Table 2.

HSCT for CGD with active inflammation/inflammatory sequelae

Patient no. (reference)CGD typeAge, ySexSex of donorMarrow MNCs infused per kg, × 108ConditioningRisk factors
(lung function)		Outcome
(lung function)		Myeloid engraftment (extent of hemopoiesis)
10 gp91phox 17 M F* 3.2 Bu, Cy Colitis; pulmonary restriction (FVC 60%) Colitis resolved; GVHD 4 → chronic → resolving 1.3 y (full) 
11 gp91phox 4.3 M M 1.9 Bu, Cy Colitis, steroid dependent; previous Aspergilluspneumonia Colitis resolved; alive and well 1.2 y (full)  
12 (Di Bartolomeo et al7) gp91phox 11 M F* 4.7 Bu 13, Cy Pulmonary restriction (FVC 34%, O2dependent) Restriction ↓ (FVC 72%, no O2); alive and well 12 y (full)  
13 (Nagler et al8) X-CGD 6 M F PBSCs Bu 8,‡ Flu 240, ATG Pulmonary restriction (FVC 28%, Sa O2 75%, clubbed) + lung cysts Lung improved (Sa O2 87%); skin GVHD 2; alive and well 4 y (full, after DLI)  
14 p47phox 5 F F 1.1 Bu, Cy Pulmonary restriction (DLCO 40%,
O2 dependent, clubbed) 		Restriction ↓ (DLCO 76%, no O2, clubbing ↓); Catch-up-growth; alive and well 3 y (full) 
15 gp91phox 4.4 M F 5.9 Bu, Cy Pulmonary restriction (clubbed) + local bronchiectases Restriction ↓ (clubbing ↓); catch-up-growth; skin GVHD 2; alive and well 1.5 y (88%) 
16 X-CGD 38.7 M M PBSCs TBI 200,‡Flu 180 Pulmonary restriction (FVC 38%, O2 dependent, clubbed, wheel chair) + bronchiectases Lung improved (no O2, out of chair); alive and well 1.6 y (full) 
Patient no. (reference)CGD typeAge, ySexSex of donorMarrow MNCs infused per kg, × 108ConditioningRisk factors
(lung function)		Outcome
(lung function)		Myeloid engraftment (extent of hemopoiesis)
10 gp91phox 17 M F* 3.2 Bu, Cy Colitis; pulmonary restriction (FVC 60%) Colitis resolved; GVHD 4 → chronic → resolving 1.3 y (full) 
11 gp91phox 4.3 M M 1.9 Bu, Cy Colitis, steroid dependent; previous Aspergilluspneumonia Colitis resolved; alive and well 1.2 y (full)  
12 (Di Bartolomeo et al7) gp91phox 11 M F* 4.7 Bu 13, Cy Pulmonary restriction (FVC 34%, O2dependent) Restriction ↓ (FVC 72%, no O2); alive and well 12 y (full)  
13 (Nagler et al8) X-CGD 6 M F PBSCs Bu 8,‡ Flu 240, ATG Pulmonary restriction (FVC 28%, Sa O2 75%, clubbed) + lung cysts Lung improved (Sa O2 87%); skin GVHD 2; alive and well 4 y (full, after DLI)  
14 p47phox 5 F F 1.1 Bu, Cy Pulmonary restriction (DLCO 40%,
O2 dependent, clubbed) 		Restriction ↓ (DLCO 76%, no O2, clubbing ↓); Catch-up-growth; alive and well 3 y (full) 
15 gp91phox 4.4 M F 5.9 Bu, Cy Pulmonary restriction (clubbed) + local bronchiectases Restriction ↓ (clubbing ↓); catch-up-growth; skin GVHD 2; alive and well 1.5 y (88%) 
16 X-CGD 38.7 M M PBSCs TBI 200,‡Flu 180 Pulmonary restriction (FVC 38%, O2 dependent, clubbed, wheel chair) + bronchiectases Lung improved (no O2, out of chair); alive and well 1.6 y (full) 

DLCO indicates diffusing capacity for CO; DLI, donor lymphocyte infusion; FVC, forced vital capacity; TBI, total body irradiation. For other abbreviations see Table 1 footnote.

*

Heterozygous carrier of X-CGD.

‡

Nonmyeloablative.

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