OBJECTIVES:To provide real-world data on the impact of autologous hematopoietic stem cell transplantation (AHSCT) on treatment costs of patients with multiple sclerosis (MS) in Poland.METHODS:Medical data of 105 patients who underwent AHSCT in the years 2011 to 2016 were obtained from the National Health Fund (NHF) database. Treatment costs were calculated from the public payer's perspective per patient-year for the total available period as well as 12 months before and after AHSCT. The statistical analysis was performed using MATLAB 2016b.RESULTS:Mean treatment-related costs covered by the NHF per patient-year before and after the transplantation were €4314.9 and €1188.8 , respectively. The average cost of disease-modifying drugs per patient was reduced from €2497.9/year before to €65.3/year after AHSCT.CONCLUSIONS:Although the initial cost of AHSCT is high, the costs involving AHSCT and post-AHSCT treatment could, according to our analysis, pay off in 3.9 years, when compared to the costs of disease-modifying drug therapy in aggressive MS. The study provides evidence that the AHSCT can lead to significant savings in treatment costs of aggressive MS from the public payer's perspective.

Autologous hematopoietic stem cell transplantation (AHSCT) is thought to be effective therapeutic approach in patients with poor prognosis systemic sclerosis; however, the toxicity remains a challenge. Between years 2003 and 2016, we enrolled 18 patients with systemic sclerosis at median age at transplant of 52 years (range 24–68). The median duration of disease before AHSCT was 14 months (range 2–85). Peripheral blood stem cells were mobilized with cyclophosphamide (CY) and granulocyte colony-stimulating factor. Conditioning regimen included CY (200 mg/kg) and alemtuzumab (median dose, 60 mg) [ n = 11], melphalan (MEL; 140 mg/m2) and alemtuzumab [ n = 2], CY and rabbit anti-thymocyte globulin (rATG; 7.5 mg/kg) [ n = 4], and CY alone ( n = 1). Four deaths occurred early after transplant. There were three males and one female at median age at death of 51 years (range 24–68). The AHSCT-related deaths have been observed on days + 1, + 4, + 9, and + 15 after procedure. The causes of death included bilateral pneumonia followed by multi-organ failure in three patients and myocardial infarction in one. Three patients expired late during post-transplant follow-up, after 5, 21, and 42 months. The causes of death were disease progression in two patients and sudden heart attack in one. Eleven patients are alive after median follow-up after AHSCT of 42.0 months (range 0–95). Before proceeding to AHSCT in systemic sclerosis, there is a strong need to optimize patient selection to reduce toxicity. The administration of alemtuzumab should be avoided due to high risk of life-threatening infectious complications.

Autologous hematopoietic stem cell transplantation (AHSCT) is a treatment option for relapsed and recurrent follicular lymphoma (R/R FL); however, its value in the rituximab era remains to be elucidated. To evaluate the safety and clinical outcome of AHSCT for relapsed FL, we present a retrospective series of AHSCT for 30 FL patients (17 male and 13 female) at median age of 49 years. Patients were transplanted in second or subsequent complete or partial response after at least one therapeutic line including chemotherapy and rituximab. Overall, seven patients achieved second or higher complete response (CR) at AHSCT, whereas 23 were transplanted in partial response. Median overall survival (OS) was not reached, whereas progression-free survival (PFS) was 4.8 years. The estimated 10-year OS and PFS were found to be 60 and 33%, respectively. There was no significant difference in OS and PFS in terms of FLIPI score and disease status at transplant. Median follow-ups from diagnosis and from AHSCT were 4.9 years (range 1.5-18.4 years) and 1.7 years (range 0.03-16.5 years), respectively. Fifteen patients relapsed, and 11 out of them (73%) died of disease recurrence and chemoresistance. At the last contact, 19 patients are alive: 12 are in CR, whereas seven patients receive salvage regimens due to active lymphoma. AHSCT for relapsed FL patients who were pretreated with rituximab remains a safe procedure with low transplant-related mortality and long-term progression-free survival in about one-third of transplanted patients.

Abstract Abstract 4399 Autologous stem cell transplantation (ASCT) is a method of choice in many hematological malignancies enabling the patients (pts) to achieve remission or to prolong life. Commonly used mobilizing regimens include high dose chemotherapy followed by G-CSF administration. Unfortunately up to 35% pts fail to mobilize the successful number of stem cells (SC). 16 pts aged 48 (23–69) were mobilized with plerixafor in our clinic over one-year period. All of the them failed to mobilize to collect satisfactory number of SC after standard mobilizing regimen. The protocol included G-CSF (10μg/kg) administered subcutaneously for 4 days, followed by plerixafor administration (240μg/kg s.c.). Only two pts failed to collect satisfactory number of SC after plerixafor. 14/16 collected the median of 2.24×106/kg (0.8–15,8), all of them underwent the ASCT. The outcome of ASCT in this group was compared to the posttransplant period in pts mobilized with standard mobilizing regimen (control group; CG). The amount of CD34+cells x106/kg was significantly higher in the CG (2.24 vs. 4.4, p0.5G/l) −10 vs. 14 days, and to PLT engraftment (PLT>20G/l) – 11vs. 14 days, were not different significantly. 43% pts relapsed in plerixafor group with a median follow-up of 14 months and 37% pts in the CG with a median follow up of 11 months (p>.05).SC mobilization with plerixafor and G-CSF provides solution for majority of pts requiring ASCT and failing mobilization with G-CSF in combination with chemotherapy. However, due to high leukocytosis, this protocol may require modification of SC collection and freezing procedures in order to avoid large volumes. In general, pts mobilized with plerixafor had similar post transplant outcome as pts who were mobilized using standard regimen. Disclosures: No relevant conflicts of interest to declare.