This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 3.0 License.
Srivastava A, Brewer AK, Mauser-Bunschoten EP, et al; Treatment Guidelines Working Group on Behalf of The World Federation of Hemophilia. Guidelines for the management of hemophilia. Haemophilia 2013; 19: e1–47. doi: 10.1111/j.1365-2516.2012.02909.x.SrivastavaABrewerAKMauser-BunschotenEPTreatment Guidelines Working Group on Behalf of The World Federation of Hemophilia. Guidelines for the management of hemophiliaHaemophilia201319e14710.1111/j.1365-2516.2012.02909.x22776238Open DOISearch in Google Scholar
Ling G, Nathwani AC, Tuddenham EGD. Recent advances in developing specific therapies for haemophilia. Br J Haematol 2018; 181: 161–172. doi: 10.1111/bjh.15084.LingGNathwaniACTuddenhamEGDRecent advances in developing specific therapies for haemophiliaBr J Haematol201818116117210.1111/bjh.1508429359795Open DOISearch in Google Scholar
Kruse-Jarres R, Oldenburg J, Santagostino E, et al. Bleeding and safety outcomes in persons with haemophilia A without inhibitors: results from a prospective non-interventional study in a real-world setting. Haemophilia 2019; 25(2): 213–220. doi: 10.1111/hae.13655.Kruse-JarresROldenburgJSantagostinoEBleeding and safety outcomes in persons with haemophilia A without inhibitors: results from a prospective non-interventional study in a real-world settingHaemophilia201925221322010.1111/hae.1365530724422Open DOISearch in Google Scholar
Kalnins W, Schelle G, Jost K, et al. Pain therapy in haemophilia in Germany. Patient survey (BESTH study). Hamostaseologie 2015; 35(2): 167–73. doi: 10.5482/HAMO-14-03-0021.KalninsWSchelleGJostKPain therapy in haemophilia in Germany. Patient survey (BESTH study)Hamostaseologie20153521677310.5482/HAMO-14-03-002125413275Open DOISearch in Google Scholar
Forsyth AL, Witkop M, Lambing A, et al. Associations of quality of life, pain, and self-reported arthritis with age, employment, bleed rate, and utilization of hemophilia treatment center and health care provider services: results in adults with hemophilia in the HERO study. Patient Prefer Adherence 2015; 9: 1549–60. doi: 10.2147/PPA.S87659.ForsythALWitkopMLambingAAssociations of quality of life, pain, and self-reported arthritis with age, employment, bleed rate, and utilization of hemophilia treatment center and health care provider services: results in adults with hemophilia in the HERO studyPatient Prefer Adherence2015915496010.2147/PPA.S87659463141926604708Open DOISearch in Google Scholar
Berntorp E, Dolan G, Hay C, et al. European retrospective study of real-life haemophilia treatment. Haemophilia 2017; 23: 105–114. doi: 10.1111/hae.13111.BerntorpEDolanGHayCEuropean retrospective study of real-life haemophilia treatmentHaemophilia20172310511410.1111/hae.1311127761962Open DOISearch in Google Scholar
Collins P, Chalmers E, Chowdary P, et al. The use of enhanced half-life coagulation factor concentrates in routine clinical practice: guidance from UKHCDO. Haemophilia 2016; 22(4): 487–98. doi: 10.1111/hae.13013.CollinsPChalmersEChowdaryPThe use of enhanced half-life coagulation factor concentrates in routine clinical practice: guidance from UKHCDOHaemophilia20162244879810.1111/hae.1301327311929Open DOISearch in Google Scholar
den Uijl IE, Fischer K, Van Der Bom JG, et al. Analysis of low frequency bleeding data: the association of joint bleeds according to baseline FVIII activity levels. Haemophilia 2011; 17(1): 41–4. doi: 10.1111/j.1365-2516.2010.02383.x.den UijlIEFischerKVan Der BomJGAnalysis of low frequency bleeding data: the association of joint bleeds according to baseline FVIII activity levelsHaemophilia201117141410.1111/j.1365-2516.2010.02383.x20825504Open DOISearch in Google Scholar
Furlan R, Krishnan S, Vietri J. Patient and parent preferences for characteristics of prophylactic treatment in hemophilia. Patient Prefer Adherence 2015; 9: 1687–94. doi: 10.2147/PPA.S92520.FurlanRKrishnanSVietriJPatient and parent preferences for characteristics of prophylactic treatment in hemophiliaPatient Prefer Adherence2015916879410.2147/PPA.S92520466454826648701Open DOISearch in Google Scholar
Steen Carlsson K, Andersson E, Berntorp E. Preference-based valuation of treatment attributes in haemophilia A using web survey. Haemophilia 2017; 23(6): 894–903. doi: 10.1111/hae.13322.Steen CarlssonKAnderssonEBerntorpEPreference-based valuation of treatment attributes in haemophilia A using web surveyHaemophilia201723689490310.1111/hae.1332228851125Open DOISearch in Google Scholar
von Mackensen S, Kalnins W, Krucker J, et al. Haemophilia patients’ unmet needs and their expectations of the new extended half-life factor concentrates. Haemophilia 2017; 23(4): 566–574. doi: 10.1111/hae.13221.von MackensenSKalninsWKruckerJHaemophilia patients’ unmet needs and their expectations of the new extended half-life factor concentratesHaemophilia201723456657410.1111/hae.13221Open DOISearch in Google Scholar
Witkop M, Guelcher C, Forsyth A, et al. Treatment outcomes, quality of life, and impact of hemophilia on young adults (aged 18–30 years) with hemophilia. Am J Hematol 2015; 90 Suppl 2: S3–10. doi: 10.1002/ajh.24220.WitkopMGuelcherCForsythATreatment outcomes, quality of life, and impact of hemophilia on young adults (aged 18–30 years) with hemophiliaAm J Hematol201590Suppl 2S31010.1002/ajh.24220Open DOISearch in Google Scholar
Soucie JM, Grosse SD, Siddiqi AE, et al; Hemophilia Treatment Centers Network. The effects of joint disease, inhibitors and other complications on health-related quality of life among males with severe haemophilia A in the United States. Haemophilia 2017; 23(4): e287–e293. doi: 10.1111/hae.13275.SoucieJMGrosseSDSiddiqiAEHemophilia Treatment Centers NetworkThe effects of joint disease, inhibitors and other complications on health-related quality of life among males with severe haemophilia A in the United StatesHaemophilia2017234e287e29310.1111/hae.13275Open DOISearch in Google Scholar
O’Hara J, Hughes D, Camp C, et al. The cost of severe haemophilia in Europe: the CHESS study. Orphanet J Rare Dis 2017; 12: 106. doi: 10.1186/s13023-017-0660-y.O’HaraJHughesDCampCThe cost of severe haemophilia in Europe: the CHESS studyOrphanet J Rare Dis20171210610.1186/s13023-017-0660-yOpen DOISearch in Google Scholar
Camp C, O’Hara J, Hughes D, et al. The relationship between bleeding and EQ-5D in severe haemophilia. Poster P-T-62. WFH World Congress, 24–28 July 2016, Orlando, USA. Available from https://www.postersessiononline.eu/173580348_eu/congresos/WFH2016/aula/-PP-T_62_WFH2016.pdf (accessed 9 July 2020).CampCO’HaraJHughesDThe relationship between bleeding and EQ-5D in severe haemophiliaPoster P-T-62. WFH World Congress24–28 July 2016Orlando, USAAvailable from https://www.postersessiononline.eu/173580348_eu/congresos/WFH2016/aula/-PP-T_62_WFH2016.pdf (accessed 9 July 2020).Search in Google Scholar
Fischer K. Low-dose prophylaxis for severe haemophilia: a little goes a long way. Haemophilia 2016; 22(3): 331–3. doi: 10.1111/hae.12853.FischerKLow-dose prophylaxis for severe haemophilia: a little goes a long wayHaemophilia2016223331310.1111/hae.12853Open DOISearch in Google Scholar
Mulder K, Llinás A. The target joint. Haemophilia 2004;10 Suppl 4:152–6. doi: 10.1111/j.1365-2516.2004.00976.x.MulderKLlinásAThe target jointHaemophilia200410Suppl 4152610.1111/j.1365-2516.2004.00976.xOpen DOISearch in Google Scholar
The EuroQol Group. EuroQol – a new facility for the measurement of health-related quality of life. Health Policy 1990; 16: 199–208. doi: 10.1016/0168-8510(90)90421-9.The EuroQol GroupEuroQol – a new facility for the measurement of health-related quality of lifeHealth Policy19901619920810.1016/0168-8510(90)90421-9Open DOISearch in Google Scholar
Brooks R. EuroQol: the current state of play. Health Policy 1996; 37: 53–72. doi: 10.1016/0168-8510(96)00822-6.BrooksREuroQol: the current state of playHealth Policy199637537210.1016/0168-8510(96)00822-6Open DOISearch in Google Scholar
Hauck JW, Donner A. Wald's test as applied to hypotheses in logit analysis. J Am Stat Assoc 1977; 72: 851–53. doi: 10.2307/2286473.HauckJWDonnerAWald's test as applied to hypotheses in logit analysisJ Am Stat Assoc1977728515310.2307/2286473Open DOISearch in Google Scholar
Nugent D, O’Mahony B, Dolan G; International Haemophilia Access Strategy Council. Value of prophylaxis vs on-demand treatment: application of a value framework in hemophilia. Haemophilia 2018; 24(5): 755–765. doi: 10.1111/hae.13589.NugentDO’MahonyBDolanGInternational Haemophilia Access Strategy CouncilValue of prophylaxis vs on-demand treatment: application of a value framework in hemophiliaHaemophilia201824575576510.1111/hae.1358930088692Open DOISearch in Google Scholar
Chowdary P, Carcao M, Holme PA, et al. Fixed doses of N8-GP prophylaxis maintain moderate-to-mild factor VIII levels in the majority of patients with severe hemophilia A. Res Pract Thromb Haemost 2019; 3: 542–554. doi: 10.1002/rth2.12220.ChowdaryPCarcaoMHolmePAFixed doses of N8-GP prophylaxis maintain moderate-to-mild factor VIII levels in the majority of patients with severe hemophilia ARes Pract Thromb Haemost2019354255410.1002/rth2.12220661147831294338Open DOISearch in Google Scholar
Fischer K, van der Bom JG, Mauser-Bunschoten EP, et al. Changes in treatment strategies for severe haemophilia over the last 3 decades: effects on clotting factor consumption and arthropathy. Haemophilia 2001; 7(5): 446–52. doi: 10.1046/j.1365-2516.2001.00545.x.FischerKvan der BomJGMauser-BunschotenEPChanges in treatment strategies for severe haemophilia over the last 3 decades: effects on clotting factor consumption and arthropathyHaemophilia2001754465210.1046/j.1365-2516.2001.00545.x11554930Open DOISearch in Google Scholar
Lambing A, Nichols CD, Munn JE, et al. Patient, caregiver, and provider perceptions of pain and pain management in adolescents and young adults with bleeding disorders. Haemophilia 2017; 23(6): 852–860. doi: 10.1111/hae.13293.LambingANicholsCDMunnJEPatient, caregiver, and provider perceptions of pain and pain management in adolescents and young adults with bleeding disordersHaemophilia201723685286010.1111/hae.1329328806860Open DOISearch in Google Scholar
Witkop M, Lambing A. Knowledge and attitudes survey in bleeding disorders providers regarding pain. Haemophilia 2015; 21(6): e465–71. doi: 10.1111/hae.12749.WitkopMLambingAKnowledge and attitudes survey in bleeding disorders providers regarding painHaemophilia2015216e4657110.1111/hae.1274926496002Open DOISearch in Google Scholar
Tagliaferri A, Franchini M, Rivolta GF, et al. Pain assessment and management in haemophilia: A survey among Italian patients and specialist physicians. Haemophilia 2018; 24(5): 766–773. doi: 10.1111/hae.13600.TagliaferriAFranchiniMRivoltaGFPain assessment and management in haemophilia: A survey among Italian patients and specialist physiciansHaemophilia201824576677310.1111/hae.1360030112811Open DOISearch in Google Scholar
Forsyth AL, Gregory M, Nugent D, et al. Haemophilia Experiences, Results and Opportunities (HERO) Study: survey methodology and population demographics. Haemophilia 2014; 20(1): 44–51. doi: 10.1111/hae.12239.ForsythALGregoryMNugentDHaemophilia Experiences, Results and Opportunities (HERO) Study: survey methodology and population demographicsHaemophilia2014201445110.1111/hae.1223923902228Open DOISearch in Google Scholar
Pérez-Robles T, Romero-Garrido JA, Rodriguez-Merchan EC, et al. Objective quantification of adherence to prophylaxis in haemophilia patients aged 12 to 25 years and its potential association with bleeding episodes. Thromb Res 2016; 143: 22–7. doi: 10.1016/j.thromres.2016.04.015.Pérez-RoblesTRomero-GarridoJARodriguez-MerchanECObjective quantification of adherence to prophylaxis in haemophilia patients aged 12 to 25 years and its potential association with bleeding episodesThromb Res201614322710.1016/j.thromres.2016.04.01527172834Open DOISearch in Google Scholar
Guedes VG, Corrente JE, Farrugia A, et al. Comparing objective and self-reported measures of adherence in haemophilia. Haemophilia 2019; 25(5): 821–830. doi: 10.1111/hae.13811.GuedesVGCorrenteJEFarrugiaAComparing objective and self-reported measures of adherence in haemophiliaHaemophilia201925582183010.1111/hae.1381131322311Open DOISearch in Google Scholar
Duncan N, Kronenberger W, Roberson C, et al. VERITAS-Pro: a new measure of adherence to prophylactic regimens in haemophilia. Haemophilia 2010; 16(2): 247–55. doi: 10.1111/j.1365-2516.2009.02129.x.DuncanNKronenbergerWRobersonCVERITAS-Pro: a new measure of adherence to prophylactic regimens in haemophiliaHaemophilia20101622475510.1111/j.1365-2516.2009.02129.x19925631Open DOISearch in Google Scholar
, 