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@ARTICLE{Kaulen:305427,
author = {L. Kaulen$^*$ and M. Martinez-Lage and J. S. Abramson and
P. Karschnia and S. Doubrovinskaia$^*$ and G. M. Shankar and
B. D. Choi and C. M. Ramundo and F. Ehret$^*$ and J. A.
Barnes and A. El-Jawahri and E. P. Hochberg and P. C.
Johnson and J. D. Soumerai and S. R. Plotkin and T. T.
Batchelor and W. Wick$^*$ and M. V. Maus and Y.-B. Chen and
M. J. Frigault and J. Dietrich},
title = {{C}linical presentation, management, and outcome of {TIAN}
in {CNS} lymphoma treated with {CD}19-{CAR} {T}-cell
therapy.},
journal = {Blood},
volume = {146},
number = {16},
issn = {0006-4971},
address = {Washington, DC},
publisher = {American Society of Hematology},
reportid = {DKFZ-2025-02156},
pages = {1902 - 1913},
year = {2025},
note = {#EA:B320#},
abstract = {Tumor inflammation-associated neurotoxicity (TIAN) was
recently proposed as a unique complication of immunotherapy
in patients with brain tumor. Here, we report a first
comprehensive characterization of TIAN in patients with
central nervous system (CNS) lymphoma (CNSL) treated with
CD19-directed chimeric antigen receptor (CD19-CAR) T cells.
TIAN occurred in 10 of 56 $(17.9\%)$ patients with CNSL,
with clinical onset at a median 3.5 days (range, 1-9) after
CD19-CAR T-cell infusion. It was less frequently associated
with cytokine release syndrome $(60\%$ vs $100\%;$ P = .009)
than immune effector cell-associated neurotoxicity syndrome
(ICANS). Although symptoms were usually transient and fully
reversible, TIAN was associated with a fatal outcome in 1
patient. Larger CNS tumor volume at baseline allowed the
identification of patients at risk for TIAN (area under the
curve, 0.847; P = .002). Maximizing Youden J statistics, a
discriminatory tumor volume threshold of >3.4 cm3 was
determined, which carried $87.5\%$ sensitivity and $80.5\%$
specificity. TIAN correlated with higher overall response
rates to CD19-CAR T cells $(90\%$ vs $52\%;$ P = .036) and
improved progression-free survival (hazard ratio, 0.22;
$95\%$ confidence interval, 0.07-0.61; P = .006) on
multivariate Cox proportional hazard regression. Postmortem
histopathological evaluation of a TIAN lesion revealed a
dense macrophage population with central necrosis and
peripheral reactive gliosis, accompanied by loss of white
matter and intracytoplasmic myelin in foamy macrophages.
Collectively, our work supports TIAN as a localized
on-tumor, on-target neurotoxicity syndrome, closely related
to preexisting CNSL lesions and distinct from ICANS. CNS
tumor volume at baseline may allow to identify patients at
risk and may guide management.},
keywords = {Humans / Male / Female / Middle Aged / Antigens, CD19:
immunology / Immunotherapy, Adoptive: adverse effects /
Central Nervous System Neoplasms: therapy / Central Nervous
System Neoplasms: immunology / Central Nervous System
Neoplasms: pathology / Aged / Adult / Neurotoxicity
Syndromes: etiology / Neurotoxicity Syndromes: therapy /
Lymphoma: therapy / Lymphoma: immunology / Lymphoma:
pathology / Receptors, Chimeric Antigen: immunology /
Treatment Outcome / Cytokine Release Syndrome: etiology /
Antigens, CD19 (NLM Chemicals) / Receptors, Chimeric Antigen
(NLM Chemicals)},
cin = {B320 / BE01},
ddc = {610},
cid = {I:(DE-He78)B320-20160331 / I:(DE-He78)BE01-20160331},
pnm = {312 - Funktionelle und strukturelle Genomforschung
(POF4-312)},
pid = {G:(DE-HGF)POF4-312},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:40663771},
doi = {10.1182/blood.2025028964},
url = {https://inrepo02.dkfz.de/record/305427},
}