Real-world clinical and psychosocial outcomes among people with mild or moderate haemophilia A treated on-demand in the Italian CHESS II cohort: a real-world data analysis
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Berntorp E, Dolan G, Hay C, et al. European retrospective study of real-life haemophilia treatment. Haemophilia 2017; 23(1): 105-14. doi: 10.1111/hae.13111.BerntorpEDolanGHayC.European retrospective study of real-life haemophilia treatment.2017;23(1):105-14. doi:10.1111/hae.13111.Open DOISearch in Google Scholar
Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia 2020; 26(Suppl 6): 1-158. doi: 10.1111/hae.14046.SrivastavaASantagostinoEDougallA.WFH Guidelines for the Management of Hemophilia, 3rd edition.2020;26(Suppl 6):1-158. doi:10.1111/hae.14046.Open DOISearch in Google Scholar
Blanchette VS, Key NS, Ljung LR, Manco-Johnson MJ, van den Berg HM, Srivastava A. Definitions in hemophilia: Communication from the SSC of the ISTH. J Thromb Haemost 2014; 12(11): 1935-9. doi: 10.1111/jth.12672.BlanchetteVSKeyNSLjungLRManco-JohnsonMJvan den BergHMSrivastavaA.Definitions in hemophilia: Communication from the SSC of the ISTH.2014;12(11):1935-9. doi:10.1111/jth.12672.Open DOISearch in Google Scholar
Iorio A, Stonebraker JS, Chambost H, et al. Establishing the prevalence and prevalence at birth of hemophilia in males: a meta-analytic approach using national registries. Ann Intern Med 2019; 171(8): 540-6. doi: 10.7326/M19-1208.IorioAStonebrakerJSChambostH.Establishing the prevalence and prevalence at birth of hemophilia in males: a meta-analytic approach using national registries.2019;171(8):540-6. doi:10.7326/M19-1208.Open DOISearch in Google Scholar
Mingot-Castellano ME, Parra R, Núñez R, Martorell M. Improvement in clinical outcomes and replacement factor VIII use in patients with haemophilia A after factor VIII pharmacokinetic-guided prophylaxis based on Bayesian models with myPKFiT®. Haemophilia 2018; 24(5): e338-43. doi: 10.1111/hae.13540.Mingot-CastellanoMEParraRNúñezRMartorellM.Improvement in clinical outcomes and replacement factor VIII use in patients with haemophilia A after factor VIII pharmacokinetic-guided prophylaxis based on Bayesian models with myPKFiT®.2018;24(5): e338-43. doi:10.1111/hae.13540.Open DOISearch in Google Scholar
O’Hara J, Walsh S, Camp C, et al. The impact of severe haemophilia and the presence of target joints on health-related quality-of-life. Health Qual Life Outcomes 2018; 16(1): 84. doi: 10.1186/s12955-018-0908-9.O’HaraJWalshSCampC.The impact of severe haemophilia and the presence of target joints on health-related quality-of-life.2018;16(1):84. doi:10.1186/s12955-018-0908-9.Open DOISearch in Google Scholar
O’Hara J, Walsh S, Camp C, et al. The relationship between target joints and direct resource use in severe haemophilia. Health Econ Rev 2018; 8(1): 1-7. doi: 10.1186/s13561-018-0185-7O’HaraJWalshSCampC.The relationship between target joints and direct resource use in severe haemophilia.2018;8(1):1-7. doi:10.1186/s13561-018-0185-7Open DOISearch in Google Scholar
Witkop M, Neff A, Buckner TW, et al. Self-reported prevalence, description and management of pain in adults with haemophilia: methods, demographics and results from the Pain, Functional Impairment, and Quality of life (P-FiQ) study. Haemophilia 2017; 23(4): 556-65. doi: 10.1111/hae.13214.WitkopMNeffABucknerTW.Self-reported prevalence, description and management of pain in adults with haemophilia: methods, demographics and results from the Pain, Functional Impairment, and Quality of life (P-FiQ) study.2017;23(4):556-65. doi:10.1111/hae.13214.Open DOISearch in Google Scholar
Ay C, Perschy L, Rejtö J, Kaider A, Pabinger I. Treatment patterns and bleeding outcomes in persons with severe hemophilia A and B in a real-world setting. Ann Hematol 2020; 99(12): 2763-2771. doi: 10.1007/s00277-020-04250-9.AyCPerschyLRejtöJKaiderAPabingerI.Treatment patterns and bleeding outcomes in persons with severe hemophilia A and B in a real-world setting.2020;99(12):2763-2771. doi:10.1007/s00277-020-04250-9.Open DOISearch in Google Scholar
Khair K, Nissen F, Silkey M, et al. Effect of moderate and severe hemophilia a on daily life in children and their caregivers: A CHESS paediatrics study analysis. Blood 2020; 136: 43-5. doi: 10.1182/blood-2020-134658.KhairKNissenFSilkeyM.Effect of moderate and severe hemophilia a on daily life in children and their caregivers: A CHESS paediatrics study analysis.2020;136:43-5. doi:10.1182/blood-2020-134658.Open DOISearch in Google Scholar
O’Hara J, Hughes D, Camp C, Burke T, Carroll L, Diego DAG. The cost of severe haemophilia in Europe: the CHESS study. Orphanet J Rare Dis 2017; 12(1): 106. doi: 10.1186/s13023-017-0660-y.O’HaraJHughesDCampCBurkeTCarrollLDiegoDAG.The cost of severe haemophilia in Europe: the CHESS study.2017;12(1):106. doi:10.1186/s13023-017-0660-y.Open DOISearch in Google Scholar
Warren BB, Thornhill D, Stein J, et al. Young adult outcomes of childhood prophylaxis for severe hemophilia A: Results of the joint outcome continuation study. Blood Adv 2020; 4(11): 2451-9. doi: 10.1182/bloodadvances.2019001311.WarrenBBThornhillDSteinJ.Young adult outcomes of childhood prophylaxis for severe hemophilia A: Results of the joint outcome continuation study.2020;4(11):2451-9. doi:10.1182/bloodadvances.2019001311.Open DOISearch in Google Scholar
Chai-Adisaksopha C, Noone D, Curtis R, et al. Non-severe haemophilia: Is it benign? – Insights from the PROBE study. Haemophilia 2021; 27 Suppl 1: 17-24. doi: 10.1111/hae.14105.Chai-AdisaksophaCNooneDCurtisR.Non-severe haemophilia: Is it benign? – Insights from the PROBE study.2021;27Suppl 1:17-24. doi:10.1111/hae.14105.Open DOISearch in Google Scholar
Benson G, Auerswald G, Dolan G, et al. Diagnosis and care of patients with mild haemophilia: practical recommendations for clinical management. Blood Transfus 2018; 16(6): 535-44. doi: 10.2450/2017.0150-17.BensonGAuerswaldGDolanG.Diagnosis and care of patients with mild haemophilia: practical recommendations for clinical management.2018;16(6):535-44. doi:10.2450/2017.0150-17.Open DOISearch in Google Scholar
Peyvandi F, Tavakkoli F, Frame D, et al. Burden of mild haemophilia A: Systematic literature review. Haemophilia 2019; 25(5): 755-63. doi: 10.1111/hae.13777.PeyvandiFTavakkoliFFrameD.Burden of mild haemophilia A: Systematic literature review.2019;25(5):755-63. doi:10.1111/hae.13777.Open DOISearch in Google Scholar
Castaman G, Peyvandi F, De Cristofaro R, Pollio B, Di Minno DMN. Mild and moderate hemophilia A: neglected conditions, still with unmet needs. J Clin Med 2023; 12(4): 1368. doi: 10.3390/jcm12041368.CastamanGPeyvandiFDe CristofaroRPollioBDi MinnoDMN.Mild and moderate hemophilia A: neglected conditions, still with unmet needs.2023;12(4):1368. doi:10.3390/jcm12041368.Open DOISearch in Google Scholar
Witkop M, Wang M, Hernandez G, Recht M, Baumann K, Cooper DL. Impact of haemophilia on patients with mild-to-moderate disease: Results from the P-FiQ and B-HERO-S studies. Haemophilia 2021; 27(S1): 8-16. doi: 10.1111/hae.14251.WitkopMWangMHernandezGRechtMBaumannKCooperDL.Impact of haemophilia on patients with mild-to-moderate disease: Results from the P-FiQ and B-HERO-S studies.2021;27(S1):8-16. doi:10.1111/hae.14251.Open DOISearch in Google Scholar
Burke T, Ferri Grazzi E, Shaikh A, Hawes C, Camp C, O’Hara J. Exploring the relationship between condition severity and health-related quality of life in haemophilia A. Abstract PB0804. ISTH 2020 Congress. Available from https://abstracts.isth.org/abstract/exploring-the-relationship-between-condition-severity-and-health-related-quality-of-life-in-haemophilia-a/.BurkeTFerri GrazziEShaikhAHawesCCampCO’HaraJ..ISTH 2020 Congress. Available from https://abstracts.isth.org/abstract/exploring-the-relationship-between-condition-severity-and-health-related-quality-of-life-in-haemophilia-a/.Search in Google Scholar
Rodriguez-Santana I, DasMahapatra P, Burke T, et al. Differential humanistic and economic burden of mild, moderate and severe haemophilia in european adults: a regression analysis of the CHESS II study. Orphanet J Rare Dis 2022; 17: 148. doi: 10.1186/s13023-022-02300-1.Rodriguez-SantanaIDasMahapatraPBurkeT.Differential humanistic and economic burden of mild, moderate and severe haemophilia in european adults: a regression analysis of the CHESS II study.2022;17:148. doi:10.1186/s13023-022-02300-1.Open DOISearch in Google Scholar
Arcieri R, Molinari AC, Farace S, et al. Uncovered needs in the management of inherited bleeding disorders in Italy. Blood Transfus 2014; 12 (Suppl 3): s563-566. doi: 10.2450/2014.0036-14s.ArcieriRMolinariACFaraceS.Uncovered needs in the management of inherited bleeding disorders in Italy.2014;12(Suppl 3): s563-566. doi:10.2450/2014.0036-14s.Open DOISearch in Google Scholar
Calizzani G, Candura F, Menichini I, et al. The Italian institutional accreditation model for Haemophilia Centres. Blood Transfus 2014; 12 (Suppl 3): s510-4. doi: 10.2450/2014.0058-14s.CalizzaniGCanduraFMenichiniI.The Italian institutional accreditation model for Haemophilia Centres.2014;12(Suppl 3): s510-4. doi:10.2450/2014.0058-14s.Open DOISearch in Google Scholar
Abbonizio F, Biffoni M, Arcieri R, Associazione Italiana Centri Emofilia, Giampaolo A. Rapporto ISTISAN 22/38 – Registro Nazionale Coagulopatie Congenite. Rapporto 2020. Francesca Abbonizio, Mauro Biffoni, Romano, Associazione Italiana Centri Emofilia (AICE), Adele Giampaolo [Internet]. Rome: Istittuto Superiore di Sanità; 2022. (ISTISAN). Report No.: 22/38. Available from https://www.iss.it/-/rappporto-istisan-22/38-registro-nazionale-coagulopatie-congenite.-rapporto-2020.-francesca-abbonizio-mauro-biffoni-romano-arcieri-associazione-italiana-centri-emofilia-aice-adele-giampaolo (accessed February 2024).AbbonizioFBiffoniMArcieriRAssociazione Italiana Centri EmofiliaGiampaoloA.Rapporto ISTISAN 22/38 – Registro Nazionale Coagulopatie Congenite. Rapporto 2020..Rome:Istittuto Superiore di Sanità;2022. (ISTISAN). Report No.: 22/38. Available from https://www.iss.it/-/rappporto-istisan-22/38-registro-nazionale-coagulopatie-congenite.-rapporto-2020.-francesca-abbonizio-mauro-biffoni-romano-arcieri-associazione-italiana-centri-emofilia-aice-adele-giampaolo(accessed February 2024).Search in Google Scholar
Centri affiliati | AICE online [Internet]. Available from: https://aiceonline.org/?page_id=758 (accessed February 2024).Centri affiliati | AICE online[]. Available from: https://aiceonline.org/?page_id=758(accessed February 2024).Search in Google Scholar
Cortesi PA, Rocino A, Preti D, et al. Haemophilia management and treatment: An Italian survey on patients’, caregivers’ and clinicians’ point of view. Haemophilia 2022; 28(2): 254-63. doi: 10.1111/hae.14504.CortesiPARocinoAPretiD.Haemophilia management and treatment: An Italian survey on patients’, caregivers’ and clinicians’ point of view.2022;28(2):254-63. doi:10.1111/hae.14504.Open DOISearch in Google Scholar
Calizzani G, Vaglio S, Arcieri R, et al. Models for institutional and professional accreditation of haemophilia centres in Italy. Haemophilia 2013; 19(4): e248-255. doi: 10.1111/hae.12141.CalizzaniGVaglioSArcieriR.Models for institutional and professional accreditation of haemophilia centres in Italy.2013;19(4): e248-255. doi:10.1111/hae.12141.Open DOISearch in Google Scholar
Benson G, Auerswald G, Dolan G, et al. Diagnosis and care of patients with mild haemophilia: practical recommendations for clinical management. Blood Transfus 2018; 16(6): 535-44. doi: 10.2450/2017.0150-17.BensonGAuerswaldGDolanG.Diagnosis and care of patients with mild haemophilia: practical recommendations for clinical management.2018;16(6):535-44. doi:10.2450/2017.0150-17.Open DOISearch in Google Scholar
McLaughlin P, Hermans C, Asghar S, et al. Problem joints and their clinical and humanistic burden in children and adults with moderate and severe hemophilia A: CHESS Paediatrics and CHESS II. Blood 2020; 136(Supplement 1): 33-4. doi: 10.1182/blood-2020-140306.McLaughlinPHermansCAsgharS.Problem joints and their clinical and humanistic burden in children and adults with moderate and severe hemophilia A: CHESS Paediatrics and CHESS II.2020;136(Supplement 1):33-4. doi:10.1182/blood-2020-140306.Open DOISearch in Google Scholar
Nissen F, Burke T, Asghar S, et al. An insight into clinical outcomes in mild, moderate, and severe hemophilia A (HA): A preliminary analysis of the CHESS II study. Abstract OC 09.3. ISTH 2020 Congress. Available from https://abstracts.isth.org/abstract/an-insight-into-clinical-outcomes-in-mild-moderate-and-severe-hemophilia-a-ha-a-preliminary-analysis-of-the-chess-ii-study/.NissenFBurkeTAsgharS..ISTH 2020 Congress. Available from https://abstracts.isth.org/abstract/an-insight-into-clinical-outcomes-in-mild-moderate-and-severe-hemophilia-a-ha-a-preliminary-analysis-of-the-chess-ii-study/.Search in Google Scholar
Ferri Grazzi E, Sun SX, Burke T, O’Hara J. The impact of pharmacokinetic-guided prophylaxis on clinical outcomes and healthcare resource utilization in hemophilia a patients: real-world evidence from the CHESS II Study. J Blood Med 2022; 505-16. doi: 10.2147/JBM.S363028.Ferri GrazziESunSXBurkeTO’HaraJ.The impact of pharmacokinetic-guided prophylaxis on clinical outcomes and healthcare resource utilization in hemophilia a patients: real-world evidence from the CHESS II Study.2022;505-16. doi:10.2147/JBM.S363028.Open DOISearch in Google Scholar
Devlin NJ, Brooks R. EQ-5D and the EuroQol Group: past, present and future. applied health economics and health policy. Appl Health Econ Health Policy 2017; 15(2): 127-137. doi: 10.1007/s40258-017-0310-5.DevlinNJBrooksR.EQ-5D and the EuroQol Group: past, present and future. applied health economics and health policy.2017;15(2):127-137. doi:10.1007/s40258-017-0310-5.Open DOISearch in Google Scholar
Reilly MC, Zbrozek AS, Dukes EM. The validity and reproducibility of a work productivity and activity impairment instrument. PharmacoEconomics 1993; 4(5): 353-65. doi: 10.2165/00019053-199304050-00006.ReillyMCZbrozekASDukesEM.The validity and reproducibility of a work productivity and activity impairment instrument.1993;4(5):353-65. doi:10.2165/00019053-199304050-00006.Open DOISearch in Google Scholar
Burke T, Rodriguez-Santana I, Chowdary P, et al. Humanistic burden of problem joints for children and adults with haemophilia. Haemophilia 2023; 29(2): 608-18. doi: 10.1111/hae.14731.BurkeTRodriguez-SantanaIChowdaryP.Humanistic burden of problem joints for children and adults with haemophilia.2023;29(2):608-18. doi:10.1111/hae.14731.Open DOISearch in Google Scholar
den Uijl I, Biesma D, Grobbee D, Fischer K. Outcome in moderate haemophilia. Blood Transfus 2014; 12(Suppl 1): s330-6. doi: 10.2450/2012.0091-12.den UijlIBiesmaDGrobbeeDFischerK.Outcome in moderate haemophilia.2014;12(Suppl 1): s330-6. doi:10.2450/2012.0091-12.Open DOISearch in Google Scholar
Den Uijl IEM, Fischer K, Van Der Bom JG, Grobbee DE, Rosendaal FR, Plug I. Clinical outcome of moderate haemophilia compared with severe and mild haemophilia. Haemophilia 2009; 15(1): 83-90. doi: 10.1111/j.1365-2516.2008.01837.x.Den UijlIEMFischerKVan Der BomJGGrobbeeDERosendaalFRPlugI.Clinical outcome of moderate haemophilia compared with severe and mild haemophilia.2009;15(1):83-90. doi:10.1111/j.1365-2516.2008.01837.x.Open DOISearch in Google Scholar
Di Minno MND, Ambrosino P, Franchini M, Coppola A, Di Minno G. Arthropathy in patients with moderate hemophilia A: A systematic review of the literature. Semin Thromb Hemost 2013; 39(7): 723-31. doi: 10.1055/s-0033-1354422.Di MinnoMNDAmbrosinoPFranchiniMCoppolaADi MinnoG.Arthropathy in patients with moderate hemophilia A: A systematic review of the literature.2013;39(7):723-31. doi:10.1055/s-0033-1354422.Open DOISearch in Google Scholar
De la Corte-Rodriguez H, Rodriguez-Merchan EC, Alvarez-Roman MT, Martin-Salces M, Rivas-Pollmar I, Jimenez-Yuste V. Arthropathy in people with mild haemophilia: Exploring risk factors. Thromb Res 2022; 211: 19-26. doi: 10.1016/j.thromres.2022.01.010.De la Corte-RodriguezHRodriguez-MerchanECAlvarez-RomanMTMartin-SalcesMRivas-PollmarIJimenez-YusteV.Arthropathy in people with mild haemophilia: Exploring risk factors.2022;211:19-26. doi:10.1016/j.thromres.2022.01.010.Open DOISearch in Google Scholar
Tagliaferri A, Di Perna C, Riccardi F, Pattacini C, Rivolta GF, Franchini M. The natural history of mild haemophilia: a 30-year single centre experience. Haemophilia 2012; 18(2): 166-74. doi: 10.1111/j.1365-2516.2011.02617.x.TagliaferriADi PernaCRiccardiFPattaciniCRivoltaGFFranchiniM.The natural history of mild haemophilia: a 30-year single centre experience.2012;18(2):166-74. doi:10.1111/j.1365-2516.2011.02617.x.Open DOISearch in Google Scholar
Nossair F, Thornburg CD. The role of patient and healthcare professionals in the era of new hemophilia treatments in developed and developing countries. Ther Adv Hematol 2018; 9(8): 239-49. doi: 10.1177/2040620718784830.NossairFThornburgCD.The role of patient and healthcare professionals in the era of new hemophilia treatments in developed and developing countries.2018;9(8):239-49. doi:10.1177/2040620718784830.Open DOISearch in Google Scholar
Makris M, Oldenburg J, Mauser-Bunschoten EP, Peerlinck K, Castaman G, Fijnvandraat K. The definition, diagnosis and management of mild hemophilia A: communication from the SSC of the ISTH. J Thromb Haemost 2018; 16(12): 2530-3. doi: 10.1111/jth.14315.MakrisMOldenburgJMauser-BunschotenEPPeerlinckKCastamanGFijnvandraatK.The definition, diagnosis and management of mild hemophilia A: communication from the SSC of the ISTH.2018;16(12):2530-3. doi:10.1111/jth.14315.Open DOISearch in Google Scholar
Lindvall K, Colstrup L, Loogna K, Wollter IM, Grönhaug S. Knowledge of disease and adherence in adult patients with haemophilia. Haemophilia 2010; 16(4): 592-6. doi: 10.1111/j.1365-2516.2009.02189.x.LindvallKColstrupLLoognaKWollterIMGrönhaugS.Knowledge of disease and adherence in adult patients with haemophilia.2010;16(4):592-6. doi:10.1111/j.1365-2516.2009.02189.x.Open DOISearch in Google Scholar
Nilson J, Schachter C, Mulder K, et al. A qualitative study identifying the knowledge, attitudes and behaviours of young men with mild haemophilia. Haemophilia 2012; 18(3): e120-5. doi: 10.1111/j.1365-2516.2011.02714.x.NilsonJSchachterCMulderK.A qualitative study identifying the knowledge, attitudes and behaviours of young men with mild haemophilia.2012;18(3): e120-5. doi:10.1111/j.1365-2516.2011.02714.x.Open DOISearch in Google Scholar