Table 1.
Clinical details of the patients studied
| Patient no. | Age, y | Sex | Previous treatment(s) | Conditioning before cellular therapy | Current therapy | Hemoglobin, g/dL | Platelets/μL | WBC, cells/μL | Differential |
|---|---|---|---|---|---|---|---|---|---|
| ADA1 | 23 | Female | PEG-ADA, T-lymphocyte gene therapy | None | PEG-ADA, TMP/SFX | 13.4 | 242 000 | 4810 | N 67.6%, L 11.4%, M 12.9%, E 7.3%, B 0.8% |
| ADA3 | 12 | Male | Haploidentical HCT | None | PEG-ADA, IVIG, TMP/SFX | 13.9 | 383 000 | 3790 | N 79.4%, L 9.0%, M 9.5%, E 1.8%, B 0.3% |
| ADA4 | 9 | Male | Haploidentical HCT | None | PEG-ADA, IVIG, TMP/SFX | 12.2 | 235 000 | 1730 | N 47.5%, L 21.3%, M 25.5%, E 5.7%, B 0.0% |
| ADA5 | 7 | Male | PEG-ADA, HSC gene therapy | Busulfan 90 mg/m2 | SCIG, Pentamidine | 11.5 | 319 000 | 4680 | N 72.7%, L 6.4%, M 13.7%, E 5.6%, B 1.5% |
| ADA6 | 6 | Male | Haploidentical HCT | None | PEG-ADA, SCIG, TMP/SFX, itraconazole | 11.2 | 425 000 | 4000 | N 45.0%, L 3.0%, M 5.0%, E 46.0%, B 1.0% |
| ADA7 | 6 | Male | Haploidentical HCT | None | PEG-ADA, IVIG, TMP/SFX | 13.5 | 453 000 | 7490 | N 70.0%, L 5.6%, M 8.2%, E 14.9%, B 1.4% |
| ADA9 | 3 | Male | None | NA | PEG-ADA, SCIG, TMP/SFX, Acyclovir | 13.5 | 277 000 | 6180 | N 20.4%, L 61.3%, M 14.7%, E 2.8%, B 0.8% |
| ADA10 | 2 | Male | HSC gene therapy | Busulfan 90 mg/m2 | PEG-ADA, IVIG, TMP/SFX | 11.2 | 267 000 | 1020 | N 31.4%, L 13.7%, M 8.8%, E 45.1%, B 1.0% |
| ADA12 | 12 | Female | HSC gene therapy | None | PEG-ADA, IVIG, TMP/SFX | 14.8 | 326 000 | 5120 | N 74.2%, L 5.7%, M 8.3%, E 11.4%, B 0.4% |
| ADA13 | 27 | Female | PEG-ADA, T-lymphocyte gene therapy, HSC gene therapy | None | PEG-ADA | 13.1 | 290 000 | 3340 | N 71.2%, L 8.7%, M 11.3%, E 8.8%, B 0.0% |
| ADA14 | 3 | Female | PEG-ADA, HSC gene therapy | Busulfan 75 mg/m2 | IVIG, TMP/SFX, itraconazole, acyclovir | 13.2 | 262 000 | 2280 | N 71.0%, L 7.0%, M 17.0%, E 3.0%, B 2.0% |
| ADA16 | 4 | Male | PEG-ADA, HSC gene therapy | Busulfan 75 mg/m2 | acyclovir, TMP/SFX | 12.3 | 293 000 | 2670 | N 60.0%, L 5.0%, M 13.0%, E 12.0%, B 2.0% V8.0% |
| ADA18 | 5 | Female | None | NA | PEG-ADA, TMP/SFX | 11.8 | 252 000 | 5040 | N 72.3%, L 8.3%, M 7.1%, E 12.1%, B 0.2% |
HCT indicates hematopoietic cell transplantation; HSC, hematopoietic stem cell; PEG-ADA, pegylated bovine adenosine deaminase; TMP/SFX, trimethoprim/sulfamethoxazole; IVIG, intravenous immunoglobulin; NA, not applicable, SCIG, subcutaneous immunoglobulin; WBC, white blood cells; N, neutrophils; L, lymphocytes; M, monocytes; E, eosinophils; B, basophils; and V, variant lymphocytes.