Oral Prednisone in Treating LCH of Bone in Childhood and Adolescence

Study Overview
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Brief Summary
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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].

Official Title
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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

Conditions
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Langerhans Cell Histiocytosis of Bone

Intervention / Treatment
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* Drug: Prednisone


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Crossover Assignment
Interventional Model Description: If the diseae progression in the control group is confirmed, the patient is allowed to transferred to the test group (Oral prednisone).
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Oral prednisone<br>Oral prednisone, 0.5mg/kg/day, first 5 days/month, 6months | Drug: Prednisone<br>* For the experimental group, the patient will be administrated oral prednisone (0.5mg/kg/day, first 5 days per month, 6 months)<br>|
| No Intervention: Regular observation<br>Regular observation and follow up without medication | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Progression-free survival | Any evidence proving the disease progression | 2 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Local control rate | radiological examinations of local lesion status | 2 years | 
| Pain relief | 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. | 2 years | 
| Adverse effect rate | side effects related to the prednisone | 2 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Langerhans cell histiocytosis, Bone, Single-system, Children, Adolescents