Alessia Leone1, Massimo Radin2, Ahlam M Almarzooqi3, Jamal Al-Saleh3, Dario Roccatello4, Savino Sciascia5, Munther Khamashta6. 1. Ashford and St Peter's Hospitals NHS Foundation Trust, Surrey, UK. 2. Center of Research of Immunopathology and Rare Diseases, Coordinating Center of Piemonte and Valle d'Aosta Network for Rare Diseases, Department of Clinical and Biological Sciences, S. Giovanni Bosco Hospital and University of Turin, Turin, Italy. 3. Department of Rheumatology, Dubai Hospital, Dubai, UAE. 4. Center of Research of Immunopathology and Rare Diseases, Coordinating Center of Piemonte and Valle d'Aosta Network for Rare Diseases, Department of Clinical and Biological Sciences, S. Giovanni Bosco Hospital and University of Turin, Turin, Italy; SCDU Nephrology and Dialysis, S. Giovanni Bosco Hospital, Turin, Italy. 5. Center of Research of Immunopathology and Rare Diseases, Coordinating Center of Piemonte and Valle d'Aosta Network for Rare Diseases, Department of Clinical and Biological Sciences, S. Giovanni Bosco Hospital and University of Turin, Turin, Italy; SCDU Nephrology and Dialysis, S. Giovanni Bosco Hospital, Turin, Italy. Electronic address: savino.sciascia@unito.it. 6. Department of Rheumatology, Dubai Hospital, Dubai, UAE; Lupus Research Unit, Guy's and St Thomas' Hospitals NHS Foundation Trust, London, UK; Lupus Research Unit, Division of Women's Health, King's College London, London, UK.
Abstract
BACKGROUND: Hematopoietic stem cell transplantation (HSCT) has been proposed as a therapeutic option for patients with Systemic Lupus Erythematosus (SLE) refractory to standard therapy. This therapeutic approach has been applied to other severe autoimmune diseases refractory to standard therapy with promising results. AIM: To systematically review the literature and analyze the available evidence on HSCT therapy in patients with SLE and antiphospholipid syndrome (APS), with a focus on therapy efficacy and occurrence of adverse events. METHODS: A detailed literature search, applied to Ovid MEDLINE, In-Process and Other Non-Indexed Citation and Ovid Medline 1986 to 2014, has been developed a priori to identify articles that reported findings from clinical and laboratory studies that investigated the effect of HCT in patients with SLE. RESULTS: Twenty-five studies met all inclusion criteria, including a total of 279 SLE patients; of those, 54 patients also fulfilled the classification criteria of APS. The majority of the studies reported an improvement after HSCT in terms of diseases activity control (assessed with SLEDAI, or time-free from diseases) or overall survival. However, one study reported no net benefit of HSCT when compared to immunosuppression alone. One retrospective study reported an overall survival at 5years of 81% in 28 SLE patients. Of note, 5 cases (9.3%) of aPL negativization were reported after HSCT in the APS patients. When combining these studies and analyzing these patients with APS, 32 out of 44 (73%) were able to discontinue anticoagulation after HSCT. Our findings also demonstrate a total of 86 infections in the pool of patients (30.8%), 3 of which resulted in the death of the patient (1.3%). We observed an annual incidence of infection of 11.9% with a mean follow up of 36.2months. CONCLUSION: Preliminary results of HSCT as a therapeutic option for SLE appear promising. Further studies are warranted in order to assess the safety of the procedure for both the occurrence of secondary autoimmune disease and the rate of infection. However, the rate of adverse effects confines this option to very selected cases of SLE patients resistant or refractory to standard approaches.
BACKGROUND: Hematopoietic stem cell transplantation (HSCT) has been proposed as a therapeutic option for patients with Systemic Lupus Erythematosus (SLE) refractory to standard therapy. This therapeutic approach has been applied to other severe autoimmune diseases refractory to standard therapy with promising results. AIM: To systematically review the literature and analyze the available evidence on HSCT therapy in patients with SLE and antiphospholipid syndrome (APS), with a focus on therapy efficacy and occurrence of adverse events. METHODS: A detailed literature search, applied to Ovid MEDLINE, In-Process and Other Non-Indexed Citation and Ovid Medline 1986 to 2014, has been developed a priori to identify articles that reported findings from clinical and laboratory studies that investigated the effect of HCT in patients with SLE. RESULTS: Twenty-five studies met all inclusion criteria, including a total of 279 SLEpatients; of those, 54 patients also fulfilled the classification criteria of APS. The majority of the studies reported an improvement after HSCT in terms of diseases activity control (assessed with SLEDAI, or time-free from diseases) or overall survival. However, one study reported no net benefit of HSCT when compared to immunosuppression alone. One retrospective study reported an overall survival at 5years of 81% in 28 SLEpatients. Of note, 5 cases (9.3%) of aPL negativization were reported after HSCT in the APSpatients. When combining these studies and analyzing these patients with APS, 32 out of 44 (73%) were able to discontinue anticoagulation after HSCT. Our findings also demonstrate a total of 86 infections in the pool of patients (30.8%), 3 of which resulted in the death of the patient (1.3%). We observed an annual incidence of infection of 11.9% with a mean follow up of 36.2months. CONCLUSION: Preliminary results of HSCT as a therapeutic option for SLE appear promising. Further studies are warranted in order to assess the safety of the procedure for both the occurrence of secondary autoimmune disease and the rate of infection. However, the rate of adverse effects confines this option to very selected cases of SLEpatients resistant or refractory to standard approaches.
Authors: Basil Sharrack; Riccardo Saccardi; Tobias Alexander; Manuela Badoglio; Joachim Burman; Dominique Farge; Raffaella Greco; Helen Jessop; Majid Kazmi; Kirill Kirgizov; Myriam Labopin; Gianluigi Mancardi; Roland Martin; John Moore; Paolo A Muraro; Montserrat Rovira; Maria Pia Sormani; John A Snowden Journal: Bone Marrow Transplant Date: 2019-09-26 Impact factor: 5.483
Authors: Ilan Volkov; Luciana Seguro; Elaine P Leon; László Kovács; Dirk Roggenbuck; Peter Schierack; Boris Gilburd; Andrea Doria; Maria G Tektonidou; Nancy Agmon-Levin Journal: Auto Immun Highlights Date: 2020-05-15