Table 3. Baselines evaluation and SSc patient selection prior to HSCT according to ADWP consensus.
| Suggested baseline evaluations | |
| History | Complete history and examination including evaluation of quality of life by SHAQ-VAS, and/ or SF-36 questionnaires and of the extent of skin thickening by the mRSS. |
| Standard hematological, biological and urinary analyses and viral serologies | Blood cell count, ESR. |
| Chemistry: electrolytes, renal and liver function tests, proteins, CK, BNP or NT-proBNP, troponin T or troponin I, CRP. | |
| Urine: protein/creatinine ratio; urine analysis, casts. | |
| Viral serologies: CMV, HSV, VZV, EBV, VDRL, HIV, (HTLV-1,2), Hepatitis B and C and autoantibody titers. | |
| Immunology according to EBMT/EULAR Consensus Core Set | ANA, anti-Scl-70, anti-centromere and anti-polymerase RNA III antibodies (indicates high risk for renal crisis). |
| Immune-phenotyping by FACS of peripheral blood mononuclear cells = CD3+, CD4+, CD8+, CD4+ CD45RA, CD4+ CD45RO, CD31, CD3− CD56+ CD16+, CD19+, CD14+; IgG, IgA, IgM). | |
| Biobanking: storage of plasma, serum, RNA, DNA. | |
| Cardiopulmonary | Chest X-ray, and exercise test (6MWT) is recommended. |
| ECG with rhythm strip, 24 h Holter monitoring in clinically symptomatic patients (for example, palpitations, dizziness) or in those with rhythm abnormality on 12-lead ECG or rhythm strip. | |
| Comprehensive TTE with Doppler and tissue Doppler imaging. | |
| CMR with contrast and deep breathing cine images to evaluate for interventricular septal flattening or bounce. | |
| Invasive RHC including a fluid challenge. | |
| Contiguous and High resolution thoracic CT (or multi slice CT-scan with 6 HR section slices). | |
| Pulmonary function studies (including TLC, VC, DLCO, DLCO/VA, RV). | |
| Gastrointestinal | For patients with history of GAVE or anemia or iron deficiency, endoscopy should be performed and vascular ectasia, even if not actively bleeding, should undergo cauterization repeated on two or three occasions several weeks later to prevent massive bleeding during transplant induced thrombocytopenia. |
| Suggested exclusion criteria for AHSCT in SSc using a cyclophosphamide-based regimen. | |
| Age | >65 years. |
| Pregnancy | Pregnancy or unwillingness to use adequate contraception throughout investigation. |
| Psychiatric | Psychiatric disease including alcohol or drug abuse. |
| Consent | Inability to provide informed consent for treatment. |
| Liver function | Twofold increase in liver transaminases or bilirubin. |
| Failure of synthetic function or evidence of cirrhosis. | |
| Neoplasms | Neoplasms myelodysplasia or serious hematological disorders contraindicating AHSCT. |
| Infection | Active acute or chronic viral infection with HIV, HTLV-1,2, hepatitis B or C. |
| Heart | LVEF <45%. |
| mPAP >25 mmHg or PASP >40 mmHg without fluid challenge. | |
| mPAP >30 mmHg or PASP >45 mmHg with 1000 cc NS infused over 10 min. | |
| Diastolic septal flattening (D-sign). | |
| Septal bounce. | |
| Constrictive pericarditis. | |
| Cardiac tamponade. | |
| Significant atherosclerotic disease. | |
| Arrhythmias that cannot be pharmacologically controlled, cardioverted or ablated. | |
| Pulmonary | FVC <65%. |
| DLCO-SB <40%. | |
| Note: if pre-HSCT echocardiogram, CMR, and cardiac catheterization with and without fluid challenge demonstrate no contraindication, patients with lower DLCO and FVC may be considered candidates. | |
| Patients should be strongly encouraged to stop smoking. | |
| Renal | SSc renal crisis in the previous 6 months. |
| Glomerular filtration rate <40 mL/min/1.73 m2. | |
| Note: patients with renal crises or renal failure may be considered candidates if blood pressure is well controlled and dialysis is performed the morning following each cyclophophamide infusion, respectively. | |
Abbreviations: 6MWT=6 minutes walking test; ADWP=Autoimmune Disease Working Party; AHSCT= autologous hematopoietic stem cell transplantation; BNP=B-type natriuretic peptide; CMR=cardiac magnetic resonance; CT=computerized tomography; DLCO=diffusion of CO; DLCO/VA=KCO carbon monoxide transfer coefficient; ECG=electrocardiography; ESR=erythrocyte sedimentation rate; FVC=forced vital capacity; HR=high resolution; HTLV=human T-lymphotropic virus; LVEF=left ventricular ejection fraction; mPAP=mean pulmonary artery pressure; mRSS=modified Rodnan skin score; NT-proBNP=N-terminal pro-BNP; RV=residual volume; SSc=systemic sclerosis; TLC=total lung capacity; VA=alveolar volume; VC=vital capacity; VDRL=venereal disease research laboratory test.