Pseudo-obstruction in children: transplant or wait?
With advances in immunosuppression intestinal transplantation has emerged as a viable option for patients with chronic intestinal failure and life threatening complications. With a one year graft survival rate of around 60% over the past few years, the current situation compares favourably with the pre-1990 results when 30% survival rates were achieved (data from the Intestinal Transplant Registry: www.lhsc.on.ca/itr). At the 7th Meeting of the European Intestinal Transplantation Study Group (Brussels, 1998), one year survival rates up to 80–90% were reported by some very experienced centres.
In children, chronic intestinal pseudo-obstruction is often a primary disorder, either congenital or acquired. Secondary pseudo-obstruction is related to various systemic diseases and is more common in older patients. According to the pathological findings, the primary disease is classified as neuropathic, myopathic, or idiopathic if no specific pattern is recognised. The disease always involves the small bowel and may occur in any other (or all) regions of the digestive tract. Signs and symptoms of obstruction, but without anatomical obstruction being evident on conventional imaging, dominate the clinical picture. The first symptoms may appear as early as the neonatal period or later in life and range from occlusion and complete intolerance to food to recurrent abdominal pain and constipation. Chronic bacterial intestinal overgrowth and chronic central venous access for parenteral nutrition can be associated with infectious episodes, which in turn can result in evolution towards liver fibrosis/cirrhosis and cholestasis.1 2 Little has been reported on the long term outcome of children with primary intestinal pseudo-obstruction, especially those presenting with severe disease early in life. Because the disease is uncommon and because it usually progress slowly, the poor long term prognosis has probably been underestimated in the past.
Sigurdsson et al’s experience with 27 young patients (1–19 years old) (see page 570) is of major importance for several reasons. As the patients were referred for transplant, this group is probably not representative of the general population but confirms, however, that severe complications can occur during childhood. Multidisciplinary, perioperative management is mandatory and surgery must be tailored to each individual because the disease can affect other portions of the digestive system and other organ systems.1 With expert care, good results can be achieved in this difficult group of patients, as illustrated in this series.
Of more concern is that of the 22 patients registered for transplantation, eight were transplanted, eight died before a graft became available and six were still awaiting transplant. This is of course a result of the current shortage of donors but also reflects the poor clinical condition of the patients at the time of registration. Poor clinical condition and pre-existing complications are clearly related to an increased risk of death.3-5 Moreover, worsening of the clinical condition while on the waiting list leads to increased post-transplant morbidity and a higher death rate.3-5
Taking into account that the long term results of transplants are still not as good as those for long term parenteral nutrition, the timing of referral and the criteria for small bowel transplantation continue to be debated. The current general indication for bowel replacement is still the development of life threatening problems in the context of gut failure (mostly progressive liver disease and/or central venous access problems). Sigurdsson et al propose a reasonable approach—patients who do not present with cholestasis or hepatic dysfunction should be managed conservatively. These patients should benefit from the best appropriate care, minimising the risk of progressive liver disease and preventing line infections.6This strategy adheres to the current consensus guidelines: transplantation should be avoid by adequate medical management but patients who develop complications (cholestasis, portal hypertension, vascular access problems) should be referred for transplantation.
Earlier referral and thus registration of patients while still in reasonable health may help to decrease both pre- and post-transplant risks. In a recent study, early referral for liver transplantation led to better results which in turn had a positive effect on patient selection.7
Bueno et al reported that a bilirubin concentration above 3 mg/l, a prothrombin time of more than 15 seconds or bridging fibrosis at liver biopsy were significantly related to poor outcome.3 Also, 80% of the children under 1 year of age when referred for transplant assessment died within two years.3 Thus, patients should be referred to transplant centres as early as possible before liver dysfunction or portal hypertension has developed, or at least before complications occur. Then, after a full evaluation of the patient’s condition, the appropriate management strategy should be discussed with the referring team, including the option for possible future transplantation and its ideal timing.
Early referral and early interactive management between local and transplant centres is essential if the general outcome of patients in potential need of bowel replacement is to be improved.