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Cyclophosphamide-free Mobilisation Increases Safety While Preserving the Efficacy of Autologous Haematopoietic.pdf (5.08 MB)

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2024-06-05

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Si us plau utilitzeu sempre aquest identificador per citar o enllaçar aquest document: https://hdl.handle.net/2445/216580

Cyclophosphamide-free Mobilisation Increases Safety While Preserving the Efficacy of Autologous Haematopoietic Stem Cell Transplantation in Refractory Crohn's Disease Patients

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https://doi.org/10.1093/ecco-jcc/jjae076

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Background and Aim Autologous haematopoietic stem cell transplantation [AHSCT] is a therapeutic option for refractory Crohn's disease [CD]. However, high adverse event rates related to chemotherapy toxicity and immunosuppression limit its applicability. This study aims to evaluate AHSCT's safety and efficacy using a cyclophosphamide [Cy]-free mobilisation regimen.Methods A prospective, observational study included 14 refractory CD patients undergoing AHSCT between June 2017 and October 2022. The protocol involved outpatient mobilisation with G-CSF 12-16 mu g/kg/daily for 5 days, and optional Plerixafor 240 mu g/d [1-2 doses] if the CD34 + cell count target was unmet. Standard conditioning with Cy and anti-thymocyte globulin was administered. Clinical, endoscopic, and radiological assessments were conducted at baseline and during follow-up.Results All patients achieved successful outpatient mobilisation [seven patients needed Plerixafor] and underwent transplantation. Median follow-up was 106 weeks (interquartile range [IQR] 52-348). No mobilisation-related serious adverse events [SAEs] or CD worsening occurred. Clinical and endoscopic remission rates were 71% and 41.7% at 26 weeks, 64% and 25% at 52 weeks, and 71% and 16.7% at the last follow-up, respectively. The percentage of patients who restarted CD therapy for clinical relapse and/or endoscopic/radiological activity was 14% at 26 weeks, 57% at 52 weeks, and 86% at the last follow-up, respectively. Peripheral blood cell populations and antibody levels post-AHSCT were comparable to Cy-based mobilisation.Conclusions Cy-free mobilisation is safe and feasible in refractory CD patients undergoing AHSCT. Although relapse occurs in a significant proportion of patients, clinical and endoscopic responses are achieved upon CD-specific therapy reintroduction. Graphical Abstract

Matèries

Malaltia de Crohn, Autotrasplantament, Ciclofosfamida

Matèries (anglès)

Crohn's disease, Autotransplantation, Cyclophosphamide

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Articles publicats en revistes (IDIBAPS: Institut d'investigacions Biomèdiques August Pi i Sunyer)

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GIORDANO, Antonio, ROVIRA TARRATS, Montserrat, VENY ÁLVAREZ-OSSORIO, Marisol, BARASTEGUI BAGET, Rebeca, MARÍN FERNÁNDEZ, Pedro josé, MARTÍNEZ MUÑOZ, Ma. carmen, FERNÁNDEZ AVILÉS, F., SUAREZ LLEDÓ GRANDE, María, DOMENECH, Ariadna, SERRAHIMA, Anna, LOZANO MOLERO, Miguel, CID VIDAL, Joan, ORDAS JIMENEZ, Ingrid, FERNÁNDEZ CLOTET, Agnès, CABALLOL OLIVA, Berta, GALLEGO BARRERO, Marta, VARA, Alejandro, MASAMUNT, Maria carme, GINER AGUDO, Àngel, TEUBEL, Iris, ESTELLER VIÑAL, Miriam, CORRALIZA MÁRQUEZ, Ana maria, PANÉS DÍAZ, Julià, SALAS MARTÍNEZ, Azucena, RICART, Elena (ricart gómez). Cyclophosphamide-free Mobilisation Increases Safety While Preserving the Efficacy of Autologous Haematopoietic Stem Cell Transplantation in Refractory Crohn's Disease Patients. _Journal Of Crohns & Colitis_. 2024. Vol. 18, núm. 10, pàgs. 1701-1712. [consulta: 24 de gener de 2026]. ISSN: 1876-4479. [Disponible a: https://hdl.handle.net/2445/216580]

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