Table 2 General considerations for HSCT in RMDs.
Criterion | Specific recommendations for ADs |
|---|---|
Age | 18–65 years old. In exceptional cases, older patients could be considered along with co-morbidity assessment and the agreement of the MDT (level III). |
Disease status and evaluation | HSCT should be considered as a therapeutic option in patients with RMDs, which are active or progressive despite the use of standard (guideline-based and/or regulatory approved) therapy (level II). |
Centre and team requirement | In patients for whom HSCT represents a treatment option, referral should be made to a centre with an appropriate experience combining haematological and RMD specialist teams. These units should be experienced in selecting and managing severe and refractory RMD patients. Such centres should have established multidisciplinary team meetings and/or similar processes for HSCT, involving both a RMD and haematology focused program in the same centre to support thorough assessment, treatment and follow-up (level II). |
General clinical assessment | Performance status, HCT-CI [86], QoL questionnaires [87], smoking absence (with date of cessation if applicable), weight/height/body mass index, blood pressure/heart rate, full physical examination and nutritional assessment should be assessed (level III). Careful MDT evaluation is needed for patients with poor performance status (ECOG ≥2, Karnofsky ≤60%) related to the underlying disease likely to reverse after HSCT (level III). |
Patient compliance | Patient compliance and understanding of the procedure and expectations is essential, as a basis for providing informed written consent for HSCT procedure and sharing registry data (level III). Social service and psychological assessment includes an evaluation for post-traumatic stress disorder and psychosocial support and structure needed to cope with disease treatment and follow up (level III). |
Fertility | Fertility assessment and gamete preservation must be proposed to RMD patients before HSCT. Consideration should be given to chemotherapy induced infertility, risk of premature menopause, and ultimate need for hormone replacement therapy, where appropriate. Fertility assessment is mandatory (level III). |
Exclusion of pregnancy | Pregnancy should be excluded within 7 days of administering mobilization or conditioning chemotherapy (level III). |
Neoplasia screening | A complete assessment to exclude neoplasia should be done according to national recommendations and/or local practices (level III). |
Baseline laboratory and imaging assessments | Haematological assessment: complete blood counts, reticulocytes; coagulation; transfusion work-up (including blood group). Biochemistry: liver function, renal function and electrolytes, bone profile, blood protein electrophoresis and albumin, blood glucose, haemoglobin A1c, vitamins, iron status and other relevant nutrients, CRP or other inflammatory parameters. Cardiac assessment: Cardiac biomarkers (Troponin hs, NT-proBNP); ECG plus a 24-h Holter according to the AD type and the presence of any clinical or electrical abnormal signs; transthoracic echocardiography with LVEF, systolic PAP and tricuspid flow measures. Extended cardiac assessment as per SSc section and other disease specific sections should be performed. Lung assessment: CT scan, pulmonary function tests with measurement of haemoglobin-corrected DLCO. Infectious assessment: Search for and/or treatment of any ongoing or past infections as clinically appropriate. Dental assessment less than 4 months. Syphilis and tuberculosis screening. HIV1 and 2, HTLV 1 and 2, HBV, HCV, CMV, HSV, EBV, VZV, toxoplasma serologies. EBV blood PCR and CMV blood PCR. SARS-COV-2 test according to local practice; aspergillosis assessment; further assessments including parasitic according to patient’s travel history to endemic areas should be considered. |
