Validation of a registry-derived risk algorithm based on treatment protocol as a proxy for disease risk in childhood acute lymphoblastic leukemia

BMC Medical Research Methodology

Lillian Sung²  Astrid Guttmann²  Jason D Pole¹  Sumit Gupta² 

[1] Institute for Clinical Evaluative Sciences, G1 06, 2075 Bayview Avenue, Toronto, Ontario, M4N 3M5, Canada;Department of Paediatrics and Institute for Health, Policy Management and Evaluation, University of Toronto, 155 College Street, Suite 425, Toronto, Ontario, M5T 3M6, Canada

关键词: Validation;    Registries;    Health services research;    Administrative data;    Acute lymphoblastic leukemia;   

Others  :  1109719
DOI  :  10.1186/1471-2288-13-68

 received in 2012-11-19, accepted in 2013-05-21,  发布年份 2013

【 摘 要 】

Background

Administrative databases and cancer registries are frequently used to conduct population-based research, but often lack clinical data necessary for risk stratification. Our objective was to determine the criterion validity of a risk-stratification algorithm based on treatment characteristics available from a pediatric cancer registry as a proxy for disease risk, by comparing it to traditional biology-based risk classifications.

Methods

We identified all children with acute lymphoblastic leukemia diagnosed at a single institution between January 2000 and June 2011, and linked them to a population-based cancer registry. Several risk algorithms were then constructed using disease risk variables collected through chart review by a pediatric oncologist, and compared to a risk algorithm based on treatment protocol name and age, available from the registry.

Results

Of 596 patients identified, 579 (97.1%) met inclusion criteria and were successfully linked. The registry-based algorithm showed almost perfect agreement with a biology-based algorithm based on age, initial white blood cell count, immunophenotype and cytogenetics (kappa=0.85, 95^th confidence interval 0.81-0.90). Discrepant cases were often due to the presence of unusual high risk features not captured by standard disease-risk variables but reflected in clinicians’ choices of higher intensity treatment protocols.

Conclusions

Protocol name represents a valid proxy of disease risk, allowing for risk stratification while conducting comparative effectiveness research using cancer registries and health services data. Future studies should examine the validity of treatment-based risk algorithms in other malignancies and using other treatment characteristics commonly found in health services data, such as the receipt of specific chemotherapeutic agents.

【 授权许可】

   
2013 Gupta et al.; licensee BioMed Central Ltd.

【 参考文献 】

• [1]Ellison LF, Pogany L, Mery LS: Childhood and adolescent cancer survival: a period analysis of data from the Canadian Cancer Registry. Eur J Cancer 2007, 43:1967-1975.
• [2]Pui C-H, Pei D, Pappo AS, Howard SC, Cheng C, Sandlund JT, Furman WL, Ribeiro RC, Spunt SL, Rubnitz JE, et al.: Treatment outcomes in black and white children with cancer: results from the SEER database and St. Jude Children's Research Hospital, 1992 through 2007. J Clin Oncol 2012, 30:2005-2012.
• [3]Smith MA, Seibel NL, Altekruse SF, Ries LAG, Melbert DL, O'Leary M, Smith FO, Reaman GH: Outcomes for children and adolescents with cancer: Challenges for the twenty-first century. J Clin Oncol 2010, 28:2625-2634.
• [4]Hunger SP, Lu X, Devidas M, Camitta BM, Gaynon PS, Winick NJ, Reaman GH, Carroll WL: Improved survivial for children and adolescents with acute lymphoblastic leukemia between 1990 and 2005: a report from the Children's Oncology Group. J Clin Oncol 2012, 30:1663-1669.
• [5]Aplenc R, Fisher BT, Huang YS, Li Y, Alonzo TA, Gerbing RB, Hall M, Bertoch D, Keren R, Seif AE, et al.: Merging of the national cancer institute-funded cooperative oncology group data with an administrative data source to develop a more effective platform for clinical trial analysis and comparitive effectiveness research: a report from the Children's oncology group. Pharmacoepidemiol Drug Saf 2012, 21(Suppl 2):37-43.
• [6]Walker DM, Fisher BT, Seif AE, Huang YS, Torp K, Li Y, Aplenc R: Dexrazoxane use in pediatric patients with acute lymphoblastic or myeloid leukemia from 1999 and 2009: analysis of a national cohort of patients in the pediatric health information systems database. Pediatr Blood Cancer 2012, 60(4):616-620.
• [7]Moller TR, Garwicz S, Barlow L, Winther JF, Glattre E, Olafsdottir G, Olsen JH, Perfekt R, Ritvanen A, Sankila R, et al.: Decreasing late mortality among five-year survivors of cancer in childhood and adolescence: a population-based study in the Nordic Countries. J Clin Oncol 2001, 19(13):3173-3181.
• [8]Fisher BT, Zaoutis TE, Leckerman KH, Localio R, Aplenc R: Risk factors for renal failure in pediatric patients with acute myeloid leukemia: a retrospective cohort study. Pediatr Blood Cancer 2010, 55(4):655-661.
• [9]Koch SV, Kejs AMT, Engholm G, Johansen C, Schmiegelow K: Educational attainment among survivors of childhood cancer: A population-based cohort study in Denmark. Br J Cancer 2004, 91(5):923-928.
• [10]Pui CH, Campana D, Pei D, Bowman WP, Sandlund JT, Kaste SC, Ribeiro RC, Rubnitz JE, Raimondi SC, Onciu M, et al.: Treating childhood acute lymphoblastic leukemia without cranial irradiation. N Engl J Med 2009, 360:2730-2741.
• [11]Maris JM: Recent advances in neuroblastoma. N Engl J Med 2010, 362:2202-2211.
• [12]Meyer AM, Carpenter WR, Abernethy AP, Sturmer T, Kosorok MR: Data for cancer comparative effectiveness research: Past, present, and future potential. Cancer 2012, 118:5186-5197.
• [13]Rubnitz JE, Inaba H, Dahl G, Ribeiro RC, Bowman WP, Taub J, Pounds S, Razzouk BI, Lacayo NJ, Cao X, et al.: Minimal residual disease-directed therapy for childhood acute myeloid leukemia: Results of the AML02 multicenter trial. Lancet Oncol 2010, 11(6):543-552.
• [14]Greenberg ML, Barr RD, DiMonte B, McLaughlin E, Greenberg C: Childhood cancer registries in Ontario, Canada: lessons learned from a comparison of two registries. Int J Cancer 2003, 105(1):88-91.
• [15]Arico M, Schrappe M, Hunger SP, Carroll WL, Conter V, Galimberti S, Manabe A, Saha V, Baruchel A, Vettenranta K, et al.: Clinical outcome of children with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia treated between 1995 and 2005. J Clin Oncol 2010, 28(31):4755-4761.
• [16]Heerema NA, Nachman JB, Sather HN, Sensel MG, Lee MK, Hutchinson R, Lange BJ, Steinherz PG, Bostrom B, Gaynon PS, et al.: Hypodiploidy with less than 45 chromosomes confers adverse risk in childhood acute lymphoblastic leukemia: a report from the children's cancer group. Blood 1999, 94(12):4036-4045.
• [17]Borowitz MJ, Devidas M, Hunger SP, Bowman WP, Carroll A, Carroll WL, Linda S, Martin PL, Pullen J, Viswanatha D, et al.: Clinical significance of minimal residual disease in childhood acute lymphoblastic leukemia and its relationship to other prognostic factors: a children's oncology group study. Blood 2008, 111:5477-5485.
• [18]Schrappe M, Camitta B, Pui CH: Long-term results of large prospective trials in childhood acute lymphoblastic leukemia. Leukemia 2000, 14(12):2193-2194.
• [19]Landis R, Koch GG: The measurement of observer agreement for categorical data. Biometrics 1977, 33(1):159-174.
• [20]Fleiss JL: Statistical methods for rates and proportions. 2nd edition. New York: John Wiley; 1981.
• [21]Campana D: Minimal residual disease in acute lymphoblastic leukemia. Hematology 2010, 2010(1):7-12.
• [22]Chen I-M, Harvey RC, Mullighan CG, Gastier-Foster F, Wharton W, Kang H, Borowitz MJ, Camitta BM, Carroll AJ, Devidas M, et al.: Outcome modeling with CRLF2, IKZF1, JAK, and minimal residual disease in pediatric acute lymphoblastic leukemia: a children's oncology group. Blood 2012, 119(15):3512-3522.
• [23]Moorman AV, Richards SM, Robinson HM, Strefford JC, Gibson BES, Kinsey SE, Eden TOB, Vora AJ, Mitchell CD, Harrison CJ: Prognosis of children with acute lymphoblastic leukemia (ALL) and intrachromosomal amplification of chromosome 21 (iAMP21). Blood 2007, 109(6):2327-2330.
• [24]Revesz T, Kardos G, Kajtar P, Schuler D: The adverse effect of prolonged prednisolone pretreatment in children with acute lymphoblastic leukemia. Cancer 1985, 55(8):1637-1640.
• [25]Bonilla M, Gupta S, Vasquez R, Fuentes SL, DeReyes G, Ribeiro KCB, Sung L: Predictors of outcome and methodological issues in children with acute lymphoblastic leukemia in El Salvador. Eur J Cancer 2010, 46(18):3280-3286.
• [26]Arndt CA, Rose PS, Folpe AL, Laack NN: Common musculoskeletal tumors of childhood and adolescence. Mayo Clin Proc 2012, 87(5):475-487.
• [27]Fisher BT, Harris T, Torp K, Seif AE, Shah A, Huang YS, Bailey LC, Kersun LS, Reilly AF, Rheingold SR, et al.: Establishment of an 11-year cohort of 8733 pediatric patients hospitalized at United States free-standing children's hospitals with de novo acute lymphoblastic leukemia from health care administrative data. Med Care 2012. [Epub ahead of print]
• [28]Bleyer A, Budd T, Montello M: Adolescents and young adults with cancer: the scope of the problem and criticality of clinical trials. Cancer 2006, 7(Suppl):1645-1655.
• [29]Bleyer A: The adolescent gap in cancer treatment. J Regist Manage 1996, 23:114-115.