Risk-adapted Treatment for Severe B-Lineage Posttransplant Lymphoproliferative Disease After Solid Organ Transplantation in Children.

BACKGROUND: Optimal management of posttransplant lymphoproliferative disease (PTLD) remains to be defined due to heterogeneity of this condition and lack of predictors of the outcome. Here we report our experience with pediatric PTLD nonresponsive to immunosuppression (IS) withdrawal, managed after stratification into high and low risk according to the presenting features. METHODS: This is a single-center retrospective review of prospectively enrolled patients. From 2001 to 2011, 17 children were diagnosed with severe B-lineage, CD20+, PTLD after a median of 37 months (range, 5-93) from liver (12), heart (4), or multiorgan (1) transplantation. Treatment was tailored on 2 risk groups: (1) standard-risk (SR) patients received IS reduction and rituximab; (2) high-risk (HR) patients received IS discontinuation, rituximab and polychemotherapy. RESULTS: The cumulative incidence of rejection at 1 and 5 years after the diagnosis of PTLD was 35% (95% confidence interval [95% CI], 18-69%) and 53% (33-85%), respectively, whereas the disease-free survival at 1 and 5 years was 94% (95% CI, 65-99%) and 75% (45-90%), respectively. Three children died, PTLD-free, from different transplant-related complications: primary nonfunction after retransplantation (liver), cytomegalovirus disease 21 months after PTLD treatment (liver), graft dysfunction 25 months after PTLD (heart). CONCLUSIONS: Severe B-lineage PTLD after solid organ transplantation may be classified as SR or HR and treated accordingly with a tailored protocol obtaining a satisfactory long-term outcome. This approach accomplishes the control of lymphoproliferation in severe forms as well as the minimization of toxicity in milder PTLDs.

Percutaneous transhepatic biliary drainage in an infant with obstructive jaundice caused by neuroblastoma.

Neuroblastoma presenting with obstructive jaundice is a rare event. Management of this condition includes surgery, chemotherapy, radiotherapy, temporary cholecystostomy tube, endoscopic retrograde cholangiopancreatography (ERCP), and internal biliary drainage (IBD). We herein describe our experience with one infant affected by neuroblastoma presenting with jaundice, who successfully underwent percutaneous transhepatic biliary drainage (PTBD). This report introduces PTBD as a viable treatment option for neuroblastoma and obstructive jaundice and provides a review of the pertinent literature.

Is multifocality a prognostic factor in childhood hepatoblastoma?

BACKGROUND: The aim of this study was to assess the prognostic value of multifocality and the effectiveness of two different therapeutic strategies in patients with newly diagnosed hepatoblastoma. PROCEDURES: Between 1998 and 2011, 31 patients diagnosed with hepatoblastoma were referred to Ospedale Papa Giovanni XXIII, Bergamo, Italy. Patients were stratified according to SIOPEL protocols into high-risk (HR if AFP <100 ng/mL and/or PRETEXT IV and/or vascular invasion and/or extra-hepatic intra-abdominal disease and/or metastases) and standard-risk (SR, all others). The patient data we evaluated were: multifocality; patient age; gender; platelet count; AFP level at diagnosis, during treatment and follow-up; histotype; gestational age; birth weight; surgery (either resection or transplantation) and chemotherapy regimen adopted before and after surgery. The outcome measures were event free survival (EFS) and overall survival (OS); survival curves were estimated according to Kaplan-Meier. RESULTS: EFS and OS were associated significantly with multifocality (3-year EFS 40% vs. 95%, P = 0.006; 3-year OS 42% vs. 95%, P = 0.004). Multivariate analysis demonstrated that multifocality predicts lower EFS (hazard ratio 10.01, P = 0.007). Other factors at diagnosis did not reach statistical significance. A marked treatment dependent improvement was associated with intensive chemotherapy given both before and after liver transplantation (P = 0.06). CONCLUSIONS: Patients diagnosed with multifocal tumors had lower EFS levels. Multifocality should be taken into account for future stratification and further studied to assess genetic profile, immunochemistry and prognostic role.

Is there a role for high dose chemotherapy and blood stem cell rescue in childhood hepatoblastoma presenting with lung metastases? A case report and literature review.

We report the use of high dose chemotherapy with peripheral blood stem cell rescue as a consolidation treatment for a 3-year-old child affected by metastatic hepatoblastoma, who achieved complete lung response only after conventional treatment. The patient is presently alive 27 months after high dose chemotherapy with blood stem cell rescue with no evidence of disease.The role of high dose chemotherapy with blood stem cell rescue to consolidate the complete clearing of lung disease in metastatic hepatoblastoma remains controversial; the data available in the literature and our experience seems to suggest to keep this treatment option open to further consideration in the clinical setting of high-risk patients.