scholarly journals Long-Term Results of Non- Fludarabine Versus Fludarabine-Based Conditioning Regimens in Severe Aplastic Anemia Patients

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 5700-5700
Author(s):  
Elifcan Aladag ◽  
Haluk Demiroglu ◽  
Rafiye Ciftciler ◽  
Yahya Buyukasik ◽  
Nilgun Sayinalp ◽  
...  
Keyword(s):  
Aplastic Anemia ◽  
Cell Transplantation ◽  
Hematopoietic Cell Transplantation ◽  
Conflicts Of Interest ◽  
Therapeutic Modality ◽  
Long Term Results ◽  
Severe Aplastic Anemia ◽  
Hematopoietic Stem ◽  
Primary Graft Failure ◽  
Conditioning Regimens

Abstract BACKGROUND AND OBJECTIVE: Allogenic hematopoietic cell transplantation (AHSCT) is the best therapeutic modality capable of correcting the hematologic manifestations of severe aplastic anemia (SAA), here in this study we have compared the results of well-tolerated fludarabine-based conditioning regimens for SAA patients who are heavily transfused undergoing AHSCT. DESIGN AND SETTINGS: This is a retrospective study consist of 25 patients diagnosed with SAA who underwent AHSCT between February 2002 and March 2015 at the Bone Marrow Transplantation Unit of Hacettepe University. All patients gave written informed consent for the procedure. PATIENTS AND METHODS: We analyzed the outcome of 25 SAA patients who had undergone AHSCT between 2002 and 2015. the median age at transplantation was 27(16-55) years. Patients received transplants from an HLA-identical sibling (n=24) and mismatched allogeneic related HSCT (n=1). Hematopoietic Stem Cells (HSC) were collected from the donors via apheresis. Eight patients underwent standard CY 50 mg/kg plus ATG-FreseniusR 5-10 mg/kg as conditioning fort he transplantation procedure and 13 patients conditioned with CY(50 mg/kg for 2 days) plus fludarabine 25 mg/m2 for 5 days and ATG-FreseniusR 5-10 mg/kg as AHSCT conditioning and received GvHD (Graft-vs-Host Disease) prophylaxis was standard CsA 3 mg/kg IV inf. plus Methotrexate(MTX) 10mg/m2 on day 1,and MTX 5 mg/m2 on days 3.,6. and 11. RESULTS: The median follow-up period for all the survivors was 117 months [minimum 3 and maximum 159 months]. The 5-year overall survival (os) for patients who did or did not receive fludarabine-based preparative regimens for the allograft was 93%, and 87%, respectively. However, there were no statistically significant differences in os rates between these two groups (P=0,58). Median neutrophil engraftment time was 11 days and median platelet engraftment time was 13 days. Primary graft failure occurred in 1 patients (4%) who received fludarabine-based conditioning regimens. CONCLUSION: Fludarabine-Based (Flu-CY-ATG) conditioning is a safe and viable option when compared to the standard CY-ATG conditioning in heavily-transfused SAA patients who undergo Allogeneic hematopoietic stem cell transplantation (allo-HSCT). Disclosures No relevant conflicts of interest to declare.

scholarly journals Hematopoietic stem cell transplantation for infantile osteopetrosis

Blood ◽  
2015 ◽  
Vol 126 (2) ◽  
pp. 270-276 ◽  
Author(s):  
Paul J. Orchard ◽  
Anders L. Fasth ◽  
Jennifer Le Rademacher ◽  
Wensheng He ◽  
Jaap Jan Boelens ◽  
...  
Keyword(s):  
Stem Cell ◽  
Cell Transplantation ◽  
Graft Failure ◽  
Hematopoietic Cell Transplantation ◽  
Term Survival ◽  
Hematopoietic Stem ◽  
Key Points ◽  
Primary Graft Failure ◽  
Very High

Key Points Hematopoietic cell transplantation results in long-term survival. Primary graft failure is very high and the predominant cause of death.

Long-Term Clinical Outcome of Children with Acquired Aplastic Anemia in Brazil.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 4216-4216
Author(s):  
Marlene Pereira Garanito ◽  
Vicente Odone Filho ◽  
Marcela Vieira dos Santos ◽  
Elvira Velloso ◽  
Frederico L. Dulley ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Aplastic Anemia ◽  
Immunosuppressive Therapy ◽  
Cell Transplantation ◽  
Survival Probability ◽  
Conflicts Of Interest ◽  
Time History ◽  
Hematopoietic Stem ◽  
Significant Difference

Abstract Abstract 4216 Introdution/ Backgound Acquired Aplastic Anemia (AAA) is a rare hematologic disorder characterized by pancytopenia and hypocelular bone marrow. The pathophysiology is immune mediated in most cases. Environmental exposures to drugs, viruses and toxins, are thought to trigger the aberrant immune response in some patients. However, 50 to 74 percent of cases are classified as idiopathic. The highest frequency occurs in young population (15 to 25 years) with a second peak at age of 65 to 69 years. Immunosuppressive therapy is the best treatment in children with AAA who do not have a suitable donor for allogeneic stem cell transplantation. Materials and methods We reviewed the medical records of patients diagnosed with severe (SAA) and very severe acquired aplastic anemia (vSAA) at the Department of Pediatrics, Instituto da Criança – Hospital das Clínicas, University of Sào Paulo, Brazil from December, 1992 to December, 2007. We analyzed the clinical characteristics of the patients at diagnosis and the response to immunosuppressive therapy (IST) and hematopoietic stem cell transplantation (HSCT). Results In this study, 47 patients (27 boys and 20 girls), younger than 16 years, were diagnosed with vSAA (n= 21) or SAA (n=26). The median age was 7,71 years, ranging from 0.5 to 16 years and the average time history (beginning of signs and symptoms related to the disease and diagnosis) of the disease was 4,82 months, ranging from 0,25 to 48 months. Of the 47 patients, 45 had idiopathic AAA and 2 had hepatitis-associated. The median follow-up was 6,91 years for the patients treated with IST and 3,10 years for the patients who underwent to HSCT. One patient died before any treatment. For the eight patients who underwent to allogenic HLA-matched HSCT the 5-years-survival probability was 50%. For the 38 patients treated with IST, ten of them received cyclosporine and a short course of corticosteroids (CsA/CE) and 28 received antithymocyte globulin plus cyclosporine (ATG/CSA). The 5 years survival probability was 40% and 55%, respectively (p:0,0054). According to the severity of AAA, we did not show a significant difference in survival (p:0,32). Eight patients received second treatment after 1 year and 6 months (6 ATG from different species and CsA, 1 CsA and 1 thalidomide) and the probably of survival at 5 years was 60%. Among the 18 patients who responded to IST, four relapsed (22%). Two patients developed acute myeloid leukemia at 5 and 12 years after diagnosis. Conclusion Our results both for patients undergoing HSCT, as well as patients undergoing IST are lower in comparison to other hematological centers. Probably, this discrepancy is related to the prolonged time of disease when patients are admitted to our service. Unfortunately, the difficulty of access to specialized centers for diagnosis and early treatment in our country is a reality and this fact contributes to the delay to the beginning of treatment. Disclosures: No relevant conflicts of interest to declare.

More Intensity Immuno-Suppression In Conditioning Regimen  may Favor Donor Stem Cell Sustained Engraftment In Allogeneic Stem Cell Transplantation For Acquired Severe Aplastic Anemia Patients

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 5452-5452
Author(s):  
Sun Can ◽  
Lin Xia ◽  
Huang Yuxian ◽  
Chen Tuzhen ◽  
Bingyi Wu
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Aplastic Anemia ◽  
Cell Transplantation ◽  
Graft Failure ◽  
Conditioning Regimen ◽  
Severe Aplastic Anemia ◽  
Hematopoietic Stem ◽  
Acquired Severe Aplastic Anemia

Abstract Background Hematopoietic stem cell transplantation (HSCT) is the first-line therapy for patients younger less than 40 years old with severe aplastic anemia (SAA). And the long-term survival for patients with SAA who received HSCT reaches to 70%-90%. Cyclophosphamide-based conditioning regimen with or without antithymocyte globulin (ATG) has been adopted in majority of HSCT for SAA patients with HLA matched related donor. However the graft rejection and graft failure in HSCT for SAA with cyclophosphamide-based conditioning regimen is still as high as 5%-16%. The aim of this study is to explore whether more immue suppression in conditioning regimen could favor the donor stem cells sustained engraftment for severe aplastic anemia patients receiving allogeneic hematopoietic stem cell transplantation. Fludarabine and busulfan were added in cyclophosphamide-based conditioning regimen to intensity immune suppression in conditioning. Methods To analyze the outcomes and chimeras of 40 patients with SAA who received HLA matched allo-HSCT from 2000 to 2012 with either fludarabine-based conditioning regimen or cyclophosphamide-based conditioning regimen retrospectively and to explore the relationship between the chimeras and conditioning regimen. Results Forty patients with SAA who received HLA matched allo-HSCT From May, 2000 to Dec. 2012. Twelve patients ( median age 25 year old  range 13-52, male 7, femal 5) received  fludarabine-based conditioning regimen which composed of fludarabine (30 mg/m2/d ×5d), busulfan ( 3 mg/kg ×2d ), cyclophosphamide ( 60mg/kg/d×2d) and ATG (2.5mg/kg/d ×5d). Twenty patients( median age 23 year old  range 12-42, male 19, femal 9)  received  cyclophosphamide-based conditioning regimen which composed of cyclophosphamide (50mg/kg/d ×4d )and ATG (2.5mg/kg/d ×2d ). The  median dose of MNC were 4.5×108/kg (range 3.8-7.0×108/kg )and 3.58×108/kg (range 3.2-6.8×108/kg ) and CD34+ cells were  4.5×108/kg and 3.58×108/kg respectively. GVHD prophylaxis were cyclosporine and short-term course methotrexate. Donor chimera was detected on day+30, +90, +180, and 360 after HSCT by short tandem repeat polymerase chain reaction, or fluorescein in situ hybridization for X and Y chromosomes in cases when patients and donors were sex mismatched. Results All patients with fludarabine-based conditioning regimen were successful Hematopoietic reconstitution and no graft failure occurred in this group patients. But two patients could not get recovery in cyclophosphamid-based conditioning regimen group. And complete donor chimeras always present when chimeras were detected by STR-PCR or FISH at day 30,day 60, day 90 and d 180 post transplantation in fludarabine-based conditioning regimen group, while seven patients with cyclophosphamide-based conditioning regimen were mixed chimeras at day 30 post transplant.  The graft was rejected in six of the seven patients at day 90 post transplant in cyclophosphamide-based conditioning regimen group. The complete donor chimera in fludarabine-based conditioning regimen group was obvious higher than that in cyclophosphamide-based conditioning regimen group ( p=0.037). The incidence of aGVHD in the fludarabine group was 16.7% and 10.7% in the cyclophosphamide-based group. There is no significant difference of aGVHD between two group (P = 0.627). The incidence of cGVHD was 8.3% and 10.7% respectively. The bacterial infections developed in 16.7% and 28.6% of patients respectively (P=0.693), The overall survival were 83.33% and 82.14% in fludarabine-based conditioning regimen group and cyclophosphamide-based group respectively (p=0.870). Conclusions More intensity immuno-suppression in conditioning regimen may favor donor stem cell sustained engraftment in allogeneic stem cell transplantation for acquired severe aplastic anemia patients. Disclosures: No relevant conflicts of interest to declare.

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