Severe systemic sclerosis
Conditions
Interventions
Autologous CD34+ stem cells-selected transplantation
Sponsors
Department of Clinical Immunology and Rheumatology/Infectious Disease, Kyushu University Graduate school of Medicine
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: (1) definite diagnosis of Systemic Sclerosis using 2013 ACR/EULAR SSc criteria (2) between the age of 16 and 65 (3) ECOG Performance Status of 0 to 2 (4) refractory to corticosteroids or immunosuppressive therapy (5) fall under any of the following a) with maximum disease duration of 4 years, minimum modified Rodnan skin score of 15 and organ involvement as follows: 1.interstitial pneumonia diagnosed by Chest Xp or CT and less than 80% in %FVC or %DLCO 2.past history of renal crisis 3.reversible heart failure, arrhythmia or pericardial effusion b) with maximum disease duration of 2 years, minimum modified Rodnan skin score of 20 and erythrocyte sedimentation rate greater than 25 mm in the first hour and/or hemoglobin less than 11 g/dl (6) able to understand and willing to sign the Informed Consent Form
Exclusion criteria
Exclusion criteria: (1) severe organ dysfunction as following a) severe arrhythmia leading to permanent implantation of a pacemaker or implantable cardioverter defibrillator, refractory chronic heart failure, reduced left ventricular ejection fraction lower than 50% at echocardiogram or severe pulmonary hypertension (mPAP >=30 mmHg) b) severe lung dysfunction (PaO2 <60 mmHg under room air conditions, %FVC <50% or %DLOC <20%) c) severe renal dysfunction (eGFR <40 ml/min or serum Cr >=2 mg/dl) d) d) liver abnormality (baseline AST, ALT or total bilirubin levels are >3 times higher than upper limit of normal) (2) presence of malignancy except for complete remission (3) presence of active infection (4) positive for HBs-Ag, HCV-Ab, HIV-Ab or HTLV1-Ab test (5) cumulative dose of more than 10 g of cyclophosphamide (6) history of anaphylaxis or hypersensitivity to murine IgG or iron-dextran complex (7) pregnancy or lactation (8) judged by the investigator to be inappropriate for study participation
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The mean change in modified Rodnan skin score at 12 months after transplantation | — |
Secondary
| Measure | Time frame |
|---|---|
| The proportion of 25% improvement in skin score at 12 months The mean change in forced vital capacity at 12 months The mean change in serum KL-6 levels at 12 months Event-free survival Overall survival Incidence of adverse events | — |
Countries
Japan
Contacts
Public ContactHiroki Mitoma
Kyushu University hospital Department of Clinical Immunology and Rheumatology/Infectious Disease
Outcome results
None listed