Table 2.
Dose, duration of treatment, and clinical outcomes of studies included
| Study | n | Type of prophylaxis | Dose | Duration of treatment | Clinical outcomes | |
|---|---|---|---|---|---|---|
| Prophylaxis | On-demand | |||||
| Articles on non-Chinese patients | ||||||
| van Dijk et al. [6, 7] |
22 Danish 58 Dutch |
– | – | Long-term |
AJBR: 1.8 (0.0–3.0) Pettersson score: 13.0 (5.0–23.0) |
AJBR: 3.2 (0.9–6.0) Pettersson score: 13.0 (0.8–23.5) |
| Khoriaty [21] | 133 | – | – | – | AJBR: 3.2 ± 6.4 | AJBR: 5.7 ± 9.1 |
| Gringeri [22] | 40 | – | Medium | Long-term |
≤ 3 y: (0.35 events/patient/month); joint bleeds (0.12 events/patient/month) 3 y at the start of prophylaxis: (0.62 and 0.25 events/patient/month) ≤ 3 y: 3 bleeds/year |
– |
| Valentino [23] | 53 | – | Standard | Long-term | AJBR: Standard dose: 1.6 ± 1.2; Pharmacokinetic tailored dose: 1.9 ± 1.1 | – |
| Manco-Johnson et al. [24] | 38 | – | Standard | – |
Joint pain: 0.6 ± 0.9 AJBR: 0 |
AJBR: Discontinued prophylaxis ≤ 12 months before study: 4.8; Discontinued prophylaxis ≥ 13 months before study: 24 |
| Oldenburg [25] | 118 | Primary and secondary | – | – |
Mean index joint score: Secondary prophylaxis: 7.5 (0–10.5) MRI score: Secondary prophylaxis: 16.5 (0–20) Joint bleeds during previous 5 years: All prophylaxis, ≤ 4.1 |
Mean index joint score: 13.8 (0.5–19.5) MRI score: 18 (2–19) Joint bleeds during previous 5 years: ≤ 14.3 |
| Kavakli [26] | 80 | – | Standard | – | AJBR: 4.9 ± 6.8 | AJBR: 57.7 ± 24.6 |
| García-Dasí [17] | 78 | Primary or secondary | – | Long-term | – | – |
| Tagliaferri [28] | 53 | Tertiary | Medium | Long-term |
AJBR: 12–25 y (1.97); 26–55 y (2.46) Pettersson score: 12–25 y: 5.5 (4.9); 26–55 y: 22.2 (18.5) |
AJBR: 12–25 y (16.8);26–55 y (16.71) Pettersson score: 12–25 y: 5.7 (6.7); 26–55 y: 35.0 (17.2) |
| Mizrahi et al. [29] | 25 | – | Low | – | HJHS score: 16 | – |
| Pérez-Robles et al. [30] | 52 | Primary or secondary | Standard | – | – | – |
| Nijdam et al. [31] | 66 | – | Medium | Long-term |
HJHS score: discontinued (23); continued (14) AJBR: discontinued (1.5); continued (1.2) |
– |
| Schrijvers et al. [32] | 241 | – | – | – | AJBR: Children (3.7); adult (3) | – |
| Manco-Johnson et al. [33] | 70 | Tertiary | – | Long-term |
Joint pain: At 3 y, 50% decrease in pain for the previous 4 weeks ([− 17.2 ± 22.9]; − 16.0 [− 75 to 21]) MRI score: LS mean from baseline to 3 years: 0.79 HRQoL: At 3 years, improved by 3.98 points AJBR: 27.3 (14.9–41.1); 28.7 ± 18.8 |
Joint pain: At 3 y, reported no change ([0.0 ± 25.1); − 3.0 (− 52 to 52]) MRI score: LS mean from baseline to 3 years: 0.96 HRQoL: At 3 years, deterioration by 6.00 points AJBR: 0.3 (0–1.2); 1.9 ± 4.1 |
| Feldman et al. [34] | 56 | Primary | Tailored frequency-escalated | Long-term |
AJBR: Index hemarthroses: 0.95 (0.44–1.35; 0.00–13.43) Other hemarthroses: 0.11 (0.00–0.30; 0.00–1.76) |
– |
| Warren et al. [35] | 37 | Primary and tertiary | – | Long-term |
MRI score: Primary prophylaxis, 1.3 (05–2.2) vs tertiary prophylaxis, 2.3 (1.3–5.0) AJBR: Primary prophylaxis, 0.8 (0–3) vs tertiary prophylaxis, 0.4 (0–3); At age 6 years: Primary prophylaxis, 0.7 ± 0.8 vs tertiary prophylaxis, 3.8 ± 2.8 |
– |
| Miesbach et al. [36] | 161 | – | – | Long-term | AJBR: Median: 1.3 (IQR: 3.6) | AJBR: Median 31.4 (IQR: 27.6) |
| Sudevan et al. [37] | 8 | Tertiary | Medium | Short-term | AJBR: 0.63 ± 0.74/month | AJBR: 5.13 ± 2.51/month |
| Zanon et al. [38] | 42 | – | Standard | Long-term | HJHS score: Baseline: 0.1 ± 0.4; post follow-up: 2.3 ± 3.2 | – |
| Dover et al. [39] | 56 | – | Individualized | Long-term |
MRI score: 20.27 ± 12.36 AJBR: 3.71 ± 1.35 |
– |
| Songnuy et al. [41] | 15 | – | Medium | Long-term |
HRQoL: 85.71 ± 8.52 HJHS score: 9.17 |
HRQoL: 72.86 ± 10.87 HJHS score: 12.75 |
| Articles on Chinese patients | ||||||
| Hua et al. [42] | 33 | Tertiary | Low | – | AJBR: 11.8 ± 7.6 | AJBR: 41.5 ± 20.4 |
| Wu et al. [43] | 23 | – | Standard | Short-term | Mean CHO-KLAT score: Child self-reported, 61.9 ± 11.4; Parent proxy reported, 58.2 ± 8.1 | Mean CHO-KLAT score: Child self-reported, 61.4 ± 10.9; Parent proxy reported, 54.4 ± 10.5 |
| Li et al. [44] | 183 | – | – | – | AJBR: 4.91 (8.110); ABR: 8.44 (10.892) | – |
| Tang et al. [45] | 269 | – | – | – | HRQoL: Child self-reported: 58.9 ± 15.6 (range 28.1–96.9); parent proxy-reported: 51.9 ± 14.9 (range 16.7–100) | – |
| Sun et al. [46, 47] | 27 | Individualized and standard | Medium | – | – | – |
| Yang et al. [14, 48] | 80 | – | Standard | Median ABRs: 2.0 (Chinese); 1.0 (Non-Chinese patients) | Median ABRs: 61.3 (Chinese); 58.5 (Non-Chinese patients) | |
| Wu et al. [49] | 21 | – | Low | Long-term |
MRI score: Total: 2–24 HJHS score: 2–27, with 0–10 for 46.7% children and > 10 for 53.3% |
– |
MRI, Magnetic Resonance Imaging; HRQoL, Health Related Quality of Life; HJHS, Hemophilia Joint Health Score; AJBR, Joint Annualized Bleeding Rate; ABR, Annualized Bleeding Rate; IQR, Interquartile range