Pediatric Transplant Centers Research Articles

Genetic Assessment of Living Kidney Transplant Donors: A Survey of Canadian Practices.

Kidney failure is a prevalent condition with tendency for familial clustering in up to 27% of the affected individuals. Living kidney donor (LKD) transplantation is the optimal treatment option; however, in Canada, more than 45% of LKDs are biologically related to their recipients which subjects recipients to worse graft survival and donors to higher future risk of kidney failure. Although not fully understood, this observation could be partially explained by genetic predisposition to kidney diseases. Genetic testing of potential LKDs may improve risk assessment and inform the safety of donation. The strategies to evaluate these donors are still evolving. In Canada, little is known about the practice of assessing for genetic conditions among LKDs. The aim was to examine the Canadian practices regarding LKDs genetic assessment. Questionnaires were sent to 23 Canadian adult transplant centers to examine their protocols for LKDs genetic assessment. The questionnaire comprised of 10 sections and 21 questions including case scenarios of different LKD encounters. Major domains of the survey addressed general demographics, information sharing practices, effect of mode of inheritance on candidacy decision, having a policy for LKD genetic evaluation, and case scenarios covering the following conditions: autosomal dominant polycystic kidney disease (ADPKD), Alport syndrome, Fabry disease, familial focal and segmental glomerulosclerosis (FSGS), atypical hemolytic uremic syndrome (aHUS), autosomal dominant tubulointerstitial kidney disease (ADTKD), sickle cell, and apolipoprotein L1 mutation (APOL1). The questionnaire was sent to the living-donor assessment committee representative (nephrologist) in adult and pediatric kidney transplant centers across Canada. In total, 16 of 23 Canadian centers responded to the survey. Of the 8 surveyed genetic conditions, ADPKD, Alport syndrome, and aHUS were the most frequently encountered. More centers have specific policies for donor evaluation for ADPKD (25%) and aHUS (21.4%) vs none to very few for other genetic conditions. The most cited guidelines are Kidney Disease Improving Global Outcomes (KDIGO), Canadian Society of Nephrology/Canadian Society of Transplantation (CSN/CST), and the Canadian Blood Services' Kidney Paired Donation Protocol. Canadian transplant centers follow a case-by-case approach rather than a standard protocol for genetic assessment of LKDs given that current guideline recommendations are based on expert opinion due to a lack of a reliable body of evidence. With the expected rise in utilization of the increasingly available genetic testing, early multidisciplinary assessment including medical geneticists has the potential to improve personalized management. Studies examining long-term donor and graft outcomes are needed to construct the basis for evidence-based recommendations and inform the safety of donations.

Belumosudil Is Efficacious for the Management of Heavily Pretreated and Prolonged Chronic Graft-Versus-Host Disease: A Retrospective Multi-Center Real-World Study By the Israel Association for Bone Marrow Transplantation and Cellular Therapy

Introduction : Chronic graft-versus-host disease (cGvHD) remains one of the most important complications of allogeneic stem cell transplantation (ASCT) and is a leading cause of morbidity, late non-relapse mortality, and impaired quality of life. Recent advances in understanding the cGVHD mechanisms have led to the development of the Rho-associated coiled-coil containing protein kinase 2 (ROCK-2) selective inhibitor, belumosudil. The pivotal ROCKstar trial demonstrated the efficacy and acceptable safety profile of belumosudil in cGVHD patients, which led to its FDA approval for the use after >2 prior lines of therapy (LOT). The current study aimed to investigate the efficacy and safety of belumosudil in patients with cGVHD in real-world settings across all transplant centers in Israel. Methods : Data on patients treated with belumosudil for cGVHD at 6 adult and 3 pediatric transplant centers via compassionate use programs were collected and analyzed. These data included patient demographics, underlying disease requiring ASCT, transplant details, cGVHD details, and outcomes of belumosudil treatment. Results : Forty-five patients were included in the final analysis: 42 were treated according to FDA indications, and 3 pediatric patients (ages 4, 4, and 6 years) were treated outside the FDA labeled indication. The median age was 46 (range, 4-75) years; 44.4% were females. GVHD prophylaxis included a calcineurin inhibitor and methotrexate in 66.7%, post-transplant cyclophosphamide in 13.3% and others in 20% of patients. Acute GVHD occurred in 48.9% of patients. The majority of patients (84.4%) had severe cGVHD with a median of 4 involved organ systems (range, 1-7) and received a median of 4 prior LOT (range, 2-6). Notably, 86.7% had prior ruxolitinib exposure, and 13.3% concurrently received belumosudil and ruxolitinib. Patients had been treated for cGVHD for a median of 2.3 (range, .08 - 19.1) years prior to belumosudil commencement. The median duration of belumosudil therapy was 7.8 (range: 1.2 - 28.8) months, with 73.3% of patients still on treatment. The median time to best response was 100 days (range: 32 - 328). Response rates were as follows: complete response (CR) 6.7%, partial response (PR) 48.9%, stable disease (SD) 40%, and progressive disease (PD) 4.4%, with an overall response rate (ORR) of 55.6%. Responses for skin cGVHD were: CR - 11.1%, PR - 50%, SD - 38.9%; for lung cGVHD: PR - 44.4%, SD - 48.1%, PD - 7.4%. The median reduction of the glucocorticoid dose was from 0.29 mg/kg/d of prednisone at the beginning of treatment to 0.02 mg/kg/d at the last follow-up or belumosudil cessation. The most common adverse events (AEs) were pulmonary infections (17.8%) and the primary reason for belumosudil discontinuation was cGHVD worsening. Only one patient stopped belumosudil due to AEs. While on treatment, 26.1% of patients had cGVHD progression. Six patients (13.3%) died, three from cGVHD worsening, two from pulmonary infections, and one from relapsed leukemia. At a median follow-up of 8 months (range 1.3-32), the GVHD relapse-free survival (RFS) was 72% (95% CI 59%-85%) and the estimated 12-month GVHD RFS was 66% (95% CI 46%-86%). Overall survival (OS) at 8 months was 92% (95% CI 81%-100%) and the estimated 12-month OS was 75% (95% CI 62%-88%). Conclusions : The findings of this real-world study, while comparable to those observed in the ROCKstar trial, are encouraging, considering the heavily pretreated patient population with prolonged and severe cGVHD. No new safety signals emerged. Future studies are warranted to optimize patient selection and timing for treatment initiation.

Evaluation and Management of Urological Complications Following Pediatric Kidney Transplantation: Experience from a Single Tertiary Center.

Background/Objectives: Kidney transplantation is the treatment of choice for children with end-stage renal disease (ESRD), but its outcome can be affected by urological complications, with incidence rates of 2.5-25%. The aim of this study was to evaluate the occurrence of urological complications and their management in a cohort of pediatric kidney transplant recipients. Materials and Methods: A retrospective analysis on 178 patients who received a renal transplant at our Pediatric Kidney Transplant Center between 2011 and 2023 was conducted. Demographic and clinical data were analyzed. Urological complications were categorized as early, intermediate, or late based on their onset time. Results: Out of 178 patients, 28 (15.7%) experienced urological complications. Most patients (61%) had a pre-existing uropathy. Early complications (7-30 days) were all obstructive, namely, ureterovesical junction obstruction and perirenal collections. Intermediate complications (1-3 months) comprised ureteral stenosis, symptomatic vesicoureteral reflux (VUR), and obstructive lymphocele. Late complications (>3 months) included symptomatic VUR and ureteral stenosis, with one case leading to ureteral rupture. Early complications were often detected due to acute graft dysfunction, while late ones were mainly identified during routine clinical, laboratory, or ultrasound follow-up. Urological complications requiring surgical or endoscopic therapy were 13.4%. Most ureteral stenoses were treated with initial endoscopic stents, followed by definitive surgery. VUR was treated with endoscopic correction with a high success rate (75%), while open surgery was reserved for cases where initial treatments failed or complications recurred. No clear correlations were found between patient characteristics and risk of urological complication. Urological complications required multiple diagnostic procedures and therapeutic interventions (+2.5 admissions in mean and approximately +EUR 24,000) compared to an uncomplicated post-transplant course. However, they did not significantly impact transplant outcomes, with a graft survival rate comparable to that of the control group. Conclusions: Regular post-transplant follow-up is crucial, especially for patients with known risk factors, to allow for timely detection and treatment of urological complications, avoiding detrimental effects on graft function and improving transplantation outcomes.

Central line-associated bloodstream infection outbreak related to Ralstonia pickettii-contaminated saline in a pediatric hematopoietic stem cell transplant center.

Ralstonia pickettii is an aerobic Gram-negative non-fermentative bacillus. It is an opportunistic pathogen that has recently prompted nosocomial outbreaks. Although it has low virulence, it can cause a wide range of invasive diseases in immunosuppressive patients. The characteristics of R. pickettii-related central line-associated bloodstream infection (CLABSI) outbreak in pediatric hematopoietic stem cell transplant (HSCT) recipients are presented in this study. This was a single-center, retrospective analysis conducted at Bahcesehir University Goztepe Medicalpark Hospital . The clinical and laboratory characteristics of twelve children with Ralstonia-related CLABSIs were analyzed. Of the twelve patients with R. pickettii growth, seven were female. The median age was 12.1 (2-17) years. Autologous HSCT was performed in two of the patients and allogeneic HSCT was performed in ten patients for both malignant and non-malignant diseases. In the conditioning regimens, all patients were given myeloablative therapy. Clinical sepsis was the most common presentation. As a result of the investigations, R. pickettii growth was observed in saline solutions. All cases were successfully treated with the appropriate antibiotic regimen and the bacteria was not found in repeat cultures. Catheter removal was required in two patients. Mortality was not observed in any patient as the outcome of the infection episode. The detection and control of the infectious source are critical in pediatric HSCT patients with severe immunosuppression, as medical equipment-related outbreaks can be life-threatening.

Challenges and Possible Solutions to Pediatric Organ Donation and Transplantation in Turkey and the Middle East: Panel Discussion/Brainstorm Session Summary Report.

Organ transplantation is the best therapeutic option for children with end-stage organ failure. Pediatric transplantation centers and prioritization of organs for children are being established around the world. However, there is an important discrepancy observed when comparing high-income countries versus low-income and middle-income countries, as programs are active in some, but not in many others. Deceased organ donation is low in many places, which has led to an increase of organ shortage; consequently, there is a need to rely on the use of living donor organs. This is frequently the case for pediatric transplantation. To gather information about the possible reasons underlying these challenges and to propose possible solutions, a brainstorming session took place during the International Symposium on Pediatric Organ Donation and Transplantation in Ankara, Turkey. A description on different topics that were discussed is presented herein.

A study of pulmonary infectious and non-infectious complications in a pediatric hematopoietic stem cell transplantation (HSCT) center in Iran

Background: Pulmonary complications are important enough to notice in hematopoietic stem cell transplantation (HSCT). The patients undergoing HSCT might have infectious and non-infectious problems associated with morbidity and mortality. The pulmonary complications of HSCT are well-recognized in adults; however, studies on children are limited, especially in Iran. This study was done to evaluate the infectious and non-infectious pulmonary complications and the corresponding factors in children who underwent HSCT. Materials and Methods: This retrospective cohort study included the patients who underwent HSCT in Mofid Children’s Hospital in Tehran, Iran, during the years 2015 -2021. Overall, 144 medical files were evaluated, out of which 128 had undergone HSCT. The extracted data were about underlying diseases, age at transplant, sex, type of HSCT, donor type, cell source, conditioning regimen, graft versus host disease (GVHD) prophylaxis, infectious and non-infectious pulmonary complications, and associated factors. The data analysis was done by the SPSS software version 26. Chi-square, Fisher exact test and regression were also used for the analysis. Results: Infectious and non-infectious pulmonary complications were reported in 26 people (20.3%) and 11 people (8.6%), respectively. Positive coronavirus disease 2019 (COVID-19) PCR was detected only in two patients. Infectious complications were significantly lower in patients with neuroblastoma compared to other underlying diseases (2.7% vs. 27.5%, P = 0.002). These complications were significantly more frequent among those with other HSCT complications compared to those without such complications (34.6% vs. 16.7%, P=0.042). Non-infectious pulmonary complications were significantly higher in boys (13.5%) than in girls (1.9%) (P = 0.024). Conclusion: Due to the high rate of pulmonary infections in bone marrow transplant patients, clear differential diagnosis and diagnostic work are essential.

Pulmonary complications post allogeneic haematopoietic stem cell transplant in children.

Haematopoietic stem cell transplant (HCT) is a cellular therapy that, whilst curative for a child's underlying disease, carries significant risk of mortality, including because of pulmonary complications. The aims of this study were to describe the burden of pulmonary complications post-HCT in a cohort of Australian children and identify risk factors for the development of these complications. Patients were identified from the HCT databases at two paediatric transplant centres in Australia. Medical records were reviewed, and demographics, HCT characteristics and pulmonary complications documented. Relative risk ratio was used to identify risk factors for developing pulmonary complications prior to first transplant episode, and survival analysis performed to determine hazard ratio. In total, 243 children underwent transplant during the study period, and pulmonary complications occurred in 48% (117/243) of children. Infectious complications were more common (55%) than non-infective complications (18%) and 26% of patients developed both. Risk factors for the development of pulmonary complications included the following: diagnoses of MPAL (RR 2.16, P = 0.02), matched unrelated donor (RR1.34, P = 0.03), peripheral blood (RR 1.36, P = 0.028) or cord blood (RR 1.73, P = 0.012) as the stem cell source and pre-existing lung disease (RR1.72, P < 0.0001). Children with a post-HCT lung complication had a significantly increased risk of mortality compared with those who did not (HR 3.9, P < 0.0001). This study demonstrates pulmonary complications continue to occur frequently in children post-HCT and contribute significantly to mortality. Highlighting the need for improved strategies to identify patients at risk pre-transplant and enhanced treatments for those who develop lung disease.

The Clinical Approach to Interstitial Lung Disease in Childhood: A Narrative Review Article.

Interstitial lung disease (ILD) comprises a group of respiratory diseases affecting the interstitium of the lungs, which occur when a lung injury triggers an abnormal healing response, and an inflammatory process leads to altered diffusion and restrictive respiratory dysfunction. The term "interstitial" may be misleading, as other components of the lungs are usually also involved (epithelium, airways, endothelium, and so on). Pediatric conditions (childhood interstitial lung disease, chILD) are different from adult forms, as growing and developing lungs are affected and more diverse and less prevalent diseases are seen in childhood. Diffuse parenchymal lung disease (DPLD) and diffuse lung disease (DLD) can be used interchangeably with ILD. Known etiologies of chILD include chronic infections, bronchopulmonary dysplasia, aspiration, genetic mutations leading to surfactant dysfunction, and hypersensitivity pneumonitis due to drugs or environmental exposures. Many forms are seen in disorders with pulmonary involvement (connective tissue disorders, storage diseases, malignancies, and so on), but several conditions have unknown origins (desquamative pneumonitis, pulmonary interstitial glycogenosis, neuroendocrine cell hyperplasia in infancy, and so on). Currently, there is no consensus on pediatric classification; however, age grouping is proposed as some specific forms are more prevalent in infancy (developmental and growth abnormalities, surfactant dysfunction mutations, etc.) and others are usually seen in older cohorts (disorders in normal or immunocompromised hosts, systemic diseases, etc.). Clinical manifestations vary from mild nonspecific symptoms (recurrent respiratory infections, exercise intolerance, failure to thrive, dry cough, etc.) to a severe clinical picture (respiratory distress) and presentation related to the child's age. The diagnostic approach relies on imaging techniques (CT), but further investigations including genetic tests, BAL, and lung biopsy (VATS) are needed in uncertain cases. Pharmacological treatment is mostly empiric and based on anti-inflammatory and immunomodulatory drugs. Lung transplantation for selected cases in a pediatric transplantation center could be an option; however, limited data and evidence are available regarding long-term survival. International collaboration is warranted to understand chILD entities better and improve the outcomes of these patients.

Treatment outcome at the pediatric stem cell transplantation center in Syria: A single-center experience.

Hematopoietic stem cell transplantation (HSCT) is a therapeutic approach known for its high success rates in treating various hematologic malignancies, hemoglobinopathies, immune deficiencies, and other disorders. Notably, pediatric HSCT commenced in Syria in 2021 amidst the prevailing crisis. This study aims to assess the demographic and clinical profiles of pediatric patients who underwent stem cell transplantation and to analyze treatment outcomes at Syria's inaugural pediatric HSCT center. This study is a single-center retrospective analysis of 25 pediatric patients who underwent HSCT underage of 14 years in the National Stem Cell Center (HAYAT) in Damascus within the period 2021-2023. The databases were created based on data that were collected from patient medical records. In autologous patients, transplant-related mortality (TRM) was 0%, with 4 (57%) experiencing disease relapse, resulting in the death of one patient. Additionally, 3 (42.8%) of patients remain alive under second-line management. The overall survival rate was 6 (85.7%), and the disease-free survival rate was 16 (88%). In allogeneic patients, TRM was 5.5% (1/18). One allogeneic patient experienced disease relapse and subsequently died. The overall survival rate and disease-free survival rate were 16 (88%). The objective of this study was to assess the outcomes of pediatric HSCT patients who have undergone transplantation thus far. Given the recent initiation of pediatric stem cell transplantation in Syria, our dataset provides a basis for comparison with international hematopoietic stem cell transplantation centers regarding treatment complications and outcomes, notwithstanding the challenges and crises faced within our country.

Turkish Hematologists’ Preferences for Related Donor Selection: Results of a Multicenter Survey

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a widely utilized treatment for various hematological diseases. While selection criteria for unrelated donors are well established, there is a lack of consistency and standardization in the selection of related donors. This study investigated the current approach of hematologists to the selection of related donors at Turkish HSCT centers. The study employed a cross-sectional survey design, distributing a self-administered questionnaire to 95 adult and pediatric transplantation centers in Türkiye to investigate their approaches to related donor selection for allo-HSCT. The questionnaire collected data on various topics including the center’s experience in performing allo-HSCT, patient groups treated, number of allo-HSCT procedures conducted between 2015 and 2021, preferences for related donors, considerations in related donor selection (such as sex and past pregnancies), guidelines utilized for related donor selection, upper age limit for related donors, and the use of specialized advanced analyses for elderly donors. The response rate to the survey was 38.9%. Variability was observed across centers in terms of sex consideration and the impact of past pregnancies on related female donor rejection. Different guidelines were employed for related donor selection, with the European Bone Marrow Transplantation guidelines being the most commonly used. Regarding the upper age limit for related donors, 8.1% of centers accepted an upper age limit of 55 years, 48.7% preferred an upper age limit of 65 years, and 43.2% selected related donors aged 65 and above. The lack of standardized guidelines for related donor selection in HSCT centers leads to variability in criteria and potential risks. Collaboration among centers is essential to establish consensus and develop standardized protocols.

Safety and infectious outcomes in pediatric kidney transplant recipients after COVID-19 vaccination: A pediatric nephrology research consortium study.

Adult kidney transplant recipients (KTRs) fully vaccinated against COVID-19 have substantial morbidity and mortality related to SARS-CoV-2 infection compared with the general population. However, little is known regarding the safety and efficacy of the COVID-19 vaccination series in pediatric KTRs. A multicenter, retrospective observational study was performed across nine pediatric transplantation centers. Eligible KTRs fully vaccinated against COVID-19 were enrolled and data were collected pertaining to SARS-CoV-2 infection incidence and severity, graft outcomes and post-vaccination safety profile, as well as overall patient survival. A total of 247 patients were included in this investigation with a median age at transplantation of 11 years (IQR 5-15). SARS-CoV-2 infection was observed in 30/110 (27.27%) of fully vaccinated patients, tested post-transplant, within the defined follow-up period. Of these patients, 6/30 (18.18%) required hospitalization and 3/30 (12.12%) required reduction in immunosuppression, with no reported deaths. De novo donor-specific antibodies (DSAs) were found in 8/86 (9.30%) of DSA-tested patients with two experiencing rejection and subsequent graft loss. The overall incidence of rejection and graft loss among the total cohort was 11/247 (4.45%) and 6/247 (3.64%), respectively. A 100% patient survival was observed. Observationally, infectious outcomes of SARS-CoV-2 in fully vaccinated pediatric KTRs are excellent, with a low incidence of infection requiring hospitalization and no associated deaths. Though de novo DSAs were observed, there was minimal graft rejection and graft loss reported in the total cohort.

Analysis of UNOS: Pediatric Heart Transplantation Over 36 Years and Contemporary Volume-Outcome Relationship

BackgroundThis study examines 36 years of national pediatric heart transplantation data to (1) identify trends in transplant volume, centers, and 1-year graft survival and (2) assess how center transplant volume affects outcomes over a contemporary 11-year period. MethodsStudy investigators utilized the United Network for Organ Sharing database and performed a retrospective review of pediatric patients (aged <18 years) who underwent heart transplantation between January 1, 1987 and December 31, 2022, inclusive. Trend analyses included the whole cohort, whereas volume-outcome analyses included a contemporary cohort (January 1, 2012 through December 31, 2022) to account for the temporal changes observed in transplant survival. Highest-volume centers were defined by the number of heart transplantations performed per center per year. ResultsOver 36 years, 11,828 pediatric heart transplantations were performed. Transplant volume steadily rose, the number of centers remained stable, and 1-year graft survival improved significantly. In the contemporary era (2012-2022), 89 centers conducted 4959 pediatric heart transplantations. The top 15% high-volume centers (13 centers) accounted for 48.3% (n = 2393) of transplantations, with an average of 16.7 ± 3.8 transplantations per center annually, compared with 3.9 ± 3.1 for lower-volume centers (P < .001). Despite performing transplantations in higher-risk patients, high-volume centers achieved similar postoperative outcomes and improved long-term survival compared with low-volume centers. ConclusionsAlthough the number of US pediatric heart transplant centers has remained stable, pediatric heart transplant volume has steadily increased, as has 1-year graft survival. In a contemporary cohort, the top 15th percentile highest-volume centers accounted for 48.3% of US pediatric heart transplants and performed transplantations in higher-risk patients with similar postoperative outcomes and improved longitudinal survival.

Growth after pediatric kidney transplantation: a 25-year study in a pediatric kidney transplant center.

Growth failure is one of the major complications of pediatric chronic kidney disease. Even after a kidney transplant (KT), up to 50 % of patients fail to achieve the expected final height. This study aimed to assess longitudinal growth after KT and identify factors influencing it. A retrospective observational study was performed. We reviewed the clinical records of all patients who underwent KT for 25 years in a single center (n=149) and performed telephone interviews. Height-for-age and body mass index (BMI)-for-age were examined at KT, 3months, 6months, 1 year, and 5 years post-transplant and at the transition to adult care. We evaluated target height, disease duration before KT, need and type of dialysis, recombinant human growth hormone pretransplant use, nutritional support, glomerular filtration rate (GFR), and cumulative corticosteroid dose. At transplant, the average height z-score was-1.38, and height z-scores showed catch-up growth at 6months (z-score-1.26, p=0.006), 1 year (z-score-1.15, p<0.001), 5 years after KT (z-score-1.08, p<0.001), and on transition to adult care (z-score-1.22, p=0.012). Regarding BMI z-scores, a significant increase was also detected at all time points (p<0.001). After KT, GFR was significantly associated with height z-score (p=0.006) and BMI z-score (p=0.006). The height in transition to adult care was-1.28 SD compared to the target height. Despite the encouraging results regarding catch-up growth after KT in this cohort, results remain far from optimum, with a lower-than-expected height at the time of transition.

Biliary strictures post pediatric liver transplantation-incidence and risk factors in a single tertiary referral transplant center.

Biliary strictures are a significant cause of morbidity and graft loss in pediatric liver transplant recipients. Risk factors for the development of biliary strictures are not fully established. We aimed to evaluate the incidence of biliary strictures and treatment modalities outcomes and to identify potential risk factors for occurrence. Pediatric patients who underwent liver transplantation in the single tertiary pediatric liver transplant center in Israel were evaluated. We compared demographics, presentation, laboratory results, imaging, treatment, and outcomes between patients with and without biliary stricture. Multivariate regression analyses were used to identify risk factors for biliary strictures. Among 121 pediatric liver transplant patients, 65 (53.7%) were males; the median age at the time of liver transplantation was 43 (3-215) months. Fifteen patients (12.4%) had biliary strictures following transplantation. One (7%) patient with biliary stricture was treated via endoscopic retrograde cholangiopancreatography, and 12 patients (80%) underwent interventions via a percutaneous transhepatic approach. Nine of the 12 patients were treated successfully, requiring one or multiple procedures, while the remaining had surgery or laser therapy. Risk factors for the development of biliary strictures were biliary leak, acute cellular rejection, and the presence of two biliary anastomoses. In our cohort, the presence of two biliary anastomoses and post-transplant complications including acute cellular rejection and early biliary leaks were associated with biliary strictures in pediatric liver transplantation recipients. Percutaneous transhepatic interventions result in good outcomes in most patients.

Public Reporting of Heart Transplant Center Performance: Promoting Clarity or Causing Confusion?

BackgroundTransplant center report cards are publicly available and used by regulators, insurance payers, and importantly patients and families. ObjectivesIn this study, the authors sought to evaluate the variability in reported public performance ratings of pediatric and adult heart transplant centers. MethodsProgram-specific reports from the Scientific Registry of Transplant Recipients from 2017-2021 were used to evaluate stability, volatility, and reliability of 3 publicly reported ratings: waitlist survival (WS), getting to a faster transplant (FT), and post-transplantation graft failure (GF). ResultsThere were 112 adult and 55 pediatric centers. Over the study period, nearly all centers (98%) had at least 1 change in rating in at least 1 of the tiers. The average time to the first rating change of any magnitude was 12-18 months for all tiers and centers. For adult centers, the most volatile rating was WS (SD: 0.77), followed by GF (SD: 0.76) and then FT (SD: 0.57). For pediatric centers, the most volatile rating was WS (SD: 0.79), followed by both GF (SD: 0.66) and FT (SD: 0.68), which were equally volatile. All tiers except adult FT had an estimated Fleiss’s kappa <0.20, indicating poor agreement/consistency across the study period. In addition, the intraclass correlation coefficient for all tiers was <0.50, indicating poor reliability. ConclusionsThe current 5-tier reporting of transplant center performance is highly volatile and has poor reliability and consistency. Given the unintended and significant negative consequences these reports can have, critical revision of these ratings is warranted.

National survey of transplant pharmacist ambulatory care practices

AbstractIntroductionThere has been sustained growth in transplant pharmacist positions. However, the scope of pharmacist involvement, frequency of patient visits with a transplant pharmacist, and description of the transplant pharmacists' role in the ongoing management of transplant recipients have not been well described.ObjectiveTo determine the prevalence and role of transplant pharmacists in the management of transplant recipients in the ambulatory care setting.MethodsA 53‐question online survey was developed with the primary outcome being the incidence of solid organ transplant (SOT) programs with ambulatory care pharmacy services. Secondary outcomes included describing the scope of ambulatory transplant pharmacist activities for posttransplant recipients. The survey was distributed via transplant listservs and remained open from April 25, 2023 to May 27, 2023.ResultsA total of 174 responses were included for analysis, with 47 (27%) being partial responses. The total number of pediatric adult and pediatric transplant centers in the United States was 248 with a total number of 748 active programs, giving an estimated response rate of 23.8%. Of those, 93.7% reported having ambulatory transplant pharmacy services. The median of ambulatory care full‐time employees was 1 (interquartile range [IQR] 1–2) across the responding institutions. Transplant pharmacists were also involved in outpatient infusions (69.2%), immunization screening and administration (82.5%), telehealth services (57.0%), prescription refill authorization (61.0%) electronic medical record in‐basket, or equivalent messaging tasks (91.1%).ConclusionThe incidence of SOT pharmacist involvement in ambulatory care is overall high (93.7%). Analysis of this project allows for a better understanding of the landscape of SOT ambulatory care pharmacy services and can be used as an assessment for standard practice activities.

(1105) - Outcomes of Young Adults Undergoing Heart Transplant at Pediatric vs Adult Transplant Centers

(1105) - Outcomes of Young Adults Undergoing Heart Transplant at Pediatric vs Adult Transplant Centers

Effect of allogeneic hematopoietic stem cell transplantation for chronic granulomatous disease in children: A multicentre, retrospective cohort study in China

Chronic granulomatous disease (CGD) in children is a rare primary immunodeficiency disorder that can lead to life-threatening infections and inflammatory complications. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is increasingly being used to treat severe CGD in children. We conducted a multicenter retrospective analysis of children with CGD who were treated with allo-HSCT at four pediatric hematopoietic stem cell transplant centers in China from September 2005 to December 2019. The study included a total of 171 patients (169 males and 2 females). The median age at the time of transplantation was 6.1 (0–16.4) years. Among them, 154 patients had X-linked recessive inheritance caused by CYBB gene mutations, 12 patients were autosomal recessive, 1 patient had DNAH11 and HYDIN gene mutations, and 4 patients had no gene mutations. The median follow-up period was 36.3 (1.9–79) months. All participating patients were applied to myeloablative conditioning (MAC) regimens. The rates of OS, EFS, and GEFS within three years were 87.5%, 85.3%, and 75.2%, respectively. The total graft failure and the total mortality rate were 5.3% and 11.1%. The cumulative incidence of acute GVHD was 53.8% and the incidence of chronic GVHD was 12.9%, The incidence of chronic GVHD was higher for patients who received unrelated donor cord blood stem cell transplantation (UD-CB) (P = 0.001). Chronic GVHD and coinfections are the risk factors for OS and EFS in patients with CGD after receiving allo-HSCT. UD-CB is a risk factor for EFS and the presence of pneumonia before transplantation is a risk factor for OS. In conclusion, through this study, we have demonstrated that allo-HSCT has excellent efficacy in the treatment of CGD in children, especially, RD-haplo is associated with a lower rate of graft failure incidence and mortality than the treatment modalities of other donor type. Therefore, allo-HSCT is strongly recommended when a well-matched donor is available. If a well-matched donor is not available, the HLA-mismatched donor should be carefully evaluated, and the conditioning regimen modified accordingly.

Single Pediatric Transplant Center Experience with Belumosudil As a Potential Treatment for Chronic Graft Vs Host Disease in Adolescents and Young Adults

Single Pediatric Transplant Center Experience with Belumosudil As a Potential Treatment for Chronic Graft Vs Host Disease in Adolescents and Young Adults

Improvement of survival after hematopoietic stem cell transplantation and trends at a pediatric transplantation center; a three-decade journey.

Advances in stem cell transplantation have resulted in improved outcomes. This is a retrospective study aimed to analyze changes in patient profile, transplantation, graft characteristics, and outcome among 241 pediatric patients who received stem cell transplantation in a single center between 1993 and 2019. In the 2010-2019, compared with the 1993-2009 period, a significantly higher 5-year overall survival (60% vs. 44%, p = .022) and an event-free survival (53% vs. 34%, p = .025) were observed. Cumulative incidence of deaths due to relapse or progression between the 1993-2009 and 2010-2019 periods were 33% and 26% respectively (p = .66). Cumulative incidence of non-relapse mortality was significantly higher during the 1993-2009 period compared with the 2010-2019 period for malignant diseases (57.7% vs. 28.3%, p = .007). The overall survival from acute graft-versus-host disease between 1993 and 2009 was 11% versus 46% between 2010 and 2019 (p = .0001). The overall survival from infection in both eras did not show any difference (p = .41). Development in transplantation technology has led to a decrease in non-relapse mortality and better control of graft-versus-host disease. However, relapse and infection remained as major causes of death. Studies evaluating institutional trends in patients undergoing HSCT and analyzing their mortality profile, can improve the management of patients, leading to a reduction in transplant-related problems.