Irinotecan for relapsed Wilms tumor in pediatric patients: SIOP experience and review of the literatureA report from the SIOP Renal Tumor Study Group

While irinotecan has been studied in various pediatric solid tumors, its potential role in Wilms tumor (WT) is less clear. We evaluated response and outcome of irinotecan‐containing regimens in relapsed WT and compared our results to the available literature. Among 14 evaluable patients, one complete response (CR) and two partial responses (PRs) were observed in patients with initial intermediate‐risk (CR and PR) and blastemal‐type histologies (PR). Two patients were alive at last follow‐up showing no evidence of disease. Our results and the reviewed literature suggest some effectiveness of irinotecan in the setting of relapsed WT

Quality of general movements and the development of minor neurological dysfunction at toddler and school age

Objective: To evaluate the reliability of assessing infants' general movements (GMs) using a new classification and its validity in predicting complex minor neurological dysfunction (MND) at toddler and at school age. Design: Prospective study of two groups of infants, each consisting of a mix of low-risk and high-risk infants. Setting: University Hospital Groningen, the Netherlands. Subjects: Group A consisted of 16 low-risk and 21 high-risk infants; group B of 28 low-risk and 24 high-risk infants. Main outcome measures: Between term age and four months post term: multiple assessments of neurological condition by means of (a) assessment of GMs, and (b) a traditional neurological examination. GMs were classified into four classes using a standardized qualitative description: two classes of normal movements (normal-optimal and normal-suboptimal) and two classes of abnormal movements (mildly and definitely abnormal movements). Follow-up neurological examination with special attention to presence of MND was carried out in group A at 1% years, in group B at 4-9 years. Results: GMs could be assessed reliably. They were stable over age in about 60% of the infants. Both the condition of the GMs and the infant neurological condition were significantly related to neurological condition at follow-up. Best prediction of complex MND was achieved when both types of infant assessment at the age of 2-4 months post term were combined. Conclusions: The assessment of GMs is a valuable tool, in particular when combined with the traditional neurological examination, to predict at early age the development of complex MND

Anaplastic Lymphoma Kinase Aberrations in Rhabdomyosarcoma: Clinical and Prognostic Implications

Purpose The aim of this study is to investigate anaplastic lymphoma kinase (ALK) protein expression and underlying genetic aberrations in rhabdomyosarcoma (RMS), with special attention to clinical and prognostic implications. Patients and Methods A total of 189 paraffin-embedded RMS tumor specimens from 145 patients were collected on tissue microarray. ALK protein expression was evaluated by immunohistochemistry. ALK gene (2p23) copy number and translocations were determined by in situ hybridization. cDNA sequencing of the receptor tyrosine kinase domain of the ALK gene was assessed in 43 samples. Results Strong cytoplasmic ALK protein expression was more frequently observed in alveolar RMS (ARMS) than in embryonal RMS (ERMS) (81% v 32%, respectively; P < .001). ALK gene copy number gain was detected in the vast majority of ARMS (88%), compared with 52% of ERMS (P < .001). ALK copy number correlated with protein expression in primary tumors (n = 107). We identified one point mutation (2%) and seven tumors harboring whole exon deletions (16%). In ERMS, specific ALK gain in the primary tumor correl Conclusion Because ALK aberrations on genomic and protein levels are frequently found in RMSs, in particular ARMS, and are associated with disease progression and outcome in ERMS, ALK may play a role in tumor biology and may provide a potential therapeutic target for these tumors. Future research should aim at the oncogenic role of ALK and the potential effect of ALK inhibitors in RMS