Lenalidomide for del(5q) and Non-del(5q) Myelodysplastic Syndromes

Seminars in Hematology

Volumn 49, Issue 4, 2012, Pages 312-322

  Giagounidis, Aristoteles A N ^a  

^a MARIEN HOSPITAL DÜSSELDORF   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

ANTIANEMIC AGENT; AZACITIDINE; CORTICOSTEROID; CYTARABINE; DAUNORUBICIN; LENALIDOMIDE; PLACEBO; PREDNISONE; PROTEIN P53; RECOMBINANT ERYTHROPOIETIN; VALPROIC ACID; VORINOSTAT;

5Q- SYNDROME; ACUTE GRANULOCYTIC LEUKEMIA; ANEMIA; ANTIANGIOGENIC ACTIVITY; ANTIGEN EXPRESSION; ANTIINFLAMMATORY ACTIVITY; APOPTOSIS; ARTICLE; BLOOD TRANSFUSION; CANCER INCIDENCE; CANCER RISK; CELL PROTECTION; CHROMOSOME ABERRATION; CLINICAL TRIAL (TOPIC); CONSTIPATION; CYTOGENETICS; DEEP VEIN THROMBOSIS; DIARRHEA; DRUG DOSE COMPARISON; DRUG EFFICACY; DRUG ERUPTION; DRUG MECHANISM; DRUG POTENTIATION; DRUG SAFETY; DRY SKIN; EPIGENETICS; ERADICATION THERAPY; ERYTHROID PRECURSOR CELL; G1 PHASE CELL CYCLE CHECKPOINT; GENE MUTATION; GENE TARGETING; HIGH RISK POPULATION; HUMAN; IMMUNOMODULATION; INTENTION TO TREAT ANALYSIS; INTERNATIONAL PROGNOSTIC SCORING SYSTEM; LEUKEMOGENESIS; MULTIPLE MYELOMA; MUSCLE CRAMP; MYELODYSPLASTIC SYNDROME; NEUTROPENIA; NONHUMAN; OPTIMAL DRUG DOSE; OVERALL SURVIVAL; PATIENT MONITORING; PHASE 1 CLINICAL TRIAL (TOPIC); PHASE 2 CLINICAL TRIAL (TOPIC); PLEIOTROPY; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); REFRACTORY ANEMIA WITH EXCESS BLASTS; REMISSION; RISK BENEFIT ANALYSIS; RISK FACTOR; SCALP PRURITUS; SCORING SYSTEM; SECOND CANCER; SURVIVAL TIME; THROMBOCYTOPENIA; TREATMENT DURATION; TREATMENT RESPONSE; WORLD HEALTH ORGANIZATION PROGNOSTIC SCORING SYSTEM;

CHROMOSOME DELETION; CHROMOSOMES, HUMAN, PAIR 5; HUMANS; LEUKEMIA, MYELOID, ACUTE; MYELODYSPLASTIC SYNDROMES; THALIDOMIDE; TREATMENT OUTCOME;

EID: 84867614529     PISSN: 00371963     EISSN: 15328686     Source Type: Journal    
DOI: 10.1053/j.seminhematol.2012.07.001     Document Type: Article

References (57)

1. Singhal S., Mehta J., Desikan R., et al. Antitumor activity of thalidomide in refractory multiple myeloma. N Engl J Med 1999, 341:1565-1571.
2. Raza A., Meyer P., Dutt D., et al. Thalidomide produces transfusion independence in long-standing refractory anemias of patients with myelodysplastic syndromes. Blood 2001, 98:958-965.
3. Strupp C., Hildebrandt B., Germing U., Haas R., Gattermann N. Cytogenetic response to thalidomide treatment in three patients with myelodysplastic syndrome. Leukemia 2003, 17:1200-1202.
4. Zeldis J.B., Knight R., Hussein M., Chopra R., Muller G. A review of the history, properties, and use of the immunomodulatory compound lenalidomide. Ann N Y Acad Sci 2011, 1222:76-82.
5. Bartlett J.B., Tozer A., Stirling D., Zeldis J.B. Recent clinical studies of the immunomodulatory drug (imid) lenalidomide. Br J Cancer 2005, 93:613-619.
6. List A., Kurtin S., Roe D.J., et al. Efficacy of lenalidomide in myelodysplastic syndromes. N Engl J Med 2005, 352:549-557.
7. Giagounidis A.A., Haase S., Heinsch M., Gohring G., Schlegelberger B., Aul C. Lenalidomide in the context of complex karyotype or interrupted treatment: case reviews of del(5q)mds patients with unexpected responses. Ann Hematol 2007, 86:133-137.
8. Himmelmann A., Tchinda J. Long-term transfusion independence in del(5q) mds patients after short term therapy with lenalidomide: 2 new cases. Leuk Res 2012, 36:656-657.
9. Jadersten M., Karsan A. Clonal evolution in myelodysplastic syndromes with isolated del(5q): the importance of genetic monitoring. Haematologica 2011, 96:177-180.
10. Bartlett J.B., Dredge K., Dalgleish A.G. The evolution of thalidomide and its imid derivatives as anticancer agents. Nat Rev Cancer 2004, 4:314-322.
11. Buesche G., Dieck S., Giagounidis A., et al. Anti-angiogenic in vivo effect of lenalidomide (cc-5013) in myelodysplastic syndrome with del(5q) chromosome abnormality and its relation to the course of disease. ASH Annual Meeting Abstracts 2005, 106:372.
12. Ximeri M., Galanopoulos A., Klaus M., et al. Effect of lenalidomide therapy on hematopoiesis of patients with myelodysplastic syndrome associated with chromosome 5q deletion. Haematologica 2010, 95:406-414.
13. Corral L.G., Haslett P.A., Muller G.W., et al. Differential cytokine modulation and t cell activation by two distinct classes of thalidomide analogues that are potent inhibitors of tnf-alpha. J Immunol 1999, 163:380-386.
14. Mitsiades C.S., Mitsiades N. Cc-5013 (celgene). Curr Opin Investig Drugs 2004, 5:635-647.
15. Melchert M., Kale V., List A. The role of lenalidomide in the treatment of patients with chromosome 5q deletion and other myelodysplastic syndromes. Curr Opin Hematol 2007, 14:123-129.
16. Boultwood J., Fidler C., Strickson A.J., et al. Narrowing and genomic annotation of the commonly deleted region of the 5q- syndrome. Blood 2002, 99:4638-4641.
17. Ebert B.L., Pretz J., Bosco J., et al. Identification of rps14 as a 5q- syndrome gene by rna interference screen. Nature 2008, 451:335-339.
18. Oliva E.N., Cuzzola M., Nobile F., et al. Changes in rps14 expression levels during lenalidomide treatment in low- and intermediate-1-risk myelodysplastic syndromes with chromosome 5q deletion. Eur J Haematol 2010, 85:231-235.
19. Pellagatti A., Jadersten M., Forsblom A.M., et al. Lenalidomide inhibits the malignant clone and up-regulates the sparc gene mapping to the commonly deleted region in 5q- syndrome patients. Proc Natl Acad Sci U S A 2007, 104:11406-11411.
20. Wei S., Chen X., Rocha K., et al. A critical role for phosphatase haplodeficiency in the selective suppression of deletion 5q mds by lenalidomide. Proc Natl Acad Sci U S A 2009, 106:12974-12979.
21. Hutchins J.R., Clarke P.R. Many fingers on the mitotic trigger: post-translational regulation of the cdc25c phosphatase. Cell Cycle 2004, 3:41-45.
22. Turowski P., Franckhauser C., Morris M.C., Vaglio P., Fernandez A., Lamb N.J. Functional cdc25c dual-specificity phosphatase is required for s-phase entry in human cells. Mol Biol Cell 2003, 14:2984-2998.
23. Vlachos A., Farrar J., Atsidaftos E., et al. 5q- syndrome in a child: Is this acquired diamond blackfan anemia (dba)?. ASH Annual Meeting Abstracts 2010, 116:4430.
24. Moutouh-de Parseval L.A., Verhelle D., Glezer E., et al. Pomalidomide and lenalidomide regulate erythropoiesis and fetal hemoglobin production in human cd34+ cells. J Clin Invest 2008, 118:248-258.
25. Hermiston M.L., Zikherman J., Zhu J.W. Cd45, cd148, and lyp/pep: critical phosphatases regulating src family kinase signaling networks in immune cells. Immunol Rev 2009, 228:288-311.
26. Irie-Sasaki J., Sasaki T., Matsumoto W., et al. Cd45 is a jak phosphatase and negatively regulates cytokine receptor signalling. Nature 2001, 409:349-354.
27. Komrokji R.S., List A.F. Role of lenalidomide in the treatment of myelodysplastic syndromes. Semin Oncol 2011, 38:648-657.
28. Basiorka A.A., McGraw K., Clark J., et al. Lenalidomide upregulates erythropoietin receptor expression in hematopoietic progenitors by modulating receptor turnover. ASH Annual Meeting Abstracts 2011, 118:2382.
29. Kotla V., Goel S., Nischal S., et al. Mechanism of action of lenalidomide in hematological malignancies. J Hematol Oncol 2009, 2:36.
30. List A., Dewald G., Bennett J., et al. Lenalidomide in the myelodysplastic syndrome with chromosome 5q deletion. N Engl J Med 2006, 355:1456-1465.
31. Fenaux P., Giagounidis A., Selleslag D., et al. A randomized phase 3 study of lenalidomide versus placebo in rbc transfusion-dependent patients with low-/intermediate-1-risk myelodysplastic syndromes with del5q. Blood 2011, 118:3765-3776.
32. Sekeres M.A., Maciejewski J.P., Giagounidis A.A., et al. Relationship of treatment-related cytopenias and response to lenalidomide in patients with lower-risk myelodysplastic syndromes. J Clin Oncol 2008, 26:5943-5949.
33. List A.F., Giagounidis A., Backstrom J.T., Fu T., Fenaux P. Early lenalidomide (len) dose intensity and durable rbc-transfusion independence (rbc-ti) in patients (pts) with low-/int-1-risk myelodysplastic syndromes (mds) and del5q. ASCO Meeting Abstracts 2011, 29:6522.
34. Raza A., Reeves J.A., Feldman E.J., et al. Phase 2 study of lenalidomide in transfusion-dependent, low-risk, and intermediate-1 risk myelodysplastic syndromes with karyotypes other than deletion 5q. Blood 2008, 111:86-93.
35. Ebert B.L., Galili N., Tamayo P., et al. An erythroid differentiation signature predicts response to lenalidomide in myelodysplastic syndrome. PLoS Med 2008, 5:e35.
36. Mallo M., Cervera J., Schanz J., et al. Impact of adjunct cytogenetic abnormalities for prognostic stratification in patients with myelodysplastic syndrome and deletion 5q. Leukemia 2011, 25:110-120.
37. Giagounidis A.A., Germing U., Haase S., et al. Clinical, morphological, cytogenetic, and prognostic features of patients with myelodysplastic syndromes and del(5q) including band q31. Leukemia 2004, 18:113-119.
38. Kantarjian H., O'Brien S., Ravandi F., et al. The heterogeneous prognosis of patients with myelodysplastic syndrome and chromosome 5 abnormalities: how does it relate to the original lenalidomide experience in mds?. Cancer 2009, 115:5202-5209.
39. Germing U., Lauseker M., Hildebrandt B., et al. Survival, prognostic factors and rates of leukemic transformation in 381 untreated patients with mds and del(5q): a multicenter study. Leukemia 2012.
40. Kuendgen A., Lauseker M., List A.F., et al. Lenalidomide treatment is not related to aml progression risk but is associated with a survival benefit in rbc transfusion-dependent patients with ipss low- or int-1-risk mds with del5q: results from a comparative study. ASH Annual Meeting Abstracts 2011, 118:119. International Working Group on MDS with del5q.
41. Hasford J., Baccarani M., Hoffmann V., et al. Predicting complete cytogenetic response and subsequent progression-free survival. 2060 patients with cml on imatinib treatment: the eutos score. Blood 2011, 118:686-692.
42. Hasford J. [Chance or exploitation?. Clinical studies from various views]. Med Monatsschr Pharm 2002, 25:241-243.
43. Pfirrmann M., Hasford J. A simulation study using validated prognostic factors to assess expected long-term survival. Methods Inf Med 2005, 44:577-583.
44. Hasford J., Bramlage P., Koch G., Lehmacher W., Einhaupl K., Rothwell P.M. Inconsistent trial assessments by the national institute for health and clinical excellence and iqwig: standards for the performance and interpretation of subgroup analyses are needed. J Clin Epidemiol 2010, 63:1298-1304.
45. Hasford J., Bramlage P., Koch G., Lehmacher W., Einhaupl K., Rothwell P.M. Standards for subgroup analyses are needed?-We couldn't agree more. J Clin Epidemiol 2011, 64:451.
46. List A.F., Lancet J.E., Melchert M., et al. Two-stage pharmacokinetic & efficacy study of lenalidomide alone or combined with recombinant erythropoietin (epo) in lower risk mds epo-failures [pk-002]. ASH Annual Meeting Abstracts 2007, 110:4626.
47. Adès L., Boehrer S., Prebet T., et al. Efficacy and safety of lenalidomide in intermediate-2 or high-risk myelodysplastic syndromes with 5q deletion: results of a phase 2 study. Blood 2009, 113:3947-3952.
48. Ades L., Prebet T., Stamatoullas A., et al. Lenalidomide (len) combined to intensive chemotherapy (ic) in aml and higher risk mds with del 5q. Results of a phase i/ii study of the groupe francophone des myelodysplasies (gfm). ASH Annual Meeting Abstracts 2010, 116:508.
49. Sekeres M.A., List A.F., Cuthbertson D., et al. Phase I combination trial of lenalidomide and azacitidine in patients with higher-risk myelodysplastic syndromes. J Clin Oncol 2010, 28:2253-2258.
50. Sekeres M.A., Komrokji R.S., Lancet J.E., et al. Final results from the phase 2 continuation study of the lenalidomide and azacitidine combination in patients with higher-risk myelodysplastic syndromes (mds). ASH Annual Meeting Abstracts 2011, 118:607.
51. Tehranchi R., Woll P.S., Anderson K., et al. Persistent malignant stem cells in del(5q) myelodysplasia in remission. N Engl J Med 2010, 363:1025-1037.
52. Giagounidis A.A., Kulasekararaj A., Germing U., et al. Long-term transfusion independence in del(5q) mds patients who discontinue lenalidomide. Leukemia 2012, 26:855-858.
53. Sharma S.V., Lee D.Y., Li B., et al. A chromatin-mediated reversible drug-tolerant state in cancer cell subpopulations. Cell 2010, 141:69-80.
54. Attal M., Lauwers-Cances V., Marit G., et al. Lenalidomide maintenance after stem-cell transplantation for multiple myeloma. N Engl J Med 2012, 366:1782-1791.
55. McCarthy P.L., Owzar K., Hofmeister C.C., et al. Lenalidomide after stem-cell transplantation for multiple myeloma. N Engl J Med 2012, 366:1770-1781.
56. Palumbo A., Hajek R., Delforge M., et al. Continuous lenalidomide treatment for newly diagnosed multiple myeloma. N Engl J Med 2012, 366:1759-1769.
57. Giagounidis A., Raza A., List A.F., et al. Analysis of second primary malignancies in lenalidomide-treated patients with ipss low- or int-1-risk myelodysplastic syndromes. ASH Annual Meeting Abstracts 2011, 118:1704.