Background Intestinal transplant in infants with severe short bowel syndrome (SBS) is an emerging therapy, yet without sufficient long-term data or established guidelines, resulting in possible variation in practice.
Objectives To assess current attitudes and counselling practices among physicians regarding intestinal transplant in infants with SBS, and to determine whether counselling and management vary between subspecialists or centres.
Methods A national sample of practicing paediatric surgeons and neonatologists was surveyed via the American Academy of Paediatrics listserves. Results were analysed by physician subspecialty and again by presence or absence of intestinal transplant at respondent's centre.
Results The survey was completed by 433 respondents, consisting of 363 neonatologists and 70 paediatric surgeons. Fifty-seven respondents (13.2%) practiced at a centre that performed intestinal transplants in children. The vast majority of respondents (91% for preterm, 95% for term neonates) felt that maintaining a neonate with SBS on total parenteral nutrition for intestinal transplant was ethically optional (neither impermissible nor obligatory), and that parents should be given an informed choice whether to pursue that option. However, only 33% indicated they often/always offer intestinal transplant as a treatment option to families in this situation.
Conclusions There is a marked disparity between individual physicians’ beliefs regarding the acceptability of intestinal transplant for severe SBS and their reported practice. Wide variability exists among physicians with respect to their knowledge, beliefs and practice regarding severe SBS, raising concerns about transparency and justice. Survival data prior to transplant, currently unavailable, are essential to rational decision making and informed parental permission.
- Clinical Ethics
- Informed Consent
- Newborns and Minors
- Technology/Risk Assessment
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Short bowel syndrome (SBS) is defined as insufficient intestinal length and/or function leading to malabsorption and often inability to thrive. In children, necrotising enterocolitis (NEC) is the primary cause of SBS, particularly in extremely premature neonates, followed by congenital bowel defects. Advancements in medicine have led to an increase in survival for infants and children with SBS, including total parenteral nutrition (TPN), intestinal adaptation and surgical lengthening procedures.1–7
Intestinal transplant is emerging as a potential treatment option for children with severe SBS who have failed these therapies. Survival data have been reported for children with severe SBS after receiving intestinal transplant, although data are lacking for survival while awaiting transplant, quality of life (QOL) for the patient and family, and long-term outcomes.1–7 Maintaining a newborn with SBS on TPN with the goal of eventual intestinal transplant is not currently standard practice, but many feel it is an ethically permissible treatment option,8 and some physicians are offering it when counselling. Like many emerging technologies, there is currently limited information regarding the frequency of its use, and no guidelines for physicians on whether, when and how to counsel families of infants with severe SBS about the possibility of intestinal transplant as a treatment option.9–12 This raises the concern of variation in counselling and potential inconsistencies in informed consent.13 Families in similar circumstances may be given very different options with no clear ethical justification for that disparity.
The aim of this study was to assess current knowledge, attitudes and counselling practices among neonatologists and paediatric surgeons regarding intestinal transplant in neonates with severe SBS. We hypothesised that physician attitudes, knowledge, and counselling differ between subspecialties and centres, and that infants with severe SBS may be managed more aggressively at centres capable of performing intestinal transplant.
The target population included practicing neonatologists and paediatric surgeons in the USA who were members of the American Academy of Paediatrics (AAP) Section on Perinatal Paediatrics (SoPPe) and Section on Surgery (SoSu), respectively, and included on the AAP email listservs. There were 2900 neonatologists and 500 paediatric surgeons registered with the AAP sections at the time of this study.
A survey was developed by the authors via Survey Monkey and reviewed by independent subject matter experts to ensure face and content validity (see online supplementary file). The study was approved by the Human Investigation Committee, and granted exemption from full Institutional Review Board review under federal regulation 45 CFR 46.101(b)(2), including a waiver of written consent. The survey was approved by both the AAP SoPPe and SoSu, who gave authorisation for membership distribution.
Using the AAP's current email listservs, the survey was sent out in July–August 2011 to practicing neonatologists and paediatric surgeons in the USA. The email, sent twice to increase response rate, described the research, asked subjects to participate by completing the survey, and provided a link to the on-line survey via Survey Monkey. There were no incentives to complete the survey.
Participants were queried on their knowledge, attitudes and counselling practices regarding intestinal transplant for infants with severe SBS. They were also asked whether they viewed the procedure as ethically impermissible (treatment should not be offered nor made available if requested), optional (parents should be given an informed choice), or obligatory (procedure should be provided unless contraindicated), and were asked to specify their preferred course of action given specific clinical case scenarios. Demographic questions included respondent subspecialty, institution type, institutional availability of intestinal transplant, and whether ethics consultation is used to help guide management in these cases.
Data were recorded without identifiers and analysed in aggregate form. Frequencies were used to describe respondent characteristics. Results were analysed by subspecialty, and again by availability of intestinal transplant at the respondent's institution via the χ2 test and Cochran–Mantel–Haenszel χ2 test for trend with 1° of freedom. A general inductive approach was used to analyse qualitative data, consisting of solicited comments.14–15 Comments were reviewed independently by coinvestigators, analysed and grouped into similar concepts and categories, ensuring thematic saturation.
Data were analysed for those respondents who completed at least 80% of the survey, yielding a final response rate of 13% (n=433). Of those, 84% self-identified as practicing neonatologists (n=363) and 16% as paediatric surgeons (n=70). Intestinal transplant was available at 13% of respondents’ institutions (n=57). Demographic characteristics were similar, however 44% respondents reported >20 years of practice (table 1).
When asked to estimate current 5-year outcomes in children with NEC after intestinal transplant (∼45% survival), only 30% answered correctly. Excluding ‘uncertain’ responses, paediatric surgeons were four times more likely to overestimate survival than neonatologists (48% vs 10%, p<0.0001), while neonatologists were twice as likely to underestimate survival (48% vs 22%, p<0.0001) (figure 1). Respondents from centres that offered intestinal transplant were twice as likely to overestimate survival (29% vs 15%, p 0.059).
The majority of respondents felt that parents of a neonate with severe SBS should be offered the option of TPN with a goal of reaching intestinal transplant (90% for preterm, 95% for full term). That is, the procedure was believed to be ‘ethically permissible’ (figure 2). Further, 90% of respondents ‘strongly agreed/agreed’ that family preference should influence this decision. When queried about their practice, however, only 33% reported that they ‘often/always’ give parents that option, while 30% ‘rarely/never’ give parents the option. Paediatric surgeons were more likely to ‘often/always’ offer intestinal transplant when counselling families versus neonatologists (p 0.0003) (figure 3). Far fewer physicians (9%) ‘often/always’ recommend transplant.
Just over half the respondents (51%) indicated they ‘often/always’ offer intestinal rehabilitation, including bowel-lengthening surgeries, while 29% ‘often/always’ recommend it. Paediatric surgeons were more than twice as likely as neonatologists to ‘often/always’ offer and recommend these therapies (p<0.0001). When counselling parents of a neonate with severe SBS, a majority of respondents (70%) ‘often/always’ offer the option of comfort care only (CCO), and 37% recommend that life-sustaining treatment be withdrawn (figure 3). Eleven percent ‘rarely/never’ give parents that option.
There was no consensus with regard to management of profound bowel loss discovered intraoperatively. Physicians were asked to consider several management approaches in a scenario involving an otherwise healthy premature infant who develops NEC totalis, confirmed in the operating room. Thirty percent of respondents indicated they would opt to close the abdomen without removing bowel, return the patient to the neonatal intensive care unit (NICU), inform the parents that the child will not survive and offer CCO, without offering intestinal rehabilitation or transplant. Neonatologists were twice as likely to choose this option as paediatric surgeons (32% vs 18.6%, p 0.005), as were respondents from centres that did not offer intestinal transplant (31.9% vs 15.8%, p 0.048). Another 1/3 of physicians would opt to have the diseased bowel removed in the OR, return the patient to the NICU, then offer the parents either CCO, life-sustaining treatment for possible intestinal transplant, or 24–48 h on life-sustaining treatment while deciding. Twenty-four percent felt the possibility of extensive bowel loss should be discussed with parents prior to surgery, with the options of resection or CCO discussed and decided then. Only 0.5% of physicians thought the diseased bowel should be removed, and the parents informed that the child would be maintained on life-sustaining therapy for possible intestinal transplant. That is, almost no respondents felt that, upon the discovery of inadequate viable bowel, a path toward transplant was obligatory. If the above scenario involved a loved one, perhaps one's own child, just over 50% respondents opted for CCO, and 25% preferred intestinal transplant, without any differences between subspecialty or centres.
The most common reason cited by respondents for not offering intestinal transplant to parents of an infant with severe SBS was the long-term burden to the child (48%), followed by the long-term burden to the family (42%), inadequate outcome data (32%), and poor outcome data, defined as sufficient data that does not support the use of the treatment (27%). Finally, 7% cited the unavailability of the procedure at their own institution as a contributing reason.
There were several notable differences in responses between specialties, and between centres that do and do not perform intestinal transplant. Paediatric surgeons were more than twice as likely as neonatologists to say the option of possible intestinal transplant for a neonate with severe SBS was ethically impermissible (11.4% vs 5.8%) (figure 2). While half the respondents (51.5%) indicated they ‘often/always’ offer intestinal rehabilitation, including bowel-lengthening surgeries, there were no respondents at centres that perform intestinal transplant who ‘never offered’ intestinal rehabilitation (p 0.0004). Paediatric surgeons were more than twice as likely as neonatologists to ‘often/always’ offer and recommend these therapies (p<0.0001). Roughly 33% of respondents ‘often/always’ offer intestinal transplant as a therapy option, and fewer than 10% recommended it. Paediatric surgeons were more likely to ‘often/always’ offer intestinal transplant when counselling families versus neonatologists (p 0.0003) (figure 3).
Hospital ethics consultations were estimated to be ‘never/rarely’ used by most healthcare teams in these situations (75%, p 0.0083). Of those who had used ethics consultation, 63% reported they were beneficial.
Themes from respondents’ comments are summarised (box 1), with selected representative comments.
Lack of physician knowledge
“I am not aware of latest information in this area.”
“It would be helpful to have a sense of where the outcomes and data are, so good counseling could be provided.”
Insufficient data regarding outcomes
QOL, burden to child and family
Morbidity/mortality while awaiting transplant
Cost–benefit analysis data
“One needs to consider not only mortality after transplant, but mortality while awaiting transplant.”
QOL, burdens to patient and family
“There remains huge burden to the child and family, plus a huge cost.”
Surgical concerns, timing of surgery and decision making
Feasibility/availability of treatment
“The paucity of centers that perform intestinal transplant increases the complexity of the issue further.”
Intestinal transplant criteria
Selection of patients and counselling
“This is a very difficult problem because there are no accepted standards for appropriate intestinal rehab and criteria for intestinal transplant.”
Counselling and decision making
Evidence-based, transparent, interdisciplinary
Deference to parental rights
“All options, with risks and benefits, should be discussed to the best of our abilities.”
Ethics consultation, use and usage
“…Ethical consults must be obtained in all cases of SBS especially when there is conflict on prognosis and management.”
Limitations of this study should be noted to place the discussion in appropriate context and avoid overgeneralisation. Limitations include a response rate of 13%, although this yielded a sample size of 433, and diverse responses within this small convenience sample. More neonatologists responded than paediatric surgeons, although this reflected similar subspecialty response rates (12.5% vs 14%), and 44% of respondents reported >20 years of practice, perhaps influencing results. Selection bias cannot be excluded; those who participated may have different or more strongly held beliefs and counselling practices than non-responders. Also, respondents answered questions based on scenarios with limited information, without additional details that may have altered responses. Finally, reporting and recall biases are possible. These data may have been more generalisable had a more aggressive recruitment approach been used. Nevertheless, these results reflect the opinions and practice attitudes of over 400 physicians in various clinical settings in the USA.
Overall, these results reveal a wide variation in knowledge, ethical beliefs and counselling practices among neonatologists and paediatric surgeons regarding intestinal transplant for neonates with severe SBS, and between centres that do and do not offer intestinal transplant. The only clear consensus appears to be that physicians believe both intestinal transplant and CCO are ethically permissible treatment options and should be offered to families during counselling.
Intestinal transplant is currently ethically permissible because it is neither impermissible nor obligatory.8 It is not impermissible because, based on available data, it appears to offer a reasonable chance of survival in a child who otherwise would not survive. While there are legitimate concerns about QOL and long-term survival, there are no compelling data to suggest that this therapy is worse than death, or that the chance of success is unacceptably low. Thus, intestinal transplant should not be impermissible at this point.
On the other hand, intestinal transplant is not obligatory, either. While recent studies demonstrate improved survival in children after intestinal transplant, data are lacking for long-term survival and QOL post-transplantation in this population.8 Further, the possibility of ‘hidden’ mortality also exists between the time a decision is made to maintain a child on TPN until transplant, and the actual procedure, potentially skewing survival statistics. Finally, a benefit/burden analysis revealed that intestinal transplant bestows significant theoretical burden to the child, as well as to others, including the child's family, using anecdotal evidence and extrapolated judgments, as these data for this population are also unavailable. Given recent advances and improved but limited outcome data, ‘intestinal transplant for neonates with severe SBS should be discussed with families as an ethically permissible treatment option’,8 but should not be mandatory at this time.8
Such wide variation in opinions and attitudes revealed in this study surrounding this procedure may be a reflection of either moral uncertainty, prognostic uncertainty or, most likely, a combination of the two. Prognostic uncertainty exists because we are lacking data on survival while awaiting transplant, as well as QOL and long-term survival post-transplant. Moral uncertainty exists because it is unclear if the burden of such a long journey (both getting to transplant and post-transplant) is worth the benefit of possible survival. More data could potentially alleviate prognostic uncertainty by illuminating best treatment options, leading to more definitive practice guidelines and improved decision making and counselling. However, if the wide variation is due to moral (normative) uncertainty, rather than reservations regarding outcomes and prognosis, then more data may not necessarily help. For instance, when weighing different treatment options, what matters most—survival alone, maximising individual happiness, maximising overall happiness, or another measure altogether? Depending on the ethical perspective taken, additional statistics may not effectively clarify the ‘right’ choice or lead to a clear consensus within the field.
Particularly when discussing emerging technologies, where outcome data may be inadequate or evolving and guidelines or consensuses are lacking, good counselling must commence with knowledge of most current data. Many responding physicians were not aware of current outcome data surrounding intestinal transplant in children. Only 30% of respondents were able to correctly identify recent 5-year survival rates for children with NEC after intestinal transplantation. Lack of knowledge on the topic was also commonly self-reported in the comments section.
The vast majority of physicians (>90%) who responded felt that intestinal transplant in this population was an ethically permissible treatment option. However, only 33% of respondents ‘often/always’ offer intestinal transplant as a therapy option, and fewer than 10% recommend it. In other words, most physicians surveyed do not offer intestinal transplant when counselling, despite believing it is an ethically permissible option. Thus, with regard to parental counselling, there is a sharp disconnect between what physicians state ought to be done and what they report doing in practice.
Potential reasons for this ethical disconnect are worth exploring. One reason may be that clinicians have not yet changed routine practice to reflect new beliefs. Practice often lags behind a new mental model or paradigm, and physicians are not immune to the inertia of habit. Physicians may also fear coercing parents into pursuing a treatment option they may not have really desired. However, this assumption and approach denies parents their fundamental right to be informed, including of data inadequacies, and decide on behalf of their child.
An alternative explanation for this ethical disconnect could be that physicians are uncomfortable with the unavailability of important outcome data, such as survival while awaiting transplant. This potential ‘hidden mortality’ was a concern for some physicians; of those who specified they would not offer intestinal transplant, about 30% cited inadequate outcome data. More studies are needed, but in the interim, we must counsel to the best of our ability with available data.
Another reason for the inconsistency between ethical belief and practice revealed in this survey may be that physicians do not want to overly burden the child or family by offering the pursuit of intestinal transplant. Responding physicians identified QOL and the social and financial burden for patients and their families as significant factors in decision making and counselling. Of those who indicated they would not offer intestinal transplant, 50% cited long-term burden to the child, and 40% indicated long-term burden to the family. It is sometimes difficult to ascertain whether a family truly grasps the benefits, burdens and long-term implications of such a treatment, despite our best efforts when counselling. However, if physicians are refraining from offering parents the option of TPN and possible intestinal transplant because of the perception of undue burden to the child and family without clear benefit, then it seems these physicians consider this treatment ethically impermissible, contrary to what they indicated earlier. Research has yet to address questions and concerns regarding burden to the child and family.
When asked what treatment one would pursue for a loved one with severe SBS, about half (53%) the respondents preferred CCO, regardless of specialty. This suggests perhaps a tolerance for others choosing the option of intestinal transplant, but a personal preference for pursuing less aggressive treatment, specifically CCO. Surprisingly, 25% respondents preferred pursuing intestinal transplant for a loved one, which was higher than expected, given that only 9% recommended it when counselling families. These findings may reflect participation or self-selection bias; those who completed the survey may have felt strongly about either CCO or intestinal transplant.
This study revealed important differences in responses between specialties and between centres that do and do not offer intestinal transplant. Interestingly, paediatric surgeons and respondents from centres that offered intestinal transplant overestimated survival after transplant, and neonatologists underestimated actual survival. When presented with a case of a premature neonate with severe NEC, neonatologists and respondents from centres that did not offer intestinal transplant were more likely to recommend CCO as a treatment option, and not offer intestinal transplant. Ideally, these specialty and centre biases should not play a role when counselling families. Treatment options should be presented based on most current, available data, while keeping in mind the preferences and values of the family.
Inconsistencies and discrepancies in physician counselling and practices may represent an injustice for patients and their families.13 Patients with similar clinical presentations should be offered similar options, unless a clearly relevant difference between them can be identified.13 This is true irrespective of the availability of the procedure in the patient's institution, unless transfer is not feasible. Regardless of the sufficiency of the data, and regardless of which specialist or centre is providing the counselling, good communication and transparency are essential. Full disclosure of any data inadequacies and all feasible and ethically defensible treatment options, even if the physician or centre does not provide them, should be made to parents.13 ,16
Appropriate timing of counselling provided to families is also of critical importance. The survey revealed wide variation in when discussions regarding the pursuit of intestinal transplantation ought to take place. There was no consensus regarding intraoperative counselling in the case of a neonate with suspected NEC totalis. Some respondents believed the medical/surgical team should decide on either CCO or resection intraoperatively, and then inform the parents postoperatively, rather than presenting options. These approaches, however, deny parents the right to decide in light of new information. It seems best to offer counselling at multiple points throughout the hospital course, including prior to surgery, intraoperatively and postoperatively. Parents should be kept abreast of new information in order to adjust decision making accordingly. For example, one reasonable approach could be that the possibility of extensive bowel loss is discussed with parents prior to surgery, with the options of CCO, resection and/or pursuit of intestinal transplant discussed and a tentative plan decided then. Decisions could be confirmed with the team and family during surgery, or even revised if necessary based upon new or unexpected intraoperative findings. Another approach, which many respondents thought was best, that could mitigate pressure placed on parents to make a rapid decision while still in shock, may be to pursue a resection and postoperative life-sustaining therapy for a day or two while parents decide. It may be more difficult, however, for parents to decide to withdraw life-sustaining treatment once started.
Given the complexity, variability and uncertainty surrounding this issue, a consensus among specialists, if possible, may aid in decision making and counselling for these families. Guidelines outlining specific criteria for intestinal transplant in children may also be helpful in improving identification of appropriate candidates for this therapy. Counselling could then be aimed at selected families, sparing other families the false hope of a treatment unlikely to benefit their child. Discussions should ideally be multidisciplinary, and occur frequently throughout the child's hospital course, providing improved communication in difficult circumstances.
Respondents reported that hospital ethics committee consults were rarely used in these situations. Reasons for this are unclear. Those who reported their use, however, felt they were very helpful when dealing with such complex cases. Ethics committees are likely being underutilised and may be beneficial for all involved.17
Physicians’ knowledge, practice, ethical beliefs and counselling vary widely for families of infants with severe SBS regarding intestinal transplant. This study highlights important inconsistencies in counselling and what treatment options are made available to patients and their families, without clear justification for the discrepancies. Most remarkably, the vast majority of physicians surveyed felt that intestinal transplant in this setting is ethically permissible, yet most do not offer it to parents. Moral and prognostic uncertainty each may play a role in such counselling variations and inconsistencies. When counselling, physicians need to be knowledgeable and honest about treatment options and latest outcomes, including data limitations and potential biases.
Parental preference should be honoured in decision making, unless there are compelling reasons to do otherwise. New data revealing grim survival to transplant, or an unbearable QOL post-transplant, may be sufficient reasons, but research to investigate this has yet to be done. These missing data are essential to rational decision making and informed parental permission, and should be the focus of ongoing research in this area. Finally, while this study and analysis are limited to the question of intestinal transplant in infancy, this same approach could serve as a model when considering other emerging technologies in medicine.
We thank the AAP for distributing this study, and Veronika Northrup, MPH, for assistance with statistical analyses.
This web only file has been produced by the BMJ Publishing Group from an electronic file supplied by the author(s) and has not been edited for content.
Files in this Data Supplement:
- Data supplement 1 - Online supplement
Contributors CLC: conceptualised and designed the study, designed and revised the data collection instrument, drafted the initial manuscript, and revised and approved the final manuscript as submitted. KAD: critically reviewed the data collection instrument, reviewed and revised the manuscript, and approved the final manuscript as submitted. MRM: conceptualised and designed the study, reviewed and revised the data collection instrument and manuscript, and approved the final manuscript as submitted.
Competing interests None.
Ethics approval The study was approved by the Yale University School of Medicine Human Investigation Committee, and granted exemption from full Institutional Review Board review under federal regulation 45 CFR 46.101(b)(2), including a waiver of written consent.
Provenance and peer review Not commissioned; externally peer reviewed.
Data sharing statement Unpublished data in raw form are available in spreadsheet format from Dr Christy Cummings.
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