Neoadjuvant therapy and liver transplantation for hilar cholangiocarcinoma: Is pretreatment pathological confirmation of diagnosis necessary?

Charles B. Rosen, Sarwa Darwish Murad, Julie K. Heimbach, Scott Nyberg, David M. Nagorney, Gregory James Gores

Research output: Contribution to journalArticle

39 Citations (Scopus)

Abstract

BACKGROUND: Neoadjuvant chemoradiotherapy followed by operative staging and liver transplantation is an effective treatment for patients with unresectable hilar cholangiocarcinoma (CCA) and CCA arising in the setting of primary sclerosing cholangitis (PSC). Pathologic confirmation of CCA is notoriously difficult, and many patients have been treated based on clinical criteria without pathological confirmation. STUDY DESIGN: We reviewed our experience with the specific aim of determining the need for pathological confirmation of CCA before treatment. RESULTS: Two hundred and fifteen patients received neoadjuvant therapy between 1992 and 2011. One hundred and eighty-two patients underwent operative staging and 38 (21%) had findings that precluded transplantation. Pathological confirmation of CCA before therapy was achieved in 45 of 87 (52%) PSC patients and 22 of 49 (45%) de novo patients who underwent transplantation. Pretreatment pathological confirmation was associated with significantly worse 5-year survival after start of therapy for PSC patients (50% vs 80%; p = 0.001), but not for de novo patients (39% vs 48%; p = 0.27). Pretreatment pathological confirmation was associated with worse 5-year survival after transplantation for PSC patients (66% vs 92%; p = 0.01), but not for de novo patients (63% vs 65%; p = 0.71). The difference in the PSC patients was not due to recurrent cancer. Absence of pretreatment pathological confirmation did not result in less detection of residual CCA in the explanted livers or in less recurrence after transplantation. CONCLUSIONS: Rates of residual CCA in liver explants and recurrences after transplantation are comparable for patients with and without pretreatment pathological confirmation of CCA and attest to the accuracy of clinical diagnostic criteria. Pretreatment pathological confirmation of CCA is desirable but should not be a requirement for treatment.

Original languageEnglish (US)
Pages (from-to)31-38
Number of pages8
JournalJournal of the American College of Surgeons
Volume215
Issue number1
DOIs
StatePublished - Jul 2012

Fingerprint

Klatskin Tumor
Neoadjuvant Therapy
Liver Transplantation
Cholangiocarcinoma
Sclerosing Cholangitis
Transplantation
Therapeutics
Recurrence
Survival
Liver
Chemoradiotherapy

ASJC Scopus subject areas

  • Surgery

Cite this

Neoadjuvant therapy and liver transplantation for hilar cholangiocarcinoma : Is pretreatment pathological confirmation of diagnosis necessary? / Rosen, Charles B.; Darwish Murad, Sarwa; Heimbach, Julie K.; Nyberg, Scott; Nagorney, David M.; Gores, Gregory James.

In: Journal of the American College of Surgeons, Vol. 215, No. 1, 07.2012, p. 31-38.

Research output: Contribution to journalArticle

@article{f6c967c242174af3ba7edf0167b2d983,
title = "Neoadjuvant therapy and liver transplantation for hilar cholangiocarcinoma: Is pretreatment pathological confirmation of diagnosis necessary?",
abstract = "BACKGROUND: Neoadjuvant chemoradiotherapy followed by operative staging and liver transplantation is an effective treatment for patients with unresectable hilar cholangiocarcinoma (CCA) and CCA arising in the setting of primary sclerosing cholangitis (PSC). Pathologic confirmation of CCA is notoriously difficult, and many patients have been treated based on clinical criteria without pathological confirmation. STUDY DESIGN: We reviewed our experience with the specific aim of determining the need for pathological confirmation of CCA before treatment. RESULTS: Two hundred and fifteen patients received neoadjuvant therapy between 1992 and 2011. One hundred and eighty-two patients underwent operative staging and 38 (21{\%}) had findings that precluded transplantation. Pathological confirmation of CCA before therapy was achieved in 45 of 87 (52{\%}) PSC patients and 22 of 49 (45{\%}) de novo patients who underwent transplantation. Pretreatment pathological confirmation was associated with significantly worse 5-year survival after start of therapy for PSC patients (50{\%} vs 80{\%}; p = 0.001), but not for de novo patients (39{\%} vs 48{\%}; p = 0.27). Pretreatment pathological confirmation was associated with worse 5-year survival after transplantation for PSC patients (66{\%} vs 92{\%}; p = 0.01), but not for de novo patients (63{\%} vs 65{\%}; p = 0.71). The difference in the PSC patients was not due to recurrent cancer. Absence of pretreatment pathological confirmation did not result in less detection of residual CCA in the explanted livers or in less recurrence after transplantation. CONCLUSIONS: Rates of residual CCA in liver explants and recurrences after transplantation are comparable for patients with and without pretreatment pathological confirmation of CCA and attest to the accuracy of clinical diagnostic criteria. Pretreatment pathological confirmation of CCA is desirable but should not be a requirement for treatment.",
author = "Rosen, {Charles B.} and {Darwish Murad}, Sarwa and Heimbach, {Julie K.} and Scott Nyberg and Nagorney, {David M.} and Gores, {Gregory James}",
year = "2012",
month = "7",
doi = "10.1016/j.jamcollsurg.2012.03.014",
language = "English (US)",
volume = "215",
pages = "31--38",
journal = "Journal of the American College of Surgeons",
issn = "1072-7515",
publisher = "Elsevier Inc.",
number = "1",

}

TY - JOUR

T1 - Neoadjuvant therapy and liver transplantation for hilar cholangiocarcinoma

T2 - Is pretreatment pathological confirmation of diagnosis necessary?

AU - Rosen, Charles B.

AU - Darwish Murad, Sarwa

AU - Heimbach, Julie K.

AU - Nyberg, Scott

AU - Nagorney, David M.

AU - Gores, Gregory James

PY - 2012/7

Y1 - 2012/7

N2 - BACKGROUND: Neoadjuvant chemoradiotherapy followed by operative staging and liver transplantation is an effective treatment for patients with unresectable hilar cholangiocarcinoma (CCA) and CCA arising in the setting of primary sclerosing cholangitis (PSC). Pathologic confirmation of CCA is notoriously difficult, and many patients have been treated based on clinical criteria without pathological confirmation. STUDY DESIGN: We reviewed our experience with the specific aim of determining the need for pathological confirmation of CCA before treatment. RESULTS: Two hundred and fifteen patients received neoadjuvant therapy between 1992 and 2011. One hundred and eighty-two patients underwent operative staging and 38 (21%) had findings that precluded transplantation. Pathological confirmation of CCA before therapy was achieved in 45 of 87 (52%) PSC patients and 22 of 49 (45%) de novo patients who underwent transplantation. Pretreatment pathological confirmation was associated with significantly worse 5-year survival after start of therapy for PSC patients (50% vs 80%; p = 0.001), but not for de novo patients (39% vs 48%; p = 0.27). Pretreatment pathological confirmation was associated with worse 5-year survival after transplantation for PSC patients (66% vs 92%; p = 0.01), but not for de novo patients (63% vs 65%; p = 0.71). The difference in the PSC patients was not due to recurrent cancer. Absence of pretreatment pathological confirmation did not result in less detection of residual CCA in the explanted livers or in less recurrence after transplantation. CONCLUSIONS: Rates of residual CCA in liver explants and recurrences after transplantation are comparable for patients with and without pretreatment pathological confirmation of CCA and attest to the accuracy of clinical diagnostic criteria. Pretreatment pathological confirmation of CCA is desirable but should not be a requirement for treatment.

AB - BACKGROUND: Neoadjuvant chemoradiotherapy followed by operative staging and liver transplantation is an effective treatment for patients with unresectable hilar cholangiocarcinoma (CCA) and CCA arising in the setting of primary sclerosing cholangitis (PSC). Pathologic confirmation of CCA is notoriously difficult, and many patients have been treated based on clinical criteria without pathological confirmation. STUDY DESIGN: We reviewed our experience with the specific aim of determining the need for pathological confirmation of CCA before treatment. RESULTS: Two hundred and fifteen patients received neoadjuvant therapy between 1992 and 2011. One hundred and eighty-two patients underwent operative staging and 38 (21%) had findings that precluded transplantation. Pathological confirmation of CCA before therapy was achieved in 45 of 87 (52%) PSC patients and 22 of 49 (45%) de novo patients who underwent transplantation. Pretreatment pathological confirmation was associated with significantly worse 5-year survival after start of therapy for PSC patients (50% vs 80%; p = 0.001), but not for de novo patients (39% vs 48%; p = 0.27). Pretreatment pathological confirmation was associated with worse 5-year survival after transplantation for PSC patients (66% vs 92%; p = 0.01), but not for de novo patients (63% vs 65%; p = 0.71). The difference in the PSC patients was not due to recurrent cancer. Absence of pretreatment pathological confirmation did not result in less detection of residual CCA in the explanted livers or in less recurrence after transplantation. CONCLUSIONS: Rates of residual CCA in liver explants and recurrences after transplantation are comparable for patients with and without pretreatment pathological confirmation of CCA and attest to the accuracy of clinical diagnostic criteria. Pretreatment pathological confirmation of CCA is desirable but should not be a requirement for treatment.

UR - http://www.scopus.com/inward/record.url?scp=84862981935&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84862981935&partnerID=8YFLogxK

U2 - 10.1016/j.jamcollsurg.2012.03.014

DO - 10.1016/j.jamcollsurg.2012.03.014

M3 - Article

C2 - 22621893

AN - SCOPUS:84862981935

VL - 215

SP - 31

EP - 38

JO - Journal of the American College of Surgeons

JF - Journal of the American College of Surgeons

SN - 1072-7515

IS - 1

ER -