Over 27 cases of liver transplant, kidney transplant and combined liverCkidney

Over 27 cases of liver transplant, kidney transplant and combined liverCkidney transplant have already been reported for the treatment of methylmalonic aciduria. and urine methylmalonic acid levels. The amount of natural protein (30% of total protein) remains much like pre-transplant dietary regimen. Importantly, the post-transplant decompensation and recorded low bicarbonate levels experienced by our patient proved that despite the presence of normal enzyme activity in both liver and kidney, he was still susceptible to metabolic derangements under conditions of stress. To date, the criteria for solid organ transplantation in MMA have not been well-established. The decision Rabbit Polyclonal to HEXIM1 to undertake a transplantation is usually a complicated one and involves: (1) a comprehensive understanding of the disease and the risks and benefits of transplantation; (2) factor from the organic history of the condition, current healing alternatives, potential potential developments, and standard of living [38]. The function of transplantation in MMA was attended to with a workshop at a global reaching on inborn mistakes of fat burning capacity [22]. GSK2656157 manufacture It had been concluded that kids with organic acidemias seem to GSK2656157 manufacture be at higher threat of problems from transplantation than various other metabolic disorders. While standard of living may be improved, transplantation does not remedy patients who remain at risk for complications. To develop clinical guidelines, a registry of all MMA patients who have been or are being considered for transplantation GSK2656157 manufacture has been suggested [22]. GSK2656157 manufacture The clinical decision-making process in our case involved a multidisciplinary conversation regarding combined liverCkidney transplantation. A splenectomy was proposed in addition to CLKT to further decrease methylmalonate levels due to the ubiquitous nature of the enzyme. The risk of contamination with encapsulated organisms was thought to contraindicate splenectomy. Although, CKLT in our patient was not curative and experienced a post-operative clinical course similar to that explained in previous reports [19,23,32,37], we conclude that the patient has benefited from an improved quality of life based on the dramatic decrease in time spent in hospital or in chronic care facilities during recovery from decompensations. Besides the single episode of iatrogenic decompensation, he has not been hospitalized for metabolic acidosis since the transplantation, reflecting the beneficial effect of the CKLT. Based on our difficulty in deciding on the proper course of action for this patient, given the suboptimal clinical details and outcomes explained in the literature, we believe that a registry of transplantation candidates in keeping with the recommendations of the Workshop: Management of Organic Acidemias and the establishment of guidelines regarding solid organ transplantation in organic acidurias by a multinational collaborative group would be of great benefit to clinicians who will need to decide on the relative benefits of such an intervention for future patients. Acknowledgments The authors thank Drs. Edwin Kirk and Felicity Collins for their contribution to this manuscript..