Table 1 Patient and transplant characteristics and outcome at latest follow-up.
Patient | Pre-HCT morbidity | %NOB | Age at HCT (years) | Stem cell source and HLA match | Conditioning regimen Serotherapy | Peri-HCT morbidity | Latest follow-up | %NOB | Outcome |
|---|---|---|---|---|---|---|---|---|---|
P1 | Recurrent pneumonia, lymphadenitis, discoid rash, colitis, polyarthralgia | 25% | 15 | 10/10 MUD PBSC | Treosulfan Fludarabine Thiotepa Alemtuzumab | HHV6 viraemia Inflammatory pneumonitis AIHA | 3.5 years | 100% | Premature ovarian failure Colitis in remission No infection |
P2 | Staphylococcal liver abscess, recurrent skin infection, oral ulcers | 10% | 18 | 10/10 MUD PBSC | Treosulfan Fludarabine Thiotepa Alemtuzumab | Encephalopathy Anuric renal failure | - | 100% (D + 30) | Death at D + 30 from encephalopathy |
P3 | Severe colitis despite infliximab, methotrexate, certolizumab | 30% | 20 | 10/10 MUD BM | Busulfan Fludarabine Alemtuzumab | Grade I acute skin GvHD | 7.5 years | 100% | Remission of colitis |
P4 | Pulmonary nocardiosis necessitating extracorporeal membrane oxygenation, discoid lupus | 6.7% | 56 | 10/10 MSD PBSC | Busulfan Alemtuzumab PTCy | Grade I acute skin GvHD Klebsiella bacteraemia | 3.0 years | 60% | Autoimmunity in remission No infection |
P5 | Pulmonary nocardiosis, extensive colitis with rectovaginal fistulae | 3.5% | 22 | 5/10 parental PBSC | Busulfan Fludarabine PTCy | Grade III liver/gastrointestinal GvHD Disseminated adenovirus infection | - | 98% (D + 100) | Death at D + 131 from multi-organ failure |
P6 | Pneumonia, Burkholderia gladioli abscess Hypogammaglobulinaemia | 15% | 7 | 10/10 MSD BM | Busulfan Fludarabine Alemtuzumab | CMV viraemia | 2.0 years | 100% | Infection free |
P7 | Bilateral pneumonia, colitis | 1.2% | 1 | 9/10 TCRαβ/CD19-deplete PBSC | Busulfan Fludarabine Alemtuzumab | Uncomplicated | 3.0 years | 100% | Colitis in remission Infection free |
