HSCT for CGD with therapy-refractory infection
| Patient no. (reference) . | CGD type . | Age, y . | Sex . | Sex of donor . | Marrow MNCs infused per kg, × 108 . | Conditioning . | Risk factors (organs involved) . | Outcome (day of death) . | Myeloid engraftment (extent of hemopoiesis) . |
|---|---|---|---|---|---|---|---|---|---|
| 1 | X-CGD | 11.5 | M | F | 1.7 | Bu, Cy | Disseminated aspergillosis (lung, heart, bone) | Multiorgan failure (died d +9) | (None) |
| 2 | X-CGD | 17 | M | F | 3.5 | Bu, Cy | Multifocal aspergillosis (lung, CNS) | Nodular pneumonia; skin GVHD IV (died d +90) | 2 mo (full) |
| 3 | X-CGD | 20 | M | F | 4.3 | Bu, Melphalan, alemtuzumab | Multifocal fungal infection* (lung, paraspinal) | Interstitial pneumonia Tracheostomy bleeding (died d +73) | 2 mo (full) |
| 4 | X-CGD carrier | 6.8 | F | M | 5† | Bu 8,‡ Flu 180, ATG | Aspergillosis (lung) | Nonengraftment; Aspergillus pneumonia; VOD (died d +46) | (None) |
| 5 | gp91phox | 5.9 | M | M | 81-153 | Cy 120,‡ Flu 125, ATG | Mycobacteriosis1-155 (lung, nodes) | Nonengraftment autologous reconstitution, alive | (None) |
| 6 (Ozsahin et al5) | gp91phox | 8 | M | F1-154 | 4 | Bu, Cy | Multifocal aspergillosis (lung, rib, psoas) | Alive and well | 5 y (full) |
| 7 | p22phox | 14.4 | F | M | 3.5 | Bu, Cy | Multifocal aspergillosis (lung, CNS) | Aspergilluspneumonia; skin GVHD 4 → chronic 4 y → resolved; alive and well | 4 y (full) |
| 8 (Bielorai et al6 | gp91phox | 3 | M | M | PBSCs | Bu, Cy, ATG | Aspergillosis (lung, rib) | Skin GVHD 3 → chronic → resolved, alive and well | 1.8 y (full) |
| 9 | X-CGD | 16.8 | M | F | 2 | Bu 16, Flu 200, ATG | Fungal gastritis# | Alive and well | 1 y (full) |
| Patient no. (reference) . | CGD type . | Age, y . | Sex . | Sex of donor . | Marrow MNCs infused per kg, × 108 . | Conditioning . | Risk factors (organs involved) . | Outcome (day of death) . | Myeloid engraftment (extent of hemopoiesis) . |
|---|---|---|---|---|---|---|---|---|---|
| 1 | X-CGD | 11.5 | M | F | 1.7 | Bu, Cy | Disseminated aspergillosis (lung, heart, bone) | Multiorgan failure (died d +9) | (None) |
| 2 | X-CGD | 17 | M | F | 3.5 | Bu, Cy | Multifocal aspergillosis (lung, CNS) | Nodular pneumonia; skin GVHD IV (died d +90) | 2 mo (full) |
| 3 | X-CGD | 20 | M | F | 4.3 | Bu, Melphalan, alemtuzumab | Multifocal fungal infection* (lung, paraspinal) | Interstitial pneumonia Tracheostomy bleeding (died d +73) | 2 mo (full) |
| 4 | X-CGD carrier | 6.8 | F | M | 5† | Bu 8,‡ Flu 180, ATG | Aspergillosis (lung) | Nonengraftment; Aspergillus pneumonia; VOD (died d +46) | (None) |
| 5 | gp91phox | 5.9 | M | M | 81-153 | Cy 120,‡ Flu 125, ATG | Mycobacteriosis1-155 (lung, nodes) | Nonengraftment autologous reconstitution, alive | (None) |
| 6 (Ozsahin et al5) | gp91phox | 8 | M | F1-154 | 4 | Bu, Cy | Multifocal aspergillosis (lung, rib, psoas) | Alive and well | 5 y (full) |
| 7 | p22phox | 14.4 | F | M | 3.5 | Bu, Cy | Multifocal aspergillosis (lung, CNS) | Aspergilluspneumonia; skin GVHD 4 → chronic 4 y → resolved; alive and well | 4 y (full) |
| 8 (Bielorai et al6 | gp91phox | 3 | M | M | PBSCs | Bu, Cy, ATG | Aspergillosis (lung, rib) | Skin GVHD 3 → chronic → resolved, alive and well | 1.8 y (full) |
| 9 | X-CGD | 16.8 | M | F | 2 | Bu 16, Flu 200, ATG | Fungal gastritis# | Alive and well | 1 y (full) |
ATG indicates antithymocyte globulin; Bu, busulphan; CNS, central nervous system; Cy, cyclophosphamid; Flu, fludarabine; GVHD, graft-versus-host disease; MNCs, mononuclear cells; PBSCs, peripheral blood stem cells; VOD, veno-occlusive disease; →, evolves into.
Scedosporium apiospermum.
0.9 × 106 CD34 cells per kilogram.
Nonmyeloablative.
11 × 106 CD34 cells per kilogram.
Mycobacterium gordonae.
Heterozygous carrier of X-CGD.
#Ustilago species.