Immunologic status at 2 years after gene therapy
| PIN . | On ERT* . | Lymphocyte subsets† . | Serum immunoglobulins‡ . | PHA (%LLN)§ . | TREC/μg PBMC DNA¶ . | |||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| CD3 (ref. range) . | CD4 (ref. range) . | CD8 (ref. range) . | CD19 (ref. range) . | CD16 (ref. range) . | %CD45RA/CD4+ (ref. range) . | IgG (ref. range) . | IgA (ref. range) . | IgM (ref. range) . | ||||
| 201C | Yes | 0.371 (1.00-2.20) | 0.181 (0.53-1.30) | 0.192 (0.33-0.92) | 0.002 (0.11-0.57) | 0.003 (0.07-0.48) | Not done | IVIg# | 171 (70-312) | 97 (56-352) | 30.3 | 2.18 |
| 202N | Yes | 0.067 (1.20-2.60) | 0.061 (0.65-1.50) | 0.004 (0.37-1.10) | 0.062 (0.27-0.86) | 0.133 (0.10-0.48) | Not done | IVIg | 41 (33-202) | 60 (48-207) | 14.8 | Not tested |
| 203N | Yes | 0.151 (1.00-2.20) | 0.095 (0.53-1.30) | 0.045 (0.33-0.92) | 0.013 (0.11-0.57) | 0.030 (0.07-0.48) | Not done | 1090 (639-1349) | 113 (70-312) | 31 (56-352) | 47.0 | Not tested |
| 204C | Yes | 0.174 (1.40-3.70) | 0.067 (0.70-2.20) | 0.095 (0.49-1.30) | 0.063 (0.39-1.40) | 0.033 (0.13-0.72) | Not done | IVIg | 98 (33-202) | 231 (48-207) | 6.7 | 78.4 |
| 301N | No | 0.512 (1.40-3.70) | 0.074 (0.70-2.20) | 0.415 (0.49-1.30) | 0.025 (0.39-1.40) | 0.036 (0.13-0.72) | 6.0% (53%-86%) | 1270 (463-1236) | 61 (25-154) | 113 (43-196) | 125.0 | 2.1 |
| 303N | No | 0.113 (1.40-3.70) | 0.070 (0.70-2.20) | 0.042 (0.49-1.30) | 0.024 (0.39-1.40) | 0.023 (0.13-0.72) | 3.1% (53%-86%) | IVIg | 9 (22-159) | 20 (47-200) | 142.8 | Not detected |
| 304C | Yes | 0.737 (1.20-2.60) | 0.254 (0.65-1.50) | 0.473 (0.37-1.10) | 0.209 (0.27-0.86) | 0.154 (0.10-0.48) | 30% (46%-77%) | IVIg | 149 (70-312) | 51 (56-352) | 108.8 | 176.9 |
| 305C | No | 0.317 (1.40-3.70) | 0.194 (0.70-2.20) | 0.090 (0.49-1.30) | 0.022 (0.39-1.40) | 0.014 (0.13-0.72) | 33% (53%-86%) | IVIg | 77 (22-159) | 24 (47-200) | 149.0 | Not detected |
| 306N | Yes | 0.174 (1.40-3.70) | 0.115 (0.70-2.20) | 0.043 (0.49-1.30) | 0.073 (0.39-1.40) | 0.166 (0.13-0.72) | 16% (53%-86%) | IVIg | 13 (22-159) | 32 (47-200) | 80.4 | Not detected |
| PIN . | On ERT* . | Lymphocyte subsets† . | Serum immunoglobulins‡ . | PHA (%LLN)§ . | TREC/μg PBMC DNA¶ . | |||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| CD3 (ref. range) . | CD4 (ref. range) . | CD8 (ref. range) . | CD19 (ref. range) . | CD16 (ref. range) . | %CD45RA/CD4+ (ref. range) . | IgG (ref. range) . | IgA (ref. range) . | IgM (ref. range) . | ||||
| 201C | Yes | 0.371 (1.00-2.20) | 0.181 (0.53-1.30) | 0.192 (0.33-0.92) | 0.002 (0.11-0.57) | 0.003 (0.07-0.48) | Not done | IVIg# | 171 (70-312) | 97 (56-352) | 30.3 | 2.18 |
| 202N | Yes | 0.067 (1.20-2.60) | 0.061 (0.65-1.50) | 0.004 (0.37-1.10) | 0.062 (0.27-0.86) | 0.133 (0.10-0.48) | Not done | IVIg | 41 (33-202) | 60 (48-207) | 14.8 | Not tested |
| 203N | Yes | 0.151 (1.00-2.20) | 0.095 (0.53-1.30) | 0.045 (0.33-0.92) | 0.013 (0.11-0.57) | 0.030 (0.07-0.48) | Not done | 1090 (639-1349) | 113 (70-312) | 31 (56-352) | 47.0 | Not tested |
| 204C | Yes | 0.174 (1.40-3.70) | 0.067 (0.70-2.20) | 0.095 (0.49-1.30) | 0.063 (0.39-1.40) | 0.033 (0.13-0.72) | Not done | IVIg | 98 (33-202) | 231 (48-207) | 6.7 | 78.4 |
| 301N | No | 0.512 (1.40-3.70) | 0.074 (0.70-2.20) | 0.415 (0.49-1.30) | 0.025 (0.39-1.40) | 0.036 (0.13-0.72) | 6.0% (53%-86%) | 1270 (463-1236) | 61 (25-154) | 113 (43-196) | 125.0 | 2.1 |
| 303N | No | 0.113 (1.40-3.70) | 0.070 (0.70-2.20) | 0.042 (0.49-1.30) | 0.024 (0.39-1.40) | 0.023 (0.13-0.72) | 3.1% (53%-86%) | IVIg | 9 (22-159) | 20 (47-200) | 142.8 | Not detected |
| 304C | Yes | 0.737 (1.20-2.60) | 0.254 (0.65-1.50) | 0.473 (0.37-1.10) | 0.209 (0.27-0.86) | 0.154 (0.10-0.48) | 30% (46%-77%) | IVIg | 149 (70-312) | 51 (56-352) | 108.8 | 176.9 |
| 305C | No | 0.317 (1.40-3.70) | 0.194 (0.70-2.20) | 0.090 (0.49-1.30) | 0.022 (0.39-1.40) | 0.014 (0.13-0.72) | 33% (53%-86%) | IVIg | 77 (22-159) | 24 (47-200) | 149.0 | Not detected |
| 306N | Yes | 0.174 (1.40-3.70) | 0.115 (0.70-2.20) | 0.043 (0.49-1.30) | 0.073 (0.39-1.40) | 0.166 (0.13-0.72) | 16% (53%-86%) | IVIg | 13 (22-159) | 32 (47-200) | 80.4 | Not detected |
ERT indicates enzyme-replacement therapy; HSCT, hematopoietic stem cell transplantation; IVIg, intravenous immunoglobulin; MUD, matched unrelated donor; n.d., not done; Neg, negative for detectable TREC qPCR product; PEG-ADA, polyethylene glycol-conjugated adenosine deaminase; PHA, phytohemagglutinin PIN, patient identification number; TREC, thymic recombination excision circles; and VCN, vector copy number.
ERT with PEG-ADA at 2 years.
Lymphocyte subsets are presented as number of cells per microliter (cpm) × 10−3. Age-based normal reference ranges for lymphocytes subsets are from Shearer et al.27
Serum immunoglobulin levels are presented as mg/dL. Age-based normal reference ranges for serum immunoglobulin levels are from Joliff et al.28
Lymphocyte proliferative responses to PHA, presented as percentage of 3H-thymidine incorporation (cpm) by subject to lower limit of normal for testing laboratory.
TREC, measured by quantitative PCR as described in supplemental Materials and Methods. Healthy adult reference range > 25 TREC/μg.29
Receiving monthly IVIg replacement therapy.