Endobronchial autologous bone marrow–mesenchymal stromal cells in idiopathic pulmonary fibrosis: a phase I trial
| Autor: | Fermín Sánchez-Guijo, Enrique J. Andreu, Juan P. de-Torres, Rosa Cordovilla, Arantza Campo, Eva Villarón, José María González-Ruiz, Miguel Barrueco, Maria del Mar Ocon, Jesus Pueyo, Jorge M. Núñez-Córdoba, Ana B. Alcaide, Javier J. Zulueta, Felipe Prosper |
|---|---|
| Jazyk: | angličtina |
| Rok vydání: | 2021 |
| Předmět: |
Pulmonary and Respiratory Medicine
medicine.medical_specialty Vital capacity Gastroenterology Interstitial Lung Disease Pulmonary function testing 03 medical and health sciences Idiopathic pulmonary fibrosis 0302 clinical medicine Internal medicine Medicine 030212 general & internal medicine Respiratory system Adverse effect business.industry Mesenchymal stem cell Original Articles medicine.disease Clinical trial medicine.anatomical_structure 030228 respiratory system Bone marrow business |
| Zdroj: | ERJ Open Research article-version (VoR) Version of Record ERJ Open Research, Vol 7, Iss 2 (2021) |
| ISSN: | 2312-0541 |
| Popis: | Rationale Idiopathic pulmonary fibrosis (IPF) has a dismal prognosis. Mesenchymal stromal cells (MSCs) have shown benefit in other inflammatory diseases. Objectives To evaluate the safety and feasibility of endobronchial administration of bone marrow autologous MSCs (BM-MSC) in patients with mild-to-moderate IPF. Methods A phase I multicentre clinical trial (ClinicalTrials.gov NCT01919827) with a single endobronchial administration of autologous adult BM-MSCs in patients diagnosed with mild-to-moderate IPF. In a first escalating-dose phase, three patients were included sequentially in three dose cohorts (10×106, 50×106 and 100×106 cells). In a second phase, nine patients received the highest tolerated dose. Follow-up with pulmonary function testing, 6-min walk test and St George's Respiratory Questionnaire was done at 1, 2, 3, 6 and 12 months, and with computed tomography at 3, 6 and 12 months. Results 21 bone marrow samples were obtained from 17 patients. Three patients were excluded from treatment due to chromosome aberrations detected in MSCs after culture, and one patient died before treatment. Finally, 13 patients received the BM-MSC infusion. No treatment-related severe adverse events were observed during follow-up. Compared to baseline, the mean forced vital capacity showed an initial decline of 8.1% at 3 months. The number of patients without functional progression was six (46%) at 3 months and three (23%) at 12 months. Conclusions The endobronchial infusion of BM-MSCs did not cause immediate serious adverse events in IPF patients, but a relevant proportion of patients suffered clinical and/or functional progression. Genomic instability of BM-MSCs during culture found in three patients may be troublesome for the use of autologous MSCs in IPF patients. Endobronchial autologous mesenchymal stromal cells (MSCs) did not cause direct serious adverse events in IPF patients. However, significant progression was seen in seven out of 13 patients. Genomic instability of autologous MSCs may limit use in IPF. https://bit.ly/39akv7z |
| Databáze: | OpenAIRE |
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