Table 2.
Summary of HSC depicting conditioning regimen, donor source, and graft type
| Study | MPS type | Number of Patients | Conditioning Regimen | Donor source (patients) | Graft Type | Donor chimerism | Incidence of GVHD (patients) | Survival |
|---|---|---|---|---|---|---|---|---|
| Souillet et al. 2003 [8] | MPS IH | 27 | 27 - Bu, Cy + ATG for unrelated mismatche dBMT | 13-MRD 14-MUD | 2-CB 17-BM | 70% ≥ 95% donor chimerism | 5-aGvHD | 85% |
| Boelens et al. 2007[35] | MPS IH | 146 | 68 - Bu-Cy 200mg/kg 30-Bu with high dose Cy 240–260mg/kg 15 - Bu-targeting 17 - Flu-based myeloablative 18 - RIC | 96 - HLA MD | 103 BM 20 PB 23 - CB | 71% - full donor chimerism after initial transplantation | 26-aGvHD grade I 15 - aGvHD grade II 3-aGvHD grade III 5-aGvHD grade IV 8 - cGvHD | 85% after initial transplantation |
| Tuberville et al. 2011[17] | MPS VI | 45 | 6-Cy + TBI ± other 30 - Bu + Cy ± other 1 - Flu + Mel 7 - Other | 15-HLA-MSD 3-MRD 27-MUD | 34 BM 1-PB 10-CB | N/A | 15-aGvHD grade II-IV 34-aGvHD grades III-IV 19-GvHD | 78% at 100 days 66% at 1 and 3 years |
| Aldenhoven et al. 2015 [37] | 52-MPS IH 2-MPS II 2-MPS III 2-MPS VI | 62 | 29 - BuCy 33 - FluBu | 44-HLA MD | 41 CB 21 BM or PB | 88.2% -full donor chimerism | 8-aGvHD grade II-IV 8-cGvHD | 95.20% |
| Wang et al. 2016 [18] | 12-MPS I 12-MPS II 4-MPS IVA 4-MPS VI 2-unknown | 34 | 21 -Bu, Cy, ATG 8 - Bu, Cy, Flu, ATG | 11–4/6 to 6/6 HLA-MUD 4-MSD 2-MRD 17-MUD | 11 - CB 23 - PB | 31-full donor chimerism | 14-aGvHD grade II to IV 4-aGv HD grade III to IV 2-modera te-to-severe chronic GvHD | 84.8% ± 6.3% at 3 years |
| Lum et al. 2017 [82] | MPS IH | 240 | 145 - Bu/Cy ± serotherap Y 40-Bu/Flu ± serotherap y 55 - other |
67 - MRD 151 - MUD 22 - mismatc hed donor | 132BM 16 - PB 92 - CB | 80% - full donor chimerism of 85 patients assessed | 70-aGvHD grade I -II 22 aGvHD grade III - IV 14-cGvHD | 85.20% |
| Rodgers et al. 2017 [84] | MPS IH | 134 | 35 - targeted Bu 99 - no targeted Bu | 35-MRD 97-MUD | 83 - BM 49 - CB 2 - PB | 100 - ≥ 90% 21 – 10–89% 12 - <10% 1 unknown | 70% at 1 year |
Note: BM – bone marrow; PB – peripheral blood; CB – cord blood; MRD – matched related donor; MUD – matched unrelated donor, MSD – matched sibling donor; MD – matched donor; misMUD – mismatched unrelated donor; TBI – total body irradiation; Cy – cyclophosphamide; Flu – Fludarabine; Mel – Melphalan; ATG – Thymoglobulin; RIC – reduced-intensity conditioning; aGVHD – acute graft-vs-host disease; cGvHD – chronic graft-vs-host disease; ivBU – intravenous busulfan