Manghisi Beatrice, Cotilli Giulia, Fedele Marilena, Perfetti Paola, Terruzzi Elisabetta, Verga Luisa, Borin Lorenza Maria, Carrer Andrea, Fumagalli Monica, Ferrari Maria Beatrice, Moretti Alex, Rona Roberto, Benini Annalisa, Vergnano Beatrice, Palumbo Giovanni, Zincone Alessandra, Maglia Oscar, Scollo Chiara, Steidl Carolina, Iovino Lorenzo, Balduzzi Adriana, Piazza Rocco, Gambacorti-Passerini Carlo, Parma Matteo, Aroldi Andrea
Hematology Division, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy.
School of Medicine and Surgery, University of Milano-Bicocca, Monza, Italy.
Front Immunol. 2025 Apr 4;16:1563736. doi: 10.3389/fimmu.2025.1563736. eCollection 2025.
Chimeric antigen receptor (CAR)-T cell therapy is a powerful adoptive immunotherapy associated with significant toxicity, including cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS). As CAR-T usage expands, hyperinflammatory toxicities resembling hemophagocytic lymphohistiocytosis (HLH) syndrome are increasingly recognized. Immune effector cell-associated HLH-like syndrome (IEC-HS) describes HLH-like symptoms attributable to CAR-T cell therapy, often presenting as CRS resolves. Treatments for IEC-HS are adapted from primary HLH, including corticosteroids, the recombinant human interleukin (IL)-1 receptor antagonist anakinra and the Janus Kinase inhibitor ruxolitinib. Emapalumab, an anti-IFN-γ antibody, is promising but underexplored in adult IEC-HS cases. We report an adult B-cell acute lymphoblastic leukemia (B-ALL) patient treated with brexucabtagene autoleucel (brexu-cel). The patient developed CRS, refractory neurotoxicity, and IEC-HS with worsening multiorgan failure and hyperinflammatory markers. Treatment included tocilizumab, high-dose corticosteroids, anakinra, siltuximab, and ruxolitinib. Despite aggressive management, hyperinflammation and neurotoxicity persisted. Emapalumab was initiated on day +11, resulting in normalization of the biochemical parameters and full neurological recovery by day +21. The patient recovered from IEC-HS and underwent allogeneic stem cell transplantation. This case highlights the role of emapalumab in managing refractory IEC-HS and persistent neurotoxicity in adults, underscoring the need for targeted interventions in severe CAR-T complications.
嵌合抗原受体(CAR)-T细胞疗法是一种强大的过继性免疫疗法,会引发包括细胞因子释放综合征(CRS)和免疫效应细胞相关神经毒性综合征(ICANS)在内的显著毒性。随着CAR-T疗法应用范围的扩大,越来越多类似噬血细胞性淋巴组织细胞增生症(HLH)综合征的高炎症毒性被识别出来。免疫效应细胞相关HLH样综合征(IEC-HS)描述了由CAR-T细胞疗法引起的HLH样症状,通常在CRS消退时出现。IEC-HS的治疗方法借鉴了原发性HLH的治疗方法,包括使用皮质类固醇、重组人白细胞介素(IL)-1受体拮抗剂阿那白滞素和Janus激酶抑制剂鲁索替尼。抗IFN-γ抗体emapalumab在治疗成人IEC-HS病例方面前景广阔,但尚未得到充分研究。我们报告了一名接受布雷西尤单抗(brexu-cel)治疗的成年B细胞急性淋巴细胞白血病(B-ALL)患者。该患者出现了CRS、难治性神经毒性和IEC-HS,并伴有多器官功能衰竭和高炎症标志物加重。治疗措施包括使用托珠单抗、大剂量皮质类固醇、阿那白滞素、西妥昔单抗和鲁索替尼。尽管进行了积极治疗,但高炎症和神经毒性仍持续存在。在第+11天开始使用emapalumab,到第+21天时生化参数恢复正常,神经功能完全恢复。患者从IEC-HS中康复,并接受了异基因干细胞移植。该病例突出了emapalumab在治疗成人难治性IEC-HS和持续性神经毒性方面的作用,强调了针对严重CAR-T并发症进行靶向干预的必要性。