Transplantation in paediatric patients with MMA requires multidisciplinary approach for achievement of good clinical outcomes

• Published in: Pediatric nephrology (Berlin, West) Vol. 38; no. 8; pp. 2887 - 2896
• Main Authors: Paessler, Alicia, Cortes-Cerisuelo, Miriam, Jassem, Wayel, Vilca-Melendez, Hector, Deep, Akash, Jain, Vandana, Pool, Andrew, Grunewald, Stephanie, Kessaris, Nicos, Stojanovic, Jelena
• Format: Journal Article
• Language: English
• Published: Berlin/Heidelberg Springer Berlin Heidelberg 01.08.2023; Springer; Springer Nature B.V
• Subjects: Abdomen; Amino Acid Metabolism, Inborn Errors - complications; Child; Child, Preschool; Children; Clinical outcomes; Complications and side effects; Disease management; Fascia; Health aspects; Humans; Kidney diseases; Kidney Failure, Chronic - complications; Kidney transplantation; Kidney Transplantation - adverse effects; Kidney Transplantation - methods; Kidney transplants; Literature reviews; Liver; Liver transplantation; Liver Transplantation - adverse effects; Liver Transplantation - methods; Liver transplants; Management Dilemma; Medicine; Medicine & Public Health; Metabolism; Nephrology; Patient outcomes; Patients; Pediatrics; Sepsis; Toxicity; Transplantation of organs, tissues, etc; Urology; What’s New in Renal Transplantation; Williams syndrome; United Kingdom; Abdominal wall transplantation; Paediatrics; Methylmalonic acidaemia; Kidney transplantation; Liver transplantation; Williams syndrome
• ISSN: 0931-041X; 1432-198X; 1432-198X
• DOI: 10.1007/s00467-023-05906-0

Background As modern medicine is advancing, younger, small, and more complex children are becoming multi-organ transplant candidates. This brings up new challenges in all aspects of their care. Methods We describe the first report of a small child receiving a simultaneous liver and kidney transplant and abdominal rectus sheath fascia transplant on the background of Williams syndrome and methylmalonic acidaemia. At the time of transplantation, the child was 3 years old, weighed 14.0 kg, had chronic kidney disease stage V, and had not yet started any other form of kidney replacement therapy. Results There were many anaesthetic, medical, metabolic, and surgical challenges to consider in this case. A long general anaesthetic time increased the risk of cardiac complications and metabolic decompensation. Additionally, the small size of the patient and the organ size mis-match meant that primary abdominal closure was not possible. The patient’s recovery was further complicated by sepsis, transient CNI toxicity, and de novo DSAs. Conclusions Through a multidisciplinary approach between 9 specialties in 4 hospitals across England and Wales, and detailed pre-operative planning, a good outcome was achieved for this child. An hour by hour management protocol was drafted to facilitate transplant and included five domains: 1. management at the time of organ offer; 2. before the admission; 3. at admission and before theatre time; 4. intra-operative management; and 5. post-operative management in the first 24 h. Importantly, gaining a clear and in depth understanding of the metabolic state of the patient pre- and peri-operatively was crucial in avoiding metabolic decompensation. Furthermore, an abdominal rectus sheath fascia transplant was required to achieve abdominal closure, which to our knowledge, had never been done before for this indication. Using our experience of this complex case, as well as our experience in transplanting other children with MMA, and through a literature review, we propose a new perioperative management pathway for this complex cohort of transplant recipients.