Clinical trial NCT01223261: Observational Study of Surgical Treatment of Necrotizing Enterocolotis
Official title: Observational Study of Surgical Treatment of Necrotizing Enterocolotis or Isolated Intestinal Perforation
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Infant, Newborn
Infant, Low Birth Weight
Infant, Small for Gestational Age
Infant, Premature
Enterocolitis, Necrotizing
Intestinal Perforation
Keywords: NICHD Neonatal Research Network
Very Low Birth Weight (VLBW)
Extremely Low Birth Weight (ELBW)
Prematurity
Laparotomy
Drainage
Isolated intestinal perforation
Focal intestinal performation
Lead sponsor: NICHD Neonatal Research Network
Sponsor class: NETWORK
Collaborators: name: National Center for Research Resources (NCRR)
First posted: 2010-10-18
Enrollment: 156
Sex: ALL
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Extremely Low Birth Weight (ELBW) infants (those with birth weights \<1000 g) born at participating NRN Centers diagnosed by a pediatric surgeon and neonatologist as having either necrotizing enterocolitis (NEC) or intestinal perforation (IP) requiring surgical intervention.

Brief summary: The purposes of this study were: 1) to compare mortality and postoperative morbidities in extremely low birth weight (ELBW) infants who underwent initial laparotomy or drainage for necrotizing enterocolitis (NEC) or isolated intestinal perforation (IP); 2) to determine the ability to distinguish NEC from IP preoperatively and the importance of this distinction on outcome measures; and 3) to evaluate the association between extent of intestinal disease determined at operation and outcome measures. All ELBW infants born at participating NRN centers were screened for the presence of NEC or IP that was thought by the pediatric surgeon and neonatologist to require surgical intervention. Data were collected enrolled infants, including: intraoperative findings recorded by the surgeon and specific post-operative complications. Neurodevelopmental examinations were conducted on surviving infants at 18-22 months corrected age.

Detailed description: Necrotizing enterocolitis (NEC) is a condition, generally affecting premature infants, in which the intestines become ischemic (lack oxygen and/or blood flow). NEC occurs in up to 5-15% of extremely low birth weight (ELBW) infants. Isolated or focal intestinal perforation (IP) is a less common condition, affecting an estimated 4% of ELBWs, in which a hole develops in the intestines leaking fluid into the abdomin. The outcomes for infants with NEC or IP are poor: 49% die and half of the surviving infants are neurodevelopmentally impaired.

Surgical options for NEC and IP include two possible procedures: peritoneal drainage, in which a tube is placed in the abdominal cavity through a small incision for fluid to drain out; or laparotomy, in which an incision is made in the abdomen and diseased intestine is removed. Infants treated with an initial drainage sometimes go on to need a laparotomy. Most surgeons now believe that a diagnosis of the intestinal perforation (IP) may actually be either true NEC or a different and distinct pathology, termed isolated intestinal perforation. The ability to distinguish these 2 conditions preoperatively, based on perinatal characteristics, physical examination findings, and findings on abdominal plain film imaging, remains unknown. If these 2 entities can be distinguished preoperatively, the intervention chosen and outcomes may be different. From the two available surgical options, tt is not known whether initial laparotomy or peritoneal drain placement is more effective for either NEC or IP.

This study was a prospective, multicenter observational study to describe the surgical outcomes (mortality, post-operative intestinal stricture, intra-abdominal abscess formation, etc.) in ELBW infants with either NEC or IP who underwent initial laparotomy or peritoneal drainage. We also evaluated the ability of surgeons to distinguish NEC and IP pre-operatively and the relevance of this distinction on outcome. Finally, an analysis of the impact of extent of intestinal involvement with NEC on outcome measures is reported.

All ELBW infants born at participating NRN centers were screened for the presence of NEC or IP that was thought by the pediatric surgeon and neonatologist to require surgical intervention. Data were collected enrolled infants, including: intraoperative findings recorded by the surgeon and specific post-operative complications.

Neurodevelopmental examinations were conducted on surviving infants at 18-22 months corrected age.

Eligibility criteria: Inclusion Criteria:

* Infants born 401-1,000 grams at birth enrolled in the NRN Generic Database
* Sage III NEC or isolated intestinal perforation
* Pediatric surgeon decision to perform surgery for suspected NEC or IP

Exclusion Criteria:

* Decision not to treat

Primary outcomes:
measure: Feasibility of conducting a randomized trial
timeFrame: 1 year
Secondary outcomes:
measure: Document variation in current surgical practices
timeFrame: Until hospital discharge or 120 days of life
measure: Prevalence of infants who would qualify for the study
timeFrame: Until hospital discharge or 120 days of life
measure: Frequency of postoperative complications
timeFrame: Until hospital discharge or 120 days of life
measure: Neurodevelopmental impairment
timeFrame: 18-22 months corrected age
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: San Diego
country: United States
facility: University of California at San Diego
state: California
city: New Haven
country: United States
facility: Yale University
state: Connecticut
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Indianapolis
country: United States
facility: Indiana University
state: Indiana
city: Detroit
country: United States
facility: Wayne State University
state: Michigan
city: Rochester
country: United States
facility: University of Rochester
state: New York
city: Charlotte
country: United States
facility: Wake Forest University
state: North Carolina
city: Durham
country: United States
facility: RTI International
state: North Carolina
city: Durham
country: United States
facility: Duke University
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Medical Center
state: Ohio
city: Cleveland
country: United States
facility: Case Western Reserve University, Rainbow Babies and Children's Hospital
state: Ohio
city: Providence
country: United States
facility: Brown University, Women & Infants Hospital of Rhode Island
state: Rhode Island
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center at Dallas
state: Texas
city: Houston
country: United States
facility: University of Texas Health Science Center at Houston
state: Texas
Study officials:
affiliation: The University of Texas Health Science Center, Houston
name: Martin L. Blakely, MD
role: STUDY_DIRECTOR
affiliation: University of Alabama at Birmingham
name: Waldemar A. Carlo, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Brown University, Women & Infants Hospital of Rhode Island
name: William Oh, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Case Western Reserve University, Rainbow Babies & Children's Hospital
name: Avroy A. Fanaroff, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Children's Hospital Medical Center, Cincinnati
name: Edward F. Donovan, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Emory University
name: Barbara J. Stoll, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Miami
name: Charles R. Bauer, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Indiana University
name: James A. Lemons, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Stanford University
name: David K. Stevenson, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Texas
name: Abbot R. Laptook, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Wayne State University
name: Seetha Shankaran, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Yale University
name: Richard A. Ehrenkranz, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Duke University
name: Ronald N. Goldberg, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Wake Forest University
name: T. Michael O'Shea, MD MPH
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Rochester
name: Dale L. Phelps, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of California, San Diego
name: Neil N. Finer, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: RTI International
name: W. Kenneth Poole, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Blakely ML, Lally KP, McDonald S, Brown RL, Barnhart DC, Ricketts RR, Thompson WR, Scherer LR, Klein MD, Letton RW, Chwals WJ, Touloukian RJ, Kurkchubasche AG, Skinner MA, Moss RL, Hilfiker ML; NEC Subcommittee of the NICHD Neonatal Research Network. Postoperative outcomes of extremely low birth-weight infants with necrotizing enterocolitis or isolated intestinal perforation: a prospective cohort study by the NICHD Neonatal Research Network. Ann Surg. 2005 Jun;241(6):984-9; discussion 989-94. doi: 10.1097/01.sla.0000164181.67862.7f.
citation: Blakely ML, Tyson JE, Lally KP, McDonald S, Stoll BJ, Stevenson DK, Poole WK, Jobe AH, Wright LL, Higgins RD; NICHD Neonatal Research Network. Laparotomy versus peritoneal drainage for necrotizing enterocolitis or isolated intestinal perforation in extremely low birth weight infants: outcomes through 18 months adjusted age. Pediatrics. 2006 Apr;117(4):e680-7. doi: 10.1542/peds.2005-1273. Epub 2006 Mar 20.