Table 1.
Patient Number | Diagnosis | Gender | Indication for Transplant | Infections pre-HSCT | Age at Transplant | Donor | Conditioning Regimen |
---|---|---|---|---|---|---|---|
1 | IPEX | Male | PID with severe eczema/allergies, enteropathy with TPN dependence, and FTT | 10 months | Haplo related | Alem, Flu, Cy, Mel, TBI | |
2 | Birc4/XIAP deficiency | Male | HLH | 11 months | Haplo related | Alem, Flu, Mel, TBI | |
3 | DKC | Female | BMF | 19 years | 10/10 MUD | Alem, Flu, TBI | |
4 | CGD | Male | PID; brother with life-threatening infections | 5 months | 10/10 MUD | Alem, Flu, Mel | |
5 | CGD | Male | PID with life-threatening infections, enteritis, FTT | Klebsiella pneumonia, Paecilomyces pneumonia | 5 years | Haplo related | Alem, Flu, Mel, TBI |
6 | DKC | Male | BMF | 14 years | 10/10 MUD | Alem, Flu, TBI | |
7 | CGD | Male | PID with life-threatening infections; enteritis, FTT | MRSA bacteremia, fungal pneumonia | 8 years | 10/10 MUD | Alem, Flu, Bu |
8 | Hyper IgM | Male | LPD | 12 years | 10/10 MUD | Alem, Flu, Mel, TBI | |
9 | DBA | Male | Steroid refractory | 3 years | 10/10 MUD | Alem, Flu, Mel | |
10 | HLH | Female | Chemotherapy refractory | 21 years | 5/8 MMUD | Alem, Flu, Mel, TBI | |
11 | GT | Female | Platelet-refractory bleeding | 8 years | Haplo related | Alem, Flu, Mel |
Alem, alemtuzumab, BMF, bone marrow failure; Bu, busulfan; CGD, chronic granulomatous disease; Cy, cyclophosphamide, DBA Diamond-Blackfan anemia; DKC, dyskeratosis congenita; Flu, fludarabine; FTT, failure to thrive; GT, Glanzmann’s thrombasthenia; HLH, hemophagocytic lymphohistiocytosis; HSCT, hematopoietic stem cell transplant; IPEX, immune dysregulation polyendocrinopathy X-linked; LPD, lymphoproliferative disease; Mel, melphalan; MMUD mismatched unrelated donor; MRSA, methicillin-resistant staphylococcus aureus; MUD, matched unrelated donor; PID, primary immunodeficiency; TBI, total body irradiation; TPN, total parenteral nutrition.