Details of XLP1 patients surviving allogeneic HSCT
Year of HSCT . | EBV . | HLH . | Age at HSCT . | Donor . | Conditioning/serotherapy/graft manipulation . | GVHD prophylaxis . | GVHD . | Chimerism . | Follow up (mo) . | Ig . |
---|---|---|---|---|---|---|---|---|---|---|
1997 | NK | 7 y | MSD | Cy, TBI | MTX, CSA | 1 S* | 100% | 148 | ||
1998 | 1 y | MUD | Bu, Cy, ATG | MTX, CSA, P | 1 S | 100% | 133 | |||
2000 | + | 4 y | MUD | Bu, Cy, Campath | CSA | 2 S | 100% | 102 | ||
2000 | + | Yes | 3 y | mMUD | Bu, Cy | MTX, CSA | 2 S, L | 100% | 107 | |
2001 | 4 y | MUD | Flu, Melph, ATG, TBI | MMF, CSA | 100% | 102 | ||||
2001 | + | 10 y | MSD | Bu, Cy, VP-16 (NHL) | MTX, CSA | 2-3 GI | 100% | 99 | ||
2001 | 4 y | MSD | Bu, Cy | CSA | 2 S | 100% | 95 | |||
2002 | 13 y | MSD | Thio, Flu, ATG | CSA | 100% | 88 | ||||
2002 | + | 7 y | MUD | Bu, Cy, ATG | MTX, CSA | 1 S | 100% | 85 | ||
2002 | 3 y | MUD | Bu, Cy, ATG | MTX, CSA | 100% | 84 | ||||
2003 | 8 mo | mMUD | Flu, Melph, ATG, TBI | TAC, MTX, P | S | 100% | 79 | |||
2003 | 19 y | MSD | Thio, Flu, ATG | CSA | 100% | 71 | ||||
2003 | 11 y | mMUD | Flu, Melph, ATG | MMF, CSA | 100% | 68 | ||||
2004 | + | 5 y | MSD | Bu, Cy | MTX, CSA | 20% PBMC | 66 | |||
2004 | + | 12 y | mMFD | Flu, Melph, Campath, 34+ | MMF, CSA | 4 S, L* | 100% | 62 | Y | |
2004 | + | 8 y | mMUD | Flu, Melph, Campath | CSA | 2-3 S, GI | 100% | 57 | ||
2005 | + | Yes | 2 y | Haplo | Bu, Cy, ATG, 34+ | CSA | 100% | 54 | Y | |
2005 | 2 y | Haplo | Bu, Cy, ATG, 34+, top up 1 year | CSA | 88% PBMC 97% M | 50 | Y | |||
2005 | 12 y | mMUD | Flu, Melph, Campath, 34+ | MMF, CSA | 3 S | 100% | 46 | |||
2005 | 18 y | MUD | NK | NK | 46 | NK | ||||
2006 | 5 y | MSD | Bu, Cy | CSA | 1 G, 3 S | 100% | 42 | |||
2006 | NK | 2 y | MUD | Bu, Flu, Campath | MTX | 100% | 42 | |||
2006 | + | 7 y | Haplo | Flu, Melph, Thio, OKT3, ATG | 100%, 75% CD3 | 39 | ||||
2006 | + | Yes | 1 y | MUD | Flu, Melph, Ritux | CSA | 1 S | 5% | 38 | |
2006 | + | 11 y | MSD | Bu, Cy, ATG | MTX, CSA | 100% | 35 | |||
2006 | 4 y | MUD | Bu, Cy, Campath | MMF, CSA | 1 S | 100% | 33 | |||
2007 | + | Yes | 6 y | MSD | Bu, Cy | MTX, CSA | 99% | 30 | ||
2007 | + | Yes | 7 y | MSD | Bu, Cy | CSA | 3 S, L, GI | 100% | 29 | |
2007 | NK | 7 y | MUD | Flu, Melph, TBI | TAC, MTX | 98% | 27 | |||
2007 | 7 y | MSD/mMUD | Bu, Cy | CSA | 2 S, GI | 100% | 26 | |||
2008 | 17 y | MFD | Flu, Melp, Campath | MMF, CSA | 100% | 13 | ||||
2008 | Yes | 3 y | MUD | Bu, Flu | TAC, MTX | 100% | 9 | Y | ||
2009 | + | 7 y | MUD | Bu, Cy | 100% | 6 | Y | |||
2009 | + | Yes | 6 y | mMUD | Flu, Melph, Campath | CSA, MMF | 1 S | 100% | 5 | Y |
2009 | + | Yes | 3 y | MUD | Thio, Cy, ATG | CSA, P | 100% | 4 | Y |
Year of HSCT . | EBV . | HLH . | Age at HSCT . | Donor . | Conditioning/serotherapy/graft manipulation . | GVHD prophylaxis . | GVHD . | Chimerism . | Follow up (mo) . | Ig . |
---|---|---|---|---|---|---|---|---|---|---|
1997 | NK | 7 y | MSD | Cy, TBI | MTX, CSA | 1 S* | 100% | 148 | ||
1998 | 1 y | MUD | Bu, Cy, ATG | MTX, CSA, P | 1 S | 100% | 133 | |||
2000 | + | 4 y | MUD | Bu, Cy, Campath | CSA | 2 S | 100% | 102 | ||
2000 | + | Yes | 3 y | mMUD | Bu, Cy | MTX, CSA | 2 S, L | 100% | 107 | |
2001 | 4 y | MUD | Flu, Melph, ATG, TBI | MMF, CSA | 100% | 102 | ||||
2001 | + | 10 y | MSD | Bu, Cy, VP-16 (NHL) | MTX, CSA | 2-3 GI | 100% | 99 | ||
2001 | 4 y | MSD | Bu, Cy | CSA | 2 S | 100% | 95 | |||
2002 | 13 y | MSD | Thio, Flu, ATG | CSA | 100% | 88 | ||||
2002 | + | 7 y | MUD | Bu, Cy, ATG | MTX, CSA | 1 S | 100% | 85 | ||
2002 | 3 y | MUD | Bu, Cy, ATG | MTX, CSA | 100% | 84 | ||||
2003 | 8 mo | mMUD | Flu, Melph, ATG, TBI | TAC, MTX, P | S | 100% | 79 | |||
2003 | 19 y | MSD | Thio, Flu, ATG | CSA | 100% | 71 | ||||
2003 | 11 y | mMUD | Flu, Melph, ATG | MMF, CSA | 100% | 68 | ||||
2004 | + | 5 y | MSD | Bu, Cy | MTX, CSA | 20% PBMC | 66 | |||
2004 | + | 12 y | mMFD | Flu, Melph, Campath, 34+ | MMF, CSA | 4 S, L* | 100% | 62 | Y | |
2004 | + | 8 y | mMUD | Flu, Melph, Campath | CSA | 2-3 S, GI | 100% | 57 | ||
2005 | + | Yes | 2 y | Haplo | Bu, Cy, ATG, 34+ | CSA | 100% | 54 | Y | |
2005 | 2 y | Haplo | Bu, Cy, ATG, 34+, top up 1 year | CSA | 88% PBMC 97% M | 50 | Y | |||
2005 | 12 y | mMUD | Flu, Melph, Campath, 34+ | MMF, CSA | 3 S | 100% | 46 | |||
2005 | 18 y | MUD | NK | NK | 46 | NK | ||||
2006 | 5 y | MSD | Bu, Cy | CSA | 1 G, 3 S | 100% | 42 | |||
2006 | NK | 2 y | MUD | Bu, Flu, Campath | MTX | 100% | 42 | |||
2006 | + | 7 y | Haplo | Flu, Melph, Thio, OKT3, ATG | 100%, 75% CD3 | 39 | ||||
2006 | + | Yes | 1 y | MUD | Flu, Melph, Ritux | CSA | 1 S | 5% | 38 | |
2006 | + | 11 y | MSD | Bu, Cy, ATG | MTX, CSA | 100% | 35 | |||
2006 | 4 y | MUD | Bu, Cy, Campath | MMF, CSA | 1 S | 100% | 33 | |||
2007 | + | Yes | 6 y | MSD | Bu, Cy | MTX, CSA | 99% | 30 | ||
2007 | + | Yes | 7 y | MSD | Bu, Cy | CSA | 3 S, L, GI | 100% | 29 | |
2007 | NK | 7 y | MUD | Flu, Melph, TBI | TAC, MTX | 98% | 27 | |||
2007 | 7 y | MSD/mMUD | Bu, Cy | CSA | 2 S, GI | 100% | 26 | |||
2008 | 17 y | MFD | Flu, Melp, Campath | MMF, CSA | 100% | 13 | ||||
2008 | Yes | 3 y | MUD | Bu, Flu | TAC, MTX | 100% | 9 | Y | ||
2009 | + | 7 y | MUD | Bu, Cy | 100% | 6 | Y | |||
2009 | + | Yes | 6 y | mMUD | Flu, Melph, Campath | CSA, MMF | 1 S | 100% | 5 | Y |
2009 | + | Yes | 3 y | MUD | Thio, Cy, ATG | CSA, P | 100% | 4 | Y |
Chronic GVHD.
PBMC indicates peripheral blood mononuclear cell; Flu, fludarabine; Melph, melphalan; 34+, CD34+ stem cell infusion; Bu, busulfan; Cy, cyclophosphamide; Thio, thiotepa; TBI, total body irradiation; CSA, cyclosporin A; MMF, mycophenalate mofetil; MTX, methotrexate; P, prednisolone; TAC, tacrolimus; S, skin; GI, gastrointestinal; L, lung; and Ig, replacement immunoglobulin.