scholarly journals Unrelated cord blood transplantation in patients with idiopathic refractory severe aplastic anemia: a nationwide phase 2 study

Blood ◽  
2018 ◽  
Vol 132 (7) ◽  
pp. 750-754 ◽  
Author(s):  
Regis Peffault de Latour ◽  
Sylvie Chevret ◽  
Charlotte Jubert ◽  
Anne Sirvent ◽  
Claire Galambrun ◽  
...  
Keyword(s):  
Cord Blood ◽  
Aplastic Anemia ◽  
Cord Blood Transplantation ◽  
Young Patients ◽  
Phase 2 ◽  
Blood Transplantation ◽  
Phase 2 Study ◽  
Key Points ◽  
Unrelated Donors ◽  
Unrelated Cord Blood

Key Points CBT after FLU-CY-ATG-2-Gy TBI with at least 4 × 107 frozen NCs per kilogram leads to satisfactory OS in refractory SAA. CBT is a valuable curative option in young patients with refractory idiopathic SAA and no available matched unrelated donors.

scholarly journals Sequential Transplantation of Haploidentical Stem Cell and Unrelated Cord Blood With Using ATG/PTCY Increases Survival of Relapsed/Refractory Hematologic Malignancies

2021 ◽  
Vol 12 ◽  
Author(s):  
Hua Li ◽  
Xiaofan Li ◽  
Yiling Chen ◽  
Duihong Li ◽  
Xianling Chen ◽  
...  
Keyword(s):  
Cord Blood ◽  
Cord Blood Transplantation ◽  
Chronic Gvhd ◽  
Disease Free Survival ◽  
Hematologic Malignancies ◽  
Post Transplantation ◽  
Blood Transplantation ◽  
Gvhd Prophylaxis ◽  
Unrelated Donors ◽  
Unrelated Cord Blood

Allogeneic haploidentical HSCT (haplo-HSCT) and unrelated umbilical cord blood transplantation(UCBT)are used in patients lacking HLA-identical sibling or unrelated donors. With myeloablative condition and GVHD prophylaxis of using low-dose ATG and post-transplantation cyclophosphamide (PTCY), we conducted a prospective clinical trial. Of eligible 122 patients from February 2015 to December 2019 in the study, 113 patients were involved. Forty-eight patients were in the group of sequential haplo-cord transplantation (haplo-cord HSCT), and 65 patients were in the group of single UCBT. The primary endpoint of 2-year disease-free survival (DFS) was no statistical difference between groups (64.1 vs. 56.5%), p>0.05. The analysis of subgroup patients with relapsed/refractory showed haplo-cord HSCT was associated with better OS (HR 0.348, 95% CI, 0.175–0.691; p=0.0025), DFS (HR 0.402, 95% CI, 0.208–0.779; p=0.0069), and GRFS (HR 0.235, 95% CI, 0.120–0.457, p<0.0001) compared to the single cord group. The 2-year’s probability in OS, DFS, and GRFS was 64.9 vs. 31.6%, 64.5 vs. 31.6%, and 60.8 vs. 15.0% in the haplo-cord group and single cord group, respectively. III-IV acute GVHD 8.3 vs. 6.2%, chronic GVHD 25.8 vs. 13.7%, and extensive chronic GVHD 5.3 vs. 1.8% were shown in corresponding group, p>0.05. The patients engrafted persistently with UCB showed better survival outcomes. Our sequential Haplo-cord HSCT with ATG/PTCY improved the survival of patients and might be an alternative transplantation approach for patients with relapsed/refractory hematologic malignancies.

Reduced-Intensity Unrelated Cord Blood Transplantation for Adult Patients with Severe Aplastic Anemia

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 4408-4408
Author(s):  
Hisashi Yamamoto ◽  
Daisuke Kato ◽  
Naoyuki Uchida ◽  
Kazuya Ishiwata ◽  
Shinsuke Takagi ◽  
...  
Keyword(s):  
Cord Blood ◽  
Aplastic Anemia ◽  
Therapeutic Option ◽  
Cord Blood Transplantation ◽  
Conditioning Regimen ◽  
Cell Number ◽  
Adult Patients ◽  
Blood Transplantation ◽  
Unrelated Cord Blood ◽  
Reduced Intensity

Abstract Although unrelated cord blood transplantation (UCBT) has become a viable therapeutic option widely applicable for adult patients with hematological diseases, UCBT for severe aplastic anemia (SAA) remains to be controversial due to higher incidence of graft failure (GF) relative to the other stem cell sources. To evaluate the feasibility of UCBT for SAA, we retrospectively analyzed results for 11 adult patients with SAA who received first UCBT after reduced-intensity conditioning regimen (RI-UCBT) in Toranomon Hospital. Median age was 49 years (range, 20–70). Nine of them were non-responders to intensive immunosuppressive therapy and were multiply transfused, and two patients were fulminant type with no neutrophils in peripheral blood at diagnosis. The conditioning regimen consisted of fludarabine 125mg/m2, melphalan 80mg/m2, and 4 Gy of total body irradiation. Graft-versus-host disease prophylaxis was composed of cyclosporine alone (n=2), tacrolimus alone (n=2), and tacrolimus plus mycophenolate mofetil (n=7). Median total nucleated cell number and median CD34+ cell number were 2.65 × 107/kg (range, 1.83–4.39) and 0.70 × 105/kg (range, 0.27–1.52), respectively. Serological HLA disparities were as follows; 6/6 (n=3), 5/6 (n=2), and 4/6 (n=6). Ten of the 11 patients achieved primary neutrophil and platelet engraftment. Median time to an absolute neutrophil count > 0.5×109/liter and an unsupported platelet count > 20×109/liter were 18 days (range, 12–28) and 42.5 days (range, 26–64) respectively. All patients who achieved engraftment resulted in complete hematological recovery with complete donor chimerism, except for one patient who developed late GF at three years after UCBT. All 2 who experienced GF (1: primary GF, 1: late GF) were rescued by second RI-UCBT. Two of the 11 patients died from IPS (n=2), and remaining 9 patients are alive, having survived for 18.7 months (range, 2–71 months). Six out of 7 patients who survived more than 6 months were free from immunosuppressant. The probabilities of overall survival and event-free survival at 2 years after UCBT were 77.8% and 68.2%, respectively. Our results here strongly indicated feasibility and effectiveness of RI-UCBT for adult SAA patients with high engraftment rate and low treatment-related mortality. RI-UCBT could be considered as a viable therapeutic option for those who lack suitable HLA-matched sibling donors and failed immunosuppressive therapy.

Unrelated Cord Blood Transplantation for Severe Acquired Aplastic Anemia Using a Reduced-Intensity Conditioning: High Graft Rejection but Good Survival

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 2368-2368
Author(s):  
Huilan Liu ◽  
Liangquan Geng ◽  
Xingbing Wang ◽  
Kaiyang Ding ◽  
Baolin Tang ◽  
...  
Keyword(s):  
Cord Blood ◽  
Total Dose ◽  
Aplastic Anemia ◽  
Graft Rejection ◽  
Research Funding ◽  
Cord Blood Transplantation ◽  
Unrelated Cord Blood Transplantation ◽  
Blood Transplantation ◽  
Unrelated Cord Blood ◽  
Reduced Intensity

Abstract Abstract 2368 We report a single center experience in treating 16 consecutive patients (nine male and seven female) with severe aplastic anemia (AA) who received unrelated cord blood transplantation (UCBT) between 2006 and 2010. The main outcomes of interest were the tolerability and toxicity of UCBT, hematopoietic reconstitution and survival. The first two patients using a conditioning regimen consisting of cyclophosphamide (total dose 120°<<200 mg/kg), rabbit anti-thymocyte globulin (ATG, total dose 10°<<15 mg/kg) and methylprednisolone (total dose 1.5g). The other 14 cases received a reduced-intensity regimen composed of cyclophosphamide (total dose1200mg/©O), ATG (total dose 30 mg/kg) and fludarabine (total dose 120°<<180 mg/m2). Cyclosporine and mycophenolate mofetil were used as GVHD prophylaxis. The median age was 17 years (range 5–61 years) and the median weight was 48 kg (range 16–65 kg). Ten of sixteen were very SAA (VSAA). The median interval between diagnosis and UCBT was 30 days (range, 15–180). All except one did not received ATG-based immunosuppressive therapy before UCBT. Twelve patients received single UCB unit, four cases received double units. Donor/recipient HLA was at least four of six loci matching. The median nucleated cell dose infused was 4.27×107 cells/kg (range 2.34–13.41×107 cells/kg) and CD34+ cell dose infused was 2.02×105 cells/kg (range 0.71–4.35×105 cells/kg). Two patients were not evaluable for engraftment because of early death on day +21 and +22 due to severe infection and intracranial hemorrhages, respectively. Only one of the fourteen cases had engraftment with complete chimerism of single UCB unit from day 7, whose ANC© f 0.5 ×109/L and platelet© f20 ×109/L occurred on day 12 and day 31 post double UCBT, respectively. But she experienced secondary graft failure after three months post transplantation and attained survival by successful haplo-identical related transplantation. Thirteen patients experienced primary graft rejection, but all of them acquired autologous recovery. The 3-month and 6-month cumulative incidence of response was 61.5% and 85.7%, respectively. Currently, 14 patients are alive, having survived for 189 to 1712 days (median, 544 days) after their transplantations. The probability of overall survival at 4 years was 87.5%.Our data indicate that UCBT for new diagnosed SAA using no irradiation but fludarabine-based conditioning still seems to be inevitable to led to the high risk of rejection, but may facilitate autologous recovery and improve survival with low risk of transplant-related mortality. Disclosures: Sun: Fund of 11th Five-Year Science and Technology Key Project of Anhui Province (06013128B): Research Funding; Fund of Anhui Provincial “115” Industrial Innovation Program: Research Funding.

scholarly journals Filgrastim enhances T-cell clearance by antithymocyte globulin exposure after unrelated cord blood transplantation

2018 ◽  
Vol 2 (5) ◽  
pp. 565-574 ◽  
Author(s):  
Coco de Koning ◽  
Julie-Anne Gabelich ◽  
Jurgen Langenhorst ◽  
Rick Admiraal ◽  
Jurgen Kuball ◽  
...  
Keyword(s):  
T Cell ◽  
Cord Blood ◽  
Antithymocyte Globulin ◽  
Cord Blood Transplantation ◽  
Cd4 T Cell ◽  
Blood Transplantation ◽  
T Cell Reconstitution ◽  
Cell Clearance ◽  
Key Points ◽  
Unrelated Cord Blood

Key Points Residual ATG exposure delays CD4+ T-cell reconstitution more severely after CBT than after BMT. Filgrastim (G-CSF), given early after CBT, enhances ATG-mediated T-cell clearance in patients with residual ATG exposure.

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