A case of liver transplantation for HCC complicating BA in an eight-month old infant is reported. HCC in BA is extremely rare. Screening of AFP and ultrasonographic examination should be performed regularly in patients with secondary biliary cirrhosis for early detection of HCC.

With conventional dietary treatment, the clinical course of methylmalonic acidemia due to cobalamin-unresponsive methylmalonyl-CoA mutase (MCM) deficiency is characterized by the persistent risk of recurrent life-threatening decompensation episodes with metabolic acidosis, hyperammonemia, and coma. Liver transplant has been proposed as an alternative treatment and anecdotally attempted in the last 2 decades with inconsistent results. Most criticisms of this approach have been directed at the continuing risk of neurologic and renal damage after transplant. Here, we report the perioperative and postoperative clinical and biochemical outcomes of 2 patients with severe MCM deficiency who underwent early liver transplant. In both cases, liver transplant allowed prevention of decompensation episodes, normalization of dietary protein intake, and a marked improvement of quality of life. No serious complications have been observed at 12 years' and 2 years' follow-up, respectively, except for mild kidney function impairment in the older patient. On the basis of our experience, we strongly suggest that liver transplant should be offered as a therapeutic option for children with cobalamin-unresponsive MCM deficiency at an early stage of the disease.

Summary In this study, the epidemiology and outcome of graft loss following primary pediatric liver transplantation (LT) were analysed, with the hypothesis that early retransplantation (reLT) might be associated with lower immunologic risks when compared with late reLT. Between March 1984 and December 2005, 745 liver grafts were transplanted to 638 children at Saint‐Luc University Hospital, Brussels. Among them, a total of 90 children (14%) underwent 107 reLT, and were categorized into two groups (early reLT, n = 58; late reLT, n = 32), according to the interval between either transplant procedures (< or >30 days). Ten‐year patient survival rate was 85% in recipients with a single LT, vs. 61% in recipients requiring reLT (P < 0.001). Ten‐year patient survival rates were 59% and 66% for early and late reLT, respectively (P = 0.423), the corresponding graft survival rates being 51% and 63% (P = 0.231). Along the successive eras, the rate of reLT decreased from 17% to 10%, whereas progressive improvement of outcome post‐reLT was observed. No recurrence of chronic rejection (CR) was observed after reLT for CR (0 of 19). Two children developed a positive cross‐match at reLT (two of 10, 20%), both retransplanted lately for CR secondary to immunosuppression withdrawal following a post‐transplant lymphoproliferative disease. In summary, the results presented could not evidence better results for late reLT when compared with early reLT. The former did not seem to be associated with higher immunologic risk, except for children having withdrawal of immunosuppression following the first graft.

Liver transplantation in severe methylmalonic acidemia: The sooner, the better To the Editor:We read with interest the article by Niemi et al 1 about liver or combined liver-kidney transplantation in methylmalonic acidemia (MMA). The implications of the encouraging results of the transplant approach adopted by the Stanford group in children with MMA are analyzed and commented by Sloan et al in an accompanying editorial. 2 The authors' main statement is that liver or liver-kidney transplantation in MMA, although not curative, result in excellent long-term survival and stabilization of the neurocognitive prospect. Open questions concern three issues: (1) who should be transplanted; (2) when transplantation should be performed; and (3) which kind of approach (isolated liver or combined liver-kidney transplantation) should be proposed.Concomitant with these interesting papers, we published independently our experience in liver transplantation for neonatal-onset, cobalamin-unresponsive MMA, providing new arguments for answering those questions. 3 First, given the dismal natural course of the disease in spite of rigorous medical management, we are persuaded that the organ transplant approach should be considered in all patients with neonatal-onset, cobalamin-unresponsive MMA. In our patients, timely liver transplantation allowed an excellent clinical course both perioperatively and in the long-term, consistent with the results by Niemi et al. Second, we are convinced that transplantation should be performed early, ideally within the first year of life, thereby avoiding repeated metabolic decompensations and the consequent neurologic deterioration. Third, we believe that isolated liver transplantation should be the preferred option, as it provides the greatest mutase enzyme activity (5-fold greater than kidney transplantation), sufficient for preserving renal function and avoiding, or at least delaying, the need for kidney transplantation.In light of these independent reports, early liver transplantation currently appears to be the best therapeutic option in patients with severe MMA. Although not fully curative, this approach will ensure healthier clinical outcome with respect to traditional medical management.

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