Immunological assessment
| Initial assessment . | P1 (7 yo) . | P2 (6 mo) . | P3 (11 yo) . | P4 (2 yo) . | P5 (6 yo) . |
|---|---|---|---|---|---|
| FBC | |||||
| Lymphocytes/µl | 780 (1,500–7,000) | 1,080 (3,000–13500) | 530 (1,200–5,200) | 1,600 (6,000–9,000) | 800 (1,500–6,500) |
| Neutrophils/µl | 7,510 (1,500–8,000) | 3,280 (1,000–8,500) | 2,480 (1,800–8,000) | 1,900 (1,500–8,000) | 9,860 (1,500–8,000) |
| Eosinophils/µl | 330 (100–800) | 160 (100–300) | 310 (100–800) | 600 (200–1,000) | 90 (40–400) |
| Platelets/µl | 109k (150k–450k) | 318k (150k–450k) | 142k (150k–450k) | 327k (150k–400k) | 478k (140k–400k) |
| LSS | |||||
| CD3+ cells/µl | 170 (1,352–3,275) | 230 (3,764–6,289) | 430 (930–3,477) | 530 (852–5,333) | 580 (1,200–2,600) |
| CD4+ cells/µl | 10 (776–1,815) | 130 (2,093–4,769) | 290 (576–1,891) | 360 (516–3,448) | 270 (650–1,500) |
| CD8+ cells/µl | 120 (366–1,171) | 100 (720–1,271) | 130 (261–1,189) | 100 (188–1,805) | 300 (370–1,100) |
| CD4+CD45RA+CD27+ cells/µl | 0 (424–1,393) | 10 (1,748–4,201) | 90 (264–1,484) | NA | NA |
| CD8+CD45RA+CD27+ cells/µl | 86 (175–730) | 10 (564–1,040) | 85 (94–986) | NA | NA |
| CD16+CD56+ cells/µl | 280 (106–1,348) | 530 (237–1,146) | 20 (109–1,021) | 590 (138–1,759) | 410 (100–480) |
| CD19+ cells/µl | 260 (157–725) | 280 (896–2,316) | 40 (173–1,194) | 680 (232–1,637) | 240 (270–860) |
| TRECS/106 T cells | Absent | 1,028 (10th %: 15,337, median: 35,982) | 7,797 (10th %: 8,538, median: 22,214) | NA | NA |
| Spectratyping | Abnormal | Abnormal | Abnormal | NA | NA |
| PHA response | Absent | Absent | Impaired | Borderlinea | Impaired |
| IgG (g/liter) | 12.50 (5.4–16.1) | 2.75 (3–9) | 4.64 (6–16) | 15.2 (6–16) | 9.0 (4.9–16.1) |
| IgA (g/liter) | 1.29 (0.7–2.5) | 0.19 (0.15–0.7) | 2.57 (0.8–2.8) | 1.8 (0.8–2.8) | 3.68 (0.50–2.4) |
| IgM (g/liter) | 0.63 (0.5–1.8) | 0.08 (0.4–1.6) | 0.47 (0.5–1.9) | 0.3 (0.5–1.9) | 0.37 (0.5–1.8) |
| Vaccine responsesb | Normal | NA | Normal | Normal | Normal |
| POST-HCT | P1 (14 years post; 24 yo) | P2 (2 years post; 6 yo) | P3 (10 years post; 32 yo) | P5 (6 mo post; 13 yo) | |
| T cell donor chimerism | 41% | 100% | 91% | 100% | |
| Lymphocytes/µl | 1,001 (895–3,684) | 2,110 (1,827–4,564) | 1,102 (895–3,684) | 930 (1,238–4,792) | |
| LSS | |||||
| CD3+ cells/µl | 780 (564–2,935) | 1,640 (1,352–3,275) | 735 (564–2,935) | 690 (930–3,477) | |
| CD4+ cells/µl | 390 (207–1,900) | 970 (776–1,815) | 396 (207–1,900) | 510 (576–1,891) | |
| CD8+ cells/µl | 320 (160–1,103) | 320 (366–1,171) | 339 (160–1,103) | 160 (261–1,189) | |
| TRECS/106 T cells | 4,225 (10th %: NA; median: 4,120) | 9,710 (10th %: 8,538, median: 22,214) | 4,418c (10th %: NA, median: 4,120) | 2,080 (10th %: NA, median: 15,284) | |
| Spectratyping | Normal | Normal | Abnormalc | Improved | |
| PHA response | Normal | Normal | Normal | Normal | |
| Initial assessment . | P1 (7 yo) . | P2 (6 mo) . | P3 (11 yo) . | P4 (2 yo) . | P5 (6 yo) . |
|---|---|---|---|---|---|
| FBC | |||||
| Lymphocytes/µl | 780 (1,500–7,000) | 1,080 (3,000–13500) | 530 (1,200–5,200) | 1,600 (6,000–9,000) | 800 (1,500–6,500) |
| Neutrophils/µl | 7,510 (1,500–8,000) | 3,280 (1,000–8,500) | 2,480 (1,800–8,000) | 1,900 (1,500–8,000) | 9,860 (1,500–8,000) |
| Eosinophils/µl | 330 (100–800) | 160 (100–300) | 310 (100–800) | 600 (200–1,000) | 90 (40–400) |
| Platelets/µl | 109k (150k–450k) | 318k (150k–450k) | 142k (150k–450k) | 327k (150k–400k) | 478k (140k–400k) |
| LSS | |||||
| CD3+ cells/µl | 170 (1,352–3,275) | 230 (3,764–6,289) | 430 (930–3,477) | 530 (852–5,333) | 580 (1,200–2,600) |
| CD4+ cells/µl | 10 (776–1,815) | 130 (2,093–4,769) | 290 (576–1,891) | 360 (516–3,448) | 270 (650–1,500) |
| CD8+ cells/µl | 120 (366–1,171) | 100 (720–1,271) | 130 (261–1,189) | 100 (188–1,805) | 300 (370–1,100) |
| CD4+CD45RA+CD27+ cells/µl | 0 (424–1,393) | 10 (1,748–4,201) | 90 (264–1,484) | NA | NA |
| CD8+CD45RA+CD27+ cells/µl | 86 (175–730) | 10 (564–1,040) | 85 (94–986) | NA | NA |
| CD16+CD56+ cells/µl | 280 (106–1,348) | 530 (237–1,146) | 20 (109–1,021) | 590 (138–1,759) | 410 (100–480) |
| CD19+ cells/µl | 260 (157–725) | 280 (896–2,316) | 40 (173–1,194) | 680 (232–1,637) | 240 (270–860) |
| TRECS/106 T cells | Absent | 1,028 (10th %: 15,337, median: 35,982) | 7,797 (10th %: 8,538, median: 22,214) | NA | NA |
| Spectratyping | Abnormal | Abnormal | Abnormal | NA | NA |
| PHA response | Absent | Absent | Impaired | Borderlinea | Impaired |
| IgG (g/liter) | 12.50 (5.4–16.1) | 2.75 (3–9) | 4.64 (6–16) | 15.2 (6–16) | 9.0 (4.9–16.1) |
| IgA (g/liter) | 1.29 (0.7–2.5) | 0.19 (0.15–0.7) | 2.57 (0.8–2.8) | 1.8 (0.8–2.8) | 3.68 (0.50–2.4) |
| IgM (g/liter) | 0.63 (0.5–1.8) | 0.08 (0.4–1.6) | 0.47 (0.5–1.9) | 0.3 (0.5–1.9) | 0.37 (0.5–1.8) |
| Vaccine responsesb | Normal | NA | Normal | Normal | Normal |
| POST-HCT | P1 (14 years post; 24 yo) | P2 (2 years post; 6 yo) | P3 (10 years post; 32 yo) | P5 (6 mo post; 13 yo) | |
| T cell donor chimerism | 41% | 100% | 91% | 100% | |
| Lymphocytes/µl | 1,001 (895–3,684) | 2,110 (1,827–4,564) | 1,102 (895–3,684) | 930 (1,238–4,792) | |
| LSS | |||||
| CD3+ cells/µl | 780 (564–2,935) | 1,640 (1,352–3,275) | 735 (564–2,935) | 690 (930–3,477) | |
| CD4+ cells/µl | 390 (207–1,900) | 970 (776–1,815) | 396 (207–1,900) | 510 (576–1,891) | |
| CD8+ cells/µl | 320 (160–1,103) | 320 (366–1,171) | 339 (160–1,103) | 160 (261–1,189) | |
| TRECS/106 T cells | 4,225 (10th %: NA; median: 4,120) | 9,710 (10th %: 8,538, median: 22,214) | 4,418c (10th %: NA, median: 4,120) | 2,080 (10th %: NA, median: 15,284) | |
| Spectratyping | Normal | Normal | Abnormalc | Improved | |
| PHA response | Normal | Normal | Normal | Normal | |
Laboratory age-matched reference ranges given in parentheses. Results that fall outside of their respective reference ranges are marked in bold. 10th %, 10th centile; FBC, full blood count; k, thousand; LSS; lymphocyte subsets; mo, months old; NA, not available; PHA, phytohemagglutinin; TRECs; TCR excision circles; yo, years old.
P4 proliferative response of 12,972 counts per minute (cpm) versus travel control 28,147 cpm (laboratory normal cut off at 12,500 cpm).
Assessed prior to the commencement of immunoglobulin replacement therapy.
Performed at 7 years post-HCT.