Long-term follow-up after nonmyeloablative allogeneic hematopoietic stem cell transplantation for systemic sclerosis

Abstract

Systemic sclerosis (SSc) characteristically consists of fibrotic changes in various organs, and immunological abnormality is the main cause of the disease. Although high-dose immunosuppressive therapies with autologous or allogeneic hematopoietic stem cell support can reverse the disease course, they have a high treatment-related mortality. We report the successful use of nonmyeloablative allogeneic hematopoietic stem cell transplantation (HSCT) for SSc. A 40-year-old woman with diffuse scleroderma and interstitial pneumonia underwent allogeneic peripheral blood stem cell transplantation from an HLA-identical sibling after conditioning with low-dose total-body irradiation and fludarabine. Prophylaxis for graft-versus-host disease (GVHD) consisted of cyclosporine and mycophenolate mofetil. No infection or acute GVHD developed. One year after transplantation, the patient developed membranous glomerulopathy caused by chronic GVHD that was successfully treated with prednisolone. The patient's skin score decreased dramatically, and her pulmonary function is stable 4 years after transplantation. Nonmyeloablative allogeneic HSCT may be more effective than conventional therapies for SSc.

Cellular-Based Therapies in Systemic Sclerosis: From Hematopoietic Stem Cell Transplant to Innovative Approaches

“…By May 2022, 776 patients with SSc have been treated with HSCT within the EBMT registry ( Figure 1 ). While autologous HSCT is considered to be a standard treatment option [ 33 ], use of allogeneic HSCT for SSc remains anecdotal due to higher risk of transplant-related complications [ 34 , 35 , 36 , 37 ]. Reduced-toxicity HSCT platforms are needed to further investigate and expand allogeneic procedure in AD patients [ 38 ].…”

Guidelines of the Brazilian Society of Bone Marrow Transplantation on hematopoietic stem cell transplantation as a treatment for the autoimmune diseases systemic sclerosis and multiple sclerosis

“…One year after transplantation, she developed membranous glomerulonephritis due to GVHD which remitted with corticosteroids. The skin score improved dramatically and lung function remained stable for four years after the procedure (29) . Overall, individual reports of allogeneic transplants have described encouraging results, but they are still insufficient to prove safety and superiority to autologous transplants.…”

Consenso brasileiro para transplante de células-tronco hematopoéticas para tratamento de doenças autoimunes

“…No ano de 2008, um grupo japonês relatou sua experiência com transplante de células-tronco alogênicas para uma paciente portadora de esclerose sistêmica. 24 Houve melhora cutânea e estabilização da função pulmonar, embora a paciente tenha desenvolvido doença do enxerto-versus-hospedeiro renal, na forma de glomerulopatia membranosa, que foi responsiva ao tratamento com corticosteroides. Em 2007, de modo semelhante, Loh e colaboradores publicaram os resultados de um transplante alogênico não mieloablativo para uma paciente portadora de acometimento cutâneo difuso e hipertensão pulmonar.…”