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Autologous haematopoietic stem cell transplantation with reduced-intensity conditioning for aggressive multiple sclerosis: the Polish experience
L. Szczechowski1, M. Smilowski1, G. Helbig2, M. Krawczyk-Kulis2, S. Kyrcz-Krzemien2
1Department of Hematology and Bone Marrow Transplantation, A.Mielecki Hospital of Medical University of Silesia, 2Department of Hematology and Bone Marrow Transplantation, School of Medicine in Katowice, Medical University of Silesia, Katowice, Poland
Background: Multiple sclerosis (MS) is an autoimmune disease of the central nervous system (CNS) that leads to an inflammatory processes resulting in demyelination and axonal degeneration. Current treatment of relapsing-remitting MS (RRMS) includes immunomodulatory and immunosuppressive agents, which are effective, but usually in earlier and more benign forms. The immunomodulatory treatment has limited efficacy in aggressive forms of RRMS, and relapses occur despite treatment continuation. Autologous haematopoietic stem cell transplantation (AHSCT) should be considered as a therapeutic approach for patients with aggressive MS who failed conventional therapy.
Objectives: The aim of this study is to confirm the efficacy and safety of reduced-intensity immunosuppression followed by AHSCT among MS patients with aggressive forms of the disease.
Methods: 39 RRMS and secondary progressive MS (SPMS) patients met the qualification criteria for autologous hematopoietic stem cell transplantation. The qualification criteria were established according to the guidelines of the European Group for Blood and Marrow Transplantation (EBMT) 2012, current literature as well as our own experience. The group consisted of 29 patients with RRMS and 10 patients with SPMS. The stem cells were mobilized with cyclophsosphamide (Cy) and granulocyte-colony stimulating factor (G-CSF). After conditioning the stem cells were re-infused into the patient. The primary endpoint was the observation of relapse free survival (RFS) defined as relapse absence in the follow-up; progression free survival (PFS) - no progression in EDSS score and MRI-event free survival (MRI-EFS) - lack of new T2 changes. Those three parameters define the overall disease free survival (DFS) - lack of any signs of disease.
Results: A 4 years RFS was 86%. MRI-EFS was 78% and PFS was 82%. The DFS was observed in 72% of patients. No mortality was observed. Short term side effects were limited to symptoms which are common due to AHSCT procedures. No long term side effects have been observed so far.
Conclusions: AHSCT should be considered as an alternative treatment option for aggressive MS if performed according to very restrictive qualification criteria in highly specialized hematological centers.
L. Szczechowski1, M. Smilowski1, G. Helbig2, M. Krawczyk-Kulis2, S. Kyrcz-Krzemien2
1Department of Hematology and Bone Marrow Transplantation, A.Mielecki Hospital of Medical University of Silesia, 2Department of Hematology and Bone Marrow Transplantation, School of Medicine in Katowice, Medical University of Silesia, Katowice, Poland
Background: Multiple sclerosis (MS) is an autoimmune disease of the central nervous system (CNS) that leads to an inflammatory processes resulting in demyelination and axonal degeneration. Current treatment of relapsing-remitting MS (RRMS) includes immunomodulatory and immunosuppressive agents, which are effective, but usually in earlier and more benign forms. The immunomodulatory treatment has limited efficacy in aggressive forms of RRMS, and relapses occur despite treatment continuation. Autologous haematopoietic stem cell transplantation (AHSCT) should be considered as a therapeutic approach for patients with aggressive MS who failed conventional therapy.
Objectives: The aim of this study is to confirm the efficacy and safety of reduced-intensity immunosuppression followed by AHSCT among MS patients with aggressive forms of the disease.
Methods: 39 RRMS and secondary progressive MS (SPMS) patients met the qualification criteria for autologous hematopoietic stem cell transplantation. The qualification criteria were established according to the guidelines of the European Group for Blood and Marrow Transplantation (EBMT) 2012, current literature as well as our own experience. The group consisted of 29 patients with RRMS and 10 patients with SPMS. The stem cells were mobilized with cyclophsosphamide (Cy) and granulocyte-colony stimulating factor (G-CSF). After conditioning the stem cells were re-infused into the patient. The primary endpoint was the observation of relapse free survival (RFS) defined as relapse absence in the follow-up; progression free survival (PFS) - no progression in EDSS score and MRI-event free survival (MRI-EFS) - lack of new T2 changes. Those three parameters define the overall disease free survival (DFS) - lack of any signs of disease.
Results: A 4 years RFS was 86%. MRI-EFS was 78% and PFS was 82%. The DFS was observed in 72% of patients. No mortality was observed. Short term side effects were limited to symptoms which are common due to AHSCT procedures. No long term side effects have been observed so far.
Conclusions: AHSCT should be considered as an alternative treatment option for aggressive MS if performed according to very restrictive qualification criteria in highly specialized hematological centers.