Patient-reported outcomes in patients with paroxysmal nocturnal hemoglobinuria treated with crovalimab and approved C5 inhibitors in the phase III COMMODORE 2 and 1 studies
Status Publisher Jazyk angličtina Země Německo Médium print-electronic
Typ dokumentu časopisecké články
PubMed
40515823
DOI
10.1007/s00277-025-06449-0
PII: 10.1007/s00277-025-06449-0
Knihovny.cz E-zdroje
- Klíčová slova
- Complement C5 inhibitor, Crovalimab, Eculizumab, Paroxysmal nocturnal hemoglobinuria, Patient-reported outcomes, Ravulizumab,
- Publikační typ
- časopisecké články MeSH
Centre for Integrated Oncology Germany
Department of Haematological Medicine King's College Hospital London UK
Department of Haematology University Hospital Saint Luc Brussels Belgium
Department of Hematology Ege University Izmir Turkey
Department of Hematology Tokyo Medical University Tokyo Japan
Division of Hematology Hospital A Beneficência Portuguesa São Paulo Brazil
F Hoffmann La Roche Ltd Basel Switzerland
Genentech Inc South San Francisco CA USA
Hematology Service Hospital Clínico San Carlos Madrid Spain
Institute of Hematology and Blood Transfusion Prague Czech Republic
Institute of Hematology and Transfusiology National Academy of Medical Science Kyiv Ukraine
Institute of Transfusion Medicine University of Ulm Ulm Germany
Medical Center Ok!Clinic of International Institute of Clinical Research LLC Kyiv Ukraine
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Devos T, Meers S, Boeckx N, Gothot A, Deeren D, Chatelain B, Chatelain C, Devalet B (2018) Diagnosis and management of PNH: review and recommendations from a Belgian expert panel. Eur J Haematol 101(6):737–749. https://doi.org/10.1111/ejh.13166 PubMed DOI
Hillmen P (2008) The role of complement Inhibition in PNH. Hematol 2008 1:116–123. https://doi.org/10.1182/asheducation-2008.1.116 DOI
Brodsky RA (2021) How I treat paroxysmal nocturnal hemoglobinuria. Blood 137(10):1304–1309. https://doi.org/10.1182/blood.2019003812 PubMed DOI PMC
Socié G, Mary J-Y, de Gramont A, Rio B, Leporrier M, Rose C, Heudier P, Rochant H, Cahn J-Y, Gluckman E (1996) Paroxysmal nocturnal haemoglobinuria: long-term follow-up and prognostic factors. Lancet 348(9027):573–577. https://doi.org/10.1016/S0140-6736(95)12360-1 PubMed DOI
Hill A, DeZern AE, Kinoshita T, Brodsky RA (2017) Paroxysmal nocturnal haemoglobinuria. Nat Rev Dis Primers 3:17028. https://doi.org/10.1038/nrdp.2017.28 PubMed DOI PMC
Risitano AM, Peffault de Latour R (2022) How we(‘ll) treat paroxysmal nocturnal haemoglobinuria: diving into the future. Br J Haematol 196(2):288–303. https://doi.org/10.1111/bjh.17753 PubMed DOI
Hillmen P, Young NS, Schubert J, Brodsky RA, Socié G, Muus P, Röth A, Szer J, Elebute MO, Nakamura R, Browne P, Risitano AM, Hill A, Schrezenmeier H, Fu CL, Maciejewski J, Rollins SA, Mojcik CF, Rother RP, Luzzatto L (2006) The complement inhibitor Eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med 355(12):1233–1243. https://doi.org/10.1056/NEJMoa061648 PubMed DOI
Lee JW, Sicre de Fontbrune F, Wong Lee Lee L, Pessoa V, Gualandro S, Füreder W, Ptushkin V, Rottinghaus ST, Volles L, Shafner L, Aguzzi R, Pradhan R, Schrezenmeier H, Hill A (2019) Ravulizumab (ALXN1210) vs Eculizumab in adult patients with PNH Naive to complement inhibitors: the 301 study. Blood 133(6):530–539. https://doi.org/10.1182/blood-2018-09-876136 PubMed DOI PMC
Kulasekararaj AG, Hill A, Rottinghaus ST, Langemeijer S, Wells R, Gonzalez-Fernandez FA, Gaya A, Lee JW, Gutierrez EO, Piatek CI, Szer J, Risitano A, Nakao S, Bachman E, Shafner L, Damokosh AI, Ortiz S, Röth A, de Peffault R (2019) Ravulizumab (ALXN1210) vs Eculizumab in C5-inhibitor-experienced adult patients with PNH: the 302 study. Blood 133(6):540–549. https://doi.org/10.1182/blood-2018-09-876805 PubMed DOI PMC
Kelly RJ, Hill A, Arnold LM, Brooksbank GL, Richards SJ, Cullen M, Mitchell LD, Cohen DR, Gregory WM, Hillmen P (2011) Long-term treatment with Eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival. Blood 117(25):6786–6792. https://doi.org/10.1182/blood-2011-02-333997 PubMed DOI
Kulasekararaj AG, Griffin M, Langemeijer S, Usuki K, Kulagin A, Ogawa M, Yu J, Mujeebuddin A, Nishimura JI, Lee JW, Peffault de Latour R, Group S (2022) Long-term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2-year results from two pivotal phase 3 studies. Eur J Haematol. https://doi.org/10.1111/ejh.13783 PubMed DOI PMC
Kelly RJ, Holt M, Vidler J, Arnold LM, Large J, Forrest B, Barnfield C, Pike A, Griffin M, Munir T, Muus P, Nagumantry SK, Varghese A, Davies JR, Trikha R, Kulasekararaj AG, Mitchell L, Gandhi S (2024) Treatment outcomes of complement protein C5 Inhibition in 509 UK patients with paroxysmal nocturnal hemoglobinuria. Blood 143(12):1157–1166. https://doi.org/10.1182/blood.2023021762 PubMed DOI
Levy AR, Dysart L, Patel Y, Briggs A, Schneider J, Myren K-J, Tomazos I (2019) Comparison of lost productivity due to Eculizumab and ravulizumab treatments for paroxysmal nocturnal hemoglobinuria in france, germany, italy, russia, spain, the united kingdom, and the united States. Blood 134(Supplement1):4803–4803. https://doi.org/10.1182/blood-2019-127443 DOI
Kaiser K, Yount SE, Martens CE, Webster KA, Shaunfield S, Sparling A, Peipert JD, Cella D, Rottinghaus ST, Donato BMK, Wells R, Tomazos I (2020) Assessing preferences for rare disease treatment: qualitative development of the paroxysmal nocturnal hemoglobinuria patient preference questionnaire (PNH-PPQ. Patient Prefer Adherence 14:705–715. https://doi.org/10.2147/PPA.S233830 PubMed DOI PMC
Groth M, Singer S, Niedeggen C, Petermann-Meyer A, Röth A, Schrezenmeier H, Höchsmann B, Brümmendorf TH, Panse J (2017) Development of a disease-specific quality of life questionnaire for patients with aplastic Anemia and/or paroxysmal nocturnal hemoglobinuria (QLQ-AA/PNH)-report on phases I and II. Ann Hematol 96(2):171–181. https://doi.org/10.1007/s00277-016-2867-8 PubMed DOI
Peipert JD, Kulasekararaj AG, Gaya A, Langemeijer SMC, Yount S, Gonzalez-Fernandez FA, Ojeda Gutierrez E, Martens C, Sparling A, Webster KA, Cella D, Tomazos I, Ogawa M, Piatek CI, Wells R, Sicre de Fontbrune F, Röth A, Mitchell L, Hill A, Kaiser K (2020) Patient preferences and quality of life implications of ravulizumab (every 8 weeks) and Eculizumab (every 2 weeks) for the treatment of paroxysmal nocturnal hemoglobinuria. PLoS ONE 15(9):e0237497. https://doi.org/10.1371/journal.pone.0237497 PubMed DOI PMC
Bektas M, Copley-Merriman C, Khan S, Sarda SP, Shammo JM (2020) Paroxysmal nocturnal hemoglobinuria: current treatments and unmet needs. J Managed Care Specialty Pharmacy 26 (12-b Suppl):S14-S20. https://doi.org/10.18553/jmcp.2020.26.12-b.s14
Peffault de Latour R, Hosokawa K, Risitano AM (2022) Hemolytic paroxysmal nocturnal hemoglobinuria: 20 years of medical progress. Semin Hematol 59(1):38–46. https://doi.org/10.1053/j.seminhematol.2022.01.001 PubMed DOI
Chugai Pharmaceutical Co. Ltd (2024) Chugai Obtains Regulatory Approval for Piasky 340 mg for Paroxysmal Nocturnal Hemoglobinuria in Japan. https://www.chugai-pharm.co.jp/english/news/detail/20240326160001_1056.html . Accessed April 1 2024
Chugai Pharmaceutical Co. Ltd (2024) Crovalimab Approved in China as the First Country, for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH). https://www.chugai-pharm.co.jp/english/news/detail/20240208113000_1047.html?year=2024&category= . Accessed February 27 2024
PiaSky (2024) (crovalimab) [prescribing information]. South San Francisco, CA: Genentech, Inc.
PiaSky (2024) (crovalimab) [summary of product characteristics]. Grenzach-Wyhlen, Germany; Roche Registration GmbH
Fukuzawa T, Sampei Z, Haraya K, Ruike Y, Shida-Kawazoe M, Shimizu Y, Gan SW, Irie M, Tsuboi Y, Tai H, Sakiyama T, Sakamoto A, Ishii S, Maeda A, Iwayanagi Y, Shibahara N, Shibuya M, Nakamura G, Nambu T, Hayasaka A, Mimoto F, Okura Y, Hori Y, Habu K, Wada M, Miura T, Tachibana T, Honda K, Tsunoda H, Kitazawa T, Kawabe Y, Igawa T, Hattori K, Nezu J (2017) Long lasting neutralization of C5 by SKY59, a novel recycling antibody, is a potential therapy for complement-mediated diseases. Sci Rep 7(1):1080. https://doi.org/10.1038/s41598-017-01087-7 PubMed DOI PMC
Röth A, Nishimura JI, Nagy Z, Gaàl-Weisinger J, Panse J, Yoon SS, Egyed M, Ichikawa S, Ito Y, Kim JS, Ninomiya H, Schrezenmeier H, Sica S, Usuki K, Sicre de Fontbrune F, Soret J, Sostelly A, Higginson J, Dieckmann A, Gentile B, Anzures-Cabrera J, Shinomiya K, Jordan G, Biedzka-Sarek M, Klughammer B, Jahreis A, Bucher C, de Peffault R (2020) The complement C5 inhibitor crovalimab in paroxysmal nocturnal hemoglobinuria. Blood 135(12):912–920. https://doi.org/10.1182/blood.2019003399 PubMed DOI PMC
Nishimura JI, Usuki K, Ramos J, Ichikawa S, Buri M, Kiialainen A, Sostelly A, Peffault de Latour R, Paz-Priel I, Röth A (2022) Crovalimab for treatment of patients with paroxysmal nocturnal haemoglobinuria and complement C5 polymorphism: subanalysis of the phase 1/2 COMPOSER study. Br J Haematol 198(3):e46–e50. https://doi.org/10.1111/bjh.18274 PubMed DOI
Röth A, He G, Tong H, Lin Z, Wang X, Chai-Adisaksopha C, Lee JH, Brodsky A, Hantaweepant C, Dumagay TE, Demichelis-Gómez R, Rojnuckarin P, Sun J, Höglund M, Jang JH, Gaya A, Silva F, Obara N, Kelly RJ, Beveridge L, Buatois S, Chebon S, Gentile B, Lundberg P, Sreckovic S, Nishimura JI, Risitano A, Han B (2024) Phase 3 randomized COMMODORE 2 trial: crovalimab versus Eculizumab in patients with paroxysmal nocturnal hemoglobinuria Naive to complement Inhibition. Am J Hematol. https://doi.org/10.1002/ajh.27412 PubMed DOI
Scheinberg P, Clé DV, Kim JS, Nur E, Yenerel MN, Barcellini W, Bonito D, Giai V, Hus M, Lee Y, Lekue CB, Panse J, Ueda Y, Buatois S, Gentile B, Kiialainen A, Patel H, Sreckovic S, Uguen M, Edwards J, Nagy Z, Kulasekararaj AG (2024) Phase 3 randomized COMMODORE 1 trial: crovalimab versus Eculizumab in complement inhibitor-experienced patients with paroxysmal nocturnal hemoglobinuria. Am J Hematol. https://doi.org/10.1002/ajh.27413 PubMed DOI
Fayers P, Aaronson NK, Bjordal K, Groenvold M, Curran D, Bottomle A (2001) EORTC QLQ-C30 scoring manual, 3rd edn. European Organisation for Research and Treatment of Cancer, Brussels
Röth A, Panse J, Gentile B, Buri M, Patel H, Nagy Z (2022) P831: Patient-reported outcomes in patients with paroxysmal nocturnal hemoglobinuria treated with crovalimab: results from the COMPOSER trial. HemaSphere 6:725–726. https://doi.org/10.1097/01.HS9.0000846208.84566.a3 DOI PMC
Atkinson MJ, Sinha A, Hass SL, Colman SS, Kumar RN, Brod M, Rowland CR (2004) Validation of a general measure of treatment satisfaction, the treatment satisfaction questionnaire for medication (TSQM), using a National panel study of chronic disease. Health Qual Life Outcomes 2:12. https://doi.org/10.1186/1477-7525-2-12 [doi] PubMed DOI PMC
Bharmal M, Payne K, Atkinson MJ, Desrosiers MP, Morisky DE, Gemmen E (2009) Validation of an abbreviated treatment satisfaction questionnaire for medication (TSQM-9) among patients on antihypertensive medications. Health Qual Life Outcomes 7:36. https://doi.org/10.1186/1477-7525-7-36 PubMed DOI PMC
Butt Z, Rao AV, Lai JS, Abernethy AP, Rosenbloom SK, Cella D (2010) Age-associated differences in fatigue among patients with cancer. J Pain Symptom Manage 40(2):217–223. https://doi.org/10.1016/j.jpainsymman.2009.12.016 PubMed DOI PMC
Cella D, Eton DT, Lai JS, Peterman AH, Merkel DE (2002) Combining anchor and distribution-based methods to derive minimal clinically important differences on the functional assessment of Cancer therapy (FACT) anemia and fatigue scales. J Pain Symptom Manage 24(6):547–561. https://doi.org/10.1016/s0885-3924(02)00529-8 PubMed DOI
Montan I, Löwe B, Cella D, Mehnert A, Hinz A (2018) General population norms for the functional assessment of chronic illness therapy (FACIT)-Fatigue scale. Value Health 21(11):1313–1321. https://doi.org/10.1016/j.jval.2018.03.013 PubMed DOI
Cella D, Johansson P, Ueda Y, Tomazos I, Gustovic P, Wang A, Patel AS, Schrezenmeier H (2023) Clinically important change for the FACIT-Fatigue scale in paroxysmal nocturnal hemoglobinuria: a derivation from international PNH registry patient data. J Patient Rep Outcomes 7(1):63. https://doi.org/10.1186/s41687-023-00609-4 PubMed DOI PMC
Dingli D, Matos JE, Lehrhaupt K, Krishnan S, Yeh M, Fishman J, Sarda SP, Baver SB (2022) The burden of illness in patients with paroxysmal nocturnal hemoglobinuria receiving treatment with the C5-inhibitors Eculizumab or ravulizumab: results from a US patient survey. Ann Hematol 101(2):251–263. https://doi.org/10.1007/s00277-021-04715-5 PubMed DOI PMC
Kulasekararaj A, Griffin M, Piatek C, Shammo J, Nishimura JI, Patriquin C, Schrezenmeier H, Barcellini W, Panse J, Gaya A, Patel Y, Liu P, Filippov G, Sicre de Fontbrune F, Risitano A, Lee JW (2025) Long-term efficacy and safety of Danicopan as add-on therapy to ravulizumab or Eculizumab in PNH with significant EVH. Blood 145(8):811–822. https://doi.org/10.1182/blood.2024026299 PubMed DOI
Risitano AM, de Castro C, Han B, Kulasekararaj A, Maciejewski JP, Scheinberg P, Ueda Y, Vallow S, Bermann G, Dahlke M, Kumar R, de Peffault R (2025) Patient-reported improvements in paroxysmal nocturnal hemoglobinuria treated with Iptacopan from 2 phase 3 studies. Blood Adv 9(8):1816–1826. https://doi.org/10.1182/bloodadvances.2024014652 PubMed DOI PMC
Cocks K, King MT, Velikova G, Fayers PM, Brown JM (2008) Quality, interpretation and presentation of European organisation for research and treatment of Cancer quality of life questionnaire core 30 data in randomised controlled trials. Eur J Cancer 44(13):1793–1798. https://doi.org/10.1016/j.ejca.2008.05.008 PubMed DOI
Osoba D, Rodrigues G, Myles J, Zee B, Pater J (1998) Interpreting the significance of changes in health-related quality-of-life scores. J Clin Oncol 16(1):139–144. https://doi.org/10.1200/JCO.1998.16.1.139 PubMed DOI
Oliva EN, Risitano AM, Di Bona E, Vannucchi AM, Delfino IM, De Vivo A, Giai V, Notaro R, Ianni G, Beggiato E, Sica S, La Barba G, Balakrishnan S, Thavarajah T, Raso S, Fontana R, Barcellini W, Fattizzo B, Trikha R, Marano L, Panse J, Kulasekararaj AG (2024) The association of anemia and patient reported outcomes in the new era of PNH treatment. Blood 144(Supplement1):2321–2323. https://doi.org/10.1182/blood-2024-204693 DOI
US Food & Drug Administration (2023) Rare Diseases: Considerations for the Development of Drugs and Biological Products. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/rare-diseases-considerations-development-drugs-and-biological-products . Accessed 22 April 2024
Anota A, Pozet A, Lemasson H, Cotté FE, Falcoz A, Eberst G, Mouillet G, Guerzider S, Charton É, Westeel V (2022) Impact of open-label versus blinded study design on patient-reported outcomes data in randomized clinical trials of immunotherapy in advanced or metastatic cancer patients: a systematic review. Qual Life Res 31(3):645–657. https://doi.org/10.1007/s11136-021-02945-8 PubMed DOI
Mouillet G, Efficace F, Thiery-Vuillemin A, Charton E, Van Hemelrijck M, Sparano F, Anota A (2020) Investigating the impact of open label design on patient-reported outcome results in prostate cancer randomized controlled trials. Cancer Med 9(20):7363–7374. https://doi.org/10.1002/cam4.3335 PubMed DOI PMC
Atkinson TM, Wagner JS, Basch E (2017) Trustworthiness of Patient-Reported outcomes in unblinded Cancer clinical trials. JAMA Oncol 3(6):738–739. https://doi.org/10.1001/jamaoncol.2016.3328 PubMed DOI PMC
Lord-Bessen J, Signorovitch J, Yang M, Georgieva M, Roydhouse J (2023) Assessing the impact of open-label designs in patient-reported outcomes: investigation in oncology clinical trials. JNCI Cancer Spectr 7(2). https://doi.org/10.1093/jncics/pkad002
Efficace F, Cella D, Aaronson NK, Calvert M, Cottone F, Di Maio M, Perrone F, Sparano F, Gamper EM, Vignetti M, Giesinger JM (2022) Impact of blinding on Patient-Reported outcome differences between treatment arms in Cancer randomized controlled trials. J Natl Cancer Inst 114(3):471–474. https://doi.org/10.1093/jnci/djab177 PubMed DOI
Moustgaard H, Clayton GL, Jones HE, Boutron I, Jørgensen L, Laursen DRT, Olsen MF, Paludan-Müller A, Ravaud P, Savović J, Sterne JAC, Higgins JPT, Hróbjartsson A (2020) Impact of blinding on estimated treatment effects in randomised clinical trials: meta-epidemiological study. BMJ 368:l6802. https://doi.org/10.1136/bmj.l6802 PubMed DOI PMC
Roydhouse JK, King-Kallimanis BL, Howie LJ, Singh H, Kluetz PG (2019) Blinding and Patient-Reported outcome completion rates in US food and drug administration Cancer trial submissions, 2007–2017. J Natl Cancer Inst 111(5):459–464. https://doi.org/10.1093/jnci/djy181 PubMed DOI
Tack M (2023) Problems with the metablind study: an examination of data on blinding patients in trials with patient-reported outcomes. J Health Psychol 28(9):861–881. https://doi.org/10.1177/1359105321105939 PubMed DOI