Lenalidomide in the Myelodysplastic Syndrome with Chromosome 5q Deletion
Abstract
BACKGROUND: Severe, often refractory anemia is characteristic of the myelodysplastic syndrome associated with chromosome 5q31 deletion. We investigated whether lenalidomide (CC5013) could reduce the transfusion requirement and suppress the abnormal 5q31- clone in patients with this disorder. METHODS: One hundred forty-eight patients received 10 mg of lenalidomide for 21 days every 4 weeks or daily. Hematologic, bone marrow, and cytogenetic changes were assessed after 24 weeks of treatment by an intention-to-treat analysis. RESULTS: Among the 148 patients, 112 had a reduced need for transfusions (76%; 95% confidence interval [CI], 68 to 82) and 99 patients (67%; 95% CI, 59 to 74) no longer required transfusions, regardless of the karyotype complexity. The response to lenalidomide was rapid (median time to response, 4.6 weeks; range, 1 to 49) and sustained; the median duration of transfusion independence had not been reached after a median of 104 weeks of follow-up. The maximum hemoglobin concentration reached a median of 13.4 g per deciliter (range, 9.2 to 18.6), with a corresponding median rise of 5.4 g per deciliter (range, 1.1 to 11.4), as compared with the baseline nadir value before transfusion. Among 85 patients who could be evaluated, 62 had cytogenetic improvement, and 38 of the 62 had a complete cytogenetic remission. There was complete resolution of cytologic abnormalities in 38 of 106 patients whose serial bone marrow samples could be evaluated. Moderate-to-severe neutropenia (in 55% of patients) and thrombocytopenia (in 44%) were the most common reasons for interrupting treatment or adjusting the dose of lenalidomide. CONCLUSIONS: Lenalidomide can reduce transfusion requirements and reverse cytologic and cytogenetic abnormalities in patients who have the myelodysplastic syndrome with the 5q31 deletion. (ClinicalTrials.gov number, NCT00065156 [ClinicalTrials.gov].).
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
Lenalidomide is a highly effective treatment for 5q-MDS, a myelodysplastic syndrome with heterozygous deletion of the long arm of chromosome 5, which can progress into more aggressive leukemia.
“the iMiD lenalidomide is a highly effective treatment for 5q-MDS, a myelodysplastic syndrome with heterozygous deletion of the long arm of chromosome 5, which can progress into more aggressive leukemia (list et al. 2006).”
From From serendipity to strategy: molecular glue degraders in cancer therapeutics (2025) · cites this paper as list et al. 2006
For older patients and/or those unfit for autologous transplantation, the preferred induction approach remains lenalidomide in combination with dexamethasone with or without daratumumab, regimens that are effective and well tolerated in this patient population.
“For older patients and/or those unfit for autologous transplantation, the preferred induction approach remains lenalidomide in combination with dexamethasone with or without daratumumab, regimens that are effective and well tolerated in this patient population27,29.”
From Cancer therapies based on targeted protein degradation — lessons learned with lenalidomide (2021) · cites this paper as [29]
In newly diagnosed MM, lenalidomide and dexamethasone are typically combined with a proteasome inhibitor such as bortezomib and/or the CD38-targeting monoclonal antibody daratumumab.
“In this population, lenalidomide and dexamethasone are typically combined with a proteasome inhibitor, such as bortezomib28, and/or the CD38-targeting monoclonal antibody daratumumab29.”
From Cancer therapies based on targeted protein degradation — lessons learned with lenalidomide (2021) · cites this paper as [29]
Lenalidomide exerts highly favorable effects against 5q-syndrome, a type of myelodysplastic syndrome.
“it has been shown that lenalidomide exerts highly favorable effects against 5q-syndrome, a type of myelodysplastic syndrome [28,29].”
From Cereblon and its downstream substrates as molecular targets of immunomodulatory drugs (2016) · cites this paper as [28]
Cites
Nonparametric Estimation from Incomplete Observations
Kaplan, E. L., & Meier, P. (1992) · Springer series in statistics · doi:10.1007/978-1-4612-4380-9_25 · Cited by 45,232 · see citing works
Nonparametric Estimation from Incomplete Observations
Kaplan, E. L., & Meier, P. (1958) · Journal of the American Statistical Association · doi:10.1080/01621459.1958.10501452 · Cited by 39,158 · see citing works
International scoring system for evaluating prognosis in myelodysplastic syndromes
Greenberg, P., Cox, C., LeBeau, M. M., Fenaux, P., Morel, P., Sanz, G., Sanz, M., Vallespi, T., Hamblin, T., Oscier, D., et al. (1997) · Blood · doi:10.1182/blood.v89.6.2079 · Cited by 4,427 · see citing works
Self-citationProposals for the classification of the myelodysplastic syndromes
Bennett, J. M., Catovsky, D., Daniel, M., Flandrin, G., Galton, D. A. G., Gralnick, H. R., & Sultan, C. (1982) · British Journal of Haematology · doi:10.1111/j.1365-2141.1982.tb08475.x · Cited by 3,619 · see citing works
Self-citationThe World Health Organization (WHO) classification of the myeloid neoplasms
Vardiman, J. W., Harris, N. L., & Brunning, R. D. (2002) · Blood · doi:10.1182/blood-2002-04-1199 · Cited by 2,191 · see citing works
Shah, N. P., Nicoll, J. M., Nagar, B., Gorre, M. E., PAQUETTE, R. L., Kuriyan, J., & Sawyers, C. L. (2002) · Cancer Cell · doi:10.1016/s1535-6108(02)00096-x · Cited by 1,608 · see citing works
Efficacy of Lenalidomide in Myelodysplastic Syndromes
List, A., Kurtin, S., Roe, D. J., Buresh, A., Mahadevan, D., Fuchs, D., Rimsza, L., Heaton, R., Knight, R., & Zeldis, J. B. (2005) · New England Journal of Medicine · doi:10.1056/nejmoa041668 · Cited by 880 · see citing works
Self-citationMalcovati, L., Porta, M. G. D., Pascutto, C., Invernizzi, R., Boni, M., Travaglino, E., Passamonti, F., Arcaini, L., Maffioli, M., BERNASCONI, P. P., et al. (2005) · Journal of Clinical Oncology · doi:10.1200/jco.2005.01.7038 · Cited by 867 · see citing works
Cheson, B. D., Bennett, J. M., Kantarjian, H. M., Pinto, A., Schiffer, C. A., Nimer, S. D., Löwenberg, B., Beran, M., de Witte, T. M., Stone, R. M., et al. (2000) · PubMed · Cited by 666 · see citing works
Self-citationDistinct haematological disorder with deletion of long arm of No. 5 chromosome
BERGHE, H. V. D., Cassiman, J., David, G., Fryns, J., Michaux, J., & SOKAL, G. (1974) · Nature · doi:10.1038/251437a0 · Cited by 442 · see citing works
Narrowing and genomic annotation of the commonly deleted region of the 5q− syndrome
Boultwood, J., Fidler, C., Strickson, A., Watkins, F., Gama, S., Kearney, L., Tosi, S., Kasprzyk, A., Cheng, J., Jaju, R. J., et al. (2002) · Blood · doi:10.1182/blood.v99.12.4638 · Cited by 264 · see citing works
Genetic pathways in therapy-related myelodysplasia and acute myeloid leukemia
Pedersen-Bjergaard, J., Andersen, M. K., Christiansen, D. H., & Nerlov, C. (2002) · Blood · doi:10.1182/blood.v99.6.1909 · Cited by 244 · see citing works
Giagounidis, A. A. N., Germing, U., Haase, S. P., Hildebrandt, B., Schlegelberger, B., Schoch, C., Wilkens, L., Heinsch, M., Willems, H. P. J., Aivado, M. A., et al. (2003) · Leukemia · doi:10.1038/sj.leu.2403189 · Cited by 210 · see citing works
Self-citationDewald, G., Mp, D., Pierre, R., et al. (1985) · Blood · doi:10.1182/blood.v66.1.189.189 · Cited by 91 · see citing works
Self-citationBERNASCONI, P. P., Klersy, C., Boni, M., Cavigliano, P. M., Calatroni, S., Giardini, I., Rocca, B., Zappatore, R., Caresana, M., Quarna, J., et al. (2005) · Leukemia · doi:10.1038/sj.leu.2403806 · Cited by 88 · see citing works
Hemoglobin level threshold for cardiac remodeling and quality of life in myelodysplastic syndrome
Olíva, E. N., Dimitrov, B. D., Benedetto, F. A., D’Angelo, A., & Nobile, F. (2005) · Leukemia Research · doi:10.1016/j.leukres.2005.03.004 · Cited by 84 · see citing works
Flagged first, then by citations
Cited by
Sorted by citations
Clinical and biological implications of driver mutations in myelodysplastic syndromes
Papaemmanuil, E., Gerstung, M., Malcovati, L., Tauro, S., Gundem, G., Van Loo, P., Yoon, C. J., Ellis, P., Wedge, D. C., Pellagatti, A., et al. (2013) · Blood · doi:10.1182/blood-2013-08-518886 · Cited by 1,832 · see citing works
Lenalidomide induces ubiquitination and degradation of CK1α in del(5q) MDS
Krönke, J., Fink, E. C., Hollenbach, P. W., MacBeth, K. J., Hurst, S. N., Udeshi, N. D., Chamberlain, P. P., Mani, D. R., Man, H. W., Gandhi, A. K., et al. (2015) · Nature · doi:10.1038/nature14610 · Cited by 863 · see citing works
The central role of inflammatory signaling in the pathogenesis of myelodysplastic syndromes
Sallman, D. A., & List, A. (2019) · Blood · doi:10.1182/blood-2018-10-844654 · Cited by 273 · see citing works
Sekeres, M. A., Schoonen, W. M., Kantarjian, H., List, A., Fryzek, J., Paquette, R., & Maciejewski, J. P. (2008) · JNCI: Journal of the National Cancer Institute · doi:10.1093/jnci/djn349 · Cited by 248 · see citing works
Self-citationMelchert, M., & List, A. (2007) · The International Journal of Biochemistry & Cell Biology · doi:10.1016/j.biocel.2007.01.022 · Cited by 246 · see citing works
Self-citationOrdered and deterministic cancer genome evolution after p53 loss
Baslan, T., Morris, J. P., Zhao, Z., Reyes, J., Ho, Y.-J., Tsanov, K. M., Bermeo, J., Tian, S., Zhang, S., Askan, G., et al. (2022) · Nature · doi:10.1038/s41586-022-05082-5 · Cited by 221 · see citing works
Sekeres, M. A., Othus, M., List, A. F., Odenike, O., Stone, R. M., Gore, S. D., Litzow, M. R., Buckstein, R., Fang, M., Roulston, D., et al. (2017) · Journal of Clinical Oncology · doi:10.1200/jco.2015.66.2510 · Cited by 237 · see citing works
Self-citationChromosomal Abnormalities in Cancer
Fröhling, S., & Döhner, H. (2008) · New England Journal of Medicine · doi:10.1056/nejmra0803109 · Cited by 229 · see citing works
The novel mechanism of lenalidomide activity
Fink, E. C., & Ebert, B. L. (2015) · Blood · doi:10.1182/blood-2015-07-567958 · Cited by 213 · see citing works
Chasing molecular glue degraders: screening approaches
Domostegui, A., Nieto-Barrado, L., Perez-Lopez, C., & Mayor-Ruiz, C. (2022) · Chemical Society Reviews · doi:10.1039/d2cs00197g · Cited by 200 · see citing works
Eichner, R., Heider, M., Fernández-Sáiz, V., van Bebber, F., Garz, A.-K., Lemeer, S., Rudelius, M., Targosz, B.-S., Jacobs, L., Knorn, A.-M., et al. (2016) · Nature Medicine · doi:10.1038/nm.4128 · Cited by 183 · see citing works
Bally, C., Adès, L., Renneville, A., Sebert, M., Eclache, V., Preudhomme, C., Mozziconacci, M.-J., de The, H., Lehmann-Che, J., & Fenaux, P. (2014) · Leukemia Research · doi:10.1016/j.leukres.2014.03.012 · Cited by 162 · see citing works
Cancer therapies based on targeted protein degradation — lessons learned with lenalidomide
Jan, M., Sperling, A. S., & Ebert, B. L. (2021) · Nature Reviews Clinical Oncology · doi:10.1038/s41571-021-00479-z · Cited by 137 · see citing works
Crbn I391V is sufficient to confer in vivo sensitivity to thalidomide and its derivatives in mice
Fink, E. C., McConkey, M., Adams, D. N., Haldar, S. D., Kennedy, J. A., Guirguis, A. A., Udeshi, N. D., Mani, D. R., Chen, M., Liddicoat, B., et al. (2018) · Blood · doi:10.1182/blood-2018-05-852798 · Cited by 131 · see citing works
Casein kinase 1α: biological mechanisms and theranostic potential
Jiang, S., Zhang, M., Sun, J., & Yang, X. (2018) · Cell Communication and Signaling · doi:10.1186/s12964-018-0236-z · Cited by 130 · see citing works
Thyroid Dysfunction as an Unintended Side Effect of Anticancer Drugs
Torino, F., Barnabei, A., Paragliola, R., Baldelli, R., Appetecchia, M., & Corsello, S. M. (2013) · Thyroid® · doi:10.1089/thy.2013.0241 · Cited by 117 · see citing works
The clinical significance of cereblon expression in multiple myeloma
Schuster, S. R., Kortuem, K. M., Zhu, Y. X., Braggio, E., Shi, C.-X., Bruins, L. A., Schmidt, J. E., Ahmann, G., Kumar, S., Rajkumar, S. V., et al. (2014) · Leukemia Research · doi:10.1016/j.leukres.2013.08.015 · Cited by 113 · see citing works
Fehniger, T. A., Uy, G. L., Trinkaus, K., Nelson, A. D., Demland, J., Abboud, C. N., Cashen, A. F., Stockerl-Goldstein, K. E., Westervelt, P., DiPersio, J. F., et al. (2011) · Blood · doi:10.1182/blood-2010-07-297143 · Cited by 107 · see citing works
Novel immunomodulatory drugs and neo-substrates
Gao, S., Wang, S., & Song, Y. (2020) · Biomarker Research · doi:10.1186/s40364-020-0182-y · Cited by 95 · see citing works
Molecular Pathophysiology of Myelodysplastic Syndromes
Lindsley, R. C., & Ebert, B. L. (2013) · Annual Review of Pathology: Mechanisms of Disease · doi:10.1146/annurev-pathol-011811-132436 · Cited by 88 · see citing works
Dose Escalation of Lenalidomide in Relapsed or Refractory Acute Leukemias
Blum, W., Klisovic, R. B., Becker, H., Yang, X., Rozewski, D. M., Phelps, M. A., Garzon, R., Walker, A., Chandler, J. C., Whitman, S. P., et al. (2010) · Journal of Clinical Oncology · doi:10.1200/jco.2010.30.3339 · Cited by 87 · see citing works
Cereblon and its downstream substrates as molecular targets of immunomodulatory drugs
Ito, T., & Handa, H. (2016) · International Journal of Hematology · doi:10.1007/s12185-016-2073-4 · Cited by 84 · see citing works
Fang, J., Liu, X., Bolanos, L., Barker, B., Rigolino, C., Cortelezzi, A., Oliva, E. N., Cuzzola, M., Grimes, H. L., Fontanillo, C., et al. (2016) · Nature Medicine · doi:10.1038/nm.4127 · Cited by 83 · see citing works
Sekeres, M. A., Gundacker, H., Lancet, J., Advani, A., Petersdorf, S., Liesveld, J., Mulford, D., Norwood, T., Willman, C. L., Appelbaum, F. R., et al. (2011) · Blood · doi:10.1182/blood-2011-02-337303 · Cited by 80 · see citing works
Self-citationThe application and biology of immunomodulatory drugs (IMiDs) in cancer
Pan, B., & Lentzsch, S. (2012) · Pharmacology & Therapeutics · doi:10.1016/j.pharmthera.2012.07.004 · Cited by 76 · see citing works
Current and emerging strategies for management of myelodysplastic syndromes
Saygin, C., & Carraway, H. E. (2021) · Blood Reviews · doi:10.1016/j.blre.2020.100791 · Cited by 70 · see citing works
Eksioglu, E. A., Chen, X., Heider, K.-H., Rueter, B., McGraw, K. L., Basiorka, A. A., Wei, M., Burnette, A., Cheng, P., Lancet, J., et al. (2017) · Leukemia · doi:10.1038/leu.2017.21 · Cited by 66 · see citing works
Self-citationAnemia as the Main Manifestation of Myelodysplastic Syndromes
Santini, V. (2015) · Seminars in Hematology · doi:10.1053/j.seminhematol.2015.06.002 · Cited by 52 · see citing works
Lodé, L., Ménard, A., Flet, L., Richebourg, S., Loirat, M., Eveillard, M., Le Bris, Y., Godon, C., Theisen, O., Gagez, A.-L., et al. (2018) · Haematologica · doi:10.3324/haematol.2017.181404 · Cited by 52 · see citing works
A decade of progress in myelodysplastic syndrome with chromosome 5q deletion
List, A., Ebert, B. L., & Fenaux, P. (2018) · Leukemia · doi:10.1038/s41375-018-0029-9 · Cited by 49 · see citing works
Self-citationScharenberg, C., Giai, V., Pellagatti, A., Saft, L., Dimitriou, M., Jansson, M., Jädersten, M., Grandien, A., Douagi, I., Neuberg, D. S., et al. (2017) · Haematologica · doi:10.3324/haematol.2016.152025 · Cited by 47 · see citing works
Figaro, M. K., Clayton, W., Usoh, C., Brown, K., Kassim, A., Lakhani, V. T., & Jagasia, S. (2011) · American Journal of Hematology · doi:10.1002/ajh.22008 · Cited by 43 · see citing works
Jonasova, A., Bokorova, R., Polak, J., Vostry, M., Kostecka, A., Hajkova, H., Neuwirtova, R., Siskova, M., Sponerova, D., Cermak, J., et al. (2015) · European Journal of Haematology · doi:10.1111/ejh.12457 · Cited by 31 · see citing works
DeAngelo, D. J., Brunner, A. M., Werner, L., Avigan, D., Fathi, A. T., Sperling, A. S., Washington, A., Stroopinsky, D., Rosenblatt, J., McMasters, M., et al. (2018) · American Journal of Hematology · doi:10.1002/ajh.24968 · Cited by 16 · see citing works
Self-citationDrugs and Other Substances Interfering with Thyroid Function
Montanelli, L., Benvenga, S., Hegedüs, L., Vitti, P., Latrofa, F., & Duntas, L. H. (2018) · Endocrinology · doi:10.1007/978-3-319-45013-1_27 · Cited by 18 · see citing works
From serendipity to strategy: molecular glue degraders in cancer therapeutics
Darling, S., Bajrami, I., & West, S. C. (2025) · Critical Reviews in Biochemistry and Molecular Biology · doi:10.1080/10409238.2025.2564068 · Cited by 10 · see citing works
“…for 5q-MDS, a myelodysplastic syndrome with heterozygous deletion of the long arm of chromosome 5, which can progress into more aggressive leukemia (list et al. 2006). initially, it was thought that the efficacy of lenalidomide was a result of cRl4 cRBN targeting genes within the eponymous deleted…”
- Authors
- List, A., Dewald, G., Bennett, J., Giagounidis, A., Raza, A., Feldman, E., Powell, B., Greenberg, P., Thomas, D., Stone, R., Reeder, C., Wride, K., Patin, J., Schmidt, M., Zeldis, J., & Knight, R.
- Affiliations
- University of South Florida (US)
- Mayo Clinic (US)
- University of Rochester (US)
- University of Massachusetts Chan Medical School (US)
- Cornell University (US)
- Wake Forest University (US)
- Stanford University (US)
- The University of Texas MD Anderson Cancer Center (US)
- Dana-Farber Cancer Institute (US)
- ORCID
- Venue
- New England Journal of Medicine
- Publisher
- Massachusetts Medical Society
- Published
- 2006-10-05
- Volume
- 355
- Issue
- 14
- Pages
- 1456-1465
- ISSN
- 0028-4793 (ISSN-L)
- 1533-4406
- OpenAlex
- W2114492363
- Cited by
- 1,319
- References
- 16
- Citations by year
- 2026: 20 · 2025: 34 · 2024: 33 · 2023: 41 · 2022: 46 · 2021: 61 · 2020: 62 · 2019: 47 · 2018: 55 · 2017: 57 · 2016: 56 · 2015: 73 · 2014: 63 · 2013: 86 · 2012: 89
- FWCI
- 56.15
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- New England Journal of Medicine (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Health Sciences › Medicine › Hematology › Acute Myeloid Leukemia Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Retinoids in leukemia and cellular processes
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Protein Degradation and Inhibitors
- Keywords
- lenalidomide, myelodysplastic syndrome, chromosome 5q deletion, transfusion independence, cytogenetic remission, anemia, hematopoietic stem cell transplantation, neutropenia, thrombocytopenia
- MeSH terms
- Lenalidomide, Adult, Aged, Aged, 80 and over, Bone Marrow, Chromosome Aberrations, Chromosome Deletion, Chromosomes, Human, Pair 5, Female, Follow-Up Studies, Humans, Male, Middle Aged, Myelodysplastic Syndromes, Remission Induction, Thalidomide, Erythrocyte Transfusion, Cytogenetic Analysis
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
