Safety and efficacy of intra-arterial bone marrow mononuclear cell transplantation in patients with acute ischaemic stroke in Spain (IBIS trial): a phase 2, randomised, open-label, standard-of-care controlled, multicentre trial.


Journal

The Lancet. Neurology
ISSN: 1474-4465
Titre abrégé: Lancet Neurol
Pays: England
ID NLM: 101139309

Informations de publication

Date de publication:
Feb 2023
Historique:
received: 21 09 2022
revised: 14 11 2022
accepted: 01 12 2022
entrez: 21 1 2023
pubmed: 22 1 2023
medline: 25 1 2023
Statut: ppublish

Résumé

Pilot clinical trials have shown the safety of intra-arterial bone marrow mononuclear cells (BMMNCs) in stroke. However, the efficacy of different doses of intra-arterial BMMNCs in patients with acute stroke has not been tested in a randomised clinical trial. We aimed to show safety and efficacy of two different doses of autologous intra-arterial BMMNC transplantation in patients with acute stroke. The IBIS trial was a multicentre phase 2, randomised, controlled, investigator-initiated, assessor-blinded, clinical trial, in four stroke centres in Spain. We included patients (aged 18-80 years) with a non-lacunar, middle cerebral artery ischaemic stroke within 1-7 days from stroke onset and with a National Institutes of Health Stroke Scale score of 6-20. We randomly assigned patients (2:1:1) with a computer-generated randomisation sequence to standard of care (control group) or intra-arterial injection of autologous BMMNCs at one of two different doses (2 × 10 Between April 1, 2015, and May 20, 2021, we assessed 114 patients for eligibility. We randomly assigned 77 (68%) patients: 38 (49%) to the control group, 20 (26%) to the low-dose BMMNC group, and 19 (25%) the high-dose BMMNC group. The mean age of participants was 62·4 years (SD 12·7), 46 (60%) were men, 31 (40%) were women, all were White, and 63 (82%) received thrombectomy. The median NIHSS score before randomisation was 12 (IQR 9-15), with intra-arterial BMMNC injection done a median of 6 days (4-7) after stroke onset. The primary efficacy outcome occurred in 14 (39%) patients in the control group versus ten (50%) in the low-dose group (adjusted odds ratio 2·08 [95% CI 0·55-7·85]; p=0·28), eight (44%) in the high-dose group (1·89 [0·52-6·96]; p=0·33), and 18 (47%) in the pooled BMMNC group (2·22 [0·72-6·85]; p=0·16). We found no differences in the proportion of patients who had adverse events or dose-related events, but two patients had a groin haematoma after cell injection in the low-dose BMMNC group. Intra-arterial BMMNCs were safe in patients with acute ischaemic stroke, but we found no significant improvement at 180 days on the mRS. Further clinical trials are warranted to investigate whether improvements might be possible at different timepoints. Instituto de Salud Carlos III co-funded by the European Regional Development Fund/European Social Fund, Mutua Madrileña, and the Regional Ministry of Health of Andalusia.

Sections du résumé

BACKGROUND BACKGROUND
Pilot clinical trials have shown the safety of intra-arterial bone marrow mononuclear cells (BMMNCs) in stroke. However, the efficacy of different doses of intra-arterial BMMNCs in patients with acute stroke has not been tested in a randomised clinical trial. We aimed to show safety and efficacy of two different doses of autologous intra-arterial BMMNC transplantation in patients with acute stroke.
METHODS METHODS
The IBIS trial was a multicentre phase 2, randomised, controlled, investigator-initiated, assessor-blinded, clinical trial, in four stroke centres in Spain. We included patients (aged 18-80 years) with a non-lacunar, middle cerebral artery ischaemic stroke within 1-7 days from stroke onset and with a National Institutes of Health Stroke Scale score of 6-20. We randomly assigned patients (2:1:1) with a computer-generated randomisation sequence to standard of care (control group) or intra-arterial injection of autologous BMMNCs at one of two different doses (2 × 10
FINDINGS RESULTS
Between April 1, 2015, and May 20, 2021, we assessed 114 patients for eligibility. We randomly assigned 77 (68%) patients: 38 (49%) to the control group, 20 (26%) to the low-dose BMMNC group, and 19 (25%) the high-dose BMMNC group. The mean age of participants was 62·4 years (SD 12·7), 46 (60%) were men, 31 (40%) were women, all were White, and 63 (82%) received thrombectomy. The median NIHSS score before randomisation was 12 (IQR 9-15), with intra-arterial BMMNC injection done a median of 6 days (4-7) after stroke onset. The primary efficacy outcome occurred in 14 (39%) patients in the control group versus ten (50%) in the low-dose group (adjusted odds ratio 2·08 [95% CI 0·55-7·85]; p=0·28), eight (44%) in the high-dose group (1·89 [0·52-6·96]; p=0·33), and 18 (47%) in the pooled BMMNC group (2·22 [0·72-6·85]; p=0·16). We found no differences in the proportion of patients who had adverse events or dose-related events, but two patients had a groin haematoma after cell injection in the low-dose BMMNC group.
INTERPRETATION CONCLUSIONS
Intra-arterial BMMNCs were safe in patients with acute ischaemic stroke, but we found no significant improvement at 180 days on the mRS. Further clinical trials are warranted to investigate whether improvements might be possible at different timepoints.
FUNDING BACKGROUND
Instituto de Salud Carlos III co-funded by the European Regional Development Fund/European Social Fund, Mutua Madrileña, and the Regional Ministry of Health of Andalusia.

Identifiants

pubmed: 36681446
pii: S1474-4422(22)00526-9
doi: 10.1016/S1474-4422(22)00526-9
pii:
doi:

Banques de données

ClinicalTrials.gov
['NCT02178657']

Types de publication

Randomized Controlled Trial Multicenter Study Clinical Trial, Phase II Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

137-146

Informations de copyright

Copyright © 2023 Elsevier Ltd. All rights reserved.

Déclaration de conflit d'intérêts

Declaration of interests We declare no competing interests.

Auteurs

Francisco Moniche (F)

Department of Neurology, Virgen del Rocío University Hospital, Seville, Spain; Neurovascular Lab, Instituto de Biomedicina de Sevilla-IBiS, Seville, Spain. Electronic address: francisco.moniche.sspa@juntadeandalucia.es.

Juan Antonio Cabezas-Rodriguez (JA)

Department of Neurology, Virgen del Rocío University Hospital, Seville, Spain.

Roberto Valverde (R)

Department of Neurology, Department of Radiology, Reina Sofía University Hospital, Cordoba, Spain.

Irene Escudero-Martinez (I)

Department of Neurology, Virgen del Rocío University Hospital, Seville, Spain; Neurovascular Lab, Instituto de Biomedicina de Sevilla-IBiS, Seville, Spain.

Lucia Lebrato-Hernandez (L)

Department of Neurology, Virgen del Rocío University Hospital, Seville, Spain.

Blanca Pardo-Galiana (B)

Department of Neurology, Virgen del Rocío University Hospital, Seville, Spain.

Leire Ainz (L)

Department of Neurology, Virgen del Rocío University Hospital, Seville, Spain.

Manuel Medina-Rodriguez (M)

Department of Neurology, Virgen del Rocío University Hospital, Seville, Spain; Neurovascular Lab, Instituto de Biomedicina de Sevilla-IBiS, Seville, Spain.

Javier de la Torre (J)

Department of Neurology, Virgen del Rocío University Hospital, Seville, Spain.

Virginia Escamilla-Gomez (V)

Department of Hematology, Virgen del Rocío University Hospital, Seville, Spain.

Joaquin Ortega-Quintanilla (J)

Interventional Neuroradiology, Virgen del Rocío University Hospital, Seville, Spain.

Elena Zapata-Arriaza (E)

Interventional Neuroradiology, Virgen del Rocío University Hospital, Seville, Spain.

Asier de Albóniga-Chindurza (A)

Interventional Neuroradiology, Virgen del Rocío University Hospital, Seville, Spain.

Fernando Mancha (F)

Neurovascular Lab, Instituto de Biomedicina de Sevilla-IBiS, Seville, Spain.

Miguel-Angel Gamero (MA)

Department of Neurology, Virgen Macarena University Hospital, Seville, Spain.

Soledad Perez (S)

Department of Neurology, Virgen Macarena University Hospital, Seville, Spain.

Raul Espinosa-Rosso (R)

Department of Neurology, Puerta del Mar University Hospital, Cadiz, Spain.

Lucia Forero-Diaz (L)

Department of Neurology, Puerta del Mar University Hospital, Cadiz, Spain.

Miguel Moya (M)

Department of Neurology, Puerta del Mar University Hospital, Cadiz, Spain.

Pilar Piñero (P)

Department of Radiology, Virgen del Rocío University Hospital, Seville, Spain.

Cristina Calderón-Cabrera (C)

Department of Hematology, Virgen del Rocío University Hospital, Seville, Spain.

Sonia Nogueras (S)

Cell Therapy Unit, Reina Sofía University Hospital, IMIBIC, University of Córdoba, Córdoba, Spain.

Rosario Jimenez (R)

Cell Therapy Unit, Reina Sofía University Hospital, IMIBIC, University of Córdoba, Córdoba, Spain.

Vanesa Martin (V)

Department of Hematology, Reina Sofía University Hospital, IMIBIC, University of Córdoba, Córdoba, Spain; Cell Therapy Unit, Reina Sofía University Hospital, IMIBIC, University of Córdoba, Córdoba, Spain.

Fernando Delgado (F)

Interventional Neuroradiology, Department of Radiology, Reina Sofía University Hospital, Cordoba, Spain.

Juan-José Ochoa-Sepúlveda (JJ)

Department of Neurology, Department of Radiology, Reina Sofía University Hospital, Cordoba, Spain.

Blanca Quijano (B)

Coordination Unit of the Andalusian Network for the design and translation of Advanced Therapies, Seville, Spain.

Rosario Mata (R)

Coordination Unit of the Andalusian Network for the design and translation of Advanced Therapies, Seville, Spain.

Monica Santos-González (M)

Production and Reprogramming Cell Unit of Seville, Andalusian Network for the Design and Translation of Advanced Therapies, Seville, Spain; Centro de Transfusiones, Tejidos y Células de Sevilla (CTTS), Fundación Pública Andaluza para la Gestión de la Investigación en Salud en Sevilla (FISEVI), Seville, Spain.

Gloria Carmona-Sanchez (G)

Coordination Unit of the Andalusian Network for the design and translation of Advanced Therapies, Seville, Spain; Production and Reprogramming Cell Unit of Seville, Andalusian Network for the Design and Translation of Advanced Therapies, Seville, Spain.

Concha Herrera (C)

Department of Hematology, Reina Sofía University Hospital, IMIBIC, University of Córdoba, Córdoba, Spain; Cell Therapy Unit, Reina Sofía University Hospital, IMIBIC, University of Córdoba, Córdoba, Spain.

Alejandro Gonzalez (A)

Interventional Neuroradiology, Virgen del Rocío University Hospital, Seville, Spain.

Joan Montaner (J)

Neurovascular Lab, Instituto de Biomedicina de Sevilla-IBiS, Seville, Spain; Department of Neurology, Virgen Macarena University Hospital, Seville, Spain.

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