Table 2

Details of transplantation and outcome (n = 17)

Case no.Age at trans-plantationDonorHLA matchGraft
ConditioningGVHD prophylaxisComplications
Severe infectionsLength of follow-up from the first trans-plantation, monthsOutcome
SourceCD3+, × 106/kgCD34+, × 106/kgAcute GVHDChronic GVHDOther
1 3 mo and day 35, day 63, day 91 Brother 10/10 PBL 100 0 None None Skin, stage I, maximum grade 1 None Transitory aregenerative anemia None 48 Alive and well 
2 6 mo and mo 45 Brother 10/10 PBL; marrow 0.4 0; 5 None None Liver, stage II, maximum grade 2 None Mild transient arthritis, foot cellulitis None 79 Alive and well 
3 13 mo and mo 6 Brother 10/10 Marrow NA NA ATG; Bu + CY CyA, MTX None None None None 12 Sudden death at home, cause unknown 
4 3 mo Sister 10/10 PBL 80 NA None None None None None CMV, rotavirus 3 Died of pneumonitis (CMV) 
5 5 mo Unrelated, male 12/12 Marrow 160 14 None None None None None None 2 Died of circulatory failure during cardiac surgery 
6 3 mo Unrelated, male 10/10 Marrow NA 500 None CyA None None None Streptococcus spp. 144 Alive, developmental delay 
7 6 mo Father 5/10 Marrow* 0.02 20 None CyA None None None None 4 Died of respiratory failure 
8 3 mo and day 55 Unrelated, female 11/12 Marrow 91 3.8 Campath CyA Skin, stage III, maximum grade 2 None None None 5 Died of circulatory failure 
9 4 mo Unrelated, male 10/10 Cord blood 77 1.8 Campath CyA, steroids Skin, stage II, maximum grade 2 None None Parainfluenza 6 Died of pneumonitis (parainfluenza) associated with recurrent GER 
10 2 mo and ? Sister 10/10 PBL; marrow NA NA None CyA Gut, stage II, maximum grade 2 Maximum grade 2 None NA 20 Alive, retransplanted with marrow December 2006; last follow-up May 15, 2007, no details given 
11 4.5 y Sister 10/10 PBL 10 0.07 None CyA, MMF None None None None 96 Alive and well, autoimmune thyroiditis and eczema resolved after transplant 
12 6 mo and day 36 Unrelated, male 9/10 Marrow 1 0.2 None None; CyA Skin and liver, stage III-IV, grade 2 None Cardiopulmonary instability, capillary leak syndrome, ileus, feeding problems, breath-holding attacks EBV reactivation (rituximab) 48 Alive, developmental delay, feeding difficulties 
13 7 mo Unrelated, male 10/10 Marrow 71 8.1 None CyA, MMF Skin, stage III; liver and gut, Stage II-IV; maximum grade 4 Skin, gut, maximum grade 4 Peritonitis associated with gastrostomy tube leakage, hypertension, renal impairment EBV reactivation (rituximab), coagulase-negative Staphylococcus spp, E coli, C albicans 8 Died of pneumonia, chronic GVHD 
14 6 mo Unrelated, male 10/10 Marrow 56 24 OKT3, steroids CyA, MTX Skin, stage II; gut, stage II; maximum grade 2 Maximum grade 2 Pneumopathy None 18 Died of hypertrophic cardiomyopathy, pneumopathy, suspected pulmonary GVHD 
15 3 mo Unrelated, male 10/10 PBL 0.5 NA Fludarabine CyA Skin, stage III, maximum grade 2 None Pulmonary VOD, chronic pleural effusions, pulmonary edema, bowel edema, anasarca, Evans syndrome B pertusis, HHV6 positivity (cleared with ganciclovir) 14 Died of acute renal failure, intractable hyperkalemia and left ventricular dysfunction after open lung biopsy 
16 5 mo Brother 10/10 Marrow NA NA None None None None None None 62 Alive and well 
17 3 mo Mother 10/10 PBL† 10 NA None None Skin, stage III-IV; lung suspected, maximum grade 2 Suspected Transient pancytopenia None 11 Died of pulmonary hemorrhage after second cardiac surgery 
Case no.Age at trans-plantationDonorHLA matchGraft
ConditioningGVHD prophylaxisComplications
Severe infectionsLength of follow-up from the first trans-plantation, monthsOutcome
SourceCD3+, × 106/kgCD34+, × 106/kgAcute GVHDChronic GVHDOther
1 3 mo and day 35, day 63, day 91 Brother 10/10 PBL 100 0 None None Skin, stage I, maximum grade 1 None Transitory aregenerative anemia None 48 Alive and well 
2 6 mo and mo 45 Brother 10/10 PBL; marrow 0.4 0; 5 None None Liver, stage II, maximum grade 2 None Mild transient arthritis, foot cellulitis None 79 Alive and well 
3 13 mo and mo 6 Brother 10/10 Marrow NA NA ATG; Bu + CY CyA, MTX None None None None 12 Sudden death at home, cause unknown 
4 3 mo Sister 10/10 PBL 80 NA None None None None None CMV, rotavirus 3 Died of pneumonitis (CMV) 
5 5 mo Unrelated, male 12/12 Marrow 160 14 None None None None None None 2 Died of circulatory failure during cardiac surgery 
6 3 mo Unrelated, male 10/10 Marrow NA 500 None CyA None None None Streptococcus spp. 144 Alive, developmental delay 
7 6 mo Father 5/10 Marrow* 0.02 20 None CyA None None None None 4 Died of respiratory failure 
8 3 mo and day 55 Unrelated, female 11/12 Marrow 91 3.8 Campath CyA Skin, stage III, maximum grade 2 None None None 5 Died of circulatory failure 
9 4 mo Unrelated, male 10/10 Cord blood 77 1.8 Campath CyA, steroids Skin, stage II, maximum grade 2 None None Parainfluenza 6 Died of pneumonitis (parainfluenza) associated with recurrent GER 
10 2 mo and ? Sister 10/10 PBL; marrow NA NA None CyA Gut, stage II, maximum grade 2 Maximum grade 2 None NA 20 Alive, retransplanted with marrow December 2006; last follow-up May 15, 2007, no details given 
11 4.5 y Sister 10/10 PBL 10 0.07 None CyA, MMF None None None None 96 Alive and well, autoimmune thyroiditis and eczema resolved after transplant 
12 6 mo and day 36 Unrelated, male 9/10 Marrow 1 0.2 None None; CyA Skin and liver, stage III-IV, grade 2 None Cardiopulmonary instability, capillary leak syndrome, ileus, feeding problems, breath-holding attacks EBV reactivation (rituximab) 48 Alive, developmental delay, feeding difficulties 
13 7 mo Unrelated, male 10/10 Marrow 71 8.1 None CyA, MMF Skin, stage III; liver and gut, Stage II-IV; maximum grade 4 Skin, gut, maximum grade 4 Peritonitis associated with gastrostomy tube leakage, hypertension, renal impairment EBV reactivation (rituximab), coagulase-negative Staphylococcus spp, E coli, C albicans 8 Died of pneumonia, chronic GVHD 
14 6 mo Unrelated, male 10/10 Marrow 56 24 OKT3, steroids CyA, MTX Skin, stage II; gut, stage II; maximum grade 2 Maximum grade 2 Pneumopathy None 18 Died of hypertrophic cardiomyopathy, pneumopathy, suspected pulmonary GVHD 
15 3 mo Unrelated, male 10/10 PBL 0.5 NA Fludarabine CyA Skin, stage III, maximum grade 2 None Pulmonary VOD, chronic pleural effusions, pulmonary edema, bowel edema, anasarca, Evans syndrome B pertusis, HHV6 positivity (cleared with ganciclovir) 14 Died of acute renal failure, intractable hyperkalemia and left ventricular dysfunction after open lung biopsy 
16 5 mo Brother 10/10 Marrow NA NA None None None None None None 62 Alive and well 
17 3 mo Mother 10/10 PBL† 10 NA None None Skin, stage III-IV; lung suspected, maximum grade 2 Suspected Transient pancytopenia None 11 Died of pulmonary hemorrhage after second cardiac surgery 

Semicolons separate data on each transplantation; time is rounded; PBL is always unmobilized.

NA indicates data not available; ATG indicates rabbit antithymocyte globulin; Bu, busulfan; CY, cyclophosphamide; CyA, cyclosporine A; MTX, methotrexate; MMF, mycophenolate mofetil; VOD, veno-occlusive disease; CMV, cytomegalovirus; EBV, Epstein-Barr virus; HHV6, human herpesvirus 6; and GER, gastroesophageal reflux.

*

T cell–depleted marrow.

†

B cell–depleted PBLs.

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