Immunoablative Therapy with Autologous Stem Cell Transplantation in the Treatment of Poor Risk Multiple Sclerosis.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 5519-5519
Author(s):  
Tomas Kozak ◽  
Eva Havrdova ◽  
Jiri Pitha ◽  
Karolina Mayerova ◽  
Gregora Evzen ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Peripheral Blood ◽  
Repeated Measure ◽  
High Dose ◽  
Hematopoietic Stem

Abstract The immunoablative therapy with hematopoietic stem cell transplantation is tested in patients with intractable form of autoimmune diseases including multiple sclerosis (MS). Thirty one patients with secondary rapidly progressive MS were included in the phase I/II clinical trial involving the high dose chemotherapy with autologous peripheral blood progenitor cell (PBPC) support. Twenty seven patients underwent high dose conditioning BEAM. T cell depletion in vitro was performed in 18 grafts. Nine patients with not purged graft received in vivo ATG 4mg/kg i.v. D+1, D+2 after transplantation. Median follow-up is 48 months (24–72). Median EDSS (Expanded Disability Status Scale) of grafted patients at the time of inclusion was 6.5 (5.0–7.5), median EDSS of grafted patients at the last follow up was 7,0 (5.5–10.0). Two patients out of 27 (7 %) remain improved significantly (by ≥ 1.0 point on EDSS), 3 patients (11 %) are improved not significantly (by 0.5 point). Five patients (19 %) are stable in their EDSS. Seven patients (26 %) gained their disability significantly (by ≥ 1.0 point on EDSS) despite the treatment, one of them died 31 months after the transplantation from disease progression (EDSS 10.0). Ten patients (37 %) worsened not significantly (by 0.5 point) on their EDSS. Patients who clearly stabilized their disability or remain improved represent 37 %. The increase of EDSS at the 48 months (m) of the follow up is significatnt (Wicoxon’s, repeated measure ANOVA, t-test), however, is not at 36, 60 and 72 m respectively. The development of disability between the group that was grafted with in vitro purged graft and the group with ATG i.v. was not significant (Wilcoxon’s, Mann-Whitney). 20 patients stabilized their MRI finding, in 2 patients decreased number and size of lesions, 5 patients worsened their MRI. No mortality has been observed in this cohort. However, the toxicity of the transplantation differed in each individual; two serious events involving respiratory tract have been observed. Changes in lymphocyte subsets in peripheral blood were followed before and after the treatment. The percentage and absolute count of CD4+ cells and CD4+CD45 as well as IRI were significantly decreased 12 months after the transplantation Conclusion: Almost 40 % of patients with otherwise intractable rapidly progressive MS remained stable or improved in their disability with median follow up 48 months after immunoablative therapy. The value of this type of therapy should be investigated in the randomized trial. The study was sponsored by Grant IGA No NF/6560-3 from Ministry of Health of the Czech Republic.

Autologous Hematopoietic Stem Cell Transplantation in Severe Multiple Sclerosis. The Brazilian Experience with Two Conditioning Regimes: BEAM and High-Dose Cyclophosphamide.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 2881-2881
Author(s):  
Julio C Voltarelli ◽  
Marina A. Coutinho ◽  
Ana Beatriz P.L. Stracieri ◽  
Maria Carolina B. Oliveira ◽  
Daniela A. Moraes ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
High Dose ◽  
Hematopoietic Stem ◽  
Sf 36

Abstract Introduction: Multiple sclerosis (MS) is a chronic, inflammatory and demyelinating disease of the central nervous system (CNS) leading to gradual axonal degeneration. It is a classical auto-immune disease, thus high-dose chemotherapy followed by autologous hematopoietic stem cell transplantation (AHSCT) could “reset” the immune disorder by controlling autoreactive clones and by restoring self-tolerance after immunological recovery. Objectives: Describe the experience of the Brazilian Cooperative Group of AHSCT for Autoimmune Diseases in 41 MS patients followed-up to five years. Methods: Stem cells were mobilized from the bone marrow with Cy (2g/m2) and Filgrastim (10μg/kg/d SC), collected by leukapheresis and cryopreserved. Patients were conditioned with BEAM (BCNU 300 mg/m2, cytarabine 200 mg/m2, etoposide 200 mg/m2 ×4, melphalan 140 mg/m2) and horse antilymphocyte globulin (ATG) 90 mg/kg) or cyclophosphamide (CY) 50 mg/kg ×4 and rabbit ATG (rATG) 4.5 mg/kg, followed by stem cell infusion. Quality of life was assessed by the Short Form-36 Health Survey Questionnaire (SF-36). Results: Between January 2001 and June 2006, 21 patients received BEAM/ATG and 20 received CY/ATG. Medium age was 42 years (27–53 years), 24 (58.5%) were females, 33 (80.4%) had secondary progressive MS form, 80.95% had EDSS (Expanded Disability Status Scale) 6.0 or higher. No significant difference was seen between the groups regarding age, sex, disease presentation and EDSS. The mean number of CD34+ infused cells was 8.8×106/kg (2,5– 25.13×106/kg). Mean engraftment was 9,3 days (7–10 days) for neutrophils and 13 days (7–24 days) for platelets. 18 patients (46%) had neutropenic fever, 8 (20.0%) had pneumonia and 5 (12.5%) had allergic reactions to lymphoglobulin. Three patients (14.3%) died in the BEAM group due to conditioning regiment toxicity, sepsis and alveolar hemorrhage. Mean hospital stay was 35.47 days (20–168 days) in the BEAM group and 20.15 days (14–32 days) in the CY group (p<0,0001). EDSS-based disease progress in six months to a year was similar in both groups (p = 0.54). Disease worsening was detected in 6/18 patients in the BEAM group and 4/14 in the Cy group. Median follow-up was 17 months. Disease-free survival 4 years post-transplant was 65%. 31/41 patients completed SF-36 after transplantation and they exhibited improvement in their general health status at 100 days post-transplant (p = 0.02). Conclusion: In our study, a less intensive conditioning regimen (CY + rATG) was associated with similar neurological outcomes but also with less toxicity when compared to BEAM regimen. The majority of patients had an improvement in their quality of live. Longer follow-up is required to assess the therapeutic effectiveness of both regimens in MS progression.

Long Term Outcomes of Autologous Hematopoietic Stem Cell Transplantation in Progressive Multiple Sclerosis

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 4438-4438
Author(s):  
Juan Xu ◽  
Tong Wu ◽  
Li Su ◽  
Bing Xin Ji ◽  
Wu Han Hui
Keyword(s):  
Multiple Sclerosis ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Conditioning Regimen ◽  
High Dose ◽  
Hematopoietic Stem ◽  
Interval Time

Abstract Background Progressive multiple sclerosis (MS) is going with continuously disability and unresponsive to high dose steroid and immunomodulation. The autologous hematopoietic stem cell transplantation (ASCT) has been introduced in treatment of the forms of multiple sclerosis. Due to hematopoitic stem cell transplantation involved two processes that are conditioning with high dose immunosuppressive agents and stem cell transfusion. The short term outcomes (within 2 years after transplantation) do not preclude the immunosuppressant roles of conditionings, therefore the long term clinical outcomes after ASCT were evaluated for patients with progressive MS. Methods From Nov. 2001 to Jun. 2008, 34 patients with secondary progressive MS were treated with ASCT in our hospital. Of which, 26 patients were followed up more than 2 years till now. The median follow-up time was 40 months (3–83). There were 25 females (73.5%) and 9 males (26.5%). The median age of the patients was 36(20–51) years. Medium duration of disease was 36 months (15–156), and medium attacking interval time was 6.5 months (4–12). Peripheral blood stem cells were obtained by leukapheresis after mobilization with granulocyte colony-stimulating factor. BEAM, Tiniposide(600 mg/m2), melphalan(140mg/ m2), carmustin (300 mg/m2)and cytosine arabinoside (800 mg/m2), were administered as conditioning regimen. Outcomes were evaluated by the expanded disability status scale (EDSS). No maintenance treatment was administered if no disease progression. Results No deaths occurred following the treatment. All patients were observed into two groups, active-free group and activity group. The former include neurological improvement and neurological stabilization after transplantation. The latter include activity with progression and relapse without progression after improvement. Among 34 patients, 27 patients were in active-free group. Of twenty-one patients were with continuous neurological improvement without any active events. Median EDSS scores decreased from 6.0 (4.5–7.5) at transplantation to 2.0 (1.0–5) at last follow-up(p=0.000). Six patients remained neurological stable compared between the time of transplantation and last follow-up. There were 7 patients were in activity group. Of which, five patients had experience of neurological relapse during the follow-up period. However, the EDSS at relapse was lower than pre-transplantation, as well as the interval time between active events was longer than pre-transplantation. The low doses steroid relieved the symptoms in clinical. It seems to back to relapse and remission phase. There are two patients experienced neurological deterioration within 7 months after transplantation and need further immnosuppression treatment. The confirmed active-free survival rate was 79.14% and progression-free survival rate was 94.12% at 83 months according to Kaplan and Meier survival curve. Median remission-lasting time reached 63 months (95%CI 52–74). It was a significant difference compared with 7 months (95%CI 6–7) of pre-transplantation (P=0.000). We compared disease activity with attacking interval time, disease duration, patient’s age and EDSS of pre-transplantation. There is a relationship between active-free event and attacking interval time, OR=5.454, P=0.01(95% CI: 1.499 to 19.844,) and without relationship with duration of disease (OR=1.009, p=0.758), patient’s age (OR=1.136, P=0.147 and EDSS (OR=1.178, p=0.864) before transplantation. Conclusions ASCT with conditioning regimen of BEAM were able to improve or stabilize of neurological manifestations in most of progressive MS patients with failure of conventional therapy in long-term. The disease activitivy of post transplantation has a relationship with attacking interval time of pre-transplantation.

Alternative Donor Hematopoietic Stem Cell Transplantation for Children with Severe Aplastic Anemia.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 4348-4348
Author(s):  
Meerim Park ◽  
Kyung Nam Koh ◽  
Keun Wook Bae ◽  
Mee Jeong Lee ◽  
Ho Joon Im ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Aplastic Anemia ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Peripheral Blood ◽  
Hematopoietic Stem ◽  
Matched Sibling

Abstract Abstract 4348 Background Hematopoietic stem cell transplantation (HSCT) from matched sibling donor is the standard first-line treatment for children with severe aplastic anemia (SAA). However, the management of SAA lacking a suitable donor remains a great challenge. For those children, HSCT using unrelated donor or mismatched related donor could be a therapeutic alternative. The purpose of this study is to evaluate the outcome in children with SAA who received HSCT from donors other than matched sibling. Patients and Method Between March 2003 and July 2009, 17 patients received HSCT from alternative donors (AD) at Asan Medical Center. We reviewed their medical records and analyzed their transplant-related parameters and outcome. Results Of a total of 17 patients, 11 were male and the median age at HSCT was 9.0 years, ranging from 3.0 to 16.7 years. Four patients had Fanconi anemia and 13 had acquired SAA including 2 who developed SAA after liver transplantation. Donors included unrelated bone marrow (U-BM) in 5, unrelated peripheral blood (U-PB) in 6, unrelated cord blood (U-CB) in 2 and related haploidentical peripheral blood (H-PB) in 4. Of 17 patients, 15 (88%) achieved sustained engraftment. Of 15 with engraftment, only 1 patient who received HSCT from U-CB died of severe GI GVHD and the other 14 patients remain on stable normal counts without transfusion support. All 2 patients (1 U-BM, 1 H-PB) who failed to engraft were dead despite DLI or 2nd HSCT. With a median follow-up of 31.9 months, the Kaplan-Meier estimated overall survival at 2 years was 76.6%. Conclusion In children with SAA, HSCT from AD including haploidentical family donor could be considered as a treatment option if the patients have no matched sibling donor. Given the limitation of this study such as small number of patients and short follow-up period, further trial will be necessary. Disclosures: No relevant conflicts of interest to declare.

Survival, and time to an advanced disease state or progression, of untreated patients with moderately severe multiple sclerosis in a multicenter observational database: relevance for design of a clinical trial for high dose immunosuppressive therapy with autologous hematopoietic stem cell transplantation

2006 ◽  
Vol 12 (2) ◽  
pp. 174-179 ◽  
Author(s):  
M Daumer ◽  
L M Griffith ◽  
W Meister ◽  
R A Nash ◽  
J S Wolinsky
Keyword(s):  
Multiple Sclerosis ◽  
Clinical Trial ◽  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Immunosuppressive Therapy ◽  
Cell Transplantation ◽  
High Dose ◽  
Hematopoietic Stem ◽  
Edss Score

Despite prolonged survival, patients with multiple sclerosis (MS) experience considerable morbidity, which adversely impacts quality of life. To assess the risk-benefit of a clinical trial of high dose immunosuppressive therapy with autologous hematopoietic stem cell transplantation for MS, we sought to determine the natural history of the disease in a comparison group of untreated patients. We identified 285 individuals with 2132 combined observation years (median: 5.6 years; 5th to 95th percentile: 1-21 years), with Expanded Disability Status Scale (EDSS) scores of 3.0-5.5 at baseline observation. Disease-related mortality was zero at five years, 5.4% at 10 years, and 22% at 15 years (40 patients contributing to the data point; 95% confidence interval: 4-32%). Risk for progression to advanced disability, defined as an EDSS score of 8, was very low for the subgroup with a baseline EDSS score of 3-3.5; however, for those with a baseline EDSS score of 4-5.5, 3% had advanced disability after two years, 5% after three years, 6% after four years, 12% after five years, and 40% after 10 years. The estimated probability of disease progression, defined as an increase in EDSS score by ≥ 1.0 sustained for at least 180 days, was 5% after one year, 14% after two years, 22% after three years, 38% after five years, 57% after 10 years, and-80% after 20 years of observation. The relevance of these features to the design of the clinical trial is discussed.

Case Report: Haploidentical Stem Cell Transplantation In a 78 Year-Old Patient

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 5468-5468
Author(s):  
Thiago Xavier Carneiro ◽  
André Domingues Pereira ◽  
Theodora Karnakis ◽  
Celso Arrais Rodrigues
Keyword(s):  
Stem Cell ◽  
T Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Peripheral Blood ◽  
High Dose ◽  
Cell Transplant ◽  
Hematopoietic Stem ◽  
Post Transplant

Abstract An older chronologic age has been a consistent predictor of poor outcomes in hematopoietic stem cell transplantation (HSCT), mainly due to non-relapse mortality (NRM). Therefore, non-curative treatment strategies are commonly adopted for these patients. However, mortality and treatment toxicity has decreased as a result of improved supportive measures, such as reduced intensity conditioning regimens and optimized infection management. T-cell replete haploidentical HSCT emerged as a feasible alternative for leukemia patients without substantial differences in outcomes when compared to fully matched related donor. We report an old adult woman treated with haploidentical HSCT. A 78 year-old female patient presented with anemia, leukocytosis, thrombocytopenia and blasts in the peripheral blood. Diagnosis of acute myelogenous leukemia was established. Conventional cytogenetic demonstrated chromosome eight trisomy, and FISH was negative for other common MDS/AML cytogenetic abnormalities. FLT3-ITD and NPM1 mutations were negative. Her medical history was negative except for heavy smoking. Considering the patients advanced age, the first attending physician chose not to administer intensive treatment and started on decitabine 20 mg/m2 for 5 days. She was refractory to the first-line treatment with persistent cytopenias and blasts in the peripheral blood four weeks after treatment was started. Comprehensive geriatric assessment was performed. She was considered independent for Basic Activities of Daily Living (ADL score 6) and Instrumental Activities of Daily Living (IADL score 27), without cognitive impairment in mini-mental state examination (MMSE score 30), at risk of malnutrition in mini nutritional assessment (MNA 9). As she was considered fit, we decided to perform high-dose chemotherapy with idarubicin and cytarabine, but, once more, the disease was refractory. A rescue regimen was attempted with high-dose cytarabine and mitoxantrone, again, with no response. After discussing pros and cons with the patient and the family, we decided to start Another regimen consisting of topotecan and high dose cytarabine immediately followed by allogeneic hematopoietic stem cell transplantation (HSCT). At day 14, she had 3% blasts in the BM aspirate a T-cell replete haploidentical HSCT using her 52 year-old son as donor and mobilized peripheral blood as stem cell source was performed. Conditioning regimen consisted of fludarabine, cyclophosphamide, TBI 2Gy and post-transplant cyclophosphamide. Graft versus host disease (GVHD) prophylaxis consisted of mycophenolate mofetil and cyclosporine. She had neutrophil engraftment with complete donor chimerism at day+15 and platelet engraftment at day+17. At day+48, she had mild (stage II) skin acute GVHD resolved with topical steroids. Cyclosporine was withdrawn at day+ 93. Due to high relapse risk, the patient was started on monthly post-transplant azacitidine 36 mg/m2. At day+100 the patient remained in complete remission, complete donor chimerism in peripheral blood and bone marrow. Functionality was preserved (ADL score 6 and IADL score 24), presented discrete cognitive impairment (MMSE 28) and malnoutrition (MNA 5). She is now at day+182, doing well and performing again all usual daily activities. To the best of our knowledge, this is the oldest patient treated with haploidentical HSCT. Post transplant cyclophosphamide as T cell depletion strategy in haploidentical HSCT is well tolerated and widely available, being therefore an excellent alternative for patients without conventional donors who require immediate transplant. Older adults with hematologic malignancies are a heterogeneous group and decisions based on chronological age alone are clearly inappropriate. Recently, geriatric assessment proved to be an important prognostic tool in acute leukemia and may be useful in HSCT. In experienced centers, haploidentical HSCT in older adults may be a safe procedure and more accurate pre-transplantation risk stratification tools should be developed. Figure 1 Timeline of main events during hematopoietic stem cell transplant. Figure 1. Timeline of main events during hematopoietic stem cell transplant. Disclosures: No relevant conflicts of interest to declare.

Early highly aggressive MS successfully treated by hematopoietic stem cell transplantation

2008 ◽  
Vol 15 (2) ◽  
pp. 229-237 ◽  
Author(s):  
J Fagius ◽  
J Lundgren ◽  
G Öberg
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
High Dose ◽  
Hematopoietic Stem ◽  
Disability Status ◽  
Relapsing Remitting

Background During the last 15 years, high-dose chemotherapy with autologous hematopoietic stem cell transplantation (HSCT) has globally been performed for severe multiple sclerosis (MS). Most patients have been in progressive phase with long disease duration. As a rule, treatment effect has been minor or moderate. Patients Since 2004, we have performed HSCT in nine young patients with “malignant” relapsing–remitting MS. Criteria for treatment were short duration of disease; very frequent, severe relapses; recent improvement periods indicating potential for recovery after strong immunosuppression. Findings Median age at treatment was 27 (range 9–34) years, MS duration 26 (4–100) months, and annualized relapse rate 10 (4–12). Median Disability Status Scale (extended disability status scale, EDSS) at HSCT was 7.0 (3.5–8.0). Median follow-up time April 2008 is 29 (23–47) months. Median EDSS improvement is 3.5 (1.0–7.0), clearly surpassing most previous reports. One patient relapsed mildly with rapid recovery 7 months after HSCT. All patients are otherwise stable, median EDSS being 2.0 (0–6.0). Before HSCT, 61 relapses occurred in 82 patient months; during follow-up, one relapse in 289 patient months. Conclusion This small series of patients with “malignant” relapsing–remitting MS suggests HSCT to be an effective treatment option for this relatively rare disease course. It further suggests that future criteria for HSCT in MS should be close to the present ones.

scholarly journals Autologous hematopoietic stem cell transplantation in combination with immunoablative protocol in secondary progressive multiple sclerosis: A 10-year follow-up of the first transplanted patient

2016 ◽  
Vol 73 (5) ◽  
pp. 504-508
Author(s):  
Dragana Obradovic ◽  
Ljiljana Tukic ◽  
Sanja Radovinovic-Tasic ◽  
Boris Petrovic ◽  
Marija Elez ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hemopoietic Stem Cell ◽  
Disability Progression ◽  
Hematopoietic Stem ◽  
Secondary Progressive ◽  
Frequent Relapses

Introduction. Multiple sclerosis (MS) is an immunemediated disease of the central nervous system that affects young individuals and leads to severe disability. High dose immunoablation followed by autologous hemopoietic stem cell transplantation (AHSCT) has been considered in the last 15 years as potentialy effective therapeutic approach for agressive MS. The most recent long-time follow-up results suggest that AHSCT is not only effective for highly aggressive MS, but for relapsing-remitting MS as well, providing long-term remission, or maybe even cure. We presented a 10- year follow-up of the first MS patient being treated by immunoablation therapy and AHSCT. Case report. A 27-year-old male experienced the first symptoms - intermitent numbness and paresthesia of arms and legs of what was treated for two years by psychiatrist as anxiety disorder. After he developed severe paraparesis he was admitted to the Neurology Clinic and diagnosed with MS. Our patient developed aggressive MS with frequent relapses, rapid disability progression and transition to secondary progressive form 6 years after MS onset [the Expanded Disability Status Scale (EDSS) 7.0 Ambulation Index (AI) 7]. AHSCT was performed, cyclophosphamide was used for hemopoietic stem cell mobilization and the BEAM protocol was used as conditionig regimen. No major adverse events followed the AHSCT. Neurological impairment improved, EDSS 6.5, AI 6 and during a 10-year followup remained unchanged. Brain MRI follow-up showed the absence of gadolinium enhancing lesions and a mild progression of brain atrophy. Conclusion. The patient with rapidly evolving, aggressive, noninflammatory MS initialy improved and remained stable, without disability progression for 10 years, after AHSCT. This kind of treatment should be considered in aggressive MS, or in disease modifying treatment nonresponsive MS patients, since appropriately timed AHSCT treatment may not only prevent disability progression but reduce the achieved level of disability, as well.

High-dose immunoablation with autologous haematopoietic stem cell transplantation in aggressive multiple sclerosis: a single centre 10-year experience

2010 ◽  
Vol 16 (6) ◽  
pp. 685-693 ◽  
Author(s):  
Eva Krasulová ◽  
Marek Trněný ◽  
Tomáš Kozák ◽  
Blanka Vacková ◽  
David Pohlreich ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Haematopoietic Stem Cell Transplantation ◽  
Haematopoietic Stem Cell ◽  
High Dose ◽  
Adequate Treatment ◽  
Multiple Sclerosis Patients

There are multiple sclerosis patients who suffer from an aggressive course of the disease with severe relapses and rapid accumulation of disability despite adequate treatment. In such cases high-dose immunoablation with autologous haematopoietic stem cell transplantation (ASCT) may be considered. Our objective was to report our experience with 26 multiple sclerosis patients treated with ASCT within the years 1998—2008. Twenty-six patients (Expanded Disability Status Scale 2.5—7.5 (median 6.0), multiple sclerosis duration 2—19 years (median 7)) with aggressive multiple sclerosis underwent autologous haematopoietic stem cell transplantation. Stem cells were mobilized by high-dose cyclophosphamide and granulocyte colony-stimulating factor, BEAM (carmustine, etoposide, cytarabine, melphalan) was used for immunoablation. Patients were evaluated at baseline and every six months post ASCT for adverse events and clinical outcome. Follow-up period was 11—132 months (median 66). Progression-free survival was calculated using the Kaplan— Meier method. At 3 and 6 years of follow-up 70.8% and 29.2% of patients respectively were free of progression. Patients with relapsing multiple sclerosis course, disease duration <5 years and age <35 years had a more favourable outcome. There was no death within 100 days after ASCT. We conclude that ASCT represents a viable and effective treatment option for aggressive multiple sclerosis.

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