Agreement between study designs: a systematic review comparing observational studies and randomized trials of surgical treatments for necrotizing enterocolitis

Heesewijk, Anne E. van; Rush, Margaret; Schmidt, Barbara; Kirpalani, Haresh; DeMauro, Sara B.

doi:10.1080/14767058.2018.1533948

Cited by 10 publications

(6 citation statements)

References 42 publications

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“…They also proposed the concept of the efficiency-effectiveness gap to describe the gap between treatment effects observed in RCTs and those observed in RWS. However, other studies ( 13 - 18 ) have shown that most RCTs in the same disease and treatment methods have very similar results to RWS. As the design and reporting quality of RWS improve, respectively, the consistency with the results of RCTs becomes higher.…”

Section: Introductionmentioning

confidence: 91%

Is there an efficacy-effectiveness gap between randomized controlled trials and real-world studies in colorectal cancer: a systematic review and meta-analysis

Zhang¹,

Fu²,

Meng³

et al. 2020

Transl Cancer Res TCR

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Background: To investigate whether patients with colorectal cancer (CRC) enrolled in randomized controlled trials (RCTs) and real-world studies (RWS) differ in terms of baseline characteristics, leading to an efficacy-effectiveness gap.Methods: A systematic literature reviews was conducted to identify RCTs and RWS with CRC, treated with bevacizumab (BEV), cetuximab (CET) or oxaliplatin combined with capecitabine (XELOX). Using random-effects meta-analyses compared the baseline characteristics and treatment effects of RCTs and RWS, overall and by drug. Correlation between treatment effects and baseline characteristics and study types were estimated using meta-regression analyses.Results: Two hundred and fifty-three studies were included. Compared with patients enrolled in RWS, the proportion of male patients in RCTs was 0.032 higher (P=0.004), the proportion of patients with Eastern Cooperative Oncology Group (ECOG) performance ≥2 was 0.085 less (P<0.001). No significant differences in treatment effects [progression-free survival (PFS), overall survival (OS), objective response rate (ORR), disease control rate (DCR)] were found by overall analysis. But the OS of patients in RCTs was 4.184 higher (P=0.023) in the CET group. Meta-regression results showed that OS difference in the CET group was related to the difference in treatment lines, not related to other baseline characteristics and study types. Conclusions:No efficacy-effectiveness gap was found in CRC between RCTs and RWS. CRC treatment effects Between RCTs and RWS had high consistency.

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Section: Introductionmentioning

confidence: 91%

Is there an efficacy-effectiveness gap between randomized controlled trials and real-world studies in colorectal cancer: a systematic review and meta-analysis

Zhang¹,

Fu²,

Meng³

et al. 2020

Transl Cancer Res TCR

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“…The standard surgical approach is laparotomy for excision of necrotic bowel, but its challenges in critically ill infants prompt discussion about peritoneal drain placement. A recent systematic review concluded that neither RCTs nor observational studies with high quality of reporting showed a difference in mortality when comparing laparotomy versus peritoneal drainage (pooled OR 0.85, 95% CI 0.47-1.54 and pooled OR 0.67, 95% CI 0.37-1.19, respectively) [92]. In the Necrotizing Enterocolitis Surgery Trial (NEST), the authors concluded that with a preoperative diagnosis of NEC, initial laparotomy is more likely than peritoneal drainage to reduce death or NDI, with a Bayesian posterior probability of 97%.…”

Section: Management Of Surgical Necmentioning

confidence: 99%

Prevention Strategies and Management of Necrotizing Enterocolitis

Colarelli,

MD,

Barbian

et al. 2024

Curr Treat Options Peds

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Necrotizing enterocolitis (NEC) is a serious intestinal disease which primarily affects preterm infants. The pathogenesis of NEC is multifactorial. Thus, it is complicated to study, prevent, and manage. Purpose of Review The purpose of this review is to provide a comprehensive summary of recent research and provide recommendations for the prevention and management of NEC. Currently, management is supportive and non-specific and long-term outcomes for surgical NEC are poor. Recent Findings The most important strategy to prevent NEC is to provide preterm infants with a human milk diet, minimize exposure to antibiotics and avoid medications that disturb the intestinal microbiome. Summary Strategies to optimize the infant’s intestinal microbiome are critical, as disturbances in the intestinal microbiome composition are a major factor in the pathogenesis of this disease. Optimizing maternal health is also vital to prevent prematurity and neonatal morbidity. Ongoing research holds promise for the implementation of new diagnostic modalities, preventive strategies, and medical treatment options to improve outcomes for premature infants.

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“…The only previous meta-analysis to include a large number of observational studies is the one published by Van Heesewijk et al [5], which compares the two surgical approaches and evaluated the mortality rates for two RCTs and 25 observational studies published between 1994 and 2016.…”

Section: Previous Systematic Reviews and Important Differences From O...mentioning

confidence: 99%

“…Peritoneal drainage (PD), which involves insertion of a Penrose drain into the peritoneum at the bedside, was first reported as a treatment for necrotizing enterocolitis and spontaneous intestinal perforation (SIP) in 1977 by Ein et al [4], in a case series of five patients who were unstable and would not tolerate laparotomy (LAP). PD aims to decompress the abdomen and remove peritoneal toxic effluents without requiring open surgery, and has established itself as an alternative to LAP, sometimes in hemodynamically unstable infants as a temporizing procedure, but also as a less aggressive and sometimes definitive first line treatment [5].…”

Section: Introductionmentioning

confidence: 99%

“…To date, given the overall high mortality [6] and poor neurodevelopmental outcomes [7,8] associated with surgical NEC (sNEC), the first-line surgical treatment approach for sNEC and SIP remains controversial. Previous meta-analysis [9,10] have analyzed data from two published randomized controlled trials (RCTs) [11,12] and numerous observational studies [5] that have compared LAP to PD as primary surgical treatment for NEC and/or SIP, analyzing differences in mortality and other outcomes. Over the last 10 years, however, new evidence, including the largest RCT so far [13] and multiple observational studies have been published, providing new data that might help us answer the question of which is the most appropriate surgical option for preterm neonates with sNEC or SIP.…”

Section: Introductionmentioning

confidence: 99%

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Laparotomy versus Peritoneal Drainage as Primary Treatment for Surgical Necrotizing Enterocolitis or Spontaneous Intestinal Perforation in Preterm Neonates: A Systematic Review and Meta-Analysis

2023

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Aim: to systematically review and meta-analyze the impact on morbidity and mortality of peritoneal drainage (PD) compared to laparotomy (LAP) in preterm neonates with surgical NEC (sNEC) or spontaneous intestinal perforation (SIP). Methods: Medical databases were searched until June 2022 for studies comparing PD and LAP as primary surgical treatment of preterm neonates with sNEC or SIP. The primary outcome was survival during hospitalization; predefined secondary outcomes included need for parenteral nutrition at 90 days, time to reach full enteral feeds, need for subsequent laparotomy, duration of hospitalization and complications. Results: Three RCTs (N = 493) and 49 observational studies (N = 19,447) were included. No differences were found in the primary outcome for RCTs, but pooled observational data showed that, compared to LAP, infants with sNEC/SIP who underwent PD had lower survival [48 studies; N = 19,416; RR 0.85; 95% CI 0.79–0.90; GRADE: low]. Observational studies also showed that the subgroup of infants with sNEC had increased survival in the LAP group (30 studies; N = 9370; RR = 0.82; 95% CI 0.72–0.91; GRADE: low). Conclusions: Compared to LAP, PD as primary surgical treatment for sNEC or SIP has similar survival rates when analyzing data from RCTs. PD was associated with lower survival rates in observational studies.

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Agreement between study designs: a systematic review comparing observational studies and randomized trials of surgical treatments for necrotizing enterocolitis

Cited by 10 publications

References 42 publications

Is there an efficacy-effectiveness gap between randomized controlled trials and real-world studies in colorectal cancer: a systematic review and meta-analysis

Is there an efficacy-effectiveness gap between randomized controlled trials and real-world studies in colorectal cancer: a systematic review and meta-analysis

Prevention Strategies and Management of Necrotizing Enterocolitis

Laparotomy versus Peritoneal Drainage as Primary Treatment for Surgical Necrotizing Enterocolitis or Spontaneous Intestinal Perforation in Preterm Neonates: A Systematic Review and Meta-Analysis

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