A Feasibility Study of the Full Outpatient Conduction of Hematopoietic Transplants in Persons with Multiple Sclerosis Employing Autologous Non-Cryopreserved Peripheral Blood Stem Cells

Abstract

Background: With the goal of achieving immune system reset, autologous hematopoietic stem cell transplantations have been performed in patients with multiple sclerosis (MS). Material and Methods: Two hundred and eighty-six consecutive patients with MS were autografted in a single center using non-frozen peripheral blood stem cells (PBSCs), on an outpatient basis and conditioning with cyclophosphamide and rituximab. The protocol was registered in ClinicalTrials.gov identifier NCT02674217. Results: One hundred and ninety-four females and 92 males were included; the median age was 47. All procedures were started on an outpatient basis and only 8 persons needed to be admitted to the hospital during the procedure. In order to obtain at least 1 × 10⁶/kg viable CD34 cells, 1-4 aphereses were performed (median 1). The total number of viable CD34+ cells infused ranged between 1 and 19.2 × 10⁶/kg (median 4.6). Patients recovered above 0.5 × 10⁹/L absolute granulocytes on median day 8 (range 0-12). Two individuals needed red blood cells but none needed platelet transfusions. There were no transplant-related deaths and the 128-month overall survival of the patients is 100%. In 82 persons followed up for 3 or more months, the Expanded Disability Status Scale diminished from a mean of 5.2-4.9, the best results being obtained in relapsing-remitting and primary progressive MS. Conclusions: It is possible to conduct autotransplants for patients with MS employing non-frozen PBSCs and outpatient conduction. Additional information is needed to assess the efficacy of these procedures in the treatment of patients with MS.

Hematopoietic Cell Transplantation for Autoimmune Diseases: A Review of History, Current State, and Future Issues

“…Other procedures of different reduced toxicity of immunoablative but not always myeloablative conditioning were published as well: mini BEAM-like, BCNU/CCNU with intermediate dose of melphalan, ±ATG or alemtuzumab, rituximab, or Cy ± thiotepa or fludarabine, etc. Some could lead to the possibility of performing this procedure in outpatient manner [43][44][45].…”

Determine safety of outpatient chemotherapy and autotransplants using refrigerated, non‐frozen grafts in persons with multiple sclerosis

“…1,2 More than 1000 autotransplants are reported with MS 2-6 but use of autotransplants may be substantially more common as many cases are not reported. 1,2 More than 1000 autotransplants are reported with MS 2-6 but use of autotransplants may be substantially more common as many cases are not reported.…”

“…2,6 We wondered whether the same modifications could be used in persons with MS? We previously reported autotransplants for several hematologic neoplasms can be done in an outpatient setting using refrigerated, non-frozen blood cells.…”

“…Persons with multiple sclerosis (MS) are sometimes treated with intermediate-or high-dose chemotherapy and an autotransplant. 1,2 More than 1000 autotransplants are reported with MS 2-6 but use of autotransplants may be substantially more common as many cases are not reported. Recent data from a small randomized trial of less-intensive chemotherapy reported better outcomes with this approach compared with disease-modifying therapy in persons with advanced relapsing-remitting MS 7 As such, autotransplant frequency is likely to increase.…”

“…We previously reported autotransplants for several hematologic neoplasms can be done in an outpatient setting using refrigerated, non-frozen blood cells. 2,6 We wondered whether the same modifications could be used in persons with MS?…”