[Therapeutic Strategies In Children And Adolescents With Langerhans Cell Histiocytosis]. / Therapiestrategien bei Kindern und Jugendlichen mit Langerhanszell Histiozytosen.

The current standard therapy for children and adolescents with newly diagnosed Langerhans cell histiocytosis (LCH) is based on the two drugs prednisone and vinblastine. In patients with insufficient treatment response or disease relapse, the choice of second-line treatment depends on risk organ involvement (liver, spleen, and hematopoietic system). This article will give an overview of current data concerning therapeutic options in the different settings of children and adolescents with LCH. Due to limited evidence, these strategies have not been described in detail in the updated guidelines on pediatric LCH. In addition, the use of targeted therapy such as MAP-kinase inhibitors will be discussed. The reference center for LCH should be contacted if therapeutic options beyond the standard regimen are considered for treatment. All children and adolescents with LCH should be enrolled in registries or prospective studies.

Progression and Relapse of Pediatric Soft Tissue Sarcoma: Individualized Approach of Treatment - Experience from a Major Pediatric Cancer Center in Europe.

BACKGROUND: The outcome of children with refractory or relapsed soft tissue sarcoma (STS) is extremely poor. Whereas larger clinical trials evaluated specific treatment modalities, real-life data on individual multimodal therapeutic strategies, given alone or in combination, are scarce. PATIENTS AND METHODS: We retrospectively analyzed the clinical course of 18 pediatric patients with progression of or relapsed STS treated between 2008 and 2018 in our institution. RESULTS: A total of 18 patients (median age 12.4 years) suffered from progression or relapse of alveolar (n=7), embryonal (n=5), undifferentiated (n=2) rhabdomyosarcoma or desmoplastic small round cell tumor (n=4). 14 patents had an initial stage IV disease. All but one patient died. Median survival was 12.5 months. Shortest survival was seen in patients with systemic progression of the disease, longest in patients with local relapse. Patients with an Oberlin score<2 at the time of relapse had a significant longer time of survival than those with a score≥2. No significant advantage of a specific therapeutic modality was observed. DISCUSSION: We critically analyzed the clinical course in the real-life setting, in which various treatment options were applied to an individual patient according to the best of available data. We observed that some patients died within a short period of time despite multiple treatment modalities, which underlines the need for better prognostic parameters. CONCLUSION: In addition to well characterized clinical factors such as local or systemic relapse, the Oberlin score could be helpful in counselling patients and their families for choosing the best strategy of care.

[Updated AWMF Guideline on the Diagnosis and Treatment of Langerhans cell Histiocytosis in Children and Adolescents]. / Aktualisierte AWMF Leitlinie zur Diagnostik und Therapie der Langerhanszell Histiozytose (LCH) im Kindes- und Jugendalter.

Langerhans cell Histiocytosis is a rare neoplastic disease, which occurs mainly in children and adolescents. The disease may affect any organ, and therefore, the clinical symptoms vary widely. Some patients have a spontaneous remission of the disease, whereas others experience a rapid and potentially lethal clinical course. The therapeutic approach depends on the extent of the disease, and reaches from a watch-and-wait strategy to chemotherapy with the standard drugs vinblastine and prednisone. The identification of mutations in the MAPK-pathway resulted in growing interest in targeted therapy using compounds such as the BRAF inhibitors. Chronic relapses and permanent sequelae are important problems of LCH and are the focus of current research.