Table 2.
Follow-up of patients who underwent stem cell transplant for IPEX
| Study | Cases | Follow-up time | Donor chimerism % (unfractionated leukocytes unless specified) | Developed GVHD: yes/no | Off IST at last follow-up yes/no | Vital status: survived/died | Notes: complications: posttransplant |
|---|---|---|---|---|---|---|---|
| Rao [67] | 1 | 25 | 100 | Yes | No | Survived | Complications: Clostridium difficile, Enterococcus faecalis, s Histoplasma capsulatum, Enterococcus faecium, and Staphylococcus hominis |
| 2 | 19 | 100 | No | Yes | Survived | ||
| 3 | 11 | 89 | No | Yes | Survived | ||
| 4 | 6 | 85 | No | No | Survived | ||
| Mazollari [68] | 1 | 16 | T 70, B 30, PMN 50 | Yes | Yes | Survived | No complications |
| Baud [69] | 1 | 23 | Mixed chimerism | No | Yes | Died | Developed hemophagocytic syndrome |
| Wildin [34] | 1 | 5 | 100 decreasing to 50 | No | Yes | Died | Pneumonia |
| 2 | 3 | 100 decreasing to 70 | No | Yes | Died | EBV and CMV infection, sclerosing pancreatitis, | |
| Zhan [70] | 1 | 30 | 100 | Yes | Yes | Survived | No complications |
| Seidel [71] | 1 | 6 years | Range: 53–91 | Yes | Yes | Survived | No complication |
| Nadeemi [72] | 1 | 10 years | T 68, B 57, CD15 57 | No | Yes | Survived | Autoimmune cytopenias |
| 2 | 9 years | 100 | No | Yes | Survived | ||
| 3 | 8 years | 100 | No | Yes | Survived | ||
| 4 | 5 years | T 32, B 36, CD15 31 | Yes | Yes | Survived | ||
| 5 | 3.5 years | T 41, B 35, CD15 34 | No | Yes | Survived | ||
| Lucas [73] | 1 | 14 months | 81 at 1 month, 98 at 8 months | Yes | Yes | Survived | EBV |
| Kucuk [74] | 1 | 197 days | Mixed | No | Yes | Died | Autoimmune cytopenias |
| 2 | 93 days | Mixed | No | Yes | Died | ||
| 3 | 8 years | Mixed | No | Yes | Survived | ||
| 4 | 7 years | Mixed | No | No | Survived | ||
| 5 | 5 years | – | Yes | No | Survived | ||
| 6 | 4 years | Mixed | No | No | Survived | ||
| 7 | 77 days | Mixed | No | No | Survived | ||
| Burroughs [75] | 1 | 4 years | 100 | Yes | Yes | Survived | No improvement in diabetes and hypothyroidism. Grade 4 pulmonary toxicity and bacteremia |
| 2 | 4 years | Mixed | Yes | Yes | Survived | ||
| Dorsey [76] | 1 | NA | 100 | No | Yes | Survived | Staphylococcus epidermides bacteremia and HTN |
| Kasow [77] | 1 | 3 years | Mixed | No | Yes | Survived | Resolution of all clinical disease |
| Horino [58] | 1 | 2 years | Mixed | Yes | No | Survived | GVHD of skin (grade 1) and development of ALL at 2 years |
ALL acute lymphoblastic leukemia, CMV cytomegalovirus, HSCT hematopoietic stem cell transplant, HTN hypertension, EBV Epstein–Barr virus, NA not applicable