Pediatric Transplantation最新文献

{"title":"Transcatheter Pulmonary Flow Restrictors as a Palliative Bridge to Heart Transplant.","authors":"Mats Steffi Jennifer Masilamani, Jonathan N Johnson, Rebecca K Ameduri, Elizabeth H Stephens, Allison K Cabalka, Jason H Anderson","doi":"10.1111/petr.70025","DOIUrl":"10.1111/petr.70025","url":null,"abstract":"<p><strong>Background: </strong>In recent years, transcatheter implantation devices to restrict pulmonary arterial flow have emerged as a potential alternative to surgical pulmonary artery banding.</p><p><strong>Case presentation: </strong>A term male was diagnosed with critical aortic stenosis (AS) and severely reduced left ventricle (LV) systolic function. He underwent aortic balloon valvuloplasty on day 2 of life, resulting in some antegrade flow, but LV ejection fraction only improved to 15%. He remained ductal dependent for systemic perfusion. Pulmonary over-circulation ensued with systemic steal evident in his second week of life. On day 14, he underwent a transcatheter stage 1 procedure including the placement of bilateral pulmonary flow restrictor (PFR) devices and ductal stenting. Post-procedure, he improved clinically, weaned off respiratory support, and transitioned to oral feeds while awaiting cardiac transplant. Twelve days later, he received a donor heart, with an uneventful recovery. He is now 3 years post-transplant and has excellent graft function, and his transplant course has been unremarkable.</p><p><strong>Conclusion: </strong>Transcatheter-based palliation for a neonate with critical AS, utilizing endoluminal PFRs, proved effective in stabilizing the infant and successfully bridging him to transplant. This highlights an opportunity for the utilization of PFRs in neonatal conditions where control of pulmonary blood flow is imperative as a bridge to transplantation.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":"29 1","pages":"e70025"},"PeriodicalIF":1.2,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143009808","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"CMV Reactivation Following Allogeneic Transplantation in Children From a High-Seroprevalence Population: A Single-Center Experience in Colombia.","authors":"Andres Arias, Natalia Builes, Laura Niño-Serna, Alejandro Diaz, Beatriz H Aristizabal, Monica Trujillo","doi":"10.1111/petr.70033","DOIUrl":"10.1111/petr.70033","url":null,"abstract":"<p><strong>Introduction: </strong>Cytomegalovirus (CMV) infection is a frequent complication among hematopoietic stem cell transplant (HSCT) recipients. Data regarding CMV reactivation in children in underdeveloped countries is scarce. This is especially notable considering the increasing utilization of haploidentical-related HSCT with the post-transplant cyclophosphamide platform. This study aimed to describe the incidence, clinical characteristics, and evolution of children with CMV reactivation after HSCT and the possible impact of unmanipulated stem cells with PTCy for GvHD prophylaxis.</p><p><strong>Methods: </strong>Retrospective cohort study of children undergoing hematopoietic stem cell transplantation from January 2012 to June 2022. Baseline characteristics and the clinical course were described. Duration of treatment, initial viral load, and time to clearance of DNAemia by type of transplant were compared using the Kruskal-Wallis test. Survival analysis was performed with the Kaplan-Meier method and log-rank test. All statistical analysis was performed using SPSS software, version 20.0.</p><p><strong>Results: </strong>One hundred sixty-six children were included. Among them, 87% of recipients and 88% of donors were CMV positive. The cumulative incidence of cytomegalovirus DNAemia was 28% at 100 days post-transplantation. There were no differences between different donor types. Overall survival at 1 year was 60%, and non-relapse mortality was observed in 28%. CMV reactivation did not appear to negatively impact 1-year overall survival (OS).</p><p><strong>Conclusions: </strong>Our study found no differences in CMV reactivation rates, treatment duration, viral clearance times, co-infections, or 1-year overall survival across different HSCT donor types. Studies are needed to establish more precise criteria for monitoring recipients, particularly in regions where unmanipulated stem cells with PTCy for GvHD prophylaxis are increasing.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":"29 1","pages":"e70033"},"PeriodicalIF":1.2,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143009770","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Risk Factors of Hypothermia During Pediatric Liver Transplantation Using a Novel Warming Method.","authors":"Taiga Ishihara, Chiaki Baba, Kaoru Ishizaki, Jun Ninagawa, Shugo Kasuya","doi":"10.1111/petr.14902","DOIUrl":"10.1111/petr.14902","url":null,"abstract":"<p><strong>Background: </strong>Intraoperative hypothermia often occurs in patients under general anesthesia, including liver transplantation (LT), leading to various life-threatening conditions. This study aimed to evaluate factors causing hypothermia in patients undergoing pediatric LT.</p><p><strong>Methods: </strong>Data were collected from patients undergoing LT who were warmed during surgery. The participants were divided into the hypothermia group, comprising patients with intraoperative hypothermia (< 35°C), and the normothermia group, comprising patients without hypothermia (≧ 35°C). These two groups were compared in terms of preoperative conditions (weight, height, Body mass index, age [months], and pediatric end-stage liver disease score), intraoperative findings (surgical duration, graft-to-recipient weight ratio, cold ischemic time, warm ischemic time, length of the an-hepatic period, volume of blood loss, and volume of blood transfusion), and postoperative findings (complications within 30 days, rejection within 30 days, and length of intensive care unit and hospital admission).</p><p><strong>Results: </strong>The hypothermia and normothermia groups differed in terms of age, weight, height, pediatric end-stage liver disease score, graft-to-recipient weight ratio, volume of blood loss, volume of blood transfusion (red cell concentrate and fresh frozen plasma), the duration of intensive care unit admission, and rejection within 30 days (all p < 0.05). After multivariate analysis, only the number of patients experiencing rejection was associated with intraoperative hypothermia (odds ratio = 32.553, 95%CI: 2.198-482.226, p = 0.011).</p><p><strong>Conclusions: </strong>Rejection was particularly related to intraoperative hypothermia.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":"29 1","pages":"e14902"},"PeriodicalIF":1.2,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142801993","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Impact of Hepatoblastoma on Infectious Complications Following Pediatric Liver Transplantation.","authors":"Ashton D Hall, Hope A Hendricks, Katherine A Bowers, James I Geller, Alexander J Bondoc, Greg M Tiao, Amy E Taylor, William R Otto, Grant C Paulsen, Lara A Danziger-Isakov","doi":"10.1111/petr.70035","DOIUrl":"10.1111/petr.70035","url":null,"abstract":"<p><strong>Background: </strong>Liver transplantation is the standard therapy for end-stage liver disease in pediatric patients with biliary atresia (BA), congenital and metabolic conditions, and for an unresectable malignant tumor like hepatoblastoma (HB). BA is the leading indication for pediatric liver transplantation, while HB is the most common childhood liver cancer. Despite improved outcomes through advanced surgical techniques and novel immunosuppression, pediatric liver transplantation (pLT) is complicated by post-transplant infections.</p><p><strong>Methods: </strong>A retrospective review was performed of pLT recipients at Cincinnati Children's Hospital Medical Center (CCHMC) and stratified patients by underlying disease to assess impact on post-transplant infectious events.</p><p><strong>Results: </strong>BA patients were youngest at pLT (12.5 months; p < 0.001) compared to other disease cohorts (HB 30.8, other 43.7). All HB patients received organs from deceased donors. In the year following pLT, 93% of the patients experienced at least one infectious event (IE). HB patients had the highest mean number of IE across disease groups (5.5 IE/patient vs. BA 4.5, other 4.0; p = 0.055), with significantly more patients with fever and neutropenia (p < 0.001) and EBV infections (p = 0.012). HB patients were more likely to develop IE earlier after pLT than non-HB groups (p = 0.013), especially Clostridioides difficile (p < 0.01) and fever and neutropenia (p < 0.01). Despite having variable IE experiences, 1-and-5-year survival across disease groups were similar.</p><p><strong>Conclusions: </strong>IE were frequently observed in HB patients after pLT, possibly related to pre-and-postoperative chemotherapy and associated neutropenia. Underlying disease may help inform targeted infection-related patient management following pLT.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":"29 1","pages":"e70035"},"PeriodicalIF":1.2,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11771635/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143047310","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Decisions Using Intra-Operative Gross Assessment of Fontan-Associated Liver Disease: Implications for Heart-Only Transplantation.","authors":"Diana Milagros Torpoco Rivera, Ke-You Zhang, Noelle Ebel, Rachel Bensen, Elisabeth Martin, Michael Ma, Carlos O Esquivel, Manchula Navaratnam, Seth A Hollander, David N Rosenthal, Sharon Chen","doi":"10.1111/petr.70036","DOIUrl":"https://doi.org/10.1111/petr.70036","url":null,"abstract":"<p><strong>Introduction: </strong>Intraoperative gross liver inspection at the time of transplant can be used in determination of which failing Fontan patients should undergo heart-only transplantation (HT) versus combined heart-liver transplantation (CHLT). We describe the outcomes of Fontan patients listed for CHLT who underwent HT based on the gross pathology of the liver at time of transplant.</p><p><strong>Case report: </strong>Six Fontan patients, ages 15 (IQR12,16) years, listed for CHLT received HT because of reassuring gross findings of the liver at the time of transplant. All patients survived to hospital discharge, after median hospital length of stay of 59 (IQR 42-77) days. Vasoplegia, prolonged need for milrinone because of diastolic dysfunction, acute kidney injury, and infection were common complications. Five patients had acute cellular rejection within 35 days of heart transplant. At a median of 17 (IQR 10, 19) months from HT, all continue to have abnormal findings by abdominal imaging, with development of new hepatic nodules in three patients.</p><p><strong>Summary: </strong>Intraoperative gross liver inspection at the time of transplant can enable successful HT in Fontan patients with liver disease. However, a prolonged recovery period should be anticipated, with serial post-HT liver assessments and vigilant monitoring for rejection. The long-term liver prognosis remains uncertain.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":"29 1","pages":"e70036"},"PeriodicalIF":1.2,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144040204","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pediatric Age-Prioritized Waitlisting Policy Potentially Disadvantages Adolescents on Dialysis Not Listed for Transplant Until Adulthood.","authors":"Justin A Steggerda, Steven A Wisel, Dechu Puliyanda, Helen Pizzo, Jonathan Garrison, Irene K Kim","doi":"10.1111/petr.70023","DOIUrl":"10.1111/petr.70023","url":null,"abstract":"<p><strong>Background: </strong>Current kidney transplant (KT) policies offer advantages in waiting time and organ allocation priority to pediatric patients waitlisted before 18 years old. This study evaluates the effects of this policy for patients who are on dialysis before, but not waitlisted until after, age 18.</p><p><strong>Methods: </strong>Patients aged 11-25 years and waitlisted between 2001 and 2022 for KT were identified in the OPTN STAR data file for analysis. Cohorts were defined by age and dialysis status at time of listing: Peds if < 18 yo, young adult (YA) if ≥ 18 yo; NYOD-not yet on dialysis or OD-on dialysis at time of listing, with YA groups further segregated by age at dialysis initiation. Cumulative incidence of transplant was calculated for waitlisted patients. Graft survival was assessed using Kaplan-Meier analysis and multivariable Cox proportional hazards modeling. p values < 0.01 were significant.</p><p><strong>Results: </strong>Amongst 35 764 KT registrations, candidates who initiated dialysis as pediatric patients but were not waitlisted until after turning 18 years old (YA + OD < 18) have the highest rate of nontransplantation (33.5%) and longest time on dialysis (median: 2103 days) before deceased donor (DD) KT. YA + OD < 18 patients were sixfold less likely than Peds + OD patients to undergo DDKT at 5 years after listing. YA + OD < 18 recipients had the worst post-KT graft survival of all groups at 5 years with adjusted hazard ratio of 1.477 (95% confidence interval: 1.218-1.792) compared to Peds-NYOD (p < 0.001).</p><p><strong>Conclusions: </strong>Current allocation policies significantly disadvantage candidates who initiate dialysis before, but are not listed until after age 18, and should be re-examined to address these inequities.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":"29 1","pages":"e70023"},"PeriodicalIF":1.2,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143009753","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Langerhans Cell Histiocytosis or Acute Cellular Rejection?","authors":"Andreas Entenmann, Hubert Kogler, Wolf-Dietrich Huber, Marita Kölz, A S Knisely, Kristijan Skok","doi":"10.1111/petr.14884","DOIUrl":"10.1111/petr.14884","url":null,"abstract":"<p><strong>Background: </strong>Langerhans cell histiocytosis (LCH) is a rare malignant disorder of epidermal antigen presenting cells. It is characterized by infiltration of various tissues with dendritic cells (Langerhans cells, LC) that express CD1a or CD207 (langerin), often leading to organ dysfunction. A patient with LCH required liver transplantation (LT) for LCH-associated biliary-tract disease. Cholangiopathy developed after LT. The question arose: In this patient, did LC in damaged liver-allograft biliary epithelium signify acute cellular rejection (ACR) or recurrent LCH?</p><p><strong>Methods: </strong>We evaluated immunohistochemical identification of LC (CD1a, CD207) in the proposita and in 14 ACR patient samples as distinguishing between ACR and recurrent LCH.</p><p><strong>Results: </strong>Among 15 patient samples, 3 (20%) marked with neither antibody. Among the remaining 12 samples (80%), 4 (26.7%)-including that from the proposita-had cells marking for both antigens within bile-duct epithelium as well as in surrounding portal-tract connective tissue, 2 (13.3%) had cells marking for both antigens in one region or the other, but not in both, and 6 (40%) had cells marking for only one antigen in one region or the other.</p><p><strong>Conclusions: </strong>Immunostaining for CD1a and CD207/langerin in the setting of ACR without suspicion of LCH identifies LC in damaged bile ducts. This biomarker pairing proved not to be LCH-specific. Our findings indicate that the presence of these cells alone is insufficient to identify recurrent LCH in the allograft liver.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":"28 8","pages":"e14884"},"PeriodicalIF":1.2,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142576507","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pediatric Cardiac Xenotransplantation and Expanded Access: Ethical Considerations.","authors":"Daniel J Hurst, Christopher Bobier, Anthony Merlocco, Luz A Padilla, Daniel Rodger, David Cleveland, John D Cleveland","doi":"10.1111/petr.14876","DOIUrl":"10.1111/petr.14876","url":null,"abstract":"<p><p>Due to the current organ shortage waitlist, alternatives to allotransplantation are necessary. Xenotransplantation is currently being pursued as one such alternative in adults in need of kidney or heart transplantation. Cardiac xenotransplantation of genetically modified pig hearts has been conducted twice in adults under the United States Food and Drug Administration (FDA) expanded access criteria. Because of the shortage of transplantable hearts for children as well as the lack of mechanical circulatory support in this population, pediatric researchers are exploring FDA expanded access in high-risk neonates and infants who lack alternative options for survival. The adult cardiac xenotransplantation experience with expanded access can provide lessons and highlight nuances for researchers preparing pediatric application. This includes aspects of informed consent, biosurveillance, and protection of bystanders from potential xenozoonoses.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":"28 7","pages":"e14876"},"PeriodicalIF":1.2,"publicationDate":"2024-11-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142472355","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Chronotropic Response to Exercise is Decreased in Patients With Congenital Heart Disease Compared to Cardiomyopathy Following Pediatric Heart Transplantation","authors":"Sebastian Otto‐Meyer, Alan P. Wang, Garett J. Griffith, Katheryn Gambetta, Kendra Ward","doi":"10.1111/petr.14856","DOIUrl":"https://doi.org/10.1111/petr.14856","url":null,"abstract":"BackgroundTwo common indications for pediatric heart transplantation are congenital heart disease and cardiomyopathy. Prior studies suggest differences in chronotropy on cardiopulmonary exercise testing outcomes depending on indication for heart transplantation. We aimed to determine whether the number of pretransplant sternotomies is associated with differences in heart rate response during exercise testing.MethodsA retrospective analysis of our institutional pediatric heart transplant data between 2004 and 2022 was performed. Patients were categorized by indication for transplantation into a cardiomyopathy (CM) group if they had a congenital or acquired cardiomyopathy or a congenital heart disease (CHD) group including all other forms of congenital cardiac anatomic abnormalities.ResultsCHD patients (<jats:italic>n</jats:italic> = 40) differed from CM patients (<jats:italic>n</jats:italic> = 53) by mean number of sternotomies prior to transplant (2.4 ± 1.8 vs. 0.5 ± 0.9, <jats:italic>p</jats:italic> &lt; 0.001). There were no significant differences in echocardiographic function or catheterization hemodynamics. In cardiopulmonary exercise testing performance, the congenital heart disease group had a significantly higher resting heart rate (91.8 ± 11.2 vs. 86.4 ± 10.2 bpm, <jats:italic>p</jats:italic> = 0.019), lower percent predicted age‐predicted maximal heart rate achieved (78.3 ± 8.5% vs. 83.2 ± 11.4%, <jats:italic>p</jats:italic> = 0.032), and lower heart rate reserve (68.6 ± 19.8 vs. 84.4 ± 24.0 bpm, <jats:italic>p</jats:italic> = 0.001) despite a similar age and average time from transplantation. Regression analysis confirmed number of pretransplant sternotomies as a main predictor of heart rate metrics.ConclusionsThere is greater chronotropic incompetence in patients who underwent transplantation due to congenital heart disease compared to cardiomyopathy. The groups differ significantly by number of sternotomies, potentially supporting the hypothesis that prior surgical disruption of cardiac innervation may cause decreased chronotropic response to exercise following transplantation.","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":"41 1","pages":""},"PeriodicalIF":1.3,"publicationDate":"2024-09-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142259770","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Long-Term Outcomes of Living Donor Liver Transplantation for Methylmalonic Acidemia.","authors":"Robert C Minnee, Seisuke Sakamoto, Akinari Fukuda, Hajime Uchida, Kazuya Hirukawa, Masaki Honda, Shinya Okumura, Takashi Ito, Tonguç U Yilmaz, Yitian Fang, Toru Ikegami, Kwang W Lee, Mureo Kasahara","doi":"10.1111/petr.14834","DOIUrl":"10.1111/petr.14834","url":null,"abstract":"<p><strong>Background: </strong>Despite early diagnosis and medical interventions, patients with methylmalonic acidemia (MMA) suffer from multi-organ damage and recurrent metabolic decompensations.</p><p><strong>Methods: </strong>We conducted the largest retrospective multi-center cohort study so far, involving five transplant centers (NCCHD, KUH, KUHP, ATAK, and EMC), and identified all MMA patients (n = 38) undergoing LDLT in the past two decades. Our primary outcome was patient survival, and secondary outcomes included death-censored graft survival and posttransplant complications.</p><p><strong>Results: </strong>The overall 10-year patient survival and death-censored graft survival rates were 92% and 97%, respectively. Patients who underwent LDLT within 2 years of MMA onset showed significantly higher 10-year patient survival compared to those with an interval more than 2 years (100% vs. 81%, p = 0.038), although the death-censored graft survival were not statistically different (100% vs. 93%, p = 0.22). Over the long-term follow-up, 14 patients (37%) experienced intellectual disability, while two patients developed neurological complications, three patients experienced renal dysfunction, and one patient had biliary anastomotic stricture. The MMA level significantly decreased from 2218.5 mmol/L preoperative to 307.5 mmol/L postoperative (p = 0.038).</p><p><strong>Conclusions: </strong>LDLT achieves favorable long-term patient and graft survival outcomes for MMA patients. While not resulting in complete cure, our findings support the consideration of early LDLT within 2 years of disease onset. This approach holds the potential to mitigate recurrent metabolic decompensations, and preserve the long-term renal function.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":"28 6","pages":"e14834"},"PeriodicalIF":1.2,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141889973","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}