Salvage Therapy Using Mesenchymal Stromal Cells “MSC-FFM” For a Severe Gastrointestinal Steroid-Refractory Acute Graft-Versus-Host Disease in a 12-Year-Old Boy with Sickle Cell Disease

Article Article scientifique Rapport de cas Salvage Therapy Using Mesenchymal Stromal Cells “MSC-FFM” For a Severe Gastrointestinal Steroid-Refractory Acute Graft-Versus-Host Disease in a 12-Year-Old Boy with Sickle Cell Disease eng 2024-01-04 2024-01-04 author Valpacos Nogueira Mélanie 753746 author Grazioli Serge 503187 Département de Pédiatrie, Gynécologie et Obstétrique (PETRI) author Baleydier Frédéric 987669 Département de Pédiatrie, Gynécologie et Obstétrique (PETRI) author Von Bueren André 0000-0003-4197-6264 885935 Département de Pédiatrie, Gynécologie et Obstétrique (PETRI) author Bernard Fanette 810499 author Rock Nathalie 826963 author Mamez Anne-Claire 918000 author Rougemont-Pidoux Anne-Laure 29140 Département de pathologie et immunologie (PATIM) author Bader Peter author Ansari Djaberi Marc Georges 61572 Département de Pédiatrie, Gynécologie et Obstétrique (PETRI) 101577 10.29011/2574-7754.101577 2574-7754 https://www.gavinpublishers.com/article/view/salvage-therapy-using-mesenchymal-stromal-cells-msc-ffm-for-a-severe-gastrointestinal-steroid-refractory-acute-graft-versus-host-disease-in-a-12-year-old-boy-with-sickle-cell-disease Acute Graft-versus-Host Disease (aGvHD) is a frequent complication after a Hematopoietic Stem Cell Transplantation (HSCT). Its gastrointestinal subtype is associated with a high mortality, especially in steroid-refractory cases. Managing Steroid-Refractory aGvHD (SR-aGvHD) remains challenging due to the lack of validated second-line immunosuppressive strategies. Mesenchymal Stromal Cells (MSCs) are a therapeutic option for SR-aGvHD, but its efficacy and clinical response in children varies. Two recent studies using pooled bone marrow mononuclear cells from multiple donors, MSC-FFM, have shown satisfactory overall response rates but the literature is still lacking in children. We report the case of a 12-year-old boy with severe sickle cell disease and exchange transfusion dependency, indicating HSCT. Following allogeneic related-donor HSCT, he developed grade IV, gastrointestinal SR-aGvHD with an unfavorable clinical outcome despite intensive treatment with mycophenolate mofetil, methylprednisolone, and ruxolitinib. After nine days of methylprednisolone, a weekly MSC–FFM treatment was initiated that led to partial resolution of the gastrointestinal symptoms at D+18 after the first infusion of MSC–FFM and transient complete clinical remission of the gastrointestinal SR-aGvHD at D+79. In summary, MSC–FFM infusion was well tolerated, leading to significant clinical improvement and transient complete clinical remission of severe gastrointestinal SR-aGvHD. Steroid-Refractory Acute Graft-Versus-Host Disease Pediatric Gastrointestinal aGvHD Mesenchymal Stromal Cells MSC–FFM Dewey 616 Dewey 616.07 Dewey 618 58 Département de pédiatrie, gynécologie et obstétrique 223 56 Département de médecine 221 137 Département de pathologie et immunologie 21F false 564 Oncologie et hématologie pédiatriques 907 Annals of case reports 8 6 9758dc7b-e080-4307-a9f5-4ec7a48907d3 Article (Published version) Public Article_scientifique_from_unpaywall.pdf 997907 application/pdf CC BY-SA-4.0 b2df0885c523d3c3333c8ee9ee80f6f624bc1ca5a56a7175c8849e15e50730ad openAccess Faculté de médecine / Section de médecine clinique / Département de pédiatrie, gynécologie et obstétrique Faculté de médecine / Section de médecine clinique / Département de médecine Faculté de médecine / Section de médecine fondamentale / Département de pathologie et immunologie

Title	Published in	Year	Views	Downloads

Salvage Therapy Using Mesenchymal Stromal Cells “MSC-FFM” For a Severe Gastrointestinal Steroid-Refractory Acute Graft-Versus-Host Disease in a 12-Year-Old Boy with Sickle Cell Disease	Annals of case reports	2024	24	143
Diagnosis and Management of Necrotizing Enterocolitis: An International Survey of Neonatologists and Pediatric Surgeons	Neonatology	2018	517	0

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Valpacos Nogueira, Mélanie