Mesenchymal Stromal Cell Product “MSC-FFM” for Treatment of Severe Multi-Organ Steroid Refractory Acute Graft-Versus-Host Disease in an Infant with Congenital Dyserythropoietic Anemia

Leonie DH Gossel1, Janina Grau1, Andrea Jarisch1, Jan Sörensen1, Eva Rettinger1, Halvard Bonig2 and Peter Bader1*

1Department for Children and Adolescents, University Hospital Frankfurt, Germany
2Goethe University School of Medicine, Institute for Transfusion Medicine, and German Red Cross Blood Service BaWüHe, Germany

^*Correspondance to: Peter Bader 

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Abstract:

Steroid-Refractory acute Graft-versus-Host disease (SR-aGvHD) remains a therapeutic challenge and carries a devastating prognosis especially in infants and small children where no treatment is available yet. Through various studies Mesenchymal Stromal Cells (MSCs) have been proposed but definitive studies are pending. A specific off-the-shelf preparation of Bone Marrow (BM)- derived MSCs, “MSC-FFM” has previously been described and is currently being tested in a multicenter trial. Nevertheless, more insight into the clinical application of “MSC-FFM” in different patient populations is needed. Especially in those with disease constellations that prompt Hematopoietic Stem Cell Transplantation (HSCT) but that haven´t been studied as extensively yet. We report the case of a 1-year-old girl with transfusion-dependent Congenital Dyserythropoietic Anemia (CDA) type 2 who developed severe multi-organ SR-aGvHD following allogeneic HSCT. The aGvHD reached an overall grade IV according to the Glucksberg grading system with severe gastrointestinal hemorrhage which led to admission to the Pediatric Intensive Care Unit (PICU). AGvHD progressed despite treatment with Cyclosporine A (CsA), Methylprednisolone and Mycophenolate-Mofetil (MMF). Administration of weekly doses of “MSC-FFM” was initiated after 15 days (d) of futile methylprednisolone treatment and resulted in Partial Resolution (PR) at d+28 and Complete Resolution (CR) at d+34 after the start of MSC treatment. No toxicities were detected, and steroids were weaned on d+38 after the first dose of “MSC-FFM”. MMF was stopped on d+93 and CsA is being tapered. She shows a promising course of immune cell recovery and no chronic GvHD; she is a full donor chimera.

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Cite the Article:

Gossel LDH, Grau J, Jarisch A, Sörensen J, Rettinger E, Bonig H, et al. Mesenchymal Stromal Cell Product “MSC-FFM” for Treatment of Severe Multi-Organ Steroid Refractory Acute Graft-Versus-Host Disease in an Infant with Congenital Dyserythropoietic Anemia. Ann Clin Case Rep. 2022; 7: 2310..