Haematopoietic SCT in severe autoimmune diseases: updated guidelines of the European Group for Blood and Marrow Transplantation

Abstract

In 1997, the first consensus guidelines for haematopoietic SCT (HSCT) in autoimmune diseases (ADs) were published, while an international coordinated clinical programme was launched. These guidelines provided broad principles for the field over the following decade and were accompanied by comprehensive data collection in the European Group for Blood and Marrow Transplantation (EBMT) AD Registry. Subsequently, retrospective analyses and prospective phase I/II studies generated evidence to support the feasibility, safety and efficacy of HSCT in several types of severe, treatment-resistant ADs, which became the basis for larger-scale phase II and III studies. In parallel, there has also been an era of immense progress in biological therapy in ADs. The aim of this document is to provide revised and updated guidelines for both the current application and future development of HSCT in ADs in relation to the benefits, risks and health economic considerations of other modern treatments. Patient safety considerations are central to guidance on patient selection and HSCT procedural aspects within appropriately experienced and Joint Accreditation Committee of International Society for Cellular Therapy and EBMT accredited centres. A need for prospective interventional and non-interventional studies, where feasible, along with systematic data reporting, in accordance with EBMT policies and procedures, is emphasized.

Autologous stem-cell transplantation in treatment-refractory Crohn's disease: an analysis of pooled data from the ASTIC trial

“…Although this was accepted practice at the initiation of the trial, 15 lower intensity mobilisation regimes are currently recommended to reduce the toxicity of the procedure. 10,23 Importantly, patients who proceeded immediately to conditioning and stem cell rescue experienced more SAEs on univariate analysis that is likely due to the additional doses of cyclophosphamide received within in a short time frame.…”

“…The trial was conducted in six European countries at eleven centres approved for allogeneic transplantation by JACIE (Joint Accreditation Committee of the International Society for Cellular Therapy) and the European Society for Blood and Marrow Transplantation (EBMT). 15,16 This manuscript reports the clinical, radiological and endoscopic outcomes from all patients with evaluable data at one year after HSCT. It includes patients initially randomised to an early transplant and those who subsequently underwent HSCT after conventional therapy.…”

“…An intermediate intensity conditioning regimen was used which comprised intravenous cyclophosphamide 50 mg/kg/day x four days and, from day three, rabbit antithymocyte globulin (Genzyme) 2.5 mg/kg/day and methylprednisolone 1 mg/kg/day x three days, with infusion of unselected autologous stem cells (minimum 3x10 6 CD34+ cells/kg) on day seven. 15 All patients were followed for one year after HSCT and could receive standard care for CD during this period, including corticosteroids, immunosuppressive agents and biological therapy that were subsequently withdrawn to protocol if appropriate.…”

“…This comprised (i) disease activity, haematology, biochemistry, adverse events according to EBMT guidelines, 15 concomitant medication every 6 weeks (ii) quality of life (IBD-Q and EQ-5D) every 6 months and (iii) endoscopic and radiological assessment of the entire intestine at baseline and one year. Baseline data was recorded prior to mobilisation in the patients undergoing immediate HSCT and prior to conditioning in those undergoing HSCT at one year.…”

Hematopoietic Stem Cell Therapy for Multiple Sclerosis: Top 10 Lessons Learned

“…This finding was translated into the clinical realm in the mid 1990s. Over the ensuing two decades the European Bone Marrow Transplant (EBMT) Registry [8] has documented that 469 patients have undergone HSCT for MS, whereas another 143 patients have been reported to the Consortium for International Bone Marrow Transplant Research (CIBMTR) [9].…”