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Long-Term Results of a Phase I/II Clinical Trial of Autologous Mesenchymal Stem Cell Therapy for Femoral Head Osteonecrosis
(1) Background: Osteonecrosis of the femoral head (ONFH) is characterized by impaired vascularization with ischemia resulting in bone cell death, leading to the deterioration of the hip joint. Mesenchymal stem/stromal cells (MSCs) are an attractive potential therapeutic approach in this setting. The...
Autores principales: | , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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MDPI
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10051457/ https://www.ncbi.nlm.nih.gov/pubmed/36983120 http://dx.doi.org/10.3390/jcm12062117 |
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author | Blanco, Juan F. Garcia-Garcia, Francisco J. Villarón, Eva M. da Casa, Carmen Fidalgo, Helena López-Parra, Miriam Santos, José A. Sánchez-Guijo, Fermín |
author_facet | Blanco, Juan F. Garcia-Garcia, Francisco J. Villarón, Eva M. da Casa, Carmen Fidalgo, Helena López-Parra, Miriam Santos, José A. Sánchez-Guijo, Fermín |
author_sort | Blanco, Juan F. |
collection | PubMed |
description | (1) Background: Osteonecrosis of the femoral head (ONFH) is characterized by impaired vascularization with ischemia resulting in bone cell death, leading to the deterioration of the hip joint. Mesenchymal stem/stromal cells (MSCs) are an attractive potential therapeutic approach in this setting. The aim of this study is to evaluate the clinical improvement in terms of pain and quality of life, as well as the safety of the procedure during the follow-up of patients. (2) Methods: A Phase I–II Open-Label Non-Randomized Prospective clinical trial was conducted. Eight patients with idiopathic ONFH and stage < IIC in the ARCO classification were included. Four weeks before therapy, 40 mL of autologous bone marrow was obtained, and MSCs were expanded under Good-Manufacturing-Practice (GMP) standards. Study medication consisted of a suspension of autologous BM-derived MSCs (suspended in a solution of 5–10 mL of saline and 5% human albumin) in a single dose of 0.5–1 × 10(6) cells/kg of the patient, administered intraosseously with a trocar and under radioscopic control. Per-protocol monitoring of patients included a postoperative period of 12 months, with a clinical and radiological assessment that included the visual analog scale (VAS), the Harris scale, the SF-36, and the radiological evolution of both hips. In addition, all patients were further followed up for eight years to assess the need for long-term total hip replacement (THR) surgery. (3) Results: Median age of patients included was 48.38 ± 7.38 years, and all patients were men. Autologous MSCs were expanded in all cases. There were no adverse effects related to cell administration. Regarding efficacy, both VAS and ODI scores improved after surgery. Radiologically, 12.5% of patients improved at the end of follow-up, whereas 50% improved clinically. No adverse effects related to the procedure were recorded, and none of the patients needed THR surgery within the first year after MSC therapy. (4) Conclusions: The use of autologous MSCs for patients with ONFH disease is feasible, safe in the long term, and potentially effective. |
format | Online Article Text |
id | pubmed-10051457 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2023 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-100514572023-03-30 Long-Term Results of a Phase I/II Clinical Trial of Autologous Mesenchymal Stem Cell Therapy for Femoral Head Osteonecrosis Blanco, Juan F. Garcia-Garcia, Francisco J. Villarón, Eva M. da Casa, Carmen Fidalgo, Helena López-Parra, Miriam Santos, José A. Sánchez-Guijo, Fermín J Clin Med Article (1) Background: Osteonecrosis of the femoral head (ONFH) is characterized by impaired vascularization with ischemia resulting in bone cell death, leading to the deterioration of the hip joint. Mesenchymal stem/stromal cells (MSCs) are an attractive potential therapeutic approach in this setting. The aim of this study is to evaluate the clinical improvement in terms of pain and quality of life, as well as the safety of the procedure during the follow-up of patients. (2) Methods: A Phase I–II Open-Label Non-Randomized Prospective clinical trial was conducted. Eight patients with idiopathic ONFH and stage < IIC in the ARCO classification were included. Four weeks before therapy, 40 mL of autologous bone marrow was obtained, and MSCs were expanded under Good-Manufacturing-Practice (GMP) standards. Study medication consisted of a suspension of autologous BM-derived MSCs (suspended in a solution of 5–10 mL of saline and 5% human albumin) in a single dose of 0.5–1 × 10(6) cells/kg of the patient, administered intraosseously with a trocar and under radioscopic control. Per-protocol monitoring of patients included a postoperative period of 12 months, with a clinical and radiological assessment that included the visual analog scale (VAS), the Harris scale, the SF-36, and the radiological evolution of both hips. In addition, all patients were further followed up for eight years to assess the need for long-term total hip replacement (THR) surgery. (3) Results: Median age of patients included was 48.38 ± 7.38 years, and all patients were men. Autologous MSCs were expanded in all cases. There were no adverse effects related to cell administration. Regarding efficacy, both VAS and ODI scores improved after surgery. Radiologically, 12.5% of patients improved at the end of follow-up, whereas 50% improved clinically. No adverse effects related to the procedure were recorded, and none of the patients needed THR surgery within the first year after MSC therapy. (4) Conclusions: The use of autologous MSCs for patients with ONFH disease is feasible, safe in the long term, and potentially effective. MDPI 2023-03-08 /pmc/articles/PMC10051457/ /pubmed/36983120 http://dx.doi.org/10.3390/jcm12062117 Text en © 2023 by the authors. https://creativecommons.org/licenses/by/4.0/Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Article Blanco, Juan F. Garcia-Garcia, Francisco J. Villarón, Eva M. da Casa, Carmen Fidalgo, Helena López-Parra, Miriam Santos, José A. Sánchez-Guijo, Fermín Long-Term Results of a Phase I/II Clinical Trial of Autologous Mesenchymal Stem Cell Therapy for Femoral Head Osteonecrosis |
title | Long-Term Results of a Phase I/II Clinical Trial of Autologous Mesenchymal Stem Cell Therapy for Femoral Head Osteonecrosis |
title_full | Long-Term Results of a Phase I/II Clinical Trial of Autologous Mesenchymal Stem Cell Therapy for Femoral Head Osteonecrosis |
title_fullStr | Long-Term Results of a Phase I/II Clinical Trial of Autologous Mesenchymal Stem Cell Therapy for Femoral Head Osteonecrosis |
title_full_unstemmed | Long-Term Results of a Phase I/II Clinical Trial of Autologous Mesenchymal Stem Cell Therapy for Femoral Head Osteonecrosis |
title_short | Long-Term Results of a Phase I/II Clinical Trial of Autologous Mesenchymal Stem Cell Therapy for Femoral Head Osteonecrosis |
title_sort | long-term results of a phase i/ii clinical trial of autologous mesenchymal stem cell therapy for femoral head osteonecrosis |
topic | Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10051457/ https://www.ncbi.nlm.nih.gov/pubmed/36983120 http://dx.doi.org/10.3390/jcm12062117 |
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