William R Lumry1, Timothy Craig2, Bruce Zuraw3, Hilary Longhurst4, James Baker5, H Henry Li6, Jonathan A Bernstein7, John Anderson8, Marc A Riedl9, Michael E Manning10, Paul K Keith11, Donald S Levy12, Teresa Caballero13, Aleena Banerji14, Richard G Gower15, Henriette Farkas16, John-Philip Lawo17, Ingo Pragst17, Thomas Machnig17, Douglas J Watson18. 1. Allergy and Asthma Research Associates Research Center, Dallas, Tex. Electronic address: LumryMD@allergyspecialists.us. 2. Department of Medicine and Pediatrics, Penn State University, Hershey, Pa. 3. San Diego School of Medicine, University of California, San Diego, La Jolla, Calif; San Diego Veterans Administration Healthcare System, San Diego, Calif. 4. Addenbrooke's Hospital, Cambridge University Hospital NHS Foundation Trust, Hills Road, Cambridge, UK. 5. Baker Allergy Asthma Dermatology, Portland, Ore. 6. Institute for Asthma and Allergy, Chevy Chase, Md. 7. Department of Internal Medicine, University of Cincinnati College of Medicine, Cincinnati, Ohio; Bernstein Clinical Research Center, Cincinnati, Ohio. 8. Clinical Research Center of Alabama, Birmingham, Ala. 9. San Diego School of Medicine, University of California, San Diego, La Jolla, Calif. 10. Allergy, Asthma & Immunology Associates, Ltd, Scottsdale, Ariz. 11. Department of Medicine, McMaster University, Hamilton, Ontario, Canada. 12. Allergy & Immunology Services, University of California, Irvine, Calif. 13. Hospital La Paz Health Research Institute (IdiPaz), CIBERER U754, Madrid, Spain. 14. Division of Rheumatology, Department of Allergy & Immunology, Massachusetts General Hospital, Boston, Mass. 15. Marycliff Clinical Research, Spokane, Wash. 16. Hungarian Angioedema Center, 3rd Department of Internal Medicine, Semmelweis University, Budapest, Hungary. 17. CSL Behring, Marburg, Germany. 18. CSL Behring, King of Prussia, Pa.
Abstract
BACKGROUND: Hereditary angioedema with C1-inhibitor deficiency (C1-INH-HAE) impairs health-related quality of life (HRQoL). OBJECTIVE: The objective of this study was to assess HRQoL outcomes in patients self-administeringsubcutaneous C1-INH (C1-INH[SC]; HAEGARDA) for routine prevention of HAE attacks. METHODS: Post hoc analysis of data from the placebo-controlled, crossover phase III COMPACT study (Clinical Studies for Optimal Management of Preventing Angioedema with Low-Volume Subcutaneous C1-Inhibitor Replacement Therapy). Ninety patients with C1-INH-HAE were randomized to 1 of 4 treatment sequences: C1-INH(SC) 40 or 60 IU/kg twice weekly for 16 weeks, preceded or followed by 16 weeks of twice weekly placebo injections. All HAE attacks were treated with open-label on-demand treatment as necessary. HRQoL assessments at week 14 (last visit) included the European Quality of Life-5 Dimensions Questionnaire (EQ-5D-3L), the Hospital Anxiety and Depression Scale (HADS), the Work Productivity and Activity Impairment Questionnaire (WPAI), and the Treatment Satisfaction Questionnaire for Medication (TSQM). RESULTS: Compared with placebo (on-demand treatment alone), treatment with twice weekly C1-INH(SC) (both doses combined) was associated with better EQ-5D visual analog scale general health, less HADS anxiety, less WPAI presenteeism, work productivity loss, and activity impairment, and greater TSQM effectiveness and overall treatment satisfaction. More patients self-reported a "good/excellent" response during routine prevention with C1-INH(SC) compared with on-demand only (placebo prophylaxis) management. For each HRQoL measure, a greater proportion of patients had a clinically meaningful improvement during C1-INH(SC) treatment compared with placebo. CONCLUSIONS: In patients with frequent HAE attacks, a treatment strategy of routine prevention with self-administered twice weekly C1-INH(SC) had a greater impact on improving multiple HAE-related HRQoL impairments, most notably anxiety and work productivity, compared with on-demand treatment alone (placebo prophylaxis).
RCT Entities:
BACKGROUND:Hereditary angioedema with C1-inhibitor deficiency (C1-INH-HAE) impairs health-related quality of life (HRQoL). OBJECTIVE: The objective of this study was to assess HRQoL outcomes in patients self-administering subcutaneous C1-INH (C1-INH[SC]; HAEGARDA) for routine prevention of HAE attacks. METHODS: Post hoc analysis of data from the placebo-controlled, crossover phase III COMPACT study (Clinical Studies for Optimal Management of Preventing Angioedema with Low-Volume Subcutaneous C1-Inhibitor Replacement Therapy). Ninety patients with C1-INH-HAE were randomized to 1 of 4 treatment sequences: C1-INH(SC) 40 or 60 IU/kg twice weekly for 16 weeks, preceded or followed by 16 weeks of twice weekly placebo injections. All HAE attacks were treated with open-label on-demand treatment as necessary. HRQoL assessments at week 14 (last visit) included the European Quality of Life-5 Dimensions Questionnaire (EQ-5D-3L), the Hospital Anxiety and Depression Scale (HADS), the Work Productivity and Activity Impairment Questionnaire (WPAI), and the Treatment Satisfaction Questionnaire for Medication (TSQM). RESULTS: Compared with placebo (on-demand treatment alone), treatment with twice weekly C1-INH(SC) (both doses combined) was associated with better EQ-5D visual analog scale general health, less HADS anxiety, less WPAI presenteeism, work productivity loss, and activity impairment, and greater TSQM effectiveness and overall treatment satisfaction. More patients self-reported a "good/excellent" response during routine prevention with C1-INH(SC) compared with on-demand only (placebo prophylaxis) management. For each HRQoL measure, a greater proportion of patients had a clinically meaningful improvement during C1-INH(SC) treatment compared with placebo. CONCLUSIONS: In patients with frequent HAE attacks, a treatment strategy of routine prevention with self-administered twice weekly C1-INH(SC) had a greater impact on improving multiple HAE-related HRQoL impairments, most notably anxiety and work productivity, compared with on-demand treatment alone (placebo prophylaxis).
Authors: Anthony J Castaldo; Christian Jervelund; Deborah Corcoran; Henrik B Boysen; Sandra C Christiansen; Bruce L Zuraw Journal: Allergy Asthma Proc Date: 2021-02-13 Impact factor: 2.587
Authors: William R Lumry; Karsten Weller; Markus Magerl; Aleena Banerji; Hilary J Longhurst; Marc A Riedl; Hannah B Lewis; Peng Lu; Giovanna Devercelli; Gagan Jain; Marcus Maurer Journal: Allergy Date: 2020-12-24 Impact factor: 13.146
Authors: Marcus Maurer; Markus Magerl; Stephen Betschel; Werner Aberer; Ignacio J Ansotegui; Emel Aygören-Pürsün; Aleena Banerji; Noémi-Anna Bara; Isabelle Boccon-Gibod; Konrad Bork; Laurence Bouillet; Henrik Balle Boysen; Nicholas Brodszki; Paula J Busse; Anette Bygum; Teresa Caballero; Mauro Cancian; Anthony J Castaldo; Danny M Cohn; Dorottya Csuka; Henriette Farkas; Mark Gompels; Richard Gower; Anete S Grumach; Guillermo Guidos-Fogelbach; Michihiro Hide; Hye-Ryun Kang; Allen P Kaplan; Constance H Katelaris; Sorena Kiani-Alikhan; Wei-Te Lei; Richard F Lockey; Hilary Longhurst; William Lumry; Andrew MacGinnitie; Alejandro Malbran; Inmaculada Martinez Saguer; Juan José Matta Campos; Alexander Nast; Dinh Nguyen; Sandra A Nieto-Martinez; Ruby Pawankar; Jonathan Peter; Grzegorz Porebski; Nieves Prior; Avner Reshef; Marc Riedl; Bruce Ritchie; Farrukh Rafique Sheikh; William B Smith; Peter J Spaeth; Marcin Stobiecki; Elias Toubi; Lilian Agnes Varga; Karsten Weller; Andrea Zanichelli; Yuxiang Zhi; Bruce Zuraw; Timothy Craig Journal: World Allergy Organ J Date: 2022-04-07 Impact factor: 5.516
Authors: John Anderson; Donald S Levy; William Lumry; Patricia Koochaki; Sally Lanar; H Henry Li Journal: Allergy Asthma Clin Immunol Date: 2021-06-27 Impact factor: 3.406