Table 3 Detailed information of all patients regarding allogeneic stem cell transplantation and outcome
From: The efficacy and safety of allogeneic stem cell transplantation in Mevalonate Kinase Deficiency
P | Age at HSCT (yrs) | Conditioning | Donor type | Graft source | HLA match | Acute GvHD | Follow-up period after SCT (months) | Chimerism in myeloid cells at last follow-up (method) | neurological manifestations at last follow-up | Normal schooling | Normal growth | Outcome |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
1 | 1.1 | ALG, cy, VP16, flu, mel. | MUD | CB | 8/8 | Grade II (skin/gut) | 1 | – | hydrocephalus | NA | NA | Death |
2 | 2.6 | Cy, busulfan | MSD | BM | 10/10 | (−) | 194 | 100% (FISH) | No | yes | low weight | Alive |
3 | 2.9 | Cy, busulfan ATG | MUD | CB | 8/12 | Grade II (skin) | 149 | 100% (STR) | PRES. Seizures a | yes | yes | Alive |
4 | 0.7 | treo, flu, TT alfa/beta/Cd19 depletion | MMRD (haplo) | PBSC | 6/12 | (−) | 307 | 100% (STR) | No | yes | yes | Alive |
5 | 1.8 | treo, flu, TT, alfa/beta/Cd19 depletion | MMRD (haplo) | PBSC | 6/12 | (−) | Graft failure | |||||
2.0 | busulfan, flu, alemtuzumab, alfa/beta/Cd19 depletion | MMRD | PBSC | 6/12 | (−) | 16 | 100% (STR) | No | yes | yes | Alive | |
6 | 6.6 | treo, flu, TT ATG, rituximab | MMRD (haplo) | PBSC | 6/12 | (−) | 60 | 100% (STR) | No | yes | yes | Alive |
7 | 0.4 | treo, flu | MSD | BM | (−) | 43%inBM | NAb | Alive | ||||
2 | busulfan, flu, ATG | MUD | BM | 10/10 | – | 38 | 100% (STR) | No | NA | yes | Alive | |
8 | 1.7 | treo, flu, TT, ATG | MMUD | BM | 9/10 | Grade IV (skin/liver) | 3 | – | No | NA | NA | Death |
9 | 8.3 | busulfan, cy | MSD | BM | 12/12 | Grade IV (skin/gut) | 127 | 100% STR | No | yes, now working | yes | Alive |