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Improved Outcomes of UM171-Expanded Cord Blood Transplantation Compared with Other Graft Sources: Real-World Evidence
Cohen, S., Bambace, N., Ahmad, I., Roy, J., Tang, X., Zhang, M. J., Burns, L. J., Barabé, F., Bernard, L., Delisle, J. S., et al
Blood advances. 2023
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Abstract
Cord blood (CB) transplantation is hampered by low cell dose and high non-relapse mortality (NRM). A phase I-II trial of UM171-expanded CB transplants demonstrated safety and favourable preliminary efficacy. The aim of the current analysis was to retrospectively compare results of the phase I-II trial to those after unmanipulated CB and matched unrelated donor (MUD) transplants. Data from recipients of CB and MUD transplants were obtained from the Center for International Blood and Marrow Transplant Research (CIBMTR) database. Patients were directly matched for the number of prior allogeneic hematopoietic stem cell transplants (alloHCT), disease and refined Disease Risk Index. Patients were further matched by propensity score for age, comorbidity index and performance status. Primary endpoints included NRM, progression-free survival (PFS), overall survival (OS) and graft-versus-host disease (GVHD)-free relapse-free survival (GRFS) at 1- and 2-years post alloHCT. Overall, 137 CIBMTR (67 CB, 70 MUD) and 22 UM171-expanded CB patients were included. NRM at 1 and 2 years was lower, PFS and GRFS at 2 years and OS at 1 year were improved for UM171-expanded CBs compared to CB controls. Compared to MUD controls, UM171 patients had lower 1- and 2-year NRM, higher 2-year PFS, and higher 1- and 2-year GRFS. Furthermore, UM171-expanded CB recipients experienced less grades III-IV acute GVHD and chronic GVHD compared to MUD graft recipients. Compared to real-world evidence with CB and MUD alloHCT, this study suggests that UM171-expanded CB recipients may benefit from lower NRM and higher GRFS.
PICO Summary
Population
Adults with haematological malignancies enrolled in a clinical trial in USA and Canada, plus matched controls from CIBMTR database (n=159)
Intervention
UM171 cord blood transplant (n=22)
Comparison
Matched controls (n=137) who received cord blood (CB, n=67) or matched unrelated donor transplant (MUD, n=70)
Outcome
Non-relapse mortality (NRM) at 1 and 2 years was lower, progression free survival (PFS) and GvHD-free relapse free survival (GRFS) at 2 years and overall (OS) survival at 1 year were improved for UM171-expanded CBs compared to CB controls. Compared to MUD controls, UM171 patients had lower 1- and 2-year NRM, higher 2-year PFS, and higher 1- and 2-year GRFS. Furthermore, UM171-expanded CB recipients experienced less grades III-IV acute GVHD and chronic GVHD compared to MUD graft recipients.
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Clinical Impact of Cryopreservation of Allogeneic Hematopoietic Cell Grafts During the Onset of the COVID-19 Pandemic
Devine, S. M., Bo-Subait, S., Kuxhausen, M., Spellman, S. R., Bupp, C., Ahn, K. W., Stefanski, H. E., Auletta, J. J., Logan, B. R., Shaw, B. E.
Blood advances. 2023
Abstract
At the onset of the COVID-19 pandemic, the National Marrow Donor Program mandated the cryopreservation of hematopoietic cell grafts from volunteer unrelated donors due to numerous patient and donor safety concerns and logistical hurdles. Using the Center for International Blood and Marrow Transplant Research (CIBMTR) outcomes database, we report the impact of cryopreservation on overall survival (OS) and other outcomes within one year following hematopoietic cell transplantation (HCT). We analyzed 1,543 recipients of cryopreserved allografts receiving HCT at US centers during the first 6-months of the pandemic and compared them to 2,499 recipients of fresh allografts during the same 6-month period in 2019. On multivariable regression analysis, we observed no difference in OS (HR, 1.12; 95% CI: 0.98-1.28; P=0.09), non-relapse mortality (HR, 1.01; 95% CI: 0.84-1.22; P=0.89), graft-versus-host disease (GVHD), or GVHD-free, relapse-free survival (HR 1.03; 95% CI 0.93-1.14; P=0.58) in recipients of cryopreserved versus fresh allografts. Disease-free survival (DFS) was lower in the cryopreserved group (HR, 1.18; 95% CI: 1.05-1.33; P=0.006) due to a higher risk of relapse (HR, 1.21; 95% CI: 1.04-1.41; P=0.01). Primary graft failure was higher with cryopreservation (OR 1.48; 95% CI: 1.10-2.00; P=0.01) and the risk of chronic GVHD was lower (HR, 0.65; 95% CI: 0.50-0.84; P=0.001). In conclusion, while there was no negative impact of cryopreservation on OS, relapse was higher and DFS was lower. Fresh grafts are recommended as the pandemic related logistical hurdles resolve. Cryopreservation should be considered an option for patients when fresh grafts are not feasible.
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Factors Affecting Human Umbilical Cord Blood Quality Before Cryopreservation: The Importance of Birth Weight and Gestational Age
Nguyen, P. H., Nguyen, V. T., Chu, T. T., Truong, L. H., Do, T. T. H., Nguyen, T. D., Bui, A. V., Ngo, T. A., Than, U. T. T., Nguyen, L. T.
Biopreservation and biobanking. 2019
Abstract
Background: Umbilical cord blood (UCB) is a rich source of hematopoietic stem cells and is useful for the treatment of blood diseases. The cost of UCB storage is high; thus, it is necessary to evaluate the quality of UCB before collection and cryopreservation. Aim: This study aimed to determine the maternal and neonatal factors that influence UCB before selection for cryopreservation. Materials and Methods: The analysis included 403 processed UCB units. The effects of maternal characteristics including maternal age and delivery method and neonatal factors such as birth weight, gestation duration, and sex on UCB quality were determined based on the collected blood volume, total nucleated cell (TNC) count, and CD34(+) cell count. Results: The neonatal birth weight influenced the collected blood volume, TNC count, and CD34(+) cell count. Neonates with higher birth weights produced better quality UCB units because of increased collected blood volumes, TNC counts, and CD34(+) cell counts. However, an increase in the gestational age from 35 to 41 weeks led to decreases in the collected blood volume and CD34(+) cell count. Conclusion: These data may be useful for determining the optimal cord blood units for collection and cryopreservation and for advising pregnant women using private banking services.
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The Cord Blood Apgar: a novel scoring system to optimize selection of banked cord blood grafts for transplantation (CME)
Page, K. M., Zhang, L., Mendizabal, A., Wease, S., Carter, S., Shoulars, K., Gentry, T., Balber, A. E., Kurtzberg, J.
Transfusion. 2012;52(2):272-83
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Abstract
BACKGROUND Engraftment failure and delays, likely due to diminished cord blood unit (CBU) potency, remain major barriers to the overall success of unrelated umbilical cord blood transplantation (UCBT). To address this problem, we developed and retrospectively validated a novel scoring system, the Cord Blood Apgar (CBA), which is predictive of engraftment after UCBT. STUDY DESIGN AND METHODS In a single-center retrospective study, utilizing a database of 435 consecutive single cord myeloablative UCBTs performed between January 1, 2000, to December 31, 2008, precryopreservation and postthaw graft variables (total nucleated cell, CD34+, colony-forming units, mononuclear cell content, and volume) were initially correlated with neutrophil engraftment. Subsequently, based on the magnitude of hazard ratios (HRs) in univariate analysis, a weighted scoring system to predict CBU potency was developed using a randomly selected training data set and internally validated on the remaining data set. RESULTS The CBA assigns transplanted CBUs three scores: a precryopreservation score (PCS), a postthaw score (PTS), and a composite score (CS), which incorporates the PCS and PTS values. CBA-PCS scores, which could be used for initial unit selection, were predictive of neutrophil (CBA-PCS > 7.75 vs. <7.75, HR 3.5; p < 0.0001) engraftment. Likewise, CBA-PTS and CS scores were strongly predictive of Day 42 neutrophil engraftment (CBA-PTS > 9.5 vs. <9.5, HR 3.16, p < 0.0001; CBA-CS > 17.75 vs. <17.75, HR 4.01, p < 0.0001). CONCLUSION The CBA is strongly predictive of engraftment after UCBT and shows promise for optimizing screening of CBU donors for transplantation. In the future, a segment could be assayed for the PTS score providing data to apply the CS for final CBU selection. Copyright © 2012 American Association of Blood Banks.