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The patient gene therapy journey: Findings from qualitative interviews with trial participants at one UK haemophilia centre


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Doshi BS, Arruda VR. Gene therapy for hemophilia: what does the future hold? Ther Adv Hematol 2018; 9(9): 273–93. doi: 10.1177/2040620718791933. DoshiBS ArrudaVR Gene therapy for hemophilia: what does the future hold? Ther Adv Hematol 2018 9 9 273 93 10.1177/2040620718791933 613009930210756 Open DOISearch in Google Scholar

Ohmori T. Advances in gene therapy for hemophilia: basis, current status, and future perspectives. Int J Hematol 2020; 111(1): 31–41. doi: 10.1007/s12185-018-2513-4. OhmoriT Advances in gene therapy for hemophilia: basis, current status, and future perspectives Int J Hematol 2020 111 1 31 41 10.1007/s12185-018-2513-4 30083852 Open DOISearch in Google Scholar

Ohmori T, Mizukami H, Ozawa K, Sakata Y, Nishimura S. New approaches to gene and cell therapy for hemophilia. J Thromb Haemost 2015; 13(suppl 1): S133–42. doi: 10.1111/jth.12926. OhmoriT MizukamiH OzawaK SakataY NishimuraS New approaches to gene and cell therapy for hemophilia J Thromb Haemost 2015 13 suppl 1 S133 42 10.1111/jth.12926 26149014 Open DOISearch in Google Scholar

Kumar SRP, Markusic DM, Biswas M, High KA, Herzog RW. Clinical development of gene therapy: results and lessons from recent successes. Mol Ther Methods Clin Dev 2016; 3: 16034. doi: 10.1038/mtm.2016.34. KumarSRP MarkusicDM BiswasM HighKA HerzogRW Clinical development of gene therapy: results and lessons from recent successes Mol Ther Methods Clin Dev 2016 3 16034 10.1038/mtm.2016.34 487999227257611 Open DOISearch in Google Scholar

Pasi KJ, Rangarajan S, Mitchell N, et al. Multiyear follow-up of AAV5-hFVIII-SQ gene therapy for hemophilia A. N Engl J Med 2020; 382: 29–40. doi: 10.1056/NEJMoa1908490. PasiKJ RangarajanS MitchellN Multiyear follow-up of AAV5-hFVIII-SQ gene therapy for hemophilia A N Engl J Med 2020 382 29 40 10.1056/NEJMoa1908490 31893514 Open DOISearch in Google Scholar

Pipe S, Becka M, Detering E, Vanevski K, Lissitchkov T. First-in-human gene therapy study of AAVhu37 capsid vector technology in severe hemophilia A. Blood 2019; 134 (Supplement 1): 4630. doi: 10.1182/blood-2019-125764. PipeS BeckaM DeteringE VanevskiK LissitchkovT First-in-human gene therapy study of AAVhu37 capsid vector technology in severe hemophilia A Blood 2019 134 Supplement 1 4630 10.1182/blood-2019-125764 Open DOISearch in Google Scholar

Konkle BA, Stine K, Visweshwar N, et al. Initial results of the Alta Study, a phase 1/2, open label, adaptive, dose-ranging study to assess the safety and tolerability of SB-525 gene therapy in adult subjects with severe hemophilia A. Blood 2019; 134 (Supplement 1): 2060. doi: 10.1182/blood-2019-122143. KonkleBA StineK VisweshwarN Initial results of the Alta Study, a phase 1/2, open label, adaptive, dose-ranging study to assess the safety and tolerability of SB-525 gene therapy in adult subjects with severe hemophilia A Blood 2019 134 Supplement 1 2060 10.1182/blood-2019-122143 Open DOISearch in Google Scholar

High KA, George LA, Eyster ME, et al. A phase 1/2 trial of investigational Spk-8011 in hemophilia A demonstrates durable expression and prevention of bleeds. Blood 2018; 132 (Supplement 1): 487. doi: 10.1182/blood-2018-99-115495. HighKA GeorgeLA EysterME A phase 1/2 trial of investigational Spk-8011 in hemophilia A demonstrates durable expression and prevention of bleeds Blood 2018 132 Supplement 1 487 10.1182/blood-2018-99-115495 Open DOISearch in Google Scholar

Nathwani AC, Reiss U, Tuddenham E, et al. Adeno-associated mediated gene transfer for hemophilia B: 8 year follow up and impact of removing “empty viral particles” on safety and efficacy of gene transfer. Blood 2018; 132 (Supplement 1): 491. doi: 10.1182/blood-2018-99-118334. NathwaniAC ReissU TuddenhamE Adeno-associated mediated gene transfer for hemophilia B: 8 year follow up and impact of removing “empty viral particles” on safety and efficacy of gene transfer Blood 2018 132 Supplement 1 491 10.1182/blood-2018-99-118334 Open DOISearch in Google Scholar

Chowdary P, Shapiro S, Boyce S, et al. FLT180a: next generation AAV vector for haemophilia B – long term, follow-up and in-depth analysis of transgenic FIX using one-stage, chromogenic and global assays. ISTH Academy 2019; 263496; PB0309. ChowdaryP ShapiroS BoyceS FLT180a: next generation AAV vector for haemophilia B – long term, follow-up and in-depth analysis of transgenic FIX using one-stage, chromogenic and global assays ISTH Academy 2019 263496; PB0309. Search in Google Scholar

Nathwani AC, Reiss UM, Tuddenham EGD, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med 2014; 371(21): 1994–2004. doi: 10.1056/NEJMoa1407309. NathwaniAC ReissUM TuddenhamEGD Long-term safety and efficacy of factor IX gene therapy in hemophilia B N Engl J Med 2014 371 21 1994 2004 10.1056/NEJMoa1407309 427880225409372 Open DOISearch in Google Scholar

Leebeek FWG, Meijer K, Coppens M, et al. Stable expression of FIX and maintained reductions in bleeding and factor IX consumption following AMT-060 gene therapy with up to 3.5 years of follow up in adults with severe or moderate-severe hemophilia B. ISTH Academy 2019; 274036; OC 01.4. LeebeekFWG MeijerK CoppensM Stable expression of FIX and maintained reductions in bleeding and factor IX consumption following AMT-060 gene therapy with up to 3.5 years of follow up in adults with severe or moderate-severe hemophilia B ISTH Academy 2019 274036; OC 01.4. Search in Google Scholar

Von Drygalski A, Giermasz A, Castaman G, et al. Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B. Blood Adv 2019; 3(21): 3241–47. doi: 10.1182/bloodadvances.2019000811. Von DrygalskiA GiermaszA CastamanG Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B Blood Adv 2019 3 21 3241 47 10.1182/bloodadvances.2019000811 685510131698454 Open DOISearch in Google Scholar

George LA, Sullivan SK, Rasko JEJ, et al. Efficacy and safety in 15 hemophilia B patients treated with the AAV gene therapy vector fidanacogene elaparvovec and followed for at least 1 year. Blood 2019; 134 (Supplement 1): 3347. doi: 10.1182/blood-2019-124091. GeorgeLA SullivanSK RaskoJEJ Efficacy and safety in 15 hemophilia B patients treated with the AAV gene therapy vector fidanacogene elaparvovec and followed for at least 1 year Blood 2019 134 Supplement 1 3347 10.1182/blood-2019-124091 Open DOISearch in Google Scholar

Stoffman J, Andersson NG, Branchford B, et al. Common themes and challenges in hemophilia care: a multinational perspective. Hematology 2019; 24: 39–48. doi: 10.1080/10245332.2018.1505225. StoffmanJ AnderssonNG BranchfordB Common themes and challenges in hemophilia care: a multinational perspective Hematology 2019 24 39 48 10.1080/10245332.2018.1505225 30073913 Open DOISearch in Google Scholar

Pollard D, Harrison C, Dodgson S, Holland M, Khair K. The UK haemophilia specialist nurse: Competencies fit for practice in the 21st century. Haemophilia 2020; 26(4): 622–30. doi: 10.1111/hae.14002. PollardD HarrisonC DodgsonS HollandM KhairK The UK haemophilia specialist nurse: Competencies fit for practice in the 21st century Haemophilia 2020 26 4 622 30 10.1111/hae.14002 749622632311205 Open DOISearch in Google Scholar

Miesbach W, Klamroth R. The patient experience of gene therapy for hemophilia: qualitative interviews with trial patients. Patient Pref Adherence 2020; 14: 767–70. doi: 10.2147/PPA.S239810. MiesbachW KlamrothR The patient experience of gene therapy for hemophilia: qualitative interviews with trial patients Patient Pref Adherence 2020 14 767 70 10.2147/PPA.S239810 718485832368018 Open DOISearch in Google Scholar

Braun V, Clarke V. Using thematic analysis in psychology. Qual Res Psychol 2008; 3: 77–101. doi: 10.1191/1478088706qp063oa. BraunV ClarkeV Using thematic analysis in psychology Qual Res Psychol 2008 3 77 101 10.1191/1478088706qp063oa Open DOISearch in Google Scholar

Sundler AJ, Lindberg E, Nilsson C, Palmér L. Qualitative thematic analysis based on descriptive phenomenology. Nurs Open 2019; 6(3): 733–9. doi: 10.1002/nop2.275. SundlerAJ LindbergE NilssonC PalmérL Qualitative thematic analysis based on descriptive phenomenology Nurs Open 2019 6 3 733 9 10.1002/nop2.275 665066131367394 Open DOISearch in Google Scholar

UKRI Medical Research Council. Is my study research? Available from http://www.hra-decisiontools.org.uk/research/ (accessed 15 March 2021). UKRI Medical Research Council Is my study research? Available from http://www.hra-decisiontools.org.uk/research/ (accessed 15 March 2021). Search in Google Scholar

Van Balen EC, Wesselo ML, Baker BL, et al. Patient perspectives on novel treatments in haemophilia: a qualitative study. Patient 2020; 13(2): 201–10. doi: 10.1007/s40271-019-00395-6. Van BalenEC WesseloML BakerBL Patient perspectives on novel treatments in haemophilia: a qualitative study Patient 2020 13 2 201 10 10.1007/s40271-019-00395-6 707583831691206 Open DOISearch in Google Scholar

Costea I, Isasi R, Knoppers BM, Lillicrap D. Haemophilia gene therapy: the patients' perspective. Haemophilia 2009; 15(5): 1159–61. doi: 10.1111/j.1365-2516.2009.02065.x. CosteaI IsasiR KnoppersBM LillicrapD Haemophilia gene therapy: the patients' perspective Haemophilia 2009 15 5 1159 61 10.1111/j.1365-2516.2009.02065.x 19601988 Open DOISearch in Google Scholar

Woollard L, Gorman R, Rosenfelt DJ. Addressing patient education priorities in the era of gene therapy for haemophilia: Towards evidence–informed shared decision–making. Haemophilia 2020 Nov 27. doi: 10.1111/hae.14214. Online ahead of print. WoollardL GormanR RosenfeltDJ Addressing patient education priorities in the era of gene therapy for haemophilia: Towards evidence–informed shared decision–making Haemophilia 2020 Nov 27 10.1111/hae.14214 Online ahead of print. 33245825 Open DOISearch in Google Scholar

Peyvandi F, Lillicrap D, Mahlangu J, et al. Hemophilia gene therapy knowledge and perceptions: Results of an international survey. Res Pract Thromb Haemost 2020; 4(4): 644–51. doi: 10.1002/rth2.12326. PeyvandiF LillicrapD MahlanguJ Hemophilia gene therapy knowledge and perceptions: Results of an international survey Res Pract Thromb Haemost 2020 4 4 644 51 10.1002/rth2.12326 729267332548564 Open DOISearch in Google Scholar

Arcé C, Branchford B, Hart DP, et al. Optimizing language for effective communication of gene therapy concepts: A qualitative study. Presented at NHF Bleeding Disorders Conference 2019; PSO71. Available from https://www.hemophilia.org/research/research-projects/optimizing-language-for-effective-communication-of-gene-therapy-concepts-a-qualitative-study (accessed 15 March 2021). ArcéC BranchfordB HartDP Optimizing language for effective communication of gene therapy concepts: A qualitative study Presented at NHF Bleeding Disorders Conference 2019 PSO71 Available from https://www.hemophilia.org/research/research-projects/optimizing-language-for-effective-communication-of-gene-therapy-concepts-a-qualitative-study (accessed 15 March 2021). Search in Google Scholar

Morris J. Patient advocacy helps patients weigh up gene therapy trial risk/benefits. J Haem Pract 2015; 2(1): 6–8. doi: 10.17225/jhp.00040. MorrisJ Patient advocacy helps patients weigh up gene therapy trial risk/benefits J Haem Pract 2015 2 1 6 8 10.17225/jhp.00040 Open DOISearch in Google Scholar

Forrester C, Bielby H, Johns S, Efford J, Holland M, Khair K, United Kingdom Haemophilia Nurse's Association. Potential for development of haemophilia link nurse role within UK hospitals. Haemophilia 2013; 19(4): 578–82. doi: 10.1111/hae.12144. ForresterC BielbyH JohnsS EffordJ HollandM KhairK United Kingdom Haemophilia Nurse's Association Potential for development of haemophilia link nurse role within UK hospitals Haemophilia 2013 19 4 578 82 10.1111/hae.12144 23560696 Open DOISearch in Google Scholar

Khair K. Supporting adherence and improving quality of life in haemophilia care. Br J Nurs 2013; 22(12): 692. doi: 10.12968/bjon.2013.22.12.692. KhairK Supporting adherence and improving quality of life in haemophilia care Br J Nurs 2013 22 12 692 10.12968/bjon.2013.22.12.692 24151707 Open DOISearch in Google Scholar

Knobe K, Berntorp E. Haemophilia and joint disease: pathophysiology, evaluation, and management. J Comorb 2011; 1: 51–9. doi: 10.15256/joc.2011.1.2. KnobeK BerntorpE Haemophilia and joint disease: pathophysiology, evaluation, and management J Comorb 2011 1 51 9 10.15256/joc.2011.1.2 555642129090136 Open DOISearch in Google Scholar

Harrington C, Bedford M, Andritschke K, et al; EAHAD Nurses Committee. A European curriculum for nurses working in haemophilia. Haemophilia 2016; 22: 103–9. doi: 10.1111/hae.12785. HarringtonC BedfordM AndritschkeK EAHAD Nurses Committee A European curriculum for nurses working in haemophilia Haemophilia 2016 22 103 9 10.1111/hae.12785 26278710 Open DOISearch in Google Scholar

Khair K. Minimizing joint damage: the role of nurses in promoting adherence to hemophilia treatment. Orthop Nurs 2010; 29(3): 193–200. doi: 10.1097/NOR.0b013e3181db53e8. KhairK Minimizing joint damage: the role of nurses in promoting adherence to hemophilia treatment Orthop Nurs 2010 29 3 193 200 10.1097/NOR.0b013e3181db53e8 20505489 Open DOISearch in Google Scholar

Pierce GF, Coffin D, members of the WFH Gene Therapy Round Table Program Committee and Organizing Committee. The 1st WFH gene therapy round table: understanding the landscape and challenges of gene therapy for haemophilia around the world. Haemophilia 2019; 25(2): 189–194. doi: 10.1111/hae.13673. PierceGF CoffinD members of the WFH Gene Therapy Round Table Program Committee and Organizing Committee The 1st WFH gene therapy round table: understanding the landscape and challenges of gene therapy for haemophilia around the world Haemophilia 2019 25 2 189 194 10.1111/hae.13673 30604914 Open DOISearch in Google Scholar

Hurst S, Warren C, Pasi KJ. Gene therapy in hemophilia: an assessment of hematologists’ knowledge gaps and attitudes. Blood; 2018; 132 (Supplement 1): 3485. doi: 10.1182/blood-2018-99-119116. HurstS WarrenC PasiKJ Gene therapy in hemophilia: an assessment of hematologists’ knowledge gaps and attitudes Blood 2018 132 Supplement 1 3485 10.1182/blood-2018-99-119116 Open DOISearch in Google Scholar

High KA, Roncarolo MG. Gene therapy. N Engl J Med 2019; 381: 455–64. doi: 10.1056/NEJMra1706910. HighKA RoncaroloMG Gene therapy N Engl J Med 2019 381 455 64 10.1056/NEJMra1706910 31365802 Open DOISearch in Google Scholar

Mulders G, De Wee EM, Vahedi Nikbakht-Van De Sande MCVM, Kruip MJHA, Elfrink EJ, Leebeek FWG. E-learning improves knowledge and practical skills in haemophilia patients on home treatment: a randomized controlled trial. Haemophilia 2012; 18(5): 693–8. doi: 10.1111/j.1365-2516.2012.02786.x. MuldersG De WeeEM Vahedi Nikbakht-Van De SandeMCVM KruipMJHA ElfrinkEJ LeebeekFWG E-learning improves knowledge and practical skills in haemophilia patients on home treatment: a randomized controlled trial Haemophilia 2012 18 5 693 8 10.1111/j.1365-2516.2012.02786.x 22458978 Open DOISearch in Google Scholar

Miesbach W, O’Mahony B, Key NS, Makris M. How to discuss gene therapy for haemophilia? A patient and physician perspective. Haemophilia 2019; 25(4): 545–57. doi: 10.1111/hae.13769. MiesbachW O’MahonyB KeyNS MakrisM How to discuss gene therapy for haemophilia? A patient and physician perspective Haemophilia 2019 25 4 545 57 10.1111/hae.13769 685220731115117 Open DOISearch in Google Scholar

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