Retinoids in pediatric onco-hematology: The model of acute promyelocytic leukemia and neuroblastoma

Advances in Therapy

Volumn 29, Issue 9, 2012, Pages 747-762

  Masetti, Riccardo ^a   Biagi, Carlotta ^a   Zama, Daniele ^a   Vendemini, Francesca ^a   Martoni, Anna ^a   Morello, William ^a   Gasperini, Pietro ^a   Pession, Andrea ^a  

^a UNIVERSITY OF BOLOGNA   (Italy)

Author keywords

13 Cis retinoic acid; All trans retinoic acid; Clinical trials; Neuroblastoma; Pediatric acute promyelocytic leukemia

Indexed keywords

ALPHA2A INTERFERON; ANTHRACYCLINE; ARSENIC TRIOXIDE; CYTARABINE; DAUNORUBICIN; ETOPOSIDE; FENRETINIDE; GRANULOCYTE COLONY STIMULATING FACTOR; IDARUBICIN; INTERLEUKIN 2; ISOTRETINOIN; MERCAPTOPURINE; METHOTREXATE; MITOXANTRONE; MONOCLONAL ANTIBODY; MONOCLONAL ANTIBODY CH14.18; PLACEBO; RETINOIC ACID; RETINOIC ACID RECEPTOR ALPHA; RETINOID; TIOGUANINE; UNCLASSIFIED DRUG; VORINOSTAT;

ABDOMINAL PAIN; APOPTOSIS; AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION; BLOOD TOXICITY; BONE PAIN; BRAIN PSEUDOTUMOR; CANCER CHEMOTHERAPY; CANCER IMMUNOTHERAPY; CANCER PROGNOSIS; CANCER RECURRENCE; CANCER STAGING; CARDIOTOXICITY; CELL DIFFERENTIATION; CHEILITIS; CHILDHOOD LEUKEMIA; DIARRHEA; DRUG BLOOD LEVEL; DRUG DOSE COMPARISON; DRUG DOSE ESCALATION; DRUG DOSE REDUCTION; DRUG FEVER; DRUG INDUCED HEADACHE; DRUG INTERMITTENT THERAPY; DRUG MECHANISM; DRUG MEGADOSE; DRY SKIN; DYSPNEA; EVENT FREE SURVIVAL; EYE DISEASE; GASTROINTESTINAL TOXICITY; GENE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HISTOPATHOLOGY; HUMAN; HYPERCALCEMIA; HYPERCHOLESTEROLEMIA; HYPERLEUKOCYTOSIS; HYPERTRANSAMINASEMIA; HYPERTRIGLYCERIDEMIA; HYPOTENSION; INFECTION; LEUKOCYTOSIS; LOW DRUG DOSE; LUNG INFILTRATE; MUCOSAL DRYNESS; MULTIPLE CYCLE TREATMENT; MYELOABLATIVE CONDITIONING; NAUSEA; NEPHROTOXICITY; NEUROBLASTOMA; PAPILLEDEMA; PATHOPHYSIOLOGY; PERICARDIAL EFFUSION; PLEURA EFFUSION; PROMYELOCYTIC LEUKEMIA; RASH; RETINOIC ACID RECEPTOR ALPHA GENE; RETINOIC ACID SYNDROME; REVIEW; SIDE EFFECT; SKIN TOXICITY; VOMITING; WEIGHT GAIN;

ANTINEOPLASTIC AGENTS; CHILD; DISEASE-FREE SURVIVAL; HUMANS; ISOTRETINOIN; LEUKEMIA, PROMYELOCYTIC, ACUTE; NEUROBLASTOMA; TRETINOIN;

EID: 84866489831     PISSN: 0741238X     EISSN: 18658652     Source Type: Journal    
DOI: 10.1007/s12325-012-0047-3     Document Type: Review

References (78)

1. Okuno M, Kojima S, Matsushima-Nishiwaki R, et al. Retinoids in cancer chemoprevention. Curr Cancer Drug Targets. 2004;4:285-98. (Pubitemid 38560076)
2. Sun SY, Lotan R. Retinoids and their receptors in cancer development and chemoprevention. Crit Rev Oncol Hematol. 2002;41:41-55. (Pubitemid 34051581)
3. Reynolds CP, Lemons RS. Retinoid therapy of childhood cancer. Hematol Oncol Clin North Am. 2001;15:867-910. (Pubitemid 34026975)
4. Bennett JM, Catovsky D, Daniel MT, et al. Proposed revised criteria for the classification of acute myeloid leukemia. A report of the French- American-British Cooperative Group. Ann Intern Med. 1985;103:620-5.
5. Stone RM, Mayer RJ. The unique aspects of acute promyelocytic leukemia. J Clin Oncol. 1990;8:1913-21. (Pubitemid 20382356)
6. Tallman MS, Kwaan HC. Reassessing the hemostatic disorder associated with acute promyelocitic leukemia. Blood. 1992;79:543-53.
7. Mistry AR, Wessel Pedersen E, Solomon E, Grimwade D. The molecular pathogenesis of acute promyelocytic leukaemia: Implications for the clinical management of the disease. Blood Rev. 2003;17:71-97. (Pubitemid 36372973)
8. Melnick A, Licht JD. Deconstructing a disease: RARα, its fusion partners, and their roles in the pathogenesis of acute promyelocytic leukemia. Blood. 1999;93:3167-215. (Pubitemid 29220886)
9. Fang J, Chen SJ, Tong JH, Wang ZG, Chen GQ, Chen Z. Treatment of acute promyelocytic leukemia with ATRA and As2O3: A model of molecular target-based cancer therapy. Cancer Biol Ther. 2002;1:614-20.
10. de Thé H, Chen Z. Acute promyelocytic leukaemia: Novel insights into the mechanisms of cure. Nat Rev Cancer. 2010;10:775-83.
11. Gregory J, Feusner J. Acute promyelocytic leukaemia in children. Curr Oncol Rep. 2009;11:439-45.
12. Fenaux P, Chomienne C, Degos L. All-trans retinoic acid and chemotherapy in the treatment of acute promyelocytic leukemia. Semin Hematol. 2001;38:13.
13. Huang ME, Ye YC, Chen SR, et al. Use of all- trans retinoic acid in the treatment of acute promyelocytic leukemia. Blood. 1988;72:567-72.
14. Reynolds CP, Matthay KK, Villablanca JG, Maurer BJ. Retinoid therapy of high-risk neuroblastoma. Cancer Lett. 2003;197:185-92. (Pubitemid 36860009)
15. Khan AA, Villablanca JG, Reynolds CP, Avramis VI. Pharmacokinetic studies of 13-cis-retinoic acid in pediatric patients with neuroblastoma following bone marrow transplantation. Cancer Chemother Pharmacol. 1996;39:34-41. (Pubitemid 26400658)
16. Reynolds CP, Schindler PF, Jones DM, Gentile JL, Proffitt RT, Einhorn PA. Comparison of 13-cis- retinoic acid to trans-retinoic acid using human neuroblastoma cell lines. Prog Clin Biol Res. 1994;385:237.
17. Reynolds CP, Seeger RC. Neuroblastoma. In: Haskell CM ed. Cancer Treatment. Philadelphia: WB Saunders; 2000:1214.
18. Cotterill SJ, Pearson AD, Pritchard J, et al. Late relapse and prognosis for neuroblastoma patients surviving 5 years or more: A report from the European Neuroblastoma Study Group "Survey". Med Pediatr Oncol. 2001;36:235-8. (Pubitemid 32053039)
19. Matthay K, Villablanca JG, Seeger RC, et al. Treatment of high risk neuroblastoma with intensive chemotherapy, radiotherapy, autologous bone marrow transplantation, and 13-cisretinoic acid. N Engl J Med. 1999;341:1165.
20. Testi AM, Biondi A, Lo-Coco F, et al. GIMEMA-AIEOP AIDA protocol for the treatment of newly diagnosed acute promyelocytic leukemia (APL) in children. Blood. 2005;106:447-53. (Pubitemid 40981236)
21. Ortega JJ, Madero L, Martín L, et al. Treatment with all-trans retinoic acid and anthracycline monochemotherapy for children with acute promyelocytic leukemia: A multicenter study by the PETHEMA group. J Clin Oncol. 2005;23:7632- 40. (Pubitemid 46291827)
22. De Botton S, Coiteux V, Chevret S, et al. Outcome of childhood acute promyelocytic leukemia with all-trans-retinoic acid and chemotherapy. J Clin Oncol. 2004;22:1404-12. (Pubitemid 41103622)
23. Creutzig U, Zimmermann M, Dworzak M, et al. Favourable outcome of patients with childhood acute promyelocytic leukaemia after treatment with reduced cumulative anthracycline doses. Br J Haematol. 2010;149:399-409.
24. Chen ZX, Xue YQ, Zhang R, et al. A clinical and experimental study on all-trans retinoic acid-treated acute promyelocytic leukemia patients. Blood. 1991;78:1413-9.
25. Castaigne S, Chomienne C, Daniel MT, et al. All-trans retinoic acid as a differentiation therapy for acute promyelocytic leukemia. I. Clinical results. Blood. 1990;76:1704-9.
26. Frankel SR, Eardley A, Heller G, et al. All-trans retinoic acid for acute promyelocytic leukemia. Results of the New York Study. Ann Intern Med. 1994;120:278-86. (Pubitemid 24050023)
27. Fenaux P, Le Deley MC, Castaigne S, et al. Effect of all-trans-retinoic acid in newly diagnosed acute promyelocytic leukemia. Results of a multicenter randomized trial. European APL 91 Group. Blood. 1993;82:3241-9. (Pubitemid 23356690)
28. Fenaux P, Chevret S, Guerci A, et al. Long-term follow-up confirms the benefit of all-trans retinoic acid in acute promyelocytic leukemia. European APL group. Leukemia. 2000;14:1371-7. (Pubitemid 30599316)
29. Chen GQ, Shen ZX, Wu F, et al. Pharmacokinetics and efficacy of low-dose all-trans retinoic acid in the treatment of acute promyelocytic leukemia. Leukemia. 1996;10:825-8. (Pubitemid 26168400)
30. Tallman MS, Andersen JW, Schiffer CA, et al. All-trans-retinoic acid in acute promyelocytic leukemia. N Engl J Med. 1997;337:1639.
31. Fenaux P, Chastang C, Chevret S, et al. A randomized comparison of all transretinoic acid (ATRA) followed by chemotherapy and ATRA plus chemotherapy and the role of maintenance therapy in newly diagnosed acute promyelocytic leukemia. The European APL Group. Blood. 1999;94:1192-2000.
32. Avvisati G, Lo-Coco F, Paoloni FP et al. AIDA 0493 protocol for newly diagnose acute promyelocitic leukemia: very long term results and role of maintenance. Blood. 2001;117:4716-25.
33. Testi AM, Foa R, Tomei G, et al. GIMEMA-AIEOP AIDA protocols for the treatment of newly diagnosed acute promyelocytic leukemia (APL) in children: Analysis of 247 patients enrolled in two sequential Italian multicenter trials [abstract]. Paper presented at: 52nd American Society of Hematology (ASH) Annual Meeting; December 4-7, 2010; Orlando, FL. Abstract 871.
34. Mahmoud HH, Hurwitz CA, Roberts WM, Santana VM, Ribeiro RC, Krance RA. Tretinoin toxicity in children with acute promyelocytic leukaemia. Lancet. 1993;342:1394-5. (Pubitemid 23361242)
35. Testi A. Treatment study for children and adolescents with acute promyelocitic leukemia. ClinicalTrials.gov. Available at: http://clinicaltrials. gov/ct2/show/NCT01226303?term=ICC+APL&ra nk=1. Accessed Aug 6 2012.
36. Gregory JJ. Combination chemotherapy in treating young patients with newly diagnosed acute promyelocytic leukemia. ClinicalTrials.gov. Available at: http://clinicaltrials.gov/ct2/show/NCT00866918?term=NCT00866918&rank=1. Accessed Aug 6 2012.
37. Niu C, Yan H, Yu T, et al. Studies on treatment of acute promyelocytic leukemia with arsenic trioxide: Remission induction, follow-up, and molecular monitoring in 11 newly diagnosed and 47 relapsed acute promyelocytic leukemia patients. Blood. 1999;94:3315-24. (Pubitemid 29536124)
38. Soignet S, Frankel S, Douer D, et al. United States multicenter study of arsenic trioxide in relapsed acute promyelocytic leukemia. J Clin Oncol. 2001;18:3852-60. (Pubitemid 32880064)
39. Powell BL, Tallman MS, Couban S, Coutre SE. Tretinoin, cytarabine, and daunorubicin with or without arsenic trioxide followed by tretinoin with or without mercaptopurine and methotrexate in treating patients with acute promyelocytic leukemia. ClinicalTrials.gov. Available at: http://clinicaltrials. gov/ct2/show/NCT00003934?term=N CT00003934&rank=1. Accessed Aug 6 2012.
40. Gore SD. Combination chemotherapy in treating patients with acute promyelocytic leukemia. ClinicalTrials.gov. Available at: http://clinicaltrials. gov/ct2/show/NCT00276601?term=NCT00276601 &rank=1. Accessed Aug 6 2012.
41. Jurcic J. Combined tretinoin and arsenic trioxide for patients with newly diagnosed acute promyelocytic leukemia followed by risk-adapted postremission therapy. ClinicalTrials.gov. Available at: http://clinicaltrials.gov/ct2/show/ NCT01404949?term=N CT01404949&rank=1. Accessed Aug 6 2012.
42. Ravandi-Kashani F. Acute promyelocytic leukemia (APL) treated with ATRA, arsenic trioxide and gemtuzumab ozogamicin. ClinicalTrials.gov. Available at: http://clinicaltrials.gov/ct2/show/NCT01409161?term=NCT01409161&rank=1. Accessed Aug 6 2012.
43. Ravandi-Kashani F. All-trans retinoic acid, and arsenic +/- gemtuzumab. ClinicalTrials.gov. Available at: http://clinicaltrials.gov/ct2/show/ NCT00413166?term=NCT00413166&rank=1. Accessed Aug 6 2012.
44. Lancet JE, Komrokji R. Willman CL, Slovak ML, Gemtuzumab and combination chemotherapy in treating patients with previously untreated acute promyelocytic leukemia. ClinicalTrials.gov. Available at: http://clinicaltrials.gov/ct2/show/ NCT00551460?term=NCT00551460&rank=1. Accessed Aug 6 2012.
45. Jurcic J. Chemotherapy plus monoclonal antibody in treating patients with acute promyelocytic leukemia. ClinicalTrials.gov. Available at: http://clinicaltrials.gov/ct2/show/NCT00016159?term=N CT00016159&rank=1. Accessed Aug 6 2012.
46. Finklestein JZ, Krailo MD, Lenarsky C, et al. 13-cisretinoic acid (NSC 122758) in the treatment of children with metastatic neuroblastoma unresponsive to conventional chemotherapy: Report from the Children's Cancer Study Group. Med Pediatr Oncol. 1992;20:307-11.
47. Kohler JA, Imeson J, Ellershaw C, Lie SO. A randomized trial of 13-cis retinoic acid in children with advanced neuroblastoma after high-dose therapy. Br J Cancer. 2000;83:1124-7.
48. Villablanca JG, Khan AA, Avramis VI, et al. Phase I trial of 13-cis retinoic acid in children with neuroblastoma following bone marrow transplantation. J Clin Oncol. 1995;13:894-901.
49. Villablanca JG, London WB, Naranjo A, et al. Phase II study of oral capsular 4-20 hydroxyphenylretinamide (4-HPR/fenretinide) in pediatric patients with refractory or recurrent neuroblastoma: A report from the Children's Oncology Group. Clin Cancer Res. 2011;17:6858-66.
50. Yu AL, Gilman AL, Ozkaynak MF, et al. Children's Oncology Group. Anti-GD2 antibody with GM-CSF, interleukin-2, and isotretinoin for neuroblastoma. N Engl J Med. 2010;363:1324-34.
51. Matthay KK, Reynolds CP, Seeger RC, et al. Long-term results for children with high-risk neuroblastoma treated on a randomized trial of myeloablative therapy followed by 13-cis-retinoic acid: A children's oncology group study. J Clin Oncol. 2009;27:1007-13.
52. Delia D, Aiello A, Lombardi L, Pelicci et al. N-(4- hydroxyphenyl) retinamide induces apoptosis of malignant hemopoietic cell lines including those unresponsive to retinoic acid. Cancer Res. 1993;53:6036-41.
53. Reynolds CP, Wang Y, Melton LJ, Einhorn PA, Slamon DJ, Maurer BJ. Retinoic-acid resistant neuroblastoma cell lines show altered MYC regulation and high sensitivity to fenretinide. Med Pediatr Oncol. 2000;35:597-602.
54. Villablanca JG, Krailo MD, Ames MM, Reid JM, Reaman GH, Reynolds CP. Phase I trial of oral fenretinide in children with high-risk solid tumors: A report from the Children's Oncology Group (CCG 09709). J Clin Oncol. 2006;24:3423-30. (Pubitemid 46638899)
55. Villablanca JG, Ames MM, Reid JM, Bagniewski PG, Krailo M, Reynolds CP. Phase I trial of oral [N-(-4- hydroxyphenyl)retinamide] (4-HPR) in children with resistant/recurrent solid tumors: A Children's Cancer Group Study (CCG 09709) [abstract]. Proc Am Soc Clin Oncol. 2002;21. Abstract 1588.
56. Kushner BH. High-dose 3F8/GM-CSF immunotherapy plus 13-cis-retinoic acid for consolidation of first remission after non-myeloablative therapy in patients with high- risk neuroblastoma. ClinicalTrials.gov. Available at: http://clinicaltrials.gov/ct2/show/NCT01183429?term. Accessed Aug 6 2012.
57. Kushner B. High-dose 3F8/GM-CSF Immunotherapy plus 13-cis-retinoic acid for consolidation of second or greater remission of high-risk neuroblastoma. ClinicalTrials.gov. Available at: http://clinicaltrials.gov/ct2/show/ NCT01183884?term. Accessed Aug 6 2012.
58. Kushner B. High-dose 3F8/GM-CSF Immunotherapy plus 13-cis-retinoic acid for consolidation of first remission after myeloablative therapy and autologous stem-cell transplantation. ClinicalTrials. gov. Available at: http://clinicaltrials.gov/ct2/show/NCT01183416?term. Accessed Aug 6 2012.
59. Kushner B. High-dose 3F8/GM-CSF immunotherapy plus 13-cis-retinoic acid for primary refractory neuroblastoma in bone marrow. ClinicalTrials. gov. Available at: http://clinicaltrials.gov/ct2/show/NCT01183897?term. Accessed Aug 6 2012.
60. Cheung NK, Saarinen UM, Neely JE, Landmeier B, Donovan D, Coccia PF. Monoclonal antibodies to a glycolipid antigen on human neuroblastoma cells. Cancer Res. 1985;45:2642-9. (Pubitemid 15024436)
61. Schulz G, Cheresh DA, Varki NM, Yu A, Staffileno LK, Reisfeld RA. Detection of ganglioside GD2 in tumor tissues and sera of neuroblastoma. Cancer Res. 1984;44:5914-20. (Pubitemid 15183611)
62. Kushner BH, Cheung NK. GM-CSF enhances 3F8 monoclonal antibody-dependent cellular cytotoxicity against human melanoma and neuroblastoma. Blood. 1989;73:1936-41. (Pubitemid 19145127)
63. Cheung NK, Lazarus H, Miraldi FD, et al. Ganglioside GD2 specific monoclonal antibody 3F8: A phase I study in patients with neuroblastoma and malignant melanoma. J Clin Oncol. 1987;5:1430-40. (Pubitemid 18049879)
64. Cheung NK, Kushner BH, Yeh SD, Larson SM. 3F8 monoclonal antibody treatment of patients with stage 4 neuroblastoma: A phase II study. Int J Oncol. 1998;12:1299-306. (Pubitemid 28239940)
65. Furman WL, Fairclough DL, Huhn RD, et al. Therapeutic effects and pharmacokinetics of recombinant human granulocyte-macrophage colony-stimulating factor in childhood cancer patients receiving myelosuppressive chemotherapy. J Clin Oncol. 1991;9:1022-8.
66. Barker E, Mueller BM, Handgretinger R, Herter M, Yu AL, Reisfeld RA. Effect of a chimeric anti-ganglioside GD2 antibody on cell-mediated lysis of human neuroblastoma cells. Cancer Res. 1991;51:144-9.
67. Kushner BH, Kramer K, Cheung NK. Phase II trial of anti-GD2 monoclonal antibody 3F8 and granulocyte-macrophage colony-stimulating factor for neuroblastoma. J Clin Oncol. 2001;19:4189-94. (Pubitemid 33081635)
68. Park JR. Vorinostat and isotretinoin in treating patients with high-risk refractory or recurrent neuroblastoma. ClinicalTrials.gov. Available at: http://clinicaltrials.gov/ct2/show/NCT01208454?term. Accessed Aug 6 2012.
69. Park JR; Children's Oncology Group. Vorinostat with or without isotretinoin in treating young patients with recurrent or refractory solid tumors, lymphoma, or leukemia. ClinicalTrials.gov. Available at: http://clinicaltrials.gov/ct2/show/study/NCT00217412?term. Accessed Aug 6 2012.
70. Muhlethaler-Mottet A, Meier R, Flahaut M, et al. Complex molecular mechanisms cooperate to mediate histone deacetylase inhibitors anti-tumour activity in neuroblastoma cells. Mol Cancer. 2008;7:55.
71. Keshelava N, Houghton PJ, Morton CL, et al. Initial testing (stage 1) of vorinostat (SAHA) by the pediatric preclinical testing program. Pediatr Blood Cancer. 2009;53:505-8.
72. Spiller SE, Ditzler SH, Pullar BJ, Olson JM. Response of preclinical medulloblastoma models to combination therapy with 13-cis retinoic acid and suberoylanilide hydroxamic acid (SAHA). J Neurooncol. 2008;87:133-14.
73. Fouladi M, Park JR, Stewart CF, et al. Pediatric phase I trial and pharmacokinetic study of vorinostat: A Children's Oncology Group phase I consortium report. J Clin Oncol. 2010;28:3623-9.
74. National Cancer Institute (NCI). A phase II trial of all-trans-retinoic acid in combination with interferon-alpha 2a in children with recurrent neuroblastoma or Wilms' tumor. ClinicalTrials.gov. Available at: http://clinicaltrials.gov/ct2/show/NCT00001509?term. Accessed Aug 6 2012.
75. Adamson PC, Reaman G, Finklestein JZ, et al. Phase I trial and pharmacokinetic study of alltransretinoic acid administered on an intermittent schedule in combination with interferon-alpha2a in pediatric patients with refractory cancer. J Clin Oncol. 1997;15:3330-7. (Pubitemid 27485851)
76. O'Dorisio MS. 90Y DOTA/retinoic acid for neuroblastoma and neuroendocrine tumor (NET). ClinicalTrials.gov. Available at: http://clinicaltrials. gov/ct2/show/study/NCT01048086?term. Accessed Aug 6 2012.
77. Menda Y, O'Dorisio MS, Kao S, et al. Phase I trial of 90 Y-DOTATOC therapy in children and young adults with refractory solid tumors that express somatostatin receptors. J Nucl Med. 2010;51:1524-31.
78. Veal G; University of Newcastle Upon-Tyne. Isotretinoin in treating young patients with high-risk neuroblastoma. ClinicalTrials.gov. Available at: http://clinicaltrials.gov/ct2/show/NCT00939965?term. Accessed Aug 6 2012.