The incidence of leukemia and mortality from sepsis in patients with severe congenital neutropenia receiving long-term G-CSF therapy

Blood

Volumn 107, Issue 12, 2006, Pages 4628-4635

  Rosenberg, Philip S ^a   Alter, Blanche P ^b   Bolyard, Audrey A ^b   Bonilla, Mary Ann ^b   Boxer, Laurence A ^b   Cham, Bonnie ^b   Fier, Carol ^b   Freedman, Melvin ^b   Kannourakis, George ^b   Kinsey, Sally ^b   Schwinzer, Beate ^b   Zeidler, Connie ^b   Welte, Karl ^b   Dale, David C ^b  

^a NATIONAL CANCER INSTITUTE   (United States)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

GRANULOCYTE COLONY STIMULATING FACTOR;

ACUTE GRANULOCYTIC LEUKEMIA; ARTICLE; CONTROLLED STUDY; HUMAN; HUMAN CELL; INCIDENCE; INTERNATIONAL COOPERATION; LONG TERM CARE; MAJOR CLINICAL STUDY; MORTALITY; NEUTROPENIA; NEUTROPHIL COUNT; PRIORITY JOURNAL; SEPSIS; SHWACHMAN SYNDROME; TREATMENT RESPONSE;

EID: 33745096897     PISSN: 00064971     EISSN: 00064971     Source Type: Journal    
DOI: 10.1182/blood-2005-11-4370     Document Type: Article

References (37)

1. Zeidler C, Welte K. Kostmann syndrome and severe congenital neutropenia. Semin Hematol. 2002;39:82-88.
2. Alter BP. Inherited bone marrow failure syndromes. In: Nathan DG, Orkin SH, Look AT, Ginsburg D, eds. Hematology of Infancy and Childhood. Philadelphia: WB Saunders Inc; 2003:280-365.
3. Miller RW. Childhood cancer and congenital defects: a study of U.S. death certificates during the period 1960-1966. Pediatr Res. 1969;3:389-397.
4. Gilman PA, Jackson DP, Guild HG. Congenital agranulocytosis: prolonged survival and terminal acute leukemia. Blood. 1970;36:576-585.
5. Rosen RB, Kang SJ. Congenital agranulocytosis terminating in acute myelomonocytic leukemia. J Pediatr. 1979;94:406-408.
6. Wong WY, Williams D, Slovak ML, et al. Terminal acute myelogenous leukemia in a patient with congenital agranulocytosis. Am J Hematol. 1993;43:133-138.
7. Mack DR, Forstner GG, Wilschanski M, Freedman MH, Durie PR. Shwachman syndrome: exocrine pancreatic dysfunction and variable phenotypic expression. Gastroenterology. 1996;111:1593-1602.
8. Dale DC, Bonilla MA, Davis MW, et al. A randomized controlled phase III trial of recombinant human granulocyte colony-stimulating factor (filgrastim) for treatment of severe chronic neutropenia. Blood. 1993;81:2496-2502.
9. Dale DC, Cottle TE, Fier CJ, et al. Severe chronic neutropenia: treatment and follow-up of patients in the Severe Chronic Neutropenia International Registry. Am J Hematol. 2003;72:82-93.
10. Freedman MH, Bonilla MA, Fier C, et al. Myelodysplasia syndrome and acute myeloid leukemia in patients with congenital neutropenia receiving G-CSF therapy. Blood. 2000;96:429-436.
11. Donadieu J, Leblanc T, Bader MB, et al. Analysis of risk factors for myelodysplasias, leukemias and death from infection among patients with congenital neutropenia: experience of the French Severe Chronic Neutropenia Study Group. Haematologica. 2005;90:45-53.
12. Ancliff PJ. Congenital neutropenia. Blood Rev. 2003;17:209-216.
13. Gaynor JJ, Feuer EJ, Tan CC, et al. On the use of cause-specific failure and conditional failure probabilities: examples from clinical oncology data. J Am Stat Assoc. 1993;88:400-409.
14. Rosenberg PS. Hazard function estimation using B-splines. Biometrics. 1995;51:874-887.
15. Cox DR. Regression models and life-tables. J R Stat Soc Ser B Methol. 1972;34:187-220.
16. McCullagh P, Nelder JA. Generalized Linear Models. London: Chapman and Hall; 1983.
17. Hastie T, Tibshirani R, Friedman J. The Elements of Statistical Learning: Data Mining, Inference, and Prediction. New York, NY: Springer-Verlag; 2002.
18. Wilcoxon F. Individual comparisons by ranking methods. Biometrics. 1945;1:80-83.
19. Liddell FD. Simple exact analysis of the standardised mortality ratio. J Epidemiol Community Health. 1984;38:85-88.
20. Banerjee A, Shannon KM. Leukemic transformation in patients with severe congenital neutropenia. J Pediatr Hematol Oncol. 2001;23:487-495.
21. Dale DC, Person RE, Bolyard AA, et al. Mutations in the gene encoding neutrophil elastase in congenital and cyclic neutropenia. Blood. 2000;96:2317-2322.
22. Boocock GR, Morrison JA, Popovic M, et al. Mutations in SBDS are associated with Shwachman-Diamond syndrome. Nat Genet. 2003;33:97-101.
23. Dong F, Brynes RK, Tidow N, et al. Mutations in the gene for the granulocyte colony-stimulating-factor receptor in patients with acute myeloid leukemia preceded by severe congenital neutropenia. N Engl J Med. 1995;333:487-493.
24. Tidow N, Pilz C, Kasper B, Welte K. Frequency of point mutations in the gene for the G-CSF receptor in patients with chronic neutropenia undergoing G-CSF therapy. Stem Cells. 1997;15:113-119.
25. Tidow N, Kasper B, Welte K. Clinical implications of G-CSF receptor mutations. Crit Rev Oncol Hematol. 1998;28:1-6.
26. Ward AC, van Aesch YM, Schelen AM, Touw IP. Defective internalization and sustained activation of truncated granulocyte colony-stimulating factor receptor found in severe congenital neutropenia/acute myeloid leukemia. Blood. 1999;93:447-458.
27. Hunter MG, Avalos BR. Deletion of a critical internalization domain in the G-CSFR in acute myelogenous leukemia preceded by severe congenital neutropenia. Blood. 1999;93:440-446.
28. Hunter MG, Avalos BR. Granulocyte colony-stimulating factor receptor mutations in severe congenital neutropenia transforming to acute myelogenous leukemia confer resistance to apoptosis and enhance cell survival. Blood. 2000;95:2132-2137.
29. Hermans MH, Ward AC, Antonissen C, et al. Perturbed granulopoiesis in mice with a targeted mutation in the granulocyte colony-stimulating factor receptor gene associated with severe chronic neutropenia. Blood. 1998;92:32-39.
30. Hermans MH, Antonissen C, Ward AC, et al. Sustained receptor activation and hyperproliferation in response to granulocyte colony-stimulating factor (G-CSF) in mice with a severe congenital neutropenia/acute myeloid leukemia-derived mutation in the G-CSF receptor gene. J Exp Med. 1999;189:683-692.
31. Bernard T, Gale RE, Evans JP, Linch DC. Mutations of the granulocyte-colony stimulating factor receptor in patients with severe congenital neutropenia are not required for transformation to acute myeloid leukaemia and may be a bystander phenomenon. Br J Haematol. 1998;101:141-149.
32. Ancliff PJ, Gale RE, Liesner R, Hann I, Linch DC. Long-term follow-up of granulocyte colony-stimulating factor receptor mutations in patients with severe congenital neutropenia: implications for leukaemogenesis and therapy. Br J Haematol. 2003;120:685-690.
33. Tschan CA, Pilz C, Zeidler C, Welte K, Germeshausen M. Time course of increasing numbers of mutations in the granulocyte colony-stimulating factor receptor gene in a patient with congenital neutropenia who developed leukemia. Blood. 2001;97:1882-1884.
34. Bellanne-Chantelot C, Clauin S, Leblanc T, et al. Mutations in the ELA2 gene correlate with more severe expression of neutropenia: a study of 81 patients from the French Neutropenia Register. Blood. 2004;103:4119-4125.
35. Choi SW, Boxer LA, Pulsipher MA, et al. Stem cell transplantation in patients with severe congenital neutropenia with evidence of leukemic transformation. Bone Marrow Transplant. 2005;35:473-477.
36. Zeidler C, Welte K, Barak Y, et al. Stem cell transplantation in patients with severe congenital neutropenia without evidence of leukemic transformation. Blood. 2000;95:1195-1198.
37. Ferry C, Ouachee M, Leblanc T, et al. Hematopoietic stem cell transplantation in severe congenital neutropenia: experience of the French SCN register. Bone Marrow Transplant. 2005;35:45-50.