scholarly journals The feasibility of conditioning regimen of fludarabine, ATG, and reduced dose of cyclophosphamide in patients with severe aplastic anemia who received HLA-matched sibling transplantation

2006 ◽  
Vol 12 (2) ◽  
pp. 7
Author(s):  
J.W. Lee ◽  
S.Y. Kim ◽  
K.S. Eom ◽  
Y.J. Kim ◽  
H.J. Kim ◽  
...  
Keyword(s):  
Aplastic Anemia ◽  
Conditioning Regimen ◽  
Severe Aplastic Anemia ◽  
Reduced Dose ◽  
Matched Sibling

Risk Factors for Graft Failure and Mortality after HLA-Matched Sibling Donor Transplant for Severe Aplastic Anemia in Brazil.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 622-622 ◽  
Author(s):  
Ricardo Pasquini ◽  
Jeanette Carreras ◽  
Mei-Jie Zhang ◽  
Marco A. Bitencourt ◽  
Jefferson Ruiz ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Overall Survival ◽  
Blood Transfusion ◽  
Aplastic Anemia ◽  
Graft Failure ◽  
Conditioning Regimen ◽  
Severe Aplastic Anemia ◽  
Failure Rates ◽  
Platelet Recovery ◽  
Matched Sibling

Abstract Higher rates of graft failure observed after conventional cyclophosphamide (Cy) at 200mg/kg and HLA-matched sibling bone marrow transplant (BMT) for severe aplastic anemia (SAA) in South America prompted the use of a different conditioning regimen: busulfan (Bu) at 12 mg/kg plus Cy at 120 mg/kg. This change was instituted to provide greater immunosuppression for heavily transfused patients (>15 blood transfusion units pre-BMT). We report transplant outcomes in 269 SAA patients who received their HLA-matched BMT at a single center in Brazil between 1990 and 2003. Median age at transplant was 19 years (range 2 – 45). Median time from diagnosis to transplant was 3 months; 78% were transplanted ≤ 6 months from diagnosis, 11%, 7–24 months and 11% > 24 months. 128 patients received Cy alone, 2, Cy plus anti-thymocyte globulin and 139, Bu plus Cy. Eighty-one patients (62%) who received Cy conditioning also received ≤15 blood transfusion units and 49 (38%) received >15 blood transfusion units pre- BMT; all patients who received Bu plus Cy conditioning received >15 blood transfusion units. All patients received T-cell replete bone marrow grafts and almost all patients received cyclosporine and short course methotrexate for graft-versus-host disease prophylaxis. Median follow-up of surviving patients after Cy is 9 years and after Bu plus Cy, 6 years. This reflects the change in practice after 1994, when Bu plus Cy was favored for patients who received >15 blood transfusion units pre-BMT. Most patients achieved neutrophil recovery; the day-60 probabilities of recovery were 95% and 86% after Cy conditioning (≤15 and >15 blood transfusion units, respectively) and 92% after Bu plus Cy. Corresponding day-100 probabilities of platelet recovery were 95%, 86% and 87%. Thirty-nine patients subsequently lost their graft; failure rates were similar after Bu plus Cy and Cy conditioning in patients who received ≤15 blood transfusion units (13% vs. 17%; RR 0.97, p=0.924). Though failure rates were higher (10 of 42, 24%) after Cy conditioning in patients who received >15 blood transfusion units this did not attain statistical significance when compared to those who received Bu plus Cy (16 of 128, 13%, p=0.077). This may be explained by limited numbers of patients in the heavily transfused Cy group. Younger age (≤10 years) was the only factor that was associated with higher rates of secondary graft failure (RR 2.91, p=0.001). As expected, the 8-year probability of overall survival was highest (87%) after Cy conditioning in patients who received ≤15 blood transfusion units. Mortality rates were higher after Bu plus Cy conditioning (RR 3.18, p<0.001) and Cy conditioning in patients who received > 15 blood transfusion units (RR 4.66, p<0.001). The corresponding 8-year probabilities of overall survival were 67% and 51% (p=0.059). The data suggest secondary graft failure rates are similar after Bu plus Cy conditioning in patients who received >15 blood transfusion units compared to those who received Cy conditioning and fewer transfusions. Though survival rates are generally lower in patients who receive >15 blood transfusion units, the use of Bu plus Cy appears to confer a survival advantage. Patients with SAA should be referred for transplantation as soon as the diagnosis is confirmed to avoid excess blood transfusion pre-BMT.

Outcome of Allogeneic Stem Cell Transplantation for Patients with Severe Aplastic Anemia after Conditioning with Procarbazine, ATG and Cyclophosphamide: A Single Center Experience.

Blood ◽  
2005 ◽  
Vol 106 (11) ◽  
pp. 2027-2027
Author(s):  
Ki Seong ◽  
Jong Wook Lee ◽  
Yoo Jin Kim ◽  
Hee Je Kim ◽  
Chang Ki ◽  
...  
Keyword(s):  
Stem Cell ◽  
Stem Cell Transplantation ◽  
Aplastic Anemia ◽  
Cell Transplantation ◽  
Chronic Gvhd ◽  
Univariate Analysis ◽  
Conditioning Regimen ◽  
Severe Aplastic Anemia ◽  
Platelet Recovery ◽  
Matched Sibling

Abstract Background: Cyclophosphamide (CY, 200 mg/kg) plus ATG seems to be accepted as a standard conditioning regimen in HLA-matched sibling stem cell transplantation (SCT) for severe aplastic anemia (SAA). We report herein the outcome of allogeneic SCT for the patients with SAA conditioned with procarbazine (PCB), ATG and CY. Methods: Between January 1995 and March 2004, consecutive one hundred and sixty two patients with SAA received matched sibling SCT after conditioning with PCB, ATG and CY. The median age of patients was 28.5 (16~50) and median interval between diagnosis and SCT was 10.5 months (1~216). Fifty-five patients (34%) had a history of immunosuppressive therapy before SCT. The conditioning regimen consisted of PCB (6.25 mg/kg/day, 6 days), CY (50 mg/kg/day, 4 days) and ATG (1.25 mg/kg/day, 3 days). No graft manipulation was performed in 92 patients (52%), and donor marrow expansion and megadose transplantation (bone marrow plus CD34+ isolated PB as a stem cell source) were done in 35 patients (21.6%). All patients received of cyclosporine and methotrexate as GVHD prophylaxis. Results: The median dose of CD34+ and CD3+ cells infused were 4.8 x 106/kg (0.8~49.6) and 5.1 x 107/kg (0 ~ 38), respectively. Median time to neutrophil recovery more than 500/μL and platelet recovery more than 20,000/μL were 13 days (0~49) and 19 days (0~150), respectively. All patients achieved successful primary engraftment, but delayed engraftment failure developed in 18 patients (11%). Ten among 18 patients who developed graft failure received second SCT, resulting survival of 9 patients. The incidence of acute (more than grade II) and chronic GVHD were 9.3% (n=15) and 13.0 % (n=21), respectively. DFS and overall survival (OS) at 6 years was also 90%. Factors influencing on DFS on univariate analysis were patient and donor’s age, development of acute and chronic GVHD, and times of pregnancy (over 2 times). Development of acute and chronic GVHD, and occurrence of rejection were factors that influence DFS on multivariate analysis. Causes of death (n=15) were pneumonia (n=4), rejection (n=4), GVHD±infection (n=2), chronic GVHD (n=1), MOF (n=1), CVA (n=1), autoimmune hemolytic anemia (n=1), and unknown (n=1). Conclusions: These data suggest that the conditioning regimen used in this study is feasible for patients with SAA who received matched sibling SCT. GVHD and rejection were the most important factors that influence DFS.

scholarly journals Bone marrow transplantation for children with severe aplastic anemia: use of donors other than HLA-identical siblings

Blood ◽  
1989 ◽  
Vol 74 (5) ◽  
pp. 1852-1857 ◽  
Author(s):  
B Camitta ◽  
R Ash ◽  
J Menitove ◽  
K Murray ◽  
C Lawton ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Aplastic Anemia ◽  
Marrow Transplantation ◽  
Chronic Gvhd ◽  
Conditioning Regimen ◽  
Pneumocystis Pneumonia ◽  
Severe Aplastic Anemia ◽  
High Dose ◽  
Gvhd Prophylaxis

Abstract Eighty-five percent of untransfused and 70% of transfused patients with severe aplastic anemia (SAA) are cured with bone marrow transplants from histocompatible sibling donors. Use of partially matched family donors or unrelated donors has been relatively unsuccessful because of high incidences of graft rejection and graft-versus-host disease (GVHD). Thirteen children with SAA received marrow grafts from alternative donors (sibling 4, parent 5, unrelated 4). The first three patients were pretreated with cyclophosphamide (CYCLO) +/- irradiation and received methotrexate for GVHD prophylaxis. Subsequent children were pretreated with CYCLO + high-dose cytosine arabinoside + methylprednisolone + total body irradiation, had monoclonal antibody T- cell depletion of the donor marrow, and received cyclosporine for GVHD prophylaxis. Three heavily transfused patients with haploidentical- related donors failed to engraft and died. All 10 patients with more closely matched donors engrafted. Acute GVHD was grade II in only one patient (non-T-depleted); this patient is the only one with severe chronic GVHD. Three engrafted patients died (Pneumocystis pneumonia, systemic parainfluenza, venocclusive disease). Seven children are alive 33+ to 2,692+ days. Donors for the survivors were siblings 3, parent 1, unrelated 3. These data suggest that bone marrow transplantation from closely matched donors other than histocompatible siblings can be effective therapy for SAA if an intensive conditioning regimen is used. These results must be confirmed with larger numbers and longer follow- up.

Reduced dose cyclophosphamide, fludarabine and antithymocyte globulin for sibling and unrelated transplant of children with severe and very severe aplastic anemia

2013 ◽  
Vol 17 (4) ◽  
pp. 387-393 ◽  
Author(s):  
Nack-Gyun Chung ◽  
Jae Wook Lee ◽  
Pil-Sang Jang ◽  
Dae-Chul Jeong ◽  
Bin Cho ◽  
...  
Keyword(s):  
Aplastic Anemia ◽  
Antithymocyte Globulin ◽  
Severe Aplastic Anemia ◽  
Reduced Dose

Comparison of HLA-matched sibling and unrelated donor transplantation in adult patients with acquired severe aplastic anemia

2020 ◽  
Vol 55 (8) ◽  
pp. 1570-1579 ◽  
Author(s):  
Seung Hwan Shin ◽  
Sung Soo Park ◽  
Jae Ho Yoon ◽  
Seung Ah Yahng ◽  
Sung Eun Lee ◽  
...  
Keyword(s):  
Aplastic Anemia ◽  
Adult Patients ◽  
Severe Aplastic Anemia ◽  
Unrelated Donor ◽  
Matched Sibling ◽  
Acquired Severe Aplastic Anemia

scholarly journals Increased incidence of solid malignant tumors after bone marrow transplantation for severe aplastic anemia [see comments]

Blood ◽  
1991 ◽  
Vol 78 (2) ◽  
pp. 277-279 ◽  
Author(s):  
G Socie ◽  
M Henry-Amar ◽  
JM Cosset ◽  
A Devergie ◽  
T Girinsky ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Aplastic Anemia ◽  
Marrow Transplantation ◽  
Malignant Tumors ◽  
Conditioning Regimen ◽  
Severe Aplastic Anemia ◽  
Male Patients

Abstract From May 1980 to December 1989, 107 consecutive patients with non- constitutional severe aplastic anemia underwent bone marrow transplantation at our institution using cyclophosphamide and thoraco- abdominal irradiation as conditioning regimen. During the same period, 40 patients with Fanconi anemia were also grafted after a similar conditioning, giving a total series of 147 patients. With a mean follow- up of 64 months, four male patients developed a solid malignant tumor, a number that leads to an 8-year cumulative incidence rate of 22% (eg, relative risk to general population = 41, P less than .001). These results should be considered as a warning to clinicians who follow these successfully grafted long-term patients.

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