Table 3.
Summary of previously reported adherence definitions and rates for pediatric cohorts of subjects with either hemophilia A or B
| Reference number | Definition of adherence | Sample size | Reported results | Calculated 95% confidence interval |
|---|---|---|---|---|
| 10 | Percentage of adherent weeks, with adherent week defined as a week where between 80% and 150% of prescribed units are given and <33% is low, 34%‐66% is moderate and 67%‐100% is high adherence | 19 | 26% of patients have high adherence | 6%‐46% |
| 11 | No definition of adherence given | 34 | 58.8% of survey respondents self‐reported that they had “excellent” adherence | 42.3%‐75.3% |
| 16 | Percentage of factor concentrate administered relative to the amount prescribed | 180 | 80%‐87% of factor administered | NA |
| 17 | Ratio of observed to expected factor usage; optimal adherence defined as infusing between 75%‐125% of prescribed factor | 453 | 65.8% of patients with optimal adherence to prophylaxis | 61.5%‐70.2% |
| 18 | Percentage of weeks per year that the patient was adherent to the infusion frequency of the prescribed prophylaxis regimen | 14 | Median (IQR) of 86% (75%‐91%) of adherent weeks per subject | 68%‐104% |
| 19 | Weekly adherence with prescribed infusion frequency | 16 | Median (IQR) of 88.6% (73.2%‐96.9%) of adherent weeks per subject | 73.0%‐104.2% |
| 20 | Adherence defined as missing <15% of prescribed infusions (adherence rate ≥85%) | 73 | 66% of patients were adherent | 55%‐77% |
| 21 | Percentage of time periods (180 d) where supply of product administered (obtained from pharmacy database) was ≥60% of supply ordered | 74 | Mean of 51% (SD, 36%) adherent time periods | 43%‐59% |
| 22 | Percentage of patients who had ≥75% adherence to prescribed regimen, based on IU dispensed/total IU required for regimen (based on pharmacy database) | 52 |
73.1% of patients ≥75% adherence Mean adherence of 85.7% (SD, 23.8%) calculated from pharmacy records (IU dispensed/IU required) |
79.3%‐92.2% |
| 23 | Percentage of prescribed daily dose received for patients on daily low‐dose prophylaxis | 17 | Median, 85% (range, 56%‐98%) | 68%‐102% |
| 24 | Adherence index defined as units administered/units prescribed, then subtracted from 100 to determine the difference (over or under) from perfect adherence | 78 | Mean, −3.1 (SD, 14.4) with a range of −64.4 to 66.7 | −6.3 to 0.1 |
| 25 | Used VERITAS‐PROa questionnaire | 55 | Mean score, 39.6 (SD, 11.7) | NA |
| 26 | Used VERITAS‐PROa questionnaire | 69 | Mean score, 49.6 (SD, 12.9) with a range of 25‐78 | NA |
| 27 | Used VERITAS‐PROa with defined cutoff of ≥51 indicating nonadherence | 78 | 18% of patients had score of ≥51 indicating nonadherence | NA |
| 28 | No definition of adherence given | 22 | 73% of patients had “excellent” adherence to a prophylaxis regimen | 54%‐92% |
IQR, interquartile range; NA, not applicable; SD, standard deviation.
a
Validated Hemophilia Regimen Treatment Adherence Scale—Prophylaxis with a score range of 24‐120, with 24 representing perfect adherence to prophylaxis.34