scholarly journals Unrelated Donor Hematopoietic Stem Cell Transplantation for Treatment of Non-Malignant Genetic Diseases Using a Myeloablative Reduced Toxicity Conditioning Regimen

2015 ◽  
Vol 21 (2) ◽  
pp. S239
Author(s):  
Hisham Abdel-Azim ◽  
Quan Zhao ◽  
Kris Michael Mahadeo ◽  
Sajad Khazal ◽  
Donald B. Kohn ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Genetic Diseases ◽  
Conditioning Regimen ◽  
Unrelated Donor ◽  
Hematopoietic Stem ◽  
Reduced Toxicity

Allogeneic hematopoietic stem cell transplantation in thalassemia major: results of a reduced-toxicity conditioning regimen based on the use of treosulfan

Blood ◽  
2012 ◽  
Vol 120 (2) ◽  
pp. 473-476 ◽  
Author(s):  
Maria Ester Bernardo ◽  
Eugenia Piras ◽  
Adriana Vacca ◽  
Giovanna Giorgiani ◽  
Marco Zecca ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Cumulative Incidence ◽  
Chronic Gvhd ◽  
Conditioning Regimen ◽  
Unrelated Donor ◽  
Hematopoietic Stem

Abstract Sixty thalassemia patients (median age, 7 years; range, 1-37) underwent allogeneic hematopoietic stem cell transplantation (HSCT) after a preparation combining thiotepa, treosulfan, and fludarabine. Before HSCT, 27 children were assigned to risk class 1 of the Pesaro classification, 17 to class 2, and 4 to class 3; 12 patients were adults. Twenty patients were transplanted from an HLA-identical sibling and 40 from an unrelated donor. The cumulative incidence of graft failure and transplantation-related mortality was 9% and 7%, respectively. Eight patients experienced grade II-IV acute GVHD, the cumulative incidence being 14%. Among 56 patients at risk, 1 developed limited chronic GVHD. With a median follow-up of 36 months (range, 4-72), the 5-year probability of survival and thalassemia-free survival are 93% and 84%, respectively. Neither the class of risk nor the donor used influenced outcome. This treosulfan-based preparation proved to be safe and effective for thalassemia patients given allogeneic HSCT.

Allogeneic Hematopoietic Stem Cell Transplantation for Acute Myeloblastic Leukemia (AML) or Myelodysplastic Syndrome (MDS) in Patients (pts) Older Than 50 Years: a Retrospective Single Center Study

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 3309-3309
Author(s):  
Marie Robin ◽  
Raphael Porcher ◽  
Lionel Ades ◽  
Emmanuel Raffoux ◽  
Régis Peffault de Latour ◽  
...  
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Conditioning Regimen ◽  
Refractory Anemia ◽  
Unrelated Donor ◽  
Hematopoietic Stem ◽  
Co Morbidity

Abstract Background: Allogeneic hematopoietic stem cell transplantation (HSCT) remains the only curative treatment in MDS, and also in many cases of AML, 2 diseases where median age is higher than 50 years. Aim: Analyse the outcome of patients older than 50 years who received an allogeneic hematopoietic stem cell transplantation (HSCT) for MDS or AML, in Saint-Louis Hospital from January 1997 to December 2007. Method: 48 patients (pts) aged from 50 to 68 years (median: 56) received an HSCT for MDS (N=28) or AML (N=20) during this period. AML pts had intermediate (n=15) or high risk cytogenetics (n=3). 8 pts had failure to initial induction chemotherapy. FAB classification for MDS was: RAEB (n=8), RAEBt (n=6), secondary AML (n=2) or refractory anemia (n=2). Maximal IPSS score was high, intermediate-2 and intermediate-1 in 11, 14 and 4 pts. Ten pts with MDS received an “AML-like” chemotherapy and 5 received demethylating agents before transplant. 18 of the AML pts were in complete remission (CR) at time of transplant (CR1, n=15; CR2, n=3) and 2 were in relapse at time of HSCT. 10, 9 and 9 patients with MDS had < 5%, 5–10% and > 10% blasts in bone marrow at time of HSCT. Results: 19 patients with AML (95%) and 16 patients with MDS (57%) received a HSCT from an HLA-identical sibling donor. Other pts received a matched unrelated donor. Thirty-two pts, including 9/20 and 23/28 had at least one co-morbidity according to Sorror score. The conditioning regimen was myeloablative (MAC) in 14 pts (29%). Reduced intensity (RIC) was fludarabine based in 34 pts, associated with either 2 Gy TBI (n=25) or chemotherapy (n=9). All pts engrafted. 35 pts had acute graft-versus-host disease (GVHD): grade I in 11, grade II in 18 and grade III in 6. GVHD incidence did not differ between pts who received a MAC or a RIC regimen. Two-year overall survival (OS) was 41% (95% CI: 26–57). OS and relapse-free-survival (RFS) were similar after MAC or RIC regimen (OS: 29% vs 35% and RFS: 29% vs 30%). Short-term non-relapse mortality (NRM) was lower in patients who received a RIC as compared to patients who received a MAC regimen but was similar at long-term (6-month NRM = 21% vs 9% and 12-month NRM = 21 vs 19 %). Patients with AML or MDS had similar OS and RFS (OS: 42 vs 41%; RFS: 37 vs 26% at 2 years for AML and MDS, respectively). NRM was not significantly higher in pts with MDS (26 vs 10% at one year) whereas relapse rate was not significantly higher in pts with AML (13 vs 6%). Conclusion: HSCT for AML or MDS after 50 years is a curative option for pts with related or unrelated HLA-identical donor regardless kind of conditioning regimen and co-morbidity at time of transplantation.

scholarly journals Role of KIR typing in donor selection prior to allogeneic hematopoietic stem cell transplantation: literature review

2019 ◽  
Vol 13 (4) ◽  
pp. 67-74
Author(s):  
V. V. Zakharova
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Nk Cell ◽  
Conditioning Regimen ◽  
Unrelated Donor ◽  
Hematopoietic Stem

Natural killer (NK) cells are the first population to recover after allogeneic hematopoietic stem cell transplantation. Since the report in 2002 by L. Rugerri et al. showing the effectiveness of NK cell alloreactivity in haploidentical stem cell transplantation, a lot of conflicting studies have appeared about the role of NK alloreactivity in haploidentical and matched unrelated donor transplantations. Current studies demonstrate that the beneficial effects of donor NK alloreactivity are dependent on the transplant protocol – conditioning regimen, graft processing procedure and graft-versus-host disease.

scholarly journals Cytomegalovirus reactivation in patients treated with allogeneic hematopoietic stem cell transplantation

2021 ◽  
Vol 2 (2) ◽  
pp. 82-91
Author(s):  
Jovana Kessler ◽  
Katarina Ivanović ◽  
Dejana Stanisavljević ◽  
Milena Todorović-Balint
Keyword(s):  
Stem Cell ◽  
Hematopoietic Stem Cell Transplantation ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Hematopoietic Stem Cell ◽  
Conditioning Regimen ◽  
Unrelated Donor ◽  
Hematopoietic Stem ◽  
Cytomegalovirus Reactivation ◽  
Cmv Reactivation

Introduction: Opportunistic CMV reactivation is the most common viral complication after allogenic hematopoietic stem cell transplantation (allo-HSCT). Aim: The aim of our study is to evaluate the frequency of CMV reactivation in relation to the serostatus od the donor and the recipient, and the correlation with the day of leukocyte (Le) and thrombocyte (Tr) engraftment. We compared the frequency of CMV reactivation in myeloablative conditioning (MAC) versus reduced intensity conditioning (RIC), as well as in match related donor (MRD) versus match unrelated donor (MUD) allo-HSCT. We analyzed whether CMV reactivation affected the overall survival (OS) after allo-HSCT. Materials and methods: In a retrospective cohort study, we inspected 42 patients over the age of 18 years, who were treated at the Clinic for Hematology of the Clinical Center of Serbia, from December 2017 to November 2019. Results: Most CMV reactivations were noticed if the recipient (R) was seropositive, and the donor (D) was seronegative (R+/D= 60.0%). The number of CMV DNA copies corelated with the day of leukocyte engraftment of (p = 0.031), but not of thrombocyte engraftment (p = 0.598). The frequency of reactivation in patients treated with RIC was 25.0%, and it was 63.5%, if they were treated with MAC. The intensity of the conditioning regimen corelated with the number of CMV DNA copies (p = 0.025%). There was no correlation found between the type of transplantation (MRD or MUD) and CMV reactivation (p = 0.515). OS after allo-HSCT was 36.39 months (95% CI 26,0 - 46,78). The mean OS in patients with CMV reactivation was 7.39 months (95% CI 5,72 - 9,06), but we did not prove that CMV reactivation had an impact on OS (p = 0.527). Conclusion: CMV reactivation was most common in the R+/Dgroup. CMV reactivation did not affect OS after allo-HSCT in our group of patients.

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