scholarly journals Cord blood transplantation for bone marrow failure syndromes: state of art

2019 ◽  
Vol 6 ◽  
pp. 39-39 ◽  
Author(s):  
Simona Pagliuca ◽  
Annalisa Ruggeri ◽  
Régis Peffault de Latour
Keyword(s):  
Bone Marrow ◽  
Cord Blood ◽  
Bone Marrow Failure ◽  
Cord Blood Transplantation ◽  
Blood Transplantation ◽  
Bone Marrow Failure Syndromes ◽  
State Of Art

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.

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

Outcomes of Cord Blood Transplantation from an HLA-Identical Sibling for Patients with Inherited or Acquired Bone Marrow Failure Disorders: A Report from Eurocord, Cord Blood Committee (CBC-CTIWP) and Severe Aplastic Anemia Working Party (SAAWP) of the European Group of Blood and Bone Marrow Transplantation (EBMT)

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 153-153
Author(s):  
Simona Pagliuca ◽  
Régis Peffault de Latour ◽  
Franco Locatelli ◽  
Jean-Hugues Dalle ◽  
Kim Vettenranta ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Cord Blood ◽  
Median Time ◽  
Graft Failure ◽  
Bone Marrow Failure ◽  
Cord Blood Transplantation ◽  
Chronic Gvhd ◽  
Sibling Donor ◽  
Blood Transplantation ◽  
Grade Ii

Abstract Bone marrow failure (BMF) syndrome is a group of rare hereditary or idiopathic disorders occurring at all ages. For BMF patients under 40 years of age and having an HLA-identical related donor, allogeneic bone marrow transplantation (HSCT) is indicated. The use of cord blood transplantation (CBT) from an HLA-identical sibling donor is of interest in non-malignant disease due to the low risk of graft-versus-host disease (GVHD) associated with this type of stem cell source and the absence of risk to the donor. We analyzed outcomes of 122 children and young adults with inherited or acquired BMF syndrome, who received a CBT from an HLA-identical related donor. Patients were transplanted in EBMT centers between 1988 and 2014 and reported to Eurocord and EBMT. Ninety-six patients had an inherited and 26 patients had an acquired BMF. The specific diagnosis were: Fanconi anemia (n=48), Diamond Blackfan anemia (n=25), Amegakariocytic thrombocytopenia (n=6), Kostmann syndrome (n=4), Dyskeratosis congenital (n=3), Schwachman-Diamond syndrome (n=2), and unclassified inherited BMF (n=8) for the inherited group and severe aplastic anemia (n=25), pure red cell aplasia (n=1) for the acquired group. Eighty-nine patients received a single cord blood (CB) unit, whereas 33 patients received a combination of CB and bone marrow (BM) cells from the same sibling donor. The main reason for adding BM was due to the low cell dose of the single CB unit alone. Of patients receiving CB+BM, 6 had an acquired and 24 had an inherited BMF. Median age at CBT was 6.7 (range 1-16) years (5.6 years for patients with acquired and 6.9 years for those with inherited BMF). Median interval between diagnosis and CBT was 21 (range 2-114) months for the acquired and 55 (range 2-160) months for the inherited group, respectively. Fifty-nine patients (53%) received a reduced intensity conditioning (RIC) and 52 (47%) were treated with myeloablative regimens (MAC) (data available for 111 of 122 patients). Fludarabine-based regimens were used in 54 patients (44%). For patients receiving a RIC the most common regimen was cyclophosphamide and fludarabine (56%), and for MAC, cyclophosphamide and busulfan (46%). Total body irradiation was used in 8 patients (both in RIC and MAC settings). GVHD prophylaxis consisted mainly of cyclosporine alone (CSA) in 53 patients (43%), CSA + micophenolate mofetil in 11 patients (9%) and CSA + methotrexate in 23 patients (19%). The median number of total nucleated cells (TNC) infused was 6.2x107/Kg (1-24.2x107/Kg) for patients receiving a single CB unit and 26.8x107/Kg (5.3-41x107/Kg) for those receiving CB+BM (median TNC was 3x107/Kg in CB, and 22x107/Kg in BM graft). Median follow up is 5 (range 0.6-25) years. The cumulative incidence (CI) of neutrophil recovery at day 60 was 91%+9%, and the median time to engraftment was 21 (range 7-100) days. The CI of platelets engraftment at day 180 was 89+3% with a median time of 35 (range 7-138) days. Thirteen patients experienced primary graft failure (PFG) (12 patients after a single CB and one after a CB+BM graft). For those patients with available information, treatment of graft failure was second BM-HSCT (n=6), and growth factors (n=1). Of the 13 patients with graft failure, 11 died within in a median time of 1.9 (0.3 to 6) months after CBT. Two patients are alive after the second HSCT. The 100-day CI of grade II-IV acute GVHD was 11+3% (15 patients: grade II, n=8; grade III, n=6; grade VI, n=1), and the 1-year CI of chronic GVHD was 12+4%. The 1-year CI of transplant-related mortality was 13+3%, 17 patients died: 11 due to PGF (infections (n=6) hemorrhagic syndrome (n=1) and other causes (n=4)) and the remaining 6 of acute respiratory distress syndrome (n=1), and infections (n=5). Five-year overall survival (OS) for the whole population was 87+3%. OS was 80+8% for patients with acquired BMF, 87+3% for Fanconi anemia patients, 95+4% for those with Diamond Blackfan, and 86+7% for other inherited BMFs. In pediatric and young adult patients with either inherited or acquired BMF syndrome, CBT from an HLA-identical sibling donor is associated with excellent long-term outcomes with particular low rates of both acute and chronic GVHD, this latter observation being of particular importance for patients with non-malignant disorders. In case of inherited BMF, collecting cord blood unit at the birth of a new sibling is strongly recommended. Disclosures Dufour: Pfizer: Consultancy.

Single-institute comparative analysis of unrelated bone marrow transplantation and cord blood transplantation for adult patients with hematologic malignancies

Blood ◽  
2004 ◽  
Vol 104 (12) ◽  
pp. 3813-3820 ◽  
Author(s):  
Satoshi Takahashi ◽  
Tohru Iseki ◽  
Jun Ooi ◽  
Akira Tomonari ◽  
Kashiya Takasugi ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Cord Blood ◽  
Marrow Transplantation ◽  
Cord Blood Transplantation ◽  
Disease Free Survival ◽  
Hematologic Malignancies ◽  
Adult Patients ◽  
Transplant Recipients ◽  
Blood Transplantation

Unrelated cord blood transplantation (CBT) has now become more common, but as yet there have been only a few reports on its outcome compared with bone marrow transplantation (BMT), especially for adults. We studied the clinical outcomes of 113 adult patients with hematologic malignancies who received unrelated BM transplants (n = 45) or unrelated CB transplants (n = 68). We analyzed the hematopoietic recovery, rates of graft-versus-host disease (GVHD), risks of transplantation-related mortality (TRM) and relapse, and disease-free survival (DFS) using Cox proportional hazards models. The time from donor search to transplantation was significantly shorter among CB transplant recipients (median, 2 months) than BM transplant recipients (median, 11 months; P < .01). Multivariate analysis demonstrated slow neutrophil (P < .01) and platelet (P < .01) recoveries in CBT patients compared with BMT patients. Despite rapid tapering of immunosuppressants after transplantation and infrequent use of steroids to treat severe acute GVHD, there were no GVHD-related deaths among CB transplant recipients compared with 10 deaths of 24 among BM transplant recipients. Unrelated CBT showed better TRM and DFS results compared with BMT (P = .02 and P < .01, respectively), despite the higher human leukocyte antigen mismatching rate and lower number of infused cells. These data strongly suggest that CBT could be safely and effectively used for adult patients with hematologic malignancies.

Cord blood transplantation provides better reconstitution of hematopoietic reservoir compared with bone marrow transplantation

Blood ◽  
2003 ◽  
Vol 102 (3) ◽  
pp. 1138-1141 ◽  
Author(s):  
Francesco Frassoni ◽  
Marina Podestà ◽  
Rita Maccario ◽  
Giovanna Giorgiani ◽  
Gabriele Rossi ◽  
...  
Keyword(s):  
Stem Cells ◽  
Bone Marrow ◽  
Cord Blood ◽  
Mononuclear Cells ◽  
Cord Blood Transplantation ◽  
Hematopoietic Progenitor Cell ◽  
Transplant Recipients ◽  
Platelet Recovery ◽  
Blood Transplantation

Abstract Delayed hematopoietic recovery is the main factor precluding a wider use of cord blood (CB) transplants. We hypothesized that this delayed engraftment might not be related to an insufficient number of stem cells in the graft, but to an intrinsic difficulty of these cells to undergo differentiation. To test our hypothesis, 2 groups of children were compared; 12 received a CB transplant and 12 an adult bone marrow (BM) transplant. We studied neutrophil and platelet recovery and, at a median time of approximately 1 year after transplantation, the frequency of colony-forming cells (CFCs) and long-term culture initiating cells (LTC-ICs) in the BM of the 2 groups. Recipients of BM transplants received 1-log more cells and had significantly faster neutrophil and platelet recovery. Conversely, the frequency of committed and early progenitors was significantly higher in the BM of children given CB cells compared with BM transplant recipients (median count of CFC/2 × 104 BM mononuclear cells, 20 versus 11, P = .007; median count of LTC-IC/106 BM mononuclear cells, 8.2 versus 0.2 P = .001). CB, but not adult BM stem cells, can better restore the host hematopoietic progenitor cell reservoir; the delayed engraftment after CB transplantation may reflect the difficulty of CB progenitors to reprogram themselves toward differentiation.

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