Characteristics of XLP1 patients receiving allogeneic HSCT
| . | Percentage . | Number . | 1-y survival . | HR . | 95% CI . | P . |
|---|---|---|---|---|---|---|
| XLP1 features | ||||||
| Previous HLH | 37.2% | 16/43 | 50% | 23.93 | 5.31-108.0 | < .0001 |
| Previous NHL | 27.9% | 12/43 | 74.2% | 0.23 | 0.05-1.06 | .06 |
| Previous dysgammaglobulinemia | 46.5% | 20/43 | 80% | 1.2 | 0.29-4.96 | .77 |
| EBV+ | 51.2% | 21/41 | 75% | 1.37 | 0.36-5.3 | .65 |
| Age at HSCT | Mean 7 y (8 mo to 19 y 7 mo) | |||||
| 0-2 y | 9.3% | 4/43 | 75% | 5.75 | 0.11-302.1 | .38 |
| 2-5 y | 34.9% | 15/43 | 78.6% | 3.61 | 0.18-71.76 | .40 |
| 5-15 y | 48.8% | 21/43 | 85.7% | 3.16 | 0.11-90.83 | .50 |
| > 15 y | 7% | 3/43 | 100% | |||
| Year of HSCT | ||||||
| < 2000 | 7.0% | 3/43 | 66.7% | |||
| 2000-2005 | 37.2% | 16/43 | 87.5% | |||
| 2005-2009 | 55.8% | 24/43 | 79.2% | |||
| Donor Type | ||||||
| MSD, MFD | 30.4% | 14/46 | 91.77% | |||
| MUD, mMFD, mMUD | 60.9% | 28/46 | 77.8% | 0.42 | 0.08-2.07 | .27 |
| Haplo | 8.7% | 4/46 | 75% | 0.24 | 0.01-6.58 | .4 |
| Source | ||||||
| Bone marrow | 58.5% | 24/41* | 82.6% | |||
| Peripheral blood | 36.6% | 15/41* | 92.9% | |||
| Umbilical cord | 4.9% | 2/41* | 50% | |||
| Conditioning | ||||||
| MA | 50% | 23/46 | 82.9% | |||
| NMA | 50% | 23/46 | 78.9% | 1.25 | 0.30-5.2 | .77 |
| Serotherapy | 30.4% | 14/46 | ||||
| GVHD | 50% | 19/38 | ||||
| Grade 1 | 18.4% | 7/38 | ||||
| Grade 2-3 | 26.3% | 10/38 | ||||
| Grade 4 | 5.3% | 2/38 | ||||
| Chronic | 5.3% | 2/38 | ||||
| Chimerism | ||||||
| Full (> 98%) | 92% | 35/38 | 100% | |||
| Mixed | 8% | 3/38 | 88.8% | 2.98 | 0.06-151.0 | .59 |
| Replacement IVIg | 20% | 7/35† | ||||
| Alive | 81.4% | 35/43 | ||||
| Follow up | 6 wk to 148 mo |
| . | Percentage . | Number . | 1-y survival . | HR . | 95% CI . | P . |
|---|---|---|---|---|---|---|
| XLP1 features | ||||||
| Previous HLH | 37.2% | 16/43 | 50% | 23.93 | 5.31-108.0 | < .0001 |
| Previous NHL | 27.9% | 12/43 | 74.2% | 0.23 | 0.05-1.06 | .06 |
| Previous dysgammaglobulinemia | 46.5% | 20/43 | 80% | 1.2 | 0.29-4.96 | .77 |
| EBV+ | 51.2% | 21/41 | 75% | 1.37 | 0.36-5.3 | .65 |
| Age at HSCT | Mean 7 y (8 mo to 19 y 7 mo) | |||||
| 0-2 y | 9.3% | 4/43 | 75% | 5.75 | 0.11-302.1 | .38 |
| 2-5 y | 34.9% | 15/43 | 78.6% | 3.61 | 0.18-71.76 | .40 |
| 5-15 y | 48.8% | 21/43 | 85.7% | 3.16 | 0.11-90.83 | .50 |
| > 15 y | 7% | 3/43 | 100% | |||
| Year of HSCT | ||||||
| < 2000 | 7.0% | 3/43 | 66.7% | |||
| 2000-2005 | 37.2% | 16/43 | 87.5% | |||
| 2005-2009 | 55.8% | 24/43 | 79.2% | |||
| Donor Type | ||||||
| MSD, MFD | 30.4% | 14/46 | 91.77% | |||
| MUD, mMFD, mMUD | 60.9% | 28/46 | 77.8% | 0.42 | 0.08-2.07 | .27 |
| Haplo | 8.7% | 4/46 | 75% | 0.24 | 0.01-6.58 | .4 |
| Source | ||||||
| Bone marrow | 58.5% | 24/41* | 82.6% | |||
| Peripheral blood | 36.6% | 15/41* | 92.9% | |||
| Umbilical cord | 4.9% | 2/41* | 50% | |||
| Conditioning | ||||||
| MA | 50% | 23/46 | 82.9% | |||
| NMA | 50% | 23/46 | 78.9% | 1.25 | 0.30-5.2 | .77 |
| Serotherapy | 30.4% | 14/46 | ||||
| GVHD | 50% | 19/38 | ||||
| Grade 1 | 18.4% | 7/38 | ||||
| Grade 2-3 | 26.3% | 10/38 | ||||
| Grade 4 | 5.3% | 2/38 | ||||
| Chronic | 5.3% | 2/38 | ||||
| Chimerism | ||||||
| Full (> 98%) | 92% | 35/38 | 100% | |||
| Mixed | 8% | 3/38 | 88.8% | 2.98 | 0.06-151.0 | .59 |
| Replacement IVIg | 20% | 7/35† | ||||
| Alive | 81.4% | 35/43 | ||||
| Follow up | 6 wk to 148 mo |
Data missing on 5 transplants, 1 died during conditioning.
Three patients < 1 year after transplant.
CI indicates confidence interval; HR, hazard ratio; MSD, matched sibling donor; MFD, matched family donor; MUD, matched unrelated donor; mMFD, mismatched family donor; mMUD, mismatched unrelated donor; Haplo, haploidentical transplant; MA, myeloablative; and NMA, nonmyeloablative.