scholarly journals Results of unrelated cord blood transplant in patients with bone marrow failure syndromes

2004 ◽  
Vol 10 (10) ◽  
pp. 736-737 ◽  
Author(s):  
E. Gluckman
Keyword(s):  
Bone Marrow ◽  
Cord Blood ◽  
Bone Marrow Failure ◽  
Bone Marrow Failure Syndromes ◽  
Cord Blood Transplant ◽  
Unrelated Cord Blood

Double Cord Blood Transplantation in Bone Marrow Failure Syndromes.

Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 2018-2018
Author(s):  
Regis Peffault de Latour ◽  
Vanderson Rocha ◽  
Marie Robin ◽  
Celso A. Rodrigues ◽  
Delphine Rea ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Stem Cell ◽  
Cord Blood ◽  
Acute Gvhd ◽  
Bone Marrow Failure ◽  
Cord Blood Transplantation ◽  
Blood Transplantation ◽  
Bone Marrow Failure Syndromes ◽  
Cord Blood Unit ◽  
Grade Ii

Abstract The outcome of severe aplastic anemia, refractory to immunosuppressive therapy or observed in case of Fanconi anemia (FA), is usually poor in the absence of Hematopoietic Stem Cell Transplantation (HSCT). Umbilical cord blood is an alternative stem cell source for patients without matched related or unrelated donors. However, single cord blood unit is associated with high transplant related mortality due to the low cell dose infused in previous highly transfused patients. We have driven the hypothesis that double cord blood transplantation (dCBT) could circumvent the cell dose problem. For the purpose of this study, we have studied 13 patients with bone marrow failure syndromes given 2 partially matched dCBT from 2004 to 2007. The diagnoses were FA (n=9), SAA (n=4) and PNH (n=1). Among those patients, 5 (39%) received a dCBT as a rescue of previous rejected transplants (2 SAA and 3 FA). All patients received a fludarabine-based regimen, with TBI (2 Gy) for 4 patients. Cord blood units were a 4/6 or 5/6 HLA A, B and DR match with the patient except one which was 3/6. Graft versus host disease (GVHD) prophylaxis consisted in CSA+MMF. Steroids were given from day 7 to day 14 and stopped in case of no GVHD. Five male (39%) and 8 female (61%) with a median age of 16 years (range 7–31) were treated. The cell doses infused were a median of 5.0 × 107 NC/Kg (4–9) and 5.3 × 105 CD34+ cells/Kg (2–8). Graft rejection was seen in 5 patients (2 previously allotransplanted). Among those patients, one displayed a temporary mixed chimerism before rejection and another presented an autologous reconstitution. Among the remaining 8 patients, the median time to an absolute neutrophils count > 500 was 25 days (range 14–42) and the median time to a platelet count > 20,000 was 39 days. In these last patients, we observed a complete donor chimerism with one cord blood unit during 100 days after dCBT. Acute GVHD grade II–III was scored in 9 patients (69%) (7 grade II, 2 grade III). No patients presented acute GVHD grade IV. Four patients out of 8 developed Chronic GVHD (3 limited and 1 extensive). Four patients died (1 GVHD, 2 fungal infection, 1 thrombotic microangiopathy). With a median follow-up of 13 months (range 1 to 19 months), the overall survival was 55% (±15%) for all patients. The median survival of patients who were transplanted twice was 50% (±25%). In conclusion, dCBT seems to be an option to treat patients with bone marrow failure syndromes and without a suitable compatible HLA donor. Those results need to be established on a large number of patients to warrant the inclusion of dCBT in the treatment strategy of diseases with high risk of rejection.

scholarly journals The Impact of Identifying the Syndromic and Genetic Diagnoses on Hematopoietic Stem Cell Transplantation Outcome in Patients with Inherited Bone Marrow Failure Syndromes

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 5015-5015
Author(s):  
Yeon Jung Lim ◽  
Omri Avraham Arbiv ◽  
Melanie Evelyn Kalbfleisch ◽  
Robert J Klaassen ◽  
Conrad Fernandez ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Overall Survival ◽  
Stem Cell ◽  
Cord Blood ◽  
Bone Marrow Failure ◽  
Stem Cell Source ◽  
Bone Marrow Failure Syndromes ◽  
Cell Source ◽  
Syndromic Diagnosis ◽  
The Impact

Background: Over the last decade major progress has been made in developing new diagnostic methods and in phenotypic and molecular classification of inherited bone marrow failure syndromes (IBMFSs). Nevertheless, data from the Canadian Inherited Marrow Failure Registry (CIMFR) indicates that 28% of patients with inherited bone marrow failure syndromes (IBMFS) cannot be assigned a specific syndromic diagnosis. These unclassified IBMFS (UIBMFS) cases may represent either novel syndromes or atypical presentations of previously described disorders. Hematopoietic stem cell transplantation (HSCT) is the only curative option for bone marrow failure and malignant myeloid transformation in IBMFSs. However, it is unknown whether the application of this treatment to UIBMFS patients without an ability to modify the procedure according to the underlying genetic and syndromic diagnosis affects outcome. To our knowledge, there are no published transplant data on cohorts of patients with UIBMFSs. The aims of this study were to evaluate the outcome and prognostic factors of HSCT in a cohort of patients with UIBMFSs and to determine whether the knowledge of the syndromic/genetic diagnosis before HSCT has an impact on transplant outcome. Methods: Patients were enrolled on the CIMFR if they were diagnosed with a specific IBMFSs (e.g. Fanconi anemia), and/or they had bone marrow failure and either a family history of bone marrow, or physical malformations or a diagnosis before the age of one year. Patients were considered as having an UIBMFS if they fulfilled the above criteria, but could not be assigned a specific syndromic diagnosis since they did not meet the diagnostic criteria for any known IBMFS. HSCT data were extracted from the CIMFR database and analyzed. Descriptive statistics were used to compare between groups. Cox proportional hazards model was used for univariate analysis to identify risk factors for worse overall survival post HSCT in patients with UIBMFSs. Results: Among the patients enrolled in the CIMFR, 22 with UIBMFSs and 68 with classified IBMFSs (CIBMFSs) underwent HSCT between January 2001 and December 31, 2017. Transplanted patients with UIBMFSs were hematologically characterized by multilineage cytopenia (n=13), single-lineage cytopenia (n=1), myelodysplastic syndrome (MDS) (n=5) or acute myeloid leukemia (AML) (n=3). Patients with CIBMFSs had Fanconi anemia (n=30), dyskeratosis congenita (n=7), Shwachman-Diamond syndrome (n=9), Kostmann syndrome (n=6), Diamond-Blackfan anemia (n=4) or others (n= 11). Median age at diagnosis of patients with UIBMFSs was 4.18 years (range; 0 to 32.0 years) and median age at HSCT for UIBMFSs was 5.74 years (range; 0.17-66.67 years). Median time between diagnosis of UIBMFS and HSCT was 0.48 years (range; 0.12 - 34.67), this was significantly shorter than that of CIBMFS (1.77 years, range; 0.17 - 15 years, P=0.014). Six patients (27.3%) of UIBMFS and 9 patients (19.7%) with CIBMFS underwent HSCT for MDS-RCEB or AML (P=0.15). The overall 5-year survival of UIBMFS patients was significantly inferior to that of CIBMFS patients: 56±11.4% vs. 76±5.5%, respectively (P=0.047). 5-year overall survival of patients with UIBMFSs was significantly worse among those whose stem cell source was cord blood (15±13.3%) vs. those who received other stem cell sources (91±8.7%, P=0.04), while stem cell source did not affect prognosis of patients with CIBMFSs. Engraftment failure among UIBMFS patients who received cord blood was significantly higher than engraftment failure among those who received bone marrow (55.6% vs. 9.1%, P=0.024). No other factors reached statistical significance when the impact of stem cell source on overall survival was analyzed, including transfusion load, transplant indications, intensity of conditioning regimens, related/non-related donor, degree of human leukocyte antigen (HLA) matching or identifying a diagnosis after HSCT. Conclusion: Identifying the syndromic diagnosis of IBMFSs is critically important when considering HSCT. The worse HSCT outcome of UIBMFSs in this study might be related to an inability to tailor the transplant approach to the patient specific phenotype and genotype. Our data suggest that cord blood should be avoided as a stem cell source in patients with UIBMFSs. Disclosures No relevant conflicts of interest to declare.

Double cord blood transplantation in patients with high risk bone marrow failure syndromes

2008 ◽  
Vol 143 (3) ◽  
pp. 404-408 ◽  
Author(s):  
A. Ruggeri ◽  
R. Peffault de Latour ◽  
V. Rocha ◽  
J. Larghero ◽  
M. Robin ◽  
...  
Keyword(s):  
Bone Marrow ◽  
High Risk ◽  
Cord Blood ◽  
Bone Marrow Failure ◽  
Cord Blood Transplantation ◽  
Blood Transplantation ◽  
Bone Marrow Failure Syndromes

Outocomes of Second Unrelated Cord Blood Transplant for Relapse After First Allogeneic Transplant In Adult Patients with Progressive Acute Myeloid Leukimia/Myelodysplastic Syndrome.

Blood ◽  
2010 ◽  
Vol 116 (21) ◽  
pp. 1295-1295
Author(s):  
Sachi Tainosho ◽  
Kazuhiro Masuoka ◽  
Aya Nishida ◽  
Nobuaki Nakano ◽  
Kazuya Ishiwata ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Mortality Rate ◽  
Myelodysplastic Syndrome ◽  
Cord Blood ◽  
Progressive Disease ◽  
Adult Patients ◽  
Cord Blood Transplant ◽  
Unrelated Cord Blood ◽  
Related Mortality ◽  
Acute Myeloid

Abstract Abstract 1295 <Objective> Disease relapse following allogeneic hematopoietic stem cell transplant (allo-HSCT) remains a major cause of treatment failure, often with a poor outcome. Some recent reports have demonstrated successful treatment receiving 2nd allo-HSCT with bone marrow (BM) and peripheral blood (PB) following relapse. However, there are few reports about unrelated cord blood transplant (UCBT). Therefore, we retrospectively analyzed the results of 2nd allo-HSCT using UCB for 34 adult patients with acute myeloid leukemia (AML) / myelodysplastic syndrome (MDS), who relapsed after 1st HSCT. <Patients and methods> We reviewed medical records of 130 adult patients with AML/MDS who received 1st HSCT between March 2006 and May 2009 at Toranomon Hospital, Tokyo, Japan. Fifty-three of these patients relapsed after 1st allo-HSCT including 12 recipients of related peripheral blood (RPB), 13 unrelated bone marrow (UBM) and 28 UCB. In these relapsed patients, 34 proceeded to 2nd transplant with UCB. The remaining 19 patients did not receive 2nd transplants because of severe organ dysfunction or uncontrolled active infection and continued complete remission by donor lymphocyte infusion. The median age at 2nd UCBT was 54 years (18-69) and interval between 1st allo-HSCT and 2nd UCBT was 9 months (1-54). Diagnoses include de novo AML (n=21) and MDS overt AML (n=13). Disease status at 1st allo-HSCT were as follows; 4 patients in CR, 25 patients in chemo-refractory and 5 patients in progressive desease without chemotherapy. All patients at 2nd UCBT were in progressive disease. All patients received a single cord blood unit with median TNC/CD34 cell dose of 2.8×107/kg (range, 1.4–4.8) and 0.7×105/kg (range, 0.2–2.2), respectively. HLA disparities were 3/6 match (n=4), 4/6 (n=25), 5/6 (n=4) and 6/6 (n=1). Conditioning regimen consisted of fludarabine and alkylating agent. Graft-versus-host disease (GVHD) prophylaxis consisted of tacrolimus (Tac) alone in 15, Tac plus mycophenolate mofetil in 11, and cyclosporine alone in 8. <Results> Ten patients died by day 28 because of progressive disease (n=5) and transplant related mortality (n=5) and 21 of 24 patients, who survived more than 28 days, were engrafted with complete donor type chimerism (median 19 days). Acute GVHD developed in 14, and chronic GVHD in 4 of these 21 engrafted patients. Estimated 3-year over all survival (OS), transplant related mortality rate and relapse mortality rate were 16%, 32%, and 52%. In univariate analysis, a variable associated with worsened survival were early relapse within 100 days after 1st allo-HSCT (0 vs 21% in late relapses; P=0.01), while other factors were not significant. Eighteen of 19 patients who did not undergo 2nd UCBT died of progressive disease or multiple organ failure. <Conclusion> 3 year-OS of 16% with 2nd UCBT following relapsed AML/ MDS was comparable with that of 2nd BMT or PBCST. Considering that all patients in this study were in non-remission and in relatively higher age (median age 54 years), 2nd UCBT following relapse of AML/MDS could be a viable therapeutic option. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Unmanipulated Bone Marrow As Third Party Donor for Unrelated Cord Blood Transplant Decreases 100-Day Mortality in Children

2015 ◽  
Vol 21 (2) ◽  
pp. S305-S306
Author(s):  
Oscar Ramirez ◽  
Margarita Quintero ◽  
Carlos Andres Portilla ◽  
Eduardo Lopez ◽  
Viviana Lotero ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Cord Blood ◽  
Third Party ◽  
Cord Blood Transplant ◽  
Unrelated Cord Blood
Sign in / Sign up

Export Citation Format

Share Document