Transplantation characteristics of group B (patients with nonmalignant diseases)
| No . | Age, y/sex . | Disease . | Indication for RIT . | Stem cell source . | RIT BM dose, Gy* . | Conditioning regimen† . | Clinical course after HCT . | Chimerism after HCT . | Survival status . |
|---|---|---|---|---|---|---|---|---|---|
| B-1 | 20/M | β-Thalassemia | Severe hemosiderosis (liver fibrosis) | MUD/PBSC | 17 | Flu (160), Mel (140), ATG | Uneventful course | 100% donor | Alive (> 49 mo) |
| B-2 | 14/M | Congenital hemolytic anemia | Severe hemosiderosis (liver fibrosis) | MFD/BM | 16 | Flu (160), Mel (70), ATG | Uneventful course | 100% donor | Alive (> 47 mo) |
| B-3 | 4/M | RAG1-SCID (B-low T-low NK+) | 2nd HCT, active infection (pneumonitis) | Haplo/TDPBSC | 20 | Flu (160), ATG | Graft failure; 3rd HSCT from 2nd haplo donor; rejection; 4th HSCT from 3rd haplo donor; GVHD; died of adenovirus pneumonitis | Rejection | Dead (13 mo) |
| B-4 | 6/F | RAG1-CID (B-low T-low NK+) | Active infection (aspergillosis, EBV lymphoma) | MUD/PBSC | 20 | Flu (160), ATG | Uneventful course | 100% donor | Alive (> 46 mo) |
| B-5 | 10/M | Hyper-IgE syndrome (AR) | Active infection (pneumonitis) | MUD/PBSC | 16 | Flu (160), Mel (140), ATG | EBV infection | 100% donor | Alive (> 43 mo) |
| B-6 | 8/F | RAG1-CID (B-low, T-low NK+) | Active infection (pneumonitis) | MUD/BM | 17 | Flu (160), Mel (140), ATG | Adenovirus and EBV infection; chronic GVHD (extended/severe); tricytopenia; died of infection | 100% donor | Dead (36 mo) |
| B-7 | 19/M | Congenital hemolytic anemia | Severe hemosiderosis (liver fibrosis) | MUD/PBSC | 16 | Flu (160), Mel (140), ATG | Candida sepsis; Candida meningitis/osteomyelitis; CMV reactivation | 100% donor | Alive (> 40 mo) |
| B-8 | 7/F | β-Thalassemia | 2nd HCT (autologous reconstitution) | MFD/BM | 20 | Mel (140) | Bacterial sepsis (Escherichia coli); CMV reactivation | 100% donor | Alive (> 35 mo) |
| B-9 | 4/M | CGD | Active infection (aspergillosis) | MUD/BM | 18 | Flu (160), Mel (70), ATG | Uneventful course | 30% donor | Alive (> 30 mo) |
| B-10 | 9/M | CGD | Active infection (pneumonia) | MUD/BM | 17 | Flu (160), Mel (70), ATG | Bacterial sepsis; acute pancreatitis; severe colitis; pneumonitis (ongoing oxygen requirement) | 100% donor | Alive (> 27 mo) |
| B-11 | 10/M | IFN-γ–receptor deficiency | Active infection (mycobacteriosis) | MUD/BM | 17 | Flu (160), Mel (140), ATG | Bacterial sepsis; CMV reactivation; colitis (adenovirus and rotavirus); mycobacteriosis | 100% donor | Alive (> 26 mo) |
| B-12 | 10/M | Hyper-IgM syndrome | Hepatopathy (cryptosporidiosis) | MFD/BM | 16 | Flu (160), Mel (70) | Prolonged FUO; deterioration of liver function/cholestasis; enteropathy | 100% donor | Alive (> 25 mo) |
| B-13 | 5/F | Griscelli syndrome | 2nd HCT (autologous reconstitution) | Haplo/TDPBSC | 17 | Nothing | Uneventful course | 100% donor | Alive (> 24 mo) |
| B-14 | 1/M | SCID (ZAP70 deficiency) | Active infection (PCP pneumonitis, cholestasis) | MUD/BM | 17 | Flu (160), Mel (140), ATG | Cholestasis/liver cirrhosis; chronic GVHD (extended/moderate) | 100% donor | Alive (> 23 mo) |
| B-15 | 14/F | IFN-γ–receptor deficiency | 2nd HSCT (autologous reconstitution) | MFD/BM | 17 | Flu (160), Mel (70), ATG | Adenovirus infection, chronic GVHD (extended/moderate) | 100% donor | Alive (23 mo) |
| B-16 | 1/M | Hyper-IgM syndrome | Hepatopathy | MMFD/BM | 18 | Flu (160), Mel (140), ATG | EBV-associated lymphoproliferative syndrome | 10% donor | Alive (22 mo) |
| No . | Age, y/sex . | Disease . | Indication for RIT . | Stem cell source . | RIT BM dose, Gy* . | Conditioning regimen† . | Clinical course after HCT . | Chimerism after HCT . | Survival status . |
|---|---|---|---|---|---|---|---|---|---|
| B-1 | 20/M | β-Thalassemia | Severe hemosiderosis (liver fibrosis) | MUD/PBSC | 17 | Flu (160), Mel (140), ATG | Uneventful course | 100% donor | Alive (> 49 mo) |
| B-2 | 14/M | Congenital hemolytic anemia | Severe hemosiderosis (liver fibrosis) | MFD/BM | 16 | Flu (160), Mel (70), ATG | Uneventful course | 100% donor | Alive (> 47 mo) |
| B-3 | 4/M | RAG1-SCID (B-low T-low NK+) | 2nd HCT, active infection (pneumonitis) | Haplo/TDPBSC | 20 | Flu (160), ATG | Graft failure; 3rd HSCT from 2nd haplo donor; rejection; 4th HSCT from 3rd haplo donor; GVHD; died of adenovirus pneumonitis | Rejection | Dead (13 mo) |
| B-4 | 6/F | RAG1-CID (B-low T-low NK+) | Active infection (aspergillosis, EBV lymphoma) | MUD/PBSC | 20 | Flu (160), ATG | Uneventful course | 100% donor | Alive (> 46 mo) |
| B-5 | 10/M | Hyper-IgE syndrome (AR) | Active infection (pneumonitis) | MUD/PBSC | 16 | Flu (160), Mel (140), ATG | EBV infection | 100% donor | Alive (> 43 mo) |
| B-6 | 8/F | RAG1-CID (B-low, T-low NK+) | Active infection (pneumonitis) | MUD/BM | 17 | Flu (160), Mel (140), ATG | Adenovirus and EBV infection; chronic GVHD (extended/severe); tricytopenia; died of infection | 100% donor | Dead (36 mo) |
| B-7 | 19/M | Congenital hemolytic anemia | Severe hemosiderosis (liver fibrosis) | MUD/PBSC | 16 | Flu (160), Mel (140), ATG | Candida sepsis; Candida meningitis/osteomyelitis; CMV reactivation | 100% donor | Alive (> 40 mo) |
| B-8 | 7/F | β-Thalassemia | 2nd HCT (autologous reconstitution) | MFD/BM | 20 | Mel (140) | Bacterial sepsis (Escherichia coli); CMV reactivation | 100% donor | Alive (> 35 mo) |
| B-9 | 4/M | CGD | Active infection (aspergillosis) | MUD/BM | 18 | Flu (160), Mel (70), ATG | Uneventful course | 30% donor | Alive (> 30 mo) |
| B-10 | 9/M | CGD | Active infection (pneumonia) | MUD/BM | 17 | Flu (160), Mel (70), ATG | Bacterial sepsis; acute pancreatitis; severe colitis; pneumonitis (ongoing oxygen requirement) | 100% donor | Alive (> 27 mo) |
| B-11 | 10/M | IFN-γ–receptor deficiency | Active infection (mycobacteriosis) | MUD/BM | 17 | Flu (160), Mel (140), ATG | Bacterial sepsis; CMV reactivation; colitis (adenovirus and rotavirus); mycobacteriosis | 100% donor | Alive (> 26 mo) |
| B-12 | 10/M | Hyper-IgM syndrome | Hepatopathy (cryptosporidiosis) | MFD/BM | 16 | Flu (160), Mel (70) | Prolonged FUO; deterioration of liver function/cholestasis; enteropathy | 100% donor | Alive (> 25 mo) |
| B-13 | 5/F | Griscelli syndrome | 2nd HCT (autologous reconstitution) | Haplo/TDPBSC | 17 | Nothing | Uneventful course | 100% donor | Alive (> 24 mo) |
| B-14 | 1/M | SCID (ZAP70 deficiency) | Active infection (PCP pneumonitis, cholestasis) | MUD/BM | 17 | Flu (160), Mel (140), ATG | Cholestasis/liver cirrhosis; chronic GVHD (extended/moderate) | 100% donor | Alive (> 23 mo) |
| B-15 | 14/F | IFN-γ–receptor deficiency | 2nd HSCT (autologous reconstitution) | MFD/BM | 17 | Flu (160), Mel (70), ATG | Adenovirus infection, chronic GVHD (extended/moderate) | 100% donor | Alive (23 mo) |
| B-16 | 1/M | Hyper-IgM syndrome | Hepatopathy | MMFD/BM | 18 | Flu (160), Mel (140), ATG | EBV-associated lymphoproliferative syndrome | 10% donor | Alive (22 mo) |
RAG1-SCID indicates RAG1-related severe combined immunodeficiency; B-low T-low NK+, B and T cells below the normal range, normal NK-cells; AR, autosomal recessive; RAG1-CID, RAG1-related combined immunodeficiency disease; CGD, chronic granulomatous disease; IFN-γ, interferon-γ; FUO, fever of unknown origin; ZAP70, ζ-chain–associated protein kinase 70; and PCP, pneumocystis pneumonia. Other abbreviations are defined as in Table 2.
Calculated absorbed doses in BM.
Drugs used in the conditioning regimen were as follows, with doses given in parentheses in the table: fludarabine (mg/m2); etoposide (mg/kg); melphalan (mg/m2); and intravenous busulfan (applied dose was weight adapted according to the prescription of the manufacturer).