scholarly journals Unrelated Donor Cord Blood Transplantation for Children with Severe Sickle Cell Disease: Results of One Cohort from the Phase II Study from the Blood and Marrow Transplant Clinical Trials Network (BMT CTN)

2012 ◽  
Vol 18 (8) ◽  
pp. 1265-1272 ◽  
Author(s):  
Naynesh R. Kamani ◽  
Mark C. Walters ◽  
Shelly Carter ◽  
Victor Aquino ◽  
Joel A. Brochstein ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Sickle Cell Disease ◽  
Cord Blood ◽  
Phase Ii Study ◽  
Cord Blood Transplantation ◽  
Marrow Transplant ◽  
Unrelated Donor ◽  
Cell Disease ◽  
Blood Transplantation ◽  
Blood And Marrow Transplant

scholarly journals Umbilical Cord Blood Transplantation for Children with Thalassemia and Sickle Cell Disease

2011 ◽  
Vol 17 (9) ◽  
pp. 1375-1382 ◽  
Author(s):  
Annalisa Ruggeri ◽  
Mary Eapen ◽  
Andromachi Scaravadou ◽  
Mitchell S. Cairo ◽  
Monica Bhatia ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Cord Blood ◽  
Umbilical Cord ◽  
Sickle Cell ◽  
Umbilical Cord Blood ◽  
Cord Blood Transplantation ◽  
Umbilical Cord Blood Transplantation ◽  
Cell Disease ◽  
Blood Transplantation

Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling

Blood ◽  
2013 ◽  
Vol 122 (6) ◽  
pp. 1072-1078 ◽  
Author(s):  
Franco Locatelli ◽  
Nabil Kabbara ◽  
Annalisa Ruggeri ◽  
Ardeshir Ghavamzadeh ◽  
Irene Roberts ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Bone Marrow Transplantation ◽  
Sickle Cell Disease ◽  
Cord Blood ◽  
Marrow Transplantation ◽  
Cord Blood Transplantation ◽  
Thalassemia Major ◽  
Cell Disease ◽  
Blood Transplantation ◽  
Key Points

Key Points Patients with thalassemia major or sickle cell disease had excellent outcomes after both CBT and BMT from an HLA-identical sibling. Related cord blood transplantation is a suitable transplant option for patients with hemoglobinopathies.

scholarly journals Use of the HLA-B leader to optimize cord-blood transplantation

Haematologica ◽  
2020 ◽  
pp. 0-0
Author(s):  
Effie W. Petersdorf ◽  
Ted Gooley ◽  
Fernanda Volt ◽  
Chantal Kenzey ◽  
Alejandro Madrigal ◽  
...  
Keyword(s):  
Cord Blood ◽  
Cord Blood Transplantation ◽  
Marrow Transplant ◽  
Unrelated Donor ◽  
Shared Allele ◽  
Blood Transplantation ◽  
Increased Risk ◽  
Cord Blood Unit ◽  
Life Threatening ◽  
Multivariable Regression Models

Cord-blood transplantation (CBT) can cure life-threatening blood disorders. The HLA-B leader affects the success of unrelated donor transplantation but its role in CBT is unknown. We tested the hypothesis that the HLA-B leader influences CBT outcomes in unrelated single-unit cord-blood transplants performed by Eurocord/European Blood and Marrow Transplant (EBMT) centers between 1990 and 2018 with data reported to Eurocord. Among 4822 transplants, 2178 had one HLA-B mismatch of which 1013 were HLA-A and HLA-DRB1-matched. The leader (M or T) was determined for each HLA-B allele in patients and units to define the genotype. Among single HLA-B-mismatched transplants, the patient/unit mismatched alleles were defined as leader-matched if they encoded the same leader, or leader-mismatched if they encoded different leaders; the leader encoded by the matched (shared) allele was determined. The risks of GVHD, relapse, non-relapse mortality and overall mortality were estimated for various leaderdefined groups using multivariable regression models. Among the 1013 HLA-A, -DRB1- matched transplants with one HLA-B mismatch, increasing numbers of cord-blood unit M-leader alleles was associated with increased risk of relapse (hazard ratio [HR] for each increase in one M-leader allele 1.30, 95% confidence interval [CI] 1.05 to 1.60, P 0.02). Furthermore, leader mismatching together with an M-leader of the shared HLA-B allele lowered non-relapse mortality (HR 0.44, 95% CI 0.23 to 0.81; P 0.009) relative to leader-matching and a shared T-leader allele. The HLA-B leader may inform relapse and non-relapse mortality risk after CBT. Future patients might benefit from the appropriate selection of units that consider the leader.

scholarly journals Use of alternative donors for allogeneic stem cell transplantation

Hematology ◽  
2015 ◽  
Vol 2015 (1) ◽  
pp. 220-224 ◽  
Author(s):  
Claudio Anasetti
Keyword(s):  
Clinical Trials ◽  
Stem Cell ◽  
Cord Blood ◽  
Graft Failure ◽  
Cord Blood Transplantation ◽  
Human Leukocyte ◽  
Unrelated Donor ◽  
Factors Affecting ◽  
Blood Transplantation ◽  
Cell Sources

Abstract For patients without a human leukocyte antigen (HLA)-matched sibling or unrelated donor, options include transplantation from HLA-mismatched related donors, HLA-mismatched unrelated donors, or unrelated cord blood units. Graft failure remains a problem in 10%-20% of cord blood transplants that contain a limited number of hematopoietic cells. Many approaches are tested in clinical trials to offset the risk of graft failure after cord blood transplantation. GVHD remains a hurdle with any HLA mismatched graft. The use of post-transplant cyclophosphamide holds the promise to overcome the HLA barrier and prevent GVHD despite donor mismatch for a full HLA haplotype. Priority should be given to enrolling patients onto transplant protocols addressing the fundamental problems of engraftment, GVHD, relapse or treatment-related mortality tested with one or more of the alternative stem cell sources. Principles for prioritization of alternative stem cell sources are discussed separately for children and adults who cannot be enrolled on clinical trials. It is difficult ranking currently available sources in the face of multiple factors affecting outcomes, rapidly changing transplant technology and without results from comparative trials.

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