Abstract
Purpose: To evaluate progression-free survival (PFS) as early surrogate endpoints for overall survival (OS) in primary CNS lymphoma (PCNSL). Methods: PubMed, Embase and Cochrane Central Library were searched up to 7 June 2022. Trial-level analyses were performed by weighted linear regression of logarithmic hazard ratios for PFS and OS. Treatment arm-level analyses were performed between PFS rates and 3- or 5-year OS rates. Results: 1471 PCNSL patients in nine randomized control trials were included. PFS was associated with OS (r = 0.750; 95% CI: 0.228–0.937). Strong linear correlations existed between 1-, 2- and 3-year PFS and 3-year OS (r = 0.896–0.928), moderate or weak correlations existed between 3- to 6-month PFS and 3-year OS, 3-month to 5-year PFS and 5-year OS. Conclusion: Short-term PFS can validly substitute for long-term OS in PCNSL.
References
- 1. European Association of Neuro-Oncology (EANO) guidelines for treatment of primary central nervous system lymphoma (PCNSL). Neuro-Oncology. 25(1), 37–53 (2022).
- 2. . Primary CNS lymphoma. J. Clin. Oncol. 35(21), 2410–2418 (2017).
- 3. . The changing incidence of primary central nervous system lymphoma is driven primarily by the changing incidence in young and middle-aged men and differs from time trends in systemic diffuse large B-cell non-Hodgkin's lymphoma. Am. J. Hematol. 88(12), 997–1000 (2013).
- 4. CBTRUS statistical report: primary brain and central nervous system tumors diagnosed in the United States in 2007–2011. Neuro-Oncology 16(Suppl. 4), iv1–iv63 (2014).
- 5. Meta-analyses evaluating surrogate endpoints for overall survival in cancer randomized trials: a critical review. Crit. Rev. Oncol. Hemat. 123, 21–41 (2018).
- 6. . Endpoints in cancer clinical trials. J. Visc. Surg. 151(1), 17–22 (2014).
- 7. . Surrogate endpoints for early-stage breast cancer: a review of the state of the art, controversies, and future prospects. Ther. Adv. Med. Oncol. 13, 17495677 (2021).
- 8. Association of progression-free or event-free survival with overall survival in diffuse large B-cell lymphoma after immunochemotherapy: a systematic review. Leukemia 34(10), 2576–2591 (2020).
- 9. Progression-free survival as a surrogate end point for overall survival in first-line diffuse large B-cell lymphoma: an individual patient-level analysis of multiple randomized trials (SEAL). J. Clin. Oncol. 36(25), 2593–2602 (2018).
- 10. US FDA. Table of surrogate endpoints that were the basis of drug approval licensure. www.fda.gov/drugs/development-resources/table-surrogate-endpoints-were-basis-drug-approval-or-licensure (Accessed 2 October 2022).
- 11. . Biased evaluation in cancer drug trials-how use of progression-free survival as the primary end point can mislead. JAMA Oncol. 8(5), 679–680 (2022).
- 12. The PRISMA 2020 statement: an updated guideline for reporting systematic reviews. BMJ Brit. Med. J. 372, n71 (2021).
- 13. High-dose cytarabine plus high-dose methotrexate versus high-dose methotrexate alone in patients with primary CNS lymphoma: a randomised phase 2 trial. Lancet 374(9700), 1512–1520 (2009).
- 14. A medical research council randomized trial in patients with primary cerebral non-Hodgkin lymphoma: cerebral radiotherapy with and without cyclophosphamide, doxorubicin, vincristine, and prednisone chemotherapy. Am. Cancer Soc. 89(6), 1359–1370 (2000).
- 15. The Cochrane Collaboration's tool for assessing risk of bias in randomised trials. BMJ Brit. Med. J. 343, d5928 (2011).
- 16. . Practical methods for incorporating summary time-to-event data into meta-analysis. Trials 8, 16 (2007).
- 17. A systematic review and recommendation for reporting of surrogate endpoint evaluation using meta-analyses. JNCI Cancer Spect. 3(1), kz2 (2019).
- 18. Long-term efficacy, safety and neurotolerability of MATRix regimen followed by autologous transplant in primary CNS lymphoma: 7-year results of the IELSG32 randomized trial. Leukemia. 36(7), 1870–1878 (2022).
- 19. . Randomized phase III study of high-dose methotrexate and whole brain radiotherapy with or without concomitant and adjuvant temozolomide in patients with newly diagnosed primary central nervous system lymphoma: JCOG1114C. J. Clin. Oncol. 38(Suppl. 15), 2500 (2020).
- 20. Rituximab in patients with primary CNS lymphoma (HOVON 105/ALLG NHL 24): a randomised, open-label, phase 3 intergroup study. Lancet Oncol. 20(2), 216–228 (2019).
- 21. . Radiotherapy or autologous stem-cell transplantation for primary CNS lymphoma in patients 60 years of age and younger: results of the intergroup ANOCEF-GOELAMS randomized phase II PRECIS study. J. Clin. Oncol. 37(10), 823–833 (2019).
- 22. . Fotemustine, teniposide and dexamethasone versus high-dose methotrexate plus cytarabine in newly diagnosed primary CNS lymphoma: a randomised phase 2 trial. J. Neurooncol. 140(2), 427–434 (2018).
- 23. Methotrexate and temozolomide versus methotrexate, procarbazine, vincristine, and cytarabine for primary CNS lymphoma in an elderly population: an intergroup ANOCEF-GOELAMS randomised phase 2 trial. Lancet Haematol. 2(6), e251–e259 (2015).
- 24. Randomized phase III study of whole-brain radiotherapy for primary CNS lymphoma. Neurology 84(12), 1242–1248 (2015).
- 25. . Systemic approach to recurrent primary CNS lymphoma: perspective on current and emerging treatment strategies. Oncotargets Ther. 13, 8323–8335 (2020).
- 26. . Relapsed primary central nervous system lymphoma: current advances. Front Oncol. 11, 649789 (2021).
- 27. Evolution of the randomized clinical trial in the era of precision oncology. JAMA Oncol. 7(5), 728–734 (2021).
- 28. A single-center retrospective analysis of outcome measures and consolidation strategies for relapsed and refractory primary CNS lymphoma. J. Neurooncol. 151(2), 193–200 (2021).
- 29. Primary CNS lymphoma at first relapse/progression: characteristics, management, and outcome of 256 patients from the French LOC network. Neuro-Oncology 18(9), 1297–1303 (2016).
- 30. Analysis of key factors associated with response to salvage high-dose methotrexate rechallenge in primary central nervous system lymphoma with first relapse. Curr. Oncol. 29(9), 6642–6656 (2022).
- 31. Phase 1b trial of an ibrutinib-based combination therapy in recurrent/refractory CNS lymphoma. Blood 133(5), 436–445 (2019).
- 32. Lenalidomide in combination with intravenous rituximab (REVRI) in relapsed/refractory primary CNS lymphoma or primary intraocular lymphoma: a multicenter prospective ‘proof of concept’ phase II study of the French Oculo-Cerebral Lymphoma (LOC) Network and the Lymphoma Study Association (LYSA)†. Ann. Oncol. 30(4), 621–628 (2019).
- 33. PD-1 blockade with nivolumab in relapsed/refractory primary central nervous system and testicular lymphoma. Blood 129(23), 3071–3073 (2017).
- 34. CAR T-cell therapy in primary central nervous system lymphoma: the clinical experience of the French LOC network. Blood 139(5), 792–796 (2022).
- 35. . Brain-restricted mTOR inhibition with binary pharmacology. Nature 609(7928), 822–828 (2022).
- 36. Report of an international workshop to standardize baseline evaluation and response criteria for primary CNS lymphoma. J. Clin. Oncol. 23(22), 5034–5043 (2005).
- 37. Changes in cerebrospinal fluid interleukin-10 levels display better performance in predicting disease relapse than conventional magnetic resonance imaging in primary central nervous system lymphoma. BMC Cancer 21(1), 183 (2021).