Late sequela after treatment of childhood low-grade gliomas: a retrospective analysis of 69 long-term survivors treated between 1983 and 2003.

The aim of the present study was to evaluate the spectrum of late effects in a large cohort of pediatric patients with low-grade gliomas (WHO grade I and II) during an observation period of 20 years. Eighty-seven patients with low-grade gliomas grouped according to tumor location (cerebellum: n=28; cerebral hemispheres: n=21; central midline: n=15; brainstem: n=12; tectum: n=5; other locations: n=6) were evaluated for tumor- and/or treatment-related late effects by analysis of medical and computer records, and personal interviews. Seventy patients underwent neurosurgery, 29 patients received additional radiotherapy and 20 additional chemotherapy. Median follow-up of survivors is 96 months with an overall survival of 79% (cerebellum: 89%; cerebral hemispheres: 95%; central midline: 80%; brainstem: 25%; tectum: 100%; other locations: 66%). Chronic medical problems (mild ataxia to multiple severe neuroendocrine deficits) are observed in 100% of patients with brainstem/central midline tumors and in 40-50% of patients with low-grade gliomas of other locations. Endocrine deficiencies were observed in 15/17 (88%) of long-term survivors who received radiotherapy. In contrast, none of the patients who underwent surgery only had endocrine deficiencies. Seven long-term survivors (10.1%) are severely disabled with permanent need of medical help. Tumor- and treatment-related late effects are common in patients with low-grade gliomas with the most severe occurring in patients with brainstem or central midline tumors. As long-term survival is excellent in patients with low-grade gliomas except for tumors located in the brainstem, future treatment studies should focus on avoiding long-term late effects.

Low-dose versus high-dose immunoglobulin for primary treatment of acute immune thrombocytopenic purpura in children: results of a prospective, randomized single-center trial.

PURPOSE: To investigate the efficacy and side effects of two different intravenous immunoglobulin (IVIG) dose regimens for the initial treatment of childhood acute immune thrombocytopenic purpura (ITP). METHODS: Thirty-four consecutive patients with a clinical diagnosis of acute ITP and a platelet count below 20x10(9)/L were randomized to receive either 1 g/kg body weight (n=17; group A) or 0.3 g/kg body weight (n=17; group B) IVIG per day for 2 consecutive days (total dose 2 g/kg and 0.6 g/kg). RESULTS: Fifteen of the 17 patients (88.2%) in group A and 13 of the 17 patients (76.5%) in group B achieved a platelet count of more than 20x10(9)/L within 72 hours. The increase in platelet counts on day 2 and 3 was more pronounced in the high-dose group. Two patients in the high-dose group and four in the low-dose group were non-responders. Chronic disease occurred in three patients receiving 2 g/kg IVIG and in five patients receiving 0.6 g/kg IVIG. Side effects of IVIG administration were more common in the high-dose group. CONCLUSIONS: The present study showed that platelet counts increased more rapidly after high-dose IVIG administration within the first 72 hours, although a platelet count of more than 20x10(9)/L can be achieved also with low-dose IVIG in most children with acute ITP. For patients with very low platelet counts, doses higher than 0.6 g/kg seem, therefore, to be more effective.

Multimodal treatment of children with unresectable or recurrent desmoid tumors: an 11-year longitudinal observational study.

The primary goal of treatment for desmoid tumors is complete surgical resection to achieve negative margins. In adults with unresectable or recurrent lesions, treatment options include noncytotoxic and cytotoxic drugs, but little is known about nonsurgical treatment in children. Between 1992 and 2003 six children (four girls, two boys) with a median age of 2.5 years (range 11 months to 9 years) received multimodal adjuvant therapy for unresectable or recurrent desmoid tumors. Primary treatment consisted of noncytotoxic treatment with tamoxifen (1 mg/kg orally, twice daily) and diclofenac (2 mg/kg rectally, twice daily), whereas two children with life-threatening tumor progression in addition received treatment intensification with weekly vinblastine (6 mg/m intravenously) and methotrexate (30 mg/m intravenously). Of the four children with unresectable tumors, two achieved remarkable tumor shrinkage and two had stable disease, whereas two patients were disease-free for 3.7 and 2.6 years after nonradical resection. Median observation time was 3.1 years (range 1-11 years). Treatment was generally well tolerated; only one patient developed pubertal acceleration after a duration of tamoxifen treatment of 9.3 years. Because of the potential life-threatening situation, the management of children with unresectable or recurrent desmoid tumors requires a multidisciplinary approach. Nonaggressive therapy with tamoxifen and diclofenac may be the first treatment choice in these patients, but in patients with progressive disease, cytotoxic chemotherapy is indicated. Weekly administration of vinblastine and methotrexate seems to be safe and effective in these children.