Phase 1 trial of durvalumab (anti–PD-L1) combined with lenalidomide in relapsed/refractory cutaneous T-cell lymphoma

Abstract: Selective targeting of the functionally exhausted malignant T cells in cutaneous T-cell lymphoma (CTCL) and distinct cells within the tumor microenvironment (TME) via programmed cell death 1/programmed cell death ligand 1 blockade (durvalumab) may restore an antitumor immune response. The...

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Main Authors: Christiane Querfeld, Joycelynne Palmer, Zhen Han, Xiwei Wu, Yate-Ching Yuan, Min-Hsuan Chen, Chingyu Su, Ni-Chun Tsai, D. Lynne Smith, Samantha N. Hammond, Liliana Crisan, Joo Y. Song, Raju Pillai, Steven T. Rosen, Jasmine Zain
Format: Article
Language:English
Published: Elsevier 2025-05-01
Series:Blood Advances
Online Access:http://www.sciencedirect.com/science/article/pii/S2473952925001065
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author Christiane Querfeld
Joycelynne Palmer
Zhen Han
Xiwei Wu
Yate-Ching Yuan
Min-Hsuan Chen
Chingyu Su
Ni-Chun Tsai
D. Lynne Smith
Samantha N. Hammond
Liliana Crisan
Joo Y. Song
Raju Pillai
Steven T. Rosen
Jasmine Zain
author_facet Christiane Querfeld
Joycelynne Palmer
Zhen Han
Xiwei Wu
Yate-Ching Yuan
Min-Hsuan Chen
Chingyu Su
Ni-Chun Tsai
D. Lynne Smith
Samantha N. Hammond
Liliana Crisan
Joo Y. Song
Raju Pillai
Steven T. Rosen
Jasmine Zain
author_sort Christiane Querfeld
collection DOAJ
description Abstract: Selective targeting of the functionally exhausted malignant T cells in cutaneous T-cell lymphoma (CTCL) and distinct cells within the tumor microenvironment (TME) via programmed cell death 1/programmed cell death ligand 1 blockade (durvalumab) may restore an antitumor immune response. The oral immunomodulator lenalidomide, which has activity in CTCL, may enhance durvalumab immune checkpoint blockade. Our phase 1/2 clinical trial of durvalumab and lenalidomide in patients with refractory/advanced CTCL sought to assess the safety and tolerability and to identify the maximum tolerated dose and recommended phase 2 dose (RP2D) of lenalidomide plus fixed-dose durvalumab. Secondary and tertiary objectives were to investigate the efficacy and effects on the TME. Thirteen patients were evaluable for toxicities and 12 for dose decisions and response. No serious adverse events (AEs) or dose-limiting toxicities (DLTs) were observed during cycles 1 to 3 (DLT evaluation period), and dose level 3 was identified as the RP2D. The most frequent AEs were tumor flare, fatigue, neutropenia, and leukopenia. Three patients developed grade 1 or 2 autoimmune thyroiditis that resolved with treatment. Best overall and skin response rates were 58.3% (95% confidence interval (95% CI), 27.7-84.8%) and 75% (95% CI: 42.8-94.5%), respectively. The median cycles of treatment were 11, and the median duration of response was 25.5 months. The combination showed clinical activity with 7 partial responses and 4 stable disease. Potentially predictive immune signatures were downregulation of -α signaling via NF-κB, interferon gamma, and phosphoinositide 3 kinase-AKT-mammalian target of rapamycin signaling pathways in responders and upregulation of MYC targets and proinflammatory pathways in nonresponders. Profiling of immune cell compositions revealed changes in individual immune cell clusters based on treatment response. This trial was registered at www.ClinicalTrials.gov as #NCT03011814.
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spelling doaj-art-e7d0d7bd4ed24fa79c2f4b4f26d48e8a2025-08-20T02:14:15ZengElsevierBlood Advances2473-95292025-05-01992247226010.1182/bloodadvances.2024014655Phase 1 trial of durvalumab (anti–PD-L1) combined with lenalidomide in relapsed/refractory cutaneous T-cell lymphomaChristiane Querfeld0Joycelynne Palmer1Zhen Han2Xiwei Wu3Yate-Ching Yuan4Min-Hsuan Chen5Chingyu Su6Ni-Chun Tsai7D. Lynne Smith8Samantha N. Hammond9Liliana Crisan10Joo Y. Song11Raju Pillai12Steven T. Rosen13Jasmine Zain14Division of Dermatology, City of Hope, Duarte, CA; Department of Pathology, City of Hope, Duarte, CA; Department of Hematology and Hematopoietic Cell Transplantation, City of Hope, Duarte, CA; Beckman Research Institute, City of Hope, Duarte, CA; Correspondence: Christiane Querfeld, Division of Dermatology, Department of Pathology, Beckman Research Institute, City of Hope National Medical Center, 1500 E Duarte Rd, Duarte, CA 91010;Beckman Research Institute, City of Hope, Duarte, CA; Division of Biostatistics, City of Hope, Duarte, CA; Department of Computational and Quantitative Medicine, City of Hope, Duarte, CADepartment of Pathology, City of Hope, Duarte, CA; Beckman Research Institute, City of Hope, Duarte, CABeckman Research Institute, City of Hope, Duarte, CA; Department of Computational and Quantitative Medicine, City of Hope, Duarte, CA; Integrative Genomics Core, City of Hope, Duarte, CABeckman Research Institute, City of Hope, Duarte, CA; Department of Computational and Quantitative Medicine, City of Hope, Duarte, CA; Department of Translational Bioinformatics, Center for Informatics, City of Hope, Duarte, CABeckman Research Institute, City of Hope, Duarte, CA; Department of Computational and Quantitative Medicine, City of Hope, Duarte, CADepartment of Pathology, City of Hope, Duarte, CA; Beckman Research Institute, City of Hope, Duarte, CABeckman Research Institute, City of Hope, Duarte, CA; Division of Biostatistics, City of Hope, Duarte, CADepartment of Hematology and Hematopoietic Cell Transplantation, City of Hope, Duarte, CABeckman Research Institute, City of Hope, Duarte, CADivision of Dermatology, City of Hope, Duarte, CA; Beckman Research Institute, City of Hope, Duarte, CADepartment of Pathology, City of Hope, Duarte, CADepartment of Pathology, City of Hope, Duarte, CADepartment of Hematology and Hematopoietic Cell Transplantation, City of Hope, Duarte, CA; Beckman Research Institute, City of Hope, Duarte, CADepartment of Hematology and Hematopoietic Cell Transplantation, City of Hope, Duarte, CAAbstract: Selective targeting of the functionally exhausted malignant T cells in cutaneous T-cell lymphoma (CTCL) and distinct cells within the tumor microenvironment (TME) via programmed cell death 1/programmed cell death ligand 1 blockade (durvalumab) may restore an antitumor immune response. The oral immunomodulator lenalidomide, which has activity in CTCL, may enhance durvalumab immune checkpoint blockade. Our phase 1/2 clinical trial of durvalumab and lenalidomide in patients with refractory/advanced CTCL sought to assess the safety and tolerability and to identify the maximum tolerated dose and recommended phase 2 dose (RP2D) of lenalidomide plus fixed-dose durvalumab. Secondary and tertiary objectives were to investigate the efficacy and effects on the TME. Thirteen patients were evaluable for toxicities and 12 for dose decisions and response. No serious adverse events (AEs) or dose-limiting toxicities (DLTs) were observed during cycles 1 to 3 (DLT evaluation period), and dose level 3 was identified as the RP2D. The most frequent AEs were tumor flare, fatigue, neutropenia, and leukopenia. Three patients developed grade 1 or 2 autoimmune thyroiditis that resolved with treatment. Best overall and skin response rates were 58.3% (95% confidence interval (95% CI), 27.7-84.8%) and 75% (95% CI: 42.8-94.5%), respectively. The median cycles of treatment were 11, and the median duration of response was 25.5 months. The combination showed clinical activity with 7 partial responses and 4 stable disease. Potentially predictive immune signatures were downregulation of -α signaling via NF-κB, interferon gamma, and phosphoinositide 3 kinase-AKT-mammalian target of rapamycin signaling pathways in responders and upregulation of MYC targets and proinflammatory pathways in nonresponders. Profiling of immune cell compositions revealed changes in individual immune cell clusters based on treatment response. This trial was registered at www.ClinicalTrials.gov as #NCT03011814.http://www.sciencedirect.com/science/article/pii/S2473952925001065
spellingShingle Christiane Querfeld
Joycelynne Palmer
Zhen Han
Xiwei Wu
Yate-Ching Yuan
Min-Hsuan Chen
Chingyu Su
Ni-Chun Tsai
D. Lynne Smith
Samantha N. Hammond
Liliana Crisan
Joo Y. Song
Raju Pillai
Steven T. Rosen
Jasmine Zain
Phase 1 trial of durvalumab (anti–PD-L1) combined with lenalidomide in relapsed/refractory cutaneous T-cell lymphoma
Blood Advances
title Phase 1 trial of durvalumab (anti–PD-L1) combined with lenalidomide in relapsed/refractory cutaneous T-cell lymphoma
title_full Phase 1 trial of durvalumab (anti–PD-L1) combined with lenalidomide in relapsed/refractory cutaneous T-cell lymphoma
title_fullStr Phase 1 trial of durvalumab (anti–PD-L1) combined with lenalidomide in relapsed/refractory cutaneous T-cell lymphoma
title_full_unstemmed Phase 1 trial of durvalumab (anti–PD-L1) combined with lenalidomide in relapsed/refractory cutaneous T-cell lymphoma
title_short Phase 1 trial of durvalumab (anti–PD-L1) combined with lenalidomide in relapsed/refractory cutaneous T-cell lymphoma
title_sort phase 1 trial of durvalumab anti pd l1 combined with lenalidomide in relapsed refractory cutaneous t cell lymphoma
url http://www.sciencedirect.com/science/article/pii/S2473952925001065
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