Systemic sclerosis.
Conditions
Interventions
This multicenter prospective randomized controlled phase III study compares efficacy and safety of high dose immunoablation and autologous hematopoietic stem cell transplantation (HSCT) (considered th
Sponsors
EBMT/EULAR Working Party Autoimmune Diseases.
Eligibility
Inclusion criteria
Inclusion criteria: Patients with diffuse systemic sclerosis, aged 16-65 yrs, and: 1. Disease duration 4 years or less, plus evidence of heart, lung or kidney involvement, plus skin score 15 or more, or: 2. Disease duration 2 years or less, plus evidence of an acute phase reaction in blood, plus skin score 20 or more.
Exclusion criteria
Exclusion criteria: Patients with concomitant severe disease, extensive pretreatment according to predefined criteria with cyclophosphamide are excluded.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary endpoint is event-free survival defined as the time in days from the day of randomization until the occurrence of death or the development of persistent major organ failure (heart, lung, kidney) during the study period of 2 years. | — |
Secondary
| Measure | Time frame |
|---|---|
| Key secondary outcomes include: treatment related mortality, treatment toxicity, and progression-free survival, defined as the time in days since the day of randomization until death or predefined changes in skin score, organ function, body weight, functional disability from baseline has been documented at two consecutive 3-month evaluations within the study period of 2 years. | — |
Contacts
Public ContactJ.M. Laar, van
Leiden University Medical Center (LUMC), Department of Rheumatology, P.O. Box 9600
Outcome results
None listed