Aledort 1994 |
Prospective observational study. |
Ali 2018 |
Includes participants with inhibitors. |
Andreeva 2015 |
Unclear if participants were previously treated (conference abstract only). |
Antunes 2013 |
Includes participants with inhibitors. |
ASPIRE 2020 |
Prospective observational study. |
Astermark 1999 |
Retrospective observational study. |
Astermark 2019 |
Intervention includes the anti‐Tissue Factor Pathway Inhibitor (TFPI),concizumab, which is not a clotting factor therapy. This could be eligible for the subsequent review on non‐clotting factor therapies. |
Bertolet 2020 |
Intervention includes emicizumab, which is not a clotting factor therapy. |
Booth 2017 |
Prospective observational study |
Brackmann 1992 |
Retrospective observational study. |
Carlsson 1997 |
Includes children previously on prophylaxis. |
Chakraborty 2018 |
Includes children on primary prophylaxis. |
Chowdary 2013 |
Intervention includes the anti‐Tissue Factor Pathway Inhibitor (TFPI), concizumab, which is not a clotting factor therapy. |
Chowdary 2017 |
Intervention is an investigational RNA interference (RNAi) therapeutic, not a clotting factor therapy. |
Chozie 2018 |
Includes children on primary prophylaxis. |
Chuansumrit 1995 |
Retrospective observational study. |
Collins 2010 |
Prospective observational cross‐over study. |
Collins 2014 |
Includes children on primary prophylaxis. |
Courter 2001 |
Prospective observational study. |
Curry 2019 |
Includes people on primary prophylaxis. |
Davydkin 2015 |
Unclear if participants were children or previously treated (conference abstract only). |
Dzinaj 1996 |
Prospective observational study. |
Eichler 2018 |
Intervention includes concizumab, which is not a clotting factor therapy. |
ENJOIH 2010 |
Includes participants with inhibitors. |
Escuriola 2019 |
Pharmacokinetics evaluated. |
ESPRIT 2011 |
Pediatric population on primary prophylaxis. |
Feldman 2006 |
Prospective observational single‐arm dose‐escalation study. |
Fernandez‐Bello 2017 |
Pharmacokinetics evaluated in participants with inhibitors. |
Fischer 2005 |
Retrospective observational study. |
HAVEN 1 2017 |
Includes participants with inhibitors. |
Hazendonk 2015 |
Pharmacokinetic parameters evaluated. |
INHIBIT 2014 |
Children on primary prophylaxis. |
Kavakli 1997 |
Prospective observational study. |
Khayat 2016 |
Children on primary prophylaxis. |
Kids B‐LONG 2017 |
Prospective observational study in children. |
Konkle 2016 |
Retrospective observational study. |
Kreuz 1998 |
Prospective observational study. |
LEOPOLD I 2015 |
Participants on primary prophylaxis |
Liesner 1996 |
Retrospective observational study. |
Lofqvist 1997 |
Retrospective observational study. |
Ma 2015 |
Retrospective observational study. |
Manco‐Johnson 1994 |
Prospective observational study. |
Manco‐Johnson 2007 |
Children on primary prophylaxis. |
NCT03315455 |
Intervention includes emicizumab, which is not a clotting factor therapy. |
NCT04303559 |
Intervention includes emicizumab, which is not a clotting factor therapy. |
Nemes 2007 |
Prospective observational single‐arm study. |
Nilsson 1970 |
Retrospective observational study with historical control. |
Nilsson 1976 |
Prospective observational study with historical control. |
Nilsson 1992 |
Retrospective observational study. |
PERSEPT 1 2017 |
Includes participants with inhibitors. |
Petrini 1991 |
Retrospective observational study. |
Pettersson 1981 |
Retrospective observational study with historical control. |
PRO‐FEIBA 2010 |
Includes participants with inhibitors. |
PROTECT VIII 2017 |
Includes participants on primary prophylaxis. |
Ragni 2017 |
Pilot feasibility study with cross‐over design. |
Ramsay 1973 |
Prospective observational study. |
Royal 2002 |
Retrospective observational study with parallel groups. |
Santagostino 2016 |
Prospective observational single‐arm study. |
Schimpf 1977 |
Prospective observational cross‐over study. |
Schobess 2008 |
Prospective observational study. |
Shah 2019 |
Pharmacokinetic parameters evaluated. |
Shapiro 2018 |
Intervention includes concizumab, which is not a clotting factor therapy. |
Smith 1996 |
Retrospective observational switch study. |
Smith 2018 |
Includes children on primary prophylaxis |
Solms 2020 |
Pharmacokinetic parameters evaluated. |
Song 2012 |
Ad‐hoc analysis of a randomized controlled trial. Protocol or full study with results not found on trial registries. Also, the comparison focused on the degree of availability of factor concentrates per country i.e. low versus high IU/capita countries. |
Szucs 1996 |
Prospective observational study. |
Tagliaferri 2008 |
Retrospective observational switch study. |
Van den Berg 2001 |
Retrospective observational single‐arm study. |
Verma 2016 |
Includes children on primary prophylaxis |
Windyga 2014 |
No randomization in the comparison arm assessing outcome of interest (hemostatic efficacy). |
Wu 2011 |
Prospective observation with historical control. |
Young 2015 |
Prospective observational study in children. |
Young 2017 |
Prospective dose and dose‐interval comparison study (conference abstract only). |