Clinical trial NCT01515501: Endoscopic Mucosal Resection for the Diagnosis of a-Ganglionosis, a Controlled Prospective Trial (EDGE Trial)
Official title: Endoscopic Mucosal Resection for the Diagnosis of a-Ganglionosis, a Controlled Prospective Trial (EDGE Trial)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Constipation
Hirschsprung Disease
Keywords: Constipation
Hirschsprung Disease
Lead sponsor: Massachusetts General Hospital
Sponsor class: OTHER
First posted: 2012-01-24
Enrollment: 12
Sex: ALL
Minimum age (years): 10
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: DIAGNOSTIC

Brief summary: Patients undergoing routine rectal suction biopsy will undergo as part of the study an additional Endoscopic mucosal resection. The biopsy results will also be correlated with patient's clinical data including clinical history, Bristol stool scale, anorectal manometry results, and SITZ marker studies. Cost and recovery time will be compared.

Detailed description: Background: Adult and pediatric patients presenting with chronic constipation and/or motility disorders may be referred for rectal biopsy to rule out aganglionosis or Hirschsprung's disease. The traditional diagnostic test, rectal suction biopsy, is a blind technique and is insensitive in confirming the diagnosis. Frequently, patients require subsequent referral for a surgical full thickness biopsy.

Hypothesis: Endoscopic mucosal resection (EMR) will improve the diagnostic yield for aganglionosis and decrease the need for subsequent surgical full thickness biopsy.

Methods: This is a prospective, single center, controlled investigation of EMR for the diagnosis of colonic aganglionosis / Hirschsprung's disease. Patients who are offered rectal suction biopsy will be offered enrollment. Enrolled patients will have one additional procedure (EMR) at the time of their rectal suction biopsy. Specimens will be analyzed pathologically for size, submucosal tissue adequacy, the presence of ganglionic tissue, and positivity by acetylcholinesterase staining. The adequacy of the tissue specimen, the proportion of diagnostic specimens, and the proportion of patients that would have required subsequent referral for full thickness biopsy, will be compared. Variable such as cost and recovery time will be compared. Biopsy results will be compared with patient's pre-endoscopy clinical data including their history of constipation, results of SITZ marker studies, Bristol stool scale, and anorectal manometry.

Results: The primary outcome variable will be the proportion of patients with a diagnostic specimen in each group. Secondary outcome variables will include the size of the specimen, the presence of ganglionic tissue, the presence of submucosal tissue, and the positivity of the acetylcholinesterase stain. The biopsy results will also be correlated with patient's clinical data including clinical history, Bristol stool scale, anorectal manometry results, and SITZ marker studies. Cost and recovery time will be compared.

Discussion: The proposed investigation may demonstrate that a simple endoscopic test, which uses direct visualization, can improve the diagnostic yield of rectal biopsies for Hirschsprung's disease, and spare patients an additional surgical full thickness rectal biopsy.

Eligibility criteria: Inclusion Criteria:

* 10 years of age or older
* offered Rectal Suction biopsy

Exclusion Criteria:

* any contraindication to general anesthesia or conscious sedation
* contraindication to endoscopy
* untreated or unmanageable coagulopathy
* thrombocytopenia (\<50)
* inability to provide informed consent.

Primary outcomes:
measure: The number of EMR Biopsies that are diagnostic specimens
timeFrame: One Year
Secondary outcomes:
Other outcomes:
Locations:
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
Study officials:
affiliation: Massachusetts General Hospital
name: Braden Kuo, M.D.
role: PRINCIPAL_INVESTIGATOR
References:
citation: Croffie JM, Davis MM, Faught PR, Corkins MR, Gupta SK, Pfefferkorn MD, Molleston JP, Fitzgerald JF. At what age is a suction rectal biopsy less likely to provide adequate tissue for identification of ganglion cells? J Pediatr Gastroenterol Nutr. 2007 Feb;44(2):198-202. doi: 10.1097/01.mpg.0000252188.12793.ee.
citation: Hurlstone DP, Cross SS, Drew K, Adam I, Shorthouse AJ, Brown S, Sanders DS, Lobo AJ. An evaluation of colorectal endoscopic mucosal resection using high-magnification chromoscopic colonoscopy: a prospective study of 1000 colonoscopies. Endoscopy. 2004 Jun;36(6):491-8. doi: 10.1055/s-2004-814397.
citation: Hirose R, Hirata Y, Yamada T, Kawana T, Taguchi T, Suita S. The simple technique of rectal mucosal biopsy for the diagnosis of Hirschsprung's disease. J Pediatr Surg. 1993 Jul;28(7):942-4. doi: 10.1016/0022-3468(93)90703-n.
citation: Pini-Prato A, Martucciello G, Jasonni V. Rectal suction biopsy in the diagnosis of intestinal dysganglionoses: 5-year experience with Solo-RBT in 389 patients. J Pediatr Surg. 2006 Jun;41(6):1043-8. doi: 10.1016/j.jpedsurg.2006.01.070.