Systemic sclerosis
Conditions
Interventions
Cyclophosphamide 2g/m2, 2 days Harvest of autologous hematopoietic stem cells Cyclophosphamide 50mg/kg, 4 days Transplantation of autologous hematopoietic stem cells
Sponsors
Kyushu University
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: A patient diagnosed as systemic sclerosis with performance status (ECOG) 0 to 2. a) Within 4 years after onset and modified Rodnan total skin thickness score (mRSS) is 15 or more, and applicable to one of the following organ complication 1) Pulmonary complications: interstitial pneumonia diagnosed by chest X-ray examination or chest CT, and %FVC or %DLCO is less than 80%. 2) Renal complications: systolic blood pressure is 160 mmHg or more or diastolic blood pressure is 110 mmHg or more, urinalysis abnormality (urine protein, urine occult blood, urinary sediment), microangiopathic hemolytic anemia, or elevated serum creatinine 3) cardiac complications: reversible heart failure, or arrhythmia (supraventricular, ventricular, AV block), or pericardial effusion b) 1) Involvement of sclerosis to the trunk within 2 years after onset, mRSS is 20 or more, and 2) ESR is 25 mm/1h or more and/or Hb is 11 g/dL or less. c) Progressive interstitial pneumonia during the course even though more than 4 years after onset or lower than 15 mRSS.
Exclusion criteria
Exclusion criteria: A patient with one of the following severe organ complication, a) Heart: uncontrollable arrhythmia, uncontrollable heart failure, LVEF is less than 50% in cardiac ultrasound examination, moderate or more pulmonary hypertension (mean pulmonary artery pressure is 40 mmHg or more) b) Lung: PaO2 is less than 60 mmHg at room air, %VC is less than 50%. %DLCO is less than 20%. c) Kidney: renal glomerular filtration rate is less than 40 ml/min, serum creatinine is 2 mg/dl or more. d) Uncontrolled malignant neoplasm e) Uncontrollable infection. f) Cyclophosphamide is used more than 10 g in total.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Event-free survival at 2 years after transplantation | — |
Secondary
| Measure | Time frame |
|---|---|
| Event-free survival at 1, 3 and 5 years after transplantation Improvement of modified Rodnan total skin thickness score at 1, 3 and 5 years after transplantation Improvement of %VC at 1, 3 and 5 years after transplantation Safety, Progression-free survival, Overall survival | — |
Countries
Japan
Contacts
Public ContactHiroki Mitoma
Kyushu University Hospital Department of Clinical Immunology and Rheumatology / Infectious Disease
Outcome results
None listed