Safety, Feasibility, and Preliminary Efficacy of Allogeneic MSCs to Treat Advanced Femoral Head Osteonecrosis (ALOFEM) - AOFEM: A Pilot Study in Young Onco-Hematological Patients

Sep 29, 2026

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Severe femoral head osteonecrosis (ONFH) caused by corticosteroid therapy in symptomatic young onco-hematological patients lacks a safer effective alternative, because early total hip replacement (THR) is high risk. This pilot phase 1 trial assessed the feasibility, safety, and early efficacy of allogeneic expanded mesenchymal stromal cells (MSCs) for bone regeneration. Open, noncontrolled, nonrandomized evaluation in seven hips determined that the approach was feasible and demonstrated safety at 1 and 4 years, with no related serious adverse events, and early efficacy signals in case analysis.

LanguageEnglish

CategoryResearch & Report

FormatPDF

Pages10

Reading Time25 min

AccessFree to download

Introduction

Objectives

Methods

Results

Conclusions

Trial registration and keywords

Wiley Stem Cells International Volume 2026, Article ID 1986839, 10 pages [https://doi.org/10.1155/sci/1986839](https://doi.org/10.1155/sci/1986839) Research Article Safety, Feasibility, and Preliminary EfﬁcacyofAllogeneic MSCsto Treat Advanced Femoral Head Osteonecrosis (ALOFEM): A Pilot Study in Young Onco-Hematological Patients Enrique Gómez-Barrena , 1,2 Norma G. Padilla-Eguiluz ,2 Juan Cabello-Blanco , 1 José Juan Pozo-Kreilinger,3 Yasmina Mozo-del-Castillo ,4 María E. Martínez-Muñoz,5 Trinidad Martín-Donaire,5 Rocío Zafra ,5 Rafael F. Duarte ,5 Ana Velasco-Iglesias ,6 Cristina Avendaño-Solà ,6 and Concepción Payares-Herrera6 1Department of Orthopedic Surgery, La Paz University Hospital-IdiPAZ, Madrid, Spain 2Department of Surgery, School of Medicine, Autonomous University of Madrid-IdiPAZ, Madrid, Spain 3Department of Pathology, La Paz University Hospital, Madrid, Spain 4Department of Pediatric of Onco-Hematology, La Paz University Hospital, Madrid, Spain 5Department of Hematology and GMP Cell Production Unit, Puerta de Hierro Majadahonda University Hospital, Madrid, Spain 6Department of Clinical Pharmacology, Puerta de Hierro-Majadahonda University Hospital, Madrid, Spain Correspondence should be addressed to Enrique Gómez-Barrena; [enrique.gomezbarrena@uam.es](enrique.gomezbarrena@uam.es) Received 18 August 2025; Revised 30 September 2025; Accepted 5 December 2025 Academic Editor: Harleen Khatra Copyright © 2026 Enrique Gómez-Barrena et al. Stem Cells International published by John Wiley & Sons Ltd. This is an open access article under the terms of the Creative Commons Attribution License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited. Background: Severe osteonecrosis of the femoral head (ONFH) secondary to corticosteroid therapy in symptomatic, hematological young patients currently has no therapeutic alternative, and early total hip replacement (THR) is a high-risk intervention in those patients. Objectives: To evaluate feasibility, safety, and early efﬁcacy of allogeneic expanded mesenchymal stromal cells (MSCs) in a pilot clinical trial. Methods: Pilot phase 1 open, noncontrolled, nonrandomized clinical trial evaluating the bone regeneration capacity in seven hips from four patients (young females 11–19 year old) with symptomatic, severe bilateral femoral head osteonecrosis (secondary to corticosteroid therapy), 1 year after being surgically treated with 140 × 106 allogenic MSC plus forage. Results: The proposed therapy proved feasibility, safety at 1 and 4 years (no related serious adverse events [SAEs]), and early efﬁcacy (nonsigniﬁcant) in the case analysis (5/7 hips avoiding THR at 4 years) . Conclusions: The implantation of expanded allogeneic MSC in young patients to prevent conversion to a THR or collapse of the femoral head due to severe osteonecrosis is feasible without safety concerns in the longer-term follow-up (FU) upto 4 years. Trial Registration: EudraCT number: 2018-000886-35 Keywords: allogenic bone marrow derived MSC; expanded MSCs; osteonecrosis of the femoral head (ONFH); pilot phase 1 clinical trial 1. Introduction Orthopedic surgeons are looking for innovative therapies when bone necrosis threatens the bone structure and function, to launch bone regeneration through tissue engineering [1] and tissue repair based on cell therapy [2]. One developed approach consists of harvesting bone marrow (BM) from the patient, isolating mesenchymal stromal cells (MSCs) by their plastic adherence, expanding the adherent cells to a sufﬁcient number, stimulate their differentiation towards osteogenesis and then 2 Stem Cells International seeding them onto a suitable synthetic scaffold prior to reimplantation [3, 4]. Implanting cells to augment the host cells after differentiation in the damaged bone has been proposed to procure osteogenesis [5]. Osteonecrosis of the femoral head (ONFH), whether i

FAQs

What condition does the ALOFEM pilot study target?

The study targets advanced osteonecrosis of the femoral head (ONFH), specifically cases arising secondary to corticosteroid therapy in young onco-hematological patients.

How was the MSC therapy evaluated in this trial?

It was assessed as a pilot phase 1 open, noncontrolled, nonrandomized clinical trial, focusing on bone regeneration capacity in seven hips from four young female patients about one year after treatment with allogeneic MSCs.

What did the study report about safety and adverse events?

The proposed therapy showed feasibility and safety at 1 and 4 years, reporting no related serious adverse events (SAEs).

Did the treatment reduce the need for total hip replacement (THR)?

In the case analysis, early efficacy was described as nonsignificant, and five of seven hips avoided THR at 4 years.

Sep 29, 2026

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