Influence of companion diagnostics on efficacy and safety of targeted anti-cancer drugs: Systematic review and meta-analyses

Oncotarget

Volumn 6, Issue 37, 2015, Pages 39538-39549

  Ocana, Alberto ^a   Ethier, Josee Lyne ^b   Díez González, Laura ^a   Corrales Sánchez, Verónica ^a   Srikanthan, Amirrtha ^b   Gascón Escribano, María J ^a   Templeton, Arnoud J ^c   Vera Badillo, Francisco ^b   Seruga, Bostjan ^d   Niraula, Saroj ^e   Pandiella, Atanasio ^f   Amir, Eitan ^b  

^a HOSPITAL GENERAL UNIVERSITARIO DE ALBACETE   (Spain)

^b UNIVERSITY OF TORONTO   (Canada)

^c CANTONAL HOSPITAL ST GALLEN   (Switzerland)

^d INSTITUTE OF ONCOLOGY   (Slovenia)

^e UNIVERSITY OF MANITOBA   (Canada)

^f UNIVERSITY OF SALAMANCA   (Spain)

Author keywords

Cancer drugs; Companion diagnostics; Efficacy; Trial design

Indexed keywords

ANTINEOPLASTIC AGENT; PLACEBO;

ADVERSE DRUG REACTION; ANEMIA; ARTICLE; ASTHENIA; COMPANION DIAGNOSTICS; DEATH; DIARRHEA; DRUG SAFETY; DRUG TOLERABILITY; DRUG WITHDRAWAL; DYSPNEA; FATIGUE; HEART DISEASE; HUMAN; HYPERTENSION; LUNG DISEASE; META ANALYSIS; MOLECULARLY TARGETED THERAPY; NEUROPATHY; NEUTROPENIA; OVERALL SURVIVAL; PERSONALIZED MEDICINE; PROGRESSION FREE SURVIVAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); SKIN TOXICITY; SOLID TUMOR; STOMATITIS; SYSTEMATIC REVIEW; THROMBOCYTOPENIA; VOMITING; CHEMICALLY INDUCED; DRUG APPROVAL; FOOD AND DRUG ADMINISTRATION; NEOPLASMS; NEUROLOGIC DISEASE; PROCEDURES; SKIN DISEASE; TREATMENT OUTCOME; UNITED STATES;

ANTINEOPLASTIC AGENTS; DIARRHEA; DRUG APPROVAL; HUMANS; MOLECULAR TARGETED THERAPY; NEOPLASMS; NERVOUS SYSTEM DISEASES; PRECISION MEDICINE; RANDOMIZED CONTROLLED TRIALS AS TOPIC; SKIN DISEASES; TREATMENT OUTCOME; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION; VOMITING;

EID: 84950151454     PISSN: None     EISSN: 19492553     Source Type: Journal    
DOI: 10.18632/oncotarget.5946     Document Type: Article

References (58)

1. Wood LD, Parsons DW, Jones S, Lin J, Sjoblom T, Leary RJ, Shen D, Boca SM, Barber T, Ptak J, Silliman N, Szabo S, Dezso Z, Ustyanksky V, Nikolskaya T, Nikolsky Y, et al. The genomic landscapes of human breast and colorectal cancers. Science. 2007; 318: 1108-1113.
2. Vogelstein B, Papadopoulos N, Velculescu VE, Zhou S, Diaz LA, Jr. and Kinzler KW. Cancer genome landscapes. Science. 2013; 339:1546-1558.
3. Kris MG, Johnson BE, Berry LD, Kwiatkowski DJ, Iafrate AJ, Wistuba, II, Varella-Garcia M, Franklin WA, Aronson SL, Su PF, Shyr Y, Camidge DR, Sequist LV, Glisson BS, Khuri FR, Garon EB, et al. Using multiplexed assays of oncogenic drivers in lung cancers to select targeted drugs. Jama. 2014; 311:1998-2006.
4. Ocana A and Pandiella A. Personalized therapies in the cancer "omics" era. Molecular cancer. 2010; 9:202.
5. Rubin EH, Allen JD, Nowak JA and Bates SE. Developing precision medicine in a global world. Clinical cancer research: an official journal of the American Association for Cancer Research. 2014; 20:1419-1427.
6. Mansfield EA. FDA perspective on companion diagnostics: an evolving paradigm. Clinical cancer research: an official journal of the American Association for Cancer Research. 2014; 20:1453-1457.
7. Demetri GD, van Oosterom AT, Garrett CR, Blackstein ME, Shah MH, Verweij J, McArthur G, Judson IR, Heinrich MC, Morgan JA, Desai J, Fletcher CD, George S, Bello CL, Huang X, Baum CM, et al. Efficacy and safety of sunitinib in patients with advanced gastrointestinal stromal tumour after failure of imatinib: a randomised controlled trial. Lancet. 2006; 368:1329-1338.
8. Demetri GD, Reichardt P, Kang YK, Blay JY, Rutkowski P, Gelderblom H, Hohenberger P, Leahy M, von Mehren M, Joensuu H, Badalamenti G, Blackstein M, Le Cesne A, Schoffski P, Maki RG, Bauer S, et al. Efficacy and safety of regorafenib for advanced gastrointestinal stromal tumours after failure of imatinib and sunitinib (GRID): an international, multicentre, randomised, placebo-controlled, phase 3 trial. Lancet. 2013; 381:295-302.
9. Elisei R, Schlumberger MJ, Muller SP, Schoffski P, Brose MS, Shah MH, Licitra L, Jarzab B, Medvedev V, Kreissl MC, Niederle B, Cohen EE, Wirth LJ, Ali H, Hessel C, Yaron Y, et al. Cabozantinib in progressive medullary thyroid cancer. Journal of clinical oncology: official journal of the American Society of Clinical Oncology. 2013; 31:3639-3646.
10. Wells SA, Jr., Robinson BG, Gagel RF, Dralle H, Fagin JA, Santoro M, Baudin E, Elisei R, Jarzab B, Vasselli JR, Read J, Langmuir P, Ryan AJ and Schlumberger MJ. Vandetanib in patients with locally advanced or metastatic medullary thyroid cancer: a randomized, double-blind phase III trial. Journal of clinical oncology: official journal of the American Society of Clinical Oncology. 2012; 30:134-141.
11. Karapetis CS, Khambata-Ford S, Jonker DJ, O'Callaghan CJ, Tu D, Tebbutt NC, Simes RJ, Chalchal H, Shapiro JD, Robitaille S, Price TJ, Shepherd L, Au HJ, Langer C, Moore MJ and Zalcberg JR. K-ras mutations and benefit from cetuximab in advanced colorectal cancer. The New England journal of medicine. 2008; 359:1757-1765.
12. Amado RG, Wolf M, Peeters M, Van Cutsem E, Siena S, Freeman DJ, Juan T, Sikorski R, Suggs S, Radinsky R, Patterson SD and Chang DD. Wild-type KRAS is required for panitumumab efficacy in patients with metastatic colorectal cancer. Journal of clinical oncology: official journal of the American Society of Clinical Oncology. 2008; 26: 1626-1634.
13. Gerlinger M, Rowan AJ, Horswell S, Larkin J, Endesfelder D, Gronroos E, Martinez P, Matthews N, Stewart A, Tarpey P, Varela I, Phillimore B, Begum S, McDonald NQ, Butler A, Jones D, et al. Intratumor heterogeneity and branched evolution revealed by multiregion sequencing. The New England journal of medicine. 2012; 366:883-892.
14. Niraula S, Amir E, Vera-Badillo F, Seruga B, Ocana A and Tannock IF. Risk of incremental toxicities and associated costs of new anticancer drugs: a meta-analysis. Journal of clinical oncology: official journal of the American Society of Clinical Oncology. 2014; 32: 3634-3642.
15. Niraula S, Seruga B, Ocana A, Shao T, Goldstein R, Tannock IF and Amir E. The price we pay for progress: a meta-analysis of harms of newly approved anticancer drugs. Journal of clinical oncology: official journal of the American Society of Clinical Oncology. 2012; 30:3012-3019.
16. Sawyers CL. The cancer biomarker problem. Nature. 2008; 452:548-552.
17. Seruga B, Sterling L, Wang L and Tannock IF. Reporting of serious adverse drug reactions of targeted anticancer agents in pivotal phase III clinical trials. Journal of clinical oncology: official journal of the American Society of Clinical Oncology. 2011; 29:174-185.
18. Pitrou I, Boutron I, Ahmad N and Ravaud P. Reporting of safety results in published reports of randomized controlled trials. Archives of internal medicine. 2009; 169:1756-1761.
19. Ioannidis JP. Adverse events in randomized trials: neglected, restricted, distorted, and silenced. Archives of internal medicine. 2009; 169:1737-1739.
20. ()
21. Deeks J.J., Higgins J.P.T., Altman D.G., editors. Analysing and presenting results. Cochrane Handbook for Systematic Reviews of Interventions 4 2 5 In: The Cochrane Library, Chichester, UK, John Wiley & Sons, Ltd 2006.
22. DerSimonian R and Laird N. Meta-analysis in clinical trials. Controlled clinical trials. 1986; 7:177-188.
23. Sweeting MJ, Sutton AJ and Lambert PC. What to add to nothing? Use and avoidance of continuity corrections in meta-analysis of sparse data. Statistics in medicine. 2004; 23: 1351-1375.
24. Baselga J, Cortes J, Kim SB, Im SA, Hegg R, Im YH, Roman L, Pedrini JL, Pienkowski T, Knott A, Clark E, Benyunes MC, Ross G, Swain SM and Group CS. Pertuzumab plus trastuzumab plus docetaxel for metastatic breast cancer. The New England journal of medicine. 2012; 366:109-119.
25. Baselga J, Campone M, Piccart M, Burris HA, 3rd, Rugo HS, Sahmoud T, Noguchi S, Gnant M, Pritchard KI, Lebrun F, Beck JT, Ito Y, Yardley D, Deleu I, Perez A, Bachelot T, et al. Everolimus in postmenopausal hormone-receptorpositive advanced breast cancer. The New England journal of medicine. 2012; 366:520-529.
26. Escudier B, Eisen T, Stadler WM, Szczylik C, Oudard S, Siebels M, Negrier S, Chevreau C, Solska E, Desai AA, Rolland F, Demkow T, Hutson TE, Gore M, Freeman S, Schwartz B, et al. Sorafenib in advanced clear-cell renalcell carcinoma. The New England journal of medicine. 2007; 356:125-134.
27. Fuchs CS, Tomasek J, Yong CJ, Dumitru F, Passalacqua R, Goswami C, Safran H, dos Santos LV, Aprile G, Ferry DR, Melichar B, Tehfe M, Topuzov E, Zalcberg JR, Chau I, Campbell W, et al. Ramucirumab monotherapy for previously treated advanced gastric or gastro-oesophageal junction adenocarcinoma (REGARD): an international, randomised, multicentre, placebo-controlled, phase 3 trial. Lancet. 2014; 383:31-39.
28. Grothey A, Van Cutsem E, Sobrero A, Siena S, Falcone A, Ychou M, Humblet Y, Bouche O, Mineur L, Barone C, Adenis A, Tabernero J, Yoshino T, Lenz HJ, Goldberg RM, Sargent DJ, et al. Regorafenib monotherapy for previously treated metastatic colorectal cancer (CORRECT): an international, multicentre, randomised, placebo-controlled, phase 3 trial. Lancet. 2013; 381:303-312.
29. Hurwitz H, Fehrenbacher L, Novotny W, Cartwright T, Hainsworth J, Heim W, Berlin J, Baron A, Griffing S, Holmgren E, Ferrara N, Fyfe G, Rogers B, Ross R and Kabbinavar F. Bevacizumab plus irinotecan, fluorouracil, and leucovorin for metastatic colorectal cancer. The New England journal of medicine. 2004; 350:2335-2342.
30. Llovet JM, Ricci S, Mazzaferro V, Hilgard P, Gane E, Blanc JF, de Oliveira AC, Santoro A, Raoul JL, Forner A, Schwartz M, Porta C, Zeuzem S, Bolondi L, Greten TF, Galle PR, et al. Sorafenib in advanced hepatocellular carcinoma. The New England journal of medicine. 2008; 359:378-390.
31. Moore MJ, Goldstein D, Hamm J, Figer A, Hecht JR, Gallinger S, Au HJ, Murawa P, Walde D, Wolff RA, Campos D, Lim R, Ding K, Clark G, Voskoglou-Nomikos T, Ptasynski M, et al. Erlotinib plus gemcitabine compared with gemcitabine alone in patients with advanced pancreatic cancer: a phase III trial of the National Cancer Institute of Canada Clinical Trials Group. Journal of clinical oncology: official journal of the American Society of Clinical Oncology. 2007; 25:1960-1966.
32. Motzer RJ, Escudier B, Oudard S, Hutson TE, Porta C, Bracarda S, Grunwald V, Thompson JA, Figlin RA, Hollaender N, Urbanowitz G, Berg WJ, Kay A, Lebwohl D, Ravaud A and Group R-S. Efficacy of everolimus in advanced renal cell carcinoma: a double-blind, randomised, placebo-controlled phase III trial. Lancet. 2008; 372:449-456.
33. Raymond E, Dahan L, Raoul JL, Bang YJ, Borbath I, Lombard-Bohas C, Valle J, Metrakos P, Smith D, Vinik A, Chen JS, Horsch D, Hammel P, Wiedenmann B, Van Cutsem E, Patyna S, et al. Sunitinib malate for the treatment of pancreatic neuroendocrine tumors. The New England journal of medicine. 2011; 364:501-513.
34. Shepherd FA, Rodrigues Pereira J, Ciuleanu T, Tan EH, Hirsh V, Thongprasert S, Campos D, Maoleekoonpiroj S, Smylie M, Martins R, van Kooten M, Dediu M, Findlay B, Tu D, Johnston D, Bezjak A, et al. Erlotinib in previously treated non-small-cell lung cancer. The New England journal of medicine. 2005; 353:123-132.
35. Sternberg CN, Davis ID, Mardiak J, Szczylik C, Lee E, Wagstaff J, Barrios CH, Salman P, Gladkov OA, Kavina A, Zarba JJ, Chen M, McCann L, Pandite L, Roychowdhury DF and Hawkins RE. Pazopanib in locally advanced or metastatic renal cell carcinoma: results of a randomized phase III trial. Journal of clinical oncology: official journal of the American Society of Clinical Oncology. 2010; 28:1061-1068.
36. van der Graaf WT, Blay JY, Chawla SP, Kim DW, Bui-Nguyen B, Casali PG, Schoffski P, Aglietta M, Staddon AP, Beppu Y, Le Cesne A, Gelderblom H, Judson IR, Araki N, Ouali M, Marreaud S, et al. Pazopanib for metastatic softtissue sarcoma (PALETTE): a randomised, double-blind, placebo-controlled phase 3 trial. Lancet. 2012; 379:1879-1886.
37. Yao JC, Shah MH, Ito T, Bohas CL, Wolin EM, Van Cutsem E, Hobday TJ, Okusaka T, Capdevila J, de Vries EG, Tomassetti P, Pavel ME, Hoosen S, Haas T, Lincy J, Lebwohl D, et al. Everolimus for advanced pancreatic neuroendocrine tumors. The New England journal of medicine. 2011; 364:514-523.
38. Escudier B, Pluzanska A, Koralewski P, Ravaud A, Bracarda S, Szczylik C, Chevreau C, Filipek M, Melichar B, Bajetta E, Gorbunova V, Bay JO, Bodrogi I, Jagiello-Gruszfeld A, Moore N and investigators AT. Bevacizumab plus interferon alfa-2a for treatment of metastatic renal cell carcinoma: a randomised, double-blind phase III trial. Lancet. 2007; 370:2103-2111.
39. Bang YJ, Van Cutsem E, Feyereislova A, Chung HC, Shen L, Sawaki A, Lordick F, Ohtsu A, Omuro Y, Satoh T, Aprile G, Kulikov E, Hill J, Lehle M, Ruschoff J, Kang YK, et al. Trastuzumab in combination with chemotherapy versus chemotherapy alone for treatment of HER2-positive advanced gastric or gastro-oesophageal junction cancer (ToGA): a phase 3, open-label, randomised controlled trial. Lancet. 2010; 376:687-697.
40. Geyer CE, Forster J, Lindquist D, Chan S, Romieu CG, Pienkowski T, Jagiello-Gruszfeld A, Crown J, Chan A, Kaufman B, Skarlos D, Campone M, Davidson N, Berger M, Oliva C, Rubin SD, et al. Lapatinib plus capecitabine for HER2-positive advanced breast cancer. The New England journal of medicine. 2006; 355:2733-2743.
41. Mok TS, Wu YL, Thongprasert S, Yang CH, Chu DT, Saijo N, Sunpaweravong P, Han B, Margono B, Ichinose Y, Nishiwaki Y, Ohe Y, Yang JJ, Chewaskulyong B, Jiang H, Duffield EL, et al. Gefitinib or carboplatin-paclitaxel in pulmonary adenocarcinoma. The New England journal of medicine. 2009; 361:947-957.
42. Sequist LV, Yang JC, Yamamoto N, O'Byrne K, Hirsh V, Mok T, Geater SL, Orlov S, Tsai CM, Boyer M, Su WC, Bennouna J, Kato T, Gorbunova V, Lee KH, Shah R, et al. Phase III study of afatinib or cisplatin plus pemetrexed in patients with metastatic lung adenocarcinoma with EGFR mutations. Journal of clinical oncology: official journal of the American Society of Clinical Oncology. 2013; 31:3327-3334.
43. Shaw AT, Kim DW, Nakagawa K, Seto T, Crino L, Ahn MJ, De Pas T, Besse B, Solomon BJ, Blackhall F, Wu YL, Thomas M, O'Byrne KJ, Moro-Sibilot D, Camidge DR, Mok T, et al. Crizotinib versus chemotherapy in advanced ALK-positive lung cancer. The New England journal of medicine. 2013; 368:2385-2394.
44. Slamon DJ, Leyland-Jones B, Shak S, Fuchs H, Paton V, Bajamonde A, Fleming T, Eiermann W, Wolter J, Pegram M, Baselga J and Norton L. Use of chemotherapy plus a monoclonal antibody against HER2 for metastatic breast cancer that overexpresses HER2. The New England journal of medicine. 2001; 344:783-792.
45. Verma S, Miles D, Gianni L, Krop IE, Welslau M, Baselga J, Pegram M, Oh DY, Dieras V, Guardino E, Fang L, Lu MW, Olsen S, Blackwell K and Group ES. Trastuzumab emtansine for HER2-positive advanced breast cancer. The New England journal of medicine. 2012; 367:1783-1791.
46. Bonner JA, Harari PM, Giralt J, Azarnia N, Shin DM, Cohen RB, Jones CU, Sur R, Raben D, Jassem J, Ove R, Kies MS, Baselga J, Youssoufian H, Amellal N, Rowinsky EK, et al. Radiotherapy plus cetuximab for squamous-cell carcinoma of the head and neck. The New England journal of medicine. 2006; 354:567-578.
47. Jonker DJ, O'Callaghan CJ, Karapetis CS, Zalcberg JR, Tu D, Au HJ, Berry SR, Krahn M, Price T, Simes RJ, Tebbutt NC, van Hazel G, Wierzbicki R, Langer C and Moore MJ. Cetuximab for the treatment of colorectal cancer. The New England journal of medicine. 2007; 357:2040-2048.
48. Giantonio BJ, Catalano PJ, Meropol NJ, O'Dwyer PJ, Mitchell EP, Alberts SR, Schwartz MA, Benson AB, 3rd and Eastern Cooperative Oncology Group Study E. Bevacizumab in combination with oxaliplatin, fluorouracil, and leucovorin (FOLFOX4) for previously treated metastatic colorectal cancer: results from the Eastern Cooperative Oncology Group Study E3200. Journal of clinical oncology: official journal of the American Society of Clinical Oncology. 2007; 25:1539-1544.
49. Hudes G, Carducci M, Tomczak P, Dutcher J, Figlin R, Kapoor A, Staroslawska E, Sosman J, McDermott D, Bodrogi I, Kovacevic Z, Lesovoy V, Schmidt-Wolf IG, Barbarash O, Gokmen E, O'Toole T, et al. Temsirolimus, interferon alfa, or both for advanced renal-cell carcinoma. The New England journal of medicine. 2007; 356:2271-2281.
50. Miller AA, Wang XF, Bogart JA, Hodgson LD, Rocha Lima CM, Radford JE, Vokes EE, Green MR, Cancer and Leukemia Group B. Phase II trial of paclitaxel-topotecanetoposide followed by consolidation chemoradiotherapy for limited-stage small cell lung cancer: CALGB 30002. Journal of thoracic oncology: official publication of the International Association for the Study of Lung Cancer. 2007; 2:645-651.
51. Rini BI, Escudier B, Tomczak P, Kaprin A, Szczylik C, Hutson TE, Michaelson MD, Gorbunova VA, Gore ME, Rusakov IG, Negrier S, Ou YC, Castellano D, Lim HY, Uemura H, Tarazi J, et al. Comparative effectiveness of axitinib versus sorafenib in advanced renal cell carcinoma (AXIS): a randomised phase 3 trial. Lancet. 2011; 378:1931-1939.
52. Sandler A, Gray R, Perry MC, Brahmer J, Schiller JH, Dowlati A, Lilenbaum R and Johnson DH. Paclitaxelcarboplatin alone or with bevacizumab for non-small-cell lung cancer. The New England journal of medicine. 2006; 355:2542-2550.
53. Van Cutsem E, Tabernero J, Lakomy R, Prenen H, Prausova J, Macarulla T, Ruff P, van Hazel GA, Moiseyenko V, Ferry D, McKendrick J, Polikoff J, Tellier A, Castan R and Allegra C. Addition of aflibercept to fluorouracil, leucovorin, and irinotecan improves survival in a phase III randomized trial in patients with metastatic colorectal cancer previously treated with an oxaliplatin-based regimen. Journal of clinical oncology: official journal of the American Society of Clinical Oncology. 2012; 30:3499-3506.
54. Chapman PB, Hauschild A, Robert C, Haanen JB, Ascierto P, Larkin J, Dummer R, Garbe C, Testori A, Maio M, Hogg D, Lorigan P, Lebbe C, Jouary T, Schadendorf D, Ribas A, et al. Improved survival with vemurafenib in melanoma with BRAF V600E mutation. The New England journal of medicine. 2011; 364:2507-2516.
55. Flaherty KT, Puzanov I, Kim KB, Ribas A, McArthur GA, Sosman JA, O'Dwyer PJ, Lee RJ, Grippo JF, Nolop K and Chapman PB. Inhibition of mutated, activated BRAF in metastatic melanoma. The New England journal of medicine. 2010; 363:809-819.
56. Hauschild A, Grob JJ, Demidov LV, Jouary T, Gutzmer R, Millward M, Rutkowski P, Blank CU, Miller WH, Jr., Kaempgen E, Martin-Algarra S, Karaszewska B, Mauch C, Chiarion-Sileni V, Martin AM, Swann S, et al. Dabrafenib in BRAF-mutated metastatic melanoma: a multicentre, open-label, phase 3 randomised controlled trial. Lancet. 2012; 380: 358-365.
57. Motzer RJ, Hutson TE, Tomczak P, Michaelson MD, Bukowski RM, Rixe O, Oudard S, Negrier S, Szczylik C, Kim ST, Chen I, Bycott PW, Baum CM and Figlin RA. Sunitinib versus interferon alfa in metastatic renal-cell carcinoma. The New England journal of medicine. 2007; 356:115-124.
58. Van Cutsem E, Peeters M, Siena S, Humblet Y, Hendlisz A, Neyns B, Canon JL, Van Laethem JL, Maurel J, Richardson G, Wolf M and Amado RG. Open-label phase III trial of panitumumab plus best supportive care compared with best supportive care alone in patients with chemotherapyrefractory metastatic colorectal cancer. Journal of clinical oncology: official journal of the American Society of Clinical Oncology. 2007; 25: 1658-1664.