Authors' objectives: Up to 30% of children have constipation at some stage in their life. Although often short-lived, in one-third of children it progresses to chronic functional constipation, potentially with overflow incontinence. Optimal management strategies remain unclear. To determine the most effective interventions, and combinations and sequences of interventions, for childhood chronic functional constipation, and understand how they can best be implemented. Chronic functional constipation (CFC) in childhood is common; it is estimated to affect 5–30% of school-aged children, becoming chronic in around one-third of cases. CFC has negative effects on quality of life (QoL) of children, families and carers, with increasing impact as the child gets older. Although rarely life-threatening, CFC is an unpleasant and distressing condition, associated with a wide range of complications, including physical discomfort, missed school, poor school performance, social isolation and reduced involvement in group activities. More than a third of children with CFC will present clinically with behavioural problems as a result of the constipation. The healthcare costs of childhood CFC are significant. Treatments are usually directed at symptom control since limited diagnostics mean no underlying cause is identified in more than 95% of cases, thus limiting ability to stratify treatments. There are a number of different interventions available for the management of CFC; however, the optimal strategy for combining and implementing interventions for individual circumstances remain unclear. National Institute for Health and Care Research (NIHR) commissioned this work to address the question: ‘What are the most effective interventions, and combinations and sequences of interventions, for childhood chronic functional constipation (CFC), and how can they best be implemented?’ Specific research questions (RQs) answered by this project were: RQ1: What is the current evidence relating to management strategies for childhood CFC? (Scoping review). RQ2: What are the most effective childhood CFC strategies and combinations of strategies in relation to outcomes of importance to stakeholders and/or cost to the patient/NHS? [Systematic review (SR) of evidence of effectiveness and cost effectiveness]. RQ3: What factors are associated with implementation success or failure of childhood CFC strategies and combinations of strategies for different subgroups? (SR of factors affecting implementation). RQ4: What are the evidence gaps in childhood CFC management strategies?

Authors' results and conclusions: Stakeholders prioritised outcomes, developed a model which informed evidence synthesis and identified evidence gaps. Management of childhood chronic functional constipation is complex. The available evidence remains limited, with small, poorly conducted and reported studies. Many evidence gaps were identified. Treatment recommendations within current clinical guidelines remain largely unchanged, but there is a need for research to move away from considering effectiveness of single interventions. Clinical care and future studies must consider the individual characteristics of children. The scoping review included 651 studies, including 190 RCTs and 236 primary studies. Forty-eight interventions (or combinations of interventions) were reported. Studies were mainly conducted in high-income countries; no studies from low-income countries were identified. The most frequently reported interventions were delivered by carers, prior to healthcare professional involvement (22%); these were primarily lifestyle interventions focused on diet. The least frequently reported were psychosocial interventions (3%). Children were recruited from a variety of settings including hospitals, clinical outpatients, and other community settings. Interventions were generally delivered face to face either at home or within a variety of hospital settings such as the emergency department. Interventions were rarely delivered in education settings (e.g. school-based settings) (n = 5) or residential care/looked after population (n = 1) settings. The most frequently reported outcome measurement was defaecation frequency, which was reported in one-third of studies within the scoping review. School attendance or absenteeism was the least frequently reported outcome (n = 8 studies). Our SRs of effectiveness included 32 studies (including 2 SRs) which explored effectiveness of interventions delivered by families/carers (‘Level 0’); 21 studies (including 2 SRs) which explored effectiveness of interventions delivered by wider children’s workforce (‘Level 1’); 31 studies (including 1 SR) which explored effectiveness of interventions delivered by continence teams (‘Level 2’); 42 studies (no SRs) which explored effectiveness of interventions delivered by specialist consultant-led teams (‘Level 3’); 15 studies (no SRs) which explored effectiveness of different models of service delivery; 15 studies (2 SRs) which explored effectiveness of complementary therapies; and 4 studies (1 SR) which explored effectiveness of psychosocial interventions. Interventions for which there was some evidence of potential benefit included, within Level 0: a trial of cows’ milk-free diet, educational interventions for parents, selenium supplements. Within Level 1: laxatives, physical exercise focused on pelvic floor muscle, combined pharmacological, diet and behaviour programme. Within Level 2: combined oral and enema therapy, transanal irrigation, biofeedback (for children with abnormal defaecation dynamics), combined treatment programmes. Within Level 3: botulinum toxin, antegrade continence enema (ACE)/Malone antegrade continence enema (MACE), sacral modulation. Models of care delivery which may be beneficial included nurse-led clinics, an algorithm, or care pathway, used in primary care settings, specialist (Level 2) services and web-based information, following an appointment with a specialist. Complementary therapies for which there was some evidence of effectiveness included: connective tissue manipulation (CTM) for children with cerebral palsy (CP), and some herbal/traditional medicines. There was some evidence in favour of behavioural therapy. Interventions which evidence suggests may not be beneficial include, probiotics, additional dietary fibre, increased fluid intake and biofeedback (for children with normal defaecation dynamics). Evidence relating to probiotics was judged to be moderate quality, but for all other interventions was considered low to very low quality. There was insufficient evidence to support conclusions relating to several other interventions. We identified 31 studies which reported some evidence relating to cost or resource use, of which 20 were cost-of-illness studies. Fewer than 30% of the studies employed a formal economic evaluation study design. Most studies were poorly reported with limited details. Data included in this review were insufficient to support any generalisable conclusions relating to cost or resource use. One hundred and six studies described multiple barriers and facilitators across the five domains of the CFIR framework. The most commonly reported factors related to ‘successfulness’ of an intervention included; whether the intervention was adaptable, flexible and offered an advantage over an alternative solution; understanding the tension for change (i.e. why clinicians and families felt that the changes were needed now); the taboo nature of constipation and the reluctance of children, families, healthcare professionals and wider society to openly engage in discussion about constipation; a lack of understanding of what children and their families need; self-efficacy, coupled with individual knowledge and beliefs; and engagement of champions to support children. Research gaps were identified through evidence maps and stakeholder discussions. Key topics considered priorities for future research relate to recognition of CFC; information provision; diet; laxatives and combinations of laxatives; behavioural therapy and psychological support. Future research studies should address what works for which individual child, and when, including children with and without additional needs. Research to explore the optimal delivery of services, including identification of key components and features of effective teams and criteria for referring children from one ‘level’ to the next, is needed. We conducted a comprehensive review of all evidence relating to interventions, and combinations of interventions, for children with CFC. The finding from our review are generally in agreement with the current guideline recommendations, where recommendations exist. A significant proportion of interventions for which we found evidence had not been addressed within current guidelines. This project has highlighted that research in this field often does not adhere to recognised standards for conduct and reporting or consider the complexities of interventions for CFC. We found no evidence which gave us high certainty in the findings; and we only had moderate certainty relating to one intervention (probiotics, with evidence demonstrating that probiotics may not have any beneficial – or harmful – effect). Our certainty about all other findings was low to very low or, in many cases, we judged that the evidence was insufficient to support any generalised conclusions. The current evidence base rarely measured outcomes deemed of highest priority to children and families, and many studies failed to describe the complex nature of the treatments that a child may be receiving. This limits the conclusions that can be made from the current evidence. Further, the limitations within the evidence base reduce confidence in recommendations and create a barrier to implementation of best practice, impairing progress in efforts to improve outcomes for this group of children. Our findings do not indicate that changes are necessarily needed to the treatment recommendations within current clinical guidelines. However, management of childhood CFC is complex, and there is no simple ‘one size fits all’ approach. Clinical care and future studies must consider the individual characteristics of each child with constipation, and the context – or environment – within which they live. Key goals of successful management of CFC should be early recognition of symptoms and delivery of interventions by families/carers, achieved by providing children and families/carers with effective education and support from members of the wider children’s workforce (primary care services). Development, evaluation and implementation of strategies to enhance the delivery of services focused on individualised care, combining lifestyle and behavioural strategies with laxatives are a priority. To avoid further research waste, it is essential that future research addresses the questions which are of the highest priority to key stakeholders and has the highest possible standards of conduct and reporting. Future research studies should address what works for which individual child, and when, including children with and without additional needs. Future research into any interventions for childhood CFC should take into account relevant evidence relating to the development and evaluation of complex interventions.

Authors' methods: Key stakeholders, comprising two parents of children with chronic functional constipation, two adults who experienced childhood chronic functional constipation and four health professional/continence experts, contributed throughout the research. We conducted pragmatic mixed-method reviews. For all reviews, included studies focused on any interventions/strategies, delivered in any setting, to improve any outcomes in children (0–18 years) with a clinical diagnosis of chronic functional constipation (excluding studies of diagnosis/assessment) included. Dual reviewers applied inclusion criteria and assessed risk of bias. One reviewer extracted data, checked by a second reviewer. Scoping review: We systematically searched electronic databases (including Medical Literature Analysis and Retrieval System Online, Excerpta Medica Database, Cumulative Index to Nursing and Allied Health Literature) (January 2011 to March 2020) and grey literature, including studies (any design) reporting any intervention/strategy. Data were coded, tabulated and mapped. Research quality was not evaluated. Systematic reviews of the evidence of effectiveness: For each different intervention, we included existing systematic reviews judged to be low risk of bias (using the Risk of Bias Assessment Tool for Systematic Reviews), updating any meta-analyses with new randomised controlled trials. Where there was no existing low risk of bias systematic reviews, we included randomised controlled trials and other primary studies. The risk of bias was judged using design-specific tools. Evidence was synthesised narratively, and a process of considered judgement was used to judge certainty in the evidence as high, moderate, low, very low or insufficient evidence. Economic synthesis: Included studies (any design, English-language) detailed intervention-related costs. Studies were categorised as cost-consequence, cost-effectiveness, cost-utility or cost-benefit, and reporting quality evaluated using the consensus health economic criteria checklist. Systematic review of implementation factors: Included studies reported data relating to implementation barriers or facilitators. Using a best-fit framework synthesis approach, factors were synthesised around the consolidated framework for implementation research domains. We conducted a three-stage pragmatic mixed-method study. In stage 1, we completed a broad, comprehensive scoping review. In stage 2, we conducted focused SRs evaluating effectiveness, cost effectiveness and factors affecting implementation. In stage 3, we integrated findings, generating interactive evidence maps, exploring complementarity between our findings and published clinical guidelines, and identifying evidence gaps. Patient and public involvement Stakeholder involvement was central to our project. We formed a stakeholder group (SG) from across the UK, comprising people with lived experience of childhood CFC, parents of children with CFC, healthcare professionals and representatives of relevant charities. We adhered to key principles for research co-production. The SG provided continuous project oversight and completed specific activities. These activities included development of an intervention taxonomy and logic model, agreement on outcomes for the review, identifying evidence gaps and reaching consensus on clinical implications.

Project Status: Completed

URL for project: https://www.journalslibrary.nihr.ac.uk/programmes/hta/NIHR128470

Year Published: 2024

URL for published report: https://www.journalslibrary.nihr.ac.uk/hta/PLTR9622

URL for additional information: English

English language abstract: An English language summary is available

Publication Type: Full HTA

Country: England, United Kingdom

DOI: 10.3310/PLTR9622

Organisation Name: NIHR Health Technology Assessment programme

Contact Address: NIHR Journals Library, National Institute for Health and Care Research, Evaluation, Trials and Studies Coordinating Centre, Alpha House, University of Southampton Science Park, Southampton SO16 7NS, UK

Contact Name: journals.library@nihr.ac.uk

Contact Email: journals.library@nihr.ac.uk