Contribution Factors on Long- term and Short- term Survival of Thalassemia Major Patients
Abstract
Background: Thalassemia major is an important health problem in Mediterranean countries, placing a tremendous emotional, psychological, and economic burden. This study aimed to evaluate the effective factors on long- and short-term survival of thalassemia major patients using mixture and non-mixture cure survival models based on generalized-Gompertz distribution. The generalized-Gompertz distribution has flexible curve of failure rate that may be appropriate for different situation of survival analysis.
Methods: In this retrospective cohort study, medical records of 300 thalassemia major patients referring to Zafar's thalassemia clinic during 1994-2017 in Tehran, Iran were reviewed. Mixture and non-mixture cure survival models based on Gompertz and generalized-Gompertz distributions were performed to investigate the survival of thalassemia major patients and the influential factors in their survival and longevity. The Akaike Information Criteria was used to compare the models. Analysis was performed using SAS software version 9.4.
Results: The mean survival time was 32.21 ± 7.47years. The censorship rate was 78.3%. In both of the mixture and non-mixture cure models, generalized Gompertz distribution, as compared to the standard Gompertz had the lower Akaike (AIC) criteria that was 200.8. Based on this model, iron deposition in liver at mild and moderate levels had a significant effect on the long-term survival of these patients.
Conclusion: Based on Akaike Information criteria, considering the generalized Gompertz mixture cure model has the best fit for the data of thalassemia major disease in which patients are long-term survivors. In order to analyze the survival of patients with thalassemia major, since iron deposition in liver at mild and moderate levels had a significant effect on the long-term survival of these patients; it is recommended to regular iron chelation therapy for extra iron extraction.
2.Surapolchai P, Satayasai W, Sinlapamongkolkul P, Udomsubpayakul U. Biopsychosocial predictors of health-related quality of life in children with thalassemia in Thammasat University Hospital. J Med Assoc Thai. 2010;93(Suppl 7):65-75.
3.Caocci G, Efficace F, Ciotti F, Roncarolo MG, Vacca A, Piras E, et al. Prospective assessment of health-related quality of life in pediatric patients with beta-thalassemia following hematopoietic stem cell transplantation. Biology of Blood and Marrow Transplantation. 2011;17(6):861-6.
4.Jafari H, Lahsaeizadeh S, Jafari P, Karimi M. Quality of life in thalassemia major: reliability and validity of the Persian version of the SF-36 questionnaire. Journal of postgraduate medicine. 2008;54(4):273.
5.Brittenham GM, Griffith PM, Nienhuis AW, McLaren CE, Young NS, Tucker EE, et al. Efficacy of deferoxamine in preventing complications of iron overload in patients with thalassemia major. New England Journal of Medicine. 1994;331(9):567-73.
6.Jensen C, Tuck S, Agnew J, Koneru S, Morris R, Yardumian A, et al. High prevalence of low bone mass in thalassaemia major. British Journal of Haematology. 1998;103(4):911-5.
7.Zurlo M, De Stefano P, Borgna-Pignatti C, Di Palma A, Melevendi C, Piga A, et al. Survival and causes of death in thalassaemia major. The Lancet. 1989;334(8653):27-30.
8.Modell B, Berdoukas V. The clinical approach to thalassaemia. GRUNE AND STRATTON, NEW YORK, NY (USA) 1983. 1984.
9.Akbari Khoei AA, Bakhshi E, Azarkeivan A, Biglarian A. Application of cure models in survival analysis of thalassemia major disease. Razi J Med Sci; 2015.22(131):71-9.
10.Ladis V, Chouliaras G, Berdousi H, Kanavakis E, Kattamis C. Longitudinal study of survival and causes of death in patients with thalassemia major in Greece. Annals of the New York Academy of Sciences. 2005;1054(1):445-50.
11.Wonke B, editor Clinical management of β-thalassemia major. Seminars in hematology; 2001: Elsevier.
12.Azarkeivan A, Hajibeigi B, Alavian SM, Lankarani MM, Assari S. Associates of poor physical and mental health-related quality of life in beta thalassemia-major/intermedia. Journal of research in medical sciences: the official journal of Isfahan University of Medical Sciences. 2009;14(6):349.
13.Dubey A, Parakh A, Dublish S. Current trends in the management of beta thalassemia. The Indian Journal of Pediatrics. 2008;75(7):739.
14.Pakbaz Z, Treadwell M, Yamashita R, Quirolo K, Foote D, Quill L, et al. Quality of life in patients with thalassemia intermedia compared to thalassemia major. Annals of the New York Academy of Sciences. 2005;1054(1):457-61.
15.Kleinbaum DG, Klein M. Survival analysis: Springer; 2010.
16.Henderson R. Problems and prediction in survival‐data analysis. Statistics in medicine. 1995;14(2):161-84.
17.Swain PK, Grover G, Goel K. Mixture and non-mixture cure fraction models based on Generalized gompertz distribution under Bayesian Approach. Tatra Mountains Mathematical Publications. 2016;66.
18.Martinez EZ, Achcar JA, Jácome AA, Santos JS. Mixture and non-mixture cure fraction models based on the Generalized modified Weibull distribution with an application to gastric cancer data. Computer methods and programs in biomedicine. 2013;112(3):343-55.
19.Boag JW. The presentation and analysis of the results of radiotherapy. Part II. The British journal of radiology. 1948;21(244):189-203.
20.Efthimiadis GK, Hassapopoulou HP, Tsikaderis DD, Karvounis HI, Giannakoulas GA, Parharidis GE, et al. Survival in thalassaemia major patients. Circulation Journal. 2006;70(8):1037-42.
21.Martinez EZ, Achcar JA. The defective Generalized Gompertz distribution and its use in the analysis of lifetime data in presence of cure fraction, censored data and covariates. Electronic Journal of Applied Statistical Analysis. 2017;10(2):463-84.
22.Corbière F, Joly P. A SAS macro for parametric and semiparametric mixture cure models. Computer methods and programs in biomedicine. 2007;85(2):173-80.
23.Maggio A, Vitrano A, Capra M, Cuccia L, Gagliardotto F, Filosa A, et al. Improving survival with deferiprone treatment in patients with thalassemia major: a prospective multicenter randomised clinical trial under the auspices of the Italian Society for Thalassemia and Hemoglobinopathies. Blood Cells, Molecules, and Diseases. 2009;42(3):247-51.
Files | ||
Issue | Vol 7 No 4 (2021) | |
Section | Original Article(s) | |
DOI | https://doi.org/10.18502/jbe.v7i4.10394 | |
Keywords | ||
Thalassemia Major Survival Analysis Mixture Cure Model Non-Mixture Cure Model Generalized Gompertz Statistical Model |
Rights and permissions | |
This work is licensed under a Creative Commons Attribution-NonCommercial 4.0 International License. |