scholarly journals Everolimus in Combination with Cyclosporin A as Pre- and Posttransplantation Immunosuppressive Therapy in Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation

2012 ◽  
Vol 18 (7) ◽  
pp. 1061-1068 ◽  
Author(s):  
Christian Junghanss ◽  
Susanne Rathsack ◽  
Rainer Wacke ◽  
Volker Weirich ◽  
Heike Vogel ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cyclosporin A ◽  
Immunosuppressive Therapy ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Hematopoietic Stem

Autologous Non-Myeloablative Hematopoietic Stem Cell Transplantation for Refractory Systemic Vasculitis.

Blood ◽  
2006 ◽  
Vol 108 (11) ◽  
pp. 5457-5457 ◽  
Author(s):  
Laisvyde Statkute ◽  
Yu Oyama ◽  
Walter G. Barr ◽  
Jolita Satkus ◽  
Yvonne Loh ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Immunosuppressive Therapy ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Systemic Vasculitis ◽  
Sustained Remission ◽  
Hematopoietic Stem ◽  
Autologous Hsct

Abstract Background Current mortality rate in systemic vasculitis (SV) patients with the use of cytotoxic / immunosuppressive therapy is over 20 % at 5 years in some studies. For patients refractory to conventional therapy new strategies aimed at aggressive induction of remission and relapse prevention are being sought. We here report our single center experience in treating 4 patients with refractory SV employing non-myeloablative autologous hematopoietic stem cell transplantation (HSCT). Patients and Methods Four patients with refractory SV (2 - with neurovascular Behcet’s disease, 1 - with neurovascular Sjogren’s syndrome, and 1 - with Wegener’s granulomatosis) were involved in an IRB and FDA approved phase I clinical trial of high dose chemotherapy and autologous HSCT. Peripheral blood stem cells were mobilized with cyclophosphamide (Cy) 2g/m2 IV and granulocyte-colony stimulating factor 10 mcg/kg/day SQ and were CD34+ enriched. Conditioning regimen consisted of Cy 200 mg/kg and rabbit anti-thymocyte globulin 5.5 mg/kg IV. Disease activity and cumulative organ damage were assessed using Birmingham Vasculitis Activity Score (BVAS) and Vasculitis Damage Index (VDI), respectively. Primary and secondary endpoints were transplant-related toxicity, survival and change in BVAS and VDI after the transplant. Results All 4 patients tolerated HSCT well without transplant related mortality or any significant toxicity. At median follow up of 28 (range 22–35) months all patients are alive. Three patients (1 each with Behcet’s, Sjogren’s, and Wegener’s) entered a sustained remission at 6, 6 and 24 months after the transplant. Both, BVAS and VDI, markedly decreased within 6 months post-transplant. In a patient with WG C-ANCA levels became undetectable at 24 month evaluation corresponding to clinical and radiological improvement. All 3 patients who achieved remission discontinued immunosuppressive therapy at the time of transplant and have not required since. Two patients with pre-transplant steroid dependency discontinued prednisone at 6 and 14 months after HSCT. One patient with BD who is positive HLA-B51, now 22 months since HSCT, has never achieved remission and is still requiring immunosuppressive therapy. Conclusion We suggest autologous HSCT utilizing a non-myeloablative regimen is a safe and effective treatment for select patients with SV refractory to conventional immunosuppressive therapies. However, in patients with significant genetic predisposition, more aggressive approach such as allogeneic HSCT or cord blood transplantation might be considered.

scholarly journals Inefficacy of Immunosuppressive Therapy for Severe Aplastic Anemia Progressing From Non-SAA: Improved Outcome After Allogeneic Hematopoietic Stem Cell Transplantation

2021 ◽  
Vol 11 ◽  
Author(s):  
Limin Liu ◽  
Xin Zhao ◽  
Miao Miao ◽  
Yanming Zhang ◽  
Wenjing Jiao ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Aplastic Anemia ◽  
Immunosuppressive Therapy ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Hematopoietic Stem ◽  
Group A ◽  
Group B

Background and AimsThis study aimed at comparing the efficacy and safety of severe aplastic anemia (SAA) cases that had met the criteria for SAA at the time of diagnosis (group A) with SAA that had progressed from non-SAA (NSAA) (group B), both undergoing first-line immunosuppressive therapy (IST). Additionally, group B was compared with SAA that had progressed from NSAA and who had been treated by allogeneic hematopoietic stem cell transplantation (allo-HSCT) (group C).MethodsWe retrospectively compared 608 consecutive patients in group A (n = 232), group B (n = 229) and group C (n = 147) between June 2002 and December 2019. Six months after treatment, the rate of overall response and the fraction of patients who had achieved normal blood values, treatment-related mortality (TRM), secondary clonal disease, 5-year overall survival (OS) and failure-free survival (FFS) were indirectly compared between group A and group B, group B and group C.ResultsSix months after treatment, the rate of overall response and the fraction of patients who had achieved normal blood values in group A was higher than in group B (65.24% vs. 40.54%, P < 0.0001; 23.33% vs. 2.25%, P < 0.0001); the same was true for group C (92.50% vs. 2.25%, P < 0.0001). The rate of relapse in group B was higher than in group C (P < 0.0001), but there were no differences in TRM and secondary clonal disease (P > 0.05). There were no differences in estimated 5-year OS between groups A and B (83.8% ± 2.6% vs. 85.8% ± 2.6%, P = 0.837), or between B and C (85.8% ± 2.6% vs. 77.9% ± 3.4%, P = 0.051). The estimated 5-year FFS in groups A and C was higher than for group B (57.1% ± 3.3% vs. 39.7% ± 3.4%, P < 0.001; 76.7% ± 3.5% vs. 39.7% ± 3.4%, P < 0.0001).ConclusionThese results indicate that IST is less effective in SAA progressing from non-SAA but allo-HSCT can improve outcomes.

scholarly journals THU0312 AUTOLOGOUS NON-MYELOABLATIVE HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR REFRACTORY TAKAYASU ARTERITIS: A RETROSPECTIVE MULTICENTRE CASE-SERIES FROM THE AUTOIMMUNE DISEASES WORKING PARTY (ADWP) OF THE EUROPEAN SOCIETY FOR BLOOD AND MARROW TRANSPLANTATION (EBMT)

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 385.2-385
Author(s):  
C. Laurent ◽  
Z. Marjanovic ◽  
J. Henes ◽  
D. Farge ◽  
M. Badoglio ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Autoimmune Diseases ◽  
Immunosuppressive Therapy ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Takayasu Arteritis ◽  
Hematopoietic Stem

Background:Takayasu arteritis (TAK) is a chronic granulomatous large-vessel vasculitis, characterized by arterial thickening and fibrosis leading to stenosis and vascular occlusions. More than 10-20% of patients are refractory to conventional immunosuppressive therapy. Autologous hematopoietic stem cell transplantation (AHSCT) has emerged as a promising treatment option in severely affected and refractory patients with various autoimmune diseases and vasculitis, particularly ANCA-positive vasculitis and Behçet’s disease.Objectives:This study,approved by the ADWP, aims to evaluate the use and outcome of AHSCT in adult TAK patients.Methods:This is a retrospective survey of patients reported to the EBMT registry between 1998 and 2019, who received AHSCT primarily for TAK. Clinical and laboratory data, including data on diagnosis, previous lines of therapy, transplant regimen, treatment-related mortality, as well as data regarding course of disease and treatment were recorded.Results:Data from six adult patients treated with AHSCT between 2003 and 2019 for refractory Takayasu have been identified. Median (ranges) follow-up was 9.9 (1-14) years. Five patients were female (83%), median age was 25 (9-39) years at diagnosis and 28 (22-41) years at HSCT. All patients were pretreated with a median of 6 (4-8) lines of therapy, including systemic steroids (6 patients), methotrexate (5 patients), cyclophosphamide, mycophenolate mofetil or infliximab (4 patients), tocilizumab or etanercept (2 patients), and other biologic or conventional-synthetic DMARDs. Conditioning included cyclophosphamide and rabbit anti-thymocyte globulin in all patients. At six months post-transplantation, remission was obtained in all cases, which persisted at 12 months in 5 cases. Four patients reactivated TAK at a median time of 27 (7-52) months after AHSCT, and 3 resumed disease-modifying therapy. At last follow-up, all patients were alive, 2 patients were in remission (off-therapy), 2 patients improved compared to baseline, and 2 patients were in complete and partial remission, respectively, under immunosuppressive treatment.Conclusion:This small retrospective series demonstrates that AHSCT has the potential to provide significant clinical responses in TAK patients who had been unresponsive to previous immunosuppressive therapy, with an acceptable safety profile.Acknowledgments:noDisclosure of Interests:CHARLOTTE LAURENT: None declared, ZORA MARJANOVIC: None declared, Jörg Henes Grant/research support from: Novartis, Roche-Chugai, Consultant of: Novartis, Roche, Celgene, Pfizer, Abbvie, Sanofi, Boehringer-Ingelheim,, DOMNIQUE FARGE: None declared, MANUELA BADOGLIO: None declared, John SNOWDEN: None declared, olivier fain: None declared, Tobias Alexander: None declared, Maria Carolina Oliveira: None declared, Arsene Mekinian: None declared

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