these patients from digesting enough nutrients and fluids to maintain proper growth, and they often require parenteral nutrition (PN). Dr. Torres’ team has helped to wean 91.3 percent of patients from PN, compared with the above study, which showed that enteral autonomy was achieved in 43 percent.

Based on the outcomes for the first 120 children with SBS treated in Children’s National’s IRP from 2007 to 2016, Dr. Torres says that with meticulous and aggressive medical/surgical management, even patients with advanced liver disease can show improvement in liver functions and nutritional parameters with the ability to discontinue parenteral nutrition and avoid the need for transplantation.

“These are very, very good results for any program and ours has been growing substantially in the last 10 years,” Dr. Torres says. “We are like a family, we are very good at teaching so everyone knows how to care for these patients.”

Cross-departmental collaboration

Her main focus as director has been spreading the word about SBS across the departments. For example, the ER knows to start IV fluids on these patients right away or to keep watch for sepsis symptoms. From nurses, pediatric residents, and surgeons to radiologists and the ER, Dr. Torres has encouraged the sharing of knowledge and teaching how to respond to SBS patients.

Dr. Torres also attributes the success of the Children’s National’s program to having a multidisciplinary intestinal rehabilitation team who are trained to follow up with these highly complex patients with SBS.  “In general, these patients have a very high morbidity-mortality rate, and it’s important to be close to follow up.”

Members of  the IRP includes, a dedicated surgeon, Anthony Sandler, M.D., and four supporting GI doctors (Parvathi Mohan, M.D., Vahe Badalyan, M.D., Sona Sehgal, M.D., and Muhammad Khan, M.D.).

Other important members are one physician assistant, two nurse practitioners, two coordinators, one dietitian, one social worker, one case manager, and devoted nurses who work in the specialized Intestinal Rehabilitation Unit.

Having a dedicated director and surgeon also is a new perspective. Focusing on this group of patients allows Drs. Torres and Sandler to become experts in the medical and surgical management of the patients with short bowel and intestinal failure.

A closer look inside the program

The goal of the IRP is to optimize bowel function through the use of multiple therapies and to eventually wean patients with intestinal failure from parenteral nutrition. The medical treatment focuses on comprehensive dietary management with very precise control of metabolic balance and prompt and effective treatment of complications.

Pro-adaptive surgery, such as stoma closure, ostomy in continuity, stricturoplasty, enteroplasty, and autologous gut reconstruction, with the longitudinal intestinal lengthening and tailoring (LILT) and serial transverse enteroplasty (STEP) procedures, may produce dramatic clinical improvement in patients with SBS.

The use of specialized enteral feeding programs by the experience medical team helps to maintain nutrition and hydration, which are important factors in long-term survival. Other important components of the program are ongoing parent education and support, and promoting an optimal quality of life. Intestinal transplantation with MedStar Georgetown University Hospital is an option for patients who fail treatment.

“The Intestinal Rehabilitation at Children’s National provides children with intestinal failure the chance to receive comprehensive medical and surgical care, giving them the chance for improved long-term survival, including weaning from parenteral nutrition and avoidance of the need for transplantation and long-term immunosuppression,” Dr. Torres says.