We studied the neurophysiological features of five patients (age range: 4-20 years) suffering from Crigler-Najjar syndrome type I (CNsI) by means of multimodal (brainstem, somatosensory, motor) evoked potentials and periodic EEG-polygraphic recordings (follow-up: 3 months-4.5 years). Two patients presented with neurological disturbances, consisting mainly of mental slowing, motor impairment and seizures. Both of them presented an abnormal EEG, characterized by slowing of background activity associated with paroxysmal discharges. Liver transplantation was performed in one of these two patients and was followed by improvement of both the neurological picture and EEG activity. In a third patient, clinically normal, after two years of follow-up, the EEG started to show paroxysmal activity during sleep or when evoked by intermittent photic stimulation. In these three patients, multimodal evoked potentials were unremarkable. The remaining two younger subjects did not show any clinical or EEG abnormality. Our findings suggest that, whereas in newborns and infants evoked potentials have been demonstrated as reliable techniques to monitor bilirubin neurotoxicity, in children and adolescents with CNsI, EEG seems to be more sensitive in evaluating patients for neurological damage and effectiveness of therapeutic strategies adopted.
557 Background: Germ-cells testicular tumors are the most common cancer in young males, usually curable in early stages. Therefore, only 25% of relapsed patients are long-term survivors. Previous non-randomized studies showed promising results with HDC, however, the only randomized trial did not support this approach. Here we report our experience in a Brazilian academic center Methods: We conducted a retrospective review of all consecutive patients treated with salvage HDC followed by Stem-Cell transplantation at our service (1996 – 2016). Survival curves were estimated by Kaplan-Maier method. Prognostic factors were determined by Cox regression model. Results: We found 36 eligible patients. The cohort median follow-up was 56 months (m). At 2 years 50.4% of the patients were alive, and 46.7% were disease-free. The median overall survival (OS) was not-reached and the median progression-free survival (PFS) was 21.8 m. The overall response-rate was 61.2% (22% had complete response and 39% had partial response). All patients who achieved complete response were alive at the last follow-up, and only one had progressive disease. Out of the patients who achieved partial response, one third were rescued with surgery (46% teratoma, 23% fibrosis, and 31% residual disease). Approximately 80% were alive in 2 years vs 20% not operated. Complete response (vs. others) and HDC as second line treatment (vs. 3rd and 4th) were good prognostic factors, (HR N/A; p 0,03, HR 0.19; CI: 0.04-0.88, p = 0.01 respectively). In 2 years, the OS in patients treated in 2nd line was approximately 80%, 50% for 3rd line, and 0% of those treated in 4th line. 89% of the patients experienced grade 3 toxicity, and 25% grade 4. The most common were neutropenic fever (69%), mucositis (36%) and diarrhea (25%). There were 3 deaths related to the treatment. Conclusions: Patients with complete response have sustained long-term survival, and represent the subgroup with evident benefit of the treatment. Surgery, when feasible, can rescue 80% of the patients with partial response. According to our data, we do not recommend this modality of treatment in 4th line.
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