Clinical characteristics and outcome of pediatric patients diagnosed with Langerhans cell histiocytosis in pediatric hematology and oncology centers in Poland

Abstract

Background Langerhans cell histiocytosis (LCH) affects 1–2 in 1,000,000 people. The disease is not associated with increased risk of treatment failure (especially among older children), but appropriate procedures implemented in advance can eliminate complications which might appear and significantly worsen the patients’ quality of life. Thus, we sought to evaluate the clinical features, management, and outcome of children with LCH treated in Polish pediatric hematology-oncology centers. Materials and methods One hundred eighty two patients with LCH were treated according to the Histiocytic Society Guidelines between 2010 and 2017. The participating centers were requested to provide the following data: demographic, clinical, as well as local or systemic treatment data and patients’ outcome. Overall survival (OS) and event free survival (EFS) were estimated by Kaplan-Meier methods and compared using the log-rank test. Results Sixty nine percent of children were classified as single system (SS). The patients with SS disease were significantly older as compared to the children with multisystem disease (MS), 6 vs. 2.3 years respectively (p 0.003). Bones were involved in 76% of patients. Systemic treatment was applied to 47% of children with SS disease and 98% with MS disease. Fourteen patients relapsed while two children died. OS and EFS in entire group were 0.99 and 0.91 respectively (with median follow-up 4.3 years). Conclusion The treatment of LCH in Polish centers was effective, however, new approaches, including mutation analyses and good inter-center cooperation, are needed to identify patients who might require modification or intensification of treatment.

Clinical and magnetic resonance imaging feature differences between solitary and multiple type Langerhans cell histiocytosis involving the craniofacial bone

“…MLG patients had a younger predominance than SLG patients. Although the classification of manifestation type differs, it is consistent with previous studies [ 31 , 32 ]. Younger children have a greater risk of multi-system disease and an unfavorable clinical course.…”

Synchronous neoplasms in a paediatric patient

“…In the treatment of LCH, depending on the organ and system involved, chemotherapy, radiotherapy, surgi- Synchronous neoplasms in a paediatric patient cal treatment, local treatment, or immunotherapy play the most important roles [10]. The overall survival rate is 84-99%, and the risk of recurrence is 10-50%, depending on the extent of the process [10,12].…”

Impact of molecular status on clinical management in children with histiocytosis treated according to POL HISTIO project

“…Histiocytic disorders are rare diseases which affect 1-2 in 1,000,000 people. This frequency increases to 5-9 in 1,000,000 children under the age of 15 [1][2][3]. At the origin of these diseases is a clonal proliferation of cells that morphologically and immunophenotypically resemble macrophage line cells.…”

“…To the M group belong malignant histiocytosis (primary and secondary). The R group represents Rosai-Dorfman disease (sporadic: classic, extranodal, with neoplasia or immune disease and unclassified and familial) [1,6].…”

“…The spectrum of clinical symptoms ranges from unifocal lesions, single organ or single system disease, to very serious life-threatening multisystemic multifocal diseases with the involvement of life-critical organs (highest risk organs: bone marrow, liver, and spleen). The course of these diseases varies from spontaneous remission to aggressive progression with life-threatening conditions including death [1,2]. Therefore, the management of LCH can vary greatly from a 'wait-and-watch' attitude through serious procedures such as intensive chemotherapy up to bone marrow stem cell transplantation.…”