Table 2.
Risk of any Serious Complications (Grade 3), Life-Threatening Complications (Grade 4), or Death (Grade 5) from Chronic Health Conditions among 2-Year Survivors of BMT for IEM
| Variable | All Survivors | Leukodystrophies | Mucopolysaccharidoses | |||
|---|---|---|---|---|---|---|
| HR (95% CI) | P | HR (95% CI) | P | HR (95% CI) | P | |
| Age at BMT | ||||||
| Per year increase | 1.0 (0.97–1.01) | .4 | 1.0 (0.97–1.03) | .9 | 0.9 (0.8–1.1) | .3 |
| Sex (reference: female) | ||||||
| Male | 0.9 (0.5–1.4) | .5 | 0.7 (0.3–1.3) | .2 | 1.1 (0.5–2.3) | .9 |
| Race (reference: non-Hispanic white) | ||||||
| Other | 0.5 (0.2–1.0) | .05 | 0.2 (0.09–0.5) | .001 | 1.5 (0.3–7.7) | .6 |
| Stem cell source (reference: cord blood) | ||||||
| Bone marrow or PBSCs | 1.6 (0.6–3.7) | .3 | 1.7 (0.4–7.5) | .5 | 1.7 (0.6–5.2) | .3 |
| Type of donor (reference: related) | ||||||
| Unrelated | 2.0 (0.9–4.2) | .08 | 1.7 (0.5–6.5) | .4 | 2.0 (0.7–5.6) | .2 |
| Type of conditioning (reference: myeloablative) | ||||||
| RIC | 2.7 (1.2–6.2) | .02 | 4.2 (1.2–14.9) | .03 | 2.2 (0.5–9.6) | .3 |
| Year of BMT (reference: <2000) | ||||||
| 2000—2009 | 0.9 (0.5–1.9) | .8 | 1.1 (0.2–5.1) | .9 | 1.5 (0.5–4.0) | .5 |
| ≥ 2010 | 0.9 (0.4–2.4) | .8 | 0.8 (0.1–5.7) | .9 | 1.9 (0.4–8.2) | .4 |
| >1 BMT | ||||||
| Yes | 1.2 (0.6–2.4) | .6 | 2.6 (0.7–9.5) | .2 | 0.7 (0.3–1.9) | .5 |
| Chronic GVHD (reference: no GvHD) | ||||||
| Yes GVHD | 1.3 (0.6–2.6) | .5 | 1.9 (0.5–7.0) | .3 | 0.9 (0.3–2.9) | .9 |
| TBI (reference: no TBI) | ||||||
| Yes TBI | 0.8 (0.4–1.6) | .5 | 0.5 (0.2–1.6) | .3 | 2.4 (0.6–8.8) | .2 |
| Cyclosporine (reference: no cyclosporine) | ||||||
| Yes cyclosporine | 0.9 (0.4–2.3) | .8 | 0.2 (0.1–0.7) | .008 | 1.0 (0.2–4.6) | .9 |
| (reference: no MMF) | ||||||
| Yes MMF | 1.3 (0.6–2.8) | .5 | 1.8 (0.5–7.1) | .4 | 0.8 (0.4–2.0) | .7 |
| T cell depletion (reference: no T cell depletion) | ||||||
| Yes T cell depletion | 1.4 (0.5–3.6) | .6 | 1.9 (0.4–9.6) | .4 | 0.5 (0.1–1.9) | .3 |
MMF, mycophenolate mofetil.
Other race includes patients of Hispanic, African-American, Asian, and other mixed race/ethnicity. Category of other IEMs could not be analyzed in these models owing to a low number of events.