Definitive treatment

Since the introduction of tacrolimus (originally FK506) as an immunosuppressive drug after organ transplantation in the early 1990s, the outcome of intestinal transplantation markedly improved. Several cases of successful transplantation for MVID have been reported.30-35 Transplantation involved isolated intestine,30-33 or intestine combined with the liver.31,32 Following the report of these cases, there has been an ongoing discussion on whether or not the colon should be transplanted together with the small bowel.

We recently evaluated the possibility and outcome of intestinal transplantation in ten consecutive patients with early-onset congenital MVID at Necker-Enfants Malades Hospital (Paris) (Rue-mmele et al, submitted for publication). Two patients died before they could be put on a waiting list for small-bowel transplantation; one patient is still waiting. We performed cadaveric intestinal transplantation in seven patients aged between 3 and 11 years by using tacrolimus, steroids and interleukin (IL-2) blockers. Three transplantations were performed with isolated intestine, and four with intestine associated with the liver. Right colon transplantation was performed in five cases (two with isolated intestine). One patient died during transplantation surgery from acute liver failure and hemodynamic shock, probably due to re-perfusion shock. The six others (86%) survived, with a median follow-up of 3 years (range 1-8 years). Graft rejections occurred in two patients (one with isolated intestinal transplantation, and one with intestine and liver), who responded favorably to methylprednisolone pulses. Complete weaning from PN was achieved in all patients: the five patients with an additional colon graft were off PN after a median of 36 days after surgery, as opposed to those without colonic transplant who obtained full intestinal autonomy several months after transplantation. Thus, intestinal transplantation alone or in combination with liver, offered MVID children for the first time a long-term perspective. In this series at Necker-Enfants Malades Hospital, additional colon grafting markedly improved outcome and quality of life after transplantation in MVID. These preliminary results of intestinal transplantation in this rare disease are very encouraging, and demonstrate that the prognosis of MVID has changed dramatically during the past decade.

Finally, in a child suspected of having MVID, a precise classification into one of three different subtypes (congenital-early-onset, late-onset or atypical) should be obtained as early as possible and confirmed by an experienced pathologist, according to clearly defined morphological criteria. Recently, Croft et al34 reported a 5-year-old girl with a late-onset form of MVID who was weaned from total PN and is thriving on a normal unrestricted diet. This rare form of MVID probably has a better prognosis than the most frequent form of early-onset MVID, with a high mortality rate.15 Given the high success rate of intestinal transplantation in MVID, we recommend transplantation, once the definitive diagnosis of early-onset MVID is made. In addition, the potential candidate for transplantation has to be in a eutrophic phase under total PN. This policy should allow avoidance of liver impairment. Conversely, patients with a late-onset or atypical form of MVID should not be automatically scheduled for small-bowel transplantation. The individual course of the disease will help in deciding whether or not a child with MVID is a candidate for intestinal transplantation.

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