Keywords
Hirschsprung's disease - outcome - characteristics
Introduction
Hirschsprung's disease (HSCR) is a rare congenital condition that affects 1 in 5,000
newborn children. It is characterized by the absence of ganglion cells in the enteric
nervous system of the rectum, with variable involvement of the colon and small bowel.
Current understanding suggests that this abnormality arises from disruptions in embryonic
development stages, particularly the migration and survival of enteric nervous system
precursor cells.[1]
[2]
Infants with HSCR present with symptoms of functional intestinal obstruction, such
as delayed passage of meconium, abdominal distention, vomiting, and chronic constipation.
Currently, establishing the diagnosis predominantly relies on rectal suction biopsies
revealing an absence of ganglion cells. Aimed at re-establishing regular bowel functionality,
the treatment generally involves resection of the aganglionic colon segment and reconnecting
the ganglionic intestine segment to the anus. Surgical strategies include the Swenson,[3] Duhamel,[4] and Soave–Boley[5]
[6] resection techniques, fashioned as single- or multi-stage, open, laparoscopic, or
total transanal procedures.[1]
[7]
Due to the rarity of HSCR, prospective, controlled, multicenter trials with adequate
patient numbers are notably scarce. Furthermore, despite the increase in HSCR-related
publications in recent decades, which could potentially contribute to more evidence-based
practice, the usability of these research findings is limited. This limitation stems
from the substantial variability in the reported research parameters and their respective
definitions, preventing the possibility of comprehensive comparisons. Although a HSCR
core outcome set has been formulated, this has not yet attained widespread recognition
or utilization within the scholarly community.[7]
[8] These circumstances further prevent the resolution of existing evidence gaps, particularly
in understanding short-term complications and long-term interactions between functional
outcomes and quality of life.[9] Presently, guidelines for the management of HSCR often rely on expert opinion or
consensus rather than comprehensive research, which implies that there is substantial
room for improvement in the evidence-based practice.
The lack of standardized, well-defined parameters not only hinders overarching comparisons
in research but also adds complexity to the evaluation and comparison of (quality
of) HSCR care across hospitals, regions, or even countries. A pivotal step in advancing
HSCR care has been the initiation of the European Pediatric Surgical Audit (EPSA)
for several congenital malformations, including HSCR. This quality-of-care measurement
tool for clinicians makes use of quality indicators to reliably identify, monitor,
and evaluate variation in clinical practices and outcomes.[10]
[11]
[12] Improvement efforts could then be aligned with the findings from this evaluation.
Indicators can be classified into three categories—structure indicators, process indicators,
and outcome indicators—each evaluating different aspects of the care pathway. Examples
of structure indicators are patient volumes and the availability of certain imaging
techniques. Process indicators can involve factors such as the elapsed time between
diagnosis and surgery, and the proportion of patients undergoing a particular diagnostic
test. Outcome indicators might encompass elements such as survival rates or complication
rates.[13] To develop a universally acknowledged set of quality indicators for evaluating and
identifying variations in the quality of care for HSCR patients, it is essential to
ascertain which parameters are considered most important by HSCR researchers and clinicians
treating this disease. These identified outcomes can then serve as the basis for a
consensus-driven process to delineate the new set of quality indicators. This initiative
requires comprehensive understanding of all possible outcomes highlighted in HSCR
research, as well as patient characteristics and treatment and care process characteristics,
to adjust for case variability when comparing care quality. Such endeavors could provide
an even deeper understanding of inter-hospital variability, further supporting future
advancements in the care of this patient population.
The primary objective of this study was to compile a list of outcome measures described
in recent peer-reviewed publications on HSCR and to explore the discrepancies in their
utilization, definition, and reporting. The secondary objective was to identify patient
characteristics and treatment and care process characteristics that would facilitate
a more informed interpretation of future comparative analysis results.
Materials and Methods
This review was performed according to the Preferred Reporting Items for Systematic
Reviews and Meta-analysis (PRISMA) statement and guidelines.[14] We developed a comprehensive search strategy in collaboration with a medical librarian
of the Erasmus University Medical Center. The search was based on the search term
“Hirschsprung's disease,” combined with the terms “morbidity” or “mortality” or “survival”
or “outcome” or “complication.” The search criteria were applied to the databases
Medline, Embase, and the Cochrane Library. Duplicate publications were removed and
subsequently all studies published from 2015 up to and September 2021 were selected.
Complete search strategies are provided in [Supplementary Material 1] (available in the online version only). The search was performed in October 2021.
Inclusion and Exclusion Criteria
All publications related to any aspect of the main HSCR care process, including surgical
and nonsurgical management, were included. To ensure contemporaneousness, articles
published before the year 2015 were excluded. Other criteria for exclusion were the
following: non-English language publications, animal research, in vitro studies, case
series with fewer than 10 patients, editorials, letters, meeting abstracts, reviews,
guidelines, and consensus statements.
Selection Process
Four reviewers participated in the selection process of the publications. N.T. and
A.L.G. separately screened the titles and abstracts of all publications resulting
from the search to determine their suitability in terms of reporting on the care for
and management of patients with HSCR. N.T., D.R., and A.L.G. then independently reviewed
the full texts of the selected publications to ascertain their relevance to the study.
Any disagreements were resolved by T.W., who served as a third reviewer. The reviewing
authors were not blinded to the title, authors, or journal names during the screening
process.
Data Collection, Analysis, and Reporting
D.R. and N.T. performed the data extraction, which included recording the origin and
year of publications, study design, the number of included patients per study (as
shown in [Table 1]), and all relevant parameters in a primary Excel-based framework. Parameters were
categorized and, when feasible, merged based on consensus among the reviewers. For
each parameter, the number of publications in which it was mentioned and the proportion
relative to the total number of included publications were calculated. The parameters
mentioned in more than 5% of the included publications were then recorded in a separate
Excel file. Additionally, all definitions of studied parameters were extracted, as
well as the types of instruments or tools, either standardized or not, with which
the parameters were assessed, such as medical scores, scales, and questionnaires.
This review did not include the extraction or interpretation of parameter estimates,
nor did it involve the evaluation and assessment of methodological quality of the
included publications. Statistical analyses were conducted utilizing a customized
data computational approach implemented within the Excel environment.
Table 1
Study characteristics of included publications
|
|
n
|
%
|
|
Originated in
|
Africa
|
6
|
3
|
|
Asia
|
99
|
49
|
|
Europe
|
62
|
31
|
|
Eurasia
|
2
|
1
|
|
North America
|
23
|
12
|
|
Oceania
|
5
|
3
|
|
South America
|
2
|
1
|
|
Intercontinental
|
1
|
1
|
|
Study timing
|
Retrospective
|
130
|
65
|
|
Prospective
|
30
|
15
|
|
Cross-sectional
|
39
|
19
|
|
Mixed design
|
1
|
1
|
|
Type of study
|
Observational
|
120
|
60
|
|
Comparative
|
80
|
40
|
|
Study design
|
Cohort
|
179
|
90
|
|
Case series
|
6
|
3
|
|
Case–control
|
7
|
3
|
|
Trial
|
8
|
4
|
|
Study scale
|
Single-center
|
150
|
75
|
|
Multicenter
|
33
|
17
|
|
National
|
17
|
8
|
|
Year of publication
|
2015
|
16
|
8
|
|
2016
|
20
|
10
|
|
2017
|
27
|
13
|
|
2018
|
27
|
13
|
|
2019
|
33
|
17
|
|
2020
|
38
|
19
|
|
2021
|
39
|
20
|
|
Number of included patients
|
< 25
|
36
|
18
|
|
25–100
|
102
|
51
|
|
101–300
|
34
|
17
|
|
301–1,000
|
17
|
9
|
|
> 1,000
|
11
|
5
|
Results
Included Publications
The search strategy resulted in 1,254 publications. After removal of duplicates and
limiting the results to articles published between 2015 and 2021, a total of 828 articles
remained, of which 200 met the inclusion criteria ([Fig. 1]). The study characteristics of all included publications are summarized in [Table 1]. An individualized overview of included studies and corresponding study characteristics
can be found in [Supplementary Material 2] (available in the online version only).
Fig. 1 PRISMA flowchart: systematic review of studied parameters in Hirschsprung's disease
research.
Data Extraction
Full-text analysis of the 200 included publications identified 1,026 studied parameters
described in at least one of the included publications. The parameters were categorized
into patient characteristics (n = 226), treatment and care process characteristics (n = 199), and outcomes (n = 601). As several parameters could arguably be included in more than one category,
categorization followed agreement between the first two authors. To facilitate the
comparison of variation in studied parameters within similar subjects, we further
arranged the parameters by topic, such as comorbidities, primary treatment, specific
complications, and long-term outcome. A list of all identified, categorized items
is attached in [Supplementary Materials 3] to [5] (available in the online version only).
Measured Parameters
Of the 1,026 identified studied parameters, 116 were described in at least 5% of included
publications ([Table 2]). Predominantly described patient characteristics were sex (88%), age at the time
of surgery (66%), and the extent of aganglionosis (histological level of transitional
zone, 50.5%). Frequently studied treatment and care process characteristics included
the type of repair (56.5%), duration of follow-up (39%), and preoperative stoma (35.5%).
The most frequently mentioned outcome measures were postoperative Hirschsprung-associated
enterocolitis (HAEC) (63.5%), incontinence (53.5%), and constipation (48%). The primarily
described complications after surgical treatment were anastomotic stricture and anastomotic
leakage, cited in 38% and 28% of the publications, respectively. Other significant
areas of focus encompassed length of primary hospital stay (highlighted in 35.5% of
the studies), mortality (26.5%), reoperation rate (42.5%), and readmission rate (12.5%).
Because the definitions of analyzed parameters and the used (standardized) measurement
tools diverged significantly across different publications, it was challenging to
derive clear-cut definitions or compare these results. Consequently, these results
specifically concerning the definitions of the extracted parameters are not delineated
in this report. An overview of the standardized clinical tools, scores, and scales
applied in the studies is presented in [Table 3].
Table 2
Identified studied parameters in HSCR research appearing in > 5% of the included articles
|
|
n
|
%
|
|
Baseline characteristics
|
Sex
|
176
|
88
|
|
Age at surgery
|
132
|
66
|
|
Extent of aganglionosis[a]
|
101
|
51
|
|
Syndromal, genetic, and chromosomal disorders
|
80
|
40
|
|
(of which) Trisomy 21
|
65
|
33
|
|
Age at follow-up
|
71
|
36
|
|
Associated anomaly (cumulative)
|
58
|
29
|
|
Initial clinical symptoms (cumulative)
|
55
|
28
|
|
Gestational age or prematurity
|
45
|
23
|
|
Enterocolitis (preoperative)
|
45
|
23
|
|
Birth weight
|
42
|
21
|
|
Cardiovascular anomalies
|
40
|
20
|
|
Weight at surgery
|
37
|
19
|
|
Age at diagnosis
|
34
|
17
|
|
Family history of HSCR[b]
|
28
|
14
|
|
Delay in evacuation/passage of meconium
|
25
|
13
|
|
Congenital heart disease
|
25
|
13
|
|
Renal/genitourinary anomalies
|
23
|
12
|
|
Abdominal distention (preoperative)
|
19
|
10
|
|
Constipation (preoperative)
|
19
|
10
|
|
Neurologic anomalies
|
19
|
10
|
|
Gastrointestinal anomalyc
|
19
|
10
|
|
Ethnicity
|
18
|
9
|
|
Vomiting (preoperative)
|
17
|
9
|
|
Age at presenting symptoms
|
15
|
8
|
|
Poor feeding/malnutrition/failure to thrive (preoperative)
|
14
|
7
|
|
Congenital anomalies of the urogenital system
|
13
|
7
|
|
Pulmonary/respiratory anomalies
|
12
|
6
|
|
Intestinal perforation (preoperative)
|
10
|
5
|
|
Congenital anomalies of the nervous system
|
10
|
5
|
|
Diagnostics, treatment, and care process
|
Type of repair
|
113
|
57
|
|
Duration follow-up
|
78
|
39
|
|
Preoperative/primary stoma
|
71
|
36
|
|
Operative time (pull-through)
|
46
|
23
|
|
Blood loss (pull-through)
|
41
|
21
|
|
Laparoscopy or laparotomy or transanal approach
|
40
|
20
|
|
Length of resected aganglionic and dilated segments
|
40
|
20
|
|
Anal dilatations (preoperative)
|
33
|
17
|
|
Contrast enema (preoperative)
|
27
|
14
|
|
Rectal biopsies (preoperative)
|
26
|
13
|
|
Follow-up rate
|
21
|
11
|
|
Preoperative/primary colostomy
|
20
|
10
|
|
Single- or multiple staged procedure
|
19
|
10
|
|
Daily preoperative colon irrigations (mechanical bowel preparation)
|
18
|
9
|
|
Intraoperative biopsies at the time of pull-through
|
18
|
9
|
|
Intraoperative complications (pull-through)
|
18
|
9
|
|
Preoperative/primary ileostomy
|
16
|
8
|
|
Conversion laparoscopy to laparotomy (pull-through)
|
14
|
7
|
|
ACEd
|
14
|
7
|
|
Anorectal manometry (preoperative)
|
13
|
7
|
|
Postoperative anal dilatation under general anesthesia
|
12
|
6
|
|
Anorectal manometry: follow-up
|
12
|
6
|
|
Time to start oral feeding
|
11
|
6
|
|
Outcome
|
Postoperative HAEC
|
127
|
64
|
|
Fecal incontinence
|
107
|
54
|
|
Constipation
|
96
|
48
|
|
(Any) reoperation
|
85
|
43
|
|
Anastomotic stricture/stenosis
|
76
|
38
|
|
Soiling
|
72
|
36
|
|
Length of primary hospital stay
|
71
|
36
|
|
Anastomotic leakage
|
56
|
28
|
|
Bowel function (standardized scoring)
|
54
|
27
|
|
Mortality
|
53
|
27
|
|
Defecation frequency
|
52
|
26
|
|
Intestinal obstruction
|
49
|
25
|
|
Gastrointestinal symptoms during follow-up
|
40
|
20
|
|
Perianal issues (cumulative)
|
38
|
19
|
|
Normal defecation function
|
37
|
19
|
|
Use of bowel regulating medication
|
32
|
16
|
|
Growth/weight/failure to thrive during follow-up
|
32
|
16
|
|
Wound issues (cumulative)
|
32
|
16
|
|
Permanent stoma
|
31
|
16
|
|
Botulinum toxin
|
31
|
16
|
|
Quality of life
|
28
|
14
|
|
Complications (mentioned as such)
|
28
|
14
|
|
Urinary function
|
27
|
14
|
|
Wound infection
|
27
|
14
|
|
Early postoperative complications (mentioned as such)
|
26
|
13
|
|
Readmission
|
25
|
13
|
|
Anal excoriations
|
24
|
12
|
|
Residual aganglionosis
|
24
|
12
|
|
Stoma at the time of follow-up
|
21
|
11
|
|
Bowel function score, Rintala et al[15]
|
20
|
10
|
|
Ileus/bowel obstruction due to adhesions
|
20
|
10
|
|
Abdominal distension
|
18
|
9
|
|
Difficulties with socializing/social adaptation/relationships
|
18
|
9
|
|
Cause of death
|
17
|
9
|
|
Fecal contamination/fecal accidents
|
17
|
9
|
|
HAEC treated by conservative management
|
16
|
8
|
|
Constipation manageable with laxatives
|
15
|
8
|
|
Form/consistency of stool
|
15
|
8
|
|
Rectal prolapse
|
15
|
8
|
|
Stoma complications (cumulative)
|
15
|
8
|
|
Constipation manageable with enema(s)
|
14
|
7
|
|
Multiple episodes of enterocolitis
|
14
|
7
|
|
HAEC recurrence
|
14
|
7
|
|
Weight at the time of follow-up
|
13
|
7
|
|
Feeling the need for defecation
|
13
|
7
|
|
Ability to hold back defecation
|
13
|
7
|
|
Use of enemas
|
13
|
7
|
|
Use of laxatives
|
13
|
7
|
|
Sepsis
|
13
|
7
|
|
Incidence of HAEC
|
13
|
7
|
|
Diarrhea
|
12
|
6
|
|
Constipation manageable with diet
|
12
|
6
|
|
Recurrent constipation
|
12
|
6
|
|
Wound dehiscence
|
11
|
6
|
|
Fever
|
10
|
5
|
|
Problems with urinary incontinence
|
10
|
5
|
|
Reoperation type
|
10
|
5
|
|
Reoperation due to complications (cumulative, no timeframe)
|
10
|
5
|
|
Reoperation due to obstruction/occlusion
|
10
|
5
|
|
(Any) reoperation due to residual aganglionosis
|
10
|
5
|
|
Abscess
|
10
|
5
|
|
Infection (cumulative)
|
10
|
5
|
|
Incomplete/transitional zone pull-through
|
10
|
5
|
a Extent of aganglionosis refers to the histological level of transitional zone, namely,
rectal, sigmoid, rectosigmoid, distal descending colon.
b Gastrointestinal anomalies: excluding Hirschsprung's disease.
Abbreviations: ACE, antegrade colonic enema; HAEC, Hirschsprung-associated enterocolitis;
HSCR, Hirschsprung's disease.
Table 3
Identified tools/instruments utilized and reported in one or more included publications
|
|
N
|
%
|
|
Incontinence scores
|
Wingspread criteria
|
4
|
2
|
|
Miller incontinence score
|
2
|
1
|
|
Baylor continence scale
|
2
|
1
|
|
Postoperative fecal incontinence score
|
1
|
< 1
|
|
Jorge–Wexner score
|
1
|
< 1
|
|
Visick score
|
1
|
< 1
|
|
Continence evaluation score
|
1
|
< 1
|
|
Scores for bowel function
|
Bowel function score (Rintala et al)[15]
|
20
|
10
|
|
Bowel function score according to Holschneider
|
7
|
4
|
|
Krickenberg classification system
|
6
|
3
|
|
Pediatric incontinence and constipation scoring system
|
3
|
2
|
|
Stooling survey (El Sawaf et al[16])
|
3
|
2
|
|
Evacuation score of the Japan Society of ARM Study group
|
2
|
1
|
|
Vancouver dysfunctional elimination syndrome survey (VDESS)
|
2
|
1
|
|
Bowel function score according to Holschneider, adapted by Lindahl et al[17]
|
1
|
1
|
|
Bowel habit diary
|
1
|
< 1
|
|
Kelly scale score for recovery of bowel function
|
1
|
< 1
|
|
Postoperative bowel function score
|
1
|
< 1
|
|
HSCR anal function criteria (proposed by Reding[18])
|
1
|
< 1
|
|
Wingspread score
|
1
|
< 1
|
|
Rome IV criteria
|
1
|
< 1
|
|
Motor- and neurologic development and behavior
|
Intelligence (RAKIT[19] | WISC-III-NL)
|
1
|
< 1
|
|
Memory (WISC-III)
|
1
|
< 1
|
|
Attention (Dot cancellation test)
|
1
|
< 1
|
|
Self-esteem (SPPC)
|
1
|
< 1
|
|
WISC IV
|
1
|
< 1
|
|
NEPSY
|
1
|
< 1
|
|
KIDSCREEN 10
|
1
|
< 1
|
|
Sexual function
|
International index of erectile function
|
1
|
< 1
|
|
Female sexual functioning index
|
1
|
< 1
|
|
Female sexual distress scale
|
1
|
< 1
|
|
Sexual education questionnaire
|
1
|
< 1
|
|
Normal erectile functioning (erectile hardness score)
|
1
|
< 1
|
|
Hirschsprung's associated enterocolitis scores
|
HAEC score (by Pastor et al[20])
|
5
|
3
|
|
Grading system by Elhalaby et al[21]
|
3
|
2
|
|
Grading system by Murphy et al
|
1
|
< 1
|
|
Quality of life
|
Pediatric Quality of Life Inventory (PedsQL)
|
8
|
4
|
|
Short Form 36 (SF-36)
|
5
|
3
|
|
HSCR and ARM QoL scale (HAQL)
|
4
|
2
|
|
Gastrointestinal Quality of Life Index (GIQLI)
|
4
|
2
|
|
PedsQL Family Impact Module
|
2
|
1
|
|
Fecal incontinence and constipation quality of life
|
2
|
1
|
|
Child Health Questionnaire-Child Form 87 items (CHQ-CF87)
|
2
|
1
|
|
World Health Organization Quality of Life 100 (WHOQOL-100)
|
2
|
1
|
|
PedsQL Generic Core Scale
|
1
|
< 1
|
|
PedsQL General Wellbeing Score
|
1
|
< 1
|
|
WHO QOL-BREF
|
1
|
< 1
|
|
Assessment of QoL in children and adolescents with fecal incontinence (AQLCAFI)
|
1
|
< 1
|
|
Child Health Questionnaire - Parent Form 50 (CHQ-PF50)
|
1
|
< 1
|
|
Scoring system by Bai et al[22] in patients with HD
|
1
|
< 1
|
|
Barrena score
|
1
|
< 1
|
|
Taiwanese Child and Caregiver Quality of Life (TCCQOL)
|
1
|
< 1
|
|
Hirschsprung's Disease/Anorectal Malformation Quality of life Questionnaire (HAQL)
|
1
|
< 1
|
|
KIDSCREEN 52
|
1
|
< 1
|
|
Skin and subcutaneous
|
Manchester scar scale
|
3
|
2
|
a Multiple instruments or tools may have been used in one publication.
Discussion
This study aimed to create a comprehensive overview of patient characteristics, treatment
and care process characteristics, and outcome measures. To our knowledge, it represents
the first attempt to compile such an extensive summary of parameters investigated
in HSCR research. From the 200 publications on HSCR spanning from 2015 to 2021 that
were included, we identified a total of 1,026 unique studied parameters, which could
be categorized into 226 patient characteristics, 199 treatment and care process characteristics,
and 601 outcomes. Among these, 116 parameters were described in at least 5% of the
included publications, of which only 6 were mentioned in more than 50% of the included
publications. These six parameters were postoperative HAEC, incontinence, type of
repair, sex, age at the time of surgery, and extent of aganglionosis.
The current body of research of interest for this review primarily consists of retrospective,
single-center studies with low patient volumes.[23] A substantial portion of the surveyed literature (69%) is based on data derived
from cohorts comprising fewer than 100 patients, and more than three-quarters of the
studies are single-center studies. This underscores the need for more multicenter
and prospective studies involving larger patient cohorts to yield more robust results.
Such approaches may include randomized controlled trials or the implementation of
standardized data collection within patient registries, such as clinical audits. This
study also confirms that contemporary research on HSCR tends to focus more on outcomes
(59% of extracted variables) rather than on characteristics used to evaluate care
processes (19%), and more on short-term than on long-term outcomes. Furthermore, considering
the 1,026 distinct parameters mentioned in the included publications, we substantiated
that data collection and reporting lacks uniformity across studies, making it harder
to consolidate and analyze findings cohesively. We also demonstrated substantial variation
in parameter definitions and the tools used across studies to measure important parameters
such as quality of life or other patient-reported outcome measures (PROMs),[24] as illustrated in [Table 3]. This lack of uniformity hinders comparing and contrasting of results across different
studies, and once more indicates an urgent need for creating and implementing standardized,
reliable, and well-defined parameters and tools that are both patient-centric and
disease-specific. Concurrently, it is essential to acknowledge that the NETS1HD study
introduced a novel HSCR core outcome set through rigorous methods.[7] In contrast to this study, our approach not only centered on outcome measures but
also encompassed a comprehensive analysis of treatment and diagnostic characteristics,
as well as patients' baseline characteristics. We focused on identifying the most
extensively studied parameters and did not consult a panel of experts for their ratings
and definitions. Promoting the adoption of this core outcome set and similar initiatives
and implementing these standardized datasets in both large and small pediatric surgical
studies is crucial, as it will enhance relevance, minimizes bias, and facilitate future
meta-analyses.
Standardization of research parameters is especially important in rare disease research,
as the rare nature of the studied conditions often inherently complicates generating
high-quality evidence, as we have confirmed with this review. Because of the demonstrated
lack of high-quality evidence, current guidelines and clinical practices still predominantly
hinge on expert opinions and consensus statements. Moreover, as opposed to the previously
mentioned HSCR core outcome set, which was developed to standardize research, it is
vital to acknowledge the current deficit in standardized measures for evaluating the
quality of HSCR care and the absence of a common benchmarking system. In the modern
healthcare landscape, there is a growing emphasis on the quality of provided care,
and standardized measures are essential for precise benchmarking and the accurate
interpretation of outcomes. Consequently, the development and implementation of standardized
benchmarking through measuring predefined quality indicators could significantly enhance
the overall quality of HSCR care. Current clinical guidelines and consensus statements
and the list of treatment and care process characteristics and outcomes resulting
from this systematic review may function as a starting point for a consensus method
to develop quality indicator sets for such tools, involving both the international
HSCR expert community and patient representatives. The European Reference Network
for Inherited Congenital Anomalies and its registry (EPSA) have the potential to play
a critical role in the more effective collection and analysis of comparative quality
data, ultimately leading to the establishment of best practices and bridging the gap
between short-term and long-term outcomes.[10]
By adhering to the PRISMA guidelines, we ensured a structured and transparent methodology.
The extensive data extraction and categorization allowed for interpretation of the
current most important parameters for clinical researchers in the field. This approach
has markedly reduced the likelihood of overlooking crucial parameters, as we have
included all parameters mentioned in at least 5% of the selected studies. Our findings
emphasize the necessity for standardization of data collection, definitions, and use
of clinical tools and instruments. By addressing a significant gap in HSCR research—namely,
the lack of uniformity in parameter measurement and reporting methods—we not only
bring to light the existing discrepancies but underscore the urgency for a cohesive
approach in future studies. Additionally, the resulting list could lay the groundwork
for determining which parameters are important for assessing the quality of HSCR care.
Our research also has some limitations. First, as we pooled data from children and
adults without providing a distinct analysis for each group, the findings are not
age-specific. It is recommendable to study the functional changes HSCR patients undergo
throughout their lifespans. Second, our research is limited by a specific time frame,
focusing solely on studies published between 2015 and 2021. While this provides insight
into recent clinical practices, it may overlook critical insights from publications
after 2021. Third, although we identified significant variations in definitions, the
abundance of these definitions prevented us from exhaustively detailing each one.
As we are transitioning into prioritizing evidence-based practice and patient-centered
outcomes, embracing a unified approach and adopting quality indicators are crucial.
This approach will not only refine our understanding and management of HSCR but also
fortify the foundations of pediatric surgical research, driving meaningful advancements
in pediatric patient care.
Conclusion
This review highlights the substantial variation in the reporting of patient characteristics
and outcomes in HSCR research, with 1,026 parameters identified in recent literature.
It suggests an urgent need for adopting the newly developed core outcome set, and
establishing a standardized core indicator set. The comprehensive list of studied
parameters identified in our review can facilitate this process. Implementing standardized
measurement and reporting systems is vital, promising enhanced interpretability of
results and the potential for effective quality-of-care benchmarking across pediatric
surgical centers in Europe. Looking ahead, the European Pediatric Surgery Audit (EPSA)
emerges as a crucial entity to realize this vision, heralding a new era in pediatric
surgical research and care.
