Oral Prednisone in Treating LCH of Bone in Childhood and Adolescence

The Efficacy of Oral Prednisone in Treating Langerhans Cell Histiocytosis of Bone in Childhood and Adolescence: A Multi-center, Open-label, Randomized-controlled, Phase II Study

First Affiliated Hospital of Zhejiang University, Peking University Third Hospital, National Sun Yat-sen University, Beijing Children's Hospital, Henan Cancer Hospital, Tianjin Hospital, First Affiliated Hospital, Sun Yat-Sen University, Second Hospital of Jilin University, Peking University People's Hospital, Jinling hospital Nanjing, Jiangsu, China, The First Affiliated Hospital of Nanchang University, The Third Affiliated Hospital of Southern Medical University

Langerhans cell histiocytosis (LCH) of bone is a benign-tumor-like osteolytic lesion in childhood and adolescence, which is characterized by the aberrant activation of antigen presenting cells. Rather than the multi-system involvements of LCH, no standard or widely-accepted therapeutic regimens were established for LCH of bone. In the previous clinical practice, several LCH patients obtained remarkable pain relief after taking prednisone. Therefore, the investigators aim to conducting a multi-center, open-labelled, randomized-controlled, Phase II study to investigate the efficacy and safety of oral prednisone in treating LCH of bone in children and adolescents. The enrolled patients will be randomly recruited to the following groups: (1) Oral prednisone [Test group); (2) Regular observation [Control group].

If the diseae progression in the control group is confirmed, the patient is allowed to transferred to the test group (Oral prednisone).

For the experimental group, the patient will be administrated oral prednisone (0.5mg/kg/day, first 5 days per month, 6 months)

The Visual Analogue Scale (Minimum: 0; Maximum: 10 scores), is used to evaluate the degree of pain relief. The higher scores mean a worse outcome.

Inclusion Criteria: Pathological diagnosis of Langerhans cell histiocytosis (LCH) of bone; Single-system involvement (skeletal system); No need of surgical intervention; Must be able to swallow tablets; Signing informed consent form. Exclusion Criteria: Multi-system involvements (≥2 systems, including bone, liver, spleen, hematologic system, central nerve system); Need of surgical intervention (e.g. pathological fracture and/or spinal cord compression) Glucocorticoid allergy; Immunodeficiency; Severe infection; Insulin dependent/independent Diabetes; Having taken glucocorticoid in the past two weeks; Not capable of swallowing tablets; Without signed informed consent inform.

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