Leptomeningeal metastasis: A Response Assessment in Neuro-Oncology critical review of endpoints and response criteria of published randomized clinical trials

Marc Chamberlain, Riccardo Soffietti, Jeffrey Raizer, Roberta Rudà, Dieta Brandsma, Willem Boogerd, Sophie Taillibert, Morris D. Groves, Emilie Le Rhun, Larry Junck, Martin Van Den Bent, Patrick Y. Wen, Kurt A. Jaeckle

Research output: Contribution to journalReview article

69 Citations (Scopus)

Abstract

Purpose. To date, response criteria and optimal methods for assessment of outcome have not been standardized in patients with leptomeningeal metastasis (LM). Methods. A Response Assessment in Neuro-Oncology working group of experts in LM critically reviewed published literature regarding randomized clinical trials (RCTs) and trial design in patients with LM. Results. A literature review determined that 6 RCTs regarding the treatment of LM have been published, all of which assessed the response to intra-CSF based chemotherapy. Amongst these RCTs, only a single trial attempted to determine whether intra-CSF chemotherapy was of benefit compared with systemic therapy. Otherwise, this pragmatic question has not been formally addressed in patients with solid cancers and LM. The methodology of the 6 RCTs varied widely with respect to pretreatment evaluation, type of treatment, and response to treatment. Additionally there was little uniformity in reporting of treatment-related toxicity. One RCT suggests no advantage of combined versus single-agent intra-CSF chemotherapy in patients with LM. No specific intra-CSF regimen has shown superior efficacy in the treatment of LM, with the exception of liposomal cytarabine in patients with lymphomatous meningitis. Problematic with all RCTs is the lack of standardization with respect to response criteria. There was considerable variation in definitions of response by clinical examination, neuroimaging, and CSF analysis. Conclusion. Based upon a review of published RCTs in LM, there exists a significant unmet need for guidelines for evaluating patients with LM in clinical practice as well as for response assessment in clinical trials.

Original languageEnglish (US)
Pages (from-to)1176-1185
Number of pages10
JournalNeuro-Oncology
Volume16
Issue number9
DOIs
StatePublished - Sep 2014

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Randomized Controlled Trials
Neoplasm Metastasis
Drug Therapy
Therapeutics
Cytarabine
Meningitis
Neuroimaging
Outcome Assessment (Health Care)
Clinical Trials
Guidelines
Neoplasms

All Science Journal Classification (ASJC) codes

  • Oncology
  • Clinical Neurology
  • Cancer Research

Cite this

Chamberlain, Marc ; Soffietti, Riccardo ; Raizer, Jeffrey ; Rudà, Roberta ; Brandsma, Dieta ; Boogerd, Willem ; Taillibert, Sophie ; Groves, Morris D. ; Le Rhun, Emilie ; Junck, Larry ; Van Den Bent, Martin ; Wen, Patrick Y. ; Jaeckle, Kurt A. / Leptomeningeal metastasis : A Response Assessment in Neuro-Oncology critical review of endpoints and response criteria of published randomized clinical trials. In: Neuro-Oncology. 2014 ; Vol. 16, No. 9. pp. 1176-1185.
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abstract = "Purpose. To date, response criteria and optimal methods for assessment of outcome have not been standardized in patients with leptomeningeal metastasis (LM). Methods. A Response Assessment in Neuro-Oncology working group of experts in LM critically reviewed published literature regarding randomized clinical trials (RCTs) and trial design in patients with LM. Results. A literature review determined that 6 RCTs regarding the treatment of LM have been published, all of which assessed the response to intra-CSF based chemotherapy. Amongst these RCTs, only a single trial attempted to determine whether intra-CSF chemotherapy was of benefit compared with systemic therapy. Otherwise, this pragmatic question has not been formally addressed in patients with solid cancers and LM. The methodology of the 6 RCTs varied widely with respect to pretreatment evaluation, type of treatment, and response to treatment. Additionally there was little uniformity in reporting of treatment-related toxicity. One RCT suggests no advantage of combined versus single-agent intra-CSF chemotherapy in patients with LM. No specific intra-CSF regimen has shown superior efficacy in the treatment of LM, with the exception of liposomal cytarabine in patients with lymphomatous meningitis. Problematic with all RCTs is the lack of standardization with respect to response criteria. There was considerable variation in definitions of response by clinical examination, neuroimaging, and CSF analysis. Conclusion. Based upon a review of published RCTs in LM, there exists a significant unmet need for guidelines for evaluating patients with LM in clinical practice as well as for response assessment in clinical trials.",
author = "Marc Chamberlain and Riccardo Soffietti and Jeffrey Raizer and Roberta Rud{\`a} and Dieta Brandsma and Willem Boogerd and Sophie Taillibert and Groves, {Morris D.} and {Le Rhun}, Emilie and Larry Junck and {Van Den Bent}, Martin and Wen, {Patrick Y.} and Jaeckle, {Kurt A.}",
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doi = "10.1093/neuonc/nou089",
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Chamberlain, M, Soffietti, R, Raizer, J, Rudà, R, Brandsma, D, Boogerd, W, Taillibert, S, Groves, MD, Le Rhun, E, Junck, L, Van Den Bent, M, Wen, PY & Jaeckle, KA 2014, 'Leptomeningeal metastasis: A Response Assessment in Neuro-Oncology critical review of endpoints and response criteria of published randomized clinical trials', Neuro-Oncology, vol. 16, no. 9, pp. 1176-1185. https://doi.org/10.1093/neuonc/nou089

Leptomeningeal metastasis : A Response Assessment in Neuro-Oncology critical review of endpoints and response criteria of published randomized clinical trials. / Chamberlain, Marc; Soffietti, Riccardo; Raizer, Jeffrey; Rudà, Roberta; Brandsma, Dieta; Boogerd, Willem; Taillibert, Sophie; Groves, Morris D.; Le Rhun, Emilie; Junck, Larry; Van Den Bent, Martin; Wen, Patrick Y.; Jaeckle, Kurt A.

In: Neuro-Oncology, Vol. 16, No. 9, 09.2014, p. 1176-1185.

Research output: Contribution to journalReview article

TY - JOUR

T1 - Leptomeningeal metastasis

T2 - A Response Assessment in Neuro-Oncology critical review of endpoints and response criteria of published randomized clinical trials

AU - Chamberlain, Marc

AU - Soffietti, Riccardo

AU - Raizer, Jeffrey

AU - Rudà, Roberta

AU - Brandsma, Dieta

AU - Boogerd, Willem

AU - Taillibert, Sophie

AU - Groves, Morris D.

AU - Le Rhun, Emilie

AU - Junck, Larry

AU - Van Den Bent, Martin

AU - Wen, Patrick Y.

AU - Jaeckle, Kurt A.

PY - 2014/9

Y1 - 2014/9

N2 - Purpose. To date, response criteria and optimal methods for assessment of outcome have not been standardized in patients with leptomeningeal metastasis (LM). Methods. A Response Assessment in Neuro-Oncology working group of experts in LM critically reviewed published literature regarding randomized clinical trials (RCTs) and trial design in patients with LM. Results. A literature review determined that 6 RCTs regarding the treatment of LM have been published, all of which assessed the response to intra-CSF based chemotherapy. Amongst these RCTs, only a single trial attempted to determine whether intra-CSF chemotherapy was of benefit compared with systemic therapy. Otherwise, this pragmatic question has not been formally addressed in patients with solid cancers and LM. The methodology of the 6 RCTs varied widely with respect to pretreatment evaluation, type of treatment, and response to treatment. Additionally there was little uniformity in reporting of treatment-related toxicity. One RCT suggests no advantage of combined versus single-agent intra-CSF chemotherapy in patients with LM. No specific intra-CSF regimen has shown superior efficacy in the treatment of LM, with the exception of liposomal cytarabine in patients with lymphomatous meningitis. Problematic with all RCTs is the lack of standardization with respect to response criteria. There was considerable variation in definitions of response by clinical examination, neuroimaging, and CSF analysis. Conclusion. Based upon a review of published RCTs in LM, there exists a significant unmet need for guidelines for evaluating patients with LM in clinical practice as well as for response assessment in clinical trials.

AB - Purpose. To date, response criteria and optimal methods for assessment of outcome have not been standardized in patients with leptomeningeal metastasis (LM). Methods. A Response Assessment in Neuro-Oncology working group of experts in LM critically reviewed published literature regarding randomized clinical trials (RCTs) and trial design in patients with LM. Results. A literature review determined that 6 RCTs regarding the treatment of LM have been published, all of which assessed the response to intra-CSF based chemotherapy. Amongst these RCTs, only a single trial attempted to determine whether intra-CSF chemotherapy was of benefit compared with systemic therapy. Otherwise, this pragmatic question has not been formally addressed in patients with solid cancers and LM. The methodology of the 6 RCTs varied widely with respect to pretreatment evaluation, type of treatment, and response to treatment. Additionally there was little uniformity in reporting of treatment-related toxicity. One RCT suggests no advantage of combined versus single-agent intra-CSF chemotherapy in patients with LM. No specific intra-CSF regimen has shown superior efficacy in the treatment of LM, with the exception of liposomal cytarabine in patients with lymphomatous meningitis. Problematic with all RCTs is the lack of standardization with respect to response criteria. There was considerable variation in definitions of response by clinical examination, neuroimaging, and CSF analysis. Conclusion. Based upon a review of published RCTs in LM, there exists a significant unmet need for guidelines for evaluating patients with LM in clinical practice as well as for response assessment in clinical trials.

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