The DOI matches its Crossref record; 18 of the 18 references it cites were found in a registry or an index.
Survival in Medically Treated Patients with Homozygous β-Thalassemia
Open accessAbstract
BACKGROUND: The prognosis of patients with homozygous beta-thalassemia (thalassemia major) has been improved by transfusion and iron-chelation therapy. We analyzed outcome and prognostic factors among patients receiving transfusions and chelation therapy who had reached the age at which iron-induced cardiac disease, the most common cause of death, usually occurs. METHODS: Using the duration of life without the need for either inotropic or antiarrhythmic drugs as a measure of survival without cardiac disease, we studied 97 patients born before 1976 who were treated with regular transfusions and chelation therapy. We used Cox proportional-hazards analysis to assess the effect of prognostic factors and life-table analysis to estimate freedom from cardiac disease over time. RESULTS: Of the 97 patients, 59 (61 percent) had no cardiac disease; 36 (37 percent) had cardiac disease, and 18 of them had died. Univariate analysis demonstrated that factors affecting cardiac disease-free survival were age at the start of chelation therapy (P < 0.001), the natural log of the serum ferritin concentration before chelation therapy began (P = 0.01), the mean ferritin concentration (P < 0.001), and the proportion of ferritin measurements exceeding 2500 ng per milliliter (P < 0.001). With stepwise Cox modeling, only the proportion of ferritin measurements exceeding 2500 ng per milliliter affected cardiac disease-free survival (P < 0.001). Patients in whom less than 33 percent of the serum ferritin values exceeded 2500 ng per milliliter had estimated rates of survival without cardiac disease of 100 percent after 10 years of chelation therapy and 91 percent after 15 years. CONCLUSIONS: The prognosis for survival without cardiac disease is excellent for patients with thalassemia major who receive regular transfusions and whose serum ferritin concentrations remain below 2500 ng per milliliter with chelation therapy.
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Cites
Bone Marrow Transplantation in Patients with Thalassemia
Lucarelli, G., Galimberti, M., Polchi, P., Angelucci, E., Baronciani, D., Giardini, C., Politi, P., Durazzi, S. M. T., Muretto, P., & Albertini, F. (1990) · New England Journal of Medicine · doi:10.1056/nejm199002153220701 · Cited by 636
SURVIVAL AND CAUSES OF DEATH IN THALASSAEMIA MAJOR
Zurlo, M., De Stefano, P., Borgna-Pignatti, C., Di Palma, A., Melevendi, C., Piga, A., Di Gregorio, F., Burattini, M., & Terzoli, S. (1989) · The Lancet · doi:10.1016/s0140-6736(89)90264-x · Cited by 611
Addison, G. M., Beamish, M. R., Hales, C. N., Hodgkins, M., Jacobs, A., & Llewellin, P. (1972) · Journal of Clinical Pathology · doi:10.1136/jcp.25.4.326 · Cited by 595
Brahams, D. (1987) · The Lancet · doi:10.1016/s0140-6736(87)90821-x · Cited by 533
Visual and Auditory Neurotoxicity in Patients Receiving Subcutaneous Deferoxamine Infusions
Olivieri, N. F., Buncic, J. R., Chew, E., Gallant, T., Harrison, R. V., Keenan, N., Logan, W., Mitchell, D., Ricci, G., Skarf, B., et al. (1986) · New England Journal of Medicine · doi:10.1056/nejm198604033141402 · Cited by 429
Self-citationMagnetic-Susceptibility Measurement of Human Iron Stores
Brittenham, G. M., Farrell, D. E., Harris, J. W., Feldman, E. S., Danish, E. H., Muir, W. A., Tripp, J. H., & Bellon, E. M. (1982) · New England Journal of Medicine · doi:10.1056/nejm198212303072703 · Cited by 367
MARROW TRANSPLANTATION FOR THALASSAEMIA
Donnall Thomas, E., Sanders, J. E., Buckner, C. D., Papayannopoulou, T., Borgna-Pignatti, C., De Stefano, P., Clift, R., Sullivan, K., & Storb, R. (1982) · The Lancet · doi:10.1016/s0140-6736(82)90319-1 · Cited by 340
Brittenham, G. M., Cohen, A. R., McLaren, C. E., Martin, M. B., Griffith, P. M., Nienhuis, A. W., Young, N. S., Allen, C. J., Farrell, D. E., & Harris, J. W. (1993) · American Journal of Hematology · doi:10.1002/ajh.2830420116 · Cited by 270
Self-citationPrevention of Cardiac Disease by Subcutaneous Deferoxamine in Patients with Thalassemia Major
Wolfe, L., Olivieri, N., Sallan, D., Colan, S., Rose, V., Propper, R., Freedman, M. H., & Nathan, D. G. (1985) · New England Journal of Medicine · doi:10.1056/nejm198506203122503 · Cited by 265
Self-citationContinuous Subcutaneous Administration of Deferoxamine in Patients with Iron Overload
Propper, R. D., Cooper, B., Rufo, R. R., Nienhuis, A. W., Anderson, W. F., Bunn, H. F., Rosenthal, A., & Nathan, D. G. (1977) · New England Journal of Medicine · doi:10.1056/nejm197708252970804 · Cited by 258
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Cited by
Taher, A. T., Weatherall, D. J., & Cappellini, M. D. (2018) · The Lancet · doi:10.1016/s0140-6736(17)31822-6 · Cited by 807
Oudit, G. Y., Sun, H., Trivieri, M. G., Koch, S. E., Dawood, F., Ackerley, C., Yazdanpanah, M., Wilson, G. J., Schwartz, A., Liu, P. P., et al. (2003) · Nature Medicine · doi:10.1038/nm920 · Cited by 480
Self-citationArmand, P., Kim, H. T., Cutler, C. S., Ho, V. T., Koreth, J., Alyea, E. P., Soiffer, R. J., & Antin, J. H. (2007) · Blood · doi:10.1182/blood-2006-10-054924 · Cited by 426
Long-Term Safety and Effectiveness of Iron-Chelation Therapy with Deferiprone for Thalassemia Major
Olivieri, N. F., Brittenham, G. M., McLaren, C. E., Templeton, D. M., Cameron, R. G., McClelland, R. A., Burt, A. D., & Fleming, K. A. (1998) · New England Journal of Medicine · doi:10.1056/nejm199808133390701 · Cited by 420
Self-citationSafety and effectiveness of long-term therapy with the oral iron chelator deferiprone
Cohen, A. R., Galanello, R., Piga, A., De Sanctis, V., & Tricta, F. (2003) · Blood · doi:10.1182/blood-2002-10-3280 · Cited by 323
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Metadata
- Authors
- Olivieri, N. F., Nathan, D. G., MacMillan, J. H., Wayne, A. S., Liu, P. P., McGee, A., Martin, M., Koren, G., & Cohen, A. R.
- Venue
- New England Journal of Medicine
- Publisher
- Massachusetts Medical Society
- Published
- 1994-09
- Volume
- 331
- Issue
- 9
- Pages
- 574-578
- ISSN
- 0028-4793 (ISSN-L)
- 1533-4406
- OpenAlex
- W2317072972
- Cited by
- 852
- References
- 18
- FWCI
- 10.92
- Type
- journal article
- Language
- en
- Open access
- bronze
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