According to the 2008 World Health Organization classification, low-grade lymphomas arising in transplant recipients are not considered as specific types of PTLD. Most such cases are not associated with EBV infections, although rare reports of post-transplant marginal zone lymphoma have been described. We describe the case of an 18-yr-old female with history of heart transplant who developed a breast mass, but was otherwise completely asymptomatic. Surgical excision of the mass and histopathologic examination showed a low-grade B-cell lymphoma most consistent with marginal zone lymphoma with massive amyloid deposition; furthermore, numerous tumor cells were positive for EBV by in situ hybridization for EBV-encoded RNA. The patient was treated with reduction in immunosuppression, and no additional lesions developed. This case describes an atypical presentation of post-transplant low-grade B-cell lymphoma, unusual in its location, histopathologic features, and association with EBV, thereby adding to the rare previous accounts of such an entity, suggesting the need to include post-transplant marginal zone lymphomas in the current classification of PTLD, and helping in determining the optimal treatment modalities for such tumors.

WAS is a severe X-linked recessive disorder characterized by microthrombocytopenia, eczema, and immunodeficiency. A six-yr-old boy with WAS diagnosed as B-cell NHL (Stage III) localized in the liver who underwent successful HSCT from HLA-one antigen mismatch sibling donor has been presented here. His conditioning regimen included ATG, busulfan, and fludarabine. He received 2.3 × 10⁶/kg CD 34(+) stem cells and 11 × 10⁸/kg nucleated cells at day 0. Neutrophil engraftment was achieved at day +14 and platelet engraftment at day +20. He has been in CR for more than two yr after transplantation. Thus, HSCT is an effective treatment for children with WAS even after development of lymphoma.

VIS, a quantitative index of pressor support, has been shown to be a predictor of morbidity and mortality in infants younger than six months who underwent CPB. Data on its prognostic utility following pediatric OHT are lacking. This study compared clinical outcomes in children with differential VIS after pediatric OHT. A retrospective cohort study of 51 consecutive heart transplants from 2004 to 2011 was performed at a pediatric tertiary care facility. Peak VIS was computed within initial 24 and 48 h after OHT and was weighted for peak dose and administration of any or all of six pressors. Patients with peak VIS ≥ 15 constituted high VIS group. Children who persistently required a higher magnitude of pressor support for the first 48 h after OHT, as reflected by high peak VIS, had significantly longer ICU stay (30.2 vs. 15.9 days, p = 0.01), pressor (11.4 vs. 6.8 days, p = 0.02) and ventilatory durations (12.4 vs. 5.9 days, p = 0.05), and higher rates of short-term morbidities. Patients with longer CPB (213 vs. 153 min, p = 0.005) time have higher peak VIS. High peak VIS at 48 h is an effective, yet simple clinical marker for adverse outcomes in pediatric OHT recipients.

OM is a frequent complication for patients undergoing HSCT. The aim of this study was to evaluate the possible risk factors for OM in children undergoing HSCT for PI. A retrospective study was carried out on 55 consecutive medical records of patients with PI (including osteopetrosis) who underwent HSCT. Age at the time of HSCT, gender, diagnosis, type of donor, conditioning regimen, engraftment, graft-versus-host disease, and severity and duration of OM were collected at the beginning of the conditioning until day 100 post-HSCT or death. OM was measured using the WHO scale. Patients' age range at the time of HSCT was quite wide; 59.2% of the patients who were under nine months (n = 13) developed OM vs. 87.8% of the patients older than nine months (n = 29) (p = 0.01). T-cell positive patients had a statistically significant higher risk of developing OM (p = 0.04), as did those receiving a more intensive conditioning regimen (p < 0.01). PI patients undergoing HSCT are at higher risk of developing OM if the PI is a T-lymphocyte-positive disease and/or if the HSCT recipient is over nine months of age.

Hypertension and reduced HRV are frequent in heart-transplanted recipients. We studied 26 young recipients to investigate the relationship between BP and HRV during simultaneous 24-h monitoring. Presence of CAV was considered. All HRV measures were significantly lower than normal values. Significant correlations were found between mean daytime systolic BP and the rMSSD (p = 0.04), and mean daytime DBP and SDANN for all 5-min segments (p = 0.03) and between rMSSD and mean nighttime DBP (p = 0.03). Four patients were hypertensive during daytime, seven had a reduced nocturnal fall and two had a nocturnal rise in BP. Eight patients showed severe CAV grade (grade IV) on the Stanford scale, 13 moderate (grade III) and five mild (grade I-II). After a follow-up time of 30 months, four of the 13 patients (30%) with CAV grade III showed an increase to grade IV and all showed abnormalities of both HRV and ABPM patterns. The relationship between HRV abnormalities and arterial hypertension and CAV should be further explored.

Transition from pediatric to adult care is a critical and difficult step for young people with transplants and for the multidisciplinary team involved. In our retrospective study, we investigated the clinical course in a two-yr period of transition. Data from 66 teenagers were collected one yr before and after their transfer to three different adult care settings: (i) a specialized transition clinic, (ii) a general transplantation clinic, and (iii) a nephrologist. Patient survival rate was 100%. Three patients developed graft loss. GFR development was comparable in the three settings (ΔGFR 1.4 ± 8.7 vs. 3.1 ± 10.6 vs. 0.8 ± 4.4 mL/min/1.73 m², p = ns). Immunosuppressive therapy was stable in setting 1, whereas the number of changes increased in setting 2 and even more in setting 3. The percentage of patients with steroids increased from 36% to 38% and 52% in settings 1–3. Patient satisfaction was highest in setting 1 (100% vs. 64% and 78%, p < 0.05). Setting 1 was associated with fewer changes in therapy (13% vs. 91% and 45%, p < 0.05). The use of a specialized transition clinic is associated with fewer changes in medication and care and a higher level of patient satisfaction. This was not associated with a lower increase in GFR one yr after transition. Long-term results are awaited.

Small children are a challenging group in whom to perform KT. This retrospective study analyzed the results of 62 KTs in children weighing <15 kg, performed between 1998 and 2010, using extraperitoneal access and anastomosis of the renal vessels of donors to the aorta and IVC or iliac vessels of the recipients. Thirty-two (51.6%) grafts were LRDTs and 30 (48.4%) were DDRTs—28 of them pediatric. The mean age at KT was 3.7 ± 2.2 yr (1–12), and the mean weight was 12.3 ± 2.1 kg (5.6–14.9). Ten children weighed <10 kg, and five (8.1%) children presented previous thrombosis of the venous system. At one and five yr, patient survival was 93.2% and 84.2%, and graft survival was 85.2% and 72.7%. There were no differences between the rates for LRDT and DDRT. There were six vascular complications (four vascular thromboses, one laceration, and one renal artery stenosis) and two perirenal collections. Extraperitoneal access is a valid KT technique in children weighing <15 kg.

PTLDs are a well-recognized and potentially fatal complication after intestinal transplantation. We analyzed the incidence, clinical features, and outcome in a 63 intestinal transplantation series performed in our unit between October 1999 and July 2011. Types of graft included ISB (n = 23), LSB (n = 20), and MV (n = 20). Patients were categorized into three groups of immunosuppression: I (n = 43) received basiliximab, tacrolimus, and steroids; II (n = 11) thymoglobulin and tacrolimus, and III (n = 9) alemtuzumab and tacrolimus. EBV status was serially assessed. All PTLD cases were biopsied to establish histopathological diagnosis. The incidence of PTLD was 14.2% (9/63). Median onset of PTLD after transplant was four months (range: 0.5–28), within first postoperative year in 6 (66.6%) patients. Fever was the most common symptom. Graft removal was needed in four patients (44%). The patient survival rate was 66.6% (6/9). We have not found any association between PTLD and immunosuppression regimen or transplant type. However, there was a statistical association with EBV active infection.

AIHA is a rare and serious complication of solid organ transplantation. Herein, we report four cases of warm or mixed AIHA in pediatric patients following combined liver, small bowel and pancreas transplant. The hemolysis was refractory to multiple treatment modalities including steroids, rituximab, IVIG, plasmapheresis, cytoxan, discontinuation of prophylactic penicillin, and a change in immunosuppression from tacrolimus to cyclosporine. All patients had resolution or marked improvement of hemolysis after discontinuation of maintenance of CNI and initiation of sirolimus immunosuppression. One patient developed nephrotic syndrome but responded to a change in immunosuppression to everolimus. Three of the four patients continue on immunosuppression with sirolimus or everolimus without further hemolysis, evidence of rejection or medication side effects. Based on our experience and review of similar cases in the literature, we have proposed a treatment algorithm for AIHA in the pediatric intestinal transplant patient population that recommends an early change in immunosuppressive regimen from CNIs to sirolimus therapy.

Amanita phalloides intoxication can lead to FHF with high mortality, especially in children. There is still ongoing discussion about the optimal treatment and decision criteria for emergency liver transplantation (LTx). Here, we summarize our experience with outcomes in five children. Five children with severe A. phalloides intoxication were treated at our tertiary center from 1995 to 2010 and studied retrospectively with respect to clinical and laboratory aspects that might help to decide between LTx or conservative therapy only. The findings are discussed with regard to recommended treatment and transplantation criteria for adults. All patients survived, of whom two of five received emergency LTx. Three patients survived with conservative treatment consisting of intravenous silibinin, NAC, detoxification measures, and intensive care. Indications for LTx in two children were progressive brain edema and cardiovascular failure. Children with FHF due to A. phalloides intoxication should be considered early for emergency LTx but should be monitored closely for the necessity of definite LTx. Early detoxification with active charcoal as well as silibinin and NAC seems to improve the outcome. Late recovery of liver function after day 4 post-ingestion is possible.

This review focuses on active clinical research in pediatric liver transplantation with special emphasis on areas that could benefit from studies utilizing the SPLIT infrastructure and data repository. Ideas were solicited by members of the SPLIT Research Committee and sections were drafted by members of the committee with expertise in those given areas. This review is intended to highlight priorities for clinical research that could successfully be conducted through the SPLIT collaborative and would have significant impact in pediatric liver transplantation.

Partial albinism with variable immunodeficiency are the two major characteristics of Griscelli syndrome type 2 (GS-2). This syndrome is usually associated with a high mortality rate and commonly results in early childhood death. Patients suffer from different infections and experience crisis of HLH. HSCT remains the sole curative treatment for GS-2. We prospectively analyzed the outcomes of transplantation with RIC regimen in five patients. The median age at transplantation was 21.6 months (range: 12–30). All of the patients underwent HSCT from HLA-matched related donors. Currently, four patients are cured, and symptoms of recurrent infections and HLH crisis are not seen in them. The only patient who died had undergone HSCT in the accelerated phase of HLH. One patient who developed acute GvHD had a favorable response to therapy. No chronic GvHD occurred in patients. It seems that the use of RIC regimen as a method of transplant preparation is effective and tolerable in this group of patients with various comorbidities. It is recommended to carry out HSCT in these patients at lower ages, before presentations of different infections and HLH crisis.

CAV remains a leading cause of late graft loss and mortality among survivors of pediatric heart transplantation. We sought to define the incidence of CAV and identify its predictors in pediatric heart transplant recipients. The OPTN/UNOS database was analyzed for pediatric recipients who underwent heart transplant between 1987 and 2011. The primary end-point is time from heart transplantation to development of CAV (CAV-free survival). To identify predictors of CAV-free survival, demographic and transplant data were analyzed by the Kaplan–Meier survival method and Cox proportional hazards regression. Of 5211 pediatric heart transplant recipients with at least one-yr follow-up, the incidence of CAV at five, 10, and 15 yr was 13%, 25%, and 54%, respectively. Multivariate analysis found that risk of CAV was associated with the following variables: Recipient age 1–4 yr (HR 1.25), 5–9 yr (1.45), 10–18 yr (1.83), donor age >18 yr (1.34), re-transplantation (2.14), recipient black race (1.55), and donor cigarette use (1.54). Older recipient and donor age, recipient black race, donor cigarette use, and re-transplantation were highly associated with shorter CAV-free survival.

Orthotopic heart transplantation remains the definitive treatment of choice for patients with end-stage heart failure; however, elevated PVRI is a reported risk factor for mortality after heart transplant and, when severely elevated, is considered an absolute contraindication. Use of a ventricular assist device has been proposed as one treatment for reducing pulmonary vascular resistance index in potential heart transplant candidates refractory to medical vasodilator therapies. We report on a teenage patient with dilated cardiomyopathy and severely elevated PVRI, unresponsive to pulmonary vasodilator therapy, who underwent left ventricular assist device implantation to safely allow for aggressive pulmonary vasodilator therapy and to decrease PVRI. The resulting dramatic improvement in PVRI in a relatively short period of time allowed for successful heart transplantation, avoiding the need for heart–lung transplant.

With a limited number of pediatric lung transplant programs, the transfer of patients will be required for appropriate candidates. Pediatric patients have been successfully transferred using extracorporeal membrane oxygenation (ECMO) with no previous reports using ambulatory single-site venovenous (VV) ECMO via a bicaval dual-lumen (BCDL) catheter as a method for transport to a lung transplant center in order to bridge to lung transplantation. Therefore, we present the successful transfer of a 13-yr-old female on ambulatory VV ECMO between two free-standing children's hospitals and then bridged to bilateral lung transplantation.

FNH is a non-malignant neoplasia of the liver rarely described in children. A significant percentage of the pediatric cases have been reported in patients with a history of malignant disease treated with chemo-radiation therapy and in children who were given HSCT. Little is known about the pathogenesis of FNH in transplanted children, but many risk factors linked to the HSCT procedure have been hypothesized. The detection of hepatic nodules, particularly in children who underwent HSCT for a previous malignancy, always raises a diagnostic dilemma. To help the physicians in the diagnostic management of this rare entity, we have retrospectively evaluated a series of transplanted children diagnosed with FNH in our Center over the last 15 yr. In this period, we found 10 new diagnoses of FNH. The diagnostic work-up included CEUS, abdominal CT, and MRI. A liver biopsy was performed in two patients. The median FUP time after diagnosing FNH was 3.8 yr, with an abdominal US and no malignant transformation were observed. Possible risk factors and indications for the management of FNH in transplanted children are reported and discussed in a comprehensive review of the literature.

Respiratory viral infections are a major cause of morbidity and mortality in solid organ transplant recipients. Early detection of a viral etiology of a LRTI in a febrile transplant recipient can theoretically reduce the use of antibiotics, trigger modification of immunosuppression and prompt appropriate isolation procedures to reduce nosocomial infections. We retrospectively evaluated pediatric abdominal organ transplant recipients hospitalized with respiratory illnesses to determine the viral pathogens identified by various methods including multiplex RT-PCR performed on nasopharyngeal or endotracheal aspirates. Among 30 symptomatic subjects (median age, 2.5 yr) evaluated using this methodology, 25 (83%) were positive for at least one virus. Rhinovirus was the most frequently identified virus (14 subjects). RSV was identified in five subjects with associated mortality of 40%. Parainfluenza, influenza, metapneumovirus, and adenovirus were also identified. This study indicates that rhinovirus is a significant cause of morbidity in this single center cohort of pediatric abdominal organ transplant recipients.

There is limited evidence regarding the utility of circulating DSA in surveillance for AMR of pediatric heart recipients. Our hypothesis is that quantitation of DSA improves their power for predicting a C4d+, an integral component in the current diagnostic criteria of AMR. All pediatric recipients transplanted between 10/2005 and 1/2011 were retrospectively reviewed for DSA determined within 48 h of EMB. C4d+ was defined as >25% endothelial cell staining by immunohistochemical methods. A total of 183 paired DSA–EMB determinations were identified in 60 patients, a median of three paired studies per patient (range: 1–9). DSA were detected in 60 of these determinations. A receiver-operating characteristic plot identified a threshold single-antibody MFI of >6000 that strongly correlated with C4d+ (p < 0.0001) with a high negative predictive value (0.97) and specificity (0.95). The sensitivity and positive predictive values were 0.71 and 0.60, respectively. The predictive power of single-antigen DSA for C4d deposition was improved in pediatric heart recipients using an institution-specific MFI threshold value. In post-transplant care, quantitative DSA should be an essential component in the surveillance for AMR.

The aim of this study is to evaluate the outcome of heart transplantation in children surviving malignancies. Pediatric heart transplant recipients were identified using the UNOS database. Follow-up data including survival and rate of malignancy were analyzed. A total of 7169 children received heart transplants between 1987 and 2011. Of these, 107 (1.5%) survived previous malignancy treatment (group I) and 7062 (98.5%) did not have prior malignancy (group II). Survival after transplant was 92.5%, 90.6%, 80.3%, and 65% at three months, one, five, and 10 yr in group I, similar to the rate in group II (90.1%, 84.4%, 73.8%, and 57.7%). Survival after transplantation was similar between group I and children who underwent OHT secondary to cardiomyopathy in group II. The rate of post-OHT malignancy in group I was higher than that in group II (14/107(13%) vs. 386/7062 (5.4%), p = 0.001). Children who developed malignancy in group I had similar survival as children who developed malignancy in group II. Post-transplant survival is similar in children with and without pretransplant malignancy in spite of higher rate of malignancy in children with pretransplant malignancy. OHT appears to be a reasonable treatment option in children who develop end-stage heart disease after malignancy treatment.

Improved neonatal medical care and renal replacement technology have improved the long-term survival of patients with ARPKD. Ten-yr survival of those surviving the first year of life is reported to be 82% and is continuing to improve further. However, despite increases in overall survival and improved treatment of systemic hypertension and other complications of their renal disease, nearly 50% of survivors will develop ESRD within the first decade of life. In addition to renal pathology, patients with ARPKD develop ductal plate malformations with cystic dilation of intra- and extrahepatic bile ducts resulting in CHF and Caroli syndrome. Many patients with CHF will develop portal hypertension with resulting esophageal varices, splenomegaly, hypersplenism, protein losing enteropathy, and gastrointestinal bleeding. Management of portal hypertension may require EBL of esophageal varices or porto-systemic shunting. Complications of hepatic involvement can include ascending cholangitis, cholestasis with malabsorption of fat-soluble vitamins, and rarely benign or malignant liver tumors. Patients with ARPKD who eventually reach ESRD, and ultimately require kidney transplantation, present a unique set of complications related to their underlying hepato-biliary disease. In this review, we focus on new approaches to these challenging patients, including the indications for liver transplantation in ARPKD patients with severe chronic kidney disease awaiting kidney transplant. While survival in patients with ARPKD and isolated kidney transplant is comparable to that of age-matched pediatric patients who have received kidney transplants due to other primary renal diseases, 64–80% of the mortality occurring in ARPKD kidney transplant patients is attributed to cholangitis/sepsis, which is related to their hepato-biliary disease. Recent data demonstrate that surgical mortality among pediatric liver transplant recipients is decreased to <10% at one yr. The immunosuppressive regimen used for kidney transplant recipients is adequate for most liver transplant recipients. We therefore suggest that in a select group of ARPKD patients with recurrent cholangitis or complications of portal hypertension, combined liver–kidney transplant is a viable option. Although further study is necessary to confirm our approach, we believe that combined liver–kidney transplantation can potentially decrease overall mortality and morbidity in carefully selected ARPKD patients with ESRD and clinically significant CHF.

Rejection with acute hemodynamic compromise after OHT is rare in children, and is associated with poor survival. We retrospectively reviewed the management, course and outcome of recipients with late (following initial hospital discharge) rejection with acute hemodynamic compromise who were supported on ECLS. Of 197 consecutive children undergoing OHT (84 male; mean [SD] age 8.3 [5.7] [range 0.1–18.8 yr]) between 2/2002 and 10/2012, 187 children survived and were discharged from hospital. Mean (SD) follow-up was 5.0 (3.1) (range 0.1–10.6) yr. During follow-up, seven presented with severe hemodynamic compromise after transplantation (of whom one patient had been transplanted elsewhere). All seven children, who presented in hemodynamic collapse with poor cardiac function refractory to inotropic support, were placed on ECLS—two following in-hospital cardiac arrest. The median duration of ECLS was 6 (range 5–15) days. All survived to decannulation, with one death from overwhelming sepsis 20 days after presentation. The median (range) duration (in days) of inotropic requirement post ECLS was 11 (5–27), the median ventilation time was 8 (7–30), median ICU length of stay was 14 (10–54), and median hospitalization was 24 (19–118). In all, ventricular function normalized (FS >28%) within 10 (7–22) days. There was significant short-term morbidity; however, over a median follow-up of 5.9 (range 0.7–9.2) yr, all survivors have good functional status with no significant apparent neurological sequelae. ECLS thus appears to be a good rescue therapy for children with severe acute rejection post OHT, refractory to conventional treatment, leading to good medium-term outcome.

RNA screening for HEV in 22 liver-transplanted children with chronic graft hepatitis out of a cohort of 267 liver-transplanted children detected a single patient with chronic HEV infection. Although this patient remained viremic for 33 months, anti-HEV-IgG was not detectable with MP assay but with Wantai assay. We present the first case of successful ribavirin therapy in an immunosuppressed child with chronic HEV infection. In conclusion, chronic HEV infection in immunosuppressed children may not be detectable employing serological assays. Therefore, the most reliably screening method is screening for HEV-RNA. Chronic HEV infection in children can successfully be treated with ribavirin.

A prospective identification of the estimated 20–50% of pediatric LTX recipients developing operational tolerance would be of great clinical advantage. So far markers of immune tolerance – T-cell subpopulations or gene expression profiles – have been investigated only retrospectively in successfully weaned patients. Fifty children aged 8–265 months (median 89) were investigated 1–180 months (median 44) after LTX under ongoing immunosuppression. T-cell subpopulations were measured during regular post-transplant visits using FACS (Vδ1- vs. Vδ2-γδ-T cells and Tregs). A Vδ1/Vδ2-γδ-T-cell ratio ≥1.42 previously reported in operational tolerance was found in 12 of 50 (24%) patients. In analogy, a Treg count ≥44 per μL was found in 35 of 50 (70%) patients and a Treg proportion ≥2.23% of CD3⁺-T cells in 39 of 50 (78%) patients. Only 9 of 50 patients (18%) fulfilled both criteria. The parameters Vδ1/Vδ2-γδ-T-cell ratio and Tregs were not significantly correlated to each other or with donor type or immunosuppression. Vδ1/Vδ2-γδ-T-cell ratio was more stable in serial examinations compared with Treg analyses. The observed proportion of 18% pediatric LTX patients with potential operational tolerance is in accordance with previous reports. However, clinical experience shows that rejections may happen even after long-time weaning of immunosuppression. This suggests that operational tolerance is a dynamic process, with uncertain prediction by Vδ1/Vδ2-γδ-T-cell ratio and/or Tregs under immunosuppression.

Psychomotor development in pediatric liver transplant (LT) recipients depends on several factors. Our aim was to evaluate the importance of parental involvement and family dynamics on psychomotor development by assessing (i) children and parents individually, (ii) the parent–child relationship, and (iii) the correlation between parental functioning and patient outcome, all before and after LT. Age-appropriate scales were used before and after LT. Twenty-one patients, 19 mothers, and 16 fathers were evaluated. Developmental quotient (DQ): No subjects scored in the “very good” range. The proportion of children with deficits increased from LT to two yr: 17.6% vs. 28.6%. Subjects 0–2 yr were more likely to have normal DQ at transplant (66.7% vs. 50% for older children). Abnormal DQ was more prevalent two yr post-LT in children older at LT (p = 0.02). The mother–child relationship was normal in 59% of families pre-LT and in 67% at two yr. The relationship was more favorable when the child received a transplant as an infant (p = 0.014 at 12 months post-LT). Normal DQ was associated with higher maternal global functioning score pre-LT (p = 0.03). Paternal performance scores were higher than maternal scores. Children transplanted after two yr of age suffer greater long-term deficits than those transplanted as infants.

To describe etiology, short-term outcomes and prognostic accuracy of serial PELD scores in PALF. Retrospective analysis of children aged ≤16 yr, admitted with PALF under the QLTS, Brisbane, Australia, between 1991 and 2011. PELD-MELD scores were ascertained at three time points (i) admission (ii), meeting PALF criteria, and (iii) peak value. Fifty-four children met criteria for PALF, median age 17 months (1 day–15.6 yr) and median weight 10.2 kg (1.9–57 kg). Etiology was known in 69%: 26% metabolic, 15% infective, 13% drug-induced, 6% autoimmune, and 9% hemophagocytic lymphohistiocytosis. Age <3 months and weight <4.7 kg predicted poor survival in non-transplanted children. Significant independent predictors of poor outcome (death or LT) were peak bilirubin > 220 μm/L and peak INR > 4. Serial PELD-MELD scores were higher in the 17 (32%) transplant recipients (mean: [i] 26.8, [ii] 31.8, [iii] 42.6); highest in the 12 (22%) non-transplanted non-survivors (mean: [i] 31.6, [ii] 37.2, [iii] 45.7) compared with the 25 (46%) transplant-free survivors (mean: [i] 25.3, [ii] 26.0, [iii] 30.3). PELD-MELD thresholds of ≥27 and ≥42 at (ii) meeting PALF criteria and (iii) peak predicted poor outcome (p < 0.001). High peak bilirubin and peak INR predict poor outcome and serial PELD-MELD is superior to single admission PELD-MELD score for predicting poor outcome.

The aim of this study was to re-evaluate the indications and timing of LT for WD. From 2000 to 2009, eight patients with WD who had been referred to our institution for LT were enrolled in this study. The mean patient age was 15.9 yr (range, 7–37 yr). Four patients could not receive LT, because there were no available donors. All four patients were treated with chelating agent medication. Three of them (two of two patients with fulminant WD and one of two with cirrhotic WD) who did not undergo LT are still alive and doing well with stable liver functional tests. Only one of the patients with cirrhotic WD who did not undergo LT died of hepatic failure. Even among the four patients who underwent LT, one with fulminant WD recovered from hepatic encephalopathy with apheresis therapy and chelating agent. He later required LT because of severe neutropenia from d-penicillamine. The other three patients who underwent LT recovered and have been doing well. Some of the patients with WD can recover and avoid LT with medical treatment. Even when WD has progressed liver cirrhosis and/or fulminant hepatic failure at the time of diagnosis, medical treatment should be tried before considering LT.

The purpose of this study is to compare the outcome of pediatric recipients of kidneys procured using a hand-assisted laparoscopic (HALDN group) to an open technique (ODN group). Twenty-eight patients ≤18 yr old (HALDN group) were compared with 17 patients (ODN group). The serum creatinine for HALDN and ODN groups at discharge were 0.93 ± 0.48 and 0.94 ± 0.54 mg/dL (p = 0.917), respectively. The serum creatinine for HALDN and ODN groups at six and 12 months was 1.01 ± 0.44 and 1.11 ± 0.55, and 1.04 ± 0.52 and 1.14 ± 0.46 mg/dL (p = 0.516, p = 0.554), respectively. The eGFR for HALDN and ODN groups at discharge was 108.66 ± 37.23 and 106.1 ± 50.55 mL/min/1.73 m² (p = 0.845), respectively. The eGFR for HALDN and ODN groups at six and 12 months was 97.77 ± 28.25 and 81.73 ± 27.46, and 94.56 ± 28.3 and 85.74 ± 30.1 mL/min/1.73 m2 (p = 0.085, p = 0.344), respectively. The patient and graft survival for both groups were 100% at 12 months post-transplant. In conclusion, the short-term outcome of recipients of kidneys procured via HALDN is comparable to that of kidneys procured via ODN in pediatric patients.

HSCT is the only proven treatment option for CML, a rare disease in children. Recently, there are promising reports on the advantageous effect of imatinib mesylate for pediatric patients with CML. We conducted a retrospective study on 33 pediatric patients suffering from CML. Fourteen underwent HSCT and the rest were treated with imatinib. With a median follow-up of 24 months, the two-yr OS in the HSCT group and the imatinib group was 84% and 87%, respectively (p = 0.714). The probabilities of two-yr DFS were 59% in the HSCT group and 82% in the imatinib group, either (p = 0.880). Relapse occurred in 5 (35.7%) patients of the HSCT group, and 8 (42.1%) patients showed relapse in the imatinib group. Among nine patients who died, five were in the HSCT group and the rest were in the imatinib group. The probability of relapse in the patients of the imatinib group followed up for several consecutive years may be higher than observed in the HSCT group, so we cannot easily conclude which way is more reliable.

We evaluated the results of a novel conditioning regimen of reduced dose cyclophosphamide (Cy, 25 mg/kg for four days), fludarabine (Flu, 30 mg/m² for four days), and rabbit ATG (2.5 mg/kg for three days) for allogeneic transplant of children with SAA, implemented since January 2009. Overall, 23 patients were treated with this regimen (16 male, seven female), including 10 diagnosed with VSAA. Donors included eight-MSD and 15 UD (five-matched UD, and 10 mismatched UD). All patients showed neutrophil and platelet engraftment. Cumulative incidence of acute (grade 2 or above) and chronic GVHD was 26.1% and 8.7%, respectively. Estimated two-yr FFS and OS for the entire cohort was 90.3 ± 6.5%. Rates of TRM and graft failure were 5.3% and 4.3%, respectively. No difference in OS was found according to disease severity (SAA vs. VSAA, p = 0.184), or according to donor type (MSD vs. UD, p = 0.699). Excellent outcomes of patients with VSAA underscore the efficacy of allogeneic transplant as a means of expediting hematopoietic recovery. Improved survival of UD transplant reaffirms its role as a valid therapeutic alternative in the absence of MSD.

HSCT can be curative for many PID. Little is known about the outcome of HSCT for patients with PID in the developing countries. We retrospectively reviewed all children with PID who received HSCT at KHCC in Jordan between August 2003 and October 2011. Twenty-eight patients were identified. The median age was 16 months (3 months–17 yr). Patients' diagnoses were SCID (n = 16), CHS (n = 3), HLH (n = 3), WAS (n = 2), Griscelli syndrome (n = 1), ALPS (n = 1), Omenn's syndrome (n = 1), and DiGeorge syndrome (n = 1). Seventeen patients received HLA-matched related HSCT, eight received maternal un-manipulated haploidentical HSCT, and three received unrelated cord blood transplantation. Nine patients (32%) developed BCGosis secondary to reactivation of pretransplant vaccination. Three died while still receiving anti-tuberculosis drugs, one still on treatment, and all others have recovered. Six patients had graft failure; four of them received no conditioning regimens. At a median follow up of 32 months (range 1-67), 21 patients are alive, with overall survival of 72%. We conclude that HSCT for PID patients can be performed with a good outcome in developing countries; however, delayed diagnosis or referral and BCG reactivation are unique challenges.

The aim of this study was to assess the immunogenicity of a vaccine against this virus in a prospective cohort of transplanted pediatric patients without previous influenza infection who received one dose of MF59^®-adjuvanted pandemic H1N1/2009 vaccine. Seventeen patients who were being regularly followed up at the Outpatient Clinic of the Children's Transplant Unit (liver and kidney transplantation) in Hospital Universitari Vall d′Hebron (Barcelona) were included. Seroconversion was demonstrated in 15 of 17 (88.2%) vaccinated children. There were no rejection episodes or major adverse events. The MF59^®-adjuvanted pandemic H1N1/2009 vaccine was safe and elicited an adequate response.

Idiopathic CD4+ lymphocytopenia (ICL) is a rare immunodeficiency disease with severe CD4 T-cell depletion, leading to serious opportunistic infections. The optimal treatment of ICL has not been determined, especially in severe form of the disease. Here, we report an eight-yr-old girl with ICL who was successfully treated with fludarabine-based conditioning HSCT. To the best of our knowledge, this is the first pediatric ICL case that was treated by HSCT. Allogeneic HSCT with a reduced intensity condition (RIC) regimen may be a feasible and curative treatment option in ICL patients with recurrent life-threatening complications.