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ID
Source
Brief title
Health condition
Langerhans Cell Histiocytosis; Langerhans Cel Histiocytose
Sponsors and support
Department of Hematology
department of Hematology
Intervention
Outcome measures
Primary outcome
Definition and implementation of an uniform treatment for patients with single system LCH, multisystem LCH and pulmonary isolated LCH; implementation of uniform initial evaluation and stratification criteria.
Secondary outcome
- to explore the therapeutic efficacy on adult patients of the standard regimen for multisystem LCH in children, i.e. the combination of vinblastine and prednisone.
Endpoints will be survival, reactivation-free survival, permanent sequelae.
- to examine if an extended continuation therapy may reduce disease reactivations occuring after treatment completion in patients with multisystem LCH.
- to describe the natural history of isolated pulmonary disease and in particular the role of smoking cessation on the disease course.
- to explore the therapeutic efficacy of steroid monotherapy in adult patients with isolated pulmonary disease showing disease progression.
Background summary
LCH-A1 study is an international multicenter study for Langerhans Cell Histiocytosis.
This is a rare, tumor-like disease thas has an unpredicatable course and can be fatal. The cause of this disease is unknown.
The targeted number of participants is 1200 patients; for the Netherlands the targeted number is 20 patients.
The study is designed for 3 groups of patients.
Patient population in group 1 are patients with single system multifocal bone lesions or localized special site involvement.
Group 2 consists of patients with multisystem disease and group 3 consists of patients with isolated pulmonary disease.
Treatment for group 1 is 6 months of treatment with Prednisone, Vinblastine and Mercaptopurine.
Treatment for group 2 is the same as in group 1. Patients are randomized for 6 months treatment versus 12 months.
Treatment in group 3 is an observation phase for 6 months after smoking cessation. In case of progression of the symptoms or pulmonary dysfunction 6 months steroid monotherapy is given.
Duration of treatment is for all patients 6 months till 12 months and a follow up period of 6 years.
Study objective
Improvement of standardization in diagnosis and treatment of adult LCH
Study design
pre-treatment, at week 6 and every 3 months during treatment (i.e. month 3, month 6 and 9 when appropriate) and then at treatment completion.
thereafter every 6 months for the first 3 years. and once a year during the following 3 years.
Intervention
- Patients in group 1 (single system disease at risk): treatment with Prednisone, Vinblastine and Mercaptopurine.
Initial treatment: Prednisone 1 mg/kg/day ( not to exceed 60 mg) as a 4-week course, tapering over a period of 2 weeks.
Vinblastine 6 mg/m2 iv bolus (not to exceed 10 mg), day 1,8,15,22,29,36.
Continuation treatment: starting at day 43 after initial treatment. Mercaptopurine: 30 mg/m2(not to exceed 50 mg) daily until completion of treatment. Prednisone: 1 mg/kg/day (not to exceed 60 mg) day 1-5 every 3 weeks until completion of treatment.
Vinblastine: 6 mg/m2 iv bolus (not to exceed10 mg) day 1 every 3 weeks until completion of treatment. (starting 3 weeks after the last vinblastine injection of the initial treatment.
total duration of treatment is 6 months.
- Patients in group 2, multisystem LCH:treatment with Prednisone, Vinblastine and Mercaptopurine for 6 months versus treatment with Prednisone, Vinblastine and Mercaptopurine for 12 months.
Intervention for group 2 is the same as for group 1; total duration of treatment will be the object of randomization: 6 months vs 12 months.
- Patients in group 3 (isolated pulmonary disease): an observational period of 6 months afer cigarette smoke withdrawal. In case of progression of the symptoms of pulmonary dysfunction, treatment phase will be started with Prednisone monotherapy for 6 monthsat the following dosage:
- 1mg/kg/day (not to exceed 60 mg), daily for 1 month.
- 0.5 mg/kg/day, daily for 1 month.
- 0.25 mg/kg/day daily for 2 months.
- 0.125 mg/kg/day daily for 2 months.
Department of Hematology
De Boelelaan 1117
A.A. Loosdrecht, van de
Amsterdam 1081 HV
The Netherlands
00-31-20-4442604
a.vandeloosdrecht@vumc.nl
Department of Hematology
De Boelelaan 1117
A.A. Loosdrecht, van de
Amsterdam 1081 HV
The Netherlands
00-31-20-4442604
a.vandeloosdrecht@vumc.nl
Inclusion criteria
1. definitive diagnosis of LCH
2. no prior cytoreductive treatment for LCH
3. age 18-50 years for group 1 and 2
4. age 18-75 years for group 3
Exclusion criteria
1. patients with severe impairment of clinical condition including severely impaired pulmonary function, long term oxygen therapy or cor pulmonale.
2. treatment with immune suppressive agents and/or bisphosphonates within 4 weeks from baseline evaluation
Design
Recruitment
Followed up by the following (possibly more current) registration
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Other (possibly less up-to-date) registrations in this register
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In other registers
| Register | ID |
|---|---|
| NTR-new | NL1171 |
| NTR-old | NTR1216 |
| Other | MEC VU University Medical Center Amsterdam : 2007/169 |
| ISRCTN | ISRCTN wordt niet meer aangevraagd |