MV analysis of factors associated with a second treatment by HCT, ERT, or GT for patients who initially received non-MSD HCT
| Variable and categories . | N (N event)* . | HR estimate (95% CI)† . | P . | Overall P‡ . |
|---|---|---|---|---|
| Age at HCT/infection | ||||
| <3.5 m/no infection (baseline) | 87 (9) | 1.00 | .050 | |
| <3.5 m/infected/active | 26 (4) | 1.35 (0.40-4.49) | .628 | |
| <3.5 m/infected/resolved | 17 (3) | 1.66 (0.44-6.32) | .455 | |
| ≥3.5 m/infected/active | 240 (54) | 2.85 (1.40-5.80) | .004 | |
| ≥3.5 m/infected/resolved | 138 (29) | 2.60 (1.22-5.54) | .013 | |
| ≥3.5 m/no infection | 43 (9) | 1.70 (0.66-4.35) | .270 | |
| Infected/unknown | 20 (1) | 0.67 (0.08-5.31) | .702 | |
| Conditioning | ||||
| No/IS (baseline) | 362 (83) | 1.00 | .009 | |
| RIC/MAC | 209 (26) | 0.51 (0.30-0.85) | .009 | |
| Decade | ||||
| 1982-1989 (baseline) | 102 (20) | 1.00 | .006 | |
| 1990-1999 | 192 (29) | 0.88 (0.50-1.58) | .689 | |
| 2000-2012 | 277 (60) | 1.88 (1.08-3.26) | .032 | |
| Donor type | ||||
| MMRD (baseline) | 413 (92) | 1.00 | .004 | |
| MORD | 28 (2) | 0.27 (0.07-1.11) | .069 | |
| URD | 130 (15) | 0.41 (0.21-0.77) | .005 | |
| Sex | ||||
| Female (baseline) | 158 (42) | 1.00 | .010 | |
| Male | 413 (67) | 0.56 (0.36-0.87) | .010 | |
| Genotype | ||||
| IL2RG/JAK3 | 198 (30) | 1.00 | .098 | |
| ADA | 40 (13) | 2.81 (1.38-5.73) | .04 | |
| DCLRE1C | 18 (3) | 0.97 (0.28-3.40) | .962 | |
| IL7Ra, CD3 (any), CD45 | 43 (11) | 1.23 (0.60-2.55) | .571 | |
| Other/unknown/ND | 234 (42) | 1.53 (0.92-2.54) | .097 | |
| RAG | 38 (10) | 1.77 (0.96-3.16) | .163 | |
| Stratum | ||||
| A (baseline) | 503 (92) | 1.00 | .066 | |
| B | 68 (17) | 1.74 (0.96-3.16) | .066 |
| Variable and categories . | N (N event)* . | HR estimate (95% CI)† . | P . | Overall P‡ . |
|---|---|---|---|---|
| Age at HCT/infection | ||||
| <3.5 m/no infection (baseline) | 87 (9) | 1.00 | .050 | |
| <3.5 m/infected/active | 26 (4) | 1.35 (0.40-4.49) | .628 | |
| <3.5 m/infected/resolved | 17 (3) | 1.66 (0.44-6.32) | .455 | |
| ≥3.5 m/infected/active | 240 (54) | 2.85 (1.40-5.80) | .004 | |
| ≥3.5 m/infected/resolved | 138 (29) | 2.60 (1.22-5.54) | .013 | |
| ≥3.5 m/no infection | 43 (9) | 1.70 (0.66-4.35) | .270 | |
| Infected/unknown | 20 (1) | 0.67 (0.08-5.31) | .702 | |
| Conditioning | ||||
| No/IS (baseline) | 362 (83) | 1.00 | .009 | |
| RIC/MAC | 209 (26) | 0.51 (0.30-0.85) | .009 | |
| Decade | ||||
| 1982-1989 (baseline) | 102 (20) | 1.00 | .006 | |
| 1990-1999 | 192 (29) | 0.88 (0.50-1.58) | .689 | |
| 2000-2012 | 277 (60) | 1.88 (1.08-3.26) | .032 | |
| Donor type | ||||
| MMRD (baseline) | 413 (92) | 1.00 | .004 | |
| MORD | 28 (2) | 0.27 (0.07-1.11) | .069 | |
| URD | 130 (15) | 0.41 (0.21-0.77) | .005 | |
| Sex | ||||
| Female (baseline) | 158 (42) | 1.00 | .010 | |
| Male | 413 (67) | 0.56 (0.36-0.87) | .010 | |
| Genotype | ||||
| IL2RG/JAK3 | 198 (30) | 1.00 | .098 | |
| ADA | 40 (13) | 2.81 (1.38-5.73) | .04 | |
| DCLRE1C | 18 (3) | 0.97 (0.28-3.40) | .962 | |
| IL7Ra, CD3 (any), CD45 | 43 (11) | 1.23 (0.60-2.55) | .571 | |
| Other/unknown/ND | 234 (42) | 1.53 (0.92-2.54) | .097 | |
| RAG | 38 (10) | 1.77 (0.96-3.16) | .163 | |
| Stratum | ||||
| A (baseline) | 503 (92) | 1.00 | .066 | |
| B | 68 (17) | 1.74 (0.96-3.16) | .066 |
For each “variable,” the categories are compared with the “baseline” reference group. The P values displayed for the individual categories of a variable help to assess the relative evidence of effect among the individual categories, relative to each other, but cannot be interpreted as common P values. The “HR” characterizes the ratio in instantaneous chance of a second treatment relative to the reference group. Another statistically significant pairwise comparison includes 1990-1999 vs 2000-2012: HR, 0.48 (95% CI, 0.30-0.78), P = .03. The type of T-cell depletion among MMRD had no effect on the need for a second treatment. Indeed, HR for CD34+ selection vs T-cell depletion by soybean lectin is 1.38 (95% CI, 0.78-2.44), P = 0.27 and HR for other methods vs T-cell depletion by soybean lectin is 1.25 (95% CI, 0.62-2.51), P = 0.53.
ND, not done.
“N” is the number of subjects in the category of the variable; the “N event” is defined by a second treatment using HCT, ERT, or GT.
The “HR estimate” is for the event of a second treatment.
“Overall P” provides the overall significance of the variable in the model.