Details of XLP1 patients not surviving allogeneic HSCT
| EBV . | HLH . | Age at HSCT, y . | Year of HSCT . | Donor . | Conditioning/serotherapy/graft manipulation . | GVHD prophylaxis . | GVHD . | Chimerism . | Cause of death . |
|---|---|---|---|---|---|---|---|---|---|
| + | Yes | 2 | 2005 | MMFD | Flu, TBI | N/A | Died during conditioning 6 wk from presentation | ||
| + | Yes | 3 | 2003 | MUD | Bu, Flu, Campath, Rituximab | CSA | Died 3 d after HSCT disease progression | ||
| Yes | 6 | 2005 | MMFD | Bu, TBI | MMF. MTX, P | Died 14 d after HSCT MDR pseudomonal sepsis | |||
| + | Yes | 3 | 2009 | Haplo | Bu, Cy, ATG | TCD | Died 3 wk after HSCT VOD, MOF, renal toxicity | ||
| + (after HSCT) | Yes | 5 | 2008 | mMUD (cord plus PBSC 4 months later) | Bu, Flu, ATG then Flu, TBI | TAC, P | 100% | Died 2 mo after second HSCT EBV, fungal, and ?PCP sepsis | |
| Yes | 3 | 1998 | MSD × 2 | Flu, Melph | CSA, P | 100% | Died 3 mo after HSCT Pseudomonas sepsis | ||
| + | Yes | 12 | 2003 | MUD | Bu, Cy, Flu, Campath | 4 S | 100% | Died 3 mo after HSCT disseminated adenovirus | |
| Yes | 1 | 2007 | MUD | Flu, Melph, ATG, 34+ | CSA | 2-3 S, L | 100% | Died 3 mo after HSCT paraflu III sepsis |
| EBV . | HLH . | Age at HSCT, y . | Year of HSCT . | Donor . | Conditioning/serotherapy/graft manipulation . | GVHD prophylaxis . | GVHD . | Chimerism . | Cause of death . |
|---|---|---|---|---|---|---|---|---|---|
| + | Yes | 2 | 2005 | MMFD | Flu, TBI | N/A | Died during conditioning 6 wk from presentation | ||
| + | Yes | 3 | 2003 | MUD | Bu, Flu, Campath, Rituximab | CSA | Died 3 d after HSCT disease progression | ||
| Yes | 6 | 2005 | MMFD | Bu, TBI | MMF. MTX, P | Died 14 d after HSCT MDR pseudomonal sepsis | |||
| + | Yes | 3 | 2009 | Haplo | Bu, Cy, ATG | TCD | Died 3 wk after HSCT VOD, MOF, renal toxicity | ||
| + (after HSCT) | Yes | 5 | 2008 | mMUD (cord plus PBSC 4 months later) | Bu, Flu, ATG then Flu, TBI | TAC, P | 100% | Died 2 mo after second HSCT EBV, fungal, and ?PCP sepsis | |
| Yes | 3 | 1998 | MSD × 2 | Flu, Melph | CSA, P | 100% | Died 3 mo after HSCT Pseudomonas sepsis | ||
| + | Yes | 12 | 2003 | MUD | Bu, Cy, Flu, Campath | 4 S | 100% | Died 3 mo after HSCT disseminated adenovirus | |
| Yes | 1 | 2007 | MUD | Flu, Melph, ATG, 34+ | CSA | 2-3 S, L | 100% | Died 3 mo after HSCT paraflu III sepsis |
PBSC indicates peripheral blood stem cell; Flu, fludarabine; Melph, melphalan; 34+, CD34+ stem cell infusion; Bu, busulphan; Cy, cyclophosphamide; Thio, thiotepa; TBI, total body irradiation; CSA, cyclosporine A; MMF, mycophenalate mofetil; MTX, methotrexate; P, prednisolone; TAC, tacrolimus; TCD, T-cell depletion; S, skin; L, lung; VOD, veno-occlusive disease; MOF, mullti-organ failure; MDR, multidrug resistant; and PCP, Pneumocystis jiroveci.