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Purpose: To evaluate clinical features at diagnosis, prognostic factors, and outcomes of malignant sacrococcygeal germ cell tumors (SC-GCTs) in patients enrolled in the Associazione Italiana Ematologia Oncologia Pediatrica (AIEOP) TCG 2004 protocol., Patients and Methods: A prospective analysis was conducted on all consecutive patients diagnosed with malignant SC-GCTs between January 2004 and May 2017. Patients with stage I underwent surgery and subsequent surveillance, the others received pediatric cisplatinum-etoposide-bleomycin (pPEB) regimen and eventual deferred surgery., Results: Of 45 patients, 35 were females. Age at diagnosis ranged from 1 day to 3.6 years (median 1.6 years); 26 were stage IV. Of 38 patients who underwent surgery, pathology revealed yolk sac tumor (YST) in 27 and teratoma + YST/embryonal carcinoma in 11, while seven patients were diagnosed based on imaging and elevated levels of alpha-fetoprotein (AFP). Of six patients approached with surgery, only one relapsed and was rescued with first-line chemotherapy. Overall, 38 out of 45 achieved complete remission, three a partial remission, and four were resistant. Ten out of 41 patients who entered remission later relapsed and nine were rescued with a second-line treatment. We observed a global failure percentage of 31% and a 5-year overall survival (OS) and event-free survival (EFS) of 95% and 69%, respectively., Conclusions: Chemotherapyis generally effective in malignant SC-GCTs, even though almost one-third of our patients experienced events salvageable with second-line treatment. Most of the relapses occurred within 1 year from diagnosis. A close follow up with serial AFP level monitoring should be done for at least 2 years after diagnosis., (© 2020 Wiley Periodicals LLC.)

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., (© 2014 Wiley Periodicals, Inc.)

Management of aggressive, usually late-occurring, post-transplant lymphoproliferative disorders (PTLDs), a life-threatening complication after solid organ transplants, remains controversial. Four children affected by aggressive CD20+ PTLDs received a chemo-immunotherapy regimen for remission induction based on fludarabine, cyclophosphamide, doxorubicin, and rituximab, associated with a rapid discontinuation of immunosuppression (IS). Subsequent consolidation chemotherapy consisted of Berlin-Frankfurt-Münster-modified blocks. All patients achieved a complete remission, which persisted for 25, 68+, 80+, and 103+ months after diagnosis. Therapy was well tolerated. No patients developed allograft rejection during PTLD treatment. Our experience suggests that this chemo-immunotherapeutic approach may be an effective treatment strategy while allowing for a concomitant discontinuation of IS., (Copyright © 2011 Wiley-Liss, Inc.)

Purpose: To ascertain whether alveolar histology retains its adverse prognostic role in the subset of paratesticular rhabdomyosarcoma (RMS) patients, generally characterized by a very good outcome., Patients and Methods: Twenty pediatric patients were treated over a 25-year period using the protocols of the Italian and German Soft Tissue Sarcoma Cooperative Groups. Clinical characteristics at presentation were much the same as in non-alveolar patients., Results: The proportion of patients with alveolar histotype (8%) in paratesticular site was lower than in the general RMS population (20-30%). With a median follow-up of 122 months, 5-year EFS and OS were 78 and 89%, respectively., Conclusion: Our data suggest a distinctly better clinical behavior of paratesticular alveolar RMS than when the disease occurs at other sites. These patients were more intensively treated than the embryonal cases, however, so-although a treatment intensity reduction may be desiderable-the idea of eliminating the alkylating agents (as in low-risk embryonal paratesticular RMS) must be considered with great caution., (Copyright 2003 Wiley-Liss, Inc.)