The DOI matches its Crossref record; 24 of the 24 references it cites were found in a registry or an index.
Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival
Open accessAbstract
Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal hematopoietic disorder with increased mortality and morbidity resulting from intravascular hemolysis. Eculizumab, a monoclonal antibody against the complement protein 5, stops the intravascular hemolysis in PNH. We evaluated 79 consecutive patients treated with eculizumab in Leeds between May 2002 and July 2010. The survival of patients treated with eculizumab was not different from age- and sex-matched normal controls (P = .46) but was significantly better than 30 similar patients managed before eculizumab (P = .030). Three patients on eculizumab, all over 50 years old, died of causes unrelated to PNH. Twenty-one patients (27%) had a thrombosis before starting eculizumab (5.6 events per 100 patient-years) compared with 2 thromboses on eculizumab (0.8 events per 100 patient-years; P < .001). Twenty-one patients with no previous thrombosis discontinued warfarin on eculizumab with no thrombotic sequelae. Forty of 61 (66%) patients on eculizumab for more than 12 months achieved transfusion independence. The 12-month mean transfusion requirement reduced from 19.3 units before eculizumab to 5.0 units in the most recent 12 months on eculizumab (P < .001). Eculizumab dramatically alters the natural course of PNH, reducing symptoms and disease complications as well as improving survival to a similar level to that of the general population.
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Cites
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The Complement Inhibitor Eculizumab in Paroxysmal Nocturnal Hemoglobinuria
Hillmen, P., Young, N. S., Schubert, J., Brodsky, R. A., Socié, G., Muus, P., Röth, A., Szer, J., Elebute, M. O., Nakamura, R., et al. (2006) · New England Journal of Medicine · doi:10.1056/nejmoa061648 · Cited by 1,303
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Natural History of Paroxysmal Nocturnal Hemoglobinuria
Hillmen, P., Lewis, S. M., Bessler, M., Luzzatto, L., & Dacie, J. V. (1995) · New England Journal of Medicine · doi:10.1056/nejm199511093331904 · Cited by 907
Self-citationDiagnosis and management of paroxysmal nocturnal hemoglobinuria
Parker, C. (2005) · Blood · doi:10.1182/blood-2005-04-1717 · Cited by 783
Brodsky, R. A., Young, N. S., Antonioli, E., Risitano, A. M., Schrezenmeier, H., Schubert, J., Gaya, A., Coyle, L., de Castro, C., Fu, C.-L., et al. (2008) · Blood · doi:10.1182/blood-2007-06-094136 · Cited by 652
Self-citationHillmen, P., Hall, C., Marsh, J. C. W., Elebute, M., Bombara, M. P., Petro, B. E., Cullen, M. J., Richards, S. J., Rollins, S. A., Mojcik, C. F., et al. (2004) · New England Journal of Medicine · doi:10.1056/nejmoa031688 · Cited by 635
Self-citationHillmen, P., Muus, P., Dührsen, U., Risitano, A. M., Schubert, J., Luzzatto, L., Schrezenmeier, H., Szer, J., Brodsky, R. A., Hill, A., et al. (2007) · Blood · doi:10.1182/blood-2007-06-095646 · Cited by 579
Self-citationParoxysmal nocturnal haemoglobinuria: long-term follow-up and prognostic factors
Socié, G., Mary, J.-Y., de Gramont, A., Rio, B., Leporrier, M., Rose, C., Heudier, P., Rochant, H., Cahn, J.-Y., & Gluckman, E. (1996) · The Lancet · doi:10.1016/s0140-6736(95)12360-1 · Cited by 518
Cited by
Paroxysmal nocturnal hemoglobinuria
Brodsky, R. A. (2014) · Blood · doi:10.1182/blood-2014-02-522128 · Cited by 648
Thrombosis in paroxysmal nocturnal hemoglobinuria
Hill, A., Kelly, R. J., & Hillmen, P. (2013) · Blood · doi:10.1182/blood-2012-09-311381 · Cited by 478
Self-citationParoxysmal nocturnal haemoglobinuria
Hill, A., DeZern, A. E., Kinoshita, T., & Brodsky, R. A. (2017) · Nature Reviews Disease Primers · doi:10.1038/nrdp.2017.28 · Cited by 448
Self-citationPegcetacoplan versus eculizumab in paroxysmal nocturnal hemoglobinuria
Hillmen, P., Szer, J., Weitz, I., Röth, A., Höchsmann, B., Panse, J., Usuki, K., Griffin, M., Kiladjian, J.-J., de Castro, C., et al. (2021) · New England Journal of Medicine · doi:10.1056/nejmoa2029073 · Cited by 440
Self-citationKulasekararaj, A. G., Hill, A., Rottinghaus, S. T., Langemeijer, S., Wells, R., Gonzalez-Fernandez, F. A., Gaya, A., Lee, J. W., Gutierrez, E. O., Piatek, C. I., et al. (2019) · Blood · doi:10.1182/blood-2018-09-876805 · Cited by 382
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Metadata
- Authors
- Kelly, R. J., Hill, A., Arnold, L. M., Brooksbank, G. L., Richards, S. J., Cullen, M., Mitchell, L. D., Cohen, D. R., Gregory, W. M., & Hillmen, P.
- Venue
- Blood
- Publisher
- American Society of Hematology
- Published
- 2011-06-23
- Volume
- 117
- Issue
- 25
- Pages
- 6786-6792
- ISSN
- 0006-4971 (ISSN-L)
- 1528-0020
- OpenAlex
- W2171068466
- Cited by
- 526
- References
- 24
- FWCI
- 13.13
- Type
- journal article
- Language
- en
- Open access
- bronze
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- Kelly, R. J., Hill, A., Arnold, L. M., Brooksbank, G. L., Richards, S. J., Cullen, M., Mitchell, L. D., Cohen, D. R., Gregory, W. M., & Hillmen, P. (2011). Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival. Blood. https://doi.org/10.1182/blood-2011-02-333997
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
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