Myelodysplasia syndrome and acute myeloid leukemia in patients with congenital neutropenia receiving G-CSF therapy

Blood

Volumn 96, Issue 2, 2000, Pages 429-436

  Freedman, Melvin H ^a   Bonilla, Mary Ann ^b   Fier, Carol ^b   Bolyard, Audrey Anna ^b   Scarlata, Debra ^b   Boxer, Laurence A ^b   Brown, Sherri ^b   Cham, Bonnie ^b   Kannourakis, George ^b   Kinsey, Sally E ^b   Mori, Pier Georgio ^b   Cottle, Tammy ^b   Welte, Karl ^b   Dale, David C ^b  

^a HOSPITAL FOR SICK CHILDREN   (Canada)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

GRANULOCYTE COLONY STIMULATING FACTOR;

ACUTE GRANULOCYTIC LEUKEMIA; ADOLESCENT; ADULT; ARTICLE; CANCER SUSCEPTIBILITY; CHILD; CHROMOSOME 7; CHROMOSOME 9; CHROMOSOME LOSS; CLINICAL ARTICLE; CYTOGENETICS; FEMALE; HUMAN; INFANT; MALE; MALIGNANT TRANSFORMATION; MYELODYSPLASTIC SYNDROME; NEUTROPENIA; PATHOGENESIS; PRIORITY JOURNAL; TRISOMY 21;

ADOLESCENT; ADULT; AGING; CELL TRANSFORMATION, NEOPLASTIC; CHILD; CHILD, PRESCHOOL; FEMALE; GRANULOCYTE COLONY-STIMULATING FACTOR; HUMANS; INFANT; LEUKEMIA, MYELOCYTIC, ACUTE; MALE; MIDDLE AGED; MYELODYSPLASTIC SYNDROMES; NEUTROPENIA; TIME FACTORS;

EID: 0011982978     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Article

References (35)

1. Kostmann R. Infantile genetic agranulocytosis: a new recessive lethal disease in man. Acta Paediatr Scand. 1956;45(suppl 105):1-368.
2. Kostmann R. Infantile genetic agranulocytosis: a review with presentation of ten new cases. Acta Paedatr Scand. 1975;64:362-368.
3. Shwachman H, Diamond LK, Oski FA, Khaw K-T. The syndrome of pancreatic insufficiency and bone marrow dysfunction. J Pediatr. 1964;65:645-663.
4. De Vries A, Peketh L, Joshua H. Leukemia and agranulocytosis in a family with hereditary leukopenia. Acta Med Orientalia. 1958;17:26-32.
5. Gilman PA, Jackson DP, Guild HG. Congenital agranulocytosis: prolonged survival and terminal acute leukemia. Blood. 1970;36:576-585.
6. Wong W, Williams D, Slovak M, et al. Terminal acute myelogenous leukemia in a patient with congenital agranulocytosis. Am J Hematol. 1993; 43:133-138.
7. Rosen RB, Kang SJ. Congenital agranulocytosis terminating in acute myelomonocytic leukemia. J Pediat. 1979;94:406-408.
8. Mack DR, Forstner GG, Wilschanski M, Freedman MH, Durie PR. Shwachman syndrome: exocrine pancreatic dysfunction and variable phenotypic expression. Gastroent. 1996;111:1593-1602.
9. Smith OP, Hann IM, Chessels JM, Reeves BR, Milla P. Haematological abnormalities in Shwachman-Diamond syndrome. Br J Haematol. 1996; 94:279-284.
10. Alter BP. Inherited bone marrow failure syndromes. In: Handin RI, Stossel TP, Lux SE, eds. Blood: Principles and Practice of Hematology. Philadelphia, PA: Lippincott; 1995:227-291.
11. Dale DC, Cottle T, Bolyard AA, et al. Severe chronic neutropenia: report on treatment and outcome from a new international registry [abstract]. Blood. 1995;86(suppl 1):425a.
12. Dale D, Bonilla MA, Boxer L, et al. Development of AML/MDS in a subset of patients (PTS) with severe chronic neutropenia (SCN) [abstract]. Blood. 1994;84(suppl 1):518a.
13. Boxer L, Dale D, Bonilla MA, et al. Administration of r-met-HuG-CSF during pregnancy in patients with severe chronic neutropenia (SCN) [abstract]. Blood. 1995;86 (suppl 1):508a.
14. Boxer L, Bonilla MA, Cham B, et al. Severe chronic neutropenia: from the laboratory to the clinic [abstract]. Int J Hematol. 1996;64(suppl 1):59.
15. Kannourakis G, Kurtzberg J, Bonilla MA, et al. Report on patients with glycogen storage disease 1b with severe chronic neutropenia treated with filgrastim [abstract]. Blood. 1996;88(suppl 1):349a.
16. Zeidler C, Bonilla MA, Boxer L, et al. Report on patients with severe chronic neutropenia receiving G-CSF [abstract]. Blood. 1996;88(suppl 1): 349a.
17. Freedman MH, Bonilla MA, Boxer L, et al. MDS/ AML in patients with severe chronic neutropenia receiving G-CSF [abstract]. Blood. 1996;88(suppl 1):448a.
18. Bonilla MA, Dale DD, Zeidler C, et al. Long-term safety of treatment with recombinant human granulocyte colony-stimulating factor (r-metHuG-CSF) in patients with severe congenital neutropenias. Br J Haematol. 1994;88:723-730.
19. Heussner P, Haase D, Kanz L, Fonatsch C, Welte K, Freund M. G-CSF in the long-term treatment of cyclic neutropenia and chronic idiopathic neutropenia in adult patients, Int J Hematol. 1995;62: 225-234.
20. Zeidler C, Reiter A, Yakisan E, Koci B, Riehm H, Welte K. Langzeitbehandlung mit rekombinantem humanen Granulozyten-Kolonien stimulierenden Faktor bei Patienten mit schwerer kongenitaler Neutropenie. Klin-Padiatr. 1993;205:264-271.
21. Colton T. Longitudinal studies and the use of the life table. In: Statistics in Medicine. Boston: Little, Brown and Company; 1974:237.
22. Cox DR. Regression models and life tables. J Royal Stat Soc. 1972;34B:187.
23. SAS Institute Inc. SAS Language: Reference. Ver 6, 1st ed. Cary, NC: SAS Institute Inc; 1990:1042.
24. Dale D, Bonilla M, Davis M, et al. A randomized controlled phase III trial of recombinant human granulocyte colony-stimulating factor (Filgrastim) for treatment of SCN. Blood. 1993;81:2496-2502.
25. Cleary PD, Morrissey G, Yver A, Oster G. The effects of rG-CSF on health-related quality of life in children with congenital agranulocytosis. Qual Life Res. 1994;3:307-315.
26. Fazio MT, Glaspy JA. The impact of granulocyte colony-stimulating factor on quality of life in patients with severe chronic neutropenia. Oncol Nurs Form. 1991;18:1411-1414.
27. Freedman MH. Safety of long-term administration of granulocyte-colony stimulating factor for severe chronic neutropenia. Current Opinion Hematol. 1997;4:217-224.
28. Freedman MH. Congenital marrow failure syndromes and malignant hematopoietic transformation. The Oncologist. 1996;1:354-360.
29. Kalra R, Dale D, Freedman MH, et al. Monosomy 7 and activating ras mutations accompany malignant transformation in patients with congenital neutropenia. Blood. 1995;86:4579-4586.
30. Dong F, Brynes RK, Tidow N, Welte K, Lowenberg B, Touw IP. 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;333:487-492.
31. Tidow N, Pilz C, Teichmann B, et al. Clinical relevance of point mutations in the cytoplasmic domain of the granulocyte colony-stimulating factor (G-CSF) receptor gene in patients with severe congenital neutropenia. Blood. 1997;89:2369-2375.
32. Dong F, Dale DC, Bonilla MA, et al. Mutations in the granulocyte-colony stimulating factor receptor gene in patients with severe congenital neutropenia. Leukemia. 1997;11:120-125.
33. Welte K, Touw I. G-CSF receptor mutations in patients with severe chronic neutropenia: a step in leukemogenesis. Blood. 1997;90(suppl 1):433a.
34. Hunter MG, Avalos BR. Deletion of a critical internalization domain in the G-CSFR in acute myelogenous leukemia preceded by severe chronic neutropenia. Blood. 1999;93:440-446.
35. Ward AC, vanAesch 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.