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Table 2 Dose, duration of treatment, and clinical outcomes of studies included

From: Factor VIII replacement prophylaxis in patients with hemophilia A transitioning to adults: a systematic literature review

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%

–

  1. 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