Phase II study of irinotecan in combination with temozolomide (TEMIRI) in children with recurrent or refractory medulloblastoma: a joint ITCC and SIOPE brain tumor study.

Background: This multicenter phase II study investigated temozolomide + irinotecan (TEMIRI) treatment in children with relapsed or refractory medulloblastoma.

Methods: Patients received temozolomide 100-125 mg/m(2)/day (days 1-5) and irinotecan 10 mg/m(2)/day (days 1-5 and 8-12) every 3 weeks. The primary endpoint was tumor response within the first 4 cycles confirmed ≥4 weeks and assessed by an external response review committee (ERRC).

From idiopathic diabetes insipidus to neurodegenerative Langerhans cell histiocytosis--an unusual presentation and progression of disease.

Diabetes insipidus (DI) is rare in childhood and has a wide-ranging aetiology including the involvement of uncontrolled proliferation of dendritic cells in the hypothalamic-pituitary axis, characteristic of Langerhans cell histiocytosis (LCH). DI may manifest as a sequela of multisystem LCH disease involving skin, bone, liver, spleen and lymph nodes. In very rare cases patients diagnosed with LCH exhibit neurodegenerative changes, such as severe ataxia, tremor, dysarthria and intellectual impairment.

Natural history and outcome of optic pathway gliomas in children.

Background: The optimal management of optic pathway gliomas (OPGs) is complicated by their variable natural history, the association with neurofibromatosis type 1 (NF1) and difficulties in defining progression and response to treatment.

Methods: This study is a retrospective review of all children presenting to a single institution with an OPG between 1990 and 2004.

Results: Of the 133 children included, 78 (59%) had NF1; 87 (71 NF1) were observed initially, of whom 23 (11 NF1) subsequently required treatment.

Assessment of chemotherapeutic response in children with proptosis due to optic nerve glioma.

Introduction: Some children with optic pathway gliomas present with proptosis related to intraorbital tumor extension. The radiological assessment of chemotherapeutic response in these patients can be complicated by irregular tumor shape and lack of relation between tumor volume and cosmetic effect.

Method: We propose that proptosis measurements and derivation of a proptosis index can be a useful adjunct to the measurement of tumor volume in the radiological assessment of chemotherapeutic response.

Outcome after pulmonary radiotherapy in Wilms' tumor patients with pulmonary metastases at diagnosis: a UK Children's Cancer Study Group, Wilms' Tumour Working Group Study.

Purpose: To evaluate the effect of whole lung radiotherapy on event-free and overall survival of children with Stage IV Wilms' tumor with pulmonary metastases at diagnosis and to ascertain factors that may have led to the decision to withhold radiotherapy.

Methods And Materials: We compared recurrence and mortality risks of patients with pulmonary metastases at diagnosis enrolled in the UKW2 and UKW3 clinical trials (1986-2001) according to treatment with pulmonary radiotherapy.

Results: Of 102 eligible patients (43 patients in UKW2 and 59 patients in UKW3), 72 (71%) received pulmonary radiotherapy; 30 (29%) did not.

Weekly vinblastine in pediatric low-grade glioma patients with carboplatin allergic reaction.

Background: Carboplatin-based regimens have demonstrated activity in unresectable low-grade glioma (LGG) in children. Despite an interesting toxicity profile, the use of these regimens has been limited by the development of carboplatin hypersensitivity reaction (HSR) in up to 30% of patients. Desensitization has been the recommended approach for HSR.