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These are a group of illnesses that all have an altered reactivity of the immune system where it reacts against its own organism. The clinical course is usually chronic and varies depending on the organs involved and the clinical evolution for each individual patient.
Immunosuppression (suppressing the immune system) is the conventional therapy that the majority of patients with a relatively benign state respond well to, whereas more aggressive forms of the disease are poorly controlled by subsequent lines of therapy.
It should be remembered that organ damage caused by the disease maybe associated with the side effects of long-term immunosuppression.
Stem Cell Transplantation for Autoimmune Diseases
The data reported in the literature show that the transplant is able to provide a clinical response in the majority of patients suffering from autoimmune diseases (AD’s) resistant to conventional treatments; the duration of the response is variable, above all depending on the diagnosis. (see reference 1)
The transplant is confirmed to be however, a useful therapeutic option in patients with poor prognosis. The role of the transplant must be established by prospective trials that are currently being carried out or are in the planning stages. The European Society of Blood & Marrow Transplantation has recently issued guidelines for the use of transplantation in autoimmune diseases (see reference 2).
Post-transplantation, the majority of patients achieve disease stabilization and in most of the diseases are able to suspend immunosuppressive treatments.
The likelihood of a recurrence depends on the disease and the transplantation technique used. In principle, success is more likely in the early stages of the disease and in patients of a younger age. In the more advanced stages, there is a reduction of the effectiveness and increased toxicity related to the procedure.
In a retrospective analysis by the European Society for Blood & Marrow Transplantation performed on 900 patients, transplant-related mortality was mainly linked to the diagnosis (certain diseases lead to damage on the organism that increases procedure-related toxicity) and to the centre’s experience, very probably due to the équipe having greater knowledge and to a better selection of eligible patients (see reference 1). It follows that this selection must be carried out by trained personnel and in accordance with the guidelines of the international scientific societies and the laws in force, in order to ensure that every patient receives a therapeutic proposal in which they are properly assessed for the risks and benefits.
Equally important is the correct information given to the patient, who must have full access to information on the treatment so that he/she is able to sign our consent form approved by our local Ethics Committee in full awareness.
The transplantation technique
1) Mobilization phase: the stem cells from the bone marrow in the blood are mobilized by a combination of chemotherapy and hormone treatment that regulates the production of the blood cells. At the end of this phase, the stem cells are collected through a procedure of extracorporeal circulation, subject to strict biological controls and then frozen at -196°C.
2) Conditioning therapy: chemotherapy drugs are administered again to reset the immune system. One side effect of the conditioning therapy is a transient toxicity on the bone marrow, resulting in a drop of blood cells (White Blood Cells, Red Cells and Platelets), see below.
The patient can be discharged when the blood cell values have returned to normal.
The Transplant Program for Autoimmune Diseases at Careggi University Hospital
Over the following 16 years, our Center has achieved considerable visibility for both the volume of activity (according to EBMT data, updated in January 2013, Careggi University Hospital is the number one European center for the number of transplants performed), as well as the number of publications in scientific journals (see references 3, 7).
The activity of the Centre is well integrated into the European and American scientific societies for bone marrow transplantations, collaborating and coordinating initiatives on an international level (see references 2, 8, 9).
Screening Process for HSC Transplant
The team may subsequently request additional test results for a better assessment as to the feasibility of the transplant. The patient will also be informed as to the economic aspects of the transplant and payment schedule.
Patients who are at this stage considered to be potential candidates will be invited to make an appointment at the Transplant Centre (Medical Assessment Team Consultation – see below).
Medical Assessment Team Consultation
2. Snowden JA, Saccardi R, Allez M, et al. Haematopoietic SCT in severe autoimmune diseases: updated guidelines of the European Group for Blood and Marrow Transplantation. Bone Marrow Transplant 2012;47:770-90.
3. Pfender N, Saccardi R, Martin R. Autologous hematopoietic stem cell transplantation as a treatment option for aggressive multiple sclerosis. Curr Treat Options Neurol 2013;15:270-80.
4. Saccardi R, Di Gioia M, Bosi A. Haematopoietic stem cell transplantation for autoimmune disorders. Curr Opin Hematol 2008;15:594-600.
5. Saccardi R, Kozak T, Bocelli-Tyndall C, et al. Autologous stem cell transplantation for progressive multiple sclerosis: update of the European Group for Blood and Marrow Transplantation autoimmune diseases working party database. Mult Scler 2006;12:814-23.
6. Mancardi G, Saccardi R. Autologous haematopoietic stem-cell transplantation in multiple sclerosis. Lancet Neurol 2008;7:626-36.
7. Saccardi R, Mancardi GL, Solari A, et al. Autologous HSCT for severe progressive multiple sclerosis in a multicenter trial: impact on disease activity and quality of life. Blood 2005;105:2601-7.
8. Saccardi R, Freedman MS, Sormani MP, et al. A prospective, randomized, controlled trial of autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: a position paper. Mult Scler 2012;18:825-34.
9. Pasquini MC, Griffith LM, Arnold DL, et al. Hematopoietic stem cell transplantation for multiple sclerosis: collaboration of the CIBMTR and EBMT to facilitate international clinical studies. Biol Blood Marrow Transplant 2010;16:1076-83.