Liver involvement with Langerhans cell histiocytosis in adults

Author:

Chang Long12,Cai Hua-Cong12,Lang Min12,Lin He12,Luo Ya-Ping23,Duan Ming-hui12,Zhou Dao-bin12,Goyal Gaurav4ORCID,Cao Xin-xin12ORCID

Affiliation:

1. Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College , Beijing , People’s Republic of China

2. State Key Laboratory of Complex Severe and Rare Diseases, Peking Union Medical College Hospital , Beijing , People’s Republic of China

3. Department of Nuclear Medicine, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College , Beijing , People’s Republic of China

4. Division of Hematology-Oncology, University of Alabama at Birmingham , Birmingham, AL , United States

Abstract

Abstract Background and Aims Liver involvement portends poor prognosis in adults. We aimed to characterize the clinical features, liver function tests, radiologic findings, molecular profiles, therapeutic approaches and outcomes of adults patients with Langerhans cell histiocytosis (LCH) with liver involvement. Methods We conducted a retrospective analysis of all adults with LCH (≥ 18 years) seen at Peking Union Medical College Hospital (Beijing, China) between January 2001 and December 2022. Results Among the 445 newly diagnosed adults with LCH, 90 patients had liver involvement at diagnosis and 22 patients at relapse. The median age was 32 years (range, 18–66 years). Of 112 evaluable patients, 108 had full liver function testing, including alanine aminotransferase, aspartate aminotransferase, alkaline phosphatase (ALP), γ-glutamyl transpeptidase (GGT), and total bilirubin and albumin. Elevated ALP was seen in 63.0% and GGT in 86.1%; 14.8% had elevated bilirubin. Next-generation sequencing of 54 patients revealed frequent BRAFN486_P490 (29.6%), BRAFV600E (18.5%), and MAP2K1 (14.8%). Outcomes After a median 40 months’ follow-up (range 1-168 months), 3-year progression-free survival (PFS) and overall survival were 49.7% and 86.6% respectively. In multivariable analyses, ≥3 abnormal liver function tests (HR 3.384, 95% CI 1.550–7.388, P = .002) associated with inferior PFS; immunomodulatory drug therapy (HR 0.073, 95% CI, 0.010-0.541, P = .010) correlated with superior PFS versus chemotherapy. Conclusions In summary, elevated GGT and ALP were common in adults with LCH liver involvement. Greater than equal to 3 abnormal liver function tests predicted poor outcomes. Immunomodulatory drug therapy was associated with favorable progression-free survival compared to chemotherapy.

Funder

Beijing Natural Science Haidian

National Key R&D Program of China, Ministry of Science and Technology of the People’s Republic of China

National High Level Hospital Clinical Research Funding

Publisher

Oxford University Press (OUP)

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