Table 1.
Characteristics and quality assessment of studies included
| SL. no | Study title | Author (s) | Year | Study design | Type of treatment | Age | Quality assessmenta |
|---|---|---|---|---|---|---|---|
| Articles on non-Chinese patients | |||||||
| 1 | Can long-term prophylaxis for severe haemophilia be stopped in adulthood? Results from Denmark and the Netherlands [7] | van Dijk | 2005 | Observational | Discontinuation of prophylaxis |
Denmark: 26.2 (23.8–29.1) years Netherlands: 26.5 (23.9–29.5) years |
6 |
| 2 | A comparison between prophylaxis and on demand treatment for severe haemophilia [21] | Khoriaty | 2005 | Observational | Prophylaxis vs on-demand | Mean age: 27.93; median age: 28 years | 4 |
| 3 | A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study) [22] | Gringeri | 2011 | RCT | Prophylaxis vs episodic treatment | 50 (10–84); 48 (14–84) years | 3 |
| 4 | A randomized comparison of two prophylaxis regimens and a paired comparison of on-demand and prophylaxis treatments in hemophilia A management [23] | Valentino | 2012 | RCT | Prophylaxis vs on-demand | 26 years (7–59) years | 3 |
| 5 | Consequences of switching from prophylactic treatment to on-demand treatment in late teens and early adults with severe haemophilia A: the TEEN/TWEN study [24] | Manco-Johnson | 2013 | Prospective | Prophylaxis vs prospective on-demand vs retrospective on-demand | 19.5 (13–31) years | 5 |
| 6 | Controlled, cross-sectional MRI evaluation of joint status in severe haemophilia A patients treated with prophylaxis vs on demand [25] | Oldenburg | 2015 | Cross-sectional | Prophylaxis vs on-demand | 12–16; 17–21; 22–26; 27–35 years | 5 |
| 7 | Prophylaxis vs. on-demand treatment with BAY 81–8973, a full-length plasma protein-free recombinant factor VIII product: results from a randomized trial (LEOPOLD II) [26] | Kavakli | 2015 | RCT | 2 low-dose and 2 high-dose prophylaxis vs 2 on-demand | 12–65 years | 3 |
| 8 | Adherence to prophylaxis and quality of life in children and adolescents with severe haemophilia A [17] | García-Dasí | 2015 | Cross-sectional | Prophylaxis | 6–20 years | 5 |
| 9 | Adherence to clotting factors among persons with hemophilia A or B [27] | Armstrong | 2015 | Retrospective | Prophylactic | Range: < 1 to 61 years | 5 |
| 10 | Benefits of prophylaxis versus on-demand treatment in adolescents and adults with severe haemophilia A: The POTTER study [28] | Tagliaferri | 2015 | Prospective | Prophylaxis vs on-demand | 12–55 (12–25 and 26–55) years (grouping based on age) | 6 |
| 11 | Adherence to treatment regimen and bleeding rates in a prospective cohort of youth and young adults on low-dose daily prophylaxis for severe hemophilia A [29] | Mizrahi | 2016 | Prospective, longitudinal | Low-dose prophylaxis | 15.2–28.4 years | 6 |
| 12 | Objective quantification of adherence to prophylaxis in haemophilia patients aged 12 to 25 years and its potential association with bleeding episodes [30] | Pérez-Robles | 2016 | Retrospective | Prophylaxis | Range: 12–15; mean age: 17.56 years | 4 |
| 13 | Discontinuing early prophylaxis in severe haemophilia leads to deterioration of joint status despite low bleeding rates [31] | Nijdam | 2016 | Observational | Prophylaxis | 15.3 years | 6 |
| 14 | Adherence to prophylaxis and bleeding outcome in haemophilia: a multicentre study [32] | Schrijvers | 2016 | Prospective | Adherence to prophylaxis | Parent-reported age: 8.4 (6.2–10.5) years; patient-reported age: 29.9 (17.1–49.8) years | 4 |
| 15 | Effect of late prophylaxis in hemophilia on joint status: a randomized trial [33] | Manco-Johnson | 2017 | RCT | Prophylaxis vs on-demand | 12–50 years | 2 |
| 16 | Tailored frequency-escalated primary prophylaxis for severe haemophilia A: results of the 16-year Canadian Hemophilia Prophylaxis Study longitudinal cohort [34] | Feldman | 2018 | Longitudinal | Prophylaxis | 1–2.5 years | 5 |
| 17 | Young adult outcomes of childhood prophylaxis for severe hemophilia A: Results of the joint outcome continuation study [35] | Warren | 2020 | Observational, partially retrospective | Prophylaxis | – | 6 |
| 18 | Long-term analysis of the benefit of prophylaxis for adult patients with severe or moderate haemophilia A [36] | Miesbach | 2020 | Prospective, noninterventional, multicenter, binational, long-term | Prophylaxis | – | 6 |
| 19 | Intermediate dose prophylaxis in adults with haemophilia: a clinical audit from a resource limited setting [37] | Sudevan | 2020 | Clinical audit | Prophylaxis vs on-demand | 31.63 ± 6.98 years | 5 |
| 20 | Physical activity improved by adherence to prophylaxis in an Italian population of children, adolescents and adults with severe haemophilia A: the SHAPE study [38] | Zanon | 2020 | Prospective | Prophylaxis | < 12 years; 12–18 years and > 18 years | 6 |
| 21 | Hemophilia prophylaxis adherence and bleeding using a tailored, frequency-escalated approach: the Canadian Hemophilia Primary Prophylaxis Study [39] | Dover | 2020 | Observational | Prophylaxis | 12–30 months | 5 |
| 22 | The perspectives of adolescents and young adults on adherence to prophylaxis in hemophilia: a qualitative study [40] | Hoefnagels | 2020 | Qualitative | Prophylaxis | Median 18 (14–25) years | 6 |
| 23 | Prophylactic vs episodic treatment to prevent bleeds and preserve joint function in Thai children with moderate and severe haemophilia A [41] | Songnuy | 2020 | Prospective cohort | Episodic vs prophylaxis | ≥ 6 months to ≤ 18 years | 5 |
| Articles on Chinese patients | |||||||
| 1 | Low-dose tertiary prophylactic therapy reduces total number of bleeds and improves the ability to perform activities of daily living in adults with severe haemophilia A: a single-centre experience from Beijing [42] | Hua | 2016 | Retrospective | Prophylaxis | 18–60 years (median 31) | 6 |
| 2 | A prospective study of health-related quality of life of boys with severe haemophilia A in China: comparing on-demand to prophylaxis treatment [43] | Wu | 2017 | Prospective | Prophylaxis Vs on-demand | 4–15.9 years | 5 |
| 3 | Long-term efficacy and safety of prophylaxis with recombinant factor VIII in Chinese pediatric patients with hemophilia A: a multi-center, retrospective, non-interventional, phase IV (ReCARE) study [44] | Li | 2017 | Retrospective | Prophylaxis | 7.1 ± 4.23 years | 5 |
| 4 | Describing the quality of life of boys with haemophilia in China: Results of a multicentre study using the CHO-KLAT [45] | Tang | 2017 | Cross-sectional | Both hemophilia A and B; Prophylaxis or on-demand | 4 to 17.9 years; median: 8.4 years | 4 |
| 5 | Efficacy of short-term full-dose prophylaxis in adult Chinese patients with severe hemophilia A [46] | Sun | 2018 | Prospective | Prophylaxis Vs on-demand | 26 (20.5–29.0) years | 6 |
| 6 | Efficacy of Short- term Individualized Prophylaxis Guided by PK and Joint Evaluation in Chinese Adult Patients with Severe Hemophilia A [47] | Sun | 2019 | Prospective | Prophylaxis Vs on-demand | – | |
| 7 | Efficacy and safety of prophylaxis with BAY 81‐8973 in Chinese patients with severe haemophilia A enrolled in the LEOPOLD II trial [48] | Yang | 2019 | RCT | Low-dose Vs high-dose Vs on-demand | 12–65 years | 3 |
| 8 | Long-term joint outcomes of regular low-dose prophylaxis in Chinese children with severe haemophilia A [49] | Wu | 2021 | Retrospective | Prophylaxis | – | 6 |
RCT, Randomized clinical trial
aQuality assessment was done using Jadad scale (0–5) for RCTs and Newcastle Ottawa scale (0–8) for non-RCTs