Pediatric Transplantation最新文献

{"title":"Pediatric Multiorgan Lung Transplantation: Four Decades of National Trends and Outcomes.","authors":"Carl Britto, Ali Demir, Eleanor D Muise, J Brian McAlvin, Don Hayes, Levent Midyat","doi":"10.1111/petr.70466","DOIUrl":"10.1111/petr.70466","url":null,"abstract":"<p><strong>Background: </strong>Pediatric lung transplantation (LTx) and multiorgan lung transplantation (MOLT) remain uncommon, and national data describing temporal trends and outcomes are limited. We characterized four decades of MOLT in the United States, with particular emphasis on the evolving pediatric experience.</p><p><strong>Methods: </strong>We analyzed Organ Procurement and Transplantation Network Standard Transplant Analysis and Research files provided by the United Network for Organ Sharing from 1987 to 2024. The cohort included 54 797 isolated LTx recipients and 1 963 MOLT recipients, classified into mutually exclusive groups of dual-organ lung-heart (n = 1557), lung-liver (n = 216), lung-kidney (n = 154), and other multiorgan configurations (n = 36). Cox regression, Fine-Gray competing-risks, landmark, and interaction analyses evaluated graft-failure associations across four transplant eras and seven age groups.</p><p><strong>Results: </strong>Pediatric transplant volume declined substantially, comprising 0.8% of isolated LTx procedures in 2020-2024. Lung-heart transplantation decreased from 9.0% of lung-involved procedures before 2000 to 1.8% in 2020-2024, whereas lung-liver and lung-kidney transplantation increased modestly, predominantly among adults. Pretransplant ECMO use increased across cohorts, reaching 27.6% among lung-heart and 27.8% among lung-kidney recipients in 2020-2024. One-year graft survival improved progressively across eras.</p><p><strong>Conclusions: </strong>Pediatric MOLT has evolved within a changing national lung-transplant landscape characterized by declining pediatric volume, shifting procedural patterns, increasing recipient acuity, and improving early graft outcomes. Continued evaluation is needed as long-term follow-up from recent eras matures.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":"30 10","pages":"e70466"},"PeriodicalIF":1.4,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13673004/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149196160","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":"Routine Central Venous Catheter Maintenance After Pediatric Hematopoietic Stem Cell Transplant: Direct Cost Observations From a Single Center and Practice Variation Across Engraft Centers.","authors":"Steffani Maier, Christopher E Dandoy, Courtney Culbertson, Taylor Fitch, Nicholas Fry, Miza Salim Hammoud, John A Craddock, John Huber, Georgia Kent, Kasiani C Myers, Aivy Nguyen, Miki Nishitani, Megan Sampson, Riley Stuckey, Anne Wofford, Malika Kapadia","doi":"10.1111/petr.70493","DOIUrl":"https://doi.org/10.1111/petr.70493","url":null,"abstract":"<p><strong>Background: </strong>Central venous catheters (CVCs) are essential during pediatric hematopoietic stem cell transplantation (HSCT), but prolonged retention exposes patients to infection, thrombosis, device complications, caregiver burden, and ongoing maintenance costs. Routine maintenance costs and variation in posttransplant CVC care have not been well characterized.</p><p><strong>Methods: </strong>We conducted a descriptive mixed-methods study combining direct observation of CVC maintenance at Cincinnati Children's Hospital Medical Center with a cross-sectional survey of Engraft Learning Health Network centers. Between January and March 2025, 15 dressing changes and 10 cap changes, predominantly on double-lumen tunneled CVCs, were observed. Procedure time, staffing, supplies, and local labor and supply costs were evaluated using itemized micro-costing. Fifteen respondents representing 10 Engraft centers reported routine dressing- and cap-change practices.</p><p><strong>Results: </strong>Dressing-change practices were consistent, with all centers reporting changes every 7 days or sooner when dressing integrity was compromised. Cap-change intervals varied from daily to every 7 days. Dressing changes required a mean of 16.5 min and cost $55.25 in labor and supplies. Cap changes cost $24.08, and daily flushing cost $13.70. Estimated routine maintenance costs were $675-$728 per patient-month.</p><p><strong>Conclusion: </strong>Routine CVC maintenance after pediatric HSCT is resource intensive and varies across centers. These findings support standardization of maintenance practices and timely CVC removal when central access is no longer clinically necessary.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":"30 10","pages":"e70493"},"PeriodicalIF":1.4,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149149209","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":"Ethical Challenges in Pediatric Liver Transplant Selection Related to Patient and Caregiver Social and Psychosocial Factors: A National Survey of Clinician Perspectives.","authors":"Danielle G Rabinowitz, Eliza J Lee, Christy L Cummings, Jenna Simes, Scott A Elisofon, Andrew Wehrman, Katherine Sweeny, Christine K Lee, Stacy A Kahn","doi":"10.1111/petr.70471","DOIUrl":"https://doi.org/10.1111/petr.70471","url":null,"abstract":"<p><strong>Background: </strong>Disparities exist in pediatric liver transplantation in the United States. Clinician perspectives on how patient and family social and psychosocial factors affect patient selection remain unknown.</p><p><strong>Methods: </strong>A national, cross-sectional, mixed-methods survey was distributed to pediatric liver transplant committee members (June-November 2024). The survey assessed respondent and center characteristics, voting/conflict resolution practices, perceived influence of professional roles, social and psychosocial listing challenges, and moral distress. Quantitative analysis included descriptive statistics and multivariable linear/logistic regression; free-text responses underwent thematic analysis.</p><p><strong>Results: </strong>Among 162 eligible complete surveys, respondents represented multiple committee roles and sociodemographic backgrounds. Listing decisions were typically made verbally, with conflicts resolved through discussion, ethics consultation, or transplant surgeon tie-breaker vote. Professional role was strongly associated with perceived strength of voice, with physicians reporting substantially greater voice than non-physicians. Respondents reported moderate moral distress related to listing decisions involving social and psychosocial concerns. In adjusted models, social workers/psychologists and women reported higher moral distress ratings; social workers/psychologists also had increased odds of greater moral distress frequency, although confidence intervals were wide. Key social concerns included patient/caregiver non-adherence, mental health, housing instability, and insurance. Qualitative data identified that social and psychosocial factors often delay but do not contraindicate listing and require team collaboration.</p><p><strong>Conclusions: </strong>Patient/family social and psychosocial factors influence pediatric liver transplant selection and contribute to clinician moral distress. To promote justice and mitigate distress, advocacy should focus on optimizing patient and family support from the listing phase onward, clinician ethics education, and more balanced decision-making frameworks.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":"30 10","pages":"e70471"},"PeriodicalIF":1.4,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149168279","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":"Effectiveness and Safety of Endoscopic Retrograde Cholangiopancreatography for Post-Transplant Biliary Strictures in Children: Experience From Two Centers in Southern Iran.","authors":"Iraj Shahramian, Mohammad Hadi Imanieh, Mahmood Haghighat, Seyed Mohsen Dehghani, Naser Honar, Maryam Ataollahi, Seyed Ali Malekhosseini, Gholamreza Sivandzadeh, Fardad Ejtehadi, Ramin Niknam, Kourosh Kazemi, Seyed Mohammad Amir Hashemi","doi":"10.1111/petr.70486","DOIUrl":"10.1111/petr.70486","url":null,"abstract":"<p><strong>Background: </strong>Biliary strictures are among the most frequent complications following pediatric liver transplantation and can compromise graft function if not promptly managed. Endoscopic retrograde cholangiopancreatography (ERCP) is increasingly recognized as a safe, minimally invasive first-line therapy, though pediatric data remain limited.</p><p><strong>Methods: </strong>We retrospectively reviewed children under 18 years who developed post-transplant biliary strictures between 2017 and 2025 at two tertiary centers in Shiraz, Iran. Demographic, clinical, and procedural data were analyzed. Procedural success was defined as restoration of bile flow, and overall patient outcomes were evaluated across all ERCP sessions.</p><p><strong>Results: </strong>Among 362 pediatric liver transplant recipients, 31 (8.5%) developed biliary strictures, and 27 met inclusion criteria (12 males, 15 females). A total of 87 ERCP sessions were performed, with an overall success rate of 70.4%. Six patients (22.2%) required percutaneous transhepatic biliary drainage, and two (7.4%) underwent surgical intervention. Serum ALT and ALP levels significantly decreased after ERCP (ALT: 123 to 46 U/L, p = 0.02; ALP: 767 to 568 U/L, p = 0.028; Wilcoxon signed-rank test), whereas total bilirubin changes were not significant. Complications occurred in 12.6% of procedures, mainly mild post-ERCP pancreatitis (6.9%), all managed conservatively.</p><p><strong>Conclusions: </strong>ERCP is a safe and effective initial treatment for post-transplant biliary strictures in children. Complications are generally minor and manageable, supporting its role as a minimally invasive therapeutic option in pediatric liver transplant centers.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":"30 10","pages":"e70486"},"PeriodicalIF":1.4,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149168494","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":"Expanding the Donor Pool in Pediatric Transplantations: Successful Domino Liver Transplantation From the Youngest Reported Pediatric DCD Donor Using Normothermic Regional Perfusion in the United States.","authors":"Anastasia M Kahan, Francisca van der Schyff, Alan G Contreras, Dave N Peterson, M Kyle Jensen, Jill Sweney, Lindsey N Troy, Jean F Botha, Zachary J Kastenberg","doi":"10.1111/petr.70498","DOIUrl":"10.1111/petr.70498","url":null,"abstract":"<p><strong>Introduction: </strong>Normothermic regional perfusion (NRP) during donation after circulatory death (DCD) improves abdominal organ utilization by limiting warm ischemia time. Its application in young pediatric donors remains exceptionally rare; the youngest previously reported DCD-NRP donor in the United States was 5 years of age.</p><p><strong>Methods: </strong>We describe DCD-NRP organ procurement from a 23-month-old donor and the subsequent use of the liver graft in a domino liver transplantation involving two pediatric recipients. The kidneys were recovered en bloc. The donor, supported on veno-arterial extracorporeal membrane oxygenation (VA-ECMO) following hypoxic-ischemic injury, underwent DCD-NRP using the preexisting ECMO cannulation after thoracic exclusion of the cerebral and coronary circulations. The recovered graft was transplanted into a 4-year-old with maple syrup urine disease (MSUD), whose explanted native liver was then used as a domino graft for an 18-month-old with biliary atresia.</p><p><strong>Results: </strong>NRP was established rapidly with restoration of metabolic activity, and both transplants were technically successful with good early graft function. The MSUD recipient underwent re-laparotomy for a suspected intra-abdominal source of sepsis (no anastomotic leak identified; cultures negative) and recovered with appropriately trending branched-chain amino acid levels, and was discharged on posttransplant Day 17. The domino recipient had an uncomplicated course and was discharged on posttransplant Day 6.</p><p><strong>Conclusion: </strong>To our knowledge, this is the youngest DCD-NRP liver donor reported in the United States and the youngest domino liver transplantation performed using a DCD-NRP graft. Deliberately pairing NRP with domino transplantation may synergistically expand the critically limited pediatric donor pool.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":"30 10","pages":"e70498"},"PeriodicalIF":1.4,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13655296/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149170269","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":"Successful Liver Transplantation in a Child With Invasive Aspergillus terreus Cholangitis Identified via Plasma Metagenomic Next-Generation Sequencing.","authors":"Katherine McAleese, Minoti Vather, Sara O Vargas, Tanvi S Sharma, Scott A Elisofon, Geoffrey Guenther","doi":"10.1111/petr.70490","DOIUrl":"10.1111/petr.70490","url":null,"abstract":"<p><strong>Background: </strong>Ascending cholangitis, one of the most common complications in patients with biliary atresia (BA) after Kasai hepatoportoenterostomy (HPE), impacts native liver survival. Typically, enteric bacterial flora are the source of infection, and fungal infections of the biliary tree are rare.</p><p><strong>Case report: </strong>We report the case of a 3-year-old female with BA status post Kasai HPE who developed Aspergillus terreus cholangitis (complicated by hepatic vein thrombus) identified through plasma metagenomic next-generation sequencing (NGS) of microbial cell-free deoxyribonucleic acid (DNA). Despite improvement in galactomannan (GM) antigen indices while on antifungal therapy with voriconazole and anticoagulation, she experienced complications with the extension of septic thrombus and persistent cholestasis; thrombus tissue sampling later confirmed the diagnosis and antimicrobial sensitivities. Ultimately, native liver explantation succeeded in achieving source control with sustained normalization of GM indices after the completion of voriconazole. Post-transplant, she received routine immunosuppression and infection prophylaxis per institutional protocol and remains well without infectious complications more than 1 year later.</p><p><strong>Discussion: </strong>This case highlights the important role of newer molecular diagnostic tools in facilitating timely species-level pathogen identification and initiation of targeted therapy (in this case, 35 days prior to confirmed tissue diagnosis), particularly in circumstances where the risk-to-benefit ratio of invasive tissue sampling is unfavorable. Despite the significant morbidity associated with invasive fungal infection, transplantation followed by standard immunosuppression is a reasonable therapeutic option with successful outcomes in patients who are otherwise suitable transplant candidates.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":"30 10","pages":"e70490"},"PeriodicalIF":1.4,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149168724","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":"Comment on \"Effects of Perioperative Management on Short-Term Renal Transplant Outcome\".","authors":"Muhammad Ahmad Khan, Nida Sher","doi":"10.1111/petr.70497","DOIUrl":"https://doi.org/10.1111/petr.70497","url":null,"abstract":"<p><p>In a recent retrospective cohort study by Wang et al., the perioperative hemodynamic variables were found to be associated with short-term graft function in pediatric renal transplant recipients, especially mean arterial pressure (MAP) before and after the graft reperfusion. However, a set age-based MAP threshold does not necessarily reflect the individual renal allograft perfusion needs. The time spent below a target MAP may not be equivalent between patients, as the MAP threshold may change depending on the recipient and donor blood vessel characteristics, and the baseline hemodynamics, as well as the donor-recipient size relationship. Future research with personalized hemodynamics targets could shed light on the effects of MAP control during surgery on early renal graft function.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":"30 10","pages":"e70497"},"PeriodicalIF":1.4,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149170832","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":"Disseminated Bartonella or Post-Transplant Lymphoproliferative Disease in Pediatric Transplant Patients: A Diagnostic Challenge.","authors":"David Thomas, Erica Merlino, Chris Lamprecht, James Davis Cury, Kiran Upadhyay","doi":"10.1111/petr.70476","DOIUrl":"10.1111/petr.70476","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Background: &lt;/strong&gt;Post transplant lymphoproliferative disease (PTLD) and infection remain the two most common complications of solid organ transplant and carry a risk of significant morbidity and mortality. PTLD is a highly variable disease with clinical signs and symptoms that are non-specific and require a high degree of suspicion to detect. The most common presenting symptoms of PTLD are malaise, fatigue, fever, and a mononucleosis-like picture; night sweats, weight loss, and lymphadenopathy are also frequent manifestations. An infection with Bartonella Henselae is another known cause of local lymphadenopathy. However, in immunocompromised individuals, Bartonella Henselae is far more likely to cause a systemic response that may mimic PTLD. The specifics regarding differentiating systemic bartonella and PTLD in pediatric solid organ transplant patients are not well described. There have been more detailed descriptions of disseminated bartonella mimicking PTLD in solid organ transplants in adult patients, but this has not been thoroughly reported or described in the pediatric population. Upon literature review, only five cases of disseminated bartonella in pediatric kidney transplant recipients have been described, with three of them possibly associated with PTLD. The past cases described antimicrobial diagnostics for bartonella and other infections, but none of these reported full histopathological findings or associated imaging studies for work-up of PTLD. In addition, past cases have only been described in Epstein-Barr virus (EBV) and Cytomegalovirus (CMV) negative children. There is no consensus for treatment duration and antibiotic choice for confirmed systemic bartonella infection in pediatric kidney transplant patients due to concerns of nephrotoxicity and drug interactions.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Methods: &lt;/strong&gt;This patient who presented with fever, lymphadenopathy, nausea, vomiting, and abdominal pain was worked up and treated for both Bartonella Henslae and PTLD. PET Scan and biopsies were taken for the diagnosis of PTLD and the patient had Bartonella serologies and PCR testing done as part of a broader infectious workup. Clinical decisions regarding his treatment plan and the cause of his symptoms were made based on response to treatment and the likelihood of underlying etiology based on the timeline which his symptoms improved.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;After an extensive workup for PTLD and altering immunosuppressive regimens temporarily for possible PTLD we ultimately determined the cause of the patients' symptoms to be secondary to B. henselae infection. The patient was treated appropriately for disseminated Bartonella and improved significantly. He has followed up as an outpatient with continued improvement in symptoms and resumed his previous immunosuppression regimen as PTLD has been ruled out.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusion: &lt;/strong&gt;Bartonella and PTLD are diagnoses that need to be carefully considered and treated in ","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":"30 10","pages":"e70476"},"PeriodicalIF":1.4,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149113796","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":"Kidney Transplantation From a Heterozygous CLDN19 Carrier in a Child With FHHNC: A 2-Year Follow-Up.","authors":"Hakan Erdoğan, Mahmut Esat Danışoğlu, Hüseyin Çelik, Murat Şamlı, Bülent Oktay","doi":"10.1111/petr.70495","DOIUrl":"10.1111/petr.70495","url":null,"abstract":"<p><strong>Background: </strong>Familial hypomagnesemia with hypercalciuria and nephrocalcinosis is a rare autosomal recessive tubulopathy caused by mutations in CLDN16 or CLDN19, leading to progressive chronic kidney disease. While kidney transplantation is the definitive treatment, the safety of using living-related heterozygous carrier donors remains debated due to potential subclinical tubular dysfunction. We report the 2-year follow-up of a pediatric patient with FHHNC and a CLDN19 mutation who received a graft from his heterozygous carrier mother.</p><p><strong>Case report: </strong>A male patient presented at 3 months of age with nephrocalcinosis, hypercalciuria, and hypomagnesemia. Genetic analysis confirmed a homozygous CLDN19 (G179S) mutation. Consistent with the CLDN19 phenotype, the patient exhibited severe ocular involvement, including high myopia and macular coloboma. He progressed to end-stage kidney disease by age 15 and underwent living-related kidney transplantation from his mother (a heterozygous carrier) at age 16. Pre-operative donor evaluation showed normal renal function without proteinuria, hypercalciuria, nephrolithiasis, or nephrocalcinosis.</p><p><strong>Results: </strong>At 24 months post-transplantation, graft function remained stable with a serum creatinine of 1.22 mg/dL and a 24-h creatinine clearance of 94 mL/min/1.73 m². Metabolic abnormalities were fully corrected; serum magnesium stabilized at 1.89 mg/dL without supplementation, the urinary calcium/creatinine ratio normalized (2.6 to 0.06), and proteinuria decreased from 2580 mg/day to 324 mg/day. The heterozygous donor maintained stable renal function (24-h creatinine clearance 86 mL/min/1.73 m²) without proteinuria, nephrolithiasis, or metabolic complications during follow-up.</p><p><strong>Conclusions: </strong>Given the shortage of deceased donor organs, heterozygous parents may be considered suitable donors for children with FHHNC, provided they undergo comprehensive genetic, biochemical, and radiological evaluation to exclude evidence of tubular dysfunction. However, this conclusion should be limited to carefully selected donors, and longer-term follow-up is required to establish the safety of kidney donation in heterozygous CLDN19 carriers.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":"30 10","pages":"e70495"},"PeriodicalIF":1.4,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149149025","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":"Early Hematopoietic Stem Cell Transplantation in Type 1 Fucosidosis: Reversal of Central Tegmental Tract Abnormalities.","authors":"Mehmet Palaz, Ahmet Taşkın, Esra Özpınar, Yasemin Topçu, Yöntem Yaman, Betül Kılıç, Kürşad Aydın","doi":"10.1111/petr.70502","DOIUrl":"https://doi.org/10.1111/petr.70502","url":null,"abstract":"<p><strong>Background: </strong>Fucosidosis is an ultra-rare lysosomal storage disorder caused by alpha-L-fucosidase deficiency, leading to progressive neurodegeneration and multisystem involvement. Hematopoietic stem cell transplantation (HSCT) has emerged as a potential disease-modifying therapy, particularly when performed early in the disease course. However, neuroradiological reversibility after HSCT remains insufficiently documented.</p><p><strong>Methods: </strong>We report a male infant diagnosed with Type 1 fucosidosis at 6 months of age based on enzymatic and genetic analyses. Brain magnetic resonance imaging (MRI) and magnetic resonance spectroscopy (MRS) demonstrated characteristic abnormalities, including central tegmental tract involvement. The patient underwent allogeneic HSCT at 14 months of age and was followed clinically and radiologically.</p><p><strong>Results: </strong>Following HSCT, the patient demonstrated stabilization and gradual improvement of neurological functions. Follow-up neuroimaging revealed marked regression of white matter abnormalities and reversal of central tegmental tract hyperintensities. Developmental progression improved after transplantation, and no major transplant-related neurological complications occurred.</p><p><strong>Conclusion: </strong>Early HSCT was accompanied by clinical improvement and resolution of central tegmental tract abnormalities in this patient. These imaging changes may help assess treatment response, though larger cohorts and longer follow-up are needed to evaluate long-term outcomes.</p>","PeriodicalId":20038,"journal":{"name":"Pediatric Transplantation","volume":"30 10","pages":"e70502"},"PeriodicalIF":1.4,"publicationDate":"2026-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149207538","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}