Gastroenterology and GI Surgery

Minimally invasive surgery brings lasting relief to pediatric achalasia patients

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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.