Langerhans Cell Histiocytosis of Bone
Conditions
Keywords
Langerhans cell histiocytosis, Bone, Single-system, Children, Adolescents
Brief summary
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 \[Control group\].
Interventions
DRUGPrednisone
For the experimental group, the patient will be administrated oral prednisone (0.5mg/kg/day, first 5 days per month, 6 months)
Sponsors
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, China
The First Affiliated Hospital of Nanchang University
The Third Affiliated Hospital of Southern Medical University
Shanghai Changzheng Hospital
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
If the diseae progression in the control group is confirmed, the patient is allowed to transferred to the test group (Oral prednisone).
Eligibility
Sex/Gender
ALL
Age
2 Years to 17 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Progression-free survival | 2 years | Any evidence proving the disease progression |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Local control rate | 2 years | radiological examinations of local lesion status |
| Pain relief | 2 years | 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. |
| Adverse effect rate | 2 years | side effects related to the prednisone |
Countries
China
Outcome results
None listed