The DOI matches its Crossref record; 37 of the 37 references it cites were found in a registry or an index.
Single-agent CEP-701, a novel FLT3 inhibitor, shows biologic and clinical activity in patients with relapsed or refractory acute myeloid leukemia
Abstract
Activating mutations of FMS-like tyrosine kinase 3 (FLT3) are present in approximately 30% of patients with de novo acute myeloid leukemia (AML) and are associated with lower cure rates from standard chemotherapy-based treatment. Targeting the mutation by inhibiting the tyrosine kinase activity of FLT3 is cytotoxic to cell lines and primary AML cells harboring FLT3 mutations. Successful FLT3 inhibition can also improve survival in mouse models of FLT3-activated leukemia. CEP-701 is an orally available, novel, receptor tyrosine kinase inhibitor that selectively inhibits FLT3 autophosphorylation. We undertook a phase 1/2 trial to determine the in vivo hematologic effects of single-agent CEP-701 as salvage treatment for patients with refractory, relapsed, or poor-risk AML expressing FLT3-activating mutations. Fourteen heavily pretreated AML patients were treated with CEP-701 at an initial dose of 60 mg orally twice daily. CEP-701–related toxicities were minimal. Five patients had clinical evidence of biologic activity and measurable clinical response, including significant reductions in bone marrow and peripheral blood blasts. Laboratory data confirmed that clinical responses correlated with sustained FLT3 inhibition to CEP-701. Our results show that FLT3 inhibition is associated with clinical activity in AML patients harboring FLT3-activating mutations and indicate that CEP-701 holds promise as a novel, molecularly targeted therapy for this disease.
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Cites
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The roles of FLT3 in hematopoiesis and leukemia
Gilliland, D. G., & Griffin, J. D. (2002) · Blood · doi:10.1182/blood-2002-02-0492 · Cited by 1,558
Kottaridis, P. D., Gale, R. E., Frew, M. E., Harrison, G., Langabeer, S. E., Belton, A. A., Walker, H., Wheatley, K., Bowen, D. T., Burnett, A. K., et al. (2001) · Blood · doi:10.1182/blood.v98.6.1752 · Cited by 1,551
Internal tandem duplication of the flt3 gene found in acute myeloid leukemia.
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Activating mutation of D835 within the activation loop of FLT3 in human hematologic malignancies
Yamamoto, Y. (2001) · Blood · doi:10.1182/blood.v97.8.2434 · Cited by 1,150
Schnittger, S., Schoch, C., Dugas, M., Kern, W., Staib, P., Wuchter, C., Löffler, H., Sauerland, C. M., Serve, H., Büchner, T., et al. (2002) · Blood · doi:10.1182/blood.v100.1.59 · Cited by 973
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SU11248 is a novel FLT3 tyrosine kinase inhibitor with potent activity in vitro and in vivo
O'Farrell, A.-M., Abrams, T. J., Yuen, H. A., Ngai, T. J., Louie, S. G., Yee, K. W. H., Wong, L. M., Hong, W., Lee, L. B., Town, A., et al. (2003) · Blood · doi:10.1182/blood-2002-07-2307 · Cited by 864
Prognostic implication of FLT3 and N-RAS gene mutations in acute myeloid leukemia.
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c-kit Ligand and Flt3 Ligand: Stem/Progenitor Cell Factors With Overlapping Yet Distinct Activities
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Cited by
Döhner, H., Estey, E. H., Amadori, S., Appelbaum, F. R., Büchner, T., Burnett, A. K., Dombret, H., Fenaux, P., Grimwade, D., Larson, R. A., et al. (2010) · Blood · doi:10.1182/blood-2009-07-235358 · Cited by 3,373
Midostaurin plus Chemotherapy for Acute Myeloid Leukemia with a FLT3 Mutation
Stone, R. M., Mandrekar, S. J., Sanford, B. L., Laumann, K., Geyer, S., Bloomfield, C. D., Thiede, C., Prior, T. W., Döhner, K., Marcucci, G., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1614359 · Cited by 2,250
Functional genomic landscape of acute myeloid leukaemia
Tyner, J. W., Tognon, C. E., Bottomly, D., Wilmot, B., Kurtz, S. E., Savage, S. L., Long, N., Schultz, A. R., Traer, E., Abel, M., et al. (2018) · Nature · doi:10.1038/s41586-018-0623-z · Cited by 1,303
Gilteritinib or Chemotherapy for Relapsed or Refractory FLT3 -Mutated AML
Perl, A. E., Martinelli, G., Cortes, J. E., Neubauer, A., Berman, E., Paolini, S., Montesinos, P., Baer, M. R., Larson, R. A., Ustun, C., et al. (2019) · New England Journal of Medicine · doi:10.1056/nejmoa1902688 · Cited by 1,287
Self-citationMolecular mechanisms of cardiotoxicity of tyrosine kinase inhibition
Force, T., Krause, D. S., & Van Etten, R. A. (2007) · Nature Reviews Cancer · doi:10.1038/nrc2106 · Cited by 801
Metadata
- Authors
- Smith, B. D., Levis, M., Beran, M., Giles, F., Kantarjian, H., Berg, K., Murphy, K. M., Dauses, T., Allebach, J., & Small, D.
- Venue
- Blood
- Publisher
- American Society of Hematology
- Published
- 2004-05-15
- Volume
- 103
- Issue
- 10
- Pages
- 3669-3676
- ISSN
- 0006-4971 (ISSN-L)
- 1528-0020
- OpenAlex
- W2125877707
- Cited by
- 632
- References
- 37
- FWCI
- 30.76
- Type
- journal article
- Language
- en
- Open access
- closed
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- CoChat. (2026). Citation check: Single-agent CEP-701, a novel FLT3 inhibitor, shows biologic and clinical activity in patients with relapsed or refractory acute myeloid leukemia. https://app.cochat.ai/papers/10.1182/blood-2003-11-3775
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- Smith, B. D., Levis, M., Beran, M., Giles, F., Kantarjian, H., Berg, K., Murphy, K. M., Dauses, T., Allebach, J., & Small, D. (2004). Single-agent CEP-701, a novel FLT3 inhibitor, shows biologic and clinical activity in patients with relapsed or refractory acute myeloid leukemia. Blood. https://doi.org/10.1182/blood-2003-11-3775
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
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