Pediatric Transplantation最新文献

Background: In order to reduce the waiting time, new strategies have been developed to safely transplant donor and recipient pairs with mismatched blood groups. The present study examined the safety of the preparatory treatments and the midterm outcome of AB0 incompatible (AB0i) kidney transplantation in children.

Methods: We retrospectively analyzed 10 children who received a kidney transplant from an AB0i donor from 2012 to 2024 and 30 patients matched by sex, height, and weight who received an AB0 compatible (AB0c) living kidney transplant in the same period.

Results: In the AB0i group, preparatory treatment before KTx was well tolerated. The number of rejection episodes was comparable in the AB0i group and in the AB0c group (three episodes in three patients vs. seven episodes in six patients) during the observation period of 36 months, with rejections occurring earlier in the AB0c group. Infections were more frequent in the AB0c group than in the AB0i group (30 episodes in 23 patients [76%] vs. eight episodes in six patients [60%]). In the AB0i group, the 3-year graft survival rate was 90%; the 3-year patient survival was 100%. In the AB0c group, the 3-year graft survival was 86.8%, and the 3-year patient survival was 93.3%. There was no difference in graft survival (p = 0.08) and patient survival (p = 0.24) between the AB0i and AB0c groups.

Conclusions: AB0 incompatible kidney transplants can be safely performed in children with equivalent midterm graft and patient survival.

Intestinal and multivisceral transplantation has evolved from an experimental to a life-saving procedure for children and adults with complications of gut failure (GF). Suboptimal long-term outcomes of transplant elicit recent advances in surgical and medical gut rehabilitation along with the introduction of glucagon-like peptide-2 (GLP-2) that has established a new paradigm achieving nutritional autonomy reserving transplantation for rescue. Notably, continued improvement of transplant has been observed over the last decade. The 2025 International Intestinal Transplant Registry (IITR) report demonstrates continued era-based improvement, with overall 5-year patient survival of 60% and graft survival of 51% for pediatric recipients, and 52% and 46%, respectively, for adult recipients. Over 90% of long-term transplant survivors achieve full nutritional autonomy. Contemporary cohorts show substantially better outcomes, reflecting advances in surgical technique and immunosuppression. Key developments include refined indications, sophisticated surgical techniques for complex anatomy, and updated immunosuppressive protocols. The integration of composite tissue transplantation, particularly abdominal wall allografts, vascularized and non-vascularized rectus sheath fascia has addressed closure challenges. Despite advances in transplantation, chronic rejection remains the primary barrier to long-term graft survival. Future directions include technological innovations in organ preservation, machine perfusion and organoid transplantation.

Background: Phosphomannomutase-2 congenital disorder of glycosylation (PMM2-CDG) is the most common congenital disorder of glycosylation, affecting protein glycosylation across multiple organ systems. Hepatic involvement may range from isolated elevations in liver transaminases to end-stage liver disease. Reported outcomes of liver transplantation as a treatment modality are sparse.

Case report: We describe one of the first reported cases of liver transplantation in a child with PMM2-CDG and interim post-transplant outcomes. This patient was diagnosed at 4 months of age after presenting with failure to thrive, lipodystrophy, hypotonia, developmental delay, elevated transaminases, hypoalbuminemia, and coagulopathy. He developed cirrhosis and portal hypertension as well as sequelae of poor protein glycosylation. All these included coagulopathy, protein-losing enteropathy, and refractory ascites requiring serial intravenous fresh frozen plasma and furosemide. He ultimately underwent a liver transplant, after which his ascites resolved. Post-transplant, he developed new-onset recurrent pericardial effusions, suspected to be from a viral etiology versus extrahepatic manifestations of PMM2-CDG, and elevated transaminases following transplantation.

Discussion/conclusions: Liver transplantation may offer clinical benefit in PMM2-CDG with severe hepatic involvement, including resolution of ascites and improved quality of life, due to its potential to restore liver glycosylation function. However, this is only a partial correction as persistent extrahepatic manifestations underscore the need for further research into transplant outcomes and systemic disease progression in CDG.

Pulmonary hypertension (PH) is a potentially life-threatening disorder characterized by abnormalities of the pulmonary vasculature, causing elevated pulmonary artery pressures, which can result in right ventricular dysfunction. Patients with suprasystemic right ventricular pressure unresponsive to aggressive medical therapy have limited treatment options, including balloon atrial septostomy, reverse Potts shunt, or lung transplantation. The decision to proceed to a palliative reverse Potts shunt or lung transplantation, and the choice of one or the other, depends on surgical, medical, and psychosocial factors. In this manuscript, we discuss the definition, pathophysiology, and current treatments for PH in relation to pediatric lung transplantation, including referral indications and special considerations for transplant candidates. We review the reverse Potts shunt as a palliative option for patients with severe PH, discussing indications, patient outcomes, surgical techniques, and the relative risks/benefits compared to lung transplantation. Finally, we propose an algorithm to assist pediatric cardiologists, pulmonologists, intensivists, surgeons, and other healthcare providers in the decision-making between a palliative reverse Potts shunt procedure and lung transplantation for patients with severe PH based on currently available data.

Background: Hemophagocytic lymphohistiocytosis (HLH) is a rare complication of solid organ transplantation and is a syndrome of systemic hyperinflammation secondary to dysregulation of the inflammatory response, primarily involving lymphocytes and macrophages. It is often fatal and therefore early recognition and treatment are crucial. Among 11 adult cases of HLH in post-lung transplant cases found in the literature, only one patient survived.

Case: We report the first known pediatric case of HLH following lung transplantation. The patient, a previously healthy adolescent, developed end-stage bullous lung disease secondary to acute respiratory distress syndrome (ARDS) and underwent bilateral lung transplantation. Two months posttransplant, he was admitted with an asymptomatic febrile illness of unclear etiology. By day four, evolving multiorgan dysfunction raised concern for HLH. Despite extensive infectious, autoimmune, and malignancy workups, no definitive trigger was identified. Treatment was initiated with dexamethasone monotherapy with subsequent clinical improvement and discharge 1 month later.

Conclusion: Solid organ transplantation appears to raise a patient's risk of developing HLH, although the underlying mechanisms are unclear. Literature review suggests patients are most likely to develop this complication within the first few months of transplantation, and a high index of suspicion must be maintained in those who present with a febrile illness of unclear etiology. Standard HLH treatment protocols may not be applicable to this patient population, and further studies are needed.

Solid organ transplantation (SOT) is a life-saving intervention for pediatric patients with end-stage organ failure. Due to the limited availability of pediatric donor organs, organs from older donors are frequently utilized, increasing the risk of age-mismatched transplants. Older donor organs are linked to heightened immunogenicity, rejection rates, and impaired long-term outcomes. Emerging evidence suggests that aged donor organs may transfer senescence to pediatric recipients, accelerating aging-like processes such as frailty, cognitive decline, and organ dysfunction. Additionally, the induction of senescence could alter pediatric conditions like chronic kidney disease (CKD), juvenile idiopathic arthritis (JIA), and pediatric brain tumors which have been linked to augmented senescence. Animal models have shown that older donor organs induce senescence-associated changes in young recipients, including immune dysfunction and physical and cognitive impairments. This review highlights the role of cellular senescence in pediatric organ transplantation and discusses strategies to mitigate its impact. Therapies targeting senescence, such as senolytics, offer a potential approach to improve outcomes in pediatric recipients. Further research is needed to validate these findings in human studies and guide clinical strategies that expand the donor pool while prioritizing age-matched transplantation for pediatric patients.

Background: Clostridioides difficile infection (CDI) poses a significant risk to pediatric hematopoietic stem cell transplant (HSCT) due to microbiome disruption, mucosal injury, and graft versus host disease (GVHD). While oral vancomycin prophylaxis (OVP) is effective for preventing recurrent CDI, evidence for its role in preventing initial infection is limited. Our institution employs empiric OVP during the first HSCT admission to prevent initial CDI.

Objectives: We sought to describe the incidence of CDI among pediatric HSCT recipients receiving OVP and to evaluate secondary outcomes related to OVP exposure.

Methods: We conducted a single center, retrospective observational study of 84 pediatric HSCT recipients at our institution receiving OVP during their initial transplant admission. Chart review captured demographics, transplant information, and clinical outcomes. The primary outcome was CDI incidence during hospitalization. Secondary outcomes included VRE infections, refractory CDI following cessation of OVP, and acute GI GVHD.

Results: Only one patient developed CDI (1.19%) while on OVP, despite universal exposure to high-risk antibiotics among the entire cohort. No VRE infections were observed. Rates of GI aGVHD were consistent with national averages. Nine patients (10.7%) developed CDI after discontinuing OVP, all managed with standard treatment.

Conclusion: Empiric OVP during pediatric HSCT hospitalization was associated with a markedly low CDI incidence. Despite theoretical risks of microbiome disruption, no adverse effects were identified in this cohort, including long-term follow-up beyond 5 years. These findings support the safety and potential efficacy of OVP as primary CDI prophylaxis in pediatric HSCT patients.

Background: Monoclonal antibodies, including rituximab and daratumumab, play a pivotal role in the management of antibody-mediated rejection (AMR) following solid organ transplantation. Although generally effective, these agents can induce rare but potentially fatal complications.

Methods: We present two cases of post-transplant AMR in which patients developed fatal complications following the administration of rituximab and daratumumab. Both cases were analyzed in detail to assess the sequence of clinical events and potential underlying mechanisms.

Results: Both patients developed severe flash pulmonary edema shortly after receiving the monoclonal antibody therapy. This was followed by rapid clinical deterioration, including multiorgan failure. The clinical features and biopsy findings were consistent with cytokine release syndrome as the probable trigger for these complications.

Conclusion: Early recognition, prompt discontinuation of the offending agent, and targeted therapeutic interventions are crucial to improving outcomes in these life-threatening scenarios.