Table 2.
Health Care Resource Utilization and Associated Costs
| Resource | No-NTBC Group (n = 21)* | Late-Intervention Group (n = 26) | Early-Intervention Group (n = 41) | p Value† |
|---|---|---|---|---|
| Hospitalization‡ | NT | |||
| No. of admissions, including PICU | 273 | 178 | 40 | |
| No. of days in hospital | 2 489 | 1 377 | 105 | |
| No. of admissions, PICU only | 17 | 15 | 1 | |
| No. of days, PICU only | 177 | 211 | 7 | |
|
| ||||
| Events per person-year | ||||
| Admissions, including PICU | 0.83 | 0.41 | 0.16 | < 0.001 |
| Days in hospital | 7.6 | 3.2 | 0.4 | < 0.001 |
| Admissions, PICU only | 0.05 | 0.04 | 0.004 | < 0.001 |
| Days, PICU only | 0.5 | 0.5 | 0.03 | < 0.001 |
| Allied professional consultations§ | 1.28 | 0.65 | 0.08 | < 0.001 |
|
| ||||
| Cumulative costs¶, $ | ||||
| Hospital admissions, including PICU | 4 371 480 | 2 304 329 | 170 170 | |
| Hospital admissions, PICU only | 576 267 | 348 886 | 19 199 | |
| Allied professional consultations§ | 31 512 | 20 596 | 1 030 | |
| Medical services | 1 079 967 | 701 140 | 192 869 | |
| Pharmaceutical services, excluding NTBC** | 301 410 | 147 437 | 11 996 | |
| NTBC only | — | 16 496 387 | 13 007 067 | |
|
| ||||
| Costs per person-year¶, $ | ||||
| Hospital admissions, including PICU | 12 980 | 5 590 | 673 | < 0.001 |
| Hospital admissions, PICU only | 1 768 | 846 | 76 | < 0.001 |
| Allied professional consultations§ | 96 | 50 | 4 | < 0.001 |
| Medical services | 3 028 | 1 632 | 763 | < 0.001 |
| Pharmaceutical services, excluding NTBC** | 3 674 | 1 144 | 144 | < 0.001 |
| NTBC only | — | 64 895 | 51 493 | < 0.001 |
NTBC = nitisinone, PICU = pediatric intensive care unit, NT = not tested (values not reported per person-year).
The original no-NTBC group had 28 patients, but 7 patients died within their first year, and no data are available for them.
Kruskal–Wallis test for events per person-year, bootstrap test for costs per person-year.
In the 2 groups treated with NTBC, at least one hospital admission was attributable to diagnosis and treatment initiation. Hospital admissions occurring after treatment (liver transplant) were included and are essentially related to post-transplant surveillance and complications.
Allied professional consultations: those performed by health care professionals, other than physicians, during hospital stays and outpatient visits.
Costs reported in 2008 Canadian dollars. Stay on the ward was estimated at $528/day in 1987 and $1 925/day in 2008. Stay in PICU was estimated at $915/day in 1987 and $3 337/day in 2008.
Pharmaceutical services include pharmacist fees and were available only for patients covered by the drug plan of the Régie de l’Assurance maladie du Québec: no-NTBC group (n = 10), late-intervention group (n = 9), and early-intervention group (n = 19); data were available starting in 1997.