Risk and surrogate benefit for pediatric Phase I trials in oncology: A systematic review with meta-analysis
Jonathan Kimmelman and colleagues report findings from their meta analysis that shows that adverse effects and response rates in phase 1 cancer clinical trials are similar for children as for adults.
Vyšlo v časopise:
Risk and surrogate benefit for pediatric Phase I trials in oncology: A systematic review with meta-analysis. PLoS Med 15(2): e32767. doi:10.1371/journal.pmed.1002505
Kategorie:
Research Article
prolekare.web.journal.doi_sk:
https://doi.org/10.1371/journal.pmed.1002505
Souhrn
Jonathan Kimmelman and colleagues report findings from their meta analysis that shows that adverse effects and response rates in phase 1 cancer clinical trials are similar for children as for adults.
Zdroje
1. Heron M. Deaths: leading causes for 2013. Natl Vital Stat Rep. 2016;65:1–95.
2. Doussau A, Asselain B, Le Deley MC, Geoerger B, Doz F, Vassal G, et al. Innovations for phase I dose-finding designs in pediatric oncology clinical trials. Contemp Clin Trials. 2016;47:217–27. doi: 10.1016/j.cct.2016.01.009 26825023
3. Moreno L, Pearson ADJ, Paoletti X, Jimenez I, Geoerger B, Kearns PR, et al. Early phase clinical trials of anticancer agents in children and adolescents—an ITCC perspective. Nat Rev Clin Oncol. 2017;14:497–507. doi: 10.1038/nrclinonc.2017.59 28508875
4. van Hasselt JG, van Eijkelenburg NK, Beijnen JH, Schellens JH, Huitema AD. Optimizing drug development of anti-cancer drugs in children using modelling and simulation. Br J Clin Pharmacol. 2013;76:30–47. doi: 10.1111/bcp.12062 23216601
5. Rieder M, Hawcutt D. Design and conduct of early phase drug studies in children: challenges and opportunities. Br J Clin Pharmacol. 2016;82:1308–14. doi: 10.1111/bcp.13058 27353241
6. Miller FG, Joffe S. Benefit in phase 1 oncology trials: therapeutic misconception or reasonable treatment option? Clin Trials. 2008;5:617–23. doi: 10.1177/1740774508097576 19029210
7. Lad PM, Dahl R. Defining the research category in pediatric Phase I oncology trials. Account Res. 2015;22:139–47. doi: 10.1080/08989621.2014.919229 25635846
8. Schuklenk U, Lowry C. Terminal illness and access to phase 1 experimental agents, surgeries and devices: reviewing the ethical arguments. Br Med Bull. 2008;89:7–22. doi: 10.1093/bmb/ldn048 19056723
9. Miller M. Phase I cancer trials: a collusion of misunderstanding. Hastings Cent Rep. 2000;30:34–43.
10. Agrawal M, Emanuel EJ. Ethics of phase 1 oncology studies: reexamining the arguments and data. JAMA. 2003;290:1075–82. doi: 10.1001/jama.290.8.1075 12941681
11. Kimmelman J. Is participation in cancer Phase I trials really therapeutic? J Clin Oncol. 2017;35:135–8. doi: 10.1200/JCO.2016.67.9902 27646944
12. King NM. Defining and describing benefit appropriately in clinical trials. J Law Med Ethics. 2000;28:332–43. 11317426
13. Barfield RC, Church C. Informed consent in pediatric clinical trials. Curr Opin Pediatr. 2005;17:20–4. 15659958
14. Kodish E. Informed consent for pediatric research: is it really possible? J Pediatr. 2003;142:89–90. doi: 10.1067/mpd.2003.64 12584521
15. Simon C, Eder M, Raiz P, Zyzanski S, Pentz R, Kodish ED. Informed consent for pediatric leukemia research: clinician perspectives. Cancer. 2001;92:691–700. 11505417
16. Horng S, Emanuel EJ, Wilfond B, Rackoff J, Martz K, Grady C. Descriptions of benefits and risks in consent forms for phase 1 oncology trials. N Engl J Med. 2002;347:2134–40. doi: 10.1056/NEJMsa021182 12501226
17. Eder ML, Yamokoski AD, Wittmann PW, Kodish ED. Improving informed consent: suggestions from parents of children with leukemia. Pediatrics. 2007;119:849–59.
18. Yap TY, Yamokoski AD, Hizlan S, Zyzanski SJ, Angiolillo AL, Rheingold SR. Informed consent for pediatric phase 1 cancer trials: physicians’ perspectives. Cancer. 2010;116:3244–50. doi: 10.1002/cncr.25158 20564626
19. Giesbertz NA, Bredenoord AL, van Delden JJ. Clarifying assent in pediatric research. Eur J Hum Genet. 2014;22:266–9. doi: 10.1038/ejhg.2013.119 23756442
20. Waligora M, Dranseika V, Piasecki J. Child’s assent in research: age threshold or personalisation? BMC Med Ethics. 2014;15:44. doi: 10.1186/1472-6939-15-44 24923433
21. Jansen-van der Weide MC, Caldwell PH, Young B, de Vries MC, Willems DL, Van’t Hoff W, et al. Clinical trial decisions in difficult circumstances: parental consent under time pressure. Pediatrics. 2015;136:e983–92. doi: 10.1542/peds.2014-3402 26416935
22. Piasecki J, Waligora M, Dranseika V. Non-beneficial pediatric research: individual and social interests. Med Health Philos. 2015,18:103–12.
23. Simon R, Freidlin B, Rubinstein L, Arbuck SG, Collins J, Christian MC. Accelerated titration designs for phase I clinical trials in oncology. J Natl Cancer Inst. 1997;89:1138–47. 9262252
24. Ross L. Phase I research and the meaning of direct benefit. J Pediatr. 2006;149:S20–4. doi: 10.1016/j.jpeds.2006.04.046 16829237
25. Paoletti X, Geoerger B, Doz F, Baruchel A, Lokiec F, Le Tourneau C. A comparative analysis of paediatric dose-finding trials of molecularly targeted agent with adults’ trials. Eur J Cancer. 2013;49:2392–402. doi: 10.1016/j.ejca.2013.02.028 23540589
26. Lee DP, Skolnik JM, Adamson PC. Pediatric phase I trials in oncology: an analysis of study conduct efficiency. J Clin Oncol. 2005;23:8431–41. doi: 10.1200/JCO.2005.02.1568 16293874
27. Onar A, Kocak M, Boyett JM. Continual reassessment method vs. traditional empirically based design: modifications motivated by Phase I trials in pediatric oncology by the Pediatric Brain Tumor Consortium. J Biopharm Stat. 2009;19:437–55. doi: 10.1080/10543400902800486 19384687
28. Vassal G, Zwaan CM, Ashley D, Le Deley MC, Hargrave D, Blanc P, et al. New drugs for children and adolescents with cancer: the need for novel development pathways. Lancet Oncol. 2013;14:e117–24. doi: 10.1016/S1470-2045(13)70013-5 23434337
29. Skolnik JM, Barrett JS, Jayaraman B, Patel D, Adamson PC. Shortening the timeline of pediatric phase I trials: the rolling six design. J Clin Oncol 2008;26:190–5. doi: 10.1200/JCO.2007.12.7712 18182661
30. Waligora M, Bala MM, Koperny M, Jaeschke RR, Kargul A, Piasecki J, et al. Risk and benefits of pediatric phase I trials in oncology, 2004 through 2014: a systematic review. CRD42015015961. PROSPERO; 2015.
31. Eisenhauer EA, Twelves C, Buyse M. Phase I cancer clinical trials: a practical guide. 1st ed. Oxford: Oxford University Press; 2006.
32. Karp DD, Falchook GS. Handbook of targeted cancer therapy. Sacramento (CA): Wolters Kluwer; 2014.
33. Cremolini C, Antoniotti C, Pietrantonio F, Berenato R, Tampellini M, Baratelli C, et al. Surrogate endpoints in second-line trials of targeted agents in metastatic colorectal cancer: a literature-based systematic review and meta-analysis. Cancer Res Treat. 2017;49:834–45. doi: 10.4143/crt.2016.249 27857020
34. Wilson MK, Karakasis K, Oza AM. Outcomes and endpoints in trials of cancer treatment: the past, present, and future. Lancet Oncol. 2015;16:e32–42. doi: 10.1016/S1470-2045(14)70375-4 25638553
35. Bruzzi P, Del Mastro L, Sormani MP, Bastholt L, Danova M, Focan C, et al. Objective response to chemotherapy as a potential surrogate end point of survival in metastatic breast cancer patients. J Clin Oncol. 2005;23:5117–25. doi: 10.1200/JCO.2005.02.106 15955906
36. Agarwal SK, Mangal N, Menon RM, Freise KJ, Salem AH. Response rates as predictors of overall survival: a meta-analysis of acute myeloid leukemia trials. J Cancer. 2017;8:1562–7. doi: 10.7150/jca.18686 28775775
37. Han K, Ren M, Wick W, Abrey L, Das A, Jin J, et al. Progression-free survival as a surrogate endpoint for overall survival in glioblastoma: a literature-based meta-analysis from 91 trials. Neuro Oncol. 2014;16:696–706. doi: 10.1093/neuonc/not236 24335699
38. Horstmann E, McCabe MS, Grochow L, Yamamoto S, Rubinstein L, Budd T, et al. Risks and benefits of phase 1 oncology trials, 1991 through 2002. N Engl J Med. 2005;352:895–904. doi: 10.1056/NEJMsa042220 15745980
39. Wong HH, Barton C, Acton G, McLeod R, Halford S. Trends in the characteristics, dose-limiting toxicities and efficacy of phase I oncology trials: the Cancer Research UK experience. Eur J Cancer. 2016;66:9–16. doi: 10.1016/j.ejca.2016.07.004 27514008
40. Roberts TG Jr, Goulart BH, Squitieri L, Stallings SC, Halpern EF, Chabner BA, et al. Trends in the risks and benefits to patients with cancer participating in phase 1 clinical trials. JAMA. 2004;292:2130–40. doi: 10.1001/jama.292.17.2130 15523074
41. Italiano A, Massard C, Bahleda R, Vataire AL, Deutsch E, Magné N, et al. Treatment outcome and survival in participants of phase I oncology trials carried out from 2003 to 2006 at Institut Gustave Roussy. Ann Oncol. 2008;19:787–92. doi: 10.1093/annonc/mdm548 18042834
42. Gupta S, Hunsberger S, Boerner SA, Rubinstein L, Royds R, Ivy P, et al. Meta-analysis of the relationship between dose and benefit in phase I targeted agent trials. J Natl Cancer Inst. 2012;104:1860–6. doi: 10.1093/jnci/djs439 23169991
43. Kodish E. Pediatric ethics and early-phase childhood cancer research: conflicted goals and the prospect of benefit. Account Res. 2003;10:17–25. doi: 10.1080/08989620300502 14552299
44. Doussau A, Asselain B, Le Deley MC, Geoerger B, Doz F, Vassal G, et al. Dose-finding designs in pediatric phase I clinical trials: comparison by simulations in a realistic timeline framework. Contemp Clin Trials. 2012;33:657–65. doi: 10.1016/j.cct.2011.11.015 22521954
45. Brennan RC, Furman W, Mao S, Wu J, Turner DC, Stewart CF, et al. Phase I dose escalation and pharmacokinetic study of oral gefitinib and irinotecan in children with refractory solid tumors. Cancer Chemother Pharmacol. 2014;74:1191–8. doi: 10.1007/s00280-014-2593-7 25257509
46. Jones CW, Keil LG, Holland WC, Caughey MC, Platts-Mills TF. Comparison of registered and published outcomes in randomized controlled trials: a systematic review. BMC Med. 2015;13:282. doi: 10.1186/s12916-015-0520-3 26581191
47. Rosati P, Porzsolt F, Ricciotti G, Testa G, Inglese R, Giustini F, et al. Major discrepancies between what clinical trial registries record and paediatric randomised controlled trials publish. Trials. 2016;17:430. doi: 10.1186/s13063-016-1551-6 27659549
48. Killeen S, Sourallous P, Hunter IA, Hartley JE, Grady HL. Registration rates, adequacy of registration, and a comparison of registered and published primary outcomes in randomized controlled trials published in surgery journals. Ann Surg. 2014;259:193–6. doi: 10.1097/SLA.0b013e318299d00b 23732270
49. Hartung DM, Zarin DA, Guise JM, McDonagh M, Paynter R, Helfand M. Reporting discrepancies between the ClinicalTrials.gov results database and peer-reviewed publications. Ann Intern Med. 2014;160:477–83. doi: 10.7326/M13-0480 24687070
50. Hay M, Thomas DW, Craighead JL, Economides C, Rosenthal J. Clinical development success rates for investigational drugs. Nat Biotechnol. 2014;32:40–51. doi: 10.1038/nbt.2786 24406927
51. Biotechnology Innovation Organization, Biomedtracker, Amplion. Clinical development success rates 2006–2015. Washington (DC): Biotechnology Innovation Organization; 2016 Jun.
52. Weber JS, Levit LA, Adamson PC, Bruinooge S, Burris HA 4th, Carducci MA, et al. American Society of Clinical Oncology policy statement update: the critical role of phase I trials in cancer research and treatment. J Clin Oncol. 2015;33:278–84. doi: 10.1200/JCO.2014.58.2635 25512456
53. US Department of Health and Human Services. Code of Federal Regulations. Title 45—public welfare. Part 46—protection of human subjects. Subpart D—additional protections for children involved as subjects in research. Washington (DC): Office of the Federal Register; 1983 Mar 8 [cited 2018 Jan 16]. Available from: https://www.gpo.gov/fdsys/pkg/CFR-2016-title45-vol1/xml/CFR-2016-title45-vol1-part46.xml.
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