Outcomes Of Transplantation In Children Research Articles

Kidney transplant outcomes in children with simultaneous versus sequential heart-kidney transplants.

Heart transplant recipients frequently require kidney transplantation for concomitant advanced chronic kidney disease. Data on simultaneous (heart and kidney transplants performed simultaneously) versus sequential (heart transplant performed before kidney) heart-kidney transplants in children are limited. Herein, we compare kidney transplant outcomes between the two groups. We used the Scientific Registry of Transplant Recipients to identify all pediatric (age <21 years) heart transplant recipients who also received a kidney transplant within 10 years of the heart transplant. We divided the study cohort into simultaneous heart-kidney and sequential heart-kidney recipients. We compared patient and death-censored graft survival using the Cox regression, adjusting for age at kidney transplant, sex, race, pre-transplant dialysis, donor type, and prior kidney transplant. We evaluated delayed graft function (defined as dialysis within the first week posttransplant) using logistic regression. Our analysis cohort included 165 recipients (86 simultaneous and 79 sequential). The incidence of delayed graft function was higher in simultaneous recipients (22.4 vs. 7.7%, p=0.017), but the difference lost statistical significance on multivariable analysis. We found no difference in patient survival (aHR 0.97; 95% CI 0.39, 2.41; p=0.95) after kidney transplant but higher death-censored kidney graft survival in sequential heart-kidney recipients compared with simultaneous heart-kidney recipients (aHR 4.26; 95% CI 1.21, 14.9; p=0.02). Sequential heart-kidney transplants are associated with higher death-censored kidney allograft survival in children compared with simultaneous heart-kidney transplants.

Change in Conditioning Regimen Improves Hematopoietic Stem Cell Transplant Outcomes in Children with Lysosomal Storage Disorders

Change in Conditioning Regimen Improves Hematopoietic Stem Cell Transplant Outcomes in Children with Lysosomal Storage Disorders

T-Cell Depleted Haploidentical Transplantation in Children With Hematological Malignancies: A Comparison Between CD3+/CD19+ and TCRαβ+/CD19+ Depletion Platforms.

BackgroundT-cell depleted (TCD) haploidentical transplantation using CD3+/CD19+ and TCRαβ+/CD19+ depletion techniques has been increasingly used in children with hematological malignancies. We present a retrospective study aimed to compare transplant outcomes in children with leukemia receiving a TCD haploidentical transplant using either CD3+/CD19+ or TCRαβ+/CD19+ platforms.MethodsA total of 159 children with leukemia (ALL=80) (AML=79) that received a TCD haploidentical transplantation using either CD3+/CD19+ (n=79) or TCRαβ+/CD19+ (n=80) platforms between 2005 and 2020 were included. Median age was 9 years in both groups. There were no differences in patient, donor, and transplant characteristics between groups except for donor KIR B genotype more frequent in the TCRαβ+/CD19+ group (91%) than in the CD3+/CD19+ group (76%) (p=0.009) and a high number of NK+ cells and lower CD19+ cells infused in the TCRαβ+/CD19+ group (35.32x106/kg and 0.06 x106/Kg) than in the CD3+/CD19 group (24.6x106/Kg and 0.25 x106/Kg) (p=0.04 and p=0.0001), respectively. Conditioning was based on TBF. Median follow-up for survivors was 11 years (range; 8-16 y) in CD3+/CD19+ group and 5 years (range; 2-9 y) in the TCRαβ+/CD19+ group.ResultsEngraftment kinetics were similar in both groups (13 days for neutrophils and 10 days for platelets). There was no difference in the incidence of acute GvHD II-IV (29 ± 5% in the CD3+/CD19+ group vs 38 ± 5% in the TCRαβ+/CD19+ group) and chronic GvHD (32 ± 5% vs 23 ± 4%, respectively). NRM was 23 ± 5% in the CD3+/CD19+group vs 21 ± 4% in the TCRαβ+/CD19+group. Relapse incidence was also similar, 32 ± 5% vs 34 ± 6%, respectively. DFS and OS were not different (45 ± 5% vs 45 ± 6% and 53 ± 6% vs 58 ± 6% respectively). As there were no differences on transplant outcomes between groups, we further analyzed all patients together for risk factors associated with transplant outcomes. On multivariate analysis, we identified that early disease status at transplant (HR: 0.16; 95%CI (0.07-0.35) (p=0.0001), presence of cGvHD (HR: 0.38; 95%CI (0.20-0.70) (p= 0.002), and donor KIR-B genotype (HR: 0.50; 95%CI (0.32-0.90) (p=0.04) were associated with better DFS.ConclusionsOur data suggest that there are no advantages in transplant outcomes between TCD platforms. Risk factors for survival are dependent on disease characteristic, donor KIR genotype, and chronic GvHD rather than the TCD platform used.

Primary Liver Transplantation vs. Transplant after Kasai Portoenterostomy for Infants with Biliary Atresia.

Introduction: Primary liver transplants (pLT) in patients with biliary atresia (BA) are infrequent, since most babies with BA undergo a prior Kasai portoenterostomy (KPE). This study compared transplant outcomes in children with BA with or without a prior KPE. We hypothesized that pLT have less morbidity and better outcomes compared to those done after a failed KPE. Methods: A retrospective review of patients with BA transplanted at our institution was performed. Patients were included if they received a pLT or if they were transplanted less than 2 years from KPE. Outcomes were compared between those groups. Comparisons were also made based on era (early: 1997–2008 vs. modern: 2009–2020). p < 0.05 was considered significant. Results: Patients who received a pLT were older at diagnosis (141.5 ± 46.0 vs. KPE 67.1 ± 25.5 days, p < 0.001). The time between diagnosis and listing for transplant was shorter in the pLT group (44.5 ± 44.7 vs. KPE 140.8 ± 102.8 days, p < 0.001). In the modern era, the calculated PELD score for the pLT was significantly higher (23 ± 8 vs. KPE 16 ± 8, p = 0.022). Two waitlist deaths occurred in the KPE group (none in pLT, p = 0.14). Both the duration of transplant surgery and transfusion requirements were similar in both groups. There was a significant improvement in graft survival in transplants after KPE between eras (early era 84.3% vs. modern era 97.8%, p = 0.025). The 1-year patient and graft survival after pLT was 100%. Conclusions: Patient and graft survival after pLT are comparable to transplants after a failed KPE but pLT avoids a prior intervention. There was no significant difference in pre- or peri-transplant morbidity between groups other than wait list mortality. A multicenter collaboration with more patients may help demonstrate the potential benefits of pLT in patients predicted to have early failure of KPE.

Alterations to the Intestinal Microbiome Are Associated with Post-Heart Transplant Outcomes in Children

<h3>Purpose</h3> Alterations to the intestinal microbiome are associated with mortality after stem cell transplant in adults. We hypothesize that alterations to the intestinal microbiome occur in children after pediatric heart transplant (HTx) and are associated with post-HTx outcomes. <h3>Methods</h3> Pre-HTx and post-HTx stool specimens were prospectively collected from 9/2018 - 7/2020. Bacterial DNA was extracted and processed through an Illumina MiSeq 16S sequencing pipeline. Separate regions of the 16S rRNA gene, V1V3 and V4, were targeted for sequencing. Resulting raw sequences were analyzed utilizing the UPARSE algorithm for clustering of sequences into operational taxonomic units (OTUs) and the SILVA v123 database for taxonomic classification of each OTU. The outcome was a composite "event" (infection, graft rejection, or transplant coronary artery disease) at the time of stool collection. <h3>Results</h3> There were 316 stool specimens (138 pre-HTx, 178 post-HTx) collected from 105 patients, including from 28 patients with sequential samples who underwent HTx during the study period. There are compositional differences between pre-HTx vs post-HTx patients (Fig 1A), most notably in the abundance of <i>Bacteroides, Enterococcus</i>, and <i>Akkermansia</i> species, and compositional shifts depending on time elapsed from HTx (Fig 1B). There were also specific compositional differences among patients with post-HTx events (most notably in the abundance of <i>Clostridium and Ruminococcus</i> species<i>;</i> Fig 1C) and among patients with post-HTx rejection (most notably in <i>Enterococcus, Bifidobacterium, and Akkermansia</i> species; Fig 1D). <h3>Conclusion</h3> Compositional alterations to the intestinal microbiome occur in children undergoing HTx. Specific differences were observed in children experiencing post-HTx composite events, including specific compositional changes among children with post-HTx rejection. Whether these compositional alterations are a cause or effect, or if they can be reversed, remains to be elucidated.

Multicentre randomised controlled trial: protocol for Plasma-Lyte Usage and Assessment of Kidney Transplant Outcomes in Children (PLUTO)

IntroductionAcute electrolyte and acid–base imbalance is experienced by many children following kidney transplantation. When severe, this can lead to complications including seizures, cerebral oedema and death. Relatively large volumes of...

Impact of initial steroid response on transplant outcomes in children with steroid-resistant nephrotic syndrome.

Limited data suggest children with secondary steroid-resistant nephrotic syndrome (secondary SRNS) have increased risk of recurrence post transplantation. There are no data on the association between secondary steroid resistance and risk of transplant loss. Children who received kidney transplantation between 2000 and 2019 for either primary or secondary SRNS in Australia and New Zealand were included. Children presenting with nephrotic syndrome before 12months were excluded. Data were gathered from chart reviews and ANZDATA. Transplant survival was estimated using the Kaplan-Meier estimator with Cox modelling used to explore predictors of survival. There wereseventy children, 38 (55%) male, median age at presentation 4years (IQR 2-7) and 46 (66%) Caucasian. Median age at transplant was 11years (IQR 7-15) and 39 (55%) received living donor transplant. Secondary SRNS occurred in 20/70 (29%). For those with secondary SRNS, 18/20 (90%) had recurrence post-transplant, compared to 18/50 (36%) with primary SRNS (p = 0.001). Every child with history of atopy (n = 11) or with hypoalbuminaemia at time of transplant (n = 13) experienced immediate recurrence. For children with secondary SRNS, 8/18 (44%) with post-transplant recurrence had no response to therapy. For children with primary SRNS, 4/18 (22%) with recurrence had no response to therapy (p = 0.3). Overall, 10-year transplant survival was 47% (95%CI 29-77%) for those with secondary SRNS, compared to 71 (95%CI 57-88%) for those with primary SRNS (p = 0.05). Secondary steroid resistance is strongly associated with SRNS recurrence. Atopy and hypoalbuminaemia at transplant may be novel risk factors for recurrence. Further research is needed to assess if secondary steroid resistance is associated with poorer transplant outcomes. "A higher resolution version of the Graphical abstract is available as Supplementary information".

Kidney transplant outcomes in children and adolescents with systemic lupus erythematosus.

Literature supports equivalent kidney transplant outcomes in adults with systemic lupus erythematosus (SLE) compared with those without SLE. However, there are conflicting and scant data on kidney transplant outcomes, as well as controversy over optimal timing of transplantation, in children and adolescents with SLE. Analysis included kidney-only transplant recipients aged 2-21years from 2000 to 2017 enrolled in the Organ Procurement and Transplant Network (OPTN). The relationship between diagnosis (SLE n = 457, non-SLE glomerular disease n = 4492, and non-SLE non-glomerular disease n = 5605) and transplant outcomes was evaluated. The association between dialysis time and outcomes was analyzed in the SLE group only. In adjusted models, SLE had higher mortality compared with non-SLE glomerular recipients (HR 1.24 CI 1.07-1.44) and non-glomerular recipients (HR 1.42 CI 1.20-1.70). SLE was associated with higher graft failure compared with non-SLE glomerular (HR 1.42 CI 1.20-1.69) and non-glomerular disease (HR 1.67 CI 1.22-2.28). SLE had a higher risk of acute rejection at 1year compared with non-glomerular disease (HR 1.39 CI 1.03-1.88). There was a decreased risk of delayed graft function compared with non-SLE glomerular disease (HR 0.54, CI 0.36-0.82). There were no significant associations between dialysis time and transplant outcomes in the SLE group. SLE in children and adolescents is associated with worse patient and graft survival compared with non-SLE diagnoses. Outcomes in children and adolescents with SLE are not associated with dialysis time. Further studies are needed to assess implications of potential earlier transplantation and shorter time on dialysis prior to transplantation.

Impact of Palifermin on Transplant Outcomes in Children and Adolescents Undergoing Allogeneic Hematopoietic Cell Transplantation.

Oral mucositis (OM) is considered to be one of the worst and most debilitating complications of conditioning for hematopoietic cell transplantation (HCT). Prevention and treatment of this complication is one of the utmost priorities of supportive therapy during transplant procedure. The objective of this study was the analysis of the influence of palifermin, keratinocyte growth factor (KGF), on transplant outcomes in patients undergoing allo-HCT. A total of 253 allo-HCTs performed between 2003-2018 in patients aged 0-19 years at a single center were analyzed. KGF was administered in 161 HCTs. Uni- and multivariate risk factor analyses were performed. In spite of reducing the duration and grade of mucositis, no prognostic impact of KGF was shown for overall survival, event-free survival, relapse incidence, acute and chronic graft-versus-host disease (GVHD), nor GVHD-free relapse-free survival. Palifermin had no impact on transplant outcomes in children and adolescents undergoing allo-HCT.

Transplant Outcomes in Children with Lupus Nephritis

Transplant Outcomes in Children with Lupus Nephritis

Fludarabine, Campath, and Low-Dose Cyclophosphamide (FCClow) with or without TBI Conditioning Results in Excellent Transplant Outcomes in Children with Severe Aplastic Anemia.

Various reduced-intensity conditioning regimens are in use for allogeneic hematopoietic cell transplant (HSCT) in patients with idiopathic severe aplastic anemia (SAA). We describe the use of fludarabine, Campath, and low-dose cyclophosphamide (FCClow) conditioning in 15 children undergoing related or unrelated donor transplants. Total body irradiation (TBI) of 2 Gy was added for unrelated donor HSCT. At a median follow-up of 2.3 years, the failure-free survival was 100%, with low rates of infection and toxicity. There was no occurrence of grade III to IV acute graft-versus-host disease (GVHD). All patients had full donor myeloid chimerism post-HSCT, even with mixed chimerism in the T cell lineage. The absence of chronic GVHD and long-term stable mixed donor T cell chimerism confirms immune tolerance following FCClow (± TBI) conditioned transplantation in children with SAA.

Heart Transplant Outcomes in Children with Ebstein's Anomaly

Large variability exists in the medical and surgical management strategies for patients with Ebstein's anomaly (EA). However, in severe cases, options may be limited to heart transplantation (HT). Despite this, little data regarding outcomes of HT in EA patients exist to guide decision-making in this population. We report outcomes following HT in children with EA and describe characteristics associated with a poor HT outcome in the EA population. The Scientific Registry of Transplant Recipients and Pediatric Health Information System databases were linked at the patient level using indirect identifiers (2002 - 2016). Pediatric HT recipients with a diagnosis of congenital heart disease (CHD) were included, and those with a diagnosis of EA were identified using pre-HT ICD9/10 codes. The log-rank test was used to compare graft survival in HT recipients with EA and those with other forms of CHD. Post-HT morbidities including extracorporeal membrane oxygenation (ECMO), dialysis, stroke and rejection were also compared between groups. Cox regression was used to analyze factors associated with graft loss in the EA population. A sub-analysis of EA patients listed for HT at age ≤6 months was performed to determine if survival in this population differed from older EA patients following HT. A total of 1453 patients were identified for inclusion and 58 (4%) had EA. Of the EA patients 26 (44.8%) were male, median age at HT was 4 years (IQR 0-11 years), and 6 (10.3%) had undergone prior cardiac surgery. Support at the time of transplant for the EA group included mechanical ventilation (n=45, 77.6%), ECMO (n=6, 10.3%) and ventricular assist device (n=4, 6.9%). There was no difference in 5-year graft survival following HT in patients with EA vs other forms of CHD (76% vs 73%, p=0.84). Pre-HT ECMO was associated with graft loss in the EA population (HR 3.6, 95%CI 1.1-11.3). Between EA and CHD groups, no difference was detected in post-HT morbidities including ECMO (5.2% vs 9.1%, p=0.3), dialysis (8.6% vs 7%, p=0.64), stroke (0% vs 3.8%, p=0.13), or rejection (8.2% vs 17%, p=0.1). Sub-analysis of 19 (32.7%) EA patients listed for HT at ≤6 months of age showed no difference in 5-year survival compared to older patients with EA (83% vs 74%, p=0.88). Children with EA have similar outcomes after HT when compared with all types of CHD. Therefore, HT should remain a consideration if otherwise indicated in this population.

Heart Transplant Outcomes in Children with Fontan-Associated Plastic Bronchitis: A Pediatric Heart Transplant Society Analysis

Plastic bronchitis is a rare post Fontan complication with limited treatment options. Heart transplantation has evolved as a potential curative option, but outcomes have not been well-defined. This study aims to assess contemporary waitlist and post-transplant outcomes in patients with plastic bronchitis. All Fontan patients were identified in the Pediatric Heart Transplant Society database (2010-2018). Waitlist and post-transplant outcomes were compared between Fontan patients with and without plastic bronchitis. Competing outcomes and Kaplan Meier analyses were used to assess the impact of plastic bronchitis on waitlist and post-transplant survival. A secondary analysis excluded those with protein losing enteropathy (PLE) from the comparison cohort. Of 645 Fontan patients listed for heart transplant, 69 (8%) had plastic bronchitis. At listing, patients with plastic bronchitis were younger (9.2 vs 10.6 years, p=0.02), but had no other differences at baseline including hemodynamic data. Fewer Fontan patients with plastic bronchitis were listed in the more recent era (46 [15.4%] in 2010-2014 vs. 23 [6.6%] in 2015-2018, p<0.01). Overall, there was no difference in waitlist (p=0.30) or post-transplant (p=0.66) survival [Figure]. The results were similar after excluding patients with PLE. Contrary to prior reports, in this large series plastic bronchitis did not have a negative impact on survival to or after heart transplantation in Fontan patients. A 50% reduction in listing in the most recent era suggests improvement in medical management of this rare and life-threatening condition.

Trends in kidney transplant outcomes in children and young adults with cystinosis.

Temporal changes in kidney transplant outcomes for cystinosis are unknown. We used the SRTR to identify all kidney transplants performed for cystinosis in patients younger than 31years between 1987 and 2017. We divided time into three equal eras (1987-1997, 1998-2007, and 2008-2017) to assess changes in outcomes using Cox proportional and linear regression models. We examined 441 transplants in 362 patients. Age at ESRD progressively increased (12.1 vs 13.3 vs 13.4; P=.046). Eras 2 and 3 had lower risk of acute rejection (aHR 2 vs 1:0.45; P<.001) (aHR 3 vs 1:0.26; P<.001) and higher 5-year mean GFR (difference 2 vs 1:9.2mL/min/1.73m2 ; P=.005) (difference 3 vs 1:12.9mL/min/1.73m2 ; P=.002) compared with era 1. Five-year graft survival was similar across eras, but 5-year patient survival was higher for era 2 (aHR: 0.25; P=.01). Seventy-nine patients underwent retransplantation. Five-year patient (94.2% vs 92.5%; P=.57) and graft survival (79.1% vs 74.1%; P=.52) were similar between primary and subsequent transplants. Age at ESRD, acute rejection, GFR at 5years, and patient survival improved over time. Kidney retransplantation is associated with excellent outcomes in children and young adults with cystinosis.

SUN-107 mTORI VERSUS CNI BASED IMMUNOSUPPRESSIVE REGIMENS FOR PAEDIATRIC KIDNEY TRANSPLANTATION

Mammalian target of rapamycin inhibitors (mTORi) have been proposed as an alternative to calcineurin inhibitors (CNI) in long term immunosuppression regimens for kidney transplantation. Their potential benefits include avoidance of the nephrotoxic effects of CNIs and lower rates of viral infection. However, some studies have shown an increased risk of rejection. Little data exists on kidney transplant outcomes in children receiving mTORi. We compared the outcomes of kidney transplantation in children on mTORi containing immunosuppressive regimens versus CNI containing regimens. Data for patients <18 years who received their first transplant between 1 January 2000 to 31 December 2015 were extracted from the Australia and New Zealand Dialysis and Transplant (ANZDATA) registry. A matched cohort study design was used to compare graft survival for patients who switched from CNI to mTORi between 2 and 24 months post transplantation and continued on the mTORi for 12 months or greater and a control group matched on age, gender, year of transplant and donor source who were receiving CNI based immunosuppression. Kaplan-Meier method with Cox regression was used to compare graft survival in the first 10 post-transplant years. Of 590 first kidney transplants in patients < 18 years of age, 40 patients switched from CNI to mTORi between 2-24 months post-transplant and remained on the mTORi for 12 months or greater. These patients were matched to 120 patients who remained on CNI. There were 4 graft losses in the mTORi group and 32 in the CNI group. Graft survival was greater in the mTORi group with 10-year graft survival 87% and 72.5% in the matched control group (X21=5.22, p =0.02). Graft survival at 10 years post first kidney transplant in paediatric patients on mTORi containing immunosuppressive regimens appears to be superior to those on CNI containing regimens. Our study is limited by small numbers and short duration of mTORi exposure in some patients. Further studies in this population are needed to determine if mTORi based immunosuppressive regimens are superior to CNI based regimens in paediatric kidney transplantation.

Risk Factors for Transplant Outcomes in Children and Adolescents with Non-Malignant Diseases Following Allogeneic Hematopoietic Stem Cell Transplantation.

BackgroundThe objective of this study was the analysis of transplant outcomes and survival in children treated with allogeneic hematopoietic cell transplantation (alloHCT) for non-malignant disorders, with a focus on risk factor analysis of transplant-related mortality (TRM).Material/MethodsThe treatment outcome was analyzed retrospectively in 10 consecutive years in 4 pediatric transplant centers in Poland. To compare the outcomes, patient data were analyzed according to the diagnosis, age at transplant, donor type, stem cell source, conditioning regimens, transplanted CD34+ cells dose, and pediatric TRM score.ResultsFrom 183 analyzed patients, 27 (14.8%) died, all of them due to transplant-related complications. TRM occurred more frequently in matched unrelated donor (MUD) transplant recipients vs. matched sibling donor (MSD) transplant recipients (p=0.02); in peripheral blood (PB) recipients vs. bone marrow (BM) recipients (p=0.004); and in patients receiving >5×106/kg CD34+ cells (p<0.0001). OS differed significantly according to underlying disease comparing to other diagnoses. Lower survival was found in patients transplanted from MUD (p=0.02). OS was higher in patients receiving BM (p=0.001) and in those receiving ≤5×106/kg CD34+ cells (p<0.001). Multivariate analysis showed lower probability of TRM in BM recipients (p=0.04). The probability of TRM was higher in SCID patients (p=0.02) and in patients receiving >5×106/kg CD34+ cells (p=0.0001).ConclusionsUnderlying disease, stem cell source, and CD34+ dose higher than 5×106/kg were the most important risk factors for TRM, and they all affected OS.

The Number of Refusals for Donor Organ Quality Does Not Impact Heart Transplant Outcomes in Children

Waitlist mortality is more than 12% for pediatric heart transplantation, with strikingly high rates of organ refusal, many of which are due to donor quality. However, some centers use these organs despite refusals by other centers for donor quality. We hypothesize that the number of refusals for donor quality (RDQ) does not affect pediatric heart transplantation outcomes. Pediatric heart transplants from 2000 to 2015 were identified using the United Network for Organ Sharing database and were matched against the potential transplant recipients dataset with donor refusal codes. Refusals for donor quality were counted for each organ. The population was divided into two groups: RDQ-low (0 to 3 RDQs, n= 3,404) and RDQ-high (>3 RDQs, n= 1,585). Posttransplant outcomes of both cohorts were analyzed. Of 4,989 pediatric heart transplants, 75% (n= 3,770) had 1 or more RDQ (median 3 RDQ; interquartile range, 1 to 7). The RDQ-lower group (0 to 3 RDQs) and the RDQ-higher group (>3 RDQ) had similar posttransplant survival (p= 0.41) and freedom from retransplantation (p= 0.37). Both groups had similar posttransplant survival even for high-risk recipient cohorts: adolescents (p= 0.06), congenital heart disease (p= 0.87), retransplantation (p= 0.47), extracorporeal membrane oxygenation (p= 0.61), mechanical ventilation (p= 0.24), and poor renal function at transplant (p= 0.46). In addition, recipient subgroups who had donors with increasing number of RDQ (>6, >9, >12, and >15 RDQ) also had similar posttransplant survival compared with the RDQ-low group (p= 0.63, p= 0.62, p= 0.92, and p= 0.50, respectively). The outcome of pediatric heart transplantation is not affected by the number of RDQ, even for high-risk recipients. The use and interpretation of donor quality refusal code should be considered carefully while selecting or refusing donors in this era of supply and demand mismatch.

Pretransplant Panel Reactive Antibodies and Lung Transplant Outcomes in Children.

Background Pre-lung transplant (LTx) panel reactive antibody (PRA) levels are associated with adverse outcomes in adult LTx recipients, but their impact in pediatric LTx recipients is unknown. Methods The United Network for Organ Sharing registry was queried from 2004 to 2013 to compare survival between pediatric LTx recipients with PRA class I and II levels = 0 versus > 0. Results Overall, 333 pediatric LTx recipients had data on class I or II PRA and were included in the analysis. Univariate analysis demonstrated that PRA > 0 was not associated with survival benefit for class I (hazard ratio [HR] = 0.985; 95% confidence interval [CI]: 0.623, 1.555; p = 0.947) or class II (HR = 1.080; 95% CI: 0.657, 1.774; p = 0.762) PRA. Multivariate Cox models confirmed no significant association with mortality hazard for both class I (HR = 1.230; 95% CI: 0.641, 2.363; p = 0.533) and class II (HR = 0.847; 95% CI: 0.359, 1.997; p = 0.704) PRA. Multivariate logistic regression models identified no association between class I or class II and acute rejection within 3 years of LTx. Conclusions Pretransplant class I and II PRA levels > 0 were not associated with mortality or acute rejection in pediatric LTx recipients.

Role of race in kidney transplant outcomes in children with focal segmental glomerulosclerosis.

It is well established that racial differences exist in kidney transplant outcomes; however, there are no studies which focus on the role of race in transplant outcomes specifically in children diagnosed with FSGS. Associations between race and transplant outcomes in FSGS children were evaluated using the Organ Procurement and Transplantation Network database from 2000 to 2012. Recipients aged 2-21years who received a kidney-only transplant were included. Multivariate regression models were used to evaluate transplant outcomes by race. Five hundred and thirty-six recipients (59.7% male, 15.6±3.9years) were black and 1134 (55.7% male, 14.3±5.0years) were non-black. Graft survival was significantly shorter in the black group (4.2±3.1 vs 4.6±3.3years, P=.005). Black race was associated with significantly higher risk of graft failure (HR 1.34, 95% CI=1.21-1.49, P<.0001), acute rejection (OR 1.66 95% CI=1.39-1.97, P<.0001), and delayed graft function (OR 1.51, 95% CI=1.33-1.72, P<.001) compared to non-black race. There were no significant differences in mortality, prolonged hospitalization, or FSGS recurrence between groups. Race is a significant predictor for worse transplant outcomes in children with FSGS.

The Impact of Palifermin Use on Hematopoietic Cell Transplant Outcomes in Children

Clinical trials evaluating palifermin have enrolled few pediatric patients, precluding safety analyses in large groups of children. We compared hematopoietic cell transplantation (HCT) outcomes among pediatric patients who did or did not receive palifermin as a preventive treatment for oral mucositis. Pediatric patients and controls, matched for HCT and donor type, disease, disease status, and age, were selected from the Center for International Blood and Marrow Transplant Research database and a 1:3 matched cohort analysis was performed. Stratified Cox proportional hazards models were built and propensity score adjustments were used to compare overall and disease-free survival outcomes between palifermin-treated and untreated patients. Three controls were identified for 90% of palifermin recipients. The remaining cases were matched with 2 (8%) controls or 1 (2%) control, for a total of 210 palifermin-treated patients matched with 606 controls. Median follow-up was 31 months in cases and 36 months in controls. Fifty-seven percent of patients underwent allogeneic HCT, mostly for acute leukemia, and 43% underwent autologous HCT, mostly for solid tumors. In univariate analyses, 2-year survival and disease-free survival rates after allogeneic HCT (58% versus 66%, P = .109; 49% versus 60%, P = .06) and after autologous HCT (73% versus 77%, P = .474; 60% versus 64%, P = .637) were similar between palifermin-treated patients and matched controls. In multivariate analysis, palifermin treatment did not significantly increase the risk of mortality (relative risk [RR], 1.20; 95% confidence interval [CI], .87 to 1.66) or of relapse (RR, 1.12; 95% CI, .78 to 1.62) compared with matched controls. No significant differences in rates of acute or chronic graft-versus-host disease (GVHD) were observed between palifermin-treated patients and matched controls. Among pediatric patients undergoing HCT, overall survival, disease-free survival, neutrophil recovery, and GVHD rates were similar between palifermin-treated patients and matched controls.